CAMBRIDGE, MA, USA I February 15, 2022 I Aquinnah Pharmaceuticals announced today that it has signed an agreement with Roche to advance the development of brain penetrant oral small molecules to treat amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Aquinnah is a leader in drug discovery in the emerging fields of stress granule biology, RNA binding proteins & bimolecular condensate formation, all which mediate a wide range of neurodegenerative disorders. Aquinnah’s small molecule compounds reduce TDP-43 pathology, which is the hallmark characteristic in more than 95% of ALS patients, also known as Lou Gehrig’s disease. These approaches hold great promise for treating this relentless, debilitating disorder.

Under the terms of the agreement Aquinnah and Roche will collaborate in research activities up to the selection of an advanced drug candidate, after which Roche will be responsible for further research, development and global commercialization.

Glenn Larsen, Ph.D., President and CEO of Aquinnah said, ‘We are very pleased to advance our pioneering small molecule program focused on preventing and eliminating pathological TDP-43 with improved neuronal cell survival for ALS, with Roche’s world-renowned development and commercial expertise. This collaboration with Roche validates our scientific approach and highlights our potential in this area of biology.’

Ben Wolozin, M.D.,Ph.D., co-founder of Aquinnah, commented that ‘Recent advances suggest that the chronic response of RNA metabolism to stress plays a key role in the pathophysiology of neurodegenerative diseases, particularly ALS, frontotemporal dementias and Alzheimer’s disease. Aquinnah has successfully leveraged these recent findings into a pipeline of novel drugs that hold great promise for treating groups of patients who have few treatment options. The collaboration with Roche will propel this exciting work forward, leading the way towards a first in class therapy for patients suffering from ALS.’

James Sabry, M.D.,Ph.D., Global Head of Roche Pharma Partnering, added that ‘Roche is committed to partnering with cutting edge research organizations like Aquinnah, bringing together scientific expertise and diverse skills to advance innovation in neuroscience. This preclinical collaboration with Aquinnah will help to maximize the chances of success for developing therapies in a devastating condition, for which no disease modifying treatments are currently available.’

About Aquinnah Pharmaceuticals

Aquinnah Pharmaceuticals is harnessing the power by integrating the fields of stress granule biology, RNA binding proteins & bimolecular condensate formation to develop oral small molecule drug candidates in Neurodegenerative Diseases. Aquinnah’s therapeutics are designed to stop or even reverse diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal disease (FTD) and Alzheimer’s disease by eliminating pathologies caused by the proteins TDP-43 or tau. Aquinnah is financially backed by Pfizer, Inc., AbbVie, Inc. and Takeda Pharmaceuticals and has received numerous awards from the ALS Association, National Institutes of Health, the Alzheimer’s Association and the Rainwater Foundation.

SOURCE: Aquinnah Pharmaceuticals