Phase II
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.
BrainStorm Cell Therapeutics announced the publication of peer-reviewed data from a Phase II trial in progressive multiple sclerosis Thursday showing “preliminary evidence of efficacy.”
There’s a new collaboration in the Parkinson’s disease development space, Cardiff Oncology kicks off a mid-phase study in colorectal cancer and Ocugen published a new review of COVAXIN.
After reporting a failed clinical trial of ELX-02 for cystic fibrosis (CF), Eloxx Pharmaceuticals plans to shift its focus and use the drug to treat Alport syndrome, a rare kidney disease.
PRESS RELEASES