ASHA-624 to be studied as potential disease-modifying therapy

Company will begin preclinical studies for ALS treatment

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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A researcher studies samples of fluids on slides placed on a microscope alongside a rack of test tubes and a beaker.

ASHA-624, an experimental therapy that blocks the activity of the SARM1 protein, will be developed as a potential disease-modifying therapy for amyotrophic lateral sclerosis (ALS).

Developer Asha Therapeutics said it will conduct preclinical studies aimed at supporting the treatment’s potential for testing in human clinical trials.

“As a veteran CNS [central nervous system] drug developer, I am excited to see this compound continue its progress towards clinical trials,” Michael Gold, MD, a member of Asha’s scientific advisory board, said in a company press release. The central nervous system consists of the brain and spinal cord.

“We are committed through our work to the transformation of patient outcomes, and believe ASHA-624 is a significant step forward towards achieving that goal,” said Bradlee Heckmann, PhD, co-founder and chief scientific officer of Asha.

ALS is a neurodegenerative condition marked by progressive damage to motor neurons, the nerve cells that control voluntary movements.

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Stabilizing the SARM1 protein to preserve nerve fibers

Research has shown that the SARM1 protein can cause damage to nerve fibers, which are important for the transmission of the electrical signals, and drive nerve cell death over time. Mutations in the gene that codes for this protein have been found in ALS patients.

ASHA-624 is an intra-molecular glue that stabilizes SARM1 in its inactive form, which is thought to reduce nerve fiber damage and promote nerve cell health. The treatment is designed to work without interfering with other proteins, which is expected to prevent side effects.

“SARM-1 is a well-validated therapeutic target that could yield novel therapies for patients suffering from a range of both central and peripheral [outside the brain and spinal cord] nervous disorders,” Gold said. “ASHA-624 prevents the activation of SARM-1 using a completely novel approach that has the potential to deliver a therapy with robust clinical efficacy and few, if any, off-target side effects.”

According to Asha, treatment with ASHA-624 showed a “robust safety profile” and effectively reversed motor impairment in animal models of ALS. In contrast, placebo-treated animals continued to exhibit progressive motor function decline.

ASHA-624 is also being developed to treat chemotherapy-induced peripheral neuropathy, a condition marked by damage to the peripheral nerves caused by chemotherapy, glaucoma, or traumatic brain or spinal cord injuries.

Asha has another lead therapeutic candidate, ASHA-091, that’s being developed for Alzheimer’s disease, Parkinson’s disease, and ALS.

“Nomination of ASHA-624 as a development candidate for clinical translation in ALS, alongside Asha’s previously nominated ASHA-091 compound with indications in Alzheimer’s disease, Parkinson’s disease, and ALS, is a key juncture for Asha in supporting our mission of providing novel, disease-modifying therapies for conditions with current treatment options limited largely to symptomatic standard of care,” Heckmann said.