“We clearly see an active drug here,” PTC Therapeutics Inc. CEO Matthew Klein said of the 15-lipoxygenase inhibitor vatiquinone for Friedreich’s ataxia (FA), tested in a phase III study called Move-FA that missed the primary endpoint of statistically significant change in modified FA Rating Scale score at 72 weeks. The company will “take one step at a time” decisions about the drug, analyzing the results and then consulting with the U.S. FDA regarding how to proceed, he said. Meanwhile, Wall Street wasn’t happy, and South Plainfield, N.J.-based PTC’s shares (NASDAQ:PTCT) closed May 24 at $46.95, down $11.46, or 19.6%. Read More
A week ahead of its May 29 PDUFA date, the U.S. FDA has approved Xacduro (sulbactam for injection; durlobactam for injection) for treating one of the toughest and most deadly infections, carbapenem-resistant Acinetobacter baumannii, in adults. Read More
In its bid to become, as Chairman and CEO David Hallal said, the “world’s most indispensable cell and gene therapy technology company,” Elevatebio LLC disclosed a $401 million series D round with support from new and existing investors. At the same time, the company’s Life Edit Therapeutics Inc. affiliate inked a potential billion-dollar collaboration focused on gene editing therapies. Read More
The U.S. Centers for Medicare & Medicaid Services (CMS) is flexing its new authority in a proposed rule intended to clamp down on drug prices by providing more transparency in the Medicaid program. Read More
Cansino Biologics Inc. reported positive data in a phase IIb trial evaluating the heterologous mRNA vaccine CS-2034 booster compared to an inactivated vaccine to prevent SARS-CoV-2 infections. Read More
India has revised its policy to make quality tests by government-certified laboratories mandatory for cough syrups to be exported beginning June 1. The decision is the result of numerous safety alerts involving the death of children. Read More
Biopharmas raising money in public or private financings, including: Ambrx, Biodexa, Dermata, Inprother, Kamada, Ocugen, Phathom, Resolutionrx. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: APT, Advanz, Alvotech, Apexigen, Astrazeneca, Area, Bioscenic, Clearpoint, Clover, Concentra, Endo, Erytech, Keyuan Xinhai, Maia, Pherecydes, Pluristyx, Pyxis, UCB. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Affimed, Antengene, Blueprint, Boehringer Ingelheim, Cumberland, Indivior, Junshi, Pharmabcine, Pharmessentia, Regenxbio, Rocket, Vega, Y-mabs. Read More
