Phase 2 Trial of Oral Therapy PTC518 Launched in US

A Phase 2 trial evaluating PTC518, an investigational treatment for Huntington’s disease, has started in the U.S., PTC Therapeutics announced. The trial, called PIVOT-HD, will run across clinical sites worldwide and is divided into two parts. First, participants will be randomly assigned to a placebo or PTC518…

ANX005 Showing Potential for Early Huntington’s in Phase 2 Trial

Annexon’s investigational therapy ANX005 is showing tolerability and a potential to treat people in the earlier stages of Huntington’s disease or at genetic risk of the disorder, according to interim data from an ongoing Phase 2 trial. ANX005, an antibody designed to inhibit the activation of the complement cascade —…

FDA Grants Orphan Drug Status to SOM Biotech’s Chorea Therapy

The U.S. Food and Drug Administration (FDA) has granted an orphan drug designation to SOM3355, an investigational treatment for chorea, or involuntary jerky movements, associated with Huntington’s disease, its developer SOM Biotech has announced. Orphan drug status helps to encourage the development of therapies for rare…

Phase 2 Trial Recruiting Early-stage Huntington’s Patients to Test Neflamapimod for Improving Cognition

EIP Pharma is launching a proof-of-concept Phase 2 clinical trial to assess the ability of therapeutic candidate neflamapimod to treat cognitive dysfunction in patients with early-stage Huntington’s disease. The trial (NCT03980938), which is currently recruiting, is taking place at the John Van Geest Centre for…