NINDS Gives $3.4M to Aquinnah to Advance ALS Therapies Targeting Stress Granules

The National Institute of Neurological Disorders and Stroke (NINDS) has awarded Aquinnah Pharmaceuticals $3.4 million to continue to develop and advance potential therapies to treat amyotrophic lateral sclerosis (ALS).

This NINDS grant is the second given to Aquinnah to help support its novel approach for the treatment of ALS.

The company’s strategy aims to prevent the accumulation of the stress granule protein complexes found in the brains of most ALS patients.

Stress granules — as the name suggests — form when cells are under stress from some sort of injury. Stress granules play a role in repairing the injury, and usually disperse quickly. But in some people, such as patients with ALS, a mutation can cause the stress granules to become “sticky” and build-up.

The build-up of these protein complexes, which mostly contain a protein called TDP-43, are thought to drive ALS progression. While it is not entirely understood how TDP-43 aggregates kill nerve cells, some studies suggest that the aggregates themselves are toxic.

Aquinnah is focused on developing small molecule compounds that attack and break down these protein complexes. The company’s goal is to move this technology into clinical development.

“Modulating stress granules in the brain represents a new and exciting approach to treating and potentially preventing a wide range of neurodegenerative diseases. We are very pleased that NINDS recognizes this, leading to funding for our innovative ALS program,” Glenn Larsen, PhD, president and CEO of Aquinnah, said in a press release.  “Our mission is to revolutionize ALS treatment and change the clinical outcome of this devastating disease by developing a new drug to slow or potentially reverse the progression of ALS.”

There currently is no effective treatment that might slow progression of ALS, a severe and rapidly advancing neurodegenerative disease that attacks nerve cells of the brain and spinal cord.

NINDS, a part of the National Institutes of Health, is a leading  source of support for research into the brain and nervous system. Its mission is to promote knowledge that can be used to treat and reduce the burden of neurological diseases.

“This support from the NINDS will greatly help advance Aquinnah’s science toward IND-enabling activities and our goal of developing novel ALS therapies,” said Ben Wolozin, MD, PhD, chief science officer for Aquinnah and a company co-founder. “Our work remains urgent knowing that our efforts could radically change the outcomes for ALS patients within a matter of years.”