Deaths associated with Astellas Pharma’s gene therapy program for patients with X-linked Myotubular Myopathy underscore some of the risks of developing potential one-and-done treatment options for serious diseases.
Alexion, the unit within global pharmaceutical firm AstraZeneca that focuses on finding treatments for rare diseases, is halting clinical trials for Ultomiris (ravulizumab) for adults with amyotrophic lateral sclerosis.
Biogen’s quest to develop additional medicines for Alzheimer’s disease beyond the company’s newly approved aducanumab hit a clinical snag as the anti-tau antibody gosuranemab failed to meet endpoints in a Phase II study and was discontinued.
Helsinn Group, based in Lugano, Switzerland, and MEI Pharma, with headquarters in San Diego, announced they were discontinuing their Phase III trial of pracinostat with azacytidine in acute myeloid leukemia (AML).
