News

Levels of KP Metabolites in Blood, CSF Unlikely as Disease Biomarkers

Metabolites seen as potential biomarkers of Huntington’s disease — called kynurenine pathway metabolites — showed little evidence of Huntington’s-specific activity in a recent study. While researchers could not entirely rule out kynurenine pathway metabolites as possible treatment targets, their results suggest that the disease is not well represented by the…

PROOF-HD Phase 3 Pridopidine Trial Reaches 25% Enrollment

Prilenia Therapeutics has enrolled more than 120 patients in PROOF-HD, a Phase 3 clinical trial investigating the efficacy and safety of oral pridopidine in early stage Huntington’s disease. That amounts to 25% of the target enrollment of up to 480 patients, the company announced. The trial is…

Nonprofit Pushes for US Bill to End Wait for Patient Benefits

U.S. lawmakers have again introduced legislation that would speed up the process for Huntington’s disease (HD) patients to get Medicare and Social Security Disability Insurance (SSDI) benefits. The Huntington’s Disease Disability Insurance Access Act of 2021 — S 868/ HR 2050 — would waive the mandatory two-year…

GENERATION HD1 Trial Fails to Show Benefits of Tominersen

Treatment with tominersen failed to show evidence of clinical benefit in adults with Huntington’s disease during the Phase 3 clinical trial GENERATION HD1. Roche, the company developing tominersen, decided to pause dosing in the clinical trial about a month ago, after a pre-planned review of trial data…

‘Let’s Talk About’ Huntington’s Disease Awareness in May

Activities are underway worldwide to mark Huntington’s Disease Awareness Month, set aside each year to call attention to the neurodegenerative disorder that is thought to affect three to seven per every 100,000 people of European ancestry. May 15 is International Huntington’s Disease Awareness Day. Patients, caregivers, family, and friends…

FDA Gives Go-ahead for Phase 1/2 Trial Testing VY-HTT01 Gene Therapy

The U.S. Food and Drug Administration (FDA) has granted Voyager Therapeutics permission to start a clinical trial testing VY-HTT01, its investigational gene therapy for Huntington’s disease. Voyager is planning to launch the Phase 1/2 clinical trial, called VYTAL, later this year. VYTAL will be a dose-escalation study, meaning that…