The U.S. Food and Drug Administration placed a clinical hold on Larimar Therapeutics’ lead Friedreich’s ataxia (FA) asset, CTI-1601, after the company reported deaths in the highest dose levels of a nonhuman primate toxicology study.
Reata Pharmaceuticals Inc.’s Part 2 portion of the MOXIe Phase 2 trial of omaveloxolone in patients with Friedreich’s ataxia (FA) met the primary endpoint of change in the modified Friedreich’s Ataxia Rating Scale relative to placebo after 48 weeks of treatment.
Rockville, Maryland-based RegenxBio entered a license deal with Pfizer for a gene therapy for Friedreich’s ataxia, the most common hereditary ataxia.
Shares of Voyager Therapeutics skyrocketed after the company announced a collaborative deal to develop a gene therapy treatment for Parkinson’s disease with Neurocrine Biosciences.
Horizon Pharma ended a late-stage trial that was testing Actimmune as a treatment for a rare neuromuscular disorder that has no approved medicines.