Ferrer Internacional SA

今日，维智基因（Verge Genomics）和Ferrer宣布达成一项战略合作，共同开发Verge的主要候选药物VRG50635，用于在欧洲、中南美洲、东南亚和日本治疗散发性和家族性肌萎缩侧索硬化症（ALS）。VRG50635是一种针对PIKfyve的潜在“best-in-class”小分子抑制剂。新闻稿指出，VRG50635是首批进入临床的完全由人工智能（AI）平台发现和开发的药物之一。 该合作将Verge用于靶点发现和药物开发的“all-in-human”技术平台，及其在临床试验方面的创新，与Ferrer在临床开发、制造和商业化方面的专长相结合。根据协议条款，Ferrer将获得在美国以外的多个地区共同开发和商业化VRG50635治疗ALS的独家权利。Verge保留了在美国和协议以外的所有国家和地区中，VRG50635开发和商业化的所有权利。根据协议，Verge可能获得高达1.125亿欧元的预付款和潜在里程碑付款。 VRG50635是一种强效、可口服的PIKfyve抑制剂，能提高ALS患者神经元的存活率，并已在ALS相关运动神经元变性模型的多项临床前研究中显示出疗效。PIKfyve是基于Verge公司的AI平台CONVERGE在患病人体组织中发现的ALS治疗靶点。目前，VRG50635正在加拿大和几个欧洲国家进行1b期概念验证（PoC）研究。Verge的PoC研究采用了创新技术，能够收集大量无偏倚、客观、与疾病相关的数据，以在临床开发早期正确评估安全性、耐受性、药理剂量反应和潜在功效。 对于人工智能在医药行业的应用，维智基因的共同创始人兼首席执行官张欣立（Alice Zhang）博士在接受药明康德内容团队采访时指出，人工智能“无偏倚”（unbiased）的算法系统有望建立起整合多个机制的疾病模型，从更广阔的角度去考虑疾病，而不让人类影响研究的方向。在她看来，“越为杂乱无章的领域，越可能是机器学习与人工智能大展拳脚的地方。机器学习在解决最为复杂的疾病问题上，能带来重要的不同。” 参考资料： [1] Verge Genomics and Ferrer Announce Agreement to Co-Develop Clinical-Stage ALS Therapy VRG50635. Retrieved March 25, 2024, from https://www.globenewswire.com/news-release/2024/03/25/2851439/0/en/Verge-Genomics-and-Ferrer-Announce-Agreement-to-Co-Develop-Clinical-Stage-ALS-Therapy-VRG50635.html [2] Ferrer signs $122M biobucks deal for ex-US license to Verge's phase 1 ALS therapy. Retrieved March 25, 2024, from https://www.fiercebiotech.com/biotech/ferrer-signs-122m-biobucks-deal-ex-us-license-verges-phase-1-als-therapy 内容来源于网络，如有侵权，请联系删除。

Amylyx will spend the next eight weeks reviewing the data with the FDA and ALS community. Image credit: Shutterstock / David Herraez Calzada. Amylyx Pharmaceuticals ’s Phase III trial for amyotrophic lateral sclerosis (ALS) Relyvrio has failed to meet any of its endpoints. Following the Phase III flop, Amylyx said it will now engage with regulatory authorities and the broader ALS community, to discuss the results from the Phoenix trial over the next eight weeks and decide how to move forward. The company added this could include withdrawing Relyvrio from the market. The candidate was approved by the US Food and Drug Administration (FDA) in September 2022 based on findings from the multicentre Phase II Centaur trial that enrolled 137 ALS patients. In October 2023, the drug, which was set to be marketed as Albrioza in Europe, was rejected for approval by the European Medicines Agency (EMA). Once the market opened, the company’s stock crashed by 85.7%. The global, 48-week, randomised, placebo-controlled Phase III clinical trial of Relyvrio (AMX0035) did not meet its primary endpoint of reaching statistical significance from baseline in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) total score. There was also no statistical significance seen in secondary endpoints. See Also: AD/PD 2024: exploring cutting-edge therapies for ALS AD/PD 2024: Vaxxinity reveals Phase I success with Parkinson’s vaccine The trial saw 664 patients randomised into two arms, Relyvrio or placebo, with both treatment groups receiving standard of care (SoC). In a statement, Amylyx co-CEOs Justin Klee and Joshua Cohen said: “We are surprised and deeply disappointed by the Phoenix results following the positive data from the Centaur trial. We will be led in our decisions by two key principles: doing what is right for people living with ALS, informed by regulatory authorities and the ALS community, and by what the science tells us.” Despite the disappointing efficacy results, Relyvrio was well-tolerated with no new safety signals. European participants who completed the 48-week randomised phase had the option to enrol in an open-label extension of the trial of up to two years in duration, which remains ongoing. Momna Ali, healthcare analyst at GlobalData, said: “When Relyvrio was initially approved, there was a swirl of controversy anyway. It’s a shame that the late-stage trial found no significant difference between ALS patients treated with Relyvrio and those on placebo after 48 weeks. “There are approximately 4,000 people in the US on Relyvrio and if Amylyx does pull its drug, sadly patients would only be left with three approved therapies. One, from Biogen , which targets a very small portion of the population with certain genetic mutations. The other two, called Radicava and riluzole, have shown modest therapeutic effects on both function and survival.” GlobalData is the parent company of the Clinical Trials Arena . Amylyx has two other ongoing studies with Relyvrio, a Phase III study in progressive supranuclear palsy (PSP) and a Phase II study in Wolfram syndrome. Relyvrio is a combination of sodium phenylbutyrate and taurursodiol, which cause a reduction of damage to nerve cells. ALS landscape Also known as classical motor neuron disease, ALS is a progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death. GlobalData has predicted global sales of Relyvrio to hit $1.33bn in 2029. Developing disease-modifying for ALS has proven challenging, with a high failure rate often attributed to the disease’s unclear aetiology and complex pathophysiology. Earlier this year, Sanofi and Denali Therapeutics’s SAR443820 failed to meet its primary endpoint in a Phase II trial and Ferrer Internacional’s FAB122 failed to meet its primary endpoint in a Phase III study.

Qalsody was licensed by Biogen from Ionis Pharmaceuticals in 2022. Image Credit: Tada Images / Shutterstock. Biogen has received a positive opinion from the European Medicines Agency (EMA) committee recommending approval for Qalsody (tofersen) to treat adult amyotrophic lateral sclerosis (ALS) patients with a mutation in the superoxide dismutase 1 (SOD1) gene. The EMA’s Committee for Medicinal Products for Human Use (CHMP) based its positive recommendation on data from the Phase III VALOR trial (NCT02623699). The final decision on the marketing authorisation for Qalsody is expected in Q2 of this year. The therapy missed the primary efficacy endpoint of ALS function, measured using the Revised ALS Functional Rating Scale (ALSFRS-R), in the VALOR study. However, Qalsody met the secondary endpoints of cutting neurofilament (NfL) and SOD1 protein levels. Qalsody is an antisense oligoneucleotide that inhibits the production of mutant superoxide dismutase 1 (SOD1) protein, which is linked to toxic effects on the motor neurons in the disease. It was developed as part of a development and licence agreement with Ionis Pharmaceuticals . In April 2023, Qalsody received accelerated approval from the US Food and Drug Administration (FDA) as a treatment for ALS, in patients with SOD1 gene mutation. Although the FDA Advisory Committee voted unanimously for accelerated approval of the drug, it advised against a traditional approval citing insufficient current efficacy data. See Also: Merck’s Keytruda regimen gains positive EMA CHMP opinion SK bioscience’s typhoid vaccine receives WHO prequalification Qalsody generated $3.3m in US sales in Q4 2023, according to Biogen’s financials. GlobalData expects the drug to maintain the upward growth trajectory and pull in $128m in global sales in 2029. GlobalData is the parent company of Pharmaceutical Technology . Biogen is evaluating Qalsody in the placebo-controlled Phase III ATLAS trial (NCT04856982). The study aims to evaluate whether the drug can delay the clinical onset of ALS in patients with the SOD1 gene. The trial is expected to enrol 150 asymptomatic adult carriers of the SOD1 gene with elevated NfL, as per ClinicalTrials.gov . The ALS treatment field has suffered some setbacks. Most recently, Ferrer International’s oral edaravone (FAB122) failed to meet the primary efficacy and key secondary endpoints in the Phase III ADORE trial (NCT0178810). As the drug did not demonstrate a significant benefit over placebo in slowing ALS progression, the company also terminated the open-label extension ADOREXT study (NCT05866926). In October 2023, the EMA rejected the marketing authorisation application for Amylyx Pharmaceuticals ’ Albrioza, citing the unreliability and efficacy of the supporting data package.