URMC Clinical Researchers Pave the Way for New Huntington’s Drug

Aug. 21, 2023

A team of researchers at the University of Rochester Medical Center (URMC) were instrumental in the approval Friday by the Food and Drug Administration (FDA) of a new drug to treat Huntington’s disease.  Valbenazine is the ninth compound for neurological disorders in which URMC has played a major role in the drug development process, and only the third drug authorized by the agency to treat this rare, inherited neurodegenerative disorder. 

The FDA approval was based on the results of KINECT-HD, a phase 3, randomized, double-blind, placebo-controlled study that enrolled 128 participants at 46 sites across North America in collaboration with the Huntington Study Group (HSG) and Neurocrine Biosciences. The URMC Center for Health + Technology (CHeT) served as the key contract research organization for the study, providing project and data management, monitoring, and quality assurance services. 

The results of the KINECT-HD clinical trial were published in The Lancet Neurology in June.  The study showed that valbenazine—a drug previously approved to treat tardive dyskinesia—alleviated chorea, involuntary movements that are a common and often debilitating symptom of Huntington’s disease. 

KINECT-HD was the first phase 3 clinical trial to use a new patient-reported outcome measure developed by Chad Heatwole, MD, director of CHeT.  The research tool, called the Huntington’s Disease Health Index (HD-HI), was developed with the support of HSG, and is designed to identify and track outcomes that are meaningful to patients and satisfy FDA guidance.  The HD-HI tool revealed that valbenazine was associated with a reduction in disease burden related to mobility, abnormal movements, and hand and arm function.

Cynthia Casaceli, MBA, a research associate professor of Neurology, along with the with the Clinical Trials Coordination Center (CTCC) operational team members, provided scientific, technical, logistical, and operational support for the KINECT-HD study, as well as the ongoing open-label follow-on study KINECT-HD2.   Frederick Marshall, MD, served as the URMC site principal investigator for the study.

CTCC, a unit of CHeT, is a unique academic-based research organization with decades of experience working in a regulated environment with industry, foundations, and governmental researchers in bringing new therapies to market. Researchers with CTCC, which is currently directed by Melissa Kostrzebski, MS, MBA, have played a central role in bringing nine new drugs to market, including pramipexole, entacapone, rasagiline, and rotigitine for Parkinson’s, tetrabenezine and deutrabenazine for Huntington’s, dichlorphenamide for periodic paralysis, and omaveloxolone for Friedreich’s ataxia.