Birinapant

STOCKHOLM, Feb. 15, 2024 /PRNewswire/ -- "The clinical efficacy of fostrox in combination with Lenvima continues to improve with median time to progression having increased to over 6 months" October – December Financial summary for the quarter Net turnover amounted to SEK 4.4 (2.3) million. The loss before interest, tax, depreciation and amortization (EBITDA) amounted to SEK -20.1 (-17.9) million. Basic and diluted earnings per share amounted to SEK -0.28 (-0.32) and SEK -0.28 (-0.32) respectively. Cash flow from operating activities amounted to SEK -4.6 (-24.7) million. Cash and cash equivalents at the end of the period amounted to SEK 169.5 (117.4) million. Significant events during the quarter In October, data from an investigator evaluation showed continued promising tumor control with fostrox + Lenvima in HCC. All patients in the phase 2a study had dosed at least two treatment cycles. In October, the Board of Directors announced that Anette Lindqvist is leaving her position as Board Member of Medivir AB due to personal reasons. In October the nomination committee was appointed ahead of the AGM in May 2024. The Nomination Committee consists of Karl Tobieson, appointed by Linc AB, Richard Torgerson, appointed by Nordea Investment Funds, Anders Hallberg, appointed by HealthInvest Partners and Uli Hacksell, Chairman of the Board, Medivir AB. The Q3 report and the subsequent webcast included in-depth interim data from the 18 patients in the phase 1b/2a study who had a minimum follow-up of 12 weeks. These data continued to demonstrate clear patient benefit for the fostrox + Lenvima combination. In November it was announced that the company's interactions with the FDA regarding fostrox's clinical development plan had intensified with a first Type D meeting with a positive response regarding critical elements of the design for the planned phase 2b study. Late November it was announced that the development of an updated formulation of fostrox, suitable for commercial manufacture, had been finalized. In December, Medivir's partner IGM Biosciences, communicated a strategic pipeline cost-saving prioritization that among other things affects IGM's DR-5-agonist, aplitabart, in combination with birinapant, which was licensed in from Medivir in 2021. In December it was announced that Medivir has signed an agreement with Lonza for the manufacture of fostrox drug substance for the planned phase 2b study. In December it was announced that the durable clinical benefit in the ongoing phase 1b/2a study continues to improve, as data become more mature. In December, a rights issue was carried out, through which the company received proceeds of approximately SEK 129 before deduction of costs attributable to the rights issue. January – December Financial summary for the period Net turnover amounted to SEK 7.6 (4.4) million. The loss before interest, tax, depreciation and amortization (EBITDA) amounted to SEK -88.7 (-84.8) million. Basic and diluted earnings per share amounted to SEK -1.48 (-1.59) and SEK -1.48 (-1.59) respectively. Cash flow from operating activities amounted to SEK -59.7 (-101.8) million. Cash and cash equivalents at the end of the period amounted to SEK 169.5 (117.4) million. Events after the end of the period In January, a directed issue to Hallberg Management AB was carried out amounting to approximately SEK 20 million before deduction of issuance costs. In January, positive data from the ongoing phase 1b/2a study in advanced liver cancer (HCC) showing further improved response and time to progression was presented at the ASCO GI Symposium in San Francisco. In January Tango Therapeutics announced that it has dosed the first patient with TNG348, a new USP1-inhibitor from the preclinical USP1 program inlicensed from Medivir in 2020 Conference call for investors, analysts and the media The Year-End Report January - December 2023 will be presented by Medivir's CEO, Jens Lindberg. Time: Thursday, February 15, 2024, at 14.00 (CET). To access the webcast and find information about the teleconference, please klick HERE! The conference call will also be streamed via a link on the website: . The presentation will be available on Medivir's website after completion of the conference. CEO's message Fostrox in combination with Lenvima continues to show improved efficacy for patients with advanced primary liver cancer (HCC). The data presented in January 2024 at the ASCO-GI congress in San Francisco showed a further increase in the proportion of patients who achieve a clinically relevant reduction of their liver tumor and that patients stay on treatment longer with interrupted tumor growth. These data attracted very strong interest, which makes us even more convinced of fostrox's future role in the treatment of HCC and creates the opportunity for a faster route to market. The unmet medical need in the treatment of advanced HCC is significant. Primary liver cancer is the third leading cause of cancer-related deaths. In today's treatment guidelines, an immunotherapy combination (Tecentriq®/Avastin®) is recommended as first-line treatment. However, when this combination has stopped working, there are no approved alternatives for second-line treatment. There is thus a large need for additional treatment options in second-line that attacks the tumor in different ways compared to what Tecentriq and Avastin does. Fostrox is an organ specific, so-called smart chemotherapy, that selectively kills cancer cells in the liver. Together with Lenvima, these two form a unique combination of complementary drugs that have shown promising interim results in an ongoing phase 1b/2a study, including a complete tumor response in one patient. All patients in the study have now had at least 18 weeks of follow-up. The more mature and in-depth data we see from the study, the more convincing the clinical benefit for patients has become. Data from this study was presented at the ASCO GI Congress on January 19, 2024. These results, evaluated by investigators and local radiologists, showed further improvement as the Overall Response Rate (ORR) had increased to 25% (RECIST 1.1), a notably higher percentage than the 5-10% shown in second-line HCC in previous studies. The update also showed continued good tolerability without any new unexpected side effects. At the time of this quarterly report over 40% of patients remain on treatment in the study, and the clinical efficacy keeps improving. The median time to progression has increased further to 6.3 months, compared to 5.1 months at ASCO GI, significantly better than previous studies in second-line HCC. The patient who has benefited the longest remains on treatment after 18 months with partial response and continued shrinking tumor. Our data was met with great interest at ASCO GI, where we had the opportunity to discuss with leading global experts what the fostrox + Lenvima combination could mean for these patients. The discussions confirmed the great need that exists in the second-line treatment of HCC, where patients are today without any approved treatment alternative. With these promising data, fostrox has the opportunity to become the first approved medicinal treatment in a market worth ~2.5 billion USD annually, through a so-called accelerated approval by the regulatory authorities. We have therefore put in a higher gear in 2023 to ensure maximum momentum in fostrox's development program based on this possibility of accelerated approval. In Q4 2023 the development of an updated, commercially adapted formulation of fostrox was completed. In parallel, we have initiated the process with the FDA for a so-called Type C meeting to discuss the final study design of the planned registrational phase 2b study. In conjunction with these measures, we can now also deepen our discussions with potential partners, in accordance with previously communicated plans. We can also note that several of the projects that Medivir has licensed out to collaboration partners is entering clinical stage. In January 2024, Tango Therapeutics initiated a phase 1/2 study and dosed the first patient with TNG348, a USP-1 inhibitor developed from the preclinical USP1 program that was in-licensed from Medivir in 2020. INFEX Therapeutics also intends to initiate phase 1 study in 2024 - with the preclinical program MBLI, previously in-licensed from Medivir. IGM Biosciences has completed the fifth dose escalation cohort in the company's phase 1 study in solid tumors with Medivir's clinical project birinapant in combination with its own DR5 agonist antibody IGM-8444, now called aplitabart. For Medivir, the clinical development of fostrox is clearly in focus. With the promising data showing further improved clinical efficacy compared to expected treatment results in second-line HCC, we are even more confident that fostrox can become an effective liver cancer drug that makes a real difference to patients. I would like to thank old and new shareholders for the trust in our rights issue and for the capital injection that benefited us in the directed share issue. I look forward to keeping you informed of Medivir's continued development. Jens Lindberg Chief Executive Officer For further information, please contact Magnus Christensen, CFO Phone: +46 (0)8 5468 3100 E-mail: [email protected] This report has not been subject to auditors' review. The information was submitted for publication at 08.30 CET on February 15, 2024 The following files are available for download: SOURCE Medivir

裁员、砍管线是资金困难的生物药企活下去最经常采用的办法。近日，总部位于美国加州的IGM Biosciences决定裁员22%，并且停止所有血液瘤项目以及其靶向细胞因子候选产品的临床开发。IGM预计通过裁员和管线消减可以将公司现金流从原来坚持到2025年下半年延长到2026年第二季度。根据美国证券交易委员会（SEC）的文件，截至2023年9月30日，IGM拥有290名全职员工。IGM首席执行官表示，虽然公司停止的项目临床和临床前的数据表现都很亮眼，但是鉴于公司在资本市场融资困难，日子越来越难过，只能决定集中资源在最可能在近期产生效益的项目上。押注实体瘤和自免IGM表示将和赛诺菲继续合作，一起开发肿瘤学、免疫学和炎症候选药物。集中资源开发其治疗结直肠癌的IgM死亡受体5（DR5）激动剂抗体，以及用于自身免疫性疾病的IgMT细胞接合抗体。死亡受体5（DR5）激动剂Aplitabart联合FOLFIRI和贝伐珠单抗治疗二线转移性结直肠癌的随机临床试验正在招募患者，目标是到2024年第一季度末招募约110名患者。除了美国的临床试验地点外，该试验还包括亚洲和欧洲的多个临床试验地点。Aplitabart在晚期结直肠癌患者中应用的单臂联合临床试验也正在进行中。预计在2024年上半年完成10mg/kg Aplitabart和FOLFIRI治疗后期结直肠癌的患者入组。Aplitabart联合FOLFIRI治疗的一组患者报告的1期数据显示，其安全性与单独化疗的预期安全性大致相当。剂量为3mg/kg的Aplitabart和不含贝伐珠单抗的FOLFIRI的组合在24例患者组中的中位无进展生存期（PFS）为5.6个月，高于标准护理三线结直肠癌治疗（不含贝伐珠单抗）约2个月的历史中位无进展生存期。该组观察到的最长无进展生存期已超过16个月，且多位患者使用Aplitabart和FOLFIRI的治疗持续时间比之前的FOLFIRI方案更长。与此同时，IGM将优先推进imvotamab的临床开发，这是一种基于IgM的CD20xCD3双特异性T细胞结合抗体，用于自身免疫性疾病。关于imvotamab目前正在进行两项1b期临床试验，一项用于严重系统性红斑狼疮（SLE），另一项用于严重类风湿性关节炎（RA）。这些临床试验正在扩大到包括多个美国和国际临床试验地点。最近IGM还获得了FDA的批准，批准了其使用imvotamab治疗特发性炎症性肌病（肌炎）的IND申请，并且正在准备推进该临床试验。在临床前体内研究中，imvotamab已证明组织内的深部B细胞耗竭，其中致病性免疫细胞的耗竭可能对自身免疫性疾病的长期临床益处至关重要。Imvotamab还在临床前体外研究中证明，与利妥昔单抗相比，它可以更有效地消耗CD20表达水平低的B细胞。此外，IGM还划提交IND，以开始IGM-2644的临床开发，IGM-2644是一种CD38xCD3T细胞接合抗体，正在研究用于治疗自身免疫性疾病。亏损扩大，裁员缩管线2023年11月14日，IGM发布Q3财报，2023财年前三财季累计收入147.90万美元，去年同期累计收入为69.70万美元，同比增长112.20%。2023财年前三财季累计净亏损1.86亿美元，去年同期累计净亏损为1.69亿美元，同比扩大10.20%。作为公司战略重整的一部分，IGM将停止所有血液肿瘤学临床开发以及其靶向细胞因子候选产品的临床开发。包括Aplitabart联合birinapant用于治疗急性髓系白血病；IGM-2644（CD38xCD3）治疗多发性骨髓瘤；以及IGM-2537（CD123xCD3）和IGM-7354（IL-15xPD-L1）。去年11月，IGM在2022年美国血液学会（ASH）年会上，以海报的形式展示了几款候选药物在血液瘤中的临床前数据。数据显示与传统IgG抗CD38抗体相比，IgM-2644具有更强的补体依赖性细胞毒性(CDC)活性，此外，IGM-2644以最小的细胞因子释放实现了对达雷妥尤单抗耐药细胞系的强效T细胞依赖性细胞毒性（TDCC）杀伤，以及对骨髓瘤患者样本的强效TDCC杀伤。在人源化异种移植模型中，IGM-2644 也被证明可以抑制CD38+肿瘤生长，且与IgG双特异性T 细胞接合剂相比，它避免了杀死免疫效应细胞。IgM-2537在细胞因子释放最少的情况下显示有效的体外和体内活性。IGM-2537 被证明与人 CD123 结合具有高亲和力和特异性。IGM-2537 与CD123和CD3抗原共同参与，导致T细胞重定向杀伤急性髓系白血病 （AML） 细胞系，同时伴随 T 细胞活化，并在离体测定中以生理相关的效应/靶标比率消除AML原始细胞。与 IgGT细胞接合分子相比，IGM-2537还显示出显着减少的细胞因子释放，例如IFN-γ、TNF-α和IL-6。虽然数据鼓舞人心，但是资金的缺乏加上创新药物的开发需要的超额预算让IGM不得不放弃血液瘤管线。值得注意的是，IGM去年和赛诺菲刚刚达成巨额交易。2022年3月29日，赛诺菲和IGM达成超过60亿美元的合作协议。根据协议，双方将利用IGM公司独有的IgM抗体技术平台，开发针对3个肿瘤学靶点和3个免疫学/炎症靶点的激动剂。IGM公司将获得1.5亿美元的前期付款，在开发、监管和商业里程碑方面的总金额可能超过60亿美元。针对每个肿瘤学靶点，IGM公司可能获得高达9.4亿美元的里程碑付款；在免疫学/炎症领域，针对每个靶点，IGM公司有资格获得高达10.65亿美元的里程碑付款。在血液瘤方面管线的缩减，可能会影响到赛诺菲给予的里程碑付款。参考来源：1.https://endpts.com/igm-biosciences-cuts-22-of-workforce-axes-hematologic-oncology-program/2.https://www.globenewswire.com/news-release/2023/12/05/2791338/0/en/IGM-Biosciences-Announces-Strategic-Pipeline-Prioritization-and-Cash-Runway-Extension.html3.https://investor.igmbio.com/news-releases/news-release-details/igm-biosciences-presents-data-t-cell-engager-portfolio本周好文推荐如需转载请联系佰傲谷并在醒目位置注明出处﹀ ···