BHVN - 2 Biotechs Tackling the Challenge of Lou Gehrig's Disease
Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease, is a progressive neurodegenerative disease in which the nerve cells in the spinal cord and brain slowly die, leading to impairment of voluntary muscle movement. Patients lose the ability to eat, breathe, walk, and talk as the disease advances. In many cases, patients become completely paralyzed. Approximately 5,000 patients are diagnosed annually in the U.S., according to the ALS Association.
Four years ago, the Ice Bucket Challenge campaign raised more than $115 million for the ALS Association. In turn, it funded more than 200 research projects to the tune of $84 million, resulting in the discovery of five new genes that could be culprits of the disease. New approaches in development provide hope that one or even a few new therapies lie on the horizon.
A cutting-edge clinical trial in ALS commenced this month at Massachusetts General Hospital in Boston. Dubbed a Platform Trial, it will compare five drugs, each from a different company, against each other and a group receiving a placebo. Traditionally, each drug would have had its own clinical trial. But in this case, 30 companies applied to have their drugs evaluated. Biohaven Pharmaceutical Holding (NYSE: BHVN) and Ra Pharmaceuticals (NASDAQ: RARX) each had a drug selected for inclusion.