Gamida Cell Reports Third Quarter 2021 Financial Results and Provides Company Update

Gamida Cell报告2021年第三季度财务业绩并提供公司更新

2021-11-15 22:00:06 BioSpace

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New data being presented at American Society of Hematology Annual Meeting demonstrating GDA-201 overall survival rate of 78% at two years with a median duration of response of 16 months and long-term clinical benefit of omidubicel with long-lasting hematopoietic recovery Finished third quarter of 2021 with $121 million in cash; reassessing expected spending and prior financial guidance due to the revised timing of the omidubicel BLA submission Company to host conference call at 8:00 a.m. ET today BOSTON--(BUSINESS WIRE)-- Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, today provided a business update and reported financial results for the quarter ended September 30, 2021. Net loss for the third quarter of 2021 was $19.6 million, compared to a net loss of $14.8 million for the same period in 2020. As of September 30, 2021, Gamida Cell had total cash and cash equivalents of $120.8 million. During the past quarter, Gamida Cell: Continued to execute on plans to submit a Biologic License Application (BLA) for omidubicel, a potentially life-saving treatment for patients with blood cancers in need of stem cell transplant. As previously disclosed, in a recent pre-BLA meeting, the FDA requested a revised analysis of the manufacturing data generated at Gamida Cell’s wholly owned commercial manufacturing facility to demonstrate the comparability to the omidubicel that was produced at the clinical manufacturing sites for the Phase 3 study. The FDA did not request additional clinical data to initiate the BLA submission once analytical comparability is demonstrated. Progressed activities with objective to address the FDA’s Clinical Hold on the Investigational New Drug (IND) application for GDA-201, which was imposed based on questions about donor eligibility procedures and sterility assay qualification prior to the initiation of the study in patients with follicular and diffuse large B-cell lymphomas. Expanded the company’s NAM-enabled natural killer (NK) cell pipeline targeting solid-tumor and hematological cancers, including genetically modified variants of proprietary NK therapies using both CRISPR/Cas9 and CAR methodologies. “We are committed to advance our programs and bring our important potential therapies to patients as quickly as possible. We are working diligently to respond to the FDA’s information requests for omidubicel and GDA-201, and now expect to submit the BLA for omidubicel to the FDA in the first half of 2022 and we hope to promptly address outstanding issues regarding our IND application relating to GDA-201.” said Julian Adams, Ph.D., chief executive officer of Gamida Cell. “Additionally, at our recent NK-focused R&D Day, we provided details on our genetically modified NK cell immunotherapy programs leveraging CAR- and CRISPR-mediated strategies against hematologic malignancies and solid tumors. The company remains focused on our goal of bringing patients with cancer potentially curative cell therapies.” Recent Developments and Planned Presentations at ASH Omidubicel: Advanced Cell Therapy BLA Submission: During a recent pre-BLA meeting, the FDA requested that Gamida Cell provide revised analysis of the manufacturing data generated at Gamida Cell’s wholly owned commercial manufacturing facility. Upon completing those requirements, the company anticipates submitting the BLA in the first half of 2022. New data to be presented at ASH: Gamida Cell will have three omidubicel presentations - two presentations of additional data from the phase III randomized trial of omidubicel, and a poster presentation summarizing long term omidubicel data from multiple studies - at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition (December 11-14, 2021). Oral presentation of “Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel is Associated with Robust Immune Reconstitution and Lower Rates of Severe Infection Compared to Standard Umbilical Cord Blood Transplantation” on Saturday, December 11, 2021, at 4:30 p.m. ET. Data collected from a subset of 37 patients in the omidubicel Phase III trial shows that, in addition to more rapid short-term hematopoietic recovery, omidubicel-treated patients had more rapid recovery of a wide variety of immune cells including CD4+ T cells, B cells, monocytes, natural killer cells, and dendritic cells. The robust recovery of the broad range of the immune system correlated with and supports clinical data showing fewer severe bacterial, fungal, and viral infections in patients treated with omidubicel. Poster presentation of “Hospitalization and Healthcare Resource Use of Omidubicel vs. Cord Blood Transplantation for Hematological Malignancies in a Global Randomized Phase III Clinical Trial” on Monday, December 13, 2021, 6:00-8:00 p.m. ET. Resource utilization data during the first 100 days after transplant were analyzed for 108 patients in the phase III trial and shows that omidubicel-treated patients has significantly shorter durations of hospitalization, intensive care unit time, consultant visits, procedures, and transfusions than the control arm. These data provide further evidence of the clinical benefit associated with the more rapid hematopoietic recovery in patients treated with omidubicel and the corresponding reduction in healthcare resource utilization. Poster presentation, “Allogeneic Stem Cell Transplantation with Omidubicel: Long-Term Follow-up from a Single Center” on Saturday, December 11, 2021, 5:30-7:30 p.m. ET. Analysis of outcomes of 22 patients with hematologic malignancies treated with omidubicel at Duke University over a 10-year period shows long-term sustained bone marrow function and immune recovery, with a 10-year overall survival of 48%. These data provide further support for the long-term clinical benefit of omidubicel with long-lasting hematopoietic recovery. Oral presentation of “Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel is Associated with Robust Immune Reconstitution and Lower Rates of Severe Infection Compared to Standard Umbilical Cord Blood Transplantation” on Saturday, December 11, 2021, at 4:30 p.m. ET. Data collected from a subset of 37 patients in the omidubicel Phase III trial shows that, in addition to more rapid short-term hematopoietic recovery, omidubicel-treated patients had more rapid recovery of a wide variety of immune cells including CD4+ T cells, B cells, monocytes, natural killer cells, and dendritic cells. The robust recovery of the broad range of the immune system correlated with and supports clinical data showing fewer severe bacterial, fungal, and viral infections in patients treated with omidubicel. Poster presentation of “Hospitalization and Healthcare Resource Use of Omidubicel vs. Cord Blood Transplantation for Hematological Malignancies in a Global Randomized Phase III Clinical Trial” on Monday, December 13, 2021, 6:00-8:00 p.m. ET. Resource utilization data during the first 100 days after transplant were analyzed for 108 patients in the phase III trial and shows that omidubicel-treated patients has significantly shorter durations of hospitalization, intensive care unit time, consultant visits, procedures, and transfusions than the control arm. These data provide further evidence of the clinical benefit associated with the more rapid hematopoietic recovery in patients treated with omidubicel and the corresponding reduction in healthcare resource utilization. Poster presentation, “Allogeneic Stem Cell Transplantation with Omidubicel: Long-Term Follow-up from a Single Center” on Saturday, December 11, 2021, 5:30-7:30 p.m. ET. Analysis of outcomes of 22 patients with hematologic malignancies treated with omidubicel at Duke University over a 10-year period shows long-term sustained bone marrow function and immune recovery, with a 10-year overall survival of 48%. These data provide further support for the long-term clinical benefit of omidubicel with long-lasting hematopoietic recovery. GDA-201: NAM-Enabled NK Cell Therapy IND for Phase 1/2 Study: Gamida Cell is working to address the clinical hold on the IND for a Phase 1/2 study of GDA-201. As a result of the clinical hold, the initiation of our planned Phase 1/2 study of GDA-201 will be delayed beyond the end of 2021, as the company previously projected. New data presented at SITC: Gamida Cell recently presented promising new preclinical data in two posters characterizing the NAM-enabled mechanisms of action that contribute to the metabolic modulation properties and enhanced tumor cytotoxicity activity of GDA-201 at the Society for Immunotherapy of Cancer’s 36th Annual Meeting (SITC 2021) held from November 10-14, 2021. New data to be presented at ASH: A poster titled “GDA-201, A Novel Metabolically Enhanced Allogeneic Natural Killer (NK) Cell Product Yields High Remission Rates in Patients with Relapsed/Refractory Non-Hodgkin Lymphoma (NHL): 2-year survival and correlation with cytokine IL7” will be presented at the upcoming ASH Annual Meeting and Exposition on Monday, December 13, 2021, 6:00-8:00 p.m. ET. This analysis provides longer follow-up in the investigator-led study of GDA-201 in patients with non-Hodgkin lymphoma and demonstrated an overall survival rate of 78% at two years, median duration of response of 16 months, and a safety profile that was similar to what had been previously reported. NAM-Enabled NK Cell Pipeline Expansion Advanced NAM-enabled genetically modified NK pipeline: During Gamida Cell’s NK-focused virtual R&D Day, the company presented new data and additional details on its genetically modified NK cell immunotherapy programs, which utilize CAR, membrane bound- and CRISPR-mediated strategies to increase targeting, potency and persistence against hematologic malignancies and solid tumors: GDA-301: Knockout of CISH (cytokine inducible SH2 containing protein) in NK cells using CRISPR/Cas9 in combination with a membrane-bound IL-15/IL-15Ra; GDA-501: anti HER2 CAR-engineered NK cells to target solid tumors expressing HER2, based on a single-chain variable fragment of the widely used humanized monoclonal antibody trastuzumab; and GDA-601: CRISPR Knockout of CD38 on NK cells combined with anti CD38 CAR. CD38 is an established immunotherapeutic target in multiple myeloma, but its expression on NK cells and its further induction during ex vivo NK cell expansion represents a barrier to the development of an anti CD38 CAR-NK cell therapy. Gamida Cell recently announced a research collaboration with the Dana-Farber Cancer Institute to study the in vitro cytotoxicity of GDA-601 in fresh samples from multiple myeloma patients. GDA-301: Knockout of CISH (cytokine inducible SH2 containing protein) in NK cells using CRISPR/Cas9 in combination with a membrane-bound IL-15/IL-15Ra; GDA-501: anti HER2 CAR-engineered NK cells to target solid tumors expressing HER2, based on a single-chain variable fragment of the widely used humanized monoclonal antibody trastuzumab; and GDA-601: CRISPR Knockout of CD38 on NK cells combined with anti CD38 CAR. CD38 is an established immunotherapeutic target in multiple myeloma, but its expression on NK cells and its further induction during ex vivo NK cell expansion represents a barrier to the development of an anti CD38 CAR-NK cell therapy. Gamida Cell recently announced a research collaboration with the Dana-Farber Cancer Institute to study the in vitro cytotoxicity of GDA-601 in fresh samples from multiple myeloma patients. New data presented at PEGS Europe: Data from early-stage studies of GDA-501 demonstrated enhanced potency and cytotoxicity against a HER2-expressing tumor cell line. Data presented on GDA-301 showed cytotoxic activity against a chronic myelogenous leukemia cell line (K562) and a multiple myeloma cell line (RPMI). These data were presented at the 13th Annual Protein and Antibody Engineering Summit (PEGS) in Barcelona, Spain November 2-4, 2021. Third Quarter 2021 Financial Results Research and development expenses in the third quarter of 2021 were $12.4 million, compared to $10.5 million for the same period in 2020. The increase was mainly due to omidubicel commercial manufacturing readiness activities, and the advancement of the GDA-201 program, including broadening scientific capabilities and talent. Commercial expenses in the third quarter of 2021 were $6.0 million, compared to $1.9 million for the third quarter of 2020. The increase was mainly attributed to progress with omidubicel commercial readiness activities. Going forward, the company anticipates reducing its near-term commercial readiness expenses in line with the revised omidubicel BLA submission timing. General and administrative expenses were $4.8 million for the third quarter of 2021, compared to $2.7 million for the same period in 2020. The increase was mainly due to professional services and the hiring of key management positions, to support business growth. Finance income, net, was $3.5 million for the third quarter of 2021, compared to $0.3 million for the third quarter of 2020. The increase was primarily due to non-cash income, resulting from revaluation of warrants offset by convertible note interest expenses. Net loss for the third quarter of 2021 was $19.6 million, compared to a net loss of $14.8 million for the same period in 2020. 2021 Financial Guidance Gamida Cell is re-assessing its planned spending and prior financial guidance as a result of the revised timing of the expected omidubicel BLA submission. Expected Milestones in 2022 Omidubicel BLA submission to the FDA in the first half of 2022 GDA-201 Initiation of a company-sponsored Phase 1/2 clinical study in NHL in 2022 NK cell pipeline expansion Establish preclinical proof of concept studies of the NAM-enabled, genetically modified NK therapeutic targets in 2022 Select pipeline candidate(s) for IND enabling studies by end of 2022 Conference Call Information Gamida Cell will host a conference call today, November 15, 2021, at 8:00 a.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the “Investors & Media” section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 4347485. A recording of the webcast will be available approximately two hours after the event, for approximately 30 days. About Omidubicel Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with blood cancers. Omidubicel is the first bone marrow transplant graft to receive Breakthrough Therapy Designation from the U.S. FDA and has also received Orphan Drug Designation in the U.S. and EU. For more information about omidubicel, please visit https://www.gamida-cell.com. Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority. About GDA-201 Gamida Cell applied the capabilities of its nicotinamide (NAM)-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells. For more information about GDA-201, please visit https://www.gamida-cell.com. GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority. About Gamida Cell Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapies for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapies with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx. Cautionary Note Regarding Forward Looking Statements This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of initiation and progress of, and data reported from, the clinical trials of Gamida Cell’s product candidates (including GDA-201), anticipated regulatory filings (including the timing of submission of the BLA for omidubicel to the FDA), commercialization planning efforts, and the potentially life-saving or curative therapeutic and commercial potential of omidubicel, and Gamida Cell’s expectations for the expected clinical development milestones set forth herein.. Any statement describing Gamida Cell’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including those related to the impact that the COVID-19 pandemic could have on our business, and including the scope, progress and expansion of Gamida Cell’s clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cell’s Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on March 9, 2021, as amended, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cell’s actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cell’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements. INTERIM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION U.S. dollars in thousands September 30, December 31, 2021 2020 2020 Unaudited Audited ASSETS CURRENT ASSETS: Cash and cash equivalents $ 80,613 $ 73,311 $ 127,170 Marketable securities 40,223 - - Prepaid expenses and other current assets 2,785 1,734 2,815 Total current assets 123,621 75,045 129,985 NON-CURRENT ASSETS: Property, plant and equipment, net 30,023 15,838 18,238 Right-of-use assets 4,918 7,023 6,474 Other assets 6,599 802 786 Total non-current assets 41,540 23,663 25,498 Total assets $ 165,161 $ 98,708 $ 155,483 INTERIM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION U.S. dollars in thousands (except share and per share data) September 30, December 31, 2021 2020 2020 Unaudited Audited LIABILITIES AND EQUITY CURRENT LIABILITIES: Trade payables $ 7,833 $ 2,704 $ 6,329 Employees and payroll accruals 5,870 3,872 4,705 Current maturities of lease liabilities 1,622 2,345 2,532 Accrued interest 525 - - Accrued expenses and other payables 7,810 5,005 7,988 Total current liabilities 23,660 13,926 21,554 NON-CURRENT LIABILITIES: Liabilities presented at fair value - 3,252 12,043 Employee benefit liabilities, net 768 773 768 Other long-term liabilities 4,621 5,460 5,378 Liability to Israel Innovation Authority 20,858 14,729 17,003 Convertible senior notes, net 69,298 - - Total non-current liabilities 95,545 24,214 35,192 SHAREHOLDERS' EQUITY: Share capital - Ordinary shares of NIS 0.01 par value - Authorized: 100,000,000 shares at September 30, 2021 and 2020 (unaudited) and December 31, 2020; Issued and outstanding: 59,298,846 and 49,556,663 shares at September 30, 2021 and 2020 (unaudited), respectively and 59,000,153 shares at December 31, 2020. 167 138 166 Share premium 381,504 304,944 375,280 Capital reserve (441) (541) (441) Reserve from financial assets measured at FVOCI (42) - - Accumulated deficit (335, 232) (243,973) (276,268) Total shareholders' equity 45,956 60,568 98,737 Total liabilities and shareholders' equity $ 165,161 $ 98,708 $ 155,483 INTERIM CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS U.S. dollars in thousands (except share and per share data) Nine months ended September 30, Three months ended September 30, Year ended December 31, 2021 2020 2021 2020 2020 Unaudited Unaudited Audited Operating expenses: Research and development, net $ 37,213 $ 27,652 $ 12,396 $ 10,454 $ 41,385 Commercial activities 15,633 4,413 5,973 1,916 8,748 General and administrative 12,004 8,180 4,774 2,690 12,167 Operating loss 64,850 40,245 23,143 15,060 62,300 Finance expense 6,330 2,367 2,218 1,001 10,640 Finance income (11,769) (2,203) (5,727) (1,309) (236) Loss before tax benefit 59,411 40,409 19,634 14,752 72,704 Tax benefit (447) - - - - Net loss 58,964 40,409 19,634 14,752 72,704 Other comprehensive loss: Items that will be reclassified subsequently to profit or loss: Actuarial net gain of defined benefit plans - - - - (100) Changes in the fair value of marketable securities 42 4 17 - 4 Total comprehensive loss $ 59,006 $ 40,413 $ 19,651 $ 14,752 $ 72,608 Net loss per share: Basic loss per share $ 1.00 $ 0.98 $ 0.33 $ 0.30 $ 1.66 Diluted loss per share $ 1.18 $ 0.98 $ 0.33 $ 0.30 $ 1.66 Weighted average share count 59,219,757 41,281,970 59,281,243 49,472,749 43,725,584 U.S. dollars in thousands Nine months ended September 30, Three months ended September 30, Year ended December 31, 2021 2020 2021 2020 2020 Unaudited Unaudited Audited Cash flows from operating activities: Net loss $ (58,964) $ (40,409) $ (19,634) $ (14,752) $ (72,704) Adjustments to reconcile net loss to net cash used in operating activities: Adjustments to the profit or loss items: Depreciation of property, plant and equipment and right-of-use assets 1,898 1,716 621 610 2,397 Financial (income) expense, net 1,613 (169) 606 91 483 Share-based compensation 3,976 1,969 1,513 748 2,864 Change in employee benefit liabilities, net - - - - 94 Amortization of premium on available-for-sale financial assets - 4 - - 4 Revaluation of liabilities presented at fair value derivatives (11,257) (1,969) (5,447) (1,299) 6,822 Revaluation of liability to IIA 3,170 2,227 1,312 912 4,302 Deferred income taxes (447) - - - (1,047) 3,778 (1,395) 1,062 16,966 Changes in asset and liability items: Decrease (increase) in prepaid expenses, other current assets, and other assets 1,005 (718) 937 347 (1,626) Increase (decrease) in trade payables 1,504 1,535 2,397 (39) 5,083 Increase (decrease) in accrued expenses and other payables (894) 516 (693) 1,141 3,454 1,615 1,333 2,641 1,449 6,911 Cash received during the period for: Interest received 1,122 359 854 2 361 Interest paid (128) (120) (43) (40) (161) 994 239 811 (38) 200 Net cash used in operating activities (57,402) (35,059) (17,577) (12,279) (48,627) Cash flows from investing activities: Purchase of property, plant and equipment (9,577) (9,792) (4,187) (2,683) (11,804) Investment in long-term deposit (5,803) - (4,803) - - Purchase of marketable securities (97,808) - (29,657) - - Investment in restricted bank deposits - - - - (158) Proceeds from maturity of marketable securities 56,717 - 38,893 - - Proceeds from sale of marketable securities - 13,551 - - 13,551 Net cash provided by (used in) investing activities $ (56,471) $ 3,759 $ 246 $ (2,683) $ 1,589 INTERIM CONSOLIDATED STATEMENTS OF CASH FLOWS U.S. dollars in thousands Nine months ended September 30 Three months ended September 30 Year ended December 31, 2021 2020 2021 2020 2020 Unaudited Unaudited Audited Cash flows from financing activities: Proceeds from secondary offering, net - - - - 133,316 Receipt of grants from the IIA 311 200 259 - 399 Proceeds from secondary offering, net - 63,860 - - - Proceeds from issuance of convertible senior notes, net of issuance costs 70,777 - - - - Payment of lease liabilities (1,782) (1,539) (653) (417) (1,985) Payment of interest of Convertible senior notes (2,191) - (2,191) - - Exercise of options 566 169 10 21 650 Payment of issuance costs related to public offering (468) - - - - Net cash provided by (used in) financing activities 67,213 62,690 (2,575) (396) 132,380 Exchange differences on balances of cash and cash equivalents 103 83 29 31 (10) Increase (decrease) in cash and cash equivalents (46,557) 31,473 (19,877) (15,327) 85,332 Cash and cash equivalents at beginning of period 127,170 41,838 100,490 88,638 41,838 Cash and cash equivalents at end of period $ 80,613 $ 73,311 $ 80,613 $ 73,311 $ 127,170 Supplemental disclosure of non-cash financing activities: Significant non-cash transactions: Lease liabilities arising from new right- of-use asset $ - $ 3,376 $ - $ - $ 3,409 IIA liability for grants to be received $ 590 $ - $ 590 $ - $ 103 Issuance expenses on credit $ - $ - $ - $ - $ 468 Purchase of property, plant and equipment on credit $ 1,561 $ - $ 1,561 $ - $ 415 Borrowing costs capitalization $ 1,287 $ - $ 713 $ - $ - View source version on businesswire.com: https://www.businesswire.com/news/home/20211115005479/en/ For investors: Courtney Turiano Stern Investor Relations, Inc. Courtney.Turiano@sternir.com 1-212-362-1200 For media: Rhiannon Jeselonis Ten Bridge Communications rhiannon@tenbridgecommunications.com 1-978-417-1946 Source: Gamida Cell Ltd. View this news release online at: http://www.businesswire.com/news/home/20211115005479/en
美国血液学会年会上公布的新数据显示,GDA-201两年的总存活率为78%,中位反应持续时间为16个月,奥米杜比克的长期临床益处与持久的造血恢复 2021年第三季度以1.21亿美元现金结束;由于奥米杜比克BLA提交时间的修订,重新评估预期支出和先前的财务指导 公司将于美国东部时间今天上午8点召开电话会议 波士顿--(商业网)--致力于治疗癌症和其他严重疾病的先进细胞治疗公司Gamida Cell Ltd.(纳斯达克代码:GMDA)今天提供了业务更新,并报告了截至2021年9月30日的季度财务业绩。2021年第三季度净亏损为1960万美元,而2020年同期净亏损为1480万美元。截至2021年9月30日,Gamida Cell的现金及现金等价物总额为1.208亿美元。 在过去的一个季度里,Gamida Cell: 继续执行为奥米杜比克提交生物许可申请(BLA)的计划,奥米杜比克是一种潜在的拯救生命的治疗方法,用于需要干细胞移植的血癌患者。如前所述,在最近的一次BLA前会议上,FDA要求对Gamida Cell全资拥有的商业制造设施产生的制造数据进行修订分析,以证明与第三阶段研究临床制造场所生产的奥米杜比克的可比性。一旦分析可比性被证明,FDA没有要求额外的临床数据来启动BLA提交。 目的:解决FDA对GDA-201的研究性新药(IND)申请的临床搁置,该搁置是基于滤泡性和弥漫性大B细胞淋巴瘤患者的研究开始前关于供体资格程序和无菌试验资格的问题而实施的。 扩展了该公司针对实体肿瘤和血液系统癌症的NAM支持的自然杀伤(NK)细胞管道,包括使用CRISPR/Cas9和CAR方法的专利NK治疗的转基因变体。 “我们致力于推进我们的项目,并尽快将我们重要的潜在疗法带给患者。我们正在努力回应美国食品和药物管理局对奥米杜比塞尔和GDA-201的信息要求,现在预计将于2022年上半年向美国食品和药物管理局提交奥米杜比塞尔的BLA,我们希望迅速解决我们与GDA-201有关的IND申请的未决问题。“Gamida Cell首席执行官朱利安·亚当斯博士说。“此外,在我们最近以NK为重点的研发日上,我们提供了利用CAR和CRISPR介导的策略对抗血液恶性肿瘤和实体瘤的转基因NK细胞免疫治疗项目的细节。该公司仍然专注于我们的目标,即为癌症患者带来潜在的治愈细胞疗法。“ 最近的发展和计划在ASH上的发言 奥米比西:高级细胞疗法 BLA提交:在最近的一次BLA前会议上,FDA要求Gamida Cell提供对Gamida Cell全资拥有的商业制造设施产生的制造数据的修订分析。在完成这些要求后,该公司预计将在2022年上半年提交BLA。 将在ASH上展示的新数据:在第63届美国血液学会(ASH)年会和博览会(2021年12月11日至14日)上,Gamida Cell将有三次奥米杜比塞尔展示--两次展示奥米杜比塞尔III期随机试验的额外数据,以及一次总结多项研究长期奥米杜比塞尔数据的海报展示。 2021年12月11日星期六,美国东部时间下午4:30口头介绍“与标准脐带血移植相比,使用奥米杜比克的造血干细胞移植(HSCT)与强大的免疫重建和较低的严重感染率有关”。在奥米比克III期试验中收集的37名患者的数据显示,除了更快的短期造血恢复外,接受奥米比克治疗的患者更快地恢复了多种免疫细胞,包括CD4+T细胞、B细胞、单核细胞、自然杀伤细胞和树突状细胞。广泛免疫系统的强劲恢复与临床数据相关,并支持使用奥米比克治疗的患者较少严重的细菌、真菌和病毒感染。 2021年12月13日星期一,美国东部时间下午6:00-8:00,“在一项全球随机三期临床试验中,奥米杜比克与脐带血移植治疗血液系统恶性肿瘤的住院和医疗资源使用”的海报展示。对III期试验中108例患者移植后前100天的资源利用数据进行了分析,结果表明,奥米比克治疗的患者住院时间、重症监护室时间、咨询就诊时间、手术时间和输血时间明显短于对照组。这些数据提供了进一步的证据,证明了在使用奥米比克治疗的患者中,更快的造血恢复和相应的医疗资源利用减少相关的临床益处。 海报展示,“奥米杜比克异基因干细胞移植:来自单个中心的长期随访”,2021年12月11日星期六,美国东部时间下午5:30-7:30。杜克大学对22例血液系统恶性肿瘤患者10年来接受奥米比克治疗的结果进行分析,显示长期持续的骨髓功能和免疫恢复,10年总生存率为48%。这些数据为奥米杜比克长期临床获益和持久造血恢复提供了进一步的支持。 2021年12月11日星期六,美国东部时间下午4:30口头介绍“与标准脐带血移植相比,使用奥米杜比克的造血干细胞移植(HSCT)与强大的免疫重建和较低的严重感染率有关”。在奥米比克III期试验中收集的37名患者的数据显示,除了更快的短期造血恢复外,接受奥米比克治疗的患者更快地恢复了多种免疫细胞,包括CD4+T细胞、B细胞、单核细胞、自然杀伤细胞和树突状细胞。广泛免疫系统的强劲恢复与临床数据相关,并支持使用奥米比克治疗的患者较少严重的细菌、真菌和病毒感染。 2021年12月13日星期一,美国东部时间下午6:00-8:00,“在一项全球随机三期临床试验中,奥米杜比克与脐带血移植治疗血液系统恶性肿瘤的住院和医疗资源使用”的海报展示。对III期试验中108例患者移植后前100天的资源利用数据进行了分析,结果表明,奥米比克治疗的患者住院时间、重症监护室时间、咨询就诊时间、手术时间和输血时间明显短于对照组。这些数据提供了进一步的证据,证明了在使用奥米比克治疗的患者中,更快的造血恢复和相应的医疗资源利用减少相关的临床益处。 海报展示,“奥米杜比克异基因干细胞移植:来自单个中心的长期随访”,2021年12月11日星期六,美国东部时间下午5:30-7:30。杜克大学对22例血液系统恶性肿瘤患者10年来接受奥米比克治疗的结果进行分析,显示长期持续的骨髓功能和免疫恢复,10年总生存率为48%。这些数据为奥米杜比克长期临床获益和持久造血恢复提供了进一步的支持。 GDA-201:NAM激活的NK细胞治疗 第1/2期研究的IND:Gamida Cell正在致力于解决GDA-201第1/2期研究的IND的临床问题。由于临床搁置,我们计划的GDA-201第1/2期研究的启动将推迟到2021年底以后,正如该公司此前预测的那样。 在SITC上公布的新数据:在2021年11月10日至14日举行的癌症免疫治疗学会第36届年会上,Gamida Cell最近在两张海报中公布了有希望的新临床前数据,描述了NAM使能的作用机制,这些机制有助于代谢调节特性和增强GDA-201的肿瘤细胞毒性活性。 将在ASH上公布的新数据:美国东部时间2021年12月13日星期一下午6:00-8:00即将举行的ASH年会和博览会上将公布一张题为“GDA-201,一种新的代谢增强异体自然杀伤(NK)细胞产品,在复发/难治性非霍奇金淋巴瘤(NHL)患者中产生高缓解率:2年生存和与细胞因子IL7的相关性”的海报。这一分析在研究者领导的非霍奇金淋巴瘤患者的GDA-201研究中提供了更长的随访,并显示两年的总存活率为78%,中位反应持续时间为16个月,安全性与以前报道的相似。 NAM使能的NK细胞流水线扩展 先进的NAM支持的转基因NK管道:在Gamida cell专注于NK的虚拟研发日期间,该公司展示了其转基因NK细胞免疫治疗计划的新数据和额外细节,该计划利用CAR、膜结合和CRISPR介导的策略来提高针对血液系统恶性肿瘤和实体肿瘤的靶向性、效力和持久性: GDA-301:用CRISPR/Cas9联合膜结合IL-15/IL-15Ra敲除NK细胞中CISH(细胞因子诱导的含SH2蛋白); GDA-501:基于广泛使用的人源化单克隆抗体曲妥珠单抗单链可变片段的抗HER2 CAR工程NK细胞靶向表达HER2的实体瘤;和 GDA-601:CRISPR基因敲除NK细胞CD38,联合抗CD38CAR。CD38在多发性骨髓瘤中是一个公认的免疫治疗靶点,但其在NK细胞上的表达及其在体外NK细胞增殖过程中的进一步诱导阻碍了抗CD38 CAR-NK细胞治疗的发展。Gamida Cell最近宣布与Dana-Farber癌症研究所合作研究GDA-601在多发性骨髓瘤患者新鲜样本中的体外细胞毒性。 GDA-301:用CRISPR/Cas9联合膜结合IL-15/IL-15Ra敲除NK细胞中CISH(细胞因子诱导的含SH2蛋白); GDA-501:基于广泛使用的人源化单克隆抗体曲妥珠单抗单链可变片段的抗HER2 CAR工程NK细胞靶向表达HER2的实体瘤;和 GDA-601:CRISPR基因敲除NK细胞CD38,联合抗CD38CAR。CD38在多发性骨髓瘤中是一个公认的免疫治疗靶点,但其在NK细胞上的表达及其在体外NK细胞增殖过程中的进一步诱导阻碍了抗CD38 CAR-NK细胞治疗的发展。Gamida Cell最近宣布与Dana-Farber癌症研究所合作研究GDA-601在多发性骨髓瘤患者新鲜样本中的体外细胞毒性。 PEGS欧洲会议上公布的新数据:来自早期阶段GDA-501研究的数据显示,GDA-501增强了对HER2表达肿瘤细胞系的效力和细胞毒性。有关GDA-301的数据显示了对慢性粒细胞白血病细胞系(K562)和多发性骨髓瘤细胞系(RPMI)的细胞毒活性。这些数据于2021年11月2日至4日在西班牙巴塞罗那举行的第13届年度蛋白质和抗体工程峰会(PEGS)上公布。 2021年第三季度财务业绩 2021年第三季度的研发费用为1240万美元,而2020年同期为1050万美元。增长主要是由于奥米杜比克商业制造准备活动,以及GDA-201计划的推进,包括扩大科学能力和人才。 2021年第三季度的商业支出为600万美元,而2020年第三季度为190万美元。增加主要归因于奥米杜比克商业准备活动的进展。展望未来,该公司预计将根据修订后的奥米杜比克BLA提交时间减少其近期商业准备费用。 2021年第三季度的一般和行政费用为480万美元,而2020年同期为270万美元。增加主要由于专业服务及聘用主要管理职位,以支持业务增长。 2021年第三季度财务净收入为350万美元,而2020年第三季度为30万美元。增加主要由于认股权证重估所产生的非现金收入被可换股票据利息开支抵销。 2021年第三季度净亏损为1960万美元,而2020年同期净亏损为1480万美元。 2021财政指导 Gamida Cell正在重新评估其计划支出和先前的财务指导,因为预期奥米杜比克BLA提交的时间已经修订。 2022年预期里程碑 奥米杜比塞尔 2022年上半年向FDA提交的BLA GDA-201 2022年在NHL启动一项由公司赞助的1/2期临床研究 NK细胞管道扩展 在2022年建立NAM支持的转基因NK治疗靶点的临床前概念验证研究 在2022年底前为IND启动研究选择管道候选项目 电话会议信息 Gamida Cell将于美国东部时间2021年11月15日上午8点主持电话会议,讨论这些财务业绩和公司更新。电话会议的网络直播可在Gamida Cell网站www.gamida-cell.com的“投资者和媒体”部分访问。参加现场通话,请拨打866-930-5560(国内)或409-216-0605(国际),并参考会议身份证号4347485。网播录音将在活动结束后约两小时提供,持续约30天。 关于奥米杜比塞尔 奥米比克是一种先进的细胞疗法,正在开发中,作为一种潜在的挽救血癌患者生命的异基因造血干细胞(骨髓)移植液。奥米比克是第一个获得美国FDA突破性疗法指定的骨髓移植移植物,也获得了美国和欧盟的孤儿药指定。欲了解更多关于奥米杜比克的信息,请访问https://www.gamida-cell.com。 奥米比克是一种研究性疗法,其安全性和有效性尚未得到FDA或任何其他卫生当局的证实。 关于GDA-201 Gamida Cell应用其烟酰胺(NAM)支持的细胞扩增技术开发了GDA-201,这是一种先天的NK细胞免疫疗法,用于治疗血液和实体瘤,并结合标准护理抗体疗法。GDA-201是NAM激活的NK细胞管道中的主要候选材料,已经展示了有希望的初步临床试验结果。GDA-201通过增加细胞毒性和在骨髓和淋巴器官中的体内滞留和增殖来解决NK细胞的关键局限性。此外,GDA-201提高了抗体依赖性细胞毒性(ADCC)和NK细胞的肿瘤靶向性。有关GDA-201的更多信息,请访问https://www.gamida-cell.com。 GDA-201是一种研究性疗法,其安全性和有效性尚未得到FDA或任何其他卫生当局的证实。 关于Gamida细胞 Gamida Cell正在开创一个多样化的免疫治疗管道,为实体瘤、血癌和其他严重血液病患者提供潜在的治愈细胞疗法。我们应用一个专有的扩展平台,利用NAM的特性,用于异基因细胞来源,包括脐带血源细胞和NK细胞,以创造有可能重新定义护理标准的治疗。其中包括omidubicel,一种有潜力作为需要骨髓移植的患者的救命替代品的研究产品,以及一系列针对实体瘤和血液系统恶性肿瘤的修饰和未修饰的NAM使能的NK细胞。欲了解更多信息,请访问www.gamida-cell.com,或在LinkedIn、Twitter、Facebook或Instagram关注Gamida Cell@gamidaCelltx。 关于前瞻性陈述的警告说明 本新闻稿包含1995年《私人证券诉讼改革法》中定义的前瞻性陈述,包括Gamida Cell候选产品(包括GDA-201)临床试验的启动和进展时间以及报告的数据,预期的监管文件(包括向FDA提交奥米杜比克BLA的时间),商业化规划工作,奥米杜比克潜在的救命或治疗性治疗和商业潜力,以及Gamida Cell对本文所述预期临床发展里程碑的预期。任何描述Gamida Cell的目标、期望、财务或其他预测、意图或信念的声明都是前瞻性声明,应被视为风险声明。该等陈述受制于多项风险、不确定性及假设,包括与新冠肺炎疫情可能对我们业务造成的影响有关的风险、不确定性及假设,以及Gamida Cell临床试验的范围、进展及扩展及其成本的影响;临床、科学、法规和技术发展;以及在开发和商业化作为人类疗法使用的安全有效的候选产品的过程中,以及在围绕这些候选产品建立业务的努力中所固有的。鉴于这些风险和不确定性,以及Gamida Cell于2021年3月9日向美国证券交易委员会(SEC)提交的经修订的年度报告表格20-F中的风险因素部分和其他部分以及Gamida Cell不时向美国证券交易委员会(SEC)提交的其他文件(可在http://www.SEC.gov查阅)中描述的其他风险和不确定性,该等前瞻性陈述中讨论的事件和情况可能不会发生,Gamida Cell的实际结果可能与由此预期或暗示的情况存在重大和不利的差异。虽然Gamida Cell的前瞻性陈述反映了其管理层的诚信判断,但这些陈述仅基于Gamida Cell目前已知的事实和因素。因此,警告您不要依赖这些前瞻性陈述。 中期综合财务状况表 千美元 9月30日, 12月31日, 2021 2020 2020 未经审计 审计的 资产 流动资产: 现金及现金等价物 $ 80,613 $ 73,311 $ 127,170 有价证券 40,223 - - 待摊费用和其他流动资产 2,785 1,734 2,815 流动资产总额 123,621 75,045 129,985 非流动资产: 不动产、厂场和设备,净额 30,023 15,838 18,238 使用权资产 4,918 7,023 6,474 其他资产 6,599 802 786 非流动资产共计 41,540 23,663 25,498 总资产 $ 165,161 $ 98,708 $ 155,483 中期综合财务状况表 以千美元计(股份和每股数据除外) 9月30日, 12月31日, 2021 2020 2020 未经审计 审计的 负债和权益 流动负债: 贸易应付款项 $ 7,833 $ 2,704 $ 6,329 雇员和应计薪金 5,870 3,872 4,705 租赁负债的当前到期日 1,622 2,345 2,532 应计利息 525 - - 应计费用和其他应付款 7,810 5,005 7,988 流动负债共计 23,660 13,926 21,554 非流动负债: 按公允价值列报的负债 - 3,252 12,043 雇员福利负债,净额 768 773 768 其他长期负债 4,621 5,460 5,378 对以色列创新局的责任 20,858 14,729 17,003 可换股优先票据净额 69,298 - - 非流动负债共计 95,545 24,214 35,192 股东权益: 股本- 面值0.01新谢克尔的普通股- 授权:于2021年9月30日100,000,000股股份 及2020年(未经审计)及2020年12月31日;发布和 未偿还:59,298,846股及49,556,663股于 分别为2021年9月30日、2020年9月30日(未经审计) 及于2020年12月31日持有59,000,153股股份。 167 138 166 股份溢价 381,504 304,944 375,280 资本公积 (441) (541) (441) 以FVOCI计量的金融资产准备金 (42) - - 累计赤字 (335,232) (243,973) (276,268) 股东权益总额 45,956 60,568 98,737 负债和股东权益合计 $ 165,161 $ 98,708 $ 155,483 中期综合全面亏损表 以千美元计(股份和每股数据除外) 九个月结束 9月30日, 三个月结束 9月30日, 终了年度 12月31日, 2021 2020 2021 2020 2020 未经审计 未经审计 审计的 业务费用: 网络研究与开发 $ 37,213 $ 27,652 $ 12,396 $ 10,454 $ 41,385 商业活动 15,633 4,413 5,973 1,916 8,748 一般和行政 12,004 8,180 4,774 2,690 12,167 营业损失 64,850 40,245 23,143 15,060 62,300 财务费用 6,330 2,367 2,218 1,001 10,640 财政收入 (11,769) (2,203) (5,727) (1,309) (236) 税前损失优惠 59,411 40,409 19,634 14,752 72,704 税收优惠 (447) - - - - 净损失 58,964 40,409 19,634 14,752 72,704 其他综合损失: 将重新分类的项目 损益之后: 设定受益计划精算净收益 - - - - (100) 有价证券公允价值变动 42 4 17 - 4 总综合损失 $ 59,006 $ 40,413 $ 19,651 $ 14,752 $ 72,608 每股净亏损: 每股基本损失 $ 1.00 $ 0.98 $ 0.33 $ 0.30 $ 1.66 每股摊薄亏损 $ 1.18 $ 0.98 $ 0.33 $ 0.30 $ 1.66 加权平均股份数 59,219,757 41,281,970 59,281,243 49,472,749 43,725,584 千美元 九个月结束 9月30日, 三个月结束 9月30日, 终了年度 12月31日, 2021 2020 2021 2020 2020 未经审计 未经审计 审计的 经营活动产生的现金流量: 净损失 $ (58,964) $ (40,409) $ (19,634) $ (14,752) $ (72,704) 调整净亏损与现金净额 在经营活动中使用的: 损益项目的调整: 不动产、厂场和设备折旧 和使用权资产 1,898 1,716 621 610 2,397 财务(收入)费用,净额 1,613 (169) 606 91 483 以股份为基础的薪酬 3,976 1,969 1,513 748 2,864 雇员福利负债变动净额 - - - - 94 可供出售的溢价摊销 金融资产 - 4 - - 4 按公允价值列报的负债的重估 衍生品 (11,257) (1969) (5,447) (1,299) 6,822 对国际投资协定负债的重估 3,170 2,227 1,312 912 4,302 递延所得税 (447) - - - (1,047) 3,778 (1,395) 1,062 16,966 资产负债项目变动: 预付费用减少(增加)额、其他 流动资产及其他资产 1,005 (718) 937 347 (1,626) 贸易应付款项增加(减少) 1,504 1,535 2,397 (39) 5,083 应计费用增加(减少)额和 其他应付款项 (894) 516 (693) 1,141 3,454 1,615 1,333 2,641 1,449 6,911 本期间收到的现金如下: 收到的利息 1,122 359 854 2 361 已付利息 (128) (120) (43) (40) (161) 994 239 811 (38) 200 经营活动中使用的现金净额 (57,402) (35,059) (17,577) (12,279) (48,627) 投资活动产生的现金流量: 购置不动产、厂场和设备 (9,577) (9,792) (4,187) (2,683) (11,804) 长期存款投资 (5,803) - (4,803) - - 有价证券购买 (97,808) - (29,657) - - 受限制银行存款的投资 - - - - (158) 有价证券到期收益 56,717 - 38,893 - - 出售有价证券所得收益 - 13,551 - - 13,551 (用于)投资提供的现金净额 活动 $ (56,471) $ 3,759 $ 246 $ (2,683) $ 1,589 中期合并现金流量表 千美元 九个月结束 9月30日 三个月结束 9月30日 终了年度 12月31日, 2021 2020 2021 2020 2020 未经审计 未经审计 审计的 筹资活动产生的现金流量: 第二次发售所得款项净额 - - - - 133,316 从IIA收到赠款 311 200 259 - 399 第二次发售所得款项净额 - 63,860 - - - 发行可换股股票所得款项 优先票据,减去发行费用后的净额 70,777 - - - - 租赁负债的支付 (1,782) (1,539) (653) (417) (1,985) 可转换债券利息的支付 优先票据 (2,191) - (2,191) - - 期权的行使 566 169 10 21 650 支付相关发行费用 公开发售 (468) - - - - (用于)融资提供的现金净额 活动 67,213 62,690 (2,575) (396) 132,380 的余额汇兑差额 现金及现金等价物 103 83 29 31 (10) 现金和现金增(减)额 等价物 (46,557) 31,473 (19,877) (15,327) 85,332 期初现金及现金等价物 周期的 127,170 41,838 100,490 88,638 41,838 期末现金及现金等价物 $ 80,613 $ 73,311 $ 80,613 $ 73,311 $ 127,170 非现金补充披露 筹资活动: 重大非现金交易: 因新权利而产生的租赁负债- 待用资产 $ - $ 3,376 $ - $ - $ 3,409 IIA应收到赠款的负债 $ 590 $ - $ 590 $ - $ 103 赊销发行费用 $ - $ - $ - $ - $ 468 购置物业、厂房及 赊销设备 $ 1,561 $ - $ 1,561 $ - $ 415 借款费用资本化 $ 1,287 $ - $ 713 $ - $ - 在businesswire.com查看源代码版本:https://www.businesswire.com/news/home/20211115005479/en/ 对于投资者: 考特尼·图里亚诺 斯特恩投资者关系公司。 @sternir.com 1-212-362-1200 对于媒体: 里安农·杰塞洛尼斯 十桥通信 @tenbridgecommunications.com 1-978-417-1946 资料来源:Gamida细胞有限公司。 在网上查看此新闻稿: http://www.businesswire.com/news/home/20211115005479/en

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