AcuraStem Awarded $3.7M to Develop Gene-related Therapy for ALS, Frontotemporal Dementia

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by Alice Melão |

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The National Institute of Neurological Disorders and Stroke (NINDS) awarded $3.7 million to AcuraStem to support the development of a small molecule drug to treat C9orf72 gene-related amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

The Small Business Innovation Research (SBIR) fast-track grant (R44NS105156) will promote the development of AS2015, a new  compound developed by AcuraStem through its precision platform iNeuroRx.

iNeuroRx was engineered by Justin Ichida, PhD, assistant professor at the University of Southern California and president and co-founder of AcuraStem, and its collaborators, to improve the ability to identify therapies that have enhanced potential to slow the progression of ALS.

The SBIR fast-track grant merges the submission and review of Phase 1 (proof of concept) and Phase 2 (research and development) applications into one submission.

This helps reduce the time between the two phases and is expected to expedite the progression of an investigative therapy that has demonstrated great potential to meet an unmet medical need.

“The team at AcuraStem is grateful for this grant as it will allow us to progress more rapidly toward a treatment, and set us on a path toward a cure for ALS,” Ichida said in a press release.

“The award is intended to facilitate the advancement of a new drug candidate for ALS, leveraging patient neurons and engaging an entirely new target that focuses on the endosomal trafficking pathway,” he said.

The iNeuroRx drug discovery platform for neurodegenerative diseases combines artificial intelligence with data collected from nerve cells derived from patient’s stem cells.

With this cutting-edge strategy, the researchers identified their first preclinical therapy candidate, AS2015, also known as AS-1. They plan to advance AS2015 into human clinical trials for the treatment of ALS.

“AcuraStem’s objective is to advance a therapeutic portfolio to the clinic to treat ALS patients where limited therapies currently exist,” said Sam Alworth, CEO and co-founder of AcuraStem.

“AS-1 is our lead program, and we are initiating multiple ALS therapeutic programs aimed at targeting the endosomal trafficking pathway in neurons,” he said.

The new financial aid adds to a $225,000 Phase 1 SBIR grant (R43AG058325) awarded in December 2017 from the National Institute on Aging, part of the National Institutes of Health.

That research grant aimed to enhance the company’s models of age-associated ALS by improving the molecular methods used to produce the motor neurons that more accurately reproduce ALS features.

“ALS affects people when they’re in the prime of their lives. Traditionally, scientists have only been able to study a small population of ALS patients, relying heavily on animal models,” said Qing Liu, PhD, chief scientific officer and co-founder of AcuraStem.

“This explains why there are no effective treatments for this disease, and why the available drugs only extend a patient’s life by a few months. We are disrupting this approach,” Liu said.