In Regeneron Pharmaceuticals Inc.’s phase I/II Chord study of 12 children with genetic hearing loss, 10 out of 11 have shown improvements after being treated with a gene therapy. “What is really remarkable about this type of therapeutic approach is that the first people who are going to see the impact are not actually the physicians – it’s the families,” Jonathon Whitton, vice president and Regeneron’s auditory global program head, told BioWorld.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Arcutis, Axsome, Biocryst, OSE, Pepgen, Redx, Sanofi, Teva.
Kyorin Pharmaceutical Co. Ltd. struck two licensing deals recently, including one with Hyfe Inc. Feb. 25 to develop the world’s potential first prescription digital therapeutic for chronic cough in Japan.
The BioWorld Infectious Disease Index (BIDI) faced challenges throughout the year but narrowed its losses, ending down 6.28% after hitting a low of -25.12% in late April. By October, the index was down 12.07% and had declined 8.86% for the year as of Nov. 22. However, in January, the BIDI rebounded slightly, gaining 1.85%.
Citing recent executive orders that suggest additional cuts to the federal workforce may be in the offing, U.S. Sens. Patty Murray, D-Wash., and Jeanne Shaheen, D-N.H., wrote to Health and Human Services Secretary Robert Kennedy urging him to end “indiscriminate cuts that will cause lasting harm to FDA’s public health mission” and to protect the agency’s statutory obligations.
Cosette Pharmaceuticals Inc. announced plans to acquire Adelaide, Australia-based Mayne Pharma Pty Ltd. for AU$672 million (US$430 million) to grow its women’s health and dermatology franchise in the U.S. Under terms, Cosette, of Bridgewater, N.J., will acquire 100% of Mayne’s shares at AU$7.40 per share in cash. The price represents a 37% premium to Mayne’s closing share price on Feb. 20. The deal is expected to close in the second quarter of 2025.
Pepgen Inc. seems to have gained a leg up on competitors in early data with PGN-EDODM1 in myotonic dystrophy type 1 (DM1), and shares of the Boston-based firm (NASDAQ:PEPG) closed Feb. 24 at $2.29, up 92 cents, or about 67%. The company unveiled initial positive data from the 5- and 10-mg/kg dose cohorts in the ongoing Freedom-DM1 phase I study with PGN-EDODM1, which deploys Boston-based Pepgen’s Enhanced Delivery Oligonucleotide technology to deliver a therapeutic oligonucleotide that is designed to restore the normal function of MBNL1, a key RNA splicing protein.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Elixirgen.
RSS
