Cymabay Therapeutics Inc. said positive interim phase II results for its primary biliary cholangitis (PBC) treatment, seladelpar, won FDA support for extended dosing of the drug, a key go-ahead enabling the company to plan its phase III program. Data on two low doses of the drug suggest it can drive down serum levels of alkaline phosphatase (ALP), a biomarker correlated with reduced need for liver transplant and risk of death, while avoiding elevation of another biomarker, transaminase, that is tied to severe liver damage and was raised in a few cases during tests of higher doses.
A new deal giving Celgene Corp. strategically important rights to a PD-1 inhibitor developed by Beijing-based Beigene Ltd. will tightly bond the companies' fortunes in both the U.S. and China, putting Celgene in charge of development and commercialization of a key Beigene asset, while Beigene takes charge of selling Celgene's top products in China and advancing new ones.
An antithrombin-targeting RNAi therapy for hemophilia, fitusiran, developed by Alnylam Pharmaceuticals Inc. with Sanofi Genzyme, is entering phase III territory. The three-part Atlas trial program, in hemophilia A and B, is expected to yield top-line data in mid to late 2019. Its start, just days before results of an ongoing phase II study are scheduled to be detailed at a Berlin medical meeting, triggers a $25 million milestone payment for Alnylam and marks the kick-off of the last leg in a race to approval with Roche Holding AG's bispecific monoclonal antibody emicizumab.
A new deal giving Celgene Corp. strategically important rights to a PD-1 inhibitor developed by Beijing-based Beigene Ltd. will tightly bond the companies' fortunes in both the U.S. and China, putting Celgene in charge of development and commercialization of a key Beigene asset, while Beigene takes charge of selling Celgene's top products in China and advancing new ones.
Sienna Biopharmaceuticals Inc., a California-based dermatology company developing small-molecule topical therapies for people with a variety of common skin issues, is seeking gross proceeds of up to $74.8 million in an IPO.
Clogged cartridge filters and the trial failure they likely caused battered shares of Windtree Therapeutics Inc. (OTCQB:WINT), sending the stock falling 61.8 percent Friday, or 53 cents, to close at 33 cents on news that its aerosolized medicine for preemies with respiratory distress syndrome, Aerosurf, failed to diminish the need for intubation, missing the trial's primary endpoint.
A new FDA strategy is targeting total elimination of the existing orphan designation request backlog by mid-September and committing to respond to all new requests for it within 90 days of receipt. To get there, Gayatri Rao, director of the Office of Orphan Products Development (OOPD), told BioWorld, her group is reorganizing review staff, working to educate sponsors about how to file better designation requests, and establishing a new FDA Orphan Products Council.
SAN DIEGO – Ambitious venture funds from Russia, China and India are seeking a growing stake in U.S. companies, creating new access to capital for startups. Though offering value beyond money to both investors and the recipients of their funds, international venture capitalists speaking at BIO 2017 said cultural and political frictions can still create challenges.
Xenon Pharmaceuticals Inc. CEO Simon Pimstone said it's going to be hard to ultimately define the reason that the firm's sodium channel Nav1.7 inhibitor, TV-45070, failed to outperform placebo in a phase II postherpetic neuralgia (PHN) trial, but that "if the drug is not fully engaging the targets at the right sites, it doesn't really matter: there's not going to be an effect."
Boston Biomedical Inc. unblinded a global phase III study of its cancer stem cell pathway inhibitor, napabucasin, in advanced gastric and gastroesophageal junction cancer after the trial's data and safety monitoring board (DSMB) determined that combining napabucasin with paclitaxel is unlikely to yield better overall survival (OS) at 36 months than paclitaxel alone.
