Caribou Biosciences Reports Second Quarter 2024 Financial Results and Provides Business Update
Caribou Biosciences Reports Second Quarter 2024 Financial Results and Provides Business Update
-- Advancing four clinical-stage programs for hematologic malignancies and autoimmune diseases; clinical data reports planned for 2024 and H1 2025 --
2024年和2025年上半年计划发布四项针对血液恶性肿瘤和自身免疫性疾病的临床数据报告。
-- Enrolling 2L LBCL and prior CD19 relapsed LBCL patients based on CB-010 ANTLER Phase 1 data presented at 2024 ASCO Annual Meeting; data to be presented H1 2025 --
基于2024年ASCO年会上Cb-010 ANTLER阶段1数据,招募2L LBCL和先前CD19复发LBCL患者,计划于2025年上半年发布数据。
-- $311.8 million in cash, cash equivalents, and marketable securities expected to fund the current operating plan into H2 2026; cash runway extension of at least 6 months versus prior guidance --
现金、现金等价物和市场证券中的31180万美元预计可支持当前运营计划至2026年下半年;与先前的指南相比,现金储备至少延长6个月。
BERKELEY, Calif., Aug. 06, 2024 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today reported financial results for the second quarter 2024 and reviewed recent pipeline progress.
2024年8月6日,加利福尼亚州伯克利(GLOBE NEWSWIRE)--载体生物科技公司(Caribou Biosciences, Inc.)(纳斯达克代码:CRBU),全球领先的临床基因编辑生物制药公司,今天宣布公布了2024年第二季度的财务结果,并回顾了最近的产品管线进展。
"We are advancing our lead off-the-shelf CAR-T cell therapy, CB-010, in the ANTLER Phase 1 trial with a partial HLA matching strategy with the objective of developing an allogeneic CAR-T cell therapy that can meaningfully rival the autologous CAR-T cell therapies," said Rachel Haurwitz, PhD, Caribou's president and chief executive officer. "We are enrolling approximately 20 second-line and 10 prior CD19 relapsed LBCL patients, and we plan to present initial data for both patient cohorts in the first half of 2025. For CB-011, we expect to report initial dose escalation data in patients with relapsed or refractory multiple myeloma by the end of this year. For CB-012, dose level 1 was cleared, and we are enrolling patients at dose level 2 in the AMpLify Phase 1 trial. We continue to focus our efforts and resources on rapidly advancing our four oncology and autoimmune disease clinical-stage programs through multiple clinical data milestones expected in 2024 and 2025."
"我们正在ANTLER一期试验中推进我们的主导的现成CAR-t细胞疗法Cb-010,并采用部分HLA匹配策略,以开发可以有意义地与自体CAR-t细胞疗法竞争的异系CAR-t细胞疗法," Caribou的总裁兼首席执行官Rachel Haurwitz博士说。 "我们计划招募大约20名二线治疗患者和10名先前CD19复发LBCL患者,并计划在2025年上半年向两类患者呈现初步数据。对于Cb-011,我们希望在今年年底前报告复发或难治性多发性骨髓瘤患者的初始剂量递增数据。对于Cb-012,剂量1已经获批,我们正在AMpLify一期试验中招募剂量2的患者。我们将继续集中我们的精力和资源,通过多个临床数据里程碑,迅速推进我们的四个肿瘤和自身免疫性疾病的临床阶段计划,预计于2024年和2025年发布。"
Clinical highlights
CB-010, a clinical-stage allogeneic anti-CD19 CAR-T cell therapy for B cell non-Hodgkin lymphoma
临床亮点
Cb-010,一种针对b细胞非霍奇金淋巴瘤的现成临床同种异体抗CD19 CAR-t细胞疗法
- In June 2024, Caribou presented clinical data from the ongoing ANTLER Phase 1 clinical trial that indicate a single dose of CB-010 has the potential to rival the safety and efficacy of approved autologous CAR-T cell therapies. The clinical results were presented during a poster presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.
- At ASCO, Caribou presented data on the first 46 patients enrolled in ANTLER. Three dose levels of CB-010 were evaluated (40x106, 80x106, and 120x106 CAR-T cells) and 80x106 CAR-T cells was selected as the recommended Phase 2 dose (RP2D). In dose escalation, 16 patients with multiple subtypes of aggressive relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) were enrolled, and, in dose expansion, 30 patients with second-line large B cell lymphoma (2L LBCL) were enrolled. As of the April 1, 2024 data cutoff date, results demonstrated:
- CB-010 was generally well tolerated. No Grade 3 or higher cytokine release syndrome (CRS) and no graft-versus-host disease (GvHD) was observed.
- A retrospective analysis of all patient data demonstrated that patients who received a dose of CB-010 manufactured from a healthy donor who shared four or more matching human leukocyte antigen ("HLA") alleles with the patient (referred to as partial HLA matching) showed the potential for improved efficacy.
- Pharmacokinetic (PK) data showed that partial HLA matching correlated with increased CAR-T cell expansion and persistence. Pharmacodynamic (PD) data showed extended B cell aplasia and rapid recovery of patients' endogenous T and NK cells.
- Based on these data, Caribou has begun dosing a cohort of approximately 20 2L LBCL patients to prospectively confirm that partial HLA matching may improve patient outcomes.
- Caribou also is enrolling a cohort of up to 10 patients who have relapsed following any prior CD19-targeted therapy in a proof-of-concept cohort in this population of unmet need. This cohort will also incorporate partial HLA matching between donors and patients.
- Caribou plans to initiate a pivotal Phase 3 trial in the second half of 2025, should data confirm improved outcomes for patients receiving a partially HLA matched dose of CB-010 and following agreement with the FDA on a pivotal trial design.
- 2024年6月,Caribou在持续进行的ANTLER一期临床试验中公布的临床数据表明,单次剂量的Cb-010有可能与已批准的自体CAR-t细胞疗法相比具有相似的安全性和疗效。这些临床结果在2024年美国临床肿瘤学会(ASCO)年会期间进行了展示。 在ASCO上,Caribou介绍了ANTLER中招募的首批46名患者的数据。评估了Cb-010的三个剂量水平(40x106、80x106和120x106 CAR-t细胞),选择80x106 CAR-t细胞作为推荐的2期剂量(RP2D)。在剂量递增中,纳入了16名多亚型侵袭性复发性或难治性b细胞非霍奇金淋巴瘤(r/r b-NHL)患者,并在剂量扩展中纳入了30名二线大b细胞淋巴瘤(2L LBCL)患者。截至2024年4月1日的数据截止日期,结果表明: Caribou also is enrolling a cohort of up to 10 patients who have relapsed following any prior CD19-targeted therapy in a proof-of-concept cohort in this population of unmet need. This cohort will also incorporate partial HLA matching between donors and patients.
- 所有患者数据的回顾性分析表明,接收来自与患者共享四个或更多的匹配人类白细胞抗原("HLA")等位基因的健康供体制造的Cb-010剂量(称为部分HLA匹配)显示了改善疗效的潜力。
- CB-010在ANTLER试验中一般耐受性良好。未观察到3级或更高级细胞因子释放综合征(CRS)和无移植物抗宿主病(GvHD)。
- 药代动力学(PK)数据显示,部分HLA匹配与CAR-t细胞扩增和持久性的增加相关。药效动力学(PD)数据显示延长的B细胞失代偿和患者内源性T和NK细胞的快速恢复。
- 基于这些数据,Caribou已经开始给大约20例二线患者进行剂量匹配以验证部分HLA匹配可能会改善患者的预后。
- Caribou还在招募多达10名在先前任何CD19靶向治疗后复发的患者,在这种未满足需求的患者中进行概念验证队列。这个队列也将在供体和受体之间包含部分HLA匹配。
- Caribou计划在数据证实接受部分HLA匹配剂量的Cb-010可以改善患者预后,并获得FDA关于关键试验设计的协议后,于2025年下半年开始开展关键的3期试验。
- Cb-010,针对红斑狼疮的现成临床同种异体抗CD19 CAR-t细胞疗法
CB-010, a clinical-stage allogeneic anti-CD19 CAR-T cell therapy for lupus
Caribou计划在年底前启动GALLOP阶段1临床试验,评估提供给红斑狼疮肾炎(LN)和肾外红斑狼疮(ERL)成人患者的单次Cb-010的效果。试验将包括供体和受体之间的部分HLA匹配。
- Caribou plans to initiate the GALLOP Phase 1 clinical trial to evaluate a single infusion of CB-010 in adult patients with lupus nephritis (LN) and extrarenal lupus (ERL). The trial will incorporate partial HLA matching between donors and patients.
- Caribou plans to initiate the GALLOP Phase 1 clinical trial in adult patients with LN and ERL by year-end 2024.
- Caribou计划在2024年年底前,在LN和ERL的成人患者中启动GALLOP阶段1临床试验。
- Cb-011, 一种针对多发性骨髓瘤的现成临床同种异体抗BCMA CAR-t细胞疗法
CB-011, a clinical-stage allogeneic anti-BCMA CAR-T cell therapy for multiple myeloma
•Caribou正在进行CaMMouflage第一阶段临床试验的剂量逐步升高环节中招募患有多发性骨髓瘤(r/r MM)的患者(https://clinicaltrials.gov/study/NCT05722418)。
- Caribou is enrolling patients with relapsed or refractory multiple myeloma (r/r MM) in the dose escalation portion of the ongoing CaMMouflage Phase 1 clinical trial.
- Caribou plans to present initial dose escalation data from the ongoing CaMMouflage Phase 1 clinical trial by year-end 2024.
- Caribou正在招募复发或难治性多发性骨髓瘤(r / r MM)患者参加正在进行的剂量递增部分的临床实验。 CaMMouflage Phase 1临床试验正在进行中。.
- Caribou计划在2024年年底之前从正在进行的CaMMouflage Phase 1临床试验中呈现初始剂量递增数据。
CB-012, a clinical-stage allogeneic anti-CLL-1 CAR-T cell therapy for acute myeloid leukemia
Cb-012是一种临床阶段的异基因抗CLL-1 CAR-t细胞疗法,用于治疗急性髓样白血病。
- In June 2024, a poster was presented at ASCO on the AMpLify Phase 1 trial design for CB-012 in adults with relapsed or refractory acute myeloid leukemia (r/r AML).
- Caribou is enrolling patients with r/r AML in the dose escalation portion of the ongoing AMpLify Phase 1 clinical trial. Enrollment has concluded for dose level 1 (25x106 CAR-T cells, N=3) and patients are being enrolled at dose level 2 (75x106 CAR-T cells).
- 2024年6月,在ASCO上展示了Cb-012在成人复发或难治性急性髓样白血病(r / r AML)的AMpLify Phase 1试验设计的海报。 海报 Cb-012在正在进行的AMpLify Phase 1临床试验中招募r / r AML患者参加剂量递增部分。
- Caribou正在招募r / r AML患者参加正在进行的剂量递增部分的AMpLify Phase 1临床试验。 AMpLify Phase 1临床试验的剂量递增部分的入组已经结束,第1级剂量(25x106 CAR-t细胞,N = 3)患者正在参加第2级剂量(75x106 CAR-t细胞)的入组。剂量1 (25x106 CAR-t细胞,N=3)的招募已经结束,正在进行剂量2 (75x106 CAR-t细胞)的招募。
Corporate updates
Appointed autoimmune expert to Caribou's scientific advisory board
企业更新
在2024年7月,Terri Laufer博士被任命为Caribou的科学顾问委员会成员之一。Laufer博士是一位知名的风湿病学家,致力于对导致自身免疫性疾病的免疫细胞调控和功能障碍进行广泛的研究。他是宾夕法尼亚大学Perelman医学院名誉副教授, 宾夕法尼亚大学长老教会医疗中心和费城VA医疗中心的风湿科医生。 科学顾问会
- In July 2024, Terri Laufer, MD, was appointed to Caribou's scientific advisory board. Dr. Laufer is a leading rheumatologist known for her extensive research into immune cell regulation and dysfunction that leads to autoimmune diseases. She is an emeritus associate professor of medicine at the Perelman School of Medicine at the University of Pennsylvania and an attending rheumatologist at the Penn Presbyterian Medical Center and Philadelphia VA Medical Center.
- 扩展现金流至2026年下半年
Extended cash runway into H2 2026
在2024年7月,Caribou停止了与其异基因CAR-Nk平台相关的临床前研究活动,并将其员工人数减少约12%。预计员工削减措施以及其他成本节约措施将至少将现金流推迟6个月,直至2026年下半年。公司将承担约50万至100万美元的一次性成本,主要包括受影响员工的现金解雇费用、福利和过渡支持服务。
- In July 2024, Caribou discontinued the preclinical research activities associated with its allogeneic CAR-NK platform and reduced its workforce by approximately 12%. The workforce reduction, together with other cost containment measures, are expected to extend the cash runway by at least 6 months, into H2 2026. The Company will incur approximately $0.5 million to $1.0 million in one-time costs consisting primarily of cash severance costs, benefits, and transition support services for impacted employees.
- 预期里程碑
Anticipated milestones
Cb-010 ANTLER: Caribou计划在2025年上半年呈现基本HLA配备的第2L和先前CD19复发LBCL患者队列的初步数据。如果数据确定Cb-010的部分HLA匹配剂量可以改善患者的预后,则Caribou计划在2025年下半年启动关键的3期临床试验。
- CB-010 ANTLER: Caribou plans to present initial data from both the additional HLA-matched 2L and prior CD19 relapsed LBCL patient cohorts in H1 2025. Caribou plans to initiate a pivotal Phase 3 clinical trial in H2 2025 should data confirm improved outcomes for patients receiving a partially HLA matched dose of CB-010.
- CB-010 GALLOP: Caribou plans to initiate the GALLOP Phase 1 clinical trial in adult patients with LN and ERL by year-end 2024.
- CB-011 CaMMouflage: Caribou plans to present initial dose escalation data from the ongoing CaMMouflage Phase 1 clinical trial by year-end 2024.
- CB-012 AMpLify: Caribou plans to provide updates on dose escalation as the AMpLify Phase 1 clinical trial in r/r AML advances.
- Cb-010 GALLOP: Caribou计划在2024年年底前启动针对LN和ERL成年患者的GALLOP Phase 1临床试验。
- Cb-011 CaMMouflage: Caribou计划在2024年底之前从正在进行的CaMMouflage Phase 1临床试验中呈现初始剂量递增数据。
- Cb-012 AMpLify: Caribou计划在AMpLify Phase 1临床试验在r / r AML领域取得进展时,就剂量递增提供更新。
- Cb-012 AMpLify: Caribou计划在r/r AML AMpLify一期临床试验逐步推进过程中,提供剂量升级的更新。
Second quarter 2024 financial results
Cash, cash equivalents, and marketable securities: Caribou had $311.8 million in cash, cash equivalents, and marketable securities as of June 30, 2024, compared to $372.4 million as of December 31, 2023. Caribou expects these cash, cash equivalents, and marketable securities will be sufficient to fund its current operating plan into H2 2026.
2024年第二季度财务结果
现金、现金等价物和可交易证券:截至2024年6月30日,Caribou的现金、现金等价物和可交易证券为31180万美元,而截至2023年12月31日为37240万美元。Caribou预计这些现金、现金等价物和可交易证券将足以支持其当前的运营计划进入2026年下半年。
Licensing and collaboration revenue: Revenue from Caribou's licensing and collaboration agreements was $3.5 million for the three months ended June 30, 2024, compared to $3.8 million for the same period in 2023. The decrease was primarily due to the now-terminated AbbVie Collaboration and License Agreement, partially offset by an increase in revenue recognized under the Information Rights Agreement Caribou entered into with Pfizer on June 29, 2023. Licensing and collaboration revenue for the three months ended June 30, 2024, includes $1.6 million in a one-time receipt of non-cash equity consideration from one of Caribou's licensees.
许可和合作收入:2024年6月30日结束的三个月中,来自Caribou的许可和合作协议的收入为350万美元,而2023年同一时期为380万美元。该下降主要是由于现已终止的AbbVie合作和许可协议,部分抵消了Caribou在2023年6月29日与辉瑞达成的信息权利协议下认可的收入增加。截至2024年6月30日的许可和合作收入,包括来自Caribou许可证持有人的一次非现金股权考虑的160万美元。
R&D expenses: Research and development expenses were $35.5 million for the three months ended June 30, 2024, compared to $26.5 million for the same period in 2023. The increase was primarily due to costs to advance pipeline programs, including the CB-010 ANTLER, CB-011 CaMMouflage, and CB-012 AMpLify Phase 1 clinical trials; personnel-related expenses, including stock-based compensation, due to headcount increases; and facilities and other allocated expenses.
研发费用:2024年6月30日结束的三个月中,研发费用为3550万美元,而2023年同一时期为2650万美元。该增加主要是由于推进管线项目的成本,包括Cb-010 ANTLER、Cb-011 CaMMouflage和Cb-012 AMpLify Phase 1临床试验; 因员工增加而导致的人事相关费用,包括股票补偿; 以及设施和其他分配的费用。
G&A expenses: General and administrative expenses were $11.5 million for the three months ended June 30, 2024, compared to $10.1 million for the same period in 2023. The increase was primarily due to personnel-related expenses, including stock-based compensation, due to headcount increases, and legal expenses and other service-related expenses. These increases were partially offset by a decrease in patent prosecution and maintenance fees.
管理费用:2024年6月30日结束的三个月中,管理费用为1150万美元,而2023年同一时期为1010万美元。该增加主要是由于人事相关费用,包括股票补偿,由于人头增加以及法律费用和其他服务相关费用。这些增加部分抵消了专利申请和维护费用的减少。
Net loss: Caribou reported a net loss of $37.7 million for the three months ended June 30, 2024, compared to $29.5 million for the same period in 2023.
净亏损:截至2024年6月30日,Caribou报告的净亏损为3770万美元,而2023年同一时期为2950万美元。
About CB-010
CB-010 is the lead clinical-stage product candidate from Caribou's allogeneic CAR-T cell therapy platform, and it is being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) in the ongoing ANTLER Phase 1 clinical trial and will be evaluated in patients with lupus nephritis (LN) and extrarenal lupus (ERL) in the GALLOP Phase 1 clinical trial. In the ANTLER clinical trial, Caribou is enrolling second-line (2L) patients with large B cell lymphoma (LBCL) comprised of different subtypes of aggressive r/r B-NHL (DLBCL NOS, PMBCL, HGBL, tFL, and tMZL) who have never received prior CD19-targeted therapy as well as LBCL patients who have relapsed on a prior CD19-targeted therapy. To Caribou's knowledge, CB-010 is the first allogeneic CAR-T cell therapy in the clinic with a PD-1 knockout, a genome-editing strategy designed to improve activity against diseases by limiting premature CAR-T cell exhaustion. CB-010 is also, to Caribou's knowledge, the first anti-CD19 allogeneic CAR-T cell therapy to be evaluated in the 2L LBCL setting and, for r/r B-NHL, CB-010 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations by the FDA. Additional information on the ANTLER trial (NCT04637763) can be found at clinicaltrials.gov.
关于CB-010:
Cb-010是Caribou的异基因CAR-t细胞疗法平台的主要临床前候选产品,正在进行ANTLER Phase 1临床试验,评估在复发或难治B细胞非霍奇金淋巴瘤(r / r b-NHL)患者中的疗效,并将评估在肾病性肾炎(LN)和肾外狼疮(ERL)患者中的疗效, GALLOP第一期临床试验。在ANTLER临床试验中,Caribou正在招募由不同亚型的侵袭性r / r b-NHL(DLBCL NOS,PMBCL,HGBL,tFL和tMZL)组成的大型B细胞淋巴瘤(LBCL)的二线(2L)患者,他们从未接受过CD19靶向治疗,以及LBCL患者,他们曾在先前接受过CD19靶向治疗后出现复发。据Caribou所知,Cb-010是诊所中第一种带有PD-1敲除的异基因CAR-t细胞疗法,这是一种基因组编辑策略,旨在通过限制过早CAR-t细胞疲劳来提高针对疾病的活性。据Caribou所知,Cb-010也是首个在2L LBCL环境中进行评估的抗CD19异基因CAR-t细胞疗法,并且对于r / r b-NHL,Cb-010已被FDA授予再生医学先进疗法(RMAT),快速进入和孤儿药品认证。有关ANTLER试验(NCT04637763)的其他信息,请参见 clinicaltrials.gov.
About CB-011
CB-011 is a product candidate from Caribou's allogeneic CAR-T cell therapy platform and is being evaluated in patients with relapsed or refractory multiple myeloma (r/r MM) in the CaMMouflage Phase 1 trial. CB-011 is an allogeneic anti-BCMA CAR-T cell therapy engineered using Cas12a chRDNA genome-editing technology. To Caribou's knowledge, CB-011 is the first allogeneic CAR-T cell therapy in the clinic that is engineered to improve antitumor activity through an immune cloaking strategy with a B2M knockout and insertion of a B2M–HLA-E fusion protein to blunt immune-mediated rejection. CB-011 has been granted Fast Track and orphan drug designations by the FDA. Additional information on the CaMMouflage trial (NCT05722418) can be found at clinicaltrials.gov.
关于Cb-011
Cb-011是来自Caribou的异基因CAR-t细胞疗法平台的产品候选人,并正在CaMMouflage Phase 1试验中评估复发或难治的多发性骨髓瘤(r / r MM)患者。 Cb-011是一种异基因抗BCMA CAR-t细胞疗法,利用Cas12a chRDNA基因组编辑技术进行改良。据Caribou所知,Cb-011是第一个在诊所中进行的异基因CAR-t细胞疗法,通过B200万敲除和插入B2M-HLA-E融合蛋白贡献,设计了一种免疫遮蔽策略,以提高抗肿瘤活性。 Cb-011已获得FDA的快速通道和孤儿药物认证。关于CaMMouflage试验(NCT05722418)的其他信息,请参见 clinicaltrials.gov.
About CB-012
CB-012 is a product candidate from Caribou's allogeneic CAR-T cell therapy platform and is being evaluated in the AMpLify Phase 1 clinical trial in patients with relapsed or refractory acute myeloid leukemia (r/r AML). CB-012 is an anti-CLL-1 CAR-T cell therapy engineered with five genome edits, enabled by Caribou's patented next-generation CRISPR technology platform, which uses Cas12a chRDNA genome editing to significantly improve the specificity of genome edits. To Caribou's knowledge, CB-012 is the first allogeneic CAR-T cell therapy with both checkpoint disruption, through a PD-1 knockout, and immune cloaking, through a B2M knockout and B2M–HLA-E fusion protein insertion; both armoring strategies are designed to improve antitumor activity. Caribou has exclusively in-licensed from Memorial Sloan Kettering Cancer Center (MSKCC) in the field of allogeneic CLL-1-targeted cell therapy a panel of fully human scFvs targeting CLL-1, from which the company has selected a scFv for the generation of the company's CAR. Additional information on the AMpLify trial (NCT06128044) can be found at clinicaltrials.gov.
关于Cb-012
Cb-012是来自Caribou的异基因CAR-t细胞疗法平台的产品候选人,并正在AMpLify Phase 1临床试验中评估复发或难治的急性髓系白血病(r / r AML)患者。Cb-012是一种具有五个基因组编辑的抗CLL-1 CAR-t细胞疗法,这是由Caribou的专利下一代CRISPR技术平台使能的,该平台使用Cas12a chRDNA基因组编辑显着提高了基因组编辑的特异性。据Caribou所知,Cb-012是第一种既打破核查点,通过PD-1敲除,也打破了免疫遮蔽,通过B200万敲除和B2M-HLA-E融合蛋白插入的CAR-t细胞疗法;这两种装甲策略均旨在提高抗肿瘤活性。Caribou已经在这一领域中独家授权了Memorial Sloan Kettering Cancer Center(MSKCC)的全人类scFvs面板,用于针对CLL-1的异基因细胞疗法,该公司已经从中选择了一种用于生成公司的CAR。有关AMpLify试验(NCT06128044)的其他信息,请参见 clinicaltrials.gov.
About Caribou's novel next-generation CRISPR platform
CRISPR genome editing uses easily designed, modular biological tools to make DNA changes in living cells. There are two basic components of Class 2 CRISPR systems: the nuclease protein that cuts DNA and the RNA molecule(s) that guide the nuclease to generate a site-specific, double-stranded break, leading to an edit at the targeted genomic site. CRISPR systems are capable of editing unintended genomic sites, known as off-target editing, which may lead to harmful effects on cellular function and phenotype. In response to this challenge, Caribou has developed CRISPR hybrid RNA-DNA guides (chRDNAs; pronounced "chardonnays") that direct substantially more precise genome editing compared to all-RNA guides. Caribou is deploying the power of its chRDNA technology to carry out high efficiency multiple edits, to develop CRISPR-edited therapies.
关于Caribou的新一代CRISPR平台
CRISPR基因组编辑使用易于设计的、模块化的生物工具在活体细胞中进行DNA改变。Class 2 CRISPR系统有两个基本元素:切割DNA的核酸酶蛋白和RNA分子,它们指导核酸酶在特定的基因组位点上产生特异性的双链断裂,从而在目标基因组位点上发生编辑。CRISPR系统能够编辑称为“非靶向编辑”的意外基因组位点,这可能对细胞功能和表型产生有害影响。针对这一挑战,Caribou开发了CRISPR杂交RNA-DNA指南(chRDNA;发音为“夏多内”),相对于全RNA指南,它们可以更精确地定向基因组编辑。Caribou正在利用其chRDNA技术的威力进行高效率的多个编辑,并开发CRISPR编辑的治疗方案。
About Caribou Biosciences, Inc.
Caribou Biosciences is a clinical-stage CRISPR genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating diseases. The company's genome-editing platform, including its Cas12a chRDNA technology, enables superior precision to develop cell therapies that are armored to potentially improve activity against diseases. Caribou is advancing a pipeline of clinical-stage off-the-shelf cell therapies from its CAR-T cell platform as readily available treatments for patients with hematologic malignancies and autoimmune diseases. Follow us @CaribouBio and visit .
关于Caribou Biosciences, Inc。
Caribou Biosciences是一家临床阶段的CRISPR基因组编辑生物制药公司,致力于为患有毁灭性疾病的患者开发变革性疗法。该公司的基因组编辑平台,包括其Cas12a chRDNA技术,能够实现更高的精度,以开发装甲细胞疗法,可能提高针对疾病的活性。 Caribou正在推进从其CAR-t细胞平台提供的一系列临床阶段现成的细胞疗法,作为治疗血液肿瘤和自身免疫性疾病的可用治疗方案。关注我们@CaribouBio并访问 .
Forward-looking statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential," or "continue," or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. These forward-looking statements include, without limitation, statements related to Caribou's strategy, plans, and objectives, and expectations regarding its clinical and preclinical development programs, including its expectations relating to (i) the timing of reporting additional dose expansion data in its ANTLER Phase 1 clinical trial for CB-010, including data from both additional HLA-matched 2L LBCL and prior CD19 cohorts, and the timing of an ANTLER pivotal Phase 3 clinical trial; (ii) the timing of and updates from its CaMMouflage Phase 1 clinical trial for CB-011 and expectations regarding the timing of presenting the initial dose escalation data; (iii) the timing of and updates from its AMpLify Phase 1 clinical trial for CB-012; (iv) the timing of and updates from its GALLOP Phase 1 clinical trial for CB-010 in patients with LN and ERL; (v) the anticipated costs associated with the workforce reduction, including specific categories of costs and future cash expenditures and the timing of when the reduction is expected to be completed and the anticipated costs recognized; and (vi) its expected funding runway of cash, cash equivalents, and marketable securities. Management believes that these forward-looking statements are reasonable as and when made. However, such forward-looking statements are subject to risks and uncertainties, and actual results may differ materially from any future results expressed or implied by the forward-looking statements. Risks and uncertainties include, without limitation, risks inherent in the development of cell therapy products; uncertainties related to the initiation, cost, timing, progress, and results of Caribou's current and future research and development programs, preclinical studies, and clinical trials; and the risk that initial, preliminary, or interim clinical trial data will not ultimately be predictive of the safety and efficacy of Caribou's product candidates or that clinical outcomes may differ as patient enrollment continues and as more patient data becomes available; the risk that preclinical study results observed will not be borne out in human patients or different conclusions or considerations are reached once additional data have been received and fully evaluated; the ability to obtain key regulatory input and approvals; as well as other risk factors described from time to time in Caribou's filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent filings. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. Except as required by law, Caribou undertakes no obligation to update publicly any forward-looking statements for any reason.
前瞻性声明
本新闻稿包含根据1995年《私人证券诉讼改革法》的前瞻性声明。在某些情况下,您可以通过诸如“可能”、“将”、“应该”、“期望”、“计划”、“预计”、“可能”、“打算”、“目标”、“项目”、“考虑”、“相信”、“估计”、“预测”,“潜在”或“继续”等类似的表述,识别前瞻性声明,尽管并非所有前瞻性声明都包含这些词。这些前瞻性声明包括但不限于与Caribou的策略、计划和目标,以及其临床和临床前开发计划有关的声明,包括其与(i)Cb-010 的 ANTLER 第1期临床试验的额外剂量扩展数据报告的时机,包括来自额外的HLA匹配的2L LBCL和前CD19队列,以及ANTLER关键的第3期临床试验的时机;(ii)Cb-011 的 CaMMouflage 第1期临床试验的时机和更新,以及有关提出初步剂量升级数据的时机的预期;(iii)Cb-012 的 AMpLify 第1期临床试验的时机和更新;(iv)Cb-010 在LN和ERL患者中的 GALLOP 第1期临床试验的时机和更新;(v)与减少劳动力相关的预期成本,包括成本的特定类别和未来的现金支出以及预计完成削减的时间和预计的成本记录;以及(vi)其预期的现金、现金等价物和可市场化证券的资金跑道。管理层认为,这些前瞻性声明是合理的,仅适用于及时做出的情况下。然而,这样的前瞻性声明受到风险和不确定性的影响,实际结果可能与前瞻性声明所表达或暗示的任何未来结果存在实质性差异。风险和不确定性包括但不限于细胞治疗产品的开发风险;与Caribou当前和未来的研究和开发计划、临床前研究和临床试验的启动、成本、时机、进度和结果有关的不确定性;以及初步、初步或 interim 临床试验数据将最终不能预测 Caribou 的产品候选者安全性和有效性,或者临床结果随着病人入组的继续和更多病人数据的可用性而产生差异的风险;还包括在人体病患中观察到的临床前研究结果最终不会在人类病患中得到证实,或者接收和完全评估进一步数据后达成不同结论或考虑选择的风险;获得关键监管部门的指导和批准的能力以及其他定期或不定期在美国证券交易委员会提交的文件中描述的风险因素,包括其截至2023年12月31日的年度报告表格10-k和后续提交的文件。考虑到这些前瞻性声明的重大不确定性,您不应将前瞻性声明作为未来活动的预测依据。除法律另有规定外,Caribou不承诺出于任何原因公开更新任何前瞻性声明。
Caution should be exercised when interpreting results from separate trials involving other CAR-T cell therapies. The results of other CAR-T cell therapies presented or referenced in this press release have been derived from publicly available reports of clinical trials not conducted by Caribou, and Caribou has not performed any head-to-head trials comparing any of these other CAR-T cell therapies with CB-010. As such, the results of these other clinical trials may not be comparable to clinical results for CB-010. The design of these other clinical trials varies in material ways from the design of the ANTLER clinical trial for CB-010, including with respect to patient populations, follow-up times, clinical trial phases, and subject characteristics. As a result, cross-trial comparisons may have no interpretive value on Caribou's existing or future clinical results. For further information and to understand these material differences, you should read the reports for the other CAR-T cell therapy clinical trials and the sources included in Caribou's corporate presentations on its website.
翻译其他CAR-t细胞疗法单独试验结果时需谨慎。在本新闻稿中提供或引用的其他CAR-t细胞疗法结果来自非Caribou进行的临床试验的公开报告中,并且Caribou没有进行任何将这些其他CAR-t细胞疗法与Cb-010进行比较的头对头试验。因此,这些其他临床试验的结果可能不适用于Cb-010的临床结果。这些其他临床试验的设计在重要方面与Cb-010的ANTLER临床试验的设计不同,包括患者人群、随访时间、临床试验阶段和受试者特征。因此,交叉试验比较可能没有解释 Caribou 其现有或未来临床结果的价值。有关进一步信息和了解这些重要区别,您应阅读其他CAR-t细胞疗法临床试验的报告以及包括在Caribou网站公司介绍中的来源。
Caribou Biosciences, Inc. Condensed Consolidated Balance Sheet Data (in thousands) (unaudited) | |||||||
June 30, 2024 |
December 31, 2023 | ||||||
Cash, cash equivalents, and marketable securities | $ | 311,773 | $ | 372,404 | |||
Total assets | 372,938 | 432,209 | |||||
Total liabilities | 62,474 | 63,808 | |||||
Total stockholders' equity | 310,464 | 368,401 | |||||
Total liabilities and stockholders' equity | $ | 372,938 | $ | 432,209 |
全球领先的CRISPR基因编辑生物制药公司Caribou Biosciences,Inc。 简明合并资产负债表数据 (以千为单位) (未经审计) | |||||||
2024年6月30日 |
2023年12月31日 | ||||||
现金、现金等价物和可交易证券 | $ | 311,773 372,404 372,938 432,209 62,474 310,464 368,401 372,938 432,209 许可和合作收入 35,480 69,268 52,212 26,128 95,396 71,241 (43,501) (32,868) | $ | 372,404 | |||
总资产 | 372,938 | 432,209 | |||||
负债合计 | 62,474 | 63,808 | |||||
股东权益合计 | 310,464 | 368,401 | |||||
负债和股东权益合计 | $ | 372,938 | $ | 432,209 |
Caribou Biosciences, Inc. Condensed Consolidated Statement of Operations (in thousands, except share and per share data) (unaudited) | |||||||||||||||
Three Months Ended June 30, |
Six Months Ended June 30, |
||||||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||||||
Licensing and collaboration revenue | $ | 3,464 | $ | 3,755 | $ | 5,893 | $ | 7,257 | |||||||
Operating expenses: | |||||||||||||||
Research and development | 35,480 | 26,503 | 69,268 | 52,212 | |||||||||||
General and administrative | 11,485 | 10,120 | 26,128 | 19,029 | |||||||||||
Total operating expenses | 46,965 | 36,623 | 95,396 | 71,241 | |||||||||||
Loss from operations | (43,501) | (32,868) | (89,503) | (63,984) | |||||||||||
Other income (expense): | |||||||||||||||
Change in fair value of equity securities | (102) | 22 | (102) | 7 | |||||||||||
Change in fair value of the MSKCC success payments liability | 1,795 | 279 | 2,098 | 534 | |||||||||||
Other income - net | 4,111 | 3,048 | 8,576 | 5,880 | |||||||||||
Total other income (expense) | 5,804 | 3,349 | 10,572 | 6,421 | |||||||||||
Net loss | (37,697) | (29,519) | (78,931) | (57,563) | |||||||||||
Other comprehensive (loss) income: | |||||||||||||||
Net unrealized (loss) gain on available-for-sale marketable securities, net of tax |
3 | (406) | (349) | 382 | |||||||||||
Net comprehensive loss | $ | (37,694) | $ | (29,925) | $ | (79,280) | $ | (57,181) | |||||||
Net loss per share, basic and diluted | $ | (0.42) | $ | (0.48) | $ | (0.88) | $ | (0.94) | |||||||
Weighted-average common shares outstanding, basic and diluted | 90,340,932 | 61,417,934 | 89,821,935 | 61,302,863 |
全球领先的CRISPR基因编辑生物制药公司Caribou Biosciences,Inc。 汇编损益统计表 (以千为单位,除股份数和每股数据外) (未经审计) | |||||||||||||||
三个月之内结束 2020年6月30日 |
销售额最高的六个月 2020年6月30日 |
||||||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||||||
许可和合作收入 | $ | 3,464 | $ | 3,755 | $ | 5,893 | $ | 7,257 | |||||||
营业费用: | |||||||||||||||
研发 | 35,480 | 26,503 | 69,268 | 52,212 | |||||||||||
普通和管理 | 11,485 | 10,120 | 26,128 | 19,029 | |||||||||||
营业费用总计 | 截至2024年3月30日,我们的总负债为 | 36,623 | 95,396 | 71,241 | |||||||||||
经营亏损 | (43,501) | (32,868) | (89,503) | (63,984) | |||||||||||
其他收入(支出): | |||||||||||||||
股权证券公允价值变动 | (102) | 22 | (102) | 7 | |||||||||||
MSKCC成功支付负债公允价值变动 | 1,795 | 279 | 2,098 | 534 | |||||||||||
其他收入-净额 | 4,111 | 3,048 | 8,576 元 | 5,880 | |||||||||||
其他收入(支出)总额 | 5,804 | 3,349 | 10,572 | 6,421 | |||||||||||
净亏损 | (37,697) | (29,519) | (78,931) | (57,563) | |||||||||||
其他综合收益(损失): | |||||||||||||||
可供出售证券未实现损益, 税后净额 |
3 | (406) | (349) | 382 | |||||||||||
综合收益净亏损 | $ | (37,694) | $ | (29,925) | $ | (79,280) | $ | (57,181) | |||||||
基本和稀释每股净亏损 | $ | (0.42) | $ | (0.48) | $ | (0.88) | $ | (0.94) | |||||||
基本和稀释加权普通股份 | 90,340,932 | 61,417,934 | 89,821,935 | 61,302,863 |
Caribou Biosciences, Inc. contacts:
Investors:
Amy Figueroa, CFA
investor.relations@cariboubio.com
Caribou Biosciences, Inc.联系人:
投资者:
Amy Figueroa,CFA
investor.relations@cariboubio.com
Media:
Peggy Vorwald, PhD
media@cariboubio.com
媒体:
Peggy Vorwald,PhD
media@cariboubio.com