#USA #Pasadena #ALS #AcuraStem #AS-241

AcuraStem Secures Hoffman ALS Clinical Trial Award

AcuraStem, the innovative biotechnology firm, has announced a pivotal achievement in its mission to address neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The company has been awarded a substantial $1 million grant from the ALS Association, which holds the distinction of being the largest nonprofit organization dedicated to ALS research globally. This funding will be instrumental in supporting the Phase 1 trial of AS-241, the leading candidate from AcuraStem's UNC13A program.

This two-year grant is designed to bolster the integrated biomarker strategy for the AS-241 trial, which encompasses vital elements related to drug exposure, safety, tolerability, and treatment response. The goal is to utilize reliable and reproducible assays to ensure that the AS-241 effectively reaches therapeutic levels within the central nervous system, enabling informed decisions as the program moves forward.

Sam Alworth, co-founder and CEO of AcuraStem, expressed optimism about the progress: "We are entirely focused on advancing a broadly acting treatment for the 97% of people living with ALS driven by TDP-43 pathology. 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."

Understanding the Hoffman ALS Clinical Trial Award

The ALS Association's Hoffman ALS Clinical Trial Awards are designed to support early- to mid-stage trials that explore novel or repurposed methods for treating ALS. Specifically, these awards aim to generate crucial data regarding safety, dosing, and biomarkers, which are essential for regulatory bodies evaluating investigational therapies. The funding covers up to $1 million over a two- to three-year period based on milestone achievements, prioritizing trials that are grounded in biomarker research.

Dr. Kuldip Dave, Executive Vice President and Chief Scientist of the ALS Association, noted: "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."

The UNC13A Program and AS-241

AcuraStem’s UNC13A program centers around developing antisense oligonucleotides (ASOs) that specifically target the gene UNC13A, which is vital for maintaining the health of motor neurons in ALS and FTD. In conditions such as ALS and FTD, the UNC13A gene can undergo cryptic splicing, leading to the loss of a crucial protein responsible for maintaining healthy synaptic signaling within motor neurons. Targeting UNC13A is highly relevant as it affects a broad spectrum of ALS patients, including those diagnosed with sporadic forms of the disease.

Designed to rectify cryptic splicing and restore healthy levels of the UNC13A protein, AS-241 emerged from AcuraStem’s iNeuroRx® platform, which leverages human patient-derived motor neuron models. Conducted studies have shown that AS-241 successfully restores normal UNC13A expression while also improving synaptic function in ALS models. Preliminary in vivo evaluations indicate a promising safety profile alongside sustained central nervous system exposure, underscored by a comprehensive nonclinical safety assessment intended to thoroughly characterize the treatment's long-term safety ahead of clinical trials.

Dedicated Support from the ALS Community

The Hoffman Clinical Trial Awards are part of the Hugh and Herbert Hoffman ALS Impact Fund, a financial initiative that underscores the ALS Association's commitment to making ALS a livable condition while striving towards a cure by backing promising therapies and expanding access to necessary care. The ALS Association's backing signifies a growing trust among key funders in AcuraStem’s direction, which emphasizes addressing the root biological mechanisms behind ALS.

AcuraStem’s latest recognition from the ALS Association follows a recent grant of $7.5 million from the California Institute for Regenerative Medicine aimed at advancing AS-241 into first-in-human trials. Additionally, the company has received support from the Target ALS Drug Discovery Consortium for another program that focuses on targeting SYF2, another important regulator of TDP-43 function. Together, these funding opportunities illustrate increasing confidence in AcuraStem's approach to combatting the complexities of ALS.

For further updates and details on AcuraStem’s groundbreaking work, interested parties can visit their website at acurastem.com and stay connected through their social media channels including LinkedIn, Facebook, and X.