Phase II
Athira Pharma announced Tuesday it will study the stand-alone effects of its Alzheimer’s candidate fosgonimeton (ATH-1017). The drug failed to meet the endpoint in a Phase II study earlier this year.
The FDA removed the clinical hold on Sarepta’s investigational treatment for certain types of DMD after the company agreed to adjust its clinical trial protocols.
Boehringer announced that the FDA approved Spevigo for generalized pustular psoriasis flares in adults, the first drug approved for this indication.
It’s been a busy week for research on rare diseases, with several clinical trials from AnaptysBio, Regenxbio, Sangamo and more posting results from ongoing activities.
Shares of AnaptysBio are down more than 16% after imsidolimab failed to meet the endpoint in a Phase II trial assessing its efficacy in hidradenitis suppurativa.
Sanofi announced FDA approval of Xenpozyme for adult and pediatric patients with the rare genetic disease acid sphingomyelinase deficiency, often referred to as Niemann-Pick disease.
Day One Biopharmaceuticals aims to provide a business solution to a moral problem: the inequity in pediatric oncology drug development.
Clover’s SCB-2019 showed a robust immune response to the Omicron BA.5 sub-variant, while BrainsWay, TikoMed and more provide updates on their businesses and pipelines.
Jounce Therapeutics announced that a Phase II trial assessing a combination treatment for non-small cell lung cancer failed to meet its primary endpoint.
Alnylam Pharmaceuticals reported positive Phase II results for cemdisiran, an RNA interference (RNAi) drug developed to treat IgA nephropathy.
PRESS RELEASES