Radical Change in the Huntington Disease Research Landscape

Michael Hayden, MBChB, PhD, CEO of Prilenia Therapeutics and a Killam Professor at the University of British Columbia, describes the current research landscape for Huntington disease.

Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic starting at around 40 years of age. Persons with Huntington disease suffer from a progressive movement disorder, cognitive decline, psychiatric disturbances, and behavioral symptoms. All symptoms progress which eventually leads to immobility, dementia, and premature death.

As Dr. Hayden explains, the research landscape for Huntington disease has changed as three large clinical trials have been terminated. The first is Roche’s phase 3 GENERATION HD1 trial of tominersen in adults with Huntington’s disease which was terminated based on the investigational therapy’s potential benefit/risk profile for study participants conducted by an unblinded Independent Data Monitoring Committee. The second and third terminations were of Wave Life Sciences’ PRECISION-HD1 and 2 trials of WVE-120101 and WVE-120102 respectively in Huntington disease patients. In both trials, there was a lack of significant change in the levels of the harmful huntingtin protein in trial participants treated with the investigational drug compared to placebo.

Though the news of these terminations has been unfortunate, Prilenia’s PROOF-HD - a phase 3 trial evaluating the efficacy and safety of pridopidine in patients with early stage Huntington disease - is currently recruiting patients and Dr. Hayden is confident that pridopidine is a safe and effective drug for these patients.