Quotient Therapeutics Ltd.

本届大会以“共建创新生态 共赢产业未来”为主题，聚焦生物医药出海与国际化创新、创新药投融资、医疗器械等产业发展风向标，打造“高端化、国际化、专业化、立体化”的年度盛会。 生物制药领域，交易合作与科学发现同样重要。如果没有合作，诸如Keytruda等很多创新成果，都不会企及如今的成就高度。 事实上，随着新的专利悬崖迫近，MNC近年都开始不遗余力地物色下一款重磅产品。而在并购、引进等合作模式之外，巨头们也尝试用更加灵活的方法，提高掘金冒险的胜率。 去年7月，辉瑞与风险投资机构Flagship Pioneering合作，利用后者创业生态系统中的技术，开发10种新的候选药物。这种新的合作模式，就标志着一种更具战略性的方法，将制药商与初创企业联系起来，共同寻找新药。 Flagship将这个合作称之为创新供应链合作伙伴关系（Innovation Supply Chain），双方将共同努力，寻找创造符合辉瑞战略的新药机会。辉瑞则将为选定的开发项目提供资金，之后可以选择引进或收购该资产。 这些项目旨在满足制药公司的需求，同时引入能够生产这些药物的初创公司，有助于加快研究的启动和运行，使研发过程更加高效。 那么，这样的合作模式对生物技术行业意味着什么？Labiotech对此进行了一些分析。 1 “预先”合作 从本质上讲，辉瑞和Flagship同意战略性地集中资源，创建一种新的研发模式。 根据合作协议，两家公司会各自提供5000万美元的前期投资，辉瑞将利用Flagship背后丰富的生物技术平台，共同探索10个新项目。若项目成功上市，Flagship及其旗下公司将有资格获得高达7亿美元的里程碑付款。 Flagship Pioneering普通合伙人兼Pioneering Medicines总裁Paul Biondi解释道，Flagship将在由40多家公司和生物技术平台组成的生态系统中，寻找可以将这种药物投入使用的初创公司。 之后，Flagship和辉瑞可以对项目进行投资，获得一些数据，证明这个想法是否可行。诸如此类的各种研究正在进行，以此确定可以开发的项目。 截至目前，两方已经官宣了两个项目：今年6月，与ProFound Therapeutics合作寻找一种新的减肥药；8月，与Quotient Therapeutics合作寻找心血管和肾脏疾病的靶点。这两家初创公司都由Flagship创立。 这种合作模式与其他类型的早期合作（如孵化器或加速器）的区别在于，辉瑞自始至终都在进行战略投入。Biondi解释说：“这像是预先合作（pre-collaboration）。” 通常情况下，大药企会在更广阔的领域寻找感兴趣的初创公司。至于孵化器和加速器，可以为初创企业提供资源和指导，还会帮助他们与大药企建立关系，不过，产品的推进仍由初创公司主导。 而在辉瑞与Flagship的合作中，是预先确定他们想要的药物，后者提供一家拥有相关技术的初创公司。按照Biondi的说法，这就是创新供应链的本质。 2 另一种外包研发 辉瑞和Flagship之间的合作被外界视为新范式的先河，也是整个行业转向利用初创公司进行研发的一部分：根据医疗保健投资公司HBM Partners的数据，2013-2018年，63%获批的药物最初来自较小的公司。 当大药厂研发管线“积压”，尤其在许多畅销药物将在十年内失去专利保护的情况下，进行内部研发的压力非常大。而与初创公司合作可以提高效率：小公司更为灵活，研发速度比巨头更快。此外，他们还可以为大药厂提供创新和突破的窗口。 “大药厂非常关注如何提高研发效率。”生物技术投资者、赛诺菲前高管Laurent Van Lerberghe表示，虽然制药公司会在内部进行一些研发，但“他们会越来越多地寻找其他资源，与不同的生物技术合作，而不总是通过收购来实现”。 包括默克、赛诺菲和诺和诺德在内的MNC，在他们的业务中加入了风险投资部门，专门关注与他们相关的领域，这使制药公司能够更快地获得初创公司的科学专业知识和他们在开发的药物。 随着人工智能等工具的迅猛发展，药企也正在寻找更多可能性。像Flagship这样的模式可能是一个答案。 3 对初创公司意味着什么？ Van Lerberghe表示，在生物技术初创公司的世界里，如果没有大药企或其他公司在某个阶段收购这些资产，大多数新药公司都无法生存下去。 Vivan Therapeutics创始人兼首席执行官Laura Towart对此表示认同，Flagship和辉瑞这样的合作模式可能会让正在开发大量资产的初创公司受益。 “我们发现了很多新药物组合和新靶点，但这需要花费大量的时间去测试。而如果和大药企建立了联系，后者可以对我们的资产进行评估，确定他们是否感兴趣。如果这家不感兴趣，我们也可以去联系下一家。”Towart补充说。 这样的模式可以帮助初创公司减少损失，确定应该推进或取消哪些项目。Towart认为：“如果你不知道有一个明确的买家，那很难在一个项目上投入大量资金。” 让初创公司有机会确保会和制药公司交易的合作模式，也可能帮助前者在充满挑战的市场中创造融资机会。 Ernst & Young的报告称，今年的行业交易活动呈上升趋势，融资轮数有所增加，但数量有所减少，处于早期阶段的企业往往被甩在后面。 Flagship的模式——提供一个全面的生物平台，根据制药公司的需求进行研发，也可以增加初创公司成功的几率。“更严格的研发过程可以大大提高成功率”。 这意味着初创公司和风投公司都可以面临更少的风险，获得更多的收益。“对于风投来说，投资25-30家公司，可能只有1-2家会成功，”Van Lerberghe说，“如果（Flagship的模式）能让它在25家公司中使2-3家获得成功，回报率将变成天文数字”。 4 要合作！ Biondi和Van Lerberghe都认为，在未来几年，制药公司、风险投资公司和初创公司之间，会出现更多的合作模式。毕竟，大药企需要填补管线，而几乎所有的初创公司都需要大药企的支持才能生存。 Flagship也与GSK建立了类似的合作关系，并计划进一步推广这种合作方式。 7月底，GSK宣布与Flagship Pioneering达成合作。Flagship旗下40多家生物制药公司将作为一个“组合”，与GSK合作开发10款新型药物和疫苗，从呼吸系统疾病和免疫疗法开始，拓展到其他疾病领域。 虽然并不是所有公司都可以选择和Flagship一模一样的合作模式，但Biondi预测，会有更多公司创造出适合自己的合作模式。可能是其他促进研发的联盟，比如与研究和医疗机构的联盟——Vivan Therapeutics正在与癌症研究所（ICR）合作开发多靶点药物。Towart表示，ICR资金充足，他们可以在整个试验过程中使用药物并进行推广。 Biondi补充道，无论如何，与大药企建立合作渠道对Biotech来说总是一件好事。“每一家成功的公司，都以某种方式或形式，在与他人合作”。 — 结语 — 生物技术合作伙伴关系是该行业的基石，为相关各方带来了许多好处。我们经常看到大型制药公司与小型生物技术公司合作，开发出具有巨大潜力的候选药物。 并购、研究合作、战略联盟、授权许可等是比较常见的方式，此外还有合资企业、联合开发协议、股权投资等不同类型的伙伴关系。而辉瑞与Flagship的尝试，给业界呈现了另外的可能。 这些生物技术伙伴关系为制药公司提供了一种新的药物或技术，这些新药物或技术可以给停滞不前的管线重新注入活力。对于生物技术公司来说，合作伙伴关系意味着他们可以获得所需的资源，继续开发技术。 归根结底，基于一个分享工作和专业知识的平台，可以加快产品推向市场的过程。 参考文章： 1、New partnership model on the rise: how VCs are helping Pharma hunt for their next blockbuster；labiotech 2、The ABC of biotech partnerships；labiotech 同写意媒体矩阵，欢迎关注↓↓↓

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here . Dear readers: After almost seven years, yesterday was my last day with Endpoints News . Starting the Endpoints Weekly and growing it from 0 to now more than 120,000 subscribers has definitely been one of the highlights of my time here. I appreciate all your feedback and kind words over the years. The Saturday recaps will keep coming — you will be in good hands as Max Gelman and Nicole DeFeudis take over this newsletter. Thank you so much for reading. — Amber Tong The FDA on Friday authorized the emergency use of Novavax’s updated Covid vaccine, just over a week after clearing mRNA boosters from Pfizer and Moderna . Unlike Moderna and Pfizer’s shots which target the variant KP.2, Novavax’s vaccine targets a “parent strain” known as JN.1. Recursion’s AI platform will be put to the test as the company prepares to read out results from 10 trials in 18 months, Andrew Dunn and Max Gelman reported this week. The first Phase 2 readout, coming next month, is for a drug that predates Recursion — a decades-old compound called tempol that’s being tested in patients with a brain condition called cerebral cavernous malformation. “It feels like we’re at that moment, people launching things,” CEO Chris Gibson told Endpoints News. The CD47 pathway, once one of immuno-oncology’s great hopes , has been plagued by safety risks and disappointing readouts. Some big pharma companies have terminated or narrowed their efforts, while other drugmakers still believe in harnessing the immune system’s “don’t eat me” signal — and some try to move CD47 beyond cancer. Endpoints’ Max Gelman dives into the challenges and what’s ahead. Pfizer is joining up with Flagship Pioneering startup Quotient Therapeutics to identify genetic mutations that appear throughout life and make individual cells more susceptible or resistant to disease. They’ll use the learnings as a starting point for new cardiovascular and renal disease treatments. The research is part of a broader collaboration between Flagship and Pfizer announced last year. Alnylam’s siRNA drug vutrisiran will face tough competition in transthyretin amyloid cardiomyopathy, with Pfizer’s Vindaquel currently dominating the market and BridgeBio’s acoramidis following close behind. Ayisha Sharma unpacks new data from the European Society of Cardiology annual meeting in London. Stay tuned for more of her conference coverage. SPOTLIGHT Once-promising cancer drugs appear to protect mice from Alzheimer’s Neurocrine’s schizophrenia data are good enough for Phase 3 — but maybe not enough to beat Karuna PHARMA R&D FDA+ STARTUPS DEALS MANUFACTURING HEALTH TECH LAW

Today, a brief rundown of news from Novo Nordisk and Ventyx Biosciences, as well as updates from Novartis and Pfizer that you may have missed.A pooled analysis of data from four trials of Novo Nordisks obesity drug semaglutide found treatment reduced the risk of cardiovascular death or worsening heart failure by 31% compared to placebo. The results, which were published in The Lancet on Friday, could help Novo expand use of the drug into heart failure. The company had previously applied to the Food and Drug Administration for such an approval, but recently withdrew it in order to submit additional relevant data. The Lancet analysis, which focused on 3,700 participants across the four trials who had heart failure with preserved ejection fraction, was post-hoc. Ned PagliaruloMartin Auster, chief financial officer of Ventyx Biosciences, has departed the inflammatory disease drug developer, the company announced Friday. He will be replaced on an interim basis by Roy Gonzales, who will serve as interim principal financial officer for SEC reporting purposes. Ventyx, which has hit setbacks in developing a so-called TYK2 inhibitor, said it will search for a permanent replacement. Ned PagliaruloEmergent BioSolutions on Thursday said the Food and Drug Administration granted extended an approval of its smallpox vaccine so it can also be used to prevent mpox in people at high risk for infection. The single-dose vaccine, called ACAM2000, was initially cleared for smallpox back in 2007. Thursdays approval was based on a previous human trial and animal testing, which showed the vaccine to be effective in protecting against mpox, according to Emergent. Mpox was declared a public health emergency by the World Health Organization on Aug. 14 after outbreaks in parts of Africa. Jynneos, from Bavarian Nordic,is the only other available vaccine for mpox in the U.S. Delilah AlvaradoCancer drugmaker Repare Therapeutics plans to lay off 25% of its workforce as part of a strategic reprioritization disclosed Wednesday. The company will focus on its more advanced, clinical-stage programs and scale back preclinical research. The layoffs are expected to mainly affect staff in its preclinical group. The company said the reorganization will generate annual savings of approximately $15 million, pushing out its cash runway into the second half of 2026. Delilah AlvaradoFlagship Pioneerings ongoing research alliance with Pfizer has yielded more leads for the pharmaceutical giant, the companies said Wednesday. The company creation firm will work with its portfolio company Quotient Therapeutics on two research programs to support development of drugs for heart and kidney diseases. In June, Flagship announced it would tap ProFound Therapeutics to look for promising obesity drugs Pfizer can choose to advance. Gwendolyn Wu '