$1M DOD Grant Supports AS-202 for Sporadic ALS

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by Forest Ray PhD |

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AcuraStem has been awarded a $1 million grant from the U.S. Department of Defense (DOD) to advance AS-202, a potentially disease-modifying therapy for sporadic amyotrophic lateral sclerosis (ALS), according to a press release.

The candidate therapy is designed to increase the number of lysosomes — cell compartments in which unwanted molecules are broken down or digested — by lowering the levels of the PIKfyve kinase enzyme. Abnormal lysosome function is a common feature of neurodegenerative disorders such as ALS.

The grant, officially called an Amyotrophic Lateral Sclerosis Research Program Therapeutic Development Award, was given through the DOD’s Congressionally Directed Medical Research Programs (CDMRP).

“The studies in my lab at the University of Southern California that discovered PIKfyve as a therapeutic opportunity in ALS were funded in part by the CDMRP,” Justin Ichida, PhD, AcuraStem’s co-founder. “We welcome CDMRP’s continued support of AcuraStem to rapidly advance this treatment towards the clinic.”

As a potential therapy for sporadic ALS, AS-202 is meant for those patients who carry no known ALS-associated genetic mutations. Given the uncertainty in how sporadic ALS originates, few disease-modifying treatments have emerged to address the condition.

Studies done in animal and patient-derived cell models of sporadic ALS have suggested that reducing PIKfyve levels may be an effective therapeutic strategy.

Wen-Hsuan Chang, PhD, the company’s head of research, will lead the project.

Chang has played a key role in the candidate therapy’s development so far and led the team that established and validated AcuraStem’s iNeuroRx drug candidate discovery platform. This platform screens potential therapies in living neurons taken from ALS patients.

Because of the ALS patient origin of the cells, AcuraStem expects this strategy to provide higher chances of finding disease-modifying treatments.

“The iNeuroRx platform enables us to discover and develop treatments that will work for the vast majority of patients, including sporadic ALS,” said Sam Alworth, AcuraStem’s co-founder and CEO.

The company will apply its award money to advance AS-202 into Good Laboratory Practice toxicology studies. These studies aim to define the margins between therapy and overdose, and help determine if a new drug is safe enough for tests in humans.

They also form the basis for the investigational new drug (IND) application needed from the U.S. Food and Drug Administration to gain clearance to conduct clinical trials. AcuraStem hopes to file an IND for AS-202 in 2022.

“This is a game-changing approach to treating neurodegeneration,” Alworth added. “Our methods are well-validated in ALS, and we are extending iNeuroRx into other neurodegenerative indications.”