Sanofi SA's Genzyme Corp. has agreed to pay Denali Therapeutics Inc. $125 million to kickstart development of multiple small-molecule inhibitors of receptor-interacting serine/threonine-protein kinase 1 (RIPK1), a signaling protein that regulates inflammation and cell death in a variety of diseases. The candidates could potentially benefit people with Alzheimer's disease (AD), amyotrophic lateral sclerosis (ALS) and multiple sclerosis. Should all succeed, Denali could earn nearly $1.1 billion in milestone payments from Sanofi.
Shares of Strongbridge Biopharma plc (NASDAQ:SBBP) climbed 58 percent Wednesday to close at $6.40 as Novo Nordisk A/S agreed to pay $145 million plus royalties for U.S. and Canadian rights to the adult growth hormone deficiency therapy (AGHD) Macrilen (macimorelin), rights that Strongbridge itself had purchased less than a year ago from Aeterna Zentaris Inc. for $24 million up front. Novo also made an equity investment in the company.
Eli Lilly and Co. has agreed to pay Dicerna Pharmaceuticals Inc. $100 million up front while investing $100 million more in the company as part of a new licensing and research collaboration agreement focused on the discovery, development and commercialization of medicines aimed at "more than 10 targets" in cardiometabolic disease, neurodegeneration and pain indications.
Shares of Syndax Pharmaceuticals Inc. (NASDAQ:SNDX) fell 17.1 percent to $5.01 Friday on news that adding its HDAC inhibitor, entinostat, to an aromatase inhibitor failed to deliver a statistically significant improvement in the length of time women with hormone receptor-positive, HER2-negative breast cancer lived without worsening of their cancers. The outcome, from the ongoing phase III study E2112, dashed hopes of an early regulatory filing, putting the focus on the trial's other co-primary endpoint, overall survival (OS).
89bio Ltd., a San Francisco-based startup focused on nonalcoholic steatohepatitis (NASH) and other liver and metabolic disorders, has closed a $60 million series A financing to advance an asset acquired from Israel's Teva Pharmaceutical Industries Ltd. through proof of concept. Currently in phase I testing, the long-acting glycopegylated fibroblast growth factor 21 (FGF21) analogue arrived at the company with positive preclinical data and an approved investigational new drug application.
Alexion Pharmaceuticals Inc. has negotiated an exclusive license to develop and commercialize up to four preclinical RNAi molecules for complement-mediated diseases using Dicerna Pharmaceuticals Inc.'s liver-focused gene-silencing platform, GalXC. Dicerna will receive $22 million up front and an equity investment of $15 million for the first two candidates and stands to receive $20 million more if Alexion exercises an option to advance preclinical GalXC RNAi molecules for two additional targets within the complement pathway.
Zymeworks Inc. has been recruited by Denmark's Leo Pharma A/S to help discover and develop new bispecific antibodies for dermatology indications. That the deal's value could exceed $480 million might be the headliner for companies that have yet to capture big pharma's attention. Zymeworks, by contrast, has already inked technology deals with Merck & Co. Inc., Glaxosmithkline plc, Janssen Biotech Inc., Daiichi Sankyo Co. Ltd., Eli Lilly and Co. and Celgene Corp.
Initial data from ongoing studies of two T-cell receptor (TCR) programs presented by Adaptimmune Therapeutics plc at the European Society for Medical Oncology 2018 Congress showed what Rafael Amado, the company's president of R&D, called encouraging safety, "dose appropriate persistence and expansion, and early but transient evidence of antitumor activity in one ovarian cancer patient."
Privately held cancer drug developer TP Therapeutics Inc. said a newly completed $80 million mezzanine financing will help move its lead candidate, repotrectinib, into a potentially pivotal phase II study in early 2019. The company, which is at first targeting ROS1-positive non-small-cell lung cancer (NSCLC) and NTRK-positive solid tumors, specializes in addressing treatment resistance.
SAN FRANCISCO – Dynamic as ever in the wake of this summer's mixed news on Biogen Inc. and Eisai Co. Ltd.'s anti-beta-amyloid approach, drug developers in the Alzheimer's field are increasingly looking to genetics and biomarkers as they seek new paths of attack for the global scourge. Now, with the success of precision approaches in cancer, scientists are also beginning to think about combination approaches to the complex neurodegenerative disease, representatives of Denali Therapeutics Inc., E-Scape Bio Inc., Alkahest Inc. and Neurotrope Inc. said Thursday at the BIO Investor Forum.
