AcuraStem, an early stage biotech company, and Justin Ichida, PhD, assistant professor of stem cell biology and regenerative medicine at the Keck School of Medicine of USC, have been awarded a $3.7 million Small Business Innovation Research (SBIR) FastTrack grant by the National Institute of Neurological Disorders and Stroke (NINDS) to continue research they are conducting with Icagen, Inc. for the development of a small molecule therapeutic focused on treating patients with the genetic form of Amyotrophic Lateral Sclerosis (ALS) caused by expansion repeats in the gene C9 ORF72. The grant supports an integrated drug discovery approach by AcuraStem to leverage the inducible motor neuron cellular models developed at Ichida’s lab in combination with expertise with in silico drug design, medicinal chemistry, and in vitro/in vivo biology capabilities at Icagen. The integration of these capabilities is expected to advance the program to an IND phase in the next four years.

The NINDS SBIR program is a highly competitive program that seeks to transform early-stage scientific discovery into societal and economic benefit by catalyzing private sector commercialization of technological innovations. Small-molecule drugs are easier to administer to patients and 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. Ichida’s human motor neuron assay helps to mitigate some of these obstacles that can hinder new therapeutic progression.

“Working with Dr. Ichida and AcuraStem has been a fantastic experience. Both teams are quick, responsive and, most importantly, their iNeuroRx platform has provided us the opportunity to test new chemical analogs we generate directly in motor neurons derived from ALS patients. AcuraStem’s platform should enable our team to employ our integrated drug discovery capabilities to rapidly optimize small molecules for safety and efficacy in these motor neurons to create a first in class therapy for ALS,” said Paul August, PhD, vice president of discovery biology at Icagen.

“The awards are 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,” said Ichida, who also is the director of the Choi Family Therapeutic Screening Facility at the Keck School.

“The ultimate objective for AcuraStem is to advance a therapeutic portfolio to the clinic to treat ALS patients where limited therapies currently exist,” said Sam Alworth, CEO 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.”

— Cristy Lytal