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请指示在最近切实可行的日期，申报人各类普通股的股份总数。
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普通股类 |
| 截至2024年11月1日的优秀股份 |
普通股，面值$0.00001 |
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1
风险因素总结
投资于我们普通股的范围涉及广泛的风险，您应该在仔细考虑这些风险后才进行投资。在购买我们的普通股之前，建议您仔细阅读并考虑“项目控制项1A. 风险因素”一节中的一些风险讨论。
我们的业务受到多种风险的影响，包括可能阻止我们实现业务目标或对我们的业务、财务状况、营业收入、现金流和前景造成不利影响的风险，您应在决定是否投资我们的普通股之前考虑这些风险。这些风险在本报告的第26页开始的“风险因素”一节中有更详细的讨论，包括以下内容：
与我们的财务状况和资本需求相关的风险
| ● | 我们有有限的运营历史，仅开展了有限数量的临床试验，目前只有有限数量的患者参与我们正在进行的试验，并且迄今尚未完成任何临床试验。我们没有任何产品获得商业销售批准，也没有产生任何营业收入，这可能使投资者难以评估我们当前业务的状况、成功的机会和可行性。 |
| ● | 我们自成立以来一直亏损，并未创造任何营业收入。我们预计在可预见的未来将继续亏损，并可能永远无法实现或维持盈利。 |
| ● | 我们的营业收入和实现盈利能力很大程度上取决于我们实现与当前或未来产品候选者的发现或识别、临床前和临床研究、监管批准和商品化相关的多项目标。 |
| ● | 我们将需要大量额外资本来资助我们的业务和实现我们的目标。如果我们无法及时筹措到资金，或无法接受我们可接受的条款，我们可能被迫延迟、减少或取消我们的研究或产品开发计划、任何未来的商品化努力或其他业务。 |
| ● | 在COVID-19大流行期间实施的政策，今天仍然存在，可能对我们的业务产生不利影响，包括我们的临床前开发、临床试验和临床试验操作。 |
与我们产品候选者开发相关的风险
| ● | 我们极大程度上依赖我们的产品候选者NXP800和NXP900的成功。 |
| ● | 临床试验非常昂贵、耗时、难以设计和实施，并涉及不确定的安全性、耐受性和功效结果。此外，较早的临床前研究和临床试验的结果可能无法预测未来临床前研究或临床试验的结果。我们目前或未来的产品候选者在早期或后期临床试验中可能没有良好的结果，如果有的话，也可能无法获得监管机构的批准。 |
| ● | 如果我们未能证明我们的任何或所有产品候选者的安全性和/或功效，我们可能需要终止开发计划，这可能损害我们的声誉和业务。 |
| ● | 药品产品的开发和商业化受到广泛的规定，我们可能无法为NXP800、NXP900或任何未来的产品候选者获得监管机构的批准。 |
| ● | 如果我们无法取得或维持产品候选药品的监管批准，最终无法商业化其中一个或多个产品，或者在此过程中遇到重大延迟，我们的业务将受到重大损害。 |
3
关于我们对第三方的依赖的风险
| ● | 我们当前和未来产品候选品的制造过程复杂。出于各种原因，我们的第三方制造商可能会遇到困难或中断，这可能会延迟或完全阻止他们制造我们当前和未来产品候选品用于临床试验，如果获得批准，则用于商业销售。 |
与管理增长和员工事项相关的风险
| ● | 我们未来的成功取决于我们保留高管和关键员工的能力，吸引、留住和激励合格人才，并管理我们的人力资本。 |
| ● | 我们目前有13名全职员工，需要扩大组织规模和能力。我们可能会在管理这种增长方面遇到困难。 |
与知识产权相关的风险
| ● | 如果我们无法获得和维持产品和技术的专利保护或其他必要的权利，或者获得的专利保护范围不够广泛，或者在许可专利下的权利不够广泛，我们的竞争对手可能会开发和商业化与我们类似或相同的产品和技术，我们成功商业化产品和技术的能力可能会受到不利影响。 |
4
第I部分-财务信息
项目1。 基本报表
NUVECTIS PHARMA, INC.
简明资产负债表
(以千美元计算，每股和股数除外)
(未经审计)
| 2021年9月30日 |
| 运营租赁负债： | ||||
2024 | 2023 |
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资产 |
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流动资产 |
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现金及现金等价物 |
| $ | |
| $ | |
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其他资产 |
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总流动资产 |
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资产总计 |
| $ | |
| $ | |
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负债和股东权益 |
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流动负债 | |||||||
应付账款 |
| $ | |
| $ | |
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应计负债 |
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员工薪酬福利 |
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流动负债合计 |
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负债合计 |
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承诺和事务，参见注释3 |
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股东权益，请参阅附注4 |
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2,885.3 |
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股票认购应收款项。 |
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累积赤字 |
| ( |
| ( |
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总股东权益 |
| |
| |
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负债合计和股东权益 |
| $ | |
| $ | |
|
* |
附注是这些未经审计的基本财务报表的一部分。
5
NUVECTIS PHARMA，INC。
捷凯收购公司二期有限公司
(以千美元计算，每股和股数除外)
(未经审计)
| |||||||||||||
截至9月30日止三个月 | 截至九月三十日的九个月 | ||||||||||||
| 2024 |
| 2023 |
| 2024 |
| 2023 | ||||||
营业费用 |
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研发 |
| $ | |
| $ | |
| $ | |
| $ | |
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一般行政 | |
| | |
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营业亏损 |
| ( |
| ( |
| ( |
| ( |
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财务收益 | |
| | |
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净损失 |
| $ | ( |
| $ | ( |
| $ | ( |
| $ | ( |
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归属于普通股股东的净亏损 | $ | ( |
| $ | ( | $ | ( |
| $ | ( |
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每股普通股的基本和稀释净损失，详见附注6 | $ | ( |
| $ | ( | $ | ( |
| $ | ( |
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基本和稀释后的平均流通股本 | |
| | |
| |
| ||||||
附注是这些未经审计的基本财务报表的一部分。
6
努维克蒂斯制药公司，INC。
股东权益变动简明报表
（以千美元计，每股份额除外）
(未经审计)
|
|
|
| ||||||||||
普通股 | 额外的 | 总计 | |||||||||||
0.00001美元的面值 | 实缴 | 累积的 | 股东的 | ||||||||||
| 股份 |
| 金额 |
| 资本 |
| 赤字 |
| 股权 | ||||
2022年12月31日的余额 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 | — |
| * |
|
| |
| — | | ||||
限制性股票授予 | | * | — | — | — | ||||||||
优先投资期权的行使 | | * | | | |||||||||
认证股证权行权 | | * | | | |||||||||
本期净损失 |
| — |
| — |
| - |
| ( |
| ( | |||
2023年3月31日的余额 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 | — | * | | — |
| | |||||||
发行受限制股奖项 | | — | — | — | — | ||||||||
行使优先投资期权，减去$的发行成本 | | * | | — | | ||||||||
认证股证权行权 | | * | | — | | ||||||||
发行普通股，减去发行成本$ | | * | | — | | ||||||||
期权的行权 | | * | | — | | ||||||||
本期净损失 |
| — |
| — |
| - |
| ( |
| ( | |||
2023年6月30日的余额 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 | — |
| * | |
| — | | ||||||
发行受限制股奖励 | | * | — | — |
| — | |||||||
普通股的发行，减去发行成本为$ | | * | | — | | ||||||||
本期净损失 |
| — |
| — |
| - |
| ( |
| ( | |||
2023年9月30日的余额 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
* | 表示低于1美元美元的金额。 |
附注是这些未经审计的基本财务报表的一部分。
7
NUVECTIS PHARMA, INC.
股东权益变动简明报表
(美元指数以千为单位，股份数量除外)
(未经审计)
|
|
|
| ||||||||||
普通股 | 额外的 | 总计 | |||||||||||
面值$0.00001 | 实缴 | 累积的 | 股东的 | ||||||||||
| 股份 |
| 金额 |
| 资本 |
| 赤字 |
| 股权 | ||||
2023年12月31日的余额 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 |
| — |
| — |
| |
| — |
| | |||
限制性股票授予 | | — | — | — | — | ||||||||
发行普通股，减去发行成本$ | | * | | — | | ||||||||
本期净损失 |
| — |
| — |
| — |
| ( |
| ( | |||
2024年3月31日的余额 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 |
| — |
| * |
| |
| — | | ||||
发行受限制的股票奖励 | | * | — | — | — | ||||||||
发行普通股，减去发行成本$ | | * | | — | | ||||||||
本期净损失 |
| — |
| — |
| — |
| ( |
| ( | |||
2024年6月30日的余额 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
股份支付 |
| — |
| * |
| |
| — |
| | |||
发行受限制的股票奖励 | | * | — | — | — | ||||||||
普通股股份发行，减去$发行成本 | | * | | — | | ||||||||
本期净损失 |
| — |
| — |
| — |
| ( |
| ( | |||
2024年9月30日的余额 |
| |
| * |
| $ | |
| $ | ( |
| $ | |
* | 表示小于1美元的金额。 |
附注是这些未经审计的基本财务报表的一部分。
8
NUVECTIS PHARMA, INC.
精简现金流量表
(以千美元计算，每股和股数除外)
(未经审计)
截至9月30日的前九个月 | |||||||
| 2024 |
| 2023 | ||||
经营活动产生的现金流量 |
|
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净损失 |
| $ | ( |
| $ | ( |
|
调整以将亏损调和到经营活动中使用的净现金： |
| |
| |
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经营性资产和负债变动: |
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其他流动资产的（增加）/减少 |
| ( |
| |
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应付账款减少/增加 | ( | | |||||
应付负债减少 | ( | ( | |||||
应计的薪酬和福利减少 |
| ( |
| ( |
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经营活动使用的净现金流量 |
| $ | ( |
| $ | ( |
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投资活动产生的现金流量 |
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投资活动产生的净现金流量 |
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筹资活动产生的现金流量 |
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普通股发行收益 - 市场发行 |
| $ | |
| $ | |
|
与市场发行相关的发行成本 | ( | ( | |||||
与首次公开发行相关的发行成本 | — | ( | |||||
行使权证、期权和优先投资选项所得款项 | — | | |||||
与行使权证和优先投资选项相关的发行成本 | — | ( | |||||
定向增发相关的发行成本 | — | ( | |||||
筹资活动产生的现金净额 |
| $ | |
| $ | |
|
现金及现金等价物的(减少)/增加 |
| $ | ( |
| $ | |
|
期初现金及现金等价物 |
| $ | |
| $ | |
|
期末现金及现金等价物 |
| $ | |
| $ | |
|
附注是这些未经审计的基本财务报表的一部分。
9
NUVECTIS PHARMA, INC.
基本报表附注
注1 - 一般：
| a. | Nuvectis Pharma, Inc.（以下简称“公司”）于2020年7月27日根据德拉华州法律成立，于2021年5月开始主要业务。 公司的主要执行办公室位于新泽西州Fort Lee。 |
公司是一家专注于开发创新精准药物治疗肿瘤等严重未满足医疗需要状况的生物制药公司。
| b. | 2021年5月，公司与CRt Pioneer Fund（“CRT”）签订了全球独家许可协议（见附注3a）。 2021年8月，公司与苏格兰爱丁堡大学达成了全球独家许可协议，用于公司的第二药物候选品（见附注3a）。 |
| c. | 2022年2月，公司股票开始在纳斯达克交易，代码为“NVCT”. |
| d. | 流动性和资本资源 |
公司自成立以来一直出现净营业亏损，并截至2024年9月30日，累计赤字为$
在截至2024年9月30日的三个月内，公司总共出售了
截至2024年9月30日的九个月内，公司共出售了
管理层认为，截至2024年9月30日，公司现有的现金及现金等价物可支持至少12个月的计划运营，包括这些简明财务报表发行日期后的时间。
公司需要筹集额外资本以完成旨在开发产品候选药物的临床试验，直至获得监管和营销批准。不能保证公司将能够获得此类额外融资，或以对公司满意的条件获得，并且此融资是否足以满足其需求。如果公司未能成功获取足够资金，这可能迫使公司延迟、限制或减少其产品开发、临床试验、商业化努力或其他运营，甚至关闭或清算。
10
注 2 - 重要会计政策:
| a. | 报告范围 |
附带的简明财务报表未经审计。公司的未经审计的简明财务报表是根据美国通用会计准则（“U.S. GAAP”）编制的，以美元表示，并遵循证券交易委员会（“SEC”）有关中期财务报告的要求。因此，它们不包括美国通用会计准则为完整财务报表所要求的所有信息和披露，因为通常需要的某些脚注或其他财务信息可以简化或省略。未经审计的简明财务报表是按照已经审计的财务报表的基础进行准备的。未经审计的简明财务报表包括公司的账目。这些附注中对适用指南的任何参考，是指财务会计准则委员会（“FASB”）的会计准则编码（“ASC”）和会计准则更新（“ASU”）中找到的权威美国通用会计准则。
管理层认为，未经审计的简明财务报告包括认为对中期期间结果的真实陈述所必需的所有正常和经常性调整。截至2024年9月30日的期间结果并不一定代表预期的截至2024年12月31日或任何未来期间的结果。2023年12月31日的简明资产负债表包含于此处的，是从该日期的已经审计的财务报表导出的，但并不包括美国通用会计准则要求的所有披露。这些未经审计的简明财务报表应与公司的审计财务报表和相关附注一起阅读，其中包括公司在2024年3月5日向SEC提交的年度10-k表上的截至2023年12月31日的财年报告。
基础报表中采用并使用的重要会计政策与上一财政年度的一致。
| b. | 财务报表的编制符合美国一般公认的会计原则，要求管理层进行估计和假设，这些估计和假设影响资产和负债的报告金额和变动以及潜在的资产和负债的披露。实际结果可能与估计的结果不一致。 |
公司财务报表的编制要求管理层进行估计和假设，影响公司财务报表及附注中资产、负债和费用的报告金额。公司财务报表中最重要的估计涉及研发费用的计提、股权奖励的估值和递延税款资产的准备金。这些估计和假设基于当前事实、未来预期以及其他各种因素，被认为在情况下是合理的，其结果构成了就资产和负债的账面价值以及从其他来源无法明显得知的费用录入的判断依据。实际结果可能与这些估计有重大和不利的差异。
c. | 公允价值计量 |
公司遵循权威会计指引，其中定义了公允价值，建立了一致的公允价值衡量框架，并扩大了对每个按公允价值计量的主要资产和负债类别的披露，无论是按照重复性还是非重复性计量。公允价值被定义为在测量日期在资产或负债的主要或最有利的市场上以有序交易方式在市场参与者之间交易时将获得的交换价格（按退出价格计算）。可能用于衡量公允价值的三个输入级别包括：
第1级：在测量日期对于资产或负债可访问的相同资产或负债在活跃市场中报出（未调整）的价格。公允价值层次结构最优先考虑第1级输入。公司的第1级资产包括货币市场基金。
11
二级：除一级价格以外的可观察输入，如活跃市场中类似资产或负债的报价价格或其他可观察或可以通过可观察市场数据证实的输入，可以基本完整地覆盖资产或负债的全部期限。
三级：由市场活动几乎没有支持的不可观察输入。公平价值层次结构最低地对三级输入优先。
在确定公允价值时，公司利用估值技术最大程度地利用可观察输入，并尽可能减少不可观察输入的使用，并在评估公允价值时考虑交易对手信用风险。
现金及现金等价物中包括的货币市场账户被视为一级。
截至2024年和2023年9月30日止三个月和九个月内，未发生在公允价值衡量水平之间的转移。其他金融工具主要包括现金及现金等价物、其他流动资产、应付账款和应计负债。这些金融工具的公允价值接近其账面价值。
d. | 最近发布的未采纳会计准则 |
管理层认为，尽管尚未生效，但最近发布的会计准则，如果目前采纳，对公司财务状况或业绩不会产生重大影响。在 期内行权。
备注3 - 承诺和 contingencies:
| a. | 许可协议 |
CRt 先锋基金许可协议
CRt 先锋基金许可协议没有发生重大变化，如公司年度报告（2023年12月31日结束的财年）中披露，该报告于2024年3月5日提交给证监会（请查看我们年度报告中财务报表注 5a）。
由于与这些事件或里程碑的实现相关的不确定性，截至2024年9月30日和2023年12月31日，任何潜在的里程碑或版税支付金额尚未计提。
爱丁堡大学许可协议
与公司提交给证监会的2023年12月31日年度报告中披露的爱丁堡大学（UoE）许可协议相比，目前尚未发生重大变化（请查看我们年度报告中的财务报表注释5a）。
由于与这些事件、里程碑或额外研究承诺的实现相关的不确定性，截至2024年9月30日和2023年12月31日，任何未来可能的研究支持、里程碑或版税支付金额均未予计提。截至2024年9月30日，公司已向UoE支付了$
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| b. | 关联交易 |
除正常业务过程中已披露在公司2023财年12月31日提交给SEC的10-K表格中的相关方交易外，无其他相关方交易（请参阅我们在年度报告10-K中财务报表附注中的附注10）。
| c. | 备用金 |
截至2024年9月30日和2023年12月31日
注释 4 – 股东权益:
| a. | 公开实体的定向增发 |
于2022年7月29日，公司完成了一项定向增发（“2022年7月定向增发”），根据2022年7月27日签订的证券购买协议（“协议”）的条款和条件。有关2022年7月定向增发，公司发行了
| b. | 市价购买计划 |
2024年9月30日结束的九个月内，公司总共出售了
在截至2024年9月30日的三个月内，公司共出售了
截至2024年9月30日，ATm计划尚有约百万美元的证券可供出售。
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注5 - 基于股份的报酬：
| a. | 2021年全球股票激励计划（“激励计划”） |
以下表格总结了公司在截至2024年9月30日的九个月内激励计划中的股票期权活动：
|
|
| 已授予和预期于2021年1月2日授予股份 |
| ||||||
股数 | Risks and Uncertainties | 价格 | 聚合的 | |||||||
分shéarsunder | 行权价格每股 | 剩余期限 | 内在价值 | |||||||
时间租船 | 时间租船 | life | （以千为单位） | |||||||
2023年12月31日余额 | |
| $ | |
|
| $ | | ||
已批准 |
| — |
| - |
|
|
|
| ||
行使 |
| — |
| - |
|
|
|
| ||
被取消 |
| — |
| - |
|
|
|
| ||
优秀 - 2024年9月30日 |
| |
| $ | |
|
| $ | | |
2024年9月30日到期可行权 |
| |
|
|
| |||||
2024年9月30日预计归属 |
| |
| $ | |
|
| $ | | |
截至2024年9月30日，未确认的股权报酬支出为$
限制性股票授予
已向员工授予限制性股票奖励(RSAs)。RSA奖励的价值取决于授予日公司的股价。公司根据激励计划授予了RSAs。
以下表格总结了截至2024年9月30日为止的九个月内公司的RSA活动情况，如上述激励计划所述：
|
| 已授予和预期于2021年1月2日授予股份 |
| Risks and Uncertainties |
| 聚合的 | ||||
股数 | 平均津贴 | 合同条款 | 内在价值 | |||||||
股份 | 日期 公允价值 | （以年为单位） | （以千为单位） | |||||||
2023年12月31日余额 | |
| $ | |
|
| $ | | ||
已批准 |
| |
| |
|
|
|
| ||
被取消 | ( | | ||||||||
34,105 |
| ( |
| |
|
|
|
| ||
未来表现优越 – 2024年9月30日 |
| |
| $ | |
|
| $ | | |
预计归属于 – 2024年9月30日 |
| |
| $ | |
|
| $ | | |
截至2023年7月31日，续借贷款协议下未偿还的借款额为
截至2024年9月30日，未确认的RSA相关补偿成本总额为$
2024年1月4日，公司发行
2023年1月12日，公司发行
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在第三个确定日期上，潜在标的的收盘价虽增长了25％，但您仅收到提前赎回支付，无法从此类增值中受益。 在授予日期的每个周年纪念日。于2024年1月4日，将2023年1月授予的首个三分之一的权益延长至2024年7月15日。于2024年7月12日，将2023年1月授予的首个三分之一的权益延长至2025年1月3日。
于2022年4月1日，公司发行
2021年7月27日，Bentsur先生，Poradosu博士和Shemesh先生被授予
股份报酬支出
截至2024年9月30日的三个月，公司确认了支出，金额为$
截至2024年9月30日的九个月，公司确认了$
注6 - 每股亏损：
| a. | Basic |
基本每股净亏损是通过将归属于公司股东的净亏损除以平均未行使普通股份的加权平均数来计算的。
| |||||||||||||
三个月 | 三个月 | 在这九个月中 | 在这九个月中 | ||||||||||
截至2024年9月30日 |
| 截至2023年9月30日 | 截至2024年9月30日 |
| 截至2023年9月30日 | ||||||||
以千美元为单位，除每股和股份数量外 | |||||||||||||
归属于普通股股东的净亏损 | $ | ( |
| $ | ( | $ | ( |
| $ | ( |
| ||
每股普通股基本和稀释净亏损 | ( |
| ( | ( |
| ( |
| ||||||
普通股加权平均持股量 | |
| | |
| |
| ||||||
15
基本每股亏损是通过将归属于公司股东的结果除以期间内发行的普通股权重平均数量来计算的。
| 截至九个月结束时 | ||||||
2024年9月30日 |
| 2024年9月30日 | |||||
普通股加权平均 | |
| |
| |||
未获授予的限制性股票的加权 | ( |
| ( |
| |||
普通股加权平均股数 | |
| |
| |||
| b. | Diluted |
由于其对所示期间的稀释净损失每股影响将属于反稀释性，以下可能具有稀释性的证券未包括在稀释每股普通股净损失的计算中：
2021年9月30日 | ||||
| 2024 |
| 2023 | |
与以下相关的普通股份可分享： |
|
|
|
|
权证 |
| |
| |
Options |
| |
| |
未获颁 RSAs* |
| |
| |
| * | 包括 |
附注7 - 关联交易：
| a. | 除正常业务外，无关联方交易。 |
附注8 - 后续事项：
| a. | 请参阅注5，了解有关延长Bentsur先生，Poradosu博士和Shemesh先生获得期权的期限的信息。 |
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项目2。经营管理层讨论及经营成果分析
您应阅读本报告其他地方出现的财务报表及相关附注与本报告一起出现的下面的讨论和分析。以下讨论和分析含有在1933年证券法第27A条和1934年证券交易法第21E条的意义内的前瞻性声明，包括但不限于，关于我们的期望、信念、意图或未来策略的声明，这些声明由“期望”、“预期”、“打算”、“相信”、“可能”、“计划”、“寻求”或类似语言表示。本文件中包含的所有前瞻性声明均基于我们在本文件日期可得的信息，我们不承担更新任何此类前瞻性声明的义务。对于此类前瞻性声明，我们宣称受1995年私人证券诉讼改革法中包含的前瞻性声明安全港保护的保护。我们的业务和财务表现面临重大风险和不确定性。实际结果可能与前瞻性声明中的预测结果大不相同。在评估我们的业务时，您应仔细考虑本报告“风险因素”标题下以及我们于2023年12月31日结束的年度10-k表中提供的信息。在下文中，“我们”、“我们”和“我们的”一词可能指的是Nuvectis Pharma公司。
概述
我们是一家专注于开发用于治疗肿瘤领域未满足的严重医疗需求的创新精准药物的生物制药公司。
NXP800（GCN2激酶激活剂）
我们已经获得了NXP800的全球独家开发和商业权利许可。 在伦敦英格兰的癌症研究所发现的一种口服小分子 在伦敦英格兰的癌症研究所（ICR）发现。
在临床前研究中，NXP800治疗抑制了人类卵巢、子宫内膜和胃癌异种移植模型中的肿瘤生长，在其中，At-rich interactive domain-containing protein 1A（ARID1a）基因的遗传突变存在，可能将ARID1a作为治疗敏感性的生物标志物，并因此提供一种患者丰度的潜在策略。基于这项工作，我们已经开始在铂金抵抗性ARID1a突变的卵巢癌中进行NXP800的临床研究，这种癌症主要由两种组织学类型构成，即卵巢透明细胞癌（OCCC）和卵巢子宫内膜癌（OEC）。我们正在探索利用ARID1a缺陷作为其他肿瘤类型患者选择标志物的实用性。对ARID1a突变的遗传筛查可以使用商用的基于下一代测序的体外诊断测试进行检测，这些测试通常在临床上为癌症患者所使用。
2021年12月，位于英国的一期研究已经开始，包括两部分：剂量递增（1a期），随后是扩展期（1b期）。在1a期中，对患有晚期实体瘤的患者评估了NXP800的安全性、耐受性和药代动力学特性，以确定1亿阶段的剂量和给药方案。该研究的10亿阶段始于2023年第二季度，旨在评估NXP800对铂金抵抗性ARID1a突变的卵巢癌患者的安全性和初步抗肿瘤活性。
2022年6月，NXP800的新药申请（IND）获得了美国食品和药物管理局（FDA）的批准，包括1期临床试验方案。2022年12月，我们宣布FDA授予NXP800用于治疗铂金抵抗性ARID1a突变的卵巢癌的开发计划快速通道认定地位。
2023年8月，我们宣布FDA授予NXP800孤儿药物认定，用于胆管癌患者的治疗。
2023年12月，我们宣布与梅奥诊所合作，在胆管癌患者中进行一项由调查员赞助的临床试验。
2024年8月，我们宣布FDA授予NXP800孤儿药物认定，用于治疗ARID1a缺乏的卵巢、输卵管和原发性腹膜癌患者。
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NXP900（SRC/YES1 激酶抑制剂）
我们已授权 NXP900 的全球独家开发和商业版权，这是一种 SRC 家族激酶（“SFK”）抑制剂，可有效抑制 c-src（“SRC”）和 YES1 激酶。NXP900 是在苏格兰爱丁堡大学发现的。
SRC 在许多癌症类型中会被异常激活，包括乳腺癌、结肠癌、前列腺瘤、胰腺瘤和卵巢瘤等实体瘤，而在非癌细胞中仍然主要处于非活性状态。SRC 活性的增加通常与晚期癌症、转移潜能和治疗耐药性有关，并与不良的临床预后相关。据报道，YES1基因扩增与多种肿瘤有关，包括肺癌、头颈癌、膀胱癌和食道癌。此外，YES1 直接磷酸化和激活 Yes相关蛋白（“YAP1”），Hippo通路的主要影响因子，已被确定为包括鳞状细胞、间皮瘤和乳头状肾癌在内的多种癌症类型的耐药性、癌症进展和转移的启动子。
在体内，NXP900 治疗可抑制乳腺癌、宫颈癌、食道癌、头颈癌和髓母细胞瘤异种移植模型中的原发性和转移性肿瘤的生长，并显示出靶向药效学效应。此外，已经发现YES1基因扩增是表皮生长因子受体（“EGFR”）、人类表皮生长因子受体2（“HER2”）和间变性淋巴瘤激酶（“ALK”）耐药的关键机制。2022年4月发表在《自然通讯》（非公司赞助）上的一项同行评审研究表明，与奥西替尼联合使用时，NXP900 能够使耐药性非小细胞肺癌（“NSCLC”）细胞对奥西替尼（Tagrisso® 中的活性成分）重新敏感，奥西替尼是治疗表皮生长因子突变阳性非小细胞肺癌的主要抑制剂。我们在细胞系模型中复制和扩展了这些发现，表明与奥西替尼联合使用以及单独与alK抑制剂阿乐替尼（Alecensa® 中的活性成分）联合使用具有统计学意义的协同作用。
2023 年 5 月，美国食品药品管理局批准了我们的 NXP900 临床试验申请，其中包括 1 期临床试验方案。
第 1 阶段研究于 2023 年 9 月启动，由两部分组成：剂量递增（第 1a 阶段），然后是扩展阶段（第 1b 阶段）。在正在进行的 1a 期中，正在评估 NXP900 在晚期实体瘤患者中的安全性、耐受性和药代动力学特性，以确定第 10期的剂量和给药时间表。
运营结果
从2020年7月27日成立到2024年9月30日，我们没有产生任何收入。 从那时起ur 从头开始 2024 年 9 月 30 日 我们的主要活动是开发我们的两种候选药物，即 NXP800 和 NXP900，包括完成许可协议、支持IND的研究、对每种候选药物进行正在进行的1期临床试验以及包括筹集资金在内的其他组织活动。对于 NXP800，我们进行了支持临床试验的研究，随后我们的临床试验申请和英国药品和医疗保健监管局的受理，IND 申请并获得 FDA 的认可，以及西班牙药品和医疗器械管理局的临床试验申请和受理。NXP800 的 1a 期和 10期临床试验分别于 2021 年 12 月和 2023 年 4 月开始。对于 NXP900，我们进行了支持临床试验的研究，随后我们申请了 IND 并获得了 FDA 的认可，并为 2023 年 9 月开始的 1a 期临床试验做了准备。
研究和开发费用
研发费用包括直接归因于开展研发计划的成本，包括许可费、工资成本、基于股份的薪酬支出、工资税和其他员工福利、分包商以及用于研发活动的材料和服务，包括临床试验、制造成本和专业服务。与研究与开发有关的所有费用均按发生时列为支出。
处于临床开发后期阶段的候选产品的开发成本通常高于临床开发早期阶段的候选产品，这主要是由于后期临床试验的规模和持续时间的延长。我们预计，在短期和将来，由于我们正在进行和计划中的临床前和临床开发活动，我们的研发费用将大幅增加。我们的候选产品的成功开发是高度不确定的。目前，我们无法准确
18
估计或了解完成我们任何产品候选药的临床前和临床开发所需工作的性质、时间和成本，我们可能永远无法获得任何产品候选药的监管批准。
一般行政费用
一般和行政费用主要包括我们行政、财务和会计人员的工资和与人员相关的成本，包括基于股票的补偿，在专利和公司事务方面的法律费用；支付给会计、审计、咨询和税务服务的专业费用；保险费用；投资者关系活动；旅行费用；以及未包括在研发费用中的设施费用。
我们预计随着增加人数以支持我们持续的研发活动，我们的一般和行政费用将在未来增加。
以下表格总结了我们截至2024年9月30日和2023年9月30日三个月的营业费用（以千为单位）。
截至9月30日的三个月 | |||||||||
| 2024 |
| 2023 |
| 变化 | ||||
营业费用: |
|
|
|
|
|
| |||
研发 |
| $ | 2,819 |
| $ | 4,486 |
| $ | (1,667) |
一般行政 |
| 1,540 |
| 1,672 |
| (132) | |||
营业亏损 |
| (4,359) |
| (6,158) |
| 1,799 | |||
财务收益 |
| 206 |
| 277 |
| (71) | |||
净损失 |
| $ | (4,153) |
| $ | (5,881) |
| $ | 1,728 |
研发费用
以下表格总结了我们截至2024年9月30日和2023年三个月的研发费用（以千元计）
| 截至9月30日的三个月 |
| 增长 | ||||||
| 2024 |
| 2023 |
| （减少） | ||||
临床费用 | $ | 1,058 |
| $ | 1,209 | $ | (151) | ||
员工薪酬福利 | 1,414 |
| 1,219 | 195 | |||||
制造业-半导体 | 333 |
| 1,494 | (1,161) | |||||
许可费 | — |
| 500 | (500) | |||||
专业服务和其他 | 14 |
| 65 | (51) | |||||
所有研发费用 | $ | 2,819 |
| $ | 4,486 | $ | (1,667) | ||
在2024年9月30日结束的三个月内，研发支出下降了170万美元，降低了37%，与2023年同期相比。2024年9月30日结束的三个月内，研发支出的减少主要是由于制造相关成本减少了120万美元，这是由于制造活动减少，NXP900的一次性许可费减少了50万美元，临床试验费用减少了20万美元，部分抵消了2023年的临床试验启动费用，员工薪酬增加了20万美元，其中包括与雇员股票补偿相关的10万美元的增加。
以下表格总结了我们在2024年9月30日和2023年结束的三个月的一般和行政支出（以千为单位）。
截至9月30日的三个月 |
| 增长 | |||||||
| 2024 | 2023 |
| （减少） | |||||
其他 |
| $ | 764 |
| $ | 831 | $ | (67) | |
员工薪酬福利 |
| 391 |
| 410 | (19) | ||||
19
保险和其他 |
| 385 |
| 431 | (46) | ||||
总管理费用 |
| $ | 1,540 |
| $ | 1,672 | $ | (132) |
截至2024年9月30日的三个月内，一般和行政费用减少了$10万，或8%，与2023年同期相比。2024年9月30日结束的三个月内，一般和行政费用的减少主要是由于与上市公司相关费用的专业和咨询服务减少$10万引起的。
因上述原因，截至2024年9月30日的三个月内，我们的营业亏损减少了$170万，或29%，相较于2023年同期，其中由于利率期货增加，财务收入增加$20万。
以下表总结了截至2024年9月30日的九个月的营业费用结果2024年和2023年：
（以千为单位）
截至9月30日的九个月内， | |||||||||
| 2024 |
| 2023 |
| 变化 | ||||
营业费用: |
|
|
|
|
|
| |||
研发 |
| $ | 8,422 |
| $ | 11,115 |
| $ | (2,693) |
一般行政 |
| 4,976 |
| 4,916 |
| 60 | |||
营业亏损 |
| (13,398) |
| (16,031) |
| 2,633 | |||
财务收益 |
| 646 |
| 393 |
| 253 | |||
净损失 |
| $ | (12,752) |
| $ | (15,638) |
| $ | 2,886 |
研发费用
以下表格总结了截至2024年9月30日和2023年的研发费用情况：
（以千为单位）
| 截至9月30日的九个月， |
| 增长 | ||||||
| 2024 |
| 2023 |
| （减少） | ||||
员工薪酬福利 | $ | 4,358 |
| $ | 3,805 | $ | 553 | ||
临床费用 | 2,651 | 3,085 | (434) | ||||||
制造业-半导体 | 1,382 |
| 3,094 | (1,712) | |||||
许可费 | — |
| 1,001 | (1,001) | |||||
专业服务和其他 | 31 |
| 131 | (100) | |||||
所有研发费用 | $ | 8,422 |
| $ | 11,115 | $ | (2,693) | ||
2024年9月30日结束的九个月里，研发费用下降了270万美元，与2023年同期相比。2024年9月30日结束的九个月里，研发费用的减少主要是由于与制造业相关成本减少了170万美元，NXP900一次性许可费减少了100万美元，临床试验费用减少了40万美元，部分抵消了员工薪酬增加了50万美元，其中包括员工股票补偿增加了40万美元。
一般行政费用
以下表格总结了我们在2024年9月30日和2023年结束的九个月里的一般和管理费用（以千美元计）。
截至9月30日止的九个月 |
| 增长 | |||||||
| 2024 | 2023 |
| （减少） | |||||
其他 |
| $ | 2,609 |
| $ | 2,272 | $ | 337 | |
员工薪酬福利 |
| 1,277 |
| 1,405 | (128) | ||||
保险和其他 |
| 1,090 |
| 1,239 | (149) | ||||
20
总管理费用 |
| $ | 4,976 |
| $ | 4,916 | $ | 60 |
截至2024年9月30日的九个月内，总部和行政费用比2023年同期增加了10万美元。2024年9月30日的九个月内，总部和行政费用的增加主要是由于与公司相关费用中的专业和咨询服务增加了30万美元，部分抵消了与员工薪酬和福利减少10万美元以及其他费用减少10万美元有关。
由于上述原因，2024年9月30日结束的九个月内，与2023年同期相比，我们的营业亏损减少了290万美元，这主要是由员工薪酬和福利、临床试验费用、制造费用以及财务收入增加30万美元推动的。
流动性和资本资源
截至2024年9月30日，我们的现金及现金等价物为1720万美元。截至2024年9月30日和2023年的三个月，我们分别报告了净损失为420万美元和590万美元。截至2024年和2023年的九个月，我们的净损失分别为1280万美元和1560万美元。
2022年2月4日，我们宣布以每股5.00美元的价格定价了我们的首次公开发行普通股(IPO)，共发行320万股普通股，扣除一定的承销折让和佣金。根据UoE许可协议，我们需要支付UoE未来基金筹集的总额的2.5％，直到累计总额达到300万美元。根据IPO，我们向UoE支付了与这次筹款有关的40万美元。
IPO于2022年2月8日结束，毛收益为1600万美元，扣除承销折让和费用后净收益为1260万美元。
此外，2022年7月29日，我们完成了2022年7月的定向增发，获得了毛收益1590万美元，在扣除费用和开支后净收益为1420万美元，不包括根据我们许可协议要求支付的款项。作为该交易的一部分，我们发行了优先投资期权，该期权于2023年1月23日行使，至2026年1月29日到期，行使价格为每股9.65美元，根据证券购买协议中定义的某些调整。截至2023年12月31日，100万1091个优先投资期权以净收益890万美元行使。截至2024年9月30日止三个月和九个月，未行使任何优先投资期权，净收益为0美元。此外，作为2022年7月的定向增发的一部分，我们向代销商发行了认股权证，最多可购买115,481股普通股。代销商认股权证与优先投资期权基本相同，唯一的区别是行使价格为每股10.31美元。截至2024年9月30日，79,104份代销商认股权证已行使，净收益80万美元。
2023年3月17日，我们以S-3表格提交了一个无注册许可书登记声明（“登记声明”）。根据登记声明，我们可以发行总共高达15000万美元的证券。与登记声明的提交相对应，我们还与H.C.Wainwright & Co.（“销售代理”）签订了销售协议，根据该协议，我们可以通过一个总额高达4000万美元的现场发行计划(ATM)发行和销售我们的普通股股票，这包括在登记声明下可提供的15000万美元的证券中。根据ATM，我们将向销售代理支付高达总收益的3.0％的佣金率作为我们的普通股任何销售的佣金。我们无需在ATM下出售任何股票。截至2024年9月30日，我们已售出了1,337,654股普通股，并在ATM下收到了净收益1290万美元。
我们相信IPO、定向增发和ATm所筹集的资金将至少支持我们在财务报表公布后的未来12个月内支付营业费用和资本支出。我们这一估计是基于可能被证明错误的假设，我们可能会比预期更早耗尽可用资本资源。我们长期的未来生存取决于我们筹集额外资金以资助我们的运营能力。
我们预计随着我们不断进行的活动，特别是推进当前或未来产品候选药物的临床试验，包括根据我们与NXP800和NXP900许可协议所涉及的里程碑和赞助研究承诺支付，我们的支出将大幅增加。此外，我们预计作为公开公司运作会导致成本费用增加。
21
随着我们的发展，公司的法律、会计、投资者关系和其他费用都在增加。我们的运营支出的时间和金额将在很大程度上取决于我们能力：
| ● | 推进我们的临床和临床前项目的发展； |
| ● | 制造或采购我们的临床前和临床药物材料，并开发后期和商业制造的流程； |
| ● | 为成功完成临床试验的任何当前或未来产品候选品寻求监管批准； |
| ● | 根据我们的许可协议实现里程碑 |
| ● | 建立销售、市场营销、医务和分销基础设施，用于商业化我们可能获得营销批准的任何现有或未来候选产品 |
| ● | 招聘更多的临床、质量控制和科学人员； |
| ● | 扩大我们的运营、财务和管理系统，增加人员，包括支持我们临床开发、制造和商业化努力以及作为上市公司的运营的人员 |
| ● | 获取、保护、扩大和保护我们的知识产权组合；和 |
| ● | 收购额外的产品候选者。 |
我们预计在寻求监管批准我们的产品候选者以及如果选择追求入许可协议或收购其他产品候选者时，将需要额外的资本。如果我们的当前或未来产品候选者获得监管批准，我们预计将承担与产品制造、销售、营销和分销有关的重大商业化费用，具体取决于我们选择进行商业化的地点。
由于与我们的产品候选者的研究、开发和商业化相关的众多风险和不确定性，我们无法估计我们的运营资本需求的确切金额。我们未来的资金需求将取决于许多因素，并且可能会因此显著增加，包括：
| ● | 研究和开发我们当前或未来产品候选者的范围、进展和成本，包括我们的临床前和临床试验的时间安排以及安全性、耐受性和有效性结果。 |
| ● | 我们当前或未来产品候选者的监管审查成本、时间和结果； |
| ● | 我们当前或未来产品候选者制造的成本、时间以及为支持我们的临床试验和临床预研工作的能力； |
| ● | 未来活动的成本，包括我们任何获得营销批准的当前或未来产品候选者的销售、医疗事务、营销、制造和分销成本； |
| ● | 生产商业级产品的成本以及支持商业上市所需的库存成本； |
| ● | 有能力获得额外的非稀释性资金，包括来自组织和基金会的资助； |
| ● | 我们的产品如果获得营销批准，从商业销售中获得的营业收入（如果有）； |
22
| ● | 准备、申请和代表我们进行专利申请，获取、维护、扩展和执法我们的知识产权以及捍卫知识产权相关诉讼的成本； |
| ● | 我们建立和维护合作伙伴关系并达成有利条款的能力，如果可能的话；而且 |
| ● | 我们获得或通过许可获取其他候选产品和技术的程度。 |
在我们能够产生大量产品营业收入之前，我们预计将通过公开或私人股权发行、债务融资、政府资助、合作、战略合作伙伴关系和联盟，或与第三方进行市场营销、分销或许可安排等多种途径来筹措运营资金。如果我们通过出售股权或可转换债务证券筹集额外资本，您的所有权可能会受到重大稀释，而这些证券的条款可能包括清算或其他优先权，这可能不利于您作为普通股股东的权益。债务融资和优先股权融资（如果有）可能涉及限制我们能够采取特定行动的限制性契约，比如增加额外债务、进行资本支出或宣布分红等。此外，债务融资会导致固定的支付义务。
如果我们通过政府资助、合作、战略伙伴关系、联盟、市场营销、分销或许可安排与第三方合作方筹集额外资金，我们可能不得不放弃对我们的技术、未来营业收入、研究项目或产品候选者的宝贵权利，或者以可能对我们不利的条款授予许可。如果我们无法及时通过股权或债务融资等其他安排筹集额外资金，可能需要延迟、限制、减少或终止我们的研究、产品开发或未来商业化努力，或授予开发和推广产品候选者的权利，而这些产品我们本来更愿意自己开发和推广。
现金流量
下表提供了所述期间我们的现金流量信息：（以千为单位）
截至9月30日止的九个月 | ||||||
| 2024 |
| 2023 | |||
经营活动使用的净现金流量 |
| $ | (9,782) |
| $ | (12,104) |
投资活动产生的净现金流出 |
| — |
| — | ||
筹资活动产生的现金净额 |
| $ | 7,825 |
| $ | 14,170 |
经营活动
在2024年9月30日结束的九个月内，运营活动中使用了980万美元现金。这主要归因于我们1280万美元的净亏损，部分抵消了370万美元的非现金费用。我们经营资产和负债的变动主要是由于向供应商支付60万美元，以及支付董事和高级管理人员保险费用10万美元。
在2023年9月30日结束的九个月内，运营活动中使用了1210万美元现金。这主要归因于我们1560万美元的净亏损，部分抵消了350万美元的非现金费用。我们经营资产和负债的变动主要是由于支付员工薪酬和福利50万美元。
筹资活动
在2024年9月30日结束的九个月中，融资活动提供的净现金为780万美元，主要包括通过ATm出售普通股所得的净收入。
在2023年9月30日结束的九个月中，融资活动提供的净现金为1420万美元，主要包括我们定向增发相关认股权证所得的1030万美元净收入以及通过ATm出售普通股所得的420万美元净收入，减少了支付的40万美元的递延发行成本。
23
与业务组合有关，在扣除发行成本后，我们获得了约38390万美元的现金收益。截至2021年9月30日，我们的现金及现金等价物为28230万美元。业务组合之后，管理层认为其现有的财务资源足以满足其至少在发布这些财务报表之日起12个月内运营和资本需求。公司可能需要额外的资本来追求某些商业机会或响应技术进步，竞争动态或技术，客户需求，挑战，收购或不可预见的情况。此外，公司已经发生了，并预计将继续发生与成为一家上市公司有关的重大成本。因此，公司可能在未来进行股权或债务融资或进入信贷机构以实现上述或其他原因；但是，公司可能无法及时以有利可图的条件安全地获得额外的债务或股权融资。如果公司通过股权融资获得了额外资金，则其现有股东可能会面临重大稀释。此外，公司在未来获得的任何债务融资都可能涉及与公司的资本筹集活动和其他财务和运营事项有关的限制性契约，这可能会使公司更难以获得额外的资本并追求商业机会。如果公司无法在需要时以令人满意的条件获得充足的融资，公司继续增长或支持业务及应对业务挑战的能力可能会受到严重限制。
我们与临床研究机构、合同制造组织和其他第三方签订合同，用于临床试验、临床前研究和测试以及制造服务，在正常业务过程中。这些合同可以在提前书面通知的情况下由我们取消。取消时应付的款项仅包括截至取消日期提供的服务或发生的费用，包括我们的服务提供商不可取消的义务。此类付款的金额和时间尚不清楚。
我们还与第三方签订了许可和合作协议，这在正常业务过程中。我们未包括这些协议下的未来付款，因为这些协议下的义务取决于未来事件，例如我们达到指定的发展、监管和商业里程碑，或者净产品销售的版税。
根据NXP800许可协议，我们需要向ICR支付某些与发展和监管里程碑相关的费用，包括最多2200万美元的预批准里程碑费用，最多17800万美元（另加2200万美元）的监管和商业销售里程碑费用，以及根据销售净额分层基础上的中位数到10％的版税，除非终止开发。此外，我们最初同意向ICR提供高达额外50万美元的研究和开发。2022年3月31日，我们同意向ICR提供额外40万美元的研究和开发支持（总计90万美元）。
根据NXP900许可协议，我们需要向UoE支付某些与发展和监管里程碑相关的费用，包括最多4500万美元的预批准里程碑费用，最多27960万美元（另外4500万美元）的监管和商业销售里程碑费用，净销售额的中位数到8％的版税以及我们将来基金募集总额的2.5％，最高累计达300万美元，除非停止开发。此外，我们将向UoE提供额外高达754,000美元的研究和开发支持。
我们目前没有任何长期租赁协议。我们在2024年5月1日签订的一年协议基础上租用新泽西州Fort Lee的办公空间。
不设为资产负债表账目之离线安排
在所呈现期间，我们并没有，也目前没有任何符合证券交易委员会规定的表外安排。
关键会计政策和重大判断和估计
我们在本季度报告的形式10-Q中的简明财务报表及相关附注，是根据美国通用会计原则编制的。简明财务报表的编制还要求我们进行对资产、负债、成本、费用及相关披露的数额产生影响的估计和假设。我们的估计基于历史经验和我们认为在该情况下合理的各种其他假设。实际结果可能与管理层所做的估计有重大不同。在我们的估计与实际结果之间存在差异的范围内，我们未来的财务报表呈现、财务状况、经营成果和现金流量将受到影响。
与我们在于2024年3月5日提交给美国证券交易委员会的年度报告中所述的“注2 - 重要会计政策摘要”中描述的相比，我们的关键会计政策和估计没有重大变化。
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最近发布的会计声明
请参阅本季度报告在表格10-Q中包含的我们精简财务报表附注2，讨论最近的会计准则。
新兴成长公司和小型报告公司的地位
2012年《创业公司启动法案》允许像我们这样的“新兴成长公司”利用延长的过渡期来遵守适用于公开公司的新或修订的会计准则，直到这些准则否则将适用于私人公司。我们选择不“选择退出”此规定，因此，我们将在私人公司采纳新或修订的会计准则时采纳新或修订的会计准则，直到我们（i）不可撤销地选择“选择退出”这种延长过渡期，或者（ii）不再符合“新兴成长公司”资格。
我们还是一家“较小的报告公司”，意味着我们非关联方持有的股票市值加上我们首次公开发行的拟议票据总额少于70000万美元，我们的年度营业收入在最近完成的财政年度内少于10000万美元。只要我们（i）非关联方持有的股票市值低于25000万美元，或者（ii）我们的年度营业收入在最近完成的财政年度内少于10000万美元且非关联方持有的股票市值低于70000万美元，我们将继续是较小的报告公司。如果在我们不再是新兴成长公司时我们仍是一家较小的报告公司，我们可以继续依靠适用于较小报告公司的某些豁免披露要求。具体来说，作为一家较小的报告公司，我们可以选择在我们的年度报告10-K中只呈现最近两个财政年度的审计财务报表，与新兴成长公司类似，较小的报告公司在执行薪酬方面有减少的披露义务。
项目3。关于市场风险的定量和定性披露
根据SEC规则和法规，由于我们被视为“较小的报告公司”，所以在本报告中不需要提供本项目要求的信息。
项目4。控制和程序
披露控件和程序的评估
我们保持“披露控制和程序”，如《证券交易法》第13a-15(e)条和第15d-15(e)条所定义，旨在确保公司在根据证券交易法规定提交的报告中需要披露的信息在SEC规则和表格规定的时间内被记录、处理、总结和报告。披露控制和程序包括但不限于旨在确保公司在根据证券交易法规定提交的报告中需要披露的信息被积累并传达给我们的管理层，包括我们的首席执行官和首席财务官，以便及时作出有关所需披露的决定。
截至2024年9月30日，管理层在我们的首席执行官和首席财务官的监督和参与下，评估了我们披露控制和程序的设计和运行的有效性（如《证券交易法》第13a-15(e)条和第15d-15(e)条所定义）。我们的披露控制和程序旨在合理保证我们在根据适用规则和表格规定提交的报告中需要披露的信息在规定的时间内被记录、处理、总结和报告。根据该评估，我们的首席执行官和首席财务官得出结论，截至2024年9月30日，我们的披露控制和程序是有效的。
关于基本报表的内部控制变化及管理报告
在适用于2024年9月30日结束的九个月内进行的根据《交易所法》规定的评估中，未发现对我们的内部财务报告控制产生实质性影响或可能产生实质性影响的内部控制变化。
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控制和程序的有效性存在固有限制
我们的管理层，包括首席执行官和财务副总裁，相信我们的披露控制程序和内部财务报告控制设计旨在提供合理保证达到其目标，并在合理保证级别上是有效的。但是，管理层并不认为我们的披露控制程序或我们的内部财务报告控制能够防止或发现所有错误和欺诈。无论控制系统构思和操作得多好，都只能提供合理的、而非绝对的保证，确保控制系统的目标得以实现。由于任何控制系统的固有局限性，并没有一项控制评估能够绝对保证在公司内是否已检测到所有的控制问题和欺诈行为。任何控制系统的设计也部分基于对未来事件发生可能性的假设，而且无法保证任何设计在各种潜在未来情况下都能成功实现其既定目标。随着时间推移，由于条件变化或遵守政策或程序的程度可能恶化，控制可能会变得不足。由于成本效益型控制系统的固有局限性，由于错误或欺诈而导致的错误可能会发生而不会被发现。
第二部分-其他信息
项目1。法律诉讼。
我们可能不时卷入在业务日常中发生的法律诉讼，我们预计解决这些诉讼不会对我们的财务状况、经营业绩或现金流产生重大不利影响。然而，无法确定任何可能发生的未来诉讼是否会对我们的业务产生重大的财务影响。截至本报告日期，我们并非涉及任何重大法律事项或索赔。
第1A项。风险因素。
本报告中包含的风险因素可能导致实际结果与我们在本报告中提出的或不时提出的前瞻性陈述有所不同。在做出投资决策之前，您应仔细考虑以下风险，除本报告及我们的其他公开文件中所包含的其他信息外。我们的业务、财务状况或业务成果可能会受到任何这些风险的损害。以下所述的风险和不确定因素并不是我们面临的唯一风险。我们目前还不知道或目前还不认为对我们业务造成重大风险的其他风险也可能影响我们的业务运营。
与我们的财务和资本需求有关的风险
由于我们的有限经营历史，您可能难以评估我们迄今为止的业务成功和评估我们未来的生存能力。我们的活动始于2010年，迄今为止，我们的工作主要集中在筹集资本、寻找和开发我们的产品和临床前计划、拓展我们在开发产品方面的专业知识以及进行临床前研究并进行早期临床试验。由于FDA于2019年12月发布的重新分类规定，我们不得不暂停在美国销售用于治疗焦虑和失眠的Gen-1器械。我们目前正在评估是否继续为失眠和焦虑治疗修订FDA的旧申请或为我们的下一代器械新申请510(k)。虽然我们开发了第二代和第三代的器械，但这些器械尚未获得FDA在美国市场销售的批准。因此，如果我们的运营历史更长，您对我们未来成功或生存能力的任何预测可能不够准确。
我们是一家临床阶段的生物制药公司，经营历史有限。我们于2020年7月在特拉华州注册成立，至今的运营范围仅限于组织和人员配备、业务规划、筹集资金、识别、调查、许可和评估潜在药物候选品，以及与第三方达成协议，生产我们的主要药物候选品和组件材料的初始数量。我们的两个药物候选品均处于早期临床开发阶段。我们尚未证明能够成功进行或完成任何药物候选品的临床开发计划，获得上市批准，生产商业规模产品，或安排第三方代表我们进行，或进行必要的销售、市场营销和分销活动，以便成功将产品商业化。因此，对于我们未来成功或生存能力的任何预测可能不够准确。
在某个时候，我们可能需要从一个以研发为重点的公司过渡为能够支持完整产品生命周期相关商业活动的公司。我们可能在这种转变中不会取得成功。
自成立以来，我们一直亏损，并预计在可预见的将来会继续亏损。我们可能永远无法实现或维持盈利。
对生物制药产品开发的投资是一项高度投机性的事业，需要大量前期资本支出，并存在当前或潜在未来产品候选者可能无法表现出充分疗效或
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具有可接受的安全性能，并获得监管批准并具备商业化的可行性。我们仍处于产品候选品开发的早期阶段，并于2021年12月启动了首个临床试验。我们没有任何获批用于商业销售的产品，也没有从产品销售中产生任何营业收入。我们将继续承担与我们正在进行业务相关的重大研发和其他支出。此外，作为一家经营历史有限的企业，我们可能会遇到未预料的支出、困难、复杂情况、延迟以及其他已知和未知因素，如新冠肺炎疫情。
自我们开始业务运营以来，各个时期都亏损。自创立以来至2024年9月30日结束，我们累积亏损达6700万美元。我们预计在可预见的未来将继续承担重大亏损，并且我们预计如果继续进行我们的主导产品候选品的研发工作； 为我们目前和未来的产品候选品进行临床前研究和临床试验； 寻求任何成功完成临床试验的目前或未来产品候选品的市场批准； 遇到任何延迟或在上述任何一个方面遇到任何问题； 建立销售、营销和分销基础设施和扩大制造能力以推广我们可能获得监管批准的任何目前或未来产品候选品； 取得、扩展、保持、实施和保护我们的知识产权组合； 雇佣更多临床、监管和科学人员； 并作为一家上市公司经营。
我们的产品候选品NXP800和NXP900均处于临床研发阶段。这两个产品候选品将需要进行额外的临床前和临床研究，经过监管审查和批准，大量投资，获得足够的临床和商业生产能力，以及在我们可能从产品销售中潜在获得任何营收之前进行重大的营销工作。NXP800的1期研究于2021年12月开始，NXP900于2023年9月开始。迄今为止，我们尚未从我们的产品候选品获得任何营业收入。我们创收的能力将取决于多个因素，包括但不限于：
| ● | 我们能够及时且成功地完成我们的临床前研究和临床试验，这可能比预期更为缓慢或更为昂贵，将取决于多个因素，包括第三方承包商的表现，我们招募患者参与临床试验的能力，以及在临床试验中产生的安全性、耐受性和疗效结果； |
| ● | 成功向FDA提交IND申请以及任何额外的类似申请； |
| ● | 完成为IND或类似提交所需的非临床研究，必要时； |
| ● | 我们是否被FDA或类似的外国监管机构要求进行额外的临床试验或其他研究，以支持我们当前或未来产品候选药物的批准和商业化； |
| ● | 美国食品药品监督管理局和类似的外国监管机构对我们当前或未来产品候选者的安全性、效力、纯度、功效和风险受益配置的接受情况; |
| ● | 我们当前或未来产品候选者可能出现的副作用或其他安全问题的盛行率、持续时间和严重程度，如果有的话; |
| ● | 从FDA和相似的外国监管机构获得必要的营销批准的时间; |
| ● | 我们当前或未来产品候选者（如果获批）的实际和感知可得性、成本、风险配置以及相对于现有和未来可替代的癌症治疗和竞争产品候选者和技术的安全性和功效，以及竞争者的产品候选者和技术; |
| ● | 我们和第三方承包方的制造业-半导体能力足够，可以制造出我们现有或未来的产品候选的足够临床和商业供应，保持良好的与监管机构合规，开发、验证和维护符合cGMP商业可行制造流程； |
| ● | 我们有能力成功制定商业策略，并在美国和国际市场上销售和分销任何现有或未来产品候选，是否获批上市、报销、销售和在这些国家和地区分销，无论是独自还是与他人合作； |
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| ● | 当批准后，患者对我们当前或未来产品候选者的需求；和 |
| ● | 我们在我们当前或未来产品候选者中建立和执行知识产权的能力。 |
上述许多因素超出了我们的控制范围，可能导致我们经历重大延迟或阻止我们获得监管批准或商业化我们当前和未来产品候选者。即使我们能够商业化任何当前或未来产品候选者，我们可能不会在生成产品销售后不久或永远实现盈利。
我们将需要大额额外资金。筹集额外资金可能导致我们现有股东的稀释，或要求我们放弃专有权利。如果我们无法按需筹集资本，我们可能被迫延迟、减少或取消我们的产品开发计划或商业化努力。
我们预计我们的支出将随着我们当前活动的增加而增加，特别是在我们继续活动以确定新的产品候选者并启动临床试验，以及寻求对我们当前或将来的任何产品候选者进行市场批准之际。此外，如果我们为我们当前或将来的任何产品候选者获得市场批准，我们预计将承担与产品销售、市场营销、制造和分销相关的重大商业化支出。此外，我们预计将承担作为一家上市公司运营的重大额外成本。因此，我们将需要在持续运营过程中获得大额额外资金。我们无法确定是否会获得额外资金以可接受的条件提供，或根本不会提供。直到在某种时候，如果有的话，我们可以获得大量产品收入，我们预计将通过公开或私人股权发行，ATM融资，债务融资，政府资助，合作，战略合作伙伴关系或与第三方的市场、分销或许可安排等方式融资我们的运营。在通过出售股权或可转换债务证券进行筹集额外资本的情况下，您的所有权权益将进一步被稀释，并且这些证券的条款可能包括可能不利影响您作为股东的权利的清算或其他优先权。债务融资和优先股权金融，如果可用，可能包括限制或限制我们采取特定行动的协议，例如增加债务、进行资本支出或宣布分红。
如果我们通过与合作伙伴、战略联盟或营销、分销或许可安排筹集额外的资金，我们可能不得不放弃我们的技术、未来收入流、研究计划或产品候选者的宝贵权利，或以对我们不利的条件授予许可。
如果我们在需要资本时无法融到资金，或者融资条件不佳，我们可能被迫推迟、减少或取消我们的发现和临床前开发计划，或任何未来的商业化努力。
重大的公共卫生事件，特别是仍在实施中的COVID-19大流行期间所采取的政策和程序，可能对我们的临床试验、财务状况、经营业绩和业务的其他方面产生不利影响。
COVID-19大流行对全球社会、经济、金融市场和商业实践产生了广泛、迅速发展和不可预测的影响。在2020年和2021年期间，采取了政策和程序，至今仍在执行，包括多种疫苗和药物的广泛分发以应对大流行。我们已将运营方式调整为与大流行共存。
COVID-19大流行对公司未来业务产生影响的不确定性影响了管理层对各种依赖于这些估计和假设的会计估计的判断和假设。自公司于2021年开始运营以来，COVID-19对这些估计和判断没有起到实质性的影响。
公司继续面临相对不确定性，关于继续实施的政策和程序、大流行的强度和持续时间，以及从COVID-19大流行中恢复的性质和时间表，以及对公司的影响，包括潜在的永久性变化临床试验运营由大流行引起的程度。公司采取了管理这种不确定性的方式（在这种情况下继续是重要因素）通过强化其资产负债表和现金资产，避免债务，同时专注于成本控制。COVID-19爆发或任何爆发形成的一些因素
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由COVID-19的任何变种引起的延误或其他不利影响我们的临床试验计划，以及总体不利影响我们的业务，包括：
| ● | 临床试验现场启动时的延迟或困难，包括招募临床试验现场的困难，以及由于感染COVID-19或任何变种而导致临床试验中的患者出现增加的退出率，被迫隔离，或者无法完成研究评估，尤其是对于年长患者或其他更容易感染COVID-19或任何变种的人； |
| ● | 医疗资源（包括临床试验研究员和工作人员）转移，从开展临床试验转向关注大流行引起的担忧，可能导致合作伙伴公司的临床试验出现延迟； |
| ● | 旅行限制，包括国内和国际旅行的限制，以及政府强制实行的隔离或由重要第三方实施的限制，可能会中断关键试验活动，如临床试验现场启动和监测； |
| ● | 由于人手短缺、生产放缓或停顿，我们从合同制造组织获取产品候选品的供应中断或延迟； |
| ● | 由潜在的工作场所、实验室和办公室关闭以及整个医疗体系员工云办公增加导致的中断和延迟； |
| ● | 由于COVID-19或任何变种传播导致的疫情对FDA或其他监管机构的运作产生影响，导致监管批准、检查、审核或其他监管活动中断或延迟； |
我们目前依赖第三方为我们临床试验和业务关键领域的某些功能或服务提供支持。如果这些第三方受COVID-19疫情限制的影响，我们可能会遇到延迟和/或额外成本。因此，我们启动和完成临床试验、获得当前和未来产品候选品的监管批准以及商业化的能力可能会延迟或受到干扰。
与我公司药品候选品开发相关的风险
我们的发展方法可能永远无法导致市场化产品。
我们产品候选人群和潜在未来产品候选人群的患者人群，可能仅限于具有特定靶点突变的人群，可能尚未完全定义，但明显小于总体治疗癌症患者人群，并且我们将需要积极筛选和识别这些患者。成功识别患者取决于几个因素，包括确定具有特定遗传突变的疾病如何响应我们目前的产品候选人或任何未来的产品候选人，并且如有必要，开发配套诊断来识别这类遗传突变。此外，即使我们成功识别患者，我们也不能肯定为我们目标适应症而产生的患者群体是否足够大以实现盈利。此外，即使我们的方法成功，我们也可能永远无法成功识别我们的产品候选人可能有效的额外疾病。我们不知道我们用特定基因定义的癌症患者对患者的治疗方法是否会成功；如果我们的方法不成功，我们的业务将受到影响。
我们在开发工作的早期阶段，极大地依赖于推进NXP800、NXP900或任何未来的其他产品候选人通过临床前和临床开发，确定我们药物候选人的安全和有效剂量和用药方案，获得监管批准并最终商业化NXP800、NXP900或任何未来的其他产品候选人的能力；如果我们在此方面遇到延误，我们的业务将受到重大损害。
我们从NXP800、NXP900或任何未来的其他产品候选人中产生产品收入的能力，极大地依赖于成功的临床开发和最终商业化。此外，我们的药物开发计划可能考虑开发配套诊断，这是基于基因突变和其他改变来识别适当患者人群的检测或测试。配套诊断属于医疗设备监管范围，必须在可以营销之前从FDA或某些其他外国监管机构获得营销授权。如果配套诊断
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对于我们目前和未来的所有产品候选品，为了能够安全有效地使用，FDA必须得出结论，配套诊断工具符合适用的安全和有效性标准或用于产品候选品的实质等同性标准，才能在美国市场推广产品候选品或配套诊断工具。
在开发我们的产品候选品的临床前期或临床研究中出现负面结果可能会阻止或延迟我们继续进行临床项目或获得监管批准的能力。例如，尽管我们基于ARID1a缄默卵巢癌模型的临床前期研究表明有抑制肿瘤生长的迹象，认为这种癌症类型可能对NXP800的治疗特别敏感，但在临床测试中可能并非如此，可能由于安全性、耐受性问题和/或疗效不足，在任何目标适应症中同样成立。此外，抗肿瘤活性在我们计划在临床试验中评估的每种肿瘤类型中可能不同。因此，我们可能需要停止开发我们的药物候选品或投入大量额外资源，延迟我们的临床试验，并最终延迟任何我们未来产品候选品的批准（如果有的话）。
由于在早期临床试验中出现安全性、耐受性和/或疗效不足等原因，我们目前或未来的产品候选品可能在早期临床试验中没有良好的结果。此外，早期临床试验的积极结果并不一定能预测未来的结果，我们推进的任何产品候选品可能在后续临床试验中没有良好的结果或获得监管批准。2024年3月，我们宣布了正在进行中的NXP800铂金耐药ARID1a突变卵巢癌10亿期研究的初步安全性和疗效结果。在这一初步分析中观察到的任何结果可能并不能预测未来的结果。
我们可能遇到挫折，可能会延迟或阻止我们的当前或未来产品候选品获得监管批准或我们进行商业化，其中包括：
| ● | 负面或不确定的功效结果和/或临床试验中的不良安全性发现，或他人的临床试验取得积极结果，类似于我们的产品候选品，导致其批准，并发展为决定或要求进行额外的临床前测试或临床试验，或放弃一个项目； |
| ● | 由我们的临床试验中的患者或被试者或正在使用我们、FDA、其他监管机构或他人视为与我们当前或未来产品候选品开发相关的药物或治疗品的个人体验到的与产品相关的副作用； |
| ● | 提交IND申请或类似的国外申请时出现延迟，或者在开始临床试验之前未能获得监管机构的必要批准，或者一旦开始就暂停或终止临床试验的延迟或失败； |
| ● | FDA或与我们的临床试验范围或设计有关的类似国外机构施加的条件，包括我们的临床终点； |
| ● | 临床试验中受试者招募出现延迟，包括难以或延迟识别合适的患者，或由于健康相关紧急事件，如COVID-19大流行，以及按照GCP或良好实验室规范（“GLP”）要求及时完成临床试验； |
| ● | 无法保持与监管要求的合规性，包括cGMP合规，并有效遵守与我们当前或未来产品候选品质相关的其他要求； |
| ● | 由于安全性和耐受性问题和/或疗效不足，临床试验中受试者的高退出率； |
| ● | 我们当前或未来产品候选品的供应或质量不足，或进行临床试验所需的其他材料不足； |
| ● | 临床试验成本高于预期; |
| ● | 无法与其他疗法竞争； |
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| ● | 我们当前或未来的产品候选者效果不佳，存在安全性和/或耐受性问题，或在临床试验过程中需要不同剂量或给药计划； |
| ● | 试验结果所需时间超出预期； |
| ● | 试验遭受欺诈、数据采集故障或其他技术故障导致试验无效； |
| ● | 我们试验的结果不支持在欧盟申请条件批准； |
| ● | 不利的FDA或其他监管机构对临床试验现场的检查和审查； |
| ● | 我们的第三方承包商或调查人员未能遵守监管要求或未能及时履行其合同义务，或根本没有履行； |
| ● | 由于COVID-19大流行的影响而导致的延迟，包括COVID-19对FDA继续正常运作的影响； |
| ● | 监管要求、政策和指导方针的延迟和变更，包括针对临床开发通常或针对我们的技术特别加强额外监管监督；或 |
| ● | FDA和类似的外国监管机构对数据的不同解读。 |
此外，由于我们财务和人员资源有限，主要专注于开发NXP800和NXP900，因此我们可能放弃或推迟追求其他未来可能拥有更大商业潜力的产品候选药物，可能未能充分利用可行的商业产品或有利可图的市场机会。如果我们不能准确评估未来产品候选药物的商业潜力或目标市场，我们可能通过合作、许可或其他版税安排放弃对这些未来产品候选药物有价值的权利，在这些情况下，我们保留独家开发和商业化权利可能更为有利。
临床药物开发涉及漫长而昂贵的过程，结果不确定，临床试验难以设计和实施，我们任何一项临床试验都可能产生失败的结果或在任何阶段失败。
临床试验在人体上进行，费用昂贵，可能需要多年才能完成，结果固有地不确定。失败可能发生在任何时间。此外，药物候选物的早期研究和早期临床数据的任何积极结果可能不能预测早期临床试验的最终结果或后续临床试验的结果，使得药物候选物可能无法根据早期临床试验的结果达到后续阶段的临床试验，或者在后续的临床试验中未能展现出期望的安全性和有效性特征，尽管在早期研究和早期临床试验中显示出这些特征的迹象。制药行业中许多公司由于缺乏功效或不良的安全性特征，在早期和爱文思控股临床试验中遭受重大挫折，尽管在早期研究或初步临床研究中取得了有希望的结果。因此，我们进行的任何现有和未来的临床试验的结果可能会失败。临床试验可能由于成本高于预期或出于各种原因而延迟、暂停或过早终止，例如：
| ● | 药品审批达成一致或与FDA或类似的外国监管机构达成一致，以执行我们能够执行的试验设计可能存在延迟或失败； |
| ● | 延迟或未能获得启动试验的授权，包括获得适当独立审查委员会(IRB)批准在人体上对候选者进行测试，或无法遵守监管机构就临床试验的范围或设计所施加的条件； |
| ● | 延迟达成或未能达成与潜在CRO和临床试验地点就可接受条款达成一致的协议，这些条款可能经过广泛谈判并在不同的CRO和试验地点之间可能有很大差异； |
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| ● | 在确定和维护足够数量的试验点方面存在无力、延迟或失败的情况，其中许多试验点可能已经参与了其他临床项目； |
| ● | 招募和注册合适的志愿者或患者参与试验方面可能存在延迟或失败的情况； |
| ● | 如果被视为必要，在及时基础上开发和验证伴随诊断工具可能存在延迟或失败的情况； |
| ● | 患者未能完成试验或未能返回进行治疗后的随访; |
| ● | 无法在治疗期间或治疗后充分监测患者; |
| ● | 临床试验现场和研究者偏离试验方案, 未能根据监管要求进行试验或退出试验; |
| ● | 由于观察到的安全问题或其他原因而由FDA或类似外国监管机构实施的临床控件导致未能启动或延迟或无法完成临床试验; |
| ● | 临床试验中出现负面或不确定结果，我们决定符合监管机构的要求进行额外的临床前研究、临床试验或放弃一个或多个产品开发项目；或者 |
| ● | 无法制造出足够数量和符合质量标准的药物候选品用于临床试验。 |
我们依靠并计划继续依靠CROs、合同制造组织（“CMOs”）和临床试验点来确保对我们临床试验的适当和及时进行。尽管我们已经并预期将来会与CROs和CMOs签订管理其承包活动和行为的协议，但我们对其实际表现具有有限影响力。因此，我们最终并不会对CRO或CMO遵守与我们可能有的任何协议条款，遵守适用的监管要求，或遵守已经达成的时间表和期限负有控制权，而未来CRO或CMO未能执行这些义务可能导致我们任何临床试验受到延迟或失败。
此外，如果临床试验被暂停，被放弃或被我们、IRB或伦理委员会、数据安全监控委员会、FDA或欧洲药品管理局（“EMA”）或其他监管机构终止，我们也可能遇到延迟。暂停或终止可能由许多因素引起，包括未能按照法规要求进行临床试验，FDA、EMA或其他监管机构对临床试验操作或试验地点的检查，使参与者面临由意外安全问题或不良副作用引起的健康风险，先前未曾见过的安全问题的发展，未能证明使用药物候选品具有益处，或者政府法规或行政行动的变化。因此，我们无法确定当前正在进行、计划或未来任何临床试验的开始或完成时间表。
导致临床试验启动或完成延迟的许多因素，也可能最终导致否决我方当前或未来产品候选者的营销批准。
如果我们在启动或完成任何药物候选者的临床试验中遇到延迟，或者出现暂停、中止、暂停或终止，该药物候选者的商业前景可能会受到损害，我们从该药物候选者中产生产品收入的能力可能会延迟或被消灭。此外，临床试验完成的任何延误都将增加我们的成本，使我们的药物候选者开发和批准流程减慢，并危及我们药物候选者的监管批准以及开始销售和产生收入的能力。凡此种种事件的发生都可能严重损害我们的业务、财务状况、运营结果和前景。
招募患者的困难可能会延迟或阻止我们当前或未来产品候选者的临床试验。
确定和资格认定参与我们当前或未来产品候选者临床研究的患者对我们的成功至关重要。我们临床研究完成的时机在一定程度上取决于我们能够招募患者参与测试我们当前或未来产品候选者的速度，如果我们在招募方面遇到困难，我们可能会在临床试验中遇到延迟。
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此外，由于我们目前专注于患有特定和罕见疾病的患者，我们招募符合条件的患者的能力可能受限，可能导致招募速度比预期慢。我们的临床试验将与其他临床试验竞争，即当前或未来的产品候选品与我们当前或未来的产品候选品属于相同的治疗领域，这可能减少可供我们使用的患者数量和类型。
由于患者招募所需时间超出预期或患者退出超出预期，临床试验可能会出现延迟。如果我们无法找到并招募足够数量的符合FDA或国外监管机构要求参与这些试验的符合条件的患者，我们可能无法启动或继续当前或未来产品候选品的临床试验。我们无法预测我们未来在招募受试者方面将取得多大成功。患者的招募取决于许多因素，包括：
| ● | 患者识别和符合/不符合方案中定义的资格和包含/排除标准； |
| ● | 临床试验主要终点分析所需的患者人口规模以及识别患者的流程； |
| ● | COVID-19大流行可能导致的潜在干扰包括启动临床试验点困难、招募和保留参与者困难、医疗保健资源转移至临床试验、可能实施的旅行或隔离政策以及其他因素； |
| ● | 患者与临床试验点的距离； |
| ● | 试验的设计； |
| ● | 我们招募具有适当能力和专业知识的临床试验研究者的能力； |
| ● | 临床医生和患者对正在研究的产品候选品相对于其他可用疗法的潜在优势和风险的看法，包括任何可能获得批准用于我们正在研究适应症的新产品； |
| ● | 市场上竞争性可用疗法的可获得性和其他竞争性产品候选品的临床试验； |
| ● | 我们获取和保持临床试验受试者知情同意的能力； |
| ● | 临床试验中被招募的受试者在完成之前退出试验的风险。 |
如果我们无法找到并招募足够符合FDA或类似监管机构要求参与的合格患者，我们可能无法启动或继续我们当前或未来产品候选品的临床试验。如有必要，我们打算与第三方合作开发伴随诊断产品，用于我们的临床试验。如果这些第三方无功而返，我们在识别患有我们临床试验所针对的遗传突变的患者方面可能会遇到困难。如果我们无法纳入患有目标遗传突变或有明确定义严重未满足医疗需求的患者，我们可能无法参与FDA的加速审核和发展计划，包括突破性疗法认定和快速路径认定，或寻求加速临床开发和监管时间表。
我们的研究可能无法充分证明我们当前或未来任何产品候选品的安全性、效力、纯度、功效或其他必要的药理特性，这可能会阻碍或延迟开发、监管批准和商业化。
在获得对我们当前或未来产品候选品进行商业销售的监管批准之前，包括NXP800和NXP900，我们必须通过冗长、复杂和昂贵的研究证明我们当前或未来产品候选品在每个目标适应症中既安全又有效。临床前和临床测试费用高昂，可能需要多年才能完成，其结果本质上是不确定的。失败可能发生在任何时候，包括临床前研究和临床试验过程中，因为我们当前的产品候选品处于早期开发阶段，所以存在很高的失败风险。
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The results of preclinical studies and early clinical trials of a drug candidate may not be predictive of the final results of later-stage clinical trials. Results of our trials could reveal a high and unacceptable severity and prevalence of adverse safety issues which may result in suspension or termination, and the FDA or comparable foreign regulatory authorities could, through a clinical hold or otherwise, order us to halt or cease further development of or deny approval. Drug-related side effects could also affect patient recruitment into the study or patient willingness to remain in the study and therefore affect our ability to complete clinical trials. Drug-related side effects could also result in potential product liability claims. Any of these occurrences may harm our business, financial condition and prospects significantly.
Moreover, our product or product candidates may cause undesirable side effects that could delay or prevent their regulatory approval or impact their availability and commercial potential after approval.
The FDA and comparable foreign regulatory authorities may not accept data from any preclinical or clinical trials we may conduct in foreign countries.
The FDA’s acceptance of data generated for patients recruited outside the United States from clinical trials conducted in whole or in part outside the United States may be subject to certain conditions, if accepted at all.
Although the FDA has the authority to accept foreign data as part or even the sole basis for marketing approval, the FDA generally does not approve an application on the basis of foreign data alone unless (i) the data is applicable to the U.S. population and U.S. medical practice, (ii) the trials were performed by clinical investigators of recognized competence and pursuant to GCP regulations, and (iii) the FDA’s clinical trial requirements were met. Many foreign regulatory authorities have similar approval requirements. In addition, any clinical study conducted in whole or in part outside of the United States would be subject to the applicable local laws of the jurisdiction where the trial was conducted. We cannot guarantee that the FDA or comparable foreign regulatory authority will accept data from trials conducted in whole or in part outside of the United States, which may result in the need for additional trials.
We may not be able to submit IND applications to commence additional clinical trials on the timelines we expect, and even if we are able to, the FDA may not permit us to proceed.
Our CTAs and INDs for NXP800 and NXP900, have been approved. However, we may be unable to submit additional CTAs, IND applications or other clinical research on our expected timelines. Moreover, while we have obtained CTA and IND approvals, we cannot be sure that issues will not arise that may lead to the delay, suspension or termination of such clinical trials. Any failure to file CTAs, IND applications or other clinical research authorizations will adversely impact our expected timelines to obtain regulatory acceptance for the commencement of our trials and may prevent us from completing our clinical trials or commercializing our products on a timely basis, if at all.
We currently have no marketing and sales organization and have limited experience in marketing products. If we are unable to establish marketing and sales capabilities or enter into agreements with third parties to market and sell any approved product candidates, we may not be able to generate product revenue.
We will have to compete with other pharmaceutical and biotechnology companies to recruit, hire, train and retain marketing and sales personnel. If we are unable or decide not to establish internal sales, marketing, and distribution capabilities, we may pursue arrangements with third-party sales, marketing, and distribution collaborators regarding the sales and marketing of our products, if approved.
There can be no assurance that we will be able to develop in-house sales and distribution capabilities or establish or maintain relationships with third-party collaborators to commercialize any product in the United States or overseas.
We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do.
While we believe that our scientific knowledge, technology, and development expertise provide us with competitive advantages, we face potential competition from many different sources, including major pharmaceuticals, specialty pharmaceuticals and biotechnology companies, academic institutions and government agencies, and public and private research institutes that conduct research, development, manufacturing, and commercialization. Many of our competitors have significantly greater financial resources and expertise in research and development, manufacturing, preclinical testing, regulatory approvals, and product marketing than we do. Our
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竞争对手可能会在招聘和留住合格的科学和管理人员、建立临床试验中心和招募临床试验患者，以及获取与我们项目相关的补充或必要的技术方面与我们竞争。结果可能是，我们的竞争对手可能会比我们更早或更成功地发现、开发、许可或商业化产品。
如果我们的产品候选药NXP800和NXP900获得批准，它们可能会与竞争对手药物和其他目前正在开发中的药物竞争。政府和其他第三方支付者对产品的报销性也将显著影响我们产品的定价和竞争力。我们的竞争对手可能会比我们更快地获得FDA或其他监管部门对其产品的批准，这可能导致我们的竞争对手在我们进入市场之前建立强劲的市场地位。
政府监管风险
未能或延迟获得必要的监管批准可能会阻止或推迟我们目前或未来产品候选品的商业化，我们产生营业收入的能力可能会受到实质性损害。
药物产品的研究、测试、制造、标签、批准、销售、营销和分销在美国受FDA和其他国家/地区的监管机构的广泛监管，各国的监管规定也不同。在我们收到FDA的NDA相应批准之前，我们将不被允许在美国推出我们目前或未来的产品候选品，也不会被允许在任何海外国家推出，直到我们收到各国家/地区的监管机构的批准。获得FDA、EMA和类似外国机构批准的时间是不可预测的，通常需要很多年的临床试验后才确定，并取决于众多因素，包括监管机构的广泛裁量权和涉及的产品候选品的类型、复杂性和新颖性。监管机构在批准过程中具有相当大的裁量权，他们可能拒绝接受任何申请，或认为我们的数据不足以获得批准，需要额外的非临床研究或临床试验。
获取监管批准需要提交广泛的非临床和临床数据以及支持信息给监管机构的每个治疗适应症，以建立产品候选药物的安全性和有效性。获得监管批准还需要提交关于产品制造过程的信息，并且在许多情况下，监管机构会对制造、加工和包装设施进行检查。我们目前或未来的产品候选药物可能无效，可能只是适度有效，或可能被证明具有不良或意外的副作用、毒性或其他特征，这可能会阻止我们获得上市批准，或阻止或限制商业使用，或者我们或我们的CMO在cGMP合规方面可能存在缺陷，这可能会导致候选药物无法获得批准。此外，我们尚未在任何司法管辖区获得任何药物候选品的监管批准，我们目前的药物候选者或未来可能寻求开发的任何药物候选者可能永远都不会获得监管批准。
出于许多原因，我们的药物候选者可能无法获得监管批准，或者可能被延迟获得监管批准，包括以下任何一个或多个原因:
| ● | 美国食品药品监督管理局（FDA）、欧洲药品管理局（EMA）或类似的外国监管机构可能不同意我们临床试验的设计或实施; |
| ● | 我们可能无法向FDA、EMA或类似的外国监管机构证明一种药物候选品对其拟议适应症是安全和有效的; |
| ● | 临床试验的结果可能不符合FDA、EMA或类似的外国监管机构批准所需的统计显著性水平; |
| ● | 我们可能无法证明候选药物的临床和其他优势超过其安全风险； |
| ● | FDA、EMA或类似的外国监管机构可能不同意我们对临床前研究或临床试验数据的解释； |
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| ● | 我们药物候选品临床试验收集的数据可能不足以支持提交新药申请或其他申请，也可能无法在美国或其他地方获得监管批准； |
| ● | 在审查我们的临床试验现场和数据时，FDA或类似的外国监管机构可能会发现我们的记录保存或我们临床试验现场的记录保存不足； |
| ● | 我们与合同制造临床和商业用品的第三方制造商的制造流程或设施可能无法满足FDA、EMA或类似的外国监管机构的要求； |
| ● | 美国食品和药物管理局（FDA）、欧洲药品管理局（EMA）或类似的外国监管机构可能未能批准我们计划与合作伙伴内部开发的伴随诊断设备;且 |
| ● | 医疗标准变更或美国FDA、欧洲EMA或类似的外国监管机构的批准政策或法规可能发生显著变化，使我们的临床数据不足以获得批准。 |
获得市场许可的审批过程所需的时间和费用，以及未来临床试验结果和其他影响因素的不可预测性，可能导致我们未能获得NXP800、NXP900或我们未来可能寻求开发的任何其他药物候选品的监管批准，这将严重损害我们的业务、运营结果和前景。在这种情况下，我们可能也没有资源进行新的临床试验和/或我们可能判断进一步开发任何此类药物候选品没有正当理由，可能会中止任何此类项目。
此外，即使我们在一个或多个司法管辖区获得监管批准，监管机构也可能只批准我们的任一药物候选品用途较少或受限制的适应症，可能不会批准我们提出的产品收费价格，可能批准取决于昂贵的后市场临床试验表现（根据司法辖区不同分别称为“有条件”的或“加速”的批准），或可能批准的药物候选品标签中不包含对该药物候选品成功商业化所必要或理想的标签声明。前述任何情况均可能对我们药物候选品的商业前景造成重大损害。
在一个司法管辖区获得我们当前或未来产品候选品的监管批准并不意味着我们将能够成功在其他司法管辖区获得我们当前或未来产品候选品的监管批准。
在一个司法管辖区获得任何我们当前或未来产品候选品的监管批准并不保证我们将能够在其他任何司法管辖区获得或保持监管批准，而在一个司法管辖区获得监管批准失败或延迟可能对其他司法管辖区的监管批准流程产生负面影响。例如，即使FDA批准了产品候选品，类似的外国监管机构也必须批准在这些国家中的产品候选品的制造、营销和促销。各司法管辖区的药品批准程序各不相同，并可能涉及比美国更多、更大的要求和行政审查期，包括额外的前期临床研究或临床试验，因为在一个司法管辖区进行的临床试验可能不被其他司法管辖区的监管机构接受。在美国以外的许多司法管辖区，产品候选品必须获得报销批准后才能在该司法管辖区销售。在某些情况下，我们打算为产品收费的价格也需获得批准。
我们也可以在其他国家提交市场申请。美国以外的司法管辖区的监管机构对产品候选者的批准有要求，我们必须在那些司法管辖区开始营销前遵守这些要求。获得类似的国外监管批准，并符合类似的国外监管要求可能导致我们遇到重大延迟、困难和成本，这可能会延迟或阻止我们在某些国家推出我们的产品。我们目前没有任何产品候选者获得批准在任何司法管辖区出售，包括国际市场，也没有获得国际市场监管批准的经验。如果我们未能遵守国际市场的监管要求和/或获得适用的市场批准，我们的目标市场将受到影响，我们无法实现当前或未来产品候选者的全部市场潜力。
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即使我们的现有或未来产品候选获得监管批准，我们将需要遵守持续的监管义务和持续的监管审查，这可能导致额外的重大开支，如果我们未能遵守监管要求或与我们的现有或未来产品候选出现意外问题，我们可能会受到处罚。
如果我们的任一现有或未来产品候选获得批准，则对产品的制造、标签、包装、存储、广告、促销、取样和产品记录将受到广泛和持续的监管要求。这些要求包括提交安全性和其他上市后信息和报告，注册，以及持续遵守cGMP法规。药品制造商和负责任何产品制造过程的任何CMO都必须遵守广泛的FDA和外国类似监管机构要求，包括确保质量控制和制造程序符合cGMP法规和任何适用的外国等效物。因此，我们及我们的CMO将接受持续的审查和检查，以评估对cGMP的遵守情况和对在任何NDA、其他市场申请和先前对检查观察的回应中所做承诺的遵守情况。因此，我们和我们合作的其他人必须继续在监管遵从的所有领域，包括制造、生产和质量控制方面，花费时间、金钱和精力。
FDA或类似的外国监管机构还可能对昂贵的上市后非临床研究或临床试验（通常称为“第4期试验”）和上市后监测提出要求，以监测产品的安全性或有效性。如果我们或监管机构发现产品存在先前未知的问题，如严重或频繁的意外不良事件、生产问题或在生产或加工产品的设施存在问题，如产品污染或不符合适用的cGMP法规在内，监管机构可能对该产品、制造工厂或我们实施限制。如果我们或第三方提供商，包括我们的CMO未能充分遵守适用法规，则我们可能需要启动产品的召回或下架。
对我们目前或未来产品候选项的原先未知问题的后期发现，包括意外严重程度或频率的不良事件，或与我们的第三方制造商或制造流程有关的问题，或未能遵守监管要求，可能导致以下情况，以及其他事项：
| ● | 对产品的制造、已批准生产商或制造流程实施限制； |
| ● | 对产品的标签或市场营销有所限制； |
| ● | 对产品分销或使用实施限制； |
| ● | 要求进行后市场研究或临床试验； |
| ● | 将产品从市场上撤回; |
| ● | 产品召回; |
| ● | 美国食品药品监督管理局（FDA）的警告信或无标题信函，或来自国外监管当局的类似违规通知； |
| ● | FDA或其他适用的监管机构拒绝批准待批准申请或批准申请的补充； |
| ● | 罚款、赔偿或返还利润或收益； |
| ● | 撤销或暂停市场准入批文； |
| ● | 暂停我们进行中的任何临床试验； |
| ● | 产品被扣押或拘留或拒绝允许进口或出口; 和 |
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| ● | consent decrees, injunctions or the imposition of civil or criminal penalties. |
In addition, regulatory authorities’ policies (such as those of the FDA or EMA) may change and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our current or future product candidates. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are otherwise not able to maintain regulatory compliance, we may lose any marketing approval that we may have obtained, which would adversely affect our business, prospects and ability to achieve or sustain profitability.
Non-compliance with European Union requirements regarding safety monitoring or pharmacovigilance can also result in significant financial penalties. Similarly, failure to comply with the European Union’s requirements regarding the protection of personal information can also lead to significant penalties and sanctions.
The FDA’s policies may change and additional government regulations may be enacted that could prevent, limit or delay marketing approval of our current or future product candidates. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, this may adversely affect, or even lead to the rescission of, the marketing approval that we may have obtained, which would adversely affect our business, prospects and ability to achieve or sustain profitability.
A variety of risks associated with marketing our current or future product candidates internationally could materially adversely affect our business.
We plan to seek regulatory approval of our current or future product candidates outside of the United States and expect that we will be subject to additional risks related to operating in foreign countries including: differing regulatory requirements; unexpected changes in tariffs, trade barriers, price and exchange controls; economic weakness, including inflation, or political instability in particular foreign economies and markets; compliance with tax, employment, immigration and labor laws for employees living or traveling abroad; foreign currency fluctuations that result in increased operating expenses, reduced revenue, and other obligations incident to doing business in another country; potential liability under the Foreign Corrupt Practices Act of 1977 or comparable foreign regulations; and challenges enforcing our contractual and intellectual property rights, especially in countries that do not recognize intellectual property rights to the same extent as the United States.
The insurance coverage and reimbursement status of newly approved products is uncertain. Our current or future product candidates may become subject to unfavorable pricing regulations, third-party coverage and reimbursement practices, or healthcare reform initiatives, which would harm our business. Failure to obtain or maintain adequate coverage and reimbursement for new or current products could limit our ability to market those products and decrease our ability to generate revenue.
Adverse pricing limitations may hinder our ability to recoup our investment in one or more of our current or future product candidates, even if any such current or future product candidate we may develop obtains marketing approval.
Our ability to successfully commercialize any current or future product candidates will depend in part on the coverage and reimbursement for the products and related treatments from government health administration authorities and third-party payors, such as private health insurers and health maintenance organizations. These organizations decide which medications they will pay for and establish reimbursement levels. If coverage and adequate reimbursement is not available, or the approved reimbursement amount is not high enough, we may be unable to establish or maintain pricing sufficient to generate a return on our investment and may be unable to successfully commercialize our current or future product candidates. Reimbursement by a third-party payor may depend upon a number of factors, including, but not limited to, the third-party payor’s determination that use of a product is a covered benefit under its health plan, safe, effective and medically necessary, appropriate for the specific patient, cost-effective, and neither experimental nor investigational. If coverage and adequate reimbursement is not available, or the approved reimbursement amount is not high enough, we may be unable to establish or maintain pricing sufficient to generate a return on our investment and may be unable to successfully commercialize our current or future product candidates.
A primary trend in the U.S. healthcare industry and elsewhere is cost containment. Government authorities and third-party payors have attempted to control costs by limiting coverage and the amount of reimbursement for particular medications. In general, the prices of medicines under such systems are substantially lower than in the United States.
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There is also significant uncertainty related to the insurance coverage and reimbursement of newly approved products, and coverage may be more limited than the purposes for which the medicine is approved by the FDA or comparable foreign regulatory authorities. In the United States, the principal decisions about reimbursement for new medicines are typically made by CMS. As a result, the coverage determination process is often a time consuming and costly process that may require us to provide scientific and clinical support for the use of our products to each payor separately, with no assurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance. It is difficult to predict what CMS will decide with respect to reimbursement for fundamentally novel products such as ours. Reimbursement agencies in Europe may be more conservative than CMS. Our inability to promptly obtain coverage and profitable payment rates from both government-funded and private payors for any approved products we may develop could have a material adverse effect on our operating results, our ability to raise capital needed to commercialize our current or future product candidates, and our overall financial condition.
Healthcare legislative measures and changes in policies, funding, staffing and leadership at the FDA and other agencies could hinder or prevent the commercial success of our products.
In the United States, there have been a number of legislative and regulatory changes to the healthcare system that could affect our future results of operations and the future results of operations of our potential customers.
In recent years, there has been heightened governmental scrutiny over the manner in which biopharmaceutical manufacturers set prices for their marketed products, which has resulted in several recent government inquiries as well as federal and state legislation designed to, among other things, increase drug price transparency, review the relationship between pricing and manufacturer patient programs, reduce the cost of drugs under Medicare, and reform government reimbursement for drug products. Congress and the executive branch have each indicated that they will continue to seek new legislative and/or administrative measures to control drug costs, making this area subject to ongoing uncertainty. At the state level in the United States, legislatures have also increasingly passed legislation and implemented regulations designed to control drug product pricing.
While we cannot predict what impact these laws or policies will have in general or specifically on any product we may commercialize in the future, such efforts by the government and payors may result in downward pressure on reimbursement, which could negatively affect market acceptance of new products. Any rebates, discounts, taxes costs or regulatory or systematic changes on healthcare may have a significant effect on our profitability in the future.
Given recent federal and state government initiatives directed at lowering the total cost of healthcare, the executive branch, Congress and state legislatures will likely continue to focus on healthcare reform and the reform of the Medicare and Medicaid programs. While we cannot predict the full outcome of any such government action or legislation, it may harm our ability to market our products and generate revenues.
Furthermore, regulatory authorities’ assessment of the data and results required to demonstrate safety and effectiveness can change over time and can be affected by many factors, such as the emergence of new information, including on other products, changing policies and agency funding, staffing and leadership. We cannot be sure whether future changes to the regulatory environment will be favorable or unfavorable to our business prospects.
Our future relationships with customers and third-party payors in the United States and elsewhere may be subject to applicable anti-kickback, fraud and abuse, false claims, transparency, health information privacy and security and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm, administrative burdens, diminished profits and future earnings.
Healthcare providers, physicians and third-party payors in the U.S. and elsewhere will play a primary role in the recommendation and prescription of any current or future product candidates for which we obtain marketing approval. Our future arrangements with third-party payors and customers may expose us to broadly applicable fraud and abuse and other healthcare laws and regulations, including, without limitation, the federal Anti-Kickback Statute and the federal False Claims Act, which may constrain the business or financial arrangements and relationships through which we sell, market and distribute any current or future product candidates for which we obtain marketing approval. In addition, we may be subject to transparency laws and patient privacy regulation by the federal and state
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governments and by governments in foreign jurisdictions in which we conduct our business. The applicable federal, state and foreign healthcare laws and regulations that may affect our ability to operate include, but are not necessarily limited to:
| ● | the federal Anti-Kickback Statute, which prohibits, among other things, persons from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under federal and state healthcare programs, such as Medicare and Medicaid; |
| ● | federal civil and criminal false claims laws and civil monetary penalty laws, including the federal False Claims Act, which impose criminal and civil penalties, including civil whistleblower or qui tam actions, against individuals or entities for knowingly presenting, or causing to be presented, to the federal government, including the Medicare and Medicaid programs, claims for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government; the federal Health Insurance Portability and Accountability Act of 1996 (“HIPAA”), which imposes criminal and civil liability for executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters; |
| ● | HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009, and their respective implementing regulations, which impose obligations on covered healthcare providers, health plans, and healthcare clearinghouses, as well as their business associates that create, receive, maintain or transmit individually identifiable health information for or on behalf of a covered entity, with respect to safeguarding the privacy, security and transmission of individually identifiable health information; |
| ● | the federal Open Payments program, which requires manufacturers of certain drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to report annually to CMS, information related to “payments or other transfers of value” made to “covered recipients,” which include physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors, and teaching hospitals) and applicable manufacturers. Applicable group purchasing organizations also are required to report annually to CMS the ownership and investment interests held by the physicians and their immediate family members. The SUPPORT for Patients and Communities Act added to the definition of covered recipient practitioners including physician assistants, nurse practitioners, clinical nurse specialists, certified registered nurse anesthetists and certified nurse-midwives effective in 2022. Data collection began on August 1, 2013 with requirements for manufacturers to submit reports to CMS by March 31, 2014 and 90 days after the end of each subsequent calendar year. Disclosure of such information was made by CMS on a publicly available website beginning in September 2014; and |
| ● | analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws, which may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers; state and foreign data privacy or data protection laws and regulations, such as state health data privacy legislation, state data breach legislation, or general state privacy legislation such as California’s Consumer Privacy Act (CCPA) and its implementing regulations; state and foreign laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government or otherwise restrict payments that may be made to healthcare providers; state and foreign laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures; and state and foreign laws governing the privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts. |
In November 2020, HHS finalized significant changes to the regulations implementing the Anti-Kickback Statute, as well as the Physician Self-Referral Law and the civil monetary penalty rules regarding beneficiary inducements, with the goal of offering the healthcare industry more flexibility and reducing the regulatory burden associated with those fraud and abuse laws, particularly with respect to value-based arrangements among industry participants.
Efforts to ensure that our business arrangements with third parties will comply with applicable healthcare laws and regulations may involve substantial costs. It is possible that governmental authorities will conclude that our business practices may not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations. If our
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operations are found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal and administrative penalties, including, without limitation, damages, fines, imprisonment, exclusion from participation in government healthcare programs, such as Medicare and Medicaid, and the curtailment or restructuring of our operations, which could have a material adverse effect on our businesses. If any of the physicians or other healthcare providers or entities with whom we expect to do business, including our collaborators, is found not to be in compliance with applicable laws, it may be subject to criminal, civil or administrative sanctions, including exclusions from participation in government healthcare programs, which could also materially affect our businesses.
If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.
We are subject to numerous environmental, health and safety laws and regulations, including those governing laboratory procedures and the handling, use, storage, treatment and disposal of hazardous materials and wastes. Our operations may involve the use of hazardous and flammable materials, including chemicals and biological and radioactive materials. Our operations also may produce hazardous waste products. We currently contract with third parties for the conduct of our manufacturing efforts and preclinical studies and clinical trials and such third parties are responsible for disposal of these materials and wastes. However, we cannot eliminate our risk of contamination or injury from these materials. In the event of contamination or injury resulting from our use of hazardous materials, we could be held liable for any resulting damages, and any liability could exceed our resources. We also could incur significant costs associated with civil or criminal fines and penalties.
Although we maintain workers’ compensation insurance to cover us for costs and expenses, we may incur due to injuries to our employees resulting from the use of hazardous materials, this insurance may not provide adequate coverage against potential liabilities. We do not maintain insurance for environmental liability or toxic tort claims that may be asserted against us in connection with our storage or disposal of biological, hazardous or radioactive materials.
Risks Related to our Intellectual Property
We currently hold a license to certain intellectual property rights relating to our lead product candidate, NXP800 and to NXP900, as well as intellectual property rights relating to other compounds that modulate HSF1 and the SRC and YES1 kinases. If we are unable to maintain patent and other intellectual property protection for NXP800 and NXP900, and to obtain and maintain patent and other intellectual property protections for our other current or future product candidates and technology, or if the scope of intellectual property protection obtained or maintained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and our ability to commercialize NXP800, NXP900 or any other current or future product candidates or technology may be adversely affected.
Our success depends in large part on our ability to obtain and maintain patent and other intellectual property protection in the United States and other countries with respect to our current or future product candidates, including NXP800 and NXP900, their respective components, formulations, combination therapies, methods used to manufacture them and methods of treatment and development that are important to our business, as well as successfully defending these patents against third-party challenges. If we do not adequately protect our intellectual property rights, or if the intellectual property rights we are able to obtain are insufficiently broad and exclusive, competitors may be able to erode or negate any competitive advantage we may have, which could harm our business and ability to achieve profitability.
We intend to rely upon a combination of patents, patent applications, confidentiality agreements, trade secret protection and license agreements to protect the intellectual property related to our current or future product candidates and technologies. Any disclosure to or misappropriation by third parties of our confidential proprietary information could enable competitors to quickly duplicate or surpass our technological achievements, thus eroding our competitive position in our market. We, or any current or future partners, collaborators, or licensees, may fail to identify patentable aspects of inventions made in the course of development and commercialization activities before it is too late to obtain patent protection on them. We may be also unable to exclusively license relevant technology and associated intellectual property developed by others. Therefore, we may miss potential opportunities to establish our patent position.
If we are unable to secure additional patent protection or maintain existing or future patent protection with respect to NXP800, NXP900, or any other proprietary products and technology we develop, our business, financial condition, results of operations, and prospects would be materially harmed.
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We currently hold a license to certain intellectual property rights relating to NXP800, including its composition of matter and to other compounds that modulate HSF1 (activate the GCN2 kinase). In addition, we hold a license to certain intellectual property relating to NXP900, including its composition of matter and to other compounds that inhibit the SRC and YES1 kinases.
We have licensed one patent family covering the composition of matter for NXP800, including two issued U.S. patents covering the composition of matter for NXP800, as well as methods for using and making NXP800. Additionally, patents have been issued in major markets, including the U.S., the European Union, and Japan. The statutory expiration for the issued U.S. patents in this family is October 2034, without considering any patent extensions that may or may not be possible.
We have licensed a patent family directed to additional compounds that modulate HSF1. A patent from this family has been granted in the U.S., and has a statutory expiration of April 2036, without considering any patent extensions that may or may not be possible.
We have also licensed a patent family directed to deuterated compounds that modulate HSF1. Any U.S. patent that grants from this family would have a statutory expiration of October 2037, without considering any patent extensions or patent disclaimers that may or may not be possible.
We have licensed one patent family covering the composition of matter for NXP900, which has been granted in the U.S., EU, Japan, China and is pending in the United Kingdom and Canada. The statutory expiration for patents in this patent family is April 2036, without considering any possible patent term extension.
If the scope of our patent protection, whether now or in the future, with respect to NXP800, NXP900 or our future product candidates and technology is not sufficiently broad, we will be unable to prevent others from using our technology or from developing or commercializing technology and products similar or identical to ours or other competing products and technologies. Any failure to obtain or maintain patent protection, through our own patents or through in-licensing, with respect to NXP800, NXP900 and our future product candidates would have a material adverse effect on our business, financial condition, results of operations and prospects.
Even if they are unchallenged, our patent applications, if issued, and any patents we may own or in-license now or in the future, may not provide us with any meaningful protection or prevent competitors from designing around our patent claims to circumvent any patents we may own or in-license in the future by developing similar or alternative technologies or therapeutics in a non-infringing manner. If the patent protection provided by our patent applications or any patents we may pursue with respect to our current or future product candidates is not sufficiently broad to impede competition, our ability to successfully commercialize our current or future product candidates could be negatively affected, which would harm our business.
Additionally, we cannot be certain that the claims in our patent applications covering composition of matter (or other related aspects) of our current or future product candidates or technology will be considered patentable by the USPTO, or by patent offices in foreign countries, or that the claims in any issued patents we may own or in-license in the future will be considered patentable by courts in the United States or foreign countries.
The issuance of a patent does not foreclose challenges to its inventorship, scope, validity or enforceability. Therefore, our owned and in-licensed patents may be challenged in the courts or patent offices in the United States and elsewhere. Such challenges may result in loss of exclusivity or freedom to operate or in patent claims being narrowed, invalidated, or held unenforceable, in whole or in part. Successful patent challenges could limit our ability to stop others from using or commercializing similar or identical technology and products or limit the duration of the patent protection of our technology and products. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such product candidates might expire before or shortly after such product candidates are commercialized. As a result, our owned and in-licensed patents may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.
Moreover, patents or patent applications owned or filed by us, or by our licensors or other collaborators, may be challenged or narrowed by third-party pre-issuance submissions of prior art to the USPTO, or by opposition, derivation, reexamination, inter parties review, post-grant review or interference proceedings. An adverse determination in any such submission, Patent Trial and Appeal Board trial, proceeding or litigation could reduce the scope of, render unenforceable, or invalidate, our patent rights, allow third parties to commercialize our technology or products and compete directly with us, without payment to us, or result in our inability to manufacture or commercialize products without infringing third party patent rights. In addition, if the breadth or strength of protection provided by
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our patents and patent applications is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future product candidates.
If we fail to comply with our obligations in our current license agreements, or in any future agreements under which we may license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our current or future licensors, we could lose license rights that are important to our business.
We are currently party to a license which grants us certain intellectual property rights relating to our lead product candidate, NXP800, as well as other related compounds, and to a license which grants us certain intellectual property rights relating to our second drug candidate, NXP900, as well as other compounds that inhibit the SRC and YES1 kinases. These agreements impose numerous obligations on us to maintain our licensing rights, including development, diligence, payment, commercialization, funding, milestone, royalty, sublicensing, insurance, patent prosecution, enforcement and other obligations. In spite of our efforts, our licensor might conclude that we have materially breached our license agreement and might therefore terminate the license agreement, thereby removing or limiting our ability to develop and commercialize NXP800 or NXP900 (and other compounds covered by the licenses).
Additionally, in the future, we may be party to other license or collaboration agreements with third parties to advance our research or allow commercialization of current or future product candidates. Such future agreements may impose numerous obligations, such as development, diligence, payment, commercialization, funding, milestone, royalty, sublicensing, insurance, patent prosecution, enforcement and other obligations on us and may require us to meet development timelines, or to exercise commercially reasonable efforts to develop and commercialize licensed products, in order to maintain the licenses. In spite of our efforts, our future licensors might conclude that we have materially breached our future license agreements and might terminate the license agreements, thereby removing or limiting our ability to develop and commercialize products and technologies covered by these license agreements.
Any termination of these current or future licenses, or failure of the underlying patents to provide the intended exclusivity, could result in the loss of significant rights and could harm our ability to commercialize our current or future product candidates, and competitors or other third parties would have the freedom to seek regulatory approval of, and to market, products identical to ours and we may be required to cease our development and commercialization of certain of our current or future product candidates. Any of the foregoing could have a material adverse effect on our competitive position, business, financial conditions, results of operations, and prospects.
If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed.
In addition to the protection afforded by patents we may own or in-license in the future, we seek to rely on trade secret protection, confidentiality agreements, and license agreements to protect proprietary know-how that is not patentable, processes for which patents are difficult to enforce, and any other elements of our product discovery and development processes that involve proprietary know-how, information, or technology that is not covered by patents. Although we require all of our employees, consultants, advisors and any third parties who have access to our proprietary know-how, information, or technology to enter into confidentiality agreements, trade secrets can be difficult to protect and we have limited control over the protection of trade secrets used by our collaborators and suppliers.
If we are unable to prevent unauthorized material disclosure of our intellectual property to third parties, we will not be able to establish or maintain a competitive advantage in our market, which could materially adversely affect our business, financial condition, results of operations and future prospects.
Third-party claims of intellectual property infringement, misappropriation or other violations may be costly and time consuming and may prevent or delay our product discovery and development efforts.
The intellectual property landscape around precision medicine is crowded, and third parties may initiate legal proceedings alleging that we are infringing, misappropriating, or otherwise violating their intellectual property rights; the outcome of which would be uncertain and could have a material adverse effect on the success of our business. We or any of our future licensors or strategic partners may be party to, exposed to, or threatened with, future adversarial proceedings or litigation by third parties having patent or other intellectual property rights alleging that our current or future product candidates and/or proprietary technologies infringe, misappropriate or otherwise violate their intellectual property rights. Thus, because of the large number of patents issued and patent applications filed in our fields, there may be a risk that third parties may allege they have patent rights encompassing our current or future product candidates, technologies or methods.
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Third parties may assert that we are employing their proprietary technology without authorization. In addition, because some patent applications in the United States may be maintained in secrecy until the patents are issued, patent applications in the United States and many foreign jurisdictions are typically not published until 18 months after filing, and publications in the scientific literature often lag behind actual discoveries, we cannot be certain that others have not filed patent applications covering our current or future product candidates or technology. If any such patent applications issue as patents, and if such patents have priority over patent applications or patents we own or in-license, we may be required to obtain rights to such patents owned by third parties which may not be available on commercially reasonable terms or at all, or may only be available on a non-exclusive basis.
In the event of a successful claim of infringement, misappropriation or other violation against us, we may have to pay substantial damages, including treble damages and attorneys’ fees for willful infringement, obtain one or more licenses from third parties, pay royalties or redesign our infringing products, which may be impossible or require substantial time and monetary expenditure.
Changes to patent law in the United States and in foreign jurisdictions could diminish the value of patents in general, thereby impairing our ability to protect our products.
Our success is heavily dependent on intellectual property, particularly patents. Recent U.S. Supreme Court rulings have narrowed the scope of patent protection available in certain circumstances and weakened the rights of patent owners in certain situations. In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of patents, once obtained. Laws and regulations governing patents could further change in unpredictable ways that could weaken our ability to obtain new patents or to enforce patents that we own or in-license now, or that we might obtain or in-license in the future.
We may be subject to claims challenging the inventorship or ownership of our intellectual property, including any patents we may own or in-license currently or in the future.
We may be subject to claims that former employees, collaborators or other third parties have an interest in any patents we may own or in-license currently or in the future, trade secrets, or other intellectual property as an inventor or co-inventor. Litigation may be necessary to defend against these and other claims challenging inventorship of any patents we may own or in-license in the future, trade secrets or other intellectual property, which may require substantial time and monetary expenditure.
We may be subject to claims that we or our employees, consultants or independent contractors have wrongfully used or disclosed confidential information or alleged trade secrets of third parties or competitors or breached non-competition or non-solicitation agreements with our competitors.
We may be subject to claims that we or our employees, consultants or independent contractors have inadvertently or otherwise used or disclosed confidential information or trade secrets of third parties or competitors or our employees’ former employers or our consultants’ or contractors’ current or former clients or customers. Litigation or arbitration may be necessary to defend against these claims, which may require substantial time and monetary expenditure.
If we do not obtain patent term extension and data exclusivity for any of our current or future product candidates we may develop, our business may be materially harmed.
Depending upon the timing, duration and specifics of any FDA marketing approval of any of our current or future product candidates we may develop, one or more U.S. patents we may own or in-license in the future may be eligible for limited patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Act. If we are unable to obtain patent term extension or the term of any such extension is shorter than what we request, our competitors may obtain approval of competing products following expiration of any patents that issue from our patent applications, and our business, financial condition, results of operations, and prospects could be materially harmed.
If our trademarks and trade names are not adequately protected, we may not be able to build name recognition in our marks of interest and our business may be adversely affected.
Our trademarks or trade names may be challenged, infringed, diluted, circumvented or declared generic or determined to be infringing on other marks. We intend to rely on both registration and common law protection for our trademarks. We may not be able to protect
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our rights to these trademarks and trade names or may be forced to stop using these names, which we need for name recognition by potential partners or customers in our markets of interest. If we are unable to obtain a registered trademark or establish name recognition based on our trademarks and trade names, we may not be able to compete effectively and our business may be adversely affected.
Risks Related to our Reliance on Third Parties
We plan to rely on third parties to conduct our preclinical studies and clinical trials. If these third parties do not properly and successfully carry out their contractual duties or meet expected deadlines, we may not be able to obtain regulatory approval of or commercialize our current or future product candidates.
We plan to utilize and depend upon independent investigators and collaborators, such as medical institutions, CROs, CMOs, and strategic partners to conduct and support our preclinical studies and clinical trials under agreements with us. We rely upon, and plan to continue to rely upon, such third-party entities to execute our clinical trials and preclinical studies and to monitor and manage data produced by and relating to those studies and trials. However, in the future we may not be able to establish arrangements with CROs when needed or on terms that are acceptable to us, or at all, which could negatively affect our development efforts with respect to our drug candidates and materially harm our business, operations and prospects. As a result of the use of third-party contractors, we will have only limited control over certain aspects of their activities. Nevertheless, we are responsible for ensuring that each of our studies, including each of our clinical trials, is conducted in accordance with the applicable protocol, legal and regulatory requirements as well as scientific standards, and our reliance on any third-party entity will not relieve us of our regulatory responsibilities.
Based on our present expectations, we and our third-party contractors will be required to comply with GCP regulations for the clinical development of all of our drug candidates. If we or any of these third parties fail to comply with applicable GLP or GCP regulations, the clinical data generated in our preclinical and clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications, which we may not have sufficient cash or other resources to support and which would delay our ability to generate revenue from future sales of such drug candidate. Any agreements governing our relationships with CROs or other contractors with whom we currently engage or may engage in the future may provide those outside contractors with certain rights to terminate a clinical trial under specified circumstances. If such an outside contractor terminates its relationship with us during the performance of a clinical trial, we would be forced to seek an engagement with a substitute contractor, which we may not be able to do on a timely basis or on commercially reasonable terms, if at all, and the applicable clinical trial would experience delays or may not be completed.
Large-scale clinical trials require significant additional financial and management resources and reliance on third-party clinical investigators, CROs, and consultants, which may cause us to encounter delays that are outside of our control. We may be unable to identify and contract with sufficient investigators, CROs, or consultants on a timely basis, if at all.
If these third parties do not successfully carry out their contractual duties or obligations or meet expected deadlines, if they need to be replaced, or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our clinical protocols, legal and regulatory requirements or for other reasons, our preclinical or clinical trials may be extended, delayed or terminated and we may not be able to complete development of, obtain regulatory approval for, or successfully commercialize, our current or future product candidates. In addition, we will be unable to control whether or not they devote sufficient time and resources to our preclinical and clinical programs. These outside contractors may not assign as great a priority to our programs or pursue them as diligently as we would if we were undertaking such programs ourselves. As a result, our operations and the commercial prospects for the effected drug candidates would be harmed, our costs could increase and our ability to generate revenues could be delayed. These contractors may also have relationships with other commercial entities, some of whom may compete with us. If our contractors assist our competitors to our detriment, our competitive position would be harmed.
If our relationships with any third parties conducting our studies are terminated, we may be unable to enter into arrangements with alternative third parties on commercially reasonable terms, or at all. Switching or adding third parties to conduct our studies involves substantial cost and requires extensive management time and focus. In addition, there is a natural transition period when a new third party commences work. As a result, delays occur, which can materially impact our ability to meet our desired clinical development timelines. Although we carefully manage our relationships with third parties conducting our studies, we cannot assure you that we will not encounter similar challenges or delays in the future or that these delays or challenges will not have a material and adverse effect on our business, financial condition and results of operations.
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We rely, and expect to continue to rely, on the third-party manufacturers to manufacture our current or future product candidates. Reliance on third parties increases the risk that we will not have sufficient quantities of our products or such quantities at an acceptable quality and cost, which could delay, prevent or impair our development or commercialization efforts.
We do not currently own any facility that may be used as our clinical-scale manufacturing and processing facility and must rely on outside vendors to manufacture our current or future product candidates. We rely on a single CMO to make the NXP800 drug substance and finished drug product, each performed at a different manufacturing facility. We rely on a single CMO to manufacture NXP900 drug substance and another single CMO to manufacture NXP900 drug product. There is no assurance that we will be able to retain these relationships, and if we are unable to maintain these relationships, we could experience delays in our development efforts. There is no assurance that our CMOs will be successful in manufacturing NXP800 and/or NXP900 drug substance or product. If NXP800, NXP900 or any other drug candidate we may develop or acquire in the future receives regulatory approval, we will likely rely on one or more CMOs to manufacture the commercial supply of such drugs.
Our anticipated reliance on a limited number of third-party manufacturers exposes us to a number of risks, including:
| ● | due to the limited number of potential manufacturers, and because the FDA requires inspection of any manufacturers’ cGMP compliance as part of our marketing application, we may be unable to identify manufacturers on acceptable terms, if at all; |
| ● | a new manufacturer would have to be educated in and develop substantially equivalent processes for, the production of our current or future product candidates; |
| ● | our third-party manufacturers might be unable to timely manufacture our current or future product candidates or produce the quantity and quality required to meet our clinical and commercial needs due to a variety of potential reasons including failure to achieve drug substance or drug product specifications, batch to batch inconsistencies, site or equipment contaminations, failure to scale up as needed in a cost-efficient manner, failed regulatory inspections, competition for production capacity and availability from other customers; |
| ● | we may not own, or may have to share, the intellectual property rights to any improvements made by our third-party manufacturers in the manufacturing process for our current or future product candidates; |
| ● | our third-party manufacturers could breach or terminate their agreements with us; |
| ● | our third-party manufacturers might be unable to formulate and manufacture our drugs in the volume and of the quality required to meet our clinical and commercial needs, if any; |
| ● | our third-party manufacturers may not perform as contractually agreed or may not remain in the contract manufacturing business for the time required to supply our clinical trials or to successfully produce, store and distribute our products; |
| ● | drug manufacturers are subject to ongoing periodic unannounced inspection by the FDA and some state agencies in the United States, as well as foreign regulatory authorities, to ensure strict compliance with cGMP regulations and other regulatory requirements; and |
| ● | raw materials and components used in the manufacturing process, particularly those for which we have no other source or supplier, may not be available or may not be suitable or acceptable for use due to material or component defects. |
Each of these risks could delay or prevent the completion of our preclinical or clinical trials or the approval of any of our current or future product candidates by the FDA or another foreign regulatory authority, result in higher costs or adversely impact commercialization of our current or future product candidates.
Although our agreements with our CMOs require them to perform according to certain cGMP requirements such as those relating to quality control, quality assurance and qualified personnel, we cannot control the conduct of our CMOs to implement and maintain these standards. If any of our CMOs cannot successfully manufacture material that conforms to our specifications and the regulatory
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requirements of the FDA, EMA or other comparable foreign authorities, we could be prevented from obtaining regulatory approval for our drug candidates unless and until we engage a substitute CMO that can comply with such requirements, which we may not be able to do. Any such failure by any of our CMOs would significantly impact our ability to develop, obtain regulatory approval for or market our drug candidates, if approved.
If our third-party manufacturers use hazardous and biological materials in a manner that causes injury or violates applicable law, we may be liable for damages.
Although we believe that our manufacturers’ procedures for using, handling, storing, and disposing of hazardous and biological materials comply with legally prescribed standards, we cannot completely eliminate the risk of contamination or injury. In the event of an accident, local, city, state or federal authorities may curtail the use of these materials and interrupt our business operations. Further, we could be held liable for damages or penalized with fines, and the liability could exceed our resources. We do not have any insurance for liabilities arising from medical or hazardous materials.
Risks Related to Managing Growth and Employee Matters
We are highly dependent on our key personnel and anticipate hiring new key personnel. If we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy.
Our ability to compete in the highly competitive biotechnology and pharmaceutical industries depends upon our ability to attract and retain highly qualified managerial, scientific and medical personnel. We are highly dependent on our management, scientific and medical personnel, including our Chairman, Chief Executive Officer and President, our Chief Scientific and Business Officer and our Chief Development and Operations Officer. While we expect to engage in an orderly transition process as we integrate newly appointed officers and managers, we face a variety of risks and uncertainties relating to management transition, including diversion of management attention from business concerns, failure to retain other key personnel or loss of institutional knowledge.
We will need to grow the size of our organization, and we may experience difficulties in managing this growth.
As of November 1, 2024, we had 13 full-time employees. We also contract for various services through consulting and vendor agreements. We intend to hire new employees to conduct our research and development activities in the future. Any delay in hiring such new employees could result in delays in our research and development activities and would harm our business. As our development and commercialization plans and strategies develop, and as we transition into operating as a public company, we expect to need additional managerial, operational, sales, marketing, financial and other personnel, as well as additional facilities to expand our operations.
If we are not able to effectively expand our organization by hiring new employees and expanding our groups of consultants and contractors, or we are not able to effectively build out new facilities to accommodate this expansion, we may not be able to successfully implement the tasks necessary to further develop and commercialize our current or future product candidates and, accordingly, may not achieve our research, development and commercialization goals.
We will incur significant increased costs as a result of operating as a public company, and our management will be required to devote substantial time to compliance activities and initiatives.
As a public company, we will incur significant legal, accounting, and other expenses that we did not incur as a private company. We are now subject to the reporting requirements of the Securities Exchange Act of 1934, as amended, which requires, among other things, that we file with the SEC, annual, quarterly, and current reports with respect to our business and financial condition. In addition, the Sarbanes-Oxley Act of 2002 (“SOX”), as well as rules subsequently adopted by the SEC and the Nasdaq Capital Market to implement provisions of SOX, impose significant requirements on public companies, including requiring establishment and maintenance of effective disclosure and financial controls and changes in corporate governance practices.
Moreover, these rules and regulations will increase our legal and financial compliance costs and make some activities more time-consuming and costly. For example, these rules and regulations make it more difficult and more expensive for us to obtain director and officer liability insurance, and we may be required to accept reduced policy limits and coverage or incur substantially higher costs to obtain the same or similar coverage. As a result, it may be more difficult for us to attract and retain qualified persons to serve on our Board of Directors, our Board committees or as executive officers.
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SOX requires, among other things, that we maintain effective internal control over financial reporting and disclosure controls and procedures. As a result, we are required to periodically perform an evaluation of our internal control over financial reporting to allow management to report on the effectiveness of those controls, as required by Section 404 of SOX. These efforts to comply with Section 404 will require the commitment of significant financial and managerial resources. While we anticipate maintaining the integrity of our internal control over financial reporting and all other aspects of Section 404, we cannot be certain that a material weakness will not be identified when we test the effectiveness of our control systems in the future. If a material weakness is identified, we could be subject to sanctions or investigations by the SEC or other regulatory authorities, which would require additional financial and management resources, costly litigation or a loss of public confidence in our internal control, which could have an adverse effect on the market price of our stock.
Our business and operations would suffer in the event of computer system failures, cyber-attacks, or deficiencies in our or third parties’ cybersecurity.
We are increasingly dependent upon information technology systems, infrastructure, and data to operate our business. In the ordinary course of business, we may collect, store, and transmit confidential information, including, but not limited to, information related to our intellectual property and proprietary business information, personal information, and other confidential information. We have outsourced elements of our operations to third party vendors, who each have access to our confidential information, which increases our disclosure risk. Although we have implemented internal security and business continuity measures, our information technology and other internal infrastructure systems may breakdown, incur damage or be interrupted by system malfunctions, natural disasters, terrorism, war, or telecommunication and electrical failures, as well as by inadvertent or intentional security breaches by our employees, contractors, consultants, business partners, and/or other third parties, or from cyber-attacks by malicious third parties, each of which could compromise our system infrastructure or lead to the loss, destruction, alteration, disclosure, or dissemination of, or damage or unauthorized access to, our data or other assets. Such a security breach may cause loss, damage, or disclosure of proprietary or confidential information, which could in turn result in significant legal and financial exposure and reputational damage that could adversely affect our business. Furthermore, the loss or corruption of clinical trial data from future clinical trials may result in delays in our regulatory approval efforts and could significantly increase our costs to recover or reproduce the data.
The costs related to significant security breaches or disruptions could be material and our insurance policies may not be adequate to compensate us for the potential losses arising from any such security breach. In addition, such insurance may not be available to us on economically reasonable terms, if at all, may not cover all claims made against us, and may have high deductibles. Furthermore, if the information technology systems of our third-party vendors and other contractors and consultants become subject to disruptions or security breaches, we may have insufficient recourse against such third parties and we may have to expend significant resources to mitigate the impact of such an event, and to develop and implement protections to prevent future events of this nature from occurring.
Risks Related to Commercial Activities
If any of our current or future product candidates do not achieve broad market acceptance among physicians, patients, healthcare payors and the medical community, the revenues from any such current or future product candidate may be limited.
The use of precision medicines as a potential cancer treatment is a recent development and may not become broadly accepted by physicians, patients, hospitals, cancer treatment centers, and others in the medical community. We cannot predict whether physicians, patients, hospitals, cancer treatment centers, and government agencies or third-party payors will determine that our product is safe, therapeutically effective, and cost effective as compared with competing treatments. If our current or potential future product candidates do not achieve an adequate level of market acceptance, we may not generate significant product revenues and may not become profitable. Factors influencing acceptance of our current or future product candidates in the market, include: the clinical indications for which our product candidates are licensed; whether our product candidates are viewed as a safe and effective treatment; our ability to demonstrate our product’s advantages, including cost advantages, over alternative treatments; the prevalence and severity of any side effects of our products and of other precision medicines; product labeling or product insert requirements of the FDA or other regulatory authorities and limitations or warnings contained in the labeling; the timing of market introduction of our product candidates and competitive products; patient willingness to pay out-of-pocket in the absence of coverage by third-party payors and government authorities; and the effectiveness of our sales and marketing efforts.
If our current or future product candidates are licensed but fail to achieve market acceptance among physicians, patients, hospitals, cancer treatment centers or others in the medical community, we will not be able to generate significant revenue. In addition, although
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our current or future product candidates may differ in certain ways from other precision medicine approaches, serious adverse events or deaths in other preclinical or clinical trials involving precision medicines, even if not ultimately attributable to our current or future products or product candidates, could result in increased government regulation, unfavorable public perception and publicity, potential regulatory delays in the testing or licensing of our current or future product candidates, stricter labeling requirements for those product candidates that are licensed, and a decrease in demand for any such product candidates.
If product liability lawsuits are brought against us, we may incur substantial liabilities and may be required to limit commercialization of our current or future product candidates.
We face an inherent risk of costly and time-consuming product liability lawsuits as a result of the planned clinical testing of our current or future product candidates and will face an even greater risk if we commercialize any products. For example, we may be sued if our current or future product candidates cause or are perceived to cause injury or are found to be otherwise unsuitable during clinical testing, manufacturing, marketing or sale. If we cannot successfully defend ourselves against product liability claims, we may incur substantial liabilities or be required to limit commercialization of our current or future product candidates. Failure to obtain or retain sufficient product liability insurance at an acceptable cost may prevent or inhibit the commercialization of products we may develop. Although we have clinical trial insurance, our insurance policies have various exclusions, and we may be subject to a claim for which we have no coverage. We may have to pay any amounts awarded by a court or negotiated in a settlement that are not covered by or which exceed our insurance coverage, and we may not have sufficient capital to pay such amounts.
Risks Related to Ownership of our Common Stock
We do not know whether an active, liquid and orderly trading market will develop for our Common Stock or what the market price of our Common Stock will be and, as a result, it may be difficult for you to sell your shares of our Common Stock.
Prior to the pricing of our initial public offering on February 4, 2022, there was no public trading market for shares of our Common Stock and after our IPO the trading price of our Common Stock has been volatile. Although our Common Stock is listed on the Nasdaq Capital Market, an active trading market for our shares is still developing and may not be sustained in the future. The lack of an active market for our Common Stock creates volatility in the price of the stock and may impair investors’ ability to sell their shares at the time they wish to sell them or at a price that they consider reasonable and may reduce the fair market value of their shares. Further, an inactive market may impair our ability to raise capital by selling shares of our Common Stock and to enter into strategic partnerships or acquire companies or products using our shares of common stock as consideration.
Our growth is subject to economic and political conditions.
Our business is affected by global and local economic and political conditions as well as the state of the financial markets, inflation, recession, financial liquidity, currency volatility, growth, and policy initiatives. There can be no assurance that global economic conditions and financial markets will not worsen and that we will not experience any adverse effects that may be material to our consolidated cash flows, results of operations, financial position or our ability to access capital, such as the adverse effects resulting from a prolonged shutdown in government operations both in the United States and internationally. Political changes, including war or other conflicts, some of which may be disruptive, could interfere with our supply chain, our customers and all of our activities in a particular location.
We do not intend to pay dividends on our Common Stock in the foreseeable future, so any returns will be limited to the value of our stock, which may be volatile.
We plan to retain future earnings for the development, operation, and expansion of our business and do not anticipate declaring or paying any cash dividends for the foreseeable future. Any return to stockholders will be limited to the appreciation of their stock, which may never occur. Further, the trading price of our common stock is likely to be highly volatile and may be subject to wide fluctuations in response to various factors, some of which are beyond our control. Broad market and industry factors may negatively affect the market price of our common stock, regardless of our actual operating performance.
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If equity research analysts do not publish research or reports about our business or if they publish negative evaluations of or downgrade our Common Stock, the price of our Common Stock could decline.
The trading market for our Common Stock relies in part on the research and reports that equity research analysts publish about us or our business. We do not control these analysts. We may never obtain research coverage by industry or financial analysts. If no or few analysts publish research reports on the Company or if analysts publish negative research reports about the Company, our stock price may significantly decline.
Raising additional capital may cause dilution to our existing stockholders, restrict our operations, or require us to relinquish rights to our current or future technologies or product candidates.
We may seek additional capital through a combination of public and private equity offerings, ATM facility, debt financings, strategic partnerships and alliances and licensing arrangements. Any equity or equity-related financing may dilute our stockholders may subject us to restrictive covenants and interest costs. If we obtain funding through a strategic collaboration or licensing arrangement, we may be required to relinquish our rights to our current product candidates or any future product candidates that we may develop.
Additional fundraising efforts may divert our management from their day-to-day activities, which may adversely affect our operations. If we are unable to raise additional capital as needed or on acceptable terms, we may be required to delay or discontinue any research, development or commercialization programs and may be unable to expand our operations or otherwise capitalize on our business opportunities. Further, we may be required to seek collaborators for potential product candidates earlier, or on less favorable terms, than might otherwise be desired, or to relinquish or license our rights to potential product candidates in markets where we otherwise would seek to pursue development or commercialization. Any of the above events could significantly harm our business, prospects, financial condition and results of operations and cause the price of our common stock to decline.
Our principal stockholders and management own a significant percentage of our stock and will be able to exert significant influence over matters subject to stockholder approval.
As of November 1, 2024, our executive officers, directors, and 5% stockholders beneficially owned approximately 56.9% of our voting stock and anticipate that the same group will hold a significant portion of our outstanding voting stock for the foreseeable future. These stockholders will have the ability to influence us through their ownership position. This may prevent or discourage unsolicited acquisition proposals or offers for our common stock.
Our failure to meet the continuing listing requirements of the NASDAQ Capital Market could result in a de-listing of our securities.
If we fail to satisfy the continuing listing requirements of NASDAQ, such as the corporate governance, stockholders’ equity or minimum closing bid price requirements, NASDAQ may take steps to delist our common stock. Such a delisting would likely have a negative effect on the price of our common stock and would impair our stockholders’ ability to sell or purchase our common stock. In the event of a delisting, we would likely take actions to restore our compliance with NASDAQ’s listing requirements, but we can provide no assurance that any such action taken by us would allow our common stock to become listed again, stabilize the market price or improve the liquidity of our securities, prevent our common stock from dropping below the NASDAQ minimum bid price requirement or prevent future non-compliance with NASDAQ’s listing requirements.
We are an emerging growth company and a smaller reporting company, and we cannot be certain if the reduced reporting requirements applicable to emerging growth companies and smaller reporting companies will make our common stock less attractive to investors.
We are an emerging growth company, as defined in the JOBS Act. For as long as we continue to be an emerging growth company, we may take advantage of exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies, including: exemption from the auditor attestation requirements of Section 404 of SOX, as amended; being permitted to provide only two years of our audited financial statements and correspondingly reduced “Management’s Discussion and Analysis of Financial Condition and Results of Operations”; exemption from any Public Company Accounting Oversight Board requirement regarding audit firm rotation or an auditor report supplement providing additional information about the audit and financial
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statements; reduced disclosure obligations regarding executive compensation; and exemption from the nonbinding advisory votes on executive compensation and stockholder approval of any golden parachute payments not previously approved.
We have elected to take advantage of certain of the reduced reporting obligations. We cannot predict whether investors will find our common stock less attractive if we rely on these exemptions. If some investors find our common stock less attractive as a result, there may be a less active trading market for our common stock and our stock price may be reduced or more volatile.
Provisions in our certificate of incorporation, our bylaws, and Delaware law may discourage, delay, or prevent a change in control of our Company or changes in our management and, as a result, depress the trading price of our stock.
Provisions of our certificate of incorporation, our bylaws and Delaware law may deter unsolicited takeovers and/or delay or prevent a change in control of our Company, including transactions in which our stockholders might otherwise receive a premium for their shares.
In addition, the Delaware General Corporation Law prohibits a publicly held Delaware corporation from engaging in a business combination with an interested stockholder, defined as a person who owns, or within the last three years has owned, 15% of our voting stock, for a period of three years after the date of the transaction in which the person became an interested stockholder, unless the business combination is approved in a prescribed manner.
The foregoing provisions and anti-takeover measures may limit the price that investors might be willing to pay in the future for shares of our Common Stock and may deter potential acquirers of our Company.
Item 2.Unregistered Sales of Equity Securities and Use of Proceeds.
During the period covered by this report, we have not issued any unregistered securities.
Use of Proceeds from Sales of Registered Securities
On February 4, 2022, our registration statement on Form S-1 (File No. 333-260099) and our registration statement on Form S-1MEF (File No. 333-262512) (collectively, the “Registration Statements”) were declared effective by the SEC. Pursuant to such Registration Statements, we sold an aggregate of 3,200,000 shares of our common stock at a price of $5.00 per share for aggregate net cash proceeds of approximately $13.1 million, which amount is net of $1.12 million in underwriter’s discounts, and commissions, and $1.8 million of other expenses incurred in connection with the offering. We closed the offering on February 8, 2022.
On August 24, 2022, our registration statement on Form S-1 (File No. 333-266857), which was filed on August 15, 2022, was declared effective by the SEC. Pursuant to such registration statement, we sold an aggregate of 1,015,598 shares of our common stock at a price of $8.25, and 909,091 of pre-funded warrants to purchase one share of common stock at $8.25 per share for aggregate net cash proceeds of approximately $14.3 million, which amount is net of $1.4 million in placement agent fees, and $0.3 million of other expenses incurred in connection with the offering. We closed the private placement on July 29, 2022. In this offering, we also issued to the investors who participated in the offering preferred investment options to purchase up to an aggregate of 1,924,689 shares of common stock, at an exercise price of $9.65 per share with a term of three and one-half years from the date of issuance.
We intend to use the net proceeds of these offerings to fund the Phase 1 development of NXP800 and NXP900, to continue development and sponsored research related to our current product candidates or any future product candidate, hiring of additional personnel, capital expenditures, costs of operating as a public company and other general corporate purposes.
There has been no material change in the expected use of the net proceeds from our initial public offering as described in our final prospectuses filed with the SEC on February 8, 2022 and August 15, 2022, respectively, pursuant to Rule 424(b) under the Securities Act. We invested the funds received in an interest-bearing money market account.
Item 3.Defaults Upon Senior Securities.
None.
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Item 6. Exhibits
Exhibit No. |
| Description |
31.1* | ||
31.2* | ||
32.1** | ||
32.2** | ||
101* | The following financial information from the Company’s quarterly report on Form 10-Q for the period ended September 30, 2024, formatted in Inline Extensible Business Reporting Language (XBRL): (i) the Condensed Balance Sheets, (ii) the Condensed Statements of Operations, (iii) the Condensed Statement of Stockholders’ Equity, (iv) the Condensed Statements of Cash Flows, and (v) Notes to the Condensed Financial Statements. | |
104* | Cover Page Interactive Data File (formatted as Inline XBRL and contained in Exhibit 101). |
* Filed herewith.
** Furnished herewith.
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Signatures
Pursuant to the requirements of the Securities Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized, in the City of Fort Lee, State of New Jersey, on this 5th day of November 2024.
| Nuvectis Pharma, Inc. | ||
|
| ||
| By: | /s/ Ron Bentsur | |
|
| Name: | Ron Bentsur |
|
| Title: | Chairman, Chief Executive Officer and President |
By: | /s/ Michael J Carson | ||
| Name: | Michael J Carson | |
| Title: | Vice President of Finance (Principal Financial and Accounting Officer) | |
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