Pridopidine

Prilenia Therapeutics can garner as much as about 500 million euros (around $568 million) in upfront and total milestone payments, plus royalties.\n Dutch biotech Prilenia Therapeutics has entered a licensing pact with Spanish pharmaceutical company Ferrer to push the biotech\'s sole asset through clinical development and beyond in Europe.Under the terms of the deal, Prilenia and Ferrer will jointly develop and potentially commercialize neurodegenerative disease candidate pridopidine in Europe and other undisclosed select markets, the companies said in an April 28 release. Prilenia will retain full rights to the oral sigma-1 receptor agonist in North America, Japan and the Asia-Pacific region.Ferrer is paying Prilenia 80 million euros upfront, with up to 45 million euros available in near-term development, regulatory and commercial milestones. In total, Prilenia can garner as much as 500 million euros (around $568 million) in upfront and milestone payments, according to the release, plus royalties on net sales.The focus of the partnership will be Huntington\'s disease and amyotrophic lateral sclerosis (ALS), with the partners planning to launch a phase 3 ALS trial in early 2026, a Ferrer spokesperson told Fierce Biotech in an email. Pridopidine previously failed to improve ALS disease severity in a phase 2 test, but the company said at the time that “positive trends” warranted further studies of the drug candidate. A post hoc analysis of the data found that some patients who were rapidly declining in speech, handwriting and other daily functions had “substantially less decline” when given pridopidine compared to those who received placebo, Prilenia said.In addition to Huntington\'s and ALS, Ferrer has first rights to develop pridopidine in other potential indications, the company\'s spokesperson said. Prilenia has already submitted a marketing authorization application to the European Medicines Agency (EMA) for the use of pridopidine in Huntington’s disease, according to the release, and the biotech is in discussions with the FDA about next steps for Huntington\'s in the U.S. A decision from the EMA\'s Committee for Medicinal Products for Human Use is expected in the second half of 2025, the Ferrer spokesperson said.“Ferrer continues to grow their already significant presence throughout Europe and key international markets with particular focus on innovative products for rare diseases,” Prilenia CEO Michael Hayden, Ph.D., said in the release. “We believe that this partnership has the potential to accelerate the delivery of pridopidine to the thousands of people who are waiting for a new treatment option as well as broaden its impact through additional indications in the future.”In addition to Huntington’s and ALS, Prilenia is also testing pridopidine in vanishing white matter disease, an inherited genetic disorder, and exploring the asset’s potential in other neurodegenerative diseases, according to the biotech’s website.Ferrer’s R&D work focuses on neurological and pulmonary diseases, and the Spanish pharma has bought into ALS assets in the past. In March 2024, the firm pledged more than $121.7 million in upfront and potential milestone payments for the ex-U.S. rights to Verge Genomics’ ALS candidate, which is currently in a phase 1b proof-of-concept trial.

来源：药讯随说 3月12日（当地时间），Clene Inc及其子公司Clene Nanomedicine宣布其纳米药物CNM-Au8在肌萎缩侧索硬化（ALS，渐冻症）临床研究（HEALEY ALS平台研究）患者的交叉方案长期生存事后分析结果积极。 HEALEY ALS平台研究（主研究：NCT04297683）为一项评估多种方案在稳定背景治疗（利鲁唑和/或依达拉奉）ALS患者中有效性、安全性、PK和PD的全球多中心、随机、双盲、安慰剂对照的确证性III期临床研究。研究主要终点为生存改善，次要有效性终点为ALS临床恶化事件延迟时间。 平台试验包括7个方案，分别为：Zilucoplan（泽勒普肽，方案A：NCT04436497）、Verdiperstat（方案B：NCT04436510）、CNM-Au8（方案C：NCT04414345）、Pridopidine（普利多匹定，方案D：NCT04615923）、Trehalose（海藻糖，方案E：NCT05136885）、ABBV-CLS -7262(方案F：NCT05740813）、DNL343（方案G：NCT05842941）。 方案A：患者3：1随机接受24周活性治疗（每天一次皮下注射Zilucoplan，0.3mg/kg）或匹配安慰剂治疗。 方案C：患者2：1随机接受108周活性治疗（30mg）或匹配安慰剂治疗. 本次公布结果如下： 1、全分析集（FAS）： 中位生存：CNM-Au8 30mg组 (方案C,n=59)和方案A对照组（n=162）中位生存天数分别为951天、753天，增加198天（6.5月）； 限制平均生存时间（RMST）：协变量校正RMST增加124天（4.1月）（95%CI：3-245；p=0.045）； 2、中-重度ALS患者增强获益： 基线血清NfL（神经丝轻链蛋白）≥33pg/ml和TRICALS风险得分-6.5至-2.5间人群中，方案C（n=51）较方案A（n=120）中位生存时间从589天提高至951天，增加11.9月； 死亡风险降低44%（Cox HR=0.556;95%CI:0.367-0.842;p=0.006),RMST增加197天（6.5月，95%CI：65-329；p=0.004）； 3、符合入选标准集【血清NfL（神经丝轻链蛋白）≥33pg/ml，TRICALS风险得分-6.5至-2.5间，基线慢肺活量≥60%，发病时间≤36月】 方案C（n=40）较方案A（n=94）中位生存时间从628天提高至1079天，增加14.8月（451天）； 死亡风险降低49%（Cox HR=0.514;95%CI:0.319-0.83;p=0.006),RMST提高215天（7.1月，95%CI：70-360；p=0.004） CNM-Au8是一种具有催化活性、表面清洁、浓缩的金元素多面纳米晶体的水悬浮液。通过提供和/或接收电子驱动大脑中关键的细胞能量生成反应，通过增加神经元和胶质细胞对疾病相关应激的耐受性，使神经修复和髓鞘再生成为可能，并减少有害的反应性分子。 免责声明： 文章内容仅供参考，不构成投资建议。投资者据此操作，风险自担, 关于对文中陈述、观点判断保持中立，不对所包含内容的准确性、可靠性或完整性提供任何明示或暗示的保证。请读者仅作参考，并请自行承担全部。 联系我们 SIT 2025 展位火热预定中！ 扫码立即咨询 电话：13816031174 （同微信） 赞助形式包括但不仅限于演讲席位、会场展位、会刊彩页、晚宴赞助、会议用品宣传等。 点击此处“阅读全文”咨询更多精彩！