AcuraStem Receives Hoffman ALS Clinical Trial Award to Support Phase 1 Trial of AS-241, the Clinical Candidate of its UNC13A Program

The ALS Association's $1 Million Award Will Fund Biomarker Development to Support First-in-Human Testing of AcuraStem's Lead ALS Therapy

PASADENA, Calif., Oct. 27, 2026 /PRNewswire/ -- ⎯ AcuraStem, a biotechnology company advancing treatments for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and other neurodegenerative diseases, today announced it has been awarded a Hoffman ALS Clinical Trial Award from the ALS Association, the largest ALS organization in the world. The two-year, $1 million award supports AcuraStem's UNC13A program and the biomarker work to support the first-in-human Phase 1 trial of the program's lead candidate, AS-241.

The grant supports the integrated biomarker strategy for AS-241's Phase 1 trial, spanning drug exposure, safety and tolerability, and treatment response, using robust, reproducible assays to confirm that AS-241 reaches the central nervous system at the intended levels and to inform data-driven go/no-go decisions as the program advances.

"We are entirely focused on advancing a broadly acting treatment for the 97% of people living with ALS driven by TDP-43 pathology," said Sam Alworth, M.S., MBA, co-founder and CEO of AcuraStem. "We are closer than ever to bringing AS-241 to the people who need it. This award from the ALS Association reflects both the strength of our science and the urgency we share with the ALS community, and it affirms the rigor of the approach we are advancing toward the clinic."

The ALS Association's Hoffman ALS Clinical Trial Awards fund early- to mid-stage trials of novel or repurposed approaches for ALS, helping to generate the safety, dosing and biomarker data regulators require to evaluate investigational therapies. These awards provide up to $1 million over two or three years on a milestone basis and prioritize biomarker-driven trials with a clear biological rationale.

"We are pleased to support the continued evaluation of AS-241 through our Hoffman ALS Clinical Trial Awards. By funding programs at this critical stage, we are striving to accelerate the development of therapeutic candidates that can help make ALS a livable disease," said ALS Association EVP and Chief Scientist Kuldip Dave, Ph.D.

AcuraStem's UNC13A program has developed antisense oligonucleotides (ASOs) that target UNC13A, a gene critical to motor neuron health in ALS and FTD. In these diseases, UNC13A undergoes cryptic splicing, and motor neurons lose a protein essential to the synaptic signaling that keeps them healthy. UNC13A is a compelling target relevant for the majority of people living with ALS, including the sporadic cases that make up most of them, rather than a narrow genetic subgroup. AS-241, the program's lead candidate, is designed to correct the cryptic splicing and restore healthy UNC13A protein levels.

AS-241 emerged from AcuraStem's iNeuroRx^® platform, which models disease directly in human patient-derived motor neurons. In these studies, AS-241 restored normal UNC13A expression and improved synaptic function in ALS motor neuron models. In vivo studies demonstrated a favorable safety profile and durable central nervous system exposure, supported by an extended nonclinical safety evaluation designed to rigorously characterize long-term safety ahead of clinical testing.

The Hoffman ALS Clinical Trial Awards are part of the Hugh and Herbert Hoffman ALS Impact Fund, which advances the ALS Association's mission of making ALS livable and curing it by supporting promising ALS therapies, strengthening ALS clinics and expanding access to care.

The Hoffman Award is the latest in a rapid succession of 2026 grants advancing AcuraStem's TDP-43-focused pipeline: $7.5 million from the California Institute for Regenerative Medicine in April to advance AS-241 toward first-in-human testing, and a Target ALS Drug Discovery Consortium grant to advance a separate program targeting SYF2, another regulator of TDP-43 function. Together, they reflect growing conviction among leading funders in AcuraStem's approach to the root biology of ALS.

About AcuraStem
AcuraStem is a biotechnology company pioneering broadly acting treatments for neurodegenerative diseases including sporadic ALS and FTD. AcuraStem's iNeuroRx^® platform enables the discovery and development of disease‑modifying therapies based on human patient biology. AcuraStem's research is supported by grants and partnerships from leading public and private organizations focused on accelerating transformative therapies for patients.

For additional information, please visit acurastem.com and follow AcuraStem on LinkedIn, Facebook, and X.

Media Contacts

Kissy Black, Director of Communications, AcuraStem
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615.310.1894

Roxan Olivas, Lotos Nile
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SOURCE AcuraStem - Patient-Based Therapeutics