Aquinnah Pharmaceuticals receives $3.4 million grant to advance ALS therapies

Aquinnah Pharmaceuticals has announced that it has been awarded a $3.4 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) to advance novel therapeutic drug candidates towards the clinic for treating patients with the neurodegenerative disease, amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.

The company, which is a leader in stress granule biology, is developing drugs designed to slow or potentially reverse the progression of ALS. There is, currently, no cure for slowing the progression of ALS, a disease that stifles nerve cell function, leading to loss of muscle control throughout the body.

The NINDS grant is Aquinnah's second and has been awarded to support its unique approach to treating ALS, which targets the prevention of pathological TDP-43 stress granule protein complexes found in the brains of most ALS patients.

The accumulation of the disease-linked protein complexes is believed to drive the progression of ALS and other neurodegenerative diseases. Aquinnah will use newly identified small molecule compounds to attack and break down the complexes, with the goal of moving a novel and effective class of ALS drugs into clinical development.

Dr Glenn Larsen, President and CEO of Aquinnah, said: "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 recognises this, leading to funding for our innovative ALS programme. Our mission is to revolutionise ALS treatment and change the clinical outcome of this disease by developing a new drug to slow or potentially reverse the progression of ALS.

"This support from the NINDS will help advance Aquinnah's science toward IND-enabling activities and our goal of developing novel ALS therapies," added Aquinnah co-founder Dr Ben Wolozin.