The National Institute of Neurological Disorders and Stroke (NINDS) awarded the Californian start-up a small business innovation research fast-track grant to advance its R&D project for small molecule therapeutic AS2015.
The candidate is designed to treat patients with the genetic form of amyotrophic lateral sclerosis and frontotemoral dementia (FTD), caused by expansion repeats in the gene C9ORF72.
“The candidate molecule works by correcting the defect in protein trafficking pathway in ALS,” AcuraStem CSO Qing Liu told us.
If approved, AS2015 will compete with the two small molecule treatments for ALS currently on the market: Sanofi’s Riluzole and Mitsubishi Tanabe’s Radicava.
“The [AS2015] mechanism is unique and in our assay, our candidate works better than Riluzole and Radicava,” said Liu.
The candidate – which will be administered in an oral tablet or liquid form – could be ‘potentially’ cheaper than Riluzole and Radicava, and “much easier than [intravenous medication] Radicava to administer,” he added.
AcuraStem does not yet have a manufacturing partner for the candidate.
Earlier this year, we reported that Takeda Pharmaceuticals was also looking to compete in the small molecule ALS market, through a $230m (€195m) collaboration with Wave Life Sciences. According to the agreement, the firms will develop nucleic acid therapies for a number of central nervous system disorders, including ALS.
While according to AcuraStem, “small-molecule drugs are easier to administer to patients, are often less expensive, and more easily penetrate the blood-brain barrier,” a number of firms are also investigating large molecule-based methods to treat the disease.
In February, Denali signed a biologics deal with Lonza to make neurodegenerative disease therapies, including for ALS. In addition, last month, AveXis – which was acquired by Novartis in May – announced plans to submit an investigational new drug (IND) application for a genetic ALS treatment in late 2018 or early 2019.