Biohaven Pharmaceuticals, Hospital for Special Care in Patient Trial Testing Treatment for ALS

A relatively new Connecticut-headquartered biopharmaceutical company and a Connecticut hospital are key participants in what is potentially the most comprehensive and promising research effort into finding an effective treatment for amyotrophic lateral sclerosis (ALS) - known as Lou Gehrig’s Disease - which has stubbornly eluded researchers.

Led by Massachusetts General Hospital, in recent weeks the Hospital for Special Care in New Britain, along with five hospitals in Florida, Pennsylvania, and Texas have started a clinical study that will initially enroll 480 volunteers at 54 clinical sites around the country, with researchers testing one of three experimental drugs on groups of 160 patients.  The platform trial will later expand to include two other experimental drugs, and two additional groups of 160 patients.  

A “platform trial” is a clinical trial that involves the testing and evaluating of multiple treatments for a disease simultaneously. The goals of this groundbreaking project are to greatly accelerate the timelines towards effective ALS treatments and provide greater trial access for all those affected by this disease.

The drugs being tested first will come from Biohaven Pharmaceuticals of New Haven; UCB, a Belgian company that recently acquired Cambridge-based Ra Pharmaceuticals; and Clene Nanomedicine of Salt Lake City. Dr. Merit Cudkowicz, a neurologist and Director of the Sean M. Healey & AMG Center for ALS at Mass. General, which is running the trial, has said “We’re going to keep going until we find effective treatments. I expect to have results of the first three [drugs] in about a year.” The Healey ALS Platform Trial is the first-ever ALS Platform Trial aimed at advancing the development of treatments for ALS.

Biohaven’s Verdiperstat, an oral myeloperoxidase inhibitor, has been chosen as one of the first group of regimens to be tested in the platform trial.  Biohaven is a clinical-stage biopharmaceutical company with experienced leadership in industry and academic settings. The company’s portfolio is comprised of innovative, late-stage product candidates targeting neurological and neuropsychiatric diseases, including rare disorders. Since their initial public offering in 2017, the company’s progress has been “fueled by an entrepreneurial organizational structure and an impressive range of experience in drug development along with the confident support of top-tier biopharma investors,” according to the company website.

Biohaven has combined internal development and research with intellectual property licensed from companies and institutions including Bristol-Myers Squibb Company, AstraZeneca AB, Yale University, Catalent, ALS Biopharma LLC and Massachusetts General Hospital, according to officials. Vlad Coric, M.D., is Chief Executive Officer of Biohaven.  Dr. Coric is an Associate Clinical Professor of Psychiatry at the Yale School of Medicine and previously served as Chief of the Yale Clinical Neuroscience Research Unit.

Hospital for Special Care’s Neuromuscular Center has been selected as the only Connecticut trials site. Several sites across the U.S. were selected to bring the trial close to people who want to participate. Hospital for Special Care is the fourth largest, free-standing long-term acute care hospital in the U.S. and the only one in the nation serving adults and children. HSC is recognized for advanced care and rehabilitation, including for neuromuscular disorders and ALS research.

ALS is a rare disorder diagnosed in just over 5,000 people a year in the United States, according to the ALS Association Connecticut Chapter. It affects the nerve cells in the brain and spinal cord, gradually taking patients ability to speak, eat, and, finally, breathe. There is no cure; the life expectancy of a person with ALS averages about two to five years from the time of diagnosis.  Most people who develop ALS are between the ages of 40 and 70, with an average age of 55 at the time of diagnosis. About 90 percent of ALS cases occur without family history. Among those to have succumbed to the disease in Connecticut in recent years were former Travelers CEO Jay Fishman and Eliot Ginsberg, former president and CEO of the Connecticut Center for Advanced Technology.

In a "platform trial" new treatments are added to the platform as they become available, thereby decreasing the gap in time from identification of an exciting therapy to testing. This model, already proven successful in the cancer field, is aimed at greatly accelerate therapy development of effective and breakthrough treatments for people with ALS, according to the Healy Center, by allowing investigators to test more drugs, increase patient access to trials and reduce the cost by quickly and efficiently evaluating the effectiveness of multiple therapies. The HEALEY ALS Platform Trial, according to an announcement by Mass. General this month, will be “perpetual and will add clinical sites, participants and experimental treatments until a cure for ALS is discovered.”

Biohaven points out that because different treatments are tested using a common master protocol and infrastructure that is shared, data from participants in all placebo groups for each regimen can be pooled. This increases the chance that a participant will be assigned active treatment instead of placebo.

Patients will initially be treated for 24 weeks in the clinical trial.  All the participants in the study, whether they receive an experimental medicine or a placebo initially, will get the actual drug under an open label provision after six months. ALS patients who want to participate in the trial can contact HEALEYALSPlatform@mgh.harvard.edu.  The Hospital for Special Care can be contacted at nschmitt@hfsc.org.