Clinical research
Poxel presented positive histology data from its Phase II DESTINY-1 trial in non-alcoholic steatohepatitis (NASH), with its primary efficacy endpoint of liver fat decrease at 36 weeks.
The FDA granted rare pediatric disease designation to AVR-RD-04, an experimental gene therapy treatment for the lysosomal storage disease cystinosis, AVROBIO announced Tuesday.
Alzheon Inc. unveiled promising data from a Phase II biomarker trial of its investigational drug ALZ-801 to treat early Alzheimer’s disease.
Wave Life Sciences presented promising early data from its Phase Ib/IIa SELECT-HD trial in Huntington’s disease with evidence of biomarker improvements.
First Wave BioPharma announced that it is restarting its adrulipase program for the treatment of exocrine pancreatic insufficiency in cystic fibrosis and chronic pancreatitis.
The past week saw promising developments in candidate therapies for cancer, congenital hyperinsulinism, chronic cough and COVID-19 vaccines.
Virios Therapeutics announced Monday its lead asset IMC-1 fell short of its primary endpoint in the Phase IIb FORTRESS study in patients with fibromyalgia.
The FDA has two PDUFA dates on its calendar this week, with two extra days slotted for an advisory committee meeting to evaluate drug applications from three companies.
Intellia Therapeutics reported early, positive signs from its in vivo gene therapies for both transthyretin (ATTR) amyloidosis and hereditary angioedema Friday.
AstraZeneca’s rare disease subsidiary Alexion posted positive late-stage results from a potential complementary treatment for paroxysmal nocturnal hemoglobinuria.
PRESS RELEASES