The biotechnology company, Aquinnah Pharmaceuticals is dedicated to identifying new therapeutic agents for ALS and Alzheimer’s disease, based on a new scientific approach of RNA binding proteins involved in neurodegenerative disease. Last week, Aquinnah announced a $10 million investment from two world leader pharmaceutical companies Pfizer, Inc. and AbbVie Inc. to support therapeutic development to treat ALS, Alzheimer’s disease and other neurodegenerative diseases. This adds to a previous $5 million investment by Takeda Pharmaceuticals in December 2015. The Association recently awarded Aquinnah two grants totaling approximately $500,000 (see below) under the Lawrence and Isabel Barnett Drug Development Program, designed to propel therapeutic targets into human clinical trials through building and fostering academic-industry partnerships.

Aquinnah’s goal is to overcome the challenges of not only ALS, but other neurodegenerative diseases, such as Alzheimer’s disease. To accomplish this, the company focuses on stress granules, which are clumps of protein and RNA that form in cells in response to stress. If stress granules persist, outside of stressful circumstances in the cell, they build up and cause neurodegeneration. With this knowledge in hand, Aquinnah is developing disease-modifying compounds targeting harmful stress granules.

According to their website, their designed compounds are “potent, orally bio-available in small doses and can get past the blood brain barrier, a critical factor that hasn’t always been tested by others in the field.” Currently, animal trials are underway with human trials on the near horizon in the next two to three years.

The Association is proud to see that our investment helped spur these critical partnerships that are vital to move these promising compounds into human clinical trials, not only for ALS, but for other neurodegenerative diseases. We look forward to hearing more exciting news out of Aquinnah!

“It was the initial support of Dr. Lucie Bruijn, Chief Scientist of The ALS Association, who recognized Aquinnah’s unique approach to focus on TDP-43, a genetically validated RNA binding protein drug target implicated in ALS, which led to The Association’s financial grant that catalyzed Aquinnah's significant advances towards developing a drug for the treatment of ALS. Importantly, this catalyzed further interest and investment by the above mentioned pharmaceutical companies into Aquinnah’s approach. Aquinnah is very appreciative of The Association’s support to help us advance our exciting new ideas to the reality of a new therapeutic drug and look forward to continuing to work together closely with the The Association and to make new therapeutic options a reality for people with ALS.” – Glenn Larsen, President & CEO, Aquinnah Pharmaceuticals

Learn more:

Read press release here.

For more information on the targeting of stress granules, watch Drs. Larsen and Wolozin’s recorded webinar, “New Therapeutic Approaches for ALS Based on RNA Binding Proteins,” here or read a summary of the webinar here.

ALS Association grants to Aquinnah Pharmaceuticals:

Glenn Larson: Characterization of ADME and brain metabolism of TDP-43 inclusion inhibitors – awarded May 2015

Benjamin Wolozin: Validating the efficacy of lead compounds that decrease TDP-43 aggregation on neurotoxicity in vitro and in vivo – awarded November 2015