Monrovia, CA (June 18, 2018)–AcuraStem (www.acurastem.com), a fast-growing and innovative biotech company located in Monrovia, California, has been awarded a $3.7 million Small Business Innovation Research (SBIR) Fast-Track grant (#R44NS105156) by the National Institute of Neurological Disorders and Stroke (NINDS) to continue research for the development of a small molecule therapeutic, “AS2015”, focused on treating patients with the genetic form of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) caused by expansion repeats in the gene C9ORF72.
The grant supports an integrated drug discovery approach by AcuraStem to leverage the induced motor neuron cellular models developed at AcuraStem President and Co-Founder Dr. Justin Ichida's lab at USC. AcuraStem’s innovative precision platform, iNeuroRx™, utilizes nerve cells derived directly from patient stem cells, in conjunction with artificial intelligence, to predict drug efficacy, and is transforming patient outcomes. By applying their cutting edge iNeuroxRx™ platform, AcuraStem can partner with patients and their clinicians to evaluate disease progression and evaluate existing approved therapeutics to identify the most efficacious treatments to help slow the progression of ALS. This grant supports AcuraStem’s platform that allows research scientists to use artificial intelligence analysis combined with stem cell biology to accurately predict personalized cures for one of our most intractable disease areas.
The NINDS SBIR program seeks to help researchers commercialize early-stage scientific discoveries. Small-molecule drugs are easier to administer to patients, are often less expensive, and more easily penetrate the blood brain barrier—a beneficial feature for drugs that treat neurodegenerative diseases. The path for small molecule drug approval is marred with hundreds to thousands of failed programs, so it is believed important to leverage relevant human disease and toxicity models as early as possible during the preclinical drug discovery phases. Dr. Ichida’s human motor neuron assay helps to mitigate some of these obstacles that can hinder new therapeutic progression.
“The team at AcuraStem is grateful for this grant as it will allow us to progress more rapidly toward a treatment, and set us on a path toward a cure for ALS,” stated Ichida. “The award is intended to facilitate the advancement of a new drug candidate for ALS leveraging patient neurons and engaging an entirely new target that focuses on the endosomal trafficking pathway.”
Dr. Qing Liu, AcuraStem, Co-Founder added, “ALS affects people when they’re in the prime of their lives. Traditionally, scientists have only been able to study a small population of ALS patients, relying heavily on animal models. This explains why there are no effective treatments for this disease, and why the available drugs only extend a patient’s life by a few months. We are disrupting this approach.”
“AcuraStem’s objective is to advance a therapeutic portfolio to the clinic to treat ALS patients where limited therapies currently exist,” stated Sam Alworth, CEO and Co-Founder of AcuraStem. “AS-1 is our lead program, and we are initiating multiple ALS therapeutic programs aimed at targeting the endosomal trafficking pathway in neurons.”
AcuraStem is a fast-growing biotech company that was formed in 2016 to create an innovative precision medicine platform, iNeuroRX™, which leverages patient stem cells and advanced machine learning technology to discover drugs for neurodegenerative diseases. The platform has revealed a novel ALS target from Dr. Justin Ichida’s lab at USC. AcuraStem is led by an exceptional team of PhDs, professors, tech entrepreneurs, and successful drug company veterans employing the latest scientific breakthroughs to first address one of the most challenging, but tractable diseases, amyotrophic lateral sclerosis (ALS).
Erin Morris Huttlinger
Public Information Officer
Director of Communications