$3.4M NIH Grant Funding New ALS Treatments

(Gerd Altmann, Pixabay)

21 June 2018. A biotechnology company is receiving a grant from National Institute of Neurological Disorders and Stroke to advance its experimental treatments for amyotrophic lateral sclerosis, or ALS. Aquinnah Pharmaceuticals Inc. in Cambridge, Massachusetts is the recipient of a $3.4 million Small Business Innovation Research award from NINDS, part of National Institutes of Health.

Aquinnah Pharmaceuticals is a developer of small molecule, or low molecular weight drugs to treat ALS and other neurodegenerative diseases. ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder where neurons or nerve cells controlling muscles in the body begin to waste away, and can no longer send or receive signals from the brain or spinal cord. As the nerve cells stop functioning, the muscles in the limbs, and later speech and breathing muscles, begin weakening and eventually stop functioning. Most people with the disease die of respiratory failure. There are currently no cures for ALS, and few effective treatments for slowing progression of the disease.

Aquinnah’s treatments for ALS and Alzheimer’s disease take aim at stress granules, normal physiological reactions to damage that can build up and become toxic, contributing to these neurological conditions. The company’s work is based in part on research by its chief scientist and co-founder Benjamin Wolozin, a pharmacologist at Boston University. Wolozin identified a protein known as transactive response DNA binding protein 43, or TDP-43, which under stress, or as a result of a mutation, accumulates as excess toxic granules. Aquinnah licensed a number of compounds from Boston University that Wolozin and colleagues identified in the lab with potential for limiting TDP-43 build-ups.

The new funding from NINDS continues an earlier project that showed the feasibility of two lead compounds against TDP-43 build-ups in lab animals. The new grant is supporting continued preclinical research in lab mice with genetically induced human ALS caused by TDP-43 accumulations. The company expects tests of the oral drug candidates will show they slow or reverse the progress of the disease. If those results occur, the company will file for an investigational new drug application with the Food and Drug Administration, to allow for the beginning of clinical trials.

Aquinnah Pharmaceuticals is a four year-old privately-owned company founded by Wolozin, Glenn Larsen, Aquinnah’s CEO, and Manfred Weigele, now a scientific advisor to the company. In addition to the NINDS grants, Aquinnah is funded by equity investments from Takeda Pharmaceuticals in 2015, and drug makers AbbVie and Pfizer in 2017.

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Disclosure: The author owns shares in Pfizer, an investor in Aquinnah Pharmaceuticals.

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