Izokibep

Lonigutamab is the first subcutaneous anti-IGF-1R to demonstrate clinical responses in thyroid eye disease (TED) patients LOS ANGELES, May 29, 2024 (GLOBE NEWSWIRE) -- ACELYRIN, INC. (Nasdaq: SLRN), a late-stage clinical biopharma company focused on accelerating the development and delivery of transformative medicines in immunology, today announced that positive proof-of-concept data from an ongoing Phase 1/2 trial of lonigutamab in thyroid eye disease (TED) will be shared as a late breaking oral presentation at the Endocrine Society’s Annual Meeting (ENDO 2024), taking place from June 1-4 in Boston, MA. Lonigutamab is a subcutaneously (SC) delivered humanized IgG1 monoclonal antibody targeting the insulin-like growth factor-1 receptor (IGF-1R). Inhibition of IGF-1R is the only FDA-approved mechanism of action for the treatment of TED. Presentation details are as follows: Title: Preliminary Safety And Efficacy Of Subcutaneous Lonigutamab (anti-IGF-1R) From A Phase 1/2 Proof Of Concept Study In Patients With Thyroid Eye DiseaseDate/Time: Monday, June 3; 2:00pm - 2:15pm ETPresenter: Dr. Shoaib Ugradar, Department of Orbital and Oculoplastic Surgery, Private Practice; The Jules Stein Eye Institute, University of California About the Phase 1/2 TrialThe Phase 1/2 clinical trial (NCT05683496) is a multi-center trial evaluating the safety and efficacy of lonigutamab dosed subcutaneously in three cohorts of patients with active thyroid eye disease (TED). Cohort 1 is placebo-controlled testing lonigutamab 40mg every three weeks (Q3W) through six weeks, cohort 2 is open label testing a 50mg loading dose followed by 25mg every week (QW), and cohort 3 is open label testing every four weeks (Q4W) dosing. For more information about the Phase 1/2 trial, please visit www.clinicaltrials.gov. About Thyroid Eye DiseaseThyroid Eye Disease (TED) is a vision-threatening autoimmune disease in which there is both inflammation and expansion of the tissues behind the eye, resulting in eye bulging – known as proptosis – that may result in the subsequent inability to close the eyelids. Double vision, or diplopia, can occur, as well as the potential for compression of the retinal nerve, which can lead to blindness. Thus, TED is a progressive, chronic inflammatory disease where longer-term treatment has the potential to improve depth and durability of response. More than 100,000 people in the United States are estimated to suffer from TED. About Lonigutamab (anti-IGF-1R)Lonigutamab is a humanized IgG1 monoclonal antibody targeting the IGF-1 receptor and is delivered subcutaneously. Relative to standard of care, lonigutamab binds to a distinct epitope, which results in internalization of the receptor within minutes, and in preclinical binding and functional laboratory assays, it has been shown to be 75-fold more potent. The characteristics of lonigutamab that enable subcutaneous delivery may also enable the potential for longer-term dosing, which we believe may improve depth and durability of clinical response. Based on our preclinical and pharmacodynamic data from our completed single ascending dose study with lonigutamab, we can potentially optimize the therapeutic window utilizing the SC route of administration. About ACELYRIN, INC.ACELYRIN, INC. (Nasdaq: SLRN) is a Los Angeles area-based late-stage clinical biopharma company – with additional operations in the San Francisco Bay area – focused on providing patients life-changing new treatment options by identifying, acquiring, and accelerating the development and commercialization of transformative medicines. ACELYRIN has two programs in late-stage clinical development. Lonigutamab is a subcutaneously delivered monoclonal antibody targeting IGF-1R advancing into Phase 2b/3 development for the treatment of thyroid eye disease. Izokibep is a next generation inhibitor of IL-17A in Phase 2b/3 development for the treatment of psoriatic arthritis, hidradenitis suppurativa and uveitis. For more information about ACELYRIN, visit us at www.acelyrin.com or follow us on LinkedIn and X. Forward Looking Statements This press release contains forward-looking statements including, but not limited to, statements related to ACELYRIN’s expectations regarding its cash runway and sufficiency of its cash resources; ACELYRIN’s plan to take lonigutamab through late-stage development milestones; the potential future success of ACELYRIN’s izokibep program; anticipated development activities including the planned initiation and timing of clinical trials, and/or the availability of clinical data; the therapeutic potential of ACELYRIN’s product candidates; and other statements that are not historical fact. These forward-looking statements are based on ACELYRIN’s current plans, objectives and projections, and are inherently subject to risks and uncertainties that may cause ACELYRIN’s actual results to materially differ from those anticipated in such forward-looking statements. Such risks and uncertainties include, without limitation, those associated with the successful completion of development and regulatory activities with respect to ACELYRIN’s product candidates; the timing and results of ACELYRIN’s clinical trials, including the risk that future clinical trial results could differ materially and adversely from prior clinical trial results or data; maintaining and defending intellectual property protection; delays or failures to secure adequate supply of its product candidates; ACELYRIN’s failure to realize the expected benefits of its acquisition of additional programs; legal proceedings and other risks and uncertainties affecting ACELYRIN including those described from time to time under the caption “Risk Factors” and elsewhere in ACELYRIN’s current and future reports filed with the Securities and Exchange Commission, including its Annual Report on Form 10-K for the year ended December 31, 2023. Forward-looking statements contained in this press release are made as of this date, and ACELYRIN undertakes no duty to update such information except as required under applicable law. ACELYRIN Contacts:Tyler MarciniakVice President of Investor Relations,Communications and Advocacyinvestors@acelyrin.com media@acelyrin.com

01CEO离职近日，Acelyrin在发布公司最新进展的同时，宣布首席执行官Shao-Lee Lin已经离职，并且2024年第一季度的财报会议也不开了。截图自Acelyrin公开信息（注，以下均为此来源）令人难解的是，一年前的Acelyrin是业内绝对的顶流，为何“刚开完上市周年庆”，公司便出现了如此大的变动？把时间拉回到2023年5月5日，仅仅成立三年的Acelyrin在火速完成累计5.58亿美元的三轮融资后，成功在纳斯达克上市，IPO募资达到5.4亿美元，是近几年来美股规模最大的IPO之一。Acelyrin能在一二级市场受到资本的热捧，是源于公司创始团队过往经历与业务模式有望创造极大商业价值，为投资人带来高额汇报。由此不得不提的是Acelyrin的创始人兼（前）首席执行官Shao-Lee Lin以及首席医学官Paul Peloso等多位创始团队成员的老东家——Horizon Therapeutics。Horizon可以说是“小鱼吃虾米”之路成功的典范，凭借两款核心产品Tepezza和Krystexxa的差异化竞争潜力以及展现出的商业化爆发力，在2022年底成功被Amgen宣布以278亿美元纳入旗下，创下了一个“造富”神话。Horizon与辉瑞收购的Seagen等药企不同的是，后者用有强大的研发技术平台，具有源源不断创造创新分子的潜能，而前者的两款核心产品都不是自研产品，其中，Tepezza是Horizon在2017年收购River Vision Development Corp而来；Krystexxa则是从杜克大学和Mountain View Pharmaceuticals处License-in而来。2019年底，Tepezza获批上市，第二年，Shao-Lee Lin等人便创办了Acelyrin。从某种程度上来说，Tepezza的商业化成功与否，已经和Acelyrin的公司命途有了深刻的绑定。2020年，Tepezza上市的首年爆发出了惊人的造血能力，全年斩获8.2亿美元销售额。Tepezza商业的成功，再到最终被Amgen天价收购，Horizon“小鱼吃虾米”之路的成功，离不开高管团队的眼光及再开发能力。因而当“原班人马”创立Acelyrin并继承了Horizon“买”的基因后，一级市场投资人都想上门“送钱”，2022年，Acelyrin是全球融资最多的biotech之一，2023年，成立不到3年的Acelyrin便火速上市，成为近几年来美股最大的IPO之一。在大额资金的支持下，Acelyrin也是快速完成了核心管线的收购，在IPO时已经布局了3款药物6个适应症，其中以IL-17单抗Izokibep为核心管线，围绕其开展的4个适应症均已经处于临床后期。在礼来的ixekizumab和诺华的secukinumab等IL-17靶点药物商业化取得巨大成功的背景下，各方对Izokibep未来的商业化潜力都给与了极高的期待，因而可以说，若Izokibep能顺利获批上市，Acelyrin将极有可能复刻Horizon的成功，创始团队也将一战成名。换言之便是，Izokibep只许成功不许失败，一旦失败，对于Acelyrin来说就是致命一击，而现实的确是如此，总是不尽如人意。02IL-17抗体临床失利股价至今暴跌82%在拿够了投资的人钱之后，Acelyrin最需要证明的是创始团队的“眼光”。即便Acelyrin引进的是经过精挑细选、处于临床后期的产品，即所谓确定性极强的产品，但药物研发“九死一生”并不是空口而谈，面对的研发风险仍然极大。Acelyrin上市后，股价在波动中不断创造新高，反应的是投资人对于核心产品极强的预期，但期望越高，失望也就越大。四个月后，“泡沫”首次被刺破。9月11日，Acelyrin公布了Izokibep治疗化脓性汗腺炎（HS）的临床2b/3期临床试验的最新数据，结果显示该研究的B部分并未达到HiSCR75（脓肿和炎性结节减少至少75%）的主要终点，受此消息影响，Acelyrin股价第二日暴跌54%，直接腰斩。Izokibep作为Acelyrin的核心产品，HS又是其开发的关键适应症（Acelyrin预测，2023年HS的市场规模为28亿美元），Acelyrin自然不愿轻易“接受”这个结果，于是这次“失败”逐渐演变成了一场“闹剧”。两个月后，Acelyrin发布消息称，临床试验的失败可能是由CRO（Fortrea）的失误造成的，而CRO方则是“极力反驳”，目前仍未有确定的结果。而且在这则“甩锅”的消息中，不知道Acelyrin是否是由于“失误”，还隐隐透露出Izokibep的另一个适应症“银屑病关节炎”的临床结果也似乎不容乐观。Acelyrin欲挽回投资人信心的“阳谋”失败，股价再次跳水，跌超30%。今年3月11日，Acelyrin宣布Izokibep治疗银屑病关节炎（PsA）的临床2b/3期试验成功，以及此前失败的HS适应症，在长期随访中也取得了不错的结果。但Acelyrin当天的股价仅不温不火的涨了10%，并在随后的一个多月时间内，跌跌不休，不断创新低，截至最新交易日收盘，股价相较于9月11日收盘（Acelyrin在盘后发布了Izokibep治疗HS的临床数据），已经跌去了82%。Acelyrin似乎已经不只受困于核心管线的研发失利，而是在于更深层次，由于管理层面的“谜之操作”而导致的投资人信心溃败。首席执行官Shao-Lee Lin的离职，以及管理层的重组，似乎也应证了这一问题。SUMMARY小结从风光上市到深陷困境，Acelyrin仅花了不到一年时间。而CEO离职，管理层重组后，Acelyrin能否重塑投资人的信心，还待更多的时间验证，毕竟如果Acelyrin成功晋级为Horizon的“翻版”，哪位投资人又能不爱呢。点击下方“药渡“，关注更多精彩内容免责声明“药渡”公众号所转载该篇文章来源于其他公众号平台，主要目的在于分享行业相关知识，传递当前最新资讯。图片、文章版权均属于原作者所有，如有侵权，请及时告知，我们会在24小时内删除相关信息。微信公众号的推送规则又双叒叕改啦，如果您不点个“在看”或者没设为"星标"，我们可能就消散在茫茫文海之中~点这里，千万不要错过药渡的最新消息哦！