Asha Therapeutics has secured a grant from the ALS Association's Lawrence and Isabel Barnett Drug Development Program to propel its novel intra-molecular glue inhibitor, ASHA-624, into clinical trials. This funding aims to support the development of ASHA-624 as a potential disease-modifying therapy for Amyotrophic Lateral Sclerosis (ALS).
ASHA-624: A Novel Approach to ALS Treatment
ASHA-624 is designed to prevent the loss of nerve cell projections (axons) by selectively inactivating SARM1, a central regulator of axon degeneration. This mechanism of action represents a novel approach in the treatment of ALS. Preclinical models of ALS have shown that ASHA-624 provides robust neuroprotection and restores motor function safely.
ALS: A Devastating Neurodegenerative Disease
ALS, also known as Lou Gehrig's disease, is a progressive neurodegenerative disorder affecting neurons in the spinal cord and brain. The loss of motor neurons leads to muscle control loss and impaired motor function, eventually affecting speech, eating, and breathing. Currently, there is no cure for ALS, making the development of new therapies crucial.
Experts Join Asha's Scientific Advisory Board
Dr. Jeffrey Milbrandt and Dr. Aaron DiAntonio, pioneers in SARM1 biology and founders of Disarm Therapeutics (acquired by Eli Lilly and Company), have joined Asha's Scientific Advisory Board. Their expertise in SARM1 and axon degeneration will be invaluable in advancing ASHA-624. Dr. Jeffrey Rothstein, a leading expert in ALS research, has also joined the board.
Preclinical Efficacy and Safety
Preclinical studies have demonstrated ASHA-624's ability to reverse motor impairment and dysfunction in ALS models, bringing motor function levels back to those of healthy controls. Placebo-treated groups, in contrast, showed continued decline. These results suggest ASHA-624's potential as a disease-modifying therapeutic.
Potential Beyond ALS
In addition to ALS, ASHA-624 has potential applications in other neurodegenerative conditions, including Chemotherapy-Induced Peripheral Neuropathy (CIPN), Multiple Sclerosis (MS), Spinal Cord Injury and TBI, Glaucoma, and rare disorders like Charcot-Marie Tooth (CMT) and Autosomal Dominant Optic Atrophy (ADOA).
About Asha Therapeutics' PRISM™ Technology
Asha Therapeutics utilizes its proprietary PRISM™ drug design technology to create new medicines that restore normal biology and provide functional cures for difficult-to-treat diseases with high unmet medical needs. ASHA-624 and ASHA-091, targeting ALS, Parkinson's Disease, and Alzheimer's Disease, are expected to enter clinical trials in early 2025.
