AcuraStem Wins $1 Million ALS Association Award for AS-241 Phase 1 Trial

What the award pays for

According to AcuraStem, the grant supports an integrated biomarker strategy for the Phase 1 trial of AS-241. The strategy spans drug exposure, safety and tolerability, and treatment response. The company says it will use 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.

AcuraStem, based in Pasadena, California, develops treatments for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and other neurodegenerative diseases. The ALS Association describes itself as the largest ALS organization in the world.

The Hoffman ALS Clinical Trial Awards fund early- to mid-stage trials of novel or repurposed approaches for ALS. They are meant to help generate the safety, dosing and biomarker data that regulators require to evaluate investigational therapies. The 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.

How AS-241 works

AcuraStem's UNC13A program has developed antisense oligonucleotides (ASOs) that target UNC13A, a gene the company describes as 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.

AS-241 is designed to correct the cryptic splicing and restore healthy UNC13A protein levels. The company says UNC13A is 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.

The candidate emerged from AcuraStem's iNeuroRx platform, which models disease directly in human patient-derived motor neurons. According to the company, 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 characterize long-term safety ahead of clinical testing.

Funding momentum and what comes next

Sam Alworth, co-founder and CEO of AcuraStem, said the company is "entirely focused on advancing a broadly acting treatment for the 97% of people living with ALS driven by TDP-43 pathology." He added that the company is "closer than ever to bringing AS-241 to the people who need it."

Kuldip Dave, EVP and Chief Scientist at the ALS Association, said the group is striving "to accelerate the development of therapeutic candidates that can help make ALS a livable disease." The awards are part of the Hugh and Herbert Hoffman ALS Impact Fund.

The Hoffman award follows other 2026 funding for AcuraStem's TDP-43-focused pipeline. The company cites $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 for a separate program targeting SYF2, another regulator of TDP-43 function. AcuraStem says the grants reflect growing conviction among leading funders in its approach.

The source does not give a start date for the Phase 1 trial. For now, the award is aimed at the biomarker groundwork that should let the company confirm drug exposure in the central nervous system and decide whether to proceed.