Applied Therapeutics has provided an unusual non-update, announcing that the biotech is still figuring out a path for its rare disease asset with the FDA, following a flurry of speculation late last week tied to the emergence of a new clinical trial listing.
Before markets opened this morning, the New York-based biotech said it had met with the FDA about its late-stage lead asset, a selective aldose reductase inhibitor known as govorestat. The drug is designed to treat Charcot-Marie-Tooth sorbitol dehydrogenase deficiency (CMT-SORD), a rare subtype of CMT that causes progressive, debilitating muscle weakness and sensory loss.
Applied Tx will wait to finalize its regulatory submission plan until official minutes from the meeting are issued, according to the Sept. 29 release. Generally, FDA meeting minutes are delivered within 30 days.
No decision has been made at this point about potential accelerated approval for govorestat, according to the biotech.
Back in May, Applied Tx said that the candidate missed in a phase 2/3 trial for the rare disease after failing to improve scores in a 10-meter walk-run test at 12 months when compared to the placebo.
The biotech caveated the failure by pointing out that the 10-meter test has since been removed from the industry’s performance-based assessment for adults with CMT. In fact, CMT-SORD was only identified as a subtype of CMT a few years ago, the biotech said.
The company instead zeroed in on the study’s secondary endpoints, which included a statistically significant improvement in a patient-reported outcome measure and a lowering blood sorbitol levels.
Analysts with William Blair deemed today’s announcement as “somewhat atypical,” but highlighted a new clinical trial listing for govorestat in the condition that emerged late last week and likely triggered many questions from investors and the public.
The new study is a phase 3, double-blinded, placebo-controlled trial that will evaluate the change in a patient-reported industry measure after 36 months of treatment, using the 10-meter walk-run test and whole blood sorbitol levels as secondary measures.
The study’s status is “not yet recruiting,” with an estimated start date slated for October of this year, according to the federal database.
William Blair analysts said they were “hesitant to draw conclusions” about this morning’s announcement, but added that they expect Applied Tx to bear “increased financial risk from its limited cash runway,” citing the $30.4 million the biotech had to hand as of June 30.
The biotech’s stock has fallen nearly 50% from its previous close of $1.24 per share, currently resting at $0.66 as of 10:45 a.m. ET this morning. The company’s stock spiked at the end of last week, likely due to speculation regarding the clinical trial listing.
Applied Tx is also developing govorestat for other rare diseases, such as classic galactosemia.
In Dec. 2024, the FDA rejected a new drug application for the drug in children with the disorder, while also reprimanding the biotech for the deletion of electronic data by a third-party vendor and the mishandling of a dosing error that led to some patients initially receiving lower levels of govorestat than intended.
The application itself was based on a failed phase 3 trial from 2023, but Applied Tx had held out hope that it still had the data needed for the rare disease approval.
Weeks after the FDA rebuff, Applied Tx’s CEO Shoshana Shendelman, Ph.D., departed, with Chief Financial Officer Les Funtleyder stepping in as interim CEO.