California Startup Exploring Novel ALS Discovery Platform

June 18, 2018

A grant from the National Institute of Neurological Disorders and Stroke, which allows for concurrent phase I and II trial design review, will speed up the development of novel treatments for patients with ALS and FTD.

Justin Ichida, PhD

Research into the small molecule inhibitor AS-1 (also known as AS2015) has received a boost from a $3.7 million Fast-Track grant for small business innovation research from the National Institute of Neurological Disorders and Stroke (NINDS). The grant, which allows for concurrent phase I and II trial design review, will speed up the development of the treatment for patients with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

“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,” stated AcuraStem president and cofounder Justin Ichida, PhD, in a recent statement. “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.”

The Fast-Track SBIR grant assists in supporting AcuraStem’s integrated drug discovery approach, which seeks to leverage the induced motor neuron cellular models developed at AcuraStem. If AS2015 were to advance through clinical trials and gain approval, it would compete with the other small molecular inhibitors riluzole and edaravone.

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

The NINDS SBIR program aims to assist researchers in commercializing early-stage scientific innovations. The program is believed to be important in leveraging relevant human disease and toxicity models as early as possible during the preclinical drug discovery phases since small molecule drug approval is marred with hundreds to thousands of failed programs.

AS2015 is being developed using AcuraStem's iNeuroRx platform, which uses nerve cells obtained directly from patient stem cells in conjunction with artificial intelligence to forecast drug efficacy. The platform helps researchers assess disease progression and to assess existing approved therapeutics to determine the most efficacious treatments to assist in slowing the progression of ALS.

The platform also provides research scientists with an artificial intelligence analysis in combination with stem cell biology to accurately forecast personalized care. Based on the promise of the platform, in December 2017, AcuraStem was awarded a separate $225,000 SBIR grant for the development of the iNeuroRx platform.

“AcuraStem’s objective is to advance a therapeutic portfolio to the clinic to treat ALS patients where limited therapies currently exist,” added Sam Alworth, AcuraStem’s CEO and co-Founder. “AS-1 is our lead program, and we are initiating multiple ALS therapeutic programs aimed at targeting the endosomal trafficking pathway in neurons.”

Data for AS2015 have not yet been made available. AcuraStem is also a relatively new company, having been founded in 2016 in Los Angeles. Even still, the potential of the research approach has generated interesting, not only through SBIR grants but other research initiatives.

In November 2017, AcuraStem was selected for a unique California tax break, which granted $69 million in tax credits. The company was 1 of only 90 selected for the credit in 2017.

“The California Competes Tax Credit encourages companies from around the world to locate, expand and add good paying jobs in California," Panorea Avdis, GO-Biz Director and Chair of the California Competes Tax Credit Panel, said in a statement. “In just 3 years, hundreds of companies have made commitments to expand in the state and GO-Biz will continue to host informational workshops and work with our regional and local partners to ensure companies of all sizes know about and apply for these tax credits.”