Asha Therapeutics Awarded Barnett Drug Development Grant by the ALS Association to Advance Novel Intra-Molecular Glue Inhibitor of SARM1 to Clinic and Announces Appointment of Disarm Therapeutics’ Founders to Company’s Scientific Advisory Board
Asha Therapeutics has received a grant from the ALS Association's Barnett Drug Development Program to advance ASHA-624, a novel brain-penetrant intra-molecular glue inhibitor of SARM1, towards clinical trials for ALS treatment. The drug, designed using Asha's PRISM™ technology, has shown promising results in preclinical trials, demonstrating neuroprotection and restored motor function. The company also announced the appointment of SARM1 experts Dr. Jeffrey Milbrandt and Dr. Aaron DiAntonio, founders of Disarm Therapeutics (acquired by Eli Lilly), along with Dr. Jeffrey Rothstein to its Scientific Advisory Board.
Asha Therapeutics ha ricevuto un finanziamento dal Barnett Drug Development Program dell'ALS Association per far progredire ASHA-624, un inibitore innovativo della colla intra-molecolare penetrante nel cervello di SARM1, verso studi clinici per il trattamento della SLA. Il farmaco, progettato utilizzando la tecnologia PRISM™ di Asha, ha mostrato risultati promettenti negli studi preclinici, dimostrando neuroprotezione e ripristino della funzione motoria. L'azienda ha anche annunciato la nomina degli esperti di SARM1, il Dr. Jeffrey Milbrandt e il Dr. Aaron DiAntonio, fondatori di Disarm Therapeutics (acquisita da Eli Lilly), insieme al Dr. Jeffrey Rothstein, nel suo Consiglio Consultivo Scientifico.
Asha Therapeutics ha recibido una subvención del Barnett Drug Development Program de la ALS Association para avanzar ASHA-624, un novedoso inhibidor de pegamento intra-molecular que penetra en el cerebro de SARM1, hacia ensayos clínicos para el tratamiento de la ELA. El fármaco, diseñado utilizando la tecnología PRISM™ de Asha, ha mostrado resultados prometedores en ensayos preclínicos, demostrando neuroprotección y restauración de la función motora. La compañía también anunció el nombramiento de los expertos en SARM1, el Dr. Jeffrey Milbrandt y el Dr. Aaron DiAntonio, fundadores de Disarm Therapeutics (adquirida por Eli Lilly), junto con el Dr. Jeffrey Rothstein en su Consejo Asesor Científico.
Asha Therapeutics는 ALS 치료를 위한 ASHA-624의 임상 시험을 진행하기 위해 ALS 협회의 Barnett 약물 개발 프로그램으로부터 보조금을 받았습니다. 이 약물은 Asha의 PRISM™ 기술을 사용하여 설계되었으며, 전임상 시험에서 신경 보호 및 운동 기능 회복을 보여주는 유망한 결과를 나타냈습니다. 또한 회사는 Disarm Therapeutics(엘리 릴리 인수)의 설립자인 SARM1 전문가인 Jeffrey Milbrandt 박사와 Aaron DiAntonio 박사를 과학 자문 위원회에 임명했다고 발표했습니다.
Asha Therapeutics a reçu une subvention du Barnett Drug Development Program de l'ALS Association pour faire progresser ASHA-624, un nouvel inhibiteur de colle intra-moléculaire pénétrant le cerveau de SARM1, vers des essais cliniques pour le traitement de la SLA. Ce médicament, conçu avec la technologie PRISM™ d'Asha, a montré des résultats prometteurs lors des essais précliniques, démontrant une neuroprotection et une restauration de la fonction motrice. L'entreprise a également annoncé la nomination d'experts en SARM1, le Dr. Jeffrey Milbrandt et le Dr. Aaron DiAntonio, fondateurs de Disarm Therapeutics (acquise par Eli Lilly), ainsi que le Dr. Jeffrey Rothstein dans son Conseil Consultatif Scientifique.
Asha Therapeutics hat einen Zuschuss des Barnett Drug Development Program der ALS Association erhalten, um ASHA-624, einen neuartigen gehirngängigen intra-molekularen Kleberinhibitor von SARM1, in klinische Studien zur Behandlung von ALS voranzubringen. Das Medikament, das mit Asha's PRISM™-Technologie entwickelt wurde, hat in präklinischen Studien vielversprechende Ergebnisse gezeigt, die Neuroprotektion und Wiederherstellung der motorischen Funktion demonstrieren. Das Unternehmen kündigte außerdem die Ernennung von SARM1-Experten, Dr. Jeffrey Milbrandt und Dr. Aaron DiAntonio, die Gründer von Disarm Therapeutics (von Eli Lilly übernommen), sowie Dr. Jeffrey Rothstein in seinen Wissenschaftlichen Beirat an.
- Received prestigious ALS Association grant for drug development
- Preclinical trials showed robust neuroprotection and motor function restoration
- Strategic addition of renowned SARM1 experts to Scientific Advisory Board
- Novel drug technology with potential disease-modifying properties
- Drug still in early development stage, far from market approval
- No clinical trial data available yet
ASHA-624 was designed using Asha’s proprietary PRISM™ drug design technology, which creates new medicines to restore normal biology and provide functional cures for difficult to treat diseases with high unmet medical need. ASHA-624 prevents the loss of nerve cell projections (axons) by selectively “gluing” the central regulator of axon degeneration, SARM1 into an inactive state, a mechanism of action which represents an industry first. In a preclinical model of ALS, treatment with ASHA-624 safely provided robust neuroprotection and restored motor function.
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 debilitating loss of muscle control and impaired motor function. ALS ultimately affects the regulation and control of muscles which are required for speaking, eating, and breathing. ALS is fatal, with no available cure.
Additionally, Asha announced SARM1 and axon degeneration experts Dr. Jeffrey Milbrandt, M.D., Ph.D. and Dr. Aaron DiAntonio, M.D., Ph.D. will join the company’s Scientific Advisory Board. Dr. Milbrandt and Dr. DiAntonio were the first to characterize the function of SARM1 in axonal degeneration and are the founders of Disarm Therapeutics, acquired by Eli Lilly and Company (NYSE: LLY) in late 2020. Dr. Milbrandt is the Executive Director of McDonnell Genome Institute (MGI), James S. McDonnell Professor of Genetics, and Professor of Pathology & Immunology, Medicine, and Neurology at Washington University School of Medicine in
“SARM1 is a compelling therapeutic target for many central, peripheral, and ocular neurodegenerative diseases. Asha Therapeutics has developed a completely novel approach for inhibiting SARM1, and I look forward to helping the team at Asha bring this therapy to patients in need," noted Dr. Aaron DiAntonio, M.D., Ph.D.
Dr. Jeffrey Milbrandt, M.D., Ph.D. added, “Asha’s novel intra-molecular glue approach for SARM1 inhibition represents a potentially highly selective and unique route for therapeutic intervention in diseases including ALS and other peripheral neuropathies. Furthermore, the support from the ALS Association to advance ASHA-624 towards clinical trials is a significant milestone for the program. I am excited to work with the Asha team to develop new SARM1 therapeutics."
“The Barnett Drug Development grant from the ALS Association represents an exceedingly competitive cornerstone of validation for therapeutic programs with the potential to reshape ALS treatment, and we are profoundly grateful and humbled by our selection as a grant recipient. This award, along with the addition of Dr. Milbrandt and Dr. DiAntonio, pioneers in SARM1 biology and therapeutic development targeting SARM1, and the addition of clinical expertise in ALS brought by Dr. Rothstein, strongly positions Asha to rapidly advance ASHA-624 to clinic. Based on our data’s demonstration of robust preclinical efficacy and safety, we are optimistic that ASHA-624 is a potential disease-modifying therapeutic for ALS," commented Dr. Bradlee Heckmann, Ph.D., Asha’s Scientific Co-founder, President & Chief Scientific Officer.
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 profile as a functional cure in models of ALS. ASHA-624 therapeutic intervention in preclinical models of ALS reversed motor impairment and dysfunction to levels similar to healthy controls, while placebo treated groups exhibited continual and exacerbated decline in motor function. In addition to ALS, ASHA-624 has potential indications in Chemotherapy-Induced Peripheral Neuropathy (CIPN), Multiple Sclerosis (MS), Spinal Cord Injury and TBI, Glaucoma, and rare disorders including Charcot-Marie Tooth (CMT) and Autosomal Dominant Optic Atrophy (ADOA).
About Asha Therapeutics: Asha Therapeutics (www.ashatherapeutics.com) 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 custom design de novo compounds to create disease modifying and curative therapeutics for 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 in early 2025.
About the ALS Association: The ALS Association is the largest philanthropic funder of ALS research in the world. The Association funds global research collaborations, assists people with ALS and their families through its nationwide network of care and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. For more information about the ALS Association, visit our website at als.org. For more information about the Lawrence and Isabel Barnett Drug Development Program, visit als.org/BarnettProgram.
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Source: Asha Therapeutics
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