Asha Therapeutics Announces the Nomination of a Novel Intra-Molecular Glue Development Candidate ASHA-624 as a Disease Modifying Therapeutic for Amyotrophic Lateral Sclerosis

2024-04-01

CINCINNATI--(BUSINESS WIRE)-- Asha Therapeutics (Asha) ( ), a life sciences company designing de novo disease modifying medicines for neurodegenerative diseases with high unmet medical need, announced today the nomination of a development candidate, ASHA-624 targeting SARM1 as a potential disease modifying therapy for Amyotrophic Lateral Sclerosis (ALS) with additional indications in Chemotherapy-Induced Peripheral Neuropathy (CIPN), Glaucoma, and traumatic brain and spinal cord injuries. This announcement follows the Company’s presentation of robust efficacy and safety data on its second lead neurology program, ASHA-091 in Alzheimer’s disease and Parkinson’s disease by Chief Scientific Officer & Scientific Co-founder Dr. Bradlee Heckmann earlier this month at the AD/PD 2024 International Conference in Lisbon, Portugal (see full release here). Dr. Michael Gold, MD, MS, a member of Asha’s Scientific Advisory Board noted, “SARM-1 is a well-validated therapeutic target that could yield novel therapies for patients suffering from a range of both central and peripheral nervous disorders. 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. As a veteran CNS drug developer, I am excited to see this compound continue its progress towards clinical trials.” “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. 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,” commented Dr. Heckmann, PhD. About ASHA-624: ASHA-624 is a first-in-class, novel intra-molecular glue compound that exploits the normal biology of SARM1, a key protein that promotes axonal degeneration and neurodegeneration by selectively “gluing” activated SARM1 into an inactive conformation, leading to robust neuroprotection through the prevention of axon and neuron loss. In ALS, the activation of SARM1 leads to axonal loss, neurodegeneration and ultimately motor dysfunction. ASHA-624 was designed to inhibit SARM1 with hyper selectivity, and in preclinical studies has demonstrated a robust safety pro a functional cure in models of ALS. ASHA-624 therapeutic intervention in preclinical models of ALS reversed motor impairment and dysfunction to similar levels as healthy controls while placebo treated groups exhibited continual and exacerbated decline in motor function. About ALS: Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder that affects nerve cells (neurons) in the spinal cord and brain. Loss of motor neurons in ALS patients leads to loss of muscle control, resulting in impaired motor function. ALS ultimately affects the regulation and control of muscles which are required for speaking, eating, and breathing. There is currently no available cure for ALS, and the disease is fatal. Asha anticipates the advancement of ASHA-624 and ASHA-091 into clinical trials by late-2024 for their respective disease indications. About Asha Therapeutics: Asha Therapeutics ( ) is a life sciences company at the forefront of a new era of precision drug design, leveraging the power of its proprietary PRISM™ technology to design de novo compounds to create disease-modifying and curative therapeutics for neurological and infectious diseases with high unmet medical need. Asha's lead therapeutic programs, ASHA-624 and ASHA-091 with indications in Amyotrophic Lateral Sclerosis, Parkinson's disease and Alzheimer’s disease, are anticipated to enter clinical trials by late-2024 and are disease modifying therapeutics. Forward-Looking Statements The matters discussed in this release that are not historical facts are “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and we intend that such forward-looking statements be subject to the safe harbor created thereby. Forward-looking statements include, but are not limited to, statements containing the words “believes,” “anticipates,” “intends,” “estimates,” “plans,” “expects,” “projects” and words of similar import. Readers are cautioned not to place undue reliance on these forward-looking statements, which are based on the information available to management at this time and which speak only as of the date of this release. The Company undertakes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual results, performance or achievements of the Company or its industry to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. While the Company believes the information contained herein is reliable, the Company makes no representations or warranties regarding the accuracy or completeness of this information.