Acasti Pharma has shared early clinical data on its betamethasone oral-mucosal metered spray, providing preliminary validation of its belief the delivery format can address an unmet need in a rare, childhood neurological disorder.
Betamethasone is a steroid that is already given orally, topically and via injection to treat diseases such as rheumatoid arthritis and inflammatory bowel disease. Acasti thinks drug delivery technology can open up a new market for the old drug by enabling children with ataxia telangiectasia, a rare disease that can make it hard to swallow oral medications, to easily take the steroid.
In a phase 1 pharmacokinetic bridging study, Acasti compared its oral spray, GTX-102, to intramuscular betamethasone and an oral solution of the steroid that is available in Europe but not the U.S. The oral solution reduced neurological symptoms in ataxia telangiectasia in an earlier study.
The high dose of GTX-102 achieved comparable blood concentrations to the intramuscular and oral formulations of betamethasone. Based on the results, Acasti plans to determine the best final dosing regimen for GTX-102 and start a phase 3 trial in the second half of 2023. The phase 3 trial is intended to support a filing for FDA approval via the 505(b)(2) pathway.
The news comes almost three years after the failure of a late-phase trial of omega-3 drug CaPre marked the beginning of the end for Acasti’s bid to challenge Amarin’s Vascepa for the hypertriglyceridemia market. Acasti initially tried to forge ahead in the face of the setback, which torpedoed its stock, but a second phase 3 failure later in the year persuaded the biotech to drop plans to seek FDA approval.
Today, Acasti is focused on three candidates, with GTX-102 joined by an intravenous infusion treatment of subarachnoid hemorrhage, GTX-104, and a topical spray for post-herpetic neuralgia, GTX-101, in the company’s clinical pipeline. GTX-102 and GTX-104 are both scheduled to enter phase 3 this year.
Acasti had $34.9 million to its name at the end of September, a sum it predicted would fund its work until at least March 2024.