Utreloxastat

iStock, JHVEPhoto Monday’s agreement comes days after PTC discontinued the development of another asset, utreloxastat, due to disappointing Phase II data in amyotrophic lateral sclerosis. Novartis and PTC Therapeutics on Monday announced that they have entered into a global license deal to advance the biotech’s Huntington’s disease drug candidate PTC518. As per the terms of the agreement, Novartis will pay $1 billion upfront and will put up to $1.9 billion on the line in developmental, regulatory and sales milestones. PTC will also be entitled to a 40% share of profits in the U.S.—while Novartis will keep 60%—as well as double-digit tiered royalties on sales outside the country. Pending regulatory clearance and other customary closing conditions, the companies expect to complete the transaction in the first quarter of 2025. After unveiling the deal, PTC jumped about 20% before Monday’s opening bell, according to Seeking Alpha . PTC518, the centerpiece of biopharma’s latest multibillion-dollar deal, is a small molecule drug candidate designed to be taken orally. The drug, which can penetrate into the brain, works by lowering the production of the mutant huntingtin protein, with the aim of preventing massive neuronal death and brain injury—both hallmarks of Huntington’s disease (HD) and drivers of its progression. In September 2024, the FDA granted PTC518 its Fast Track designation for Huntington’s. PTC518 is currently being studied in the Phase II PIVOT-HD trial. An interim readout in June 2024 showed a “dose-dependent lowering” of mutant huntingtin protein in the blood and cerebrospinal fluids at 12 months. PTC did not reveal specific data at the time, however noting that there were “favorable trends” in clinical assessments, including Total Motor Score. As per Monday’s agreement, PTC will still be responsible for completing PIVOT-HD—which is likely to occur in the first half of 2025—after which Novartis will take charge of PTC518’s development, manufacturing and completion. Novartis has long been interested in Huntington’s. In March 2022, after discovering that the small molecule drug candidate branaplam could lower levels of mutant huntingtin, the pharma decided to study the drug for Huntington’s instead of its original plan in spinal muscular atrophy. Branaplam is an SMN2 RNA splicing modulator that works by slightly modifying huntingtin mRNA and tagging it for degradation, in turn reducing the overall expression of the huntingtin protein. A few months later, however, the pharma stalled its Huntington’s plans for branaplam after detecting possible side effects. Novartis formally discontinued branaplam for Huntington’s in February 2023. Earlier this year, the pharma again bet on Huntington’s with an up to $1.3 billion deal with Voyager Therapeutics and its pipeline candidates for HD and spinal muscular atrophy. Specifically, Novartis wants to leverage Voyager’s TRACER Capsid Discovery Platform to discover adeno-associated virus capsids to deliver gene therapy payloads into the brain. For PTC, the Novartis agreement comes just days after it axed another asset, utreloxastat, due to disappointing Phase II data in amyotrophic lateral sclerosis.

以下文章转自生物药大时代 声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。 近日，PTC Therapeutics宣布，一项针对实验性渐冻症药物utreloxastat的2期临床试验在主要终点和所有次要终点均失败。研究人员根据ALSFRS-R的综合症状评分，正在研究这种药物是否可以减缓疾病的进展，最终确定不能，公司表示并不打算进一步开发 utreloxastat。 此外，公司在周三宣布，以1.5 亿美元的价格出售其罕见儿科疾病优先审查券 （PRV），买家暂未透露，但在 SEC 文件中被描述为一家大型制药公司的子公司。 该PRV是在2024年11月13日美国食品和药物管理局(FDA)批准KEBILIDI™(eladocagene exuparvovec-tneq)后授予公司的，这是一种用于治疗儿童和成人AADC缺乏症的药物。 PRV的出售正在等待惯例的成交条件，包括Hart-Scott-Rodino (HSR)反垄断改进法案规定的等待期届满。 公司研发管线 PTC Therapeutics专注于发现和开发罕见疾病药物。公司的使命是为选择有限的患者提供治疗方法，利用科学和临床专业知识将疗法推向市场。这笔交易是PTC通过全球商业化和多元化管线为利益相关者创造最大价值的战略的一部分。 此前，公司透露对其2024年总收入前景表示乐观，现预计将增至7.5亿至8亿美元之间。这一乐观预测根据公司强劲的第三季度盈利，报告总收入为1.97亿美元。杜氏肌营养不良症(DMD)特许经营，包括Emflaza，贡献了1.24亿美元，对这一财务表现做出了重大贡献。 参考资料: www.endpts.com www.ptcbio.com