附录99.1
ocugen提供业务更新与2024年第三季度财报解读
今天早上8:30美东时间举行电话会议和网络研讨会
•季末后，进行了3000万美元的债务融资
•OCU400第3期验明灯视网膜色素性视网膜症临床试验顺利，预计2025年上半年完成招募
•OCU410目前处于第1/2 ArMaDa临床试验第2期进行中
•OCU410St GARDian临床试验的数据和安全监控委员会(DSMB)批准了第1/2临床试验第2期的招募
•有关OCU410、OCU410St和OCU400的第1/2期临床试验新数据将在即将举行的临床展示中发表
宾夕法尼亚州马尔文，2024年11月8日（全球新闻社） - 生物技术公司ocugen, Inc.（ocugen或公司）（纳斯达克: OCGN）致力于发现、开发和商业化新型基因和细胞疗法、生物制品和疫苗。今天报告了2024年第三季度财务业绩，并宣布了一般业务更新。
“我对我们基因治疗项目的进展和2024年第三季度所达到的临床和监管里程碑感到非常鼓舞，包括将OCU400第3期liMeLiGht临床试验扩展至加拿大，” ocugen公司的主席、首席执行官兼共同创始人Shankar Musunuri博士表示。 “通过最近的股本和债务融资，我们有足够现金储备，可继续支持我们强大的眼科医疗领域项目，并将我们的现金财务马拉松延长至2026年第1季度。”
随著OCU400在第3期临床发展中前进，为了早期至晚期RP的成年患者扩展使用计划（EAP）使得可以触及更广泛的RP患者人口 - 美国、加拿大和欧洲合计30万人。此外，将加拿大患者纳入OCU400第3期liMeliGht试验可能会促进与美国和欧洲更广泛的商业化。这些成就和持续的试验招募使公司更接近为RP患者提供潜在的一次性治疗。
OCU410第1/2期ArMaDa临床试验第2期正在进行，将评估OCU410在较大群患者中的安全性和效力，这些患者将随机分入两组治疗（中、高剂量）或对照组。 OCU410正在开发用于地理性萎缩（GA），是干性老年性黄斑变性（dAMD）的晚期阶段。 GA影响约200-300万美国和欧盟人口。目前美国食品药物管理局批准的治疗仅涉及补体系统，每年约需要6-12次眼内注射，而OCU410涉及与dAMD发病机制相关的所有4条通路，通过单次眼下注射进行。目前在欧盟尚无批准用于治疗GA的产品。
在2024年第三季度的一系列会议中，ocugen有机会向重要投资者和行业决策者提供三个临床阶段的基因调节治疗更新，如由再生医学联盟主办的Cell & Gene Meeting on the Mesa等会议。
“继续向关键利益相关者介绍我们基因不特异性调节基因治疗平台的差异化作用机制至关重要，” Musunuri博士表示。“与其他用于治疗视网膜疾病的产品候选物不同，我们的方法利用能够重置功能网络的主要基因调节因子，而不是针对单一突变，并恢复视网膜的整体健康。我们的数据继续支持通过一次性疗法治疗多个疾病突变的潜力。”
虽然基因治疗仍然是公司的主要焦点，但ocugen继续寻求整个投资组合的资金来确保其创新平台达到需要的人们手中。
2024年11月12日将举行一项临床展示，提供ocugen正在进行的基因治疗试验的更新，并将包括OCU410 ArMaDa初步的安全性和有效性数据，以及OCU410St GARDian关于stargardt疾病的第一/二期临床试验数据，以及来自OCU400第一/二期临床试验的RP和LCA数据更新。
眼科基因治疗—领先业界
•OCU400 — 参与Phase 3 liMeliGht临床试验的招募仍在进行中，加拿大最多5个地点已获Health Canada批准。FDA批准了OCU400机制治疗可能受益于RP的成人患者的应急使用计划。
•OCU410 — Actively recruiting patients in Phase 2 of the Phase 1/2 ArMaDa clinical trial. Preliminary safety and efficacy update on OCU410 Phase 1/2 ArMaDa clinical trial will be shared at upcoming clinical showcase.
•OCU410ST — DSMb approved proceeding to Phase 2 of the Phase 1/2 GARDian clinical trial. Preliminary safety and efficacy update will be shared at upcoming clinical showcase.
Ophthalmic Biologic Product
•OCU200 – FDA cleared the investigational new drug application for the Phase 1 clinical trial evaluating OCU200. The Company is planning to initiate the OCU200 Phase I clinical trial this quarter.
2024年第三季度财务结果
•With the recent $3000万 debt financing and $3500万 equity financing in the third quarter, the cash runway now extends into 1Q2026.
•截至2024年9月30日，公司的现金和受限现金总额为3900万美元，相比之下，截至2023年12月31日为3950万美元。
•截至2024年9月30日为三个月的总营业费用为1440万美元，其中包括810万美元的研发支出和630万美元的管理和行政费用。相比之下，截至2023年9月30日为三个月的总营业费用为1610万美元，其中包括700万美元的研发支出和910万美元的管理和行政费用。
会议看涨和网路转播详情
ocugen已排定于Et时间上午8:30举行电话会议和网路直播，以讨论财务业绩和最近的业务重点。Ocugen的高级管理团队将主持此电话会议，将对所有听众开放。之后还将有一个环节来回答提前准备的问题。
参与者可利用以下详细信息参加电话会议或网路直播：
拨打号码：(800) 715-9871（美国），(646) 307-1963（国际）
会议ID：9923172
网路直播：可于ocugen投资者网站活动部分中找到
A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.
对前瞻性陈述的谨慎提醒
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, expected cash runway into the first quarter of 2026, the ability to initiate new clinical programs; statements
regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
联系人：
Tiffany Hamilton
通信主管
Tiffany.Hamilton@ocugen.com
ocugen, INC.
合并资产负债表
（以千为单位）
(未经查核)
| 2024年9月30日 | 2023年12月31日 | ||||||||||
| 资产 | |||||||||||
| 流动资产合计 | |||||||||||
| 现金 | $ | 38,696 | $ | 39,462 | |||||||
| 预付费用及其他流动资产 | 1,977 | 3,509 | |||||||||
| 全部流动资产 | 40,673 | 42,971 | |||||||||
| 物业及设备，扣除折旧后净值 | 17,130 | 17,290 | |||||||||
| 限制性现金 | 305 | — | |||||||||
| 其他资产 | 3,828 | 4,286 | |||||||||
| 资产总额 | $ | 61,936 | $ | 64,547 | |||||||
| 负债及股东权益 | |||||||||||
| 流动负债 | |||||||||||
| 应付账款 | $ | 1,494 | $ | 3,172 | |||||||
| 应计费用及其他流动负债 | 12,475 | 13,343 | |||||||||
| 营运租赁负债 | 477 | 574 | |||||||||
| 长期负债的当前部分 | 1,316 | — | |||||||||
| 流动负债合计 | 15,762 | 17,089 | |||||||||
| 非流动负债 | |||||||||||
| 营运租赁负债扣除当期部分 | 3,419 | 3,567 | |||||||||
| 长期负债，净额 | 1,571 | 2,800 | |||||||||
| 其他非流动负债 | 554 | 527 | |||||||||
| 非流动负债总额 | 5,544 | 6,894 | |||||||||
| 总负债 | 21,306 | 23,983 | |||||||||
| 股东权益总额 | 40,630 | 40,564 | |||||||||
| 负债总额及股东权益合计 | $ | 61,936 | $ | 64,547 | |||||||
ocugen, INC.
综合营运状况表
(以千为单位，股票和每股金额除外)
(未经查核)
| 截至9月30日的三个月内， | 截至九月三十日止九个月， | ||||||||||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||||||||||
| 合作安排营业收入 | $ | 1,136 | $ | 3,699 | $ | 3,291 | $ | 4,627 | |||||||||||||||
| 营业总收入 | 1,136 | 3,699 | 3,291 | 4,627 | |||||||||||||||||||
| 营业费用 | |||||||||||||||||||||||
| 研发费用 | 8,108 | 7,048 | 23,836 | 31,794 | |||||||||||||||||||
| 总务与行政 | 6,280 | 9,082 | 20,372 | 26,839 | |||||||||||||||||||
| 营业费用总计 | 14,388 | 16,130 | 44,208 | 58,633 | |||||||||||||||||||
| 营运亏损 | (13,252) | (12,431) | (40,917) | (54,006) | |||||||||||||||||||
| 其他收入（费用），净额 | 282 | 714 | 743 | 1,898 | |||||||||||||||||||
| 净损失 | $ | (12,970) | $ | (11,717) | $ | (40,174) | $ | (52,108) | |||||||||||||||