Amylyx’s ALS Drug to Enter a Clinical Trial in 2016

Amylyx Pharmaceuticals was recently awarded $600,000 from the ALS Finding a Cure Foundation and the Cure Alzheimer’s Fund, in addition to an estimated $1.3 million raised through private investors, to develop its lead drug candidate AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS) in a clinical trial planned for 2016.

ALS is characterized by inflammation and nerve cell death resulting from the progressive degeneration of motor neurons. Subsequent muscle atrophy in patients causes difficulties in speaking, swallowing, and eventually breathing.

“Across all neurodegenerative diseases from Alzheimer’s to ALS, one of the major sources of neuronal cell death is inflammation and oxidative damage,” said Rudy Tanzi, PhD, Professor of Neurology at Harvard Medical School, Director of the Genetics and Aging Unit at Massachusetts General Hospital, and Chairman of Amylyx’s Scientific Advisory Board, in a press release. “If we can prevent neuroinflammation, there is an excellent chance that we will significantly ameliorate or postpone onset of clinical symptoms across many common neurological disorders.”

Riluzole, the only drug approved thus far to specifically treat ALS, has been shown to slow disease progression and improve patient survival by about 10%. In several preclinical models of ALS and Alzheimer’s disease, Amylyx’s combinatory drug AMX0035 has shown to limit cell death and neurotoxic inflammation, making it a promising candidate for further study.

“The ALS Finding a Cure Foundation is committed to pursuing multiple avenues toward a cure for ALS,” said Merit Cudkowicz, MD, the Foundation’s Chief Medical Officer, a Professor of Neurology at Harvard, and Chief of Neurology at Massachusetts General Hospital. “AMX0035 has shown promise in preclinical models. This grant will underwrite studies needed to begin clinical trials.”

Added Joshua Cohen, Co-Founder and CEO of Amylyx, “We are honored by the support from ALS Finding a Cure, the Cure Alzheimer’s Fund, and our investors. We are excited to work toward a meaningful therapy for patients suffering from this devastating disease and anticipate launching an initial clinical trial in ALS in 2016.”

ALS, classified as a rare disease, affects an average of 30,000 people in the U.S. and 450,000 worldwide. The causes underlying ALS are not yet fully understood, but evidence suggests that many risk factors contribute toward disease development, ranging from genetics and exposure to chemicals and electromagnetic fields, to higher levels of the neurotransmitter glutamate and protein misfolding.