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年上半年計劃發佈四項針對血液惡性腫瘤和自身免疫性疾病的臨床數據報告。
-- 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 --
基於2024年ASCO年會上Cb-010 ANTLER階段1數據,招募2L LBCL和先前CD19復發LBCL患者,計劃於2025年上半年發佈數據。
-- $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 --
現金、現金等價物和市場證券中的31180萬美元預計可支持當前運營計劃至2026年下半年;與先前的指南相比,現金儲備至少延長6個月。
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,加利福尼亞州伯克利(GLOBE NEWSWIRE)--載體生物科技公司(Caribou Biosciences, Inc.)(納斯達克代碼:CRBU),全球領先的臨床基因編輯生物製藥公司,今天宣佈公佈了2024年第二季度的財務結果,並回顧了最近的產品管線進展。
"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."
"我們正在ANTLER一期試驗中推進我們的主導的現成CAR-t細胞療法Cb-010,並採用部分HLA匹配策略,以開發可以有意義地與自體CAR-t細胞療法競爭的異系CAR-t細胞療法," Caribou的總裁兼首席執行官Rachel Haurwitz博士說。 "我們計劃招募大約20名二線治療患者和10名先前CD19復發LBCL患者,並計劃在2025年上半年向兩類患者呈現初步數據。對於Cb-011,我們希望在今年年底前報告復發或難治性多發性骨髓瘤患者的初始劑量遞增數據。對於Cb-012,劑量1已經獲批,我們正在AMpLify一期試驗中招募劑量2的患者。我們將繼續集中我們的精力和資源,通過多個臨床數據里程碑,迅速推進我們的四個腫瘤和自身免疫性疾病的臨床階段計劃,預計於2024年和2025年發佈。"
Clinical highlights
CB-010, a clinical-stage allogeneic anti-CD19 CAR-T cell therapy for B cell non-Hodgkin lymphoma
臨床亮點
Cb-010,一種針對b細胞非霍奇金淋巴瘤的現成臨床同種異體抗CD19 CAR-t細胞療法
- 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在持續進行的ANTLER一期臨床試驗中公佈的臨床數據表明,單次劑量的Cb-010有可能與已批准的自體CAR-t細胞療法相比具有相似的安全性和療效。這些臨床結果在2024年美國臨床腫瘤學會(ASCO)年會期間進行了展示。 在ASCO上,Caribou介紹了ANTLER中招募的首批46名患者的數據。評估了Cb-010的三個劑量水平(40x106、80x106和120x106 CAR-t細胞),選擇80x106 CAR-t細胞作爲推薦的2期劑量(RP2D)。在劑量遞增中,納入了16名多亞型侵襲性複發性或難治性b細胞非霍奇金淋巴瘤(r/r b-NHL)患者,並在劑量擴展中納入了30名二線大b細胞淋巴瘤(2L LBCL)患者。截至2024年4月1日的數據截止日期,結果表明: 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.
- 所有患者數據的回顧性分析表明,接收來自與患者共享四個或更多的匹配人類白細胞抗原("HLA")等位基因的健康供體制造的Cb-010劑量(稱爲部分HLA匹配)顯示了改善療效的潛力。
- CB-010在ANTLER試驗中一般耐受性良好。未觀察到3級或更高級細胞因子釋放綜合徵(CRS)和無移植物抗宿主病(GvHD)。
- 藥代動力學(PK)數據顯示,部分HLA匹配與CAR-t細胞擴增和持久性的增加相關。藥效動力學(PD)數據顯示延長的B細胞失代償和患者內源性T和NK細胞的快速恢復。
- 基於這些數據,Caribou已經開始給大約20例二線患者進行劑量匹配以驗證部分HLA匹配可能會改善患者的預後。
- Caribou還在招募多達10名在先前任何CD19靶向治療後復發的患者,在這種未滿足需求的患者中進行概念驗證隊列。這個隊列也將在供體和受體之間包含部分HLA匹配。
- Caribou計劃在數據證實接受部分HLA匹配劑量的Cb-010可以改善患者預後,並獲得FDA關於關鍵試驗設計的協議後,於2025年下半年開始開展關鍵的3期試驗。
- Cb-010,針對紅斑狼瘡的現成臨床同種異體抗CD19 CAR-t細胞療法
CB-010, a clinical-stage allogeneic anti-CD19 CAR-T cell therapy for lupus
Caribou計劃在年底前啓動GALLOP階段1臨床試驗,評估提供給紅斑狼瘡腎炎(LN)和腎外紅斑狼瘡(ERL)成人患者的單次Cb-010的效果。試驗將包括供體和受體之間的部分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計劃在2024年年底前,在LN和ERL的成人患者中啓動GALLOP階段1臨床試驗。
- Cb-011, 一種針對多發性骨髓瘤的現成臨床同種異體抗BCMA CAR-t細胞療法
CB-011, a clinical-stage allogeneic anti-BCMA CAR-T cell therapy for multiple myeloma
•Caribou正在進行CaMMouflage第一階段臨床試驗的劑量逐步升高環節中招募患有多發性骨髓瘤(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正在招募復發或難治性多發性骨髓瘤(r / r MM)患者參加正在進行的劑量遞增部分的臨床實驗。 CaMMouflage Phase 1臨床試驗正在進行中。.
- Caribou計劃在2024年年底之前從正在進行的CaMMouflage Phase 1臨床試驗中呈現初始劑量遞增數據。
CB-012, a clinical-stage allogeneic anti-CLL-1 CAR-T cell therapy for acute myeloid leukemia
Cb-012是一種臨床階段的異基因抗CLL-1 CAR-t細胞療法,用於治療急性髓樣白血病。
- 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在成人復發或難治性急性髓樣白血病(r / r AML)的AMpLify Phase 1試驗設計的海報。 海報 Cb-012在正在進行的AMpLify Phase 1臨床試驗中招募r / r AML患者參加劑量遞增部分。
- Caribou正在招募r / r AML患者參加正在進行的劑量遞增部分的AMpLify Phase 1臨床試驗。 AMpLify Phase 1臨床試驗的劑量遞增部分的入組已經結束,第1級劑量(25x106 CAR-t細胞,N = 3)患者正在參加第2級劑量(75x106 CAR-t細胞)的入組。劑量1 (25x106 CAR-t細胞,N=3)的招募已經結束,正在進行劑量2 (75x106 CAR-t細胞)的招募。
Corporate updates
Appointed autoimmune expert to Caribou's scientific advisory board
企業更新
在2024年7月,Terri Laufer博士被任命爲Caribou的科學顧問委員會成員之一。Laufer博士是一位知名的風溼病學家,致力於對導致自身免疫性疾病的免疫細胞調控和功能障礙進行廣泛的研究。他是賓夕法尼亞大學Perelman醫學院名譽副教授, 賓夕法尼亞大學長老教會醫療中心和費城VA醫療中心的風溼科醫生。 科學顧問會
- 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.
- 擴展現金流至2026年下半年
Extended cash runway into H2 2026
在2024年7月,Caribou停止了與其異基因CAR-Nk平台相關的臨床前研究活動,並將其員工人數減少約12%。預計員工削減措施以及其他成本節約措施將至少將現金流推遲6個月,直至2026年下半年。公司將承擔約50萬至100萬美元的一次性成本,主要包括受影響員工的現金解僱費用、福利和過渡支持服務。
- 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.
- 預期里程碑
Anticipated milestones
Cb-010 ANTLER: Caribou計劃在2025年上半年呈現基本HLA配備的第2L和先前CD19復發LBCL患者隊列的初步數據。如果數據確定Cb-010的部分HLA匹配劑量可以改善患者的預後,則Caribou計劃在2025年下半年啓動關鍵的3期臨床試驗。
- 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計劃在2024年年底前啓動針對LN和ERL成年患者的GALLOP Phase 1臨床試驗。
- Cb-011 CaMMouflage: Caribou計劃在2024年底之前從正在進行的CaMMouflage Phase 1臨床試驗中呈現初始劑量遞增數據。
- Cb-012 AMpLify: Caribou計劃在AMpLify Phase 1臨床試驗在r / r AML領域取得進展時,就劑量遞增提供更新。
- Cb-012 AMpLify: Caribou計劃在r/r AML AMpLify一期臨床試驗逐步推進過程中,提供劑量升級的更新。
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年第二季度財務結果
現金、現金等價物和可交易證券:截至2024年6月30日,Caribou的現金、現金等價物和可交易證券爲31180萬美元,而截至2023年12月31日爲37240萬美元。Caribou預計這些現金、現金等價物和可交易證券將足以支持其當前的運營計劃進入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.
許可和合作收入:2024年6月30日結束的三個月中,來自Caribou的許可和合作協議的收入爲350萬美元,而2023年同一時期爲380萬美元。該下降主要是由於現已終止的AbbVie合作和許可協議,部分抵消了Caribou在2023年6月29日與輝瑞達成的信息權利協議下認可的收入增加。截至2024年6月30日的許可和合作收入,包括來自Caribou許可證持有人的一次非現金股權考慮的160萬美元。
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.
研發費用:2024年6月30日結束的三個月中,研發費用爲3550萬美元,而2023年同一時期爲2650萬美元。該增加主要是由於推進管線項目的成本,包括Cb-010 ANTLER、Cb-011 CaMMouflage和Cb-012 AMpLify Phase 1臨床試驗; 因員工增加而導致的人事相關費用,包括股票補償; 以及設施和其他分配的費用。
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.
管理費用:2024年6月30日結束的三個月中,管理費用爲1150萬美元,而2023年同一時期爲1010萬美元。該增加主要是由於人事相關費用,包括股票補償,由於人頭增加以及法律費用和其他服務相關費用。這些增加部分抵消了專利申請和維護費用的減少。
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.
淨虧損:截至2024年6月30日,Caribou報告的淨虧損爲3770萬美元,而2023年同一時期爲2950萬美元。
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.
關於CB-010:
Cb-010是Caribou的異基因CAR-t細胞療法平台的主要臨床前候選產品,正在進行ANTLER Phase 1臨床試驗,評估在復發或難治B細胞非霍奇金淋巴瘤(r / r b-NHL)患者中的療效,並將評估在腎病性腎炎(LN)和腎外狼瘡(ERL)患者中的療效, GALLOP第一期臨床試驗。在ANTLER臨床試驗中,Caribou正在招募由不同亞型的侵襲性r / r b-NHL(DLBCL NOS,PMBCL,HGBL,tFL和tMZL)組成的大型B細胞淋巴瘤(LBCL)的二線(2L)患者,他們從未接受過CD19靶向治療,以及LBCL患者,他們曾在先前接受過CD19靶向治療後出現復發。據Caribou所知,Cb-010是診所中第一種帶有PD-1敲除的異基因CAR-t細胞療法,這是一種基因組編輯策略,旨在通過限制過早CAR-t細胞疲勞來提高針對疾病的活性。據Caribou所知,Cb-010也是首個在2L LBCL環境中進行評估的抗CD19異基因CAR-t細胞療法,並且對於r / r b-NHL,Cb-010已被FDA授予再生醫學先進療法(RMAT),快速進入和孤兒藥品認證。有關ANTLER試驗(NCT04637763)的其他信息,請參見 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.
關於Cb-011
Cb-011是來自Caribou的異基因CAR-t細胞療法平台的產品候選人,並正在CaMMouflage Phase 1試驗中評估復發或難治的多發性骨髓瘤(r / r MM)患者。 Cb-011是一種異基因抗BCMA CAR-t細胞療法,利用Cas12a chRDNA基因組編輯技術進行改良。據Caribou所知,Cb-011是第一個在診所中進行的異基因CAR-t細胞療法,通過B200萬敲除和插入B2M-HLA-E融合蛋白貢獻,設計了一種免疫遮蔽策略,以提高抗腫瘤活性。 Cb-011已獲得FDA的快速通道和孤兒藥物認證。關於CaMMouflage試驗(NCT05722418)的其他信息,請參見 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.
關於Cb-012
Cb-012是來自Caribou的異基因CAR-t細胞療法平台的產品候選人,並正在AMpLify Phase 1臨床試驗中評估復發或難治的急性髓系白血病(r / r AML)患者。Cb-012是一種具有五個基因組編輯的抗CLL-1 CAR-t細胞療法,這是由Caribou的專利下一代CRISPR技術平台使能的,該平台使用Cas12a chRDNA基因組編輯顯着提高了基因組編輯的特異性。據Caribou所知,Cb-012是第一種既打破覈查點,通過PD-1敲除,也打破了免疫遮蔽,通過B200萬敲除和B2M-HLA-E融合蛋白插入的CAR-t細胞療法;這兩種裝甲策略均旨在提高抗腫瘤活性。Caribou已經在這一領域中獨家授權了Memorial Sloan Kettering Cancer Center(MSKCC)的全人類scFvs面板,用於針對CLL-1的異基因細胞療法,該公司已經從中選擇了一種用於生成公司的CAR。有關AMpLify試驗(NCT06128044)的其他信息,請參見 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.
關於Caribou的新一代CRISPR平台
CRISPR基因組編輯使用易於設計的、模塊化的生物工具在活體細胞中進行DNA改變。Class 2 CRISPR系統有兩個基本元素:切割DNA的核酸酶蛋白和RNA分子,它們指導核酸酶在特定的基因組位點上產生特異性的雙鏈斷裂,從而在目標基因組位點上發生編輯。CRISPR系統能夠編輯稱爲“非靶向編輯”的意外基因組位點,這可能對細胞功能和表型產生有害影響。針對這一挑戰,Caribou開發了CRISPR雜交RNA-DNA指南(chRDNA;發音爲“夏多內”),相對於全RNA指南,它們可以更精確地定向基因組編輯。Caribou正在利用其chRDNA技術的威力進行高效率的多個編輯,並開發CRISPR編輯的治療方案。
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 .
關於Caribou Biosciences, Inc。
Caribou Biosciences是一家臨床階段的CRISPR基因組編輯生物製藥公司,致力於爲患有毀滅性疾病的患者開發變革性療法。該公司的基因組編輯平台,包括其Cas12a chRDNA技術,能夠實現更高的精度,以開發裝甲細胞療法,可能提高針對疾病的活性。 Caribou正在推進從其CAR-t細胞平台提供的一系列臨床階段現成的細胞療法,作爲治療血液腫瘤和自身免疫性疾病的可用治療方案。關注我們@CaribouBio並訪問 .
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.
前瞻性聲明
本新聞稿包含根據1995年《私人證券訴訟改革法》的前瞻性聲明。在某些情況下,您可以通過諸如“可能”、“將”、“應該”、“期望”、“計劃”、“預計”、“可能”、“打算”、“目標”、“項目”、“考慮”、“相信”、“估計”、“預測”,“潛在”或“繼續”等類似的表述,識別前瞻性聲明,儘管並非所有前瞻性聲明都包含這些詞。這些前瞻性聲明包括但不限於與Caribou的策略、計劃和目標,以及其臨床和臨床前開發計劃有關的聲明,包括其與(i)Cb-010 的 ANTLER 第1期臨床試驗的額外劑量擴展數據報告的時機,包括來自額外的HLA匹配的2L LBCL和前CD19隊列,以及ANTLER關鍵的第3期臨床試驗的時機;(ii)Cb-011 的 CaMMouflage 第1期臨床試驗的時機和更新,以及有關提出初步劑量升級數據的時機的預期;(iii)Cb-012 的 AMpLify 第1期臨床試驗的時機和更新;(iv)Cb-010 在LN和ERL患者中的 GALLOP 第1期臨床試驗的時機和更新;(v)與減少勞動力相關的預期成本,包括成本的特定類別和未來的現金支出以及預計完成削減的時間和預計的成本記錄;以及(vi)其預期的現金、現金等價物和可市場化證券的資金跑道。管理層認爲,這些前瞻性聲明是合理的,僅適用於及時做出的情況下。然而,這樣的前瞻性聲明受到風險和不確定性的影響,實際結果可能與前瞻性聲明所表達或暗示的任何未來結果存在實質性差異。風險和不確定性包括但不限於細胞治療產品的開發風險;與Caribou當前和未來的研究和開發計劃、臨床前研究和臨床試驗的啓動、成本、時機、進度和結果有關的不確定性;以及初步、初步或 interim 臨床試驗數據將最終不能預測 Caribou 的產品候選者安全性和有效性,或者臨床結果隨着病人入組的繼續和更多病人數據的可用性而產生差異的風險;還包括在人體病患中觀察到的臨床前研究結果最終不會在人類病患中得到證實,或者接收和完全評估進一步數據後達成不同結論或考慮選擇的風險;獲得關鍵監管部門的指導和批准的能力以及其他定期或不定期在美國證券交易委員會提交的文件中描述的風險因素,包括其截至2023年12月31日的年度報告表格10-k和後續提交的文件。考慮到這些前瞻性聲明的重大不確定性,您不應將前瞻性聲明作爲未來活動的預測依據。除法律另有規定外,Caribou不承諾出於任何原因公開更新任何前瞻性聲明。
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.
翻譯其他CAR-t細胞療法單獨試驗結果時需謹慎。在本新聞稿中提供或引用的其他CAR-t細胞療法結果來自非Caribou進行的臨床試驗的公開報告中,並且Caribou沒有進行任何將這些其他CAR-t細胞療法與Cb-010進行比較的頭對頭試驗。因此,這些其他臨床試驗的結果可能不適用於Cb-010的臨床結果。這些其他臨床試驗的設計在重要方面與Cb-010的ANTLER臨床試驗的設計不同,包括患者人群、隨訪時間、臨床試驗階段和受試者特徵。因此,交叉試驗比較可能沒有解釋 Caribou 其現有或未來臨床結果的價值。有關進一步信息和了解這些重要區別,您應閱讀其他CAR-t細胞療法臨床試驗的報告以及包括在Caribou網站公司介紹中的來源。
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 |
全球領先的CRISPR基因編輯生物製藥公司Caribou Biosciences,Inc。 簡明合併資產負債表數據 (以千爲單位) (未經審計) | |||||||
2024年6月30日 |
2023年12月31日 | ||||||
現金、現金等價物和可交易證券 | $ | 311,773 372,404 372,938 432,209 62,474 310,464 368,401 372,938 432,209 許可和合作收入 35,480 69,268 52,212 26,128 95,396 71,241 (43,501) (32,868) | $ | 372,404 | |||
總資產 | 372,938 | 432,209 | |||||
負債合計 | 62,474 | 63,808 | |||||
股東權益合計 | 310,464 | 368,401 | |||||
負債和股東權益合計 | $ | 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 |
全球領先的CRISPR基因編輯生物製藥公司Caribou Biosciences,Inc。 彙編損益統計表 (以千爲單位,除股份數和每股數據外) (未經審計) | |||||||||||||||
三個月之內結束 2020年6月30日 |
銷售額最高的六個月 2020年6月30日 |
||||||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||||||
許可和合作收入 | $ | 3,464 | $ | 3,755 | $ | 5,893 | $ | 7,257 | |||||||
營業費用: | |||||||||||||||
研發 | 35,480 | 26,503 | 69,268 | 52,212 | |||||||||||
普通和管理 | 11,485 | 10,120 | 26,128 | 19,029 | |||||||||||
營業費用總計 | 截至2024年3月30日,我們的總負債爲 | 36,623 | 95,396 | 71,241 | |||||||||||
經營虧損 | (43,501) | (32,868) | (89,503) | (63,984) | |||||||||||
其他收入(支出): | |||||||||||||||
股權證券公允價值變動 | (102) | 22 | (102) | 7 | |||||||||||
MSKCC成功支付負債公允價值變動 | 1,795 | 279 | 2,098 | 534 | |||||||||||
其他收入-淨額 | 4,111 | 3,048 | 8,576 元 | 5,880 | |||||||||||
其他收入(支出)總額 | 5,804 | 3,349 | 10,572 | 6,421 | |||||||||||
淨虧損 | (37,697) | (29,519) | (78,931) | (57,563) | |||||||||||
其他綜合收益(損失): | |||||||||||||||
可供出售證券未實現損益, 稅後淨額 |
3 | (406) | (349) | 382 | |||||||||||
綜合收益淨虧損 | $ | (37,694) | $ | (29,925) | $ | (79,280) | $ | (57,181) | |||||||
基本和稀釋每股淨虧損 | $ | (0.42) | $ | (0.48) | $ | (0.88) | $ | (0.94) | |||||||
基本和稀釋加權普通股份 | 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.聯繫人:
投資者:
Amy Figueroa,CFA
investor.relations@cariboubio.com
Media:
Peggy Vorwald, PhD
media@cariboubio.com
媒體:
Peggy Vorwald,PhD
media@cariboubio.com