Applied Therapeutics has yet to confirm whether the study, posted on Clinicaltrials.gov on Thursday, means it has indeed aligned with the FDA on govorestat’s development.
Applied Therapeutics has posted a new study for its aldose reductase inhibitor govorestat on clinicaltrials.gov, suggesting that the company has reached an agreement with the FDA on a path forward for the drug in sorbitol dehydrogenase deficiency.
In a note on Friday, William Blair suggested that the new confirmatory study indicates the FDA may have found previously collected data sufficient to support an accelerated approval.
The study page went live on Sept. 25 and sets an enrollment target of 155 patients across 16 sites worldwide. To qualify for enrollment, patients must have been diagnosed with Charcot-Marie-Tooth Type 2 or distal Hereditary Motor Neuropathy due to sorbitol dehydrogenase deficiency (SORD). The study is designed to look at the long-term effects of govorestat on functional, clinical and patient-reported outcomes, compared with placebo. The confirmatory study has yet to start enrolling and has a primary completion date of October 2028.
In May, govorestat failed the Phase II/III INSPIRE trial, failing to significantly improve scores on the 10-meter walk-run test at 12 months compared with placebo. Applied Therapeutics at the time nevertheless doubled down on a new drug application for govorestat, focusing on positive secondary outcomes, including improvements in mobility and quality of life, alongside significant reductions in blood sorbitol levels, a key disease biomarker.
“We are encouraged by the breadth of data supporting govorestat’s ability to significantly lower blood sorbitol levels and positively impact both functional and patient-reported outcomes,” Evan Bailey, senior vice president of Clinical Development at Applied, said at the time. The biotech also indicated that it would be working with the FDA to find the best path forward for govorestat, aiming for a 2025 filing.
“The entry suggests the meeting with the agency was productive and may have concluded with the FDA finding the data sufficient to support the accelerated approval of govorestat in CMT-SORD,” according to analysts at William Blair, who flagged the database entry on Friday and noted that the new study is meant to be a confirmatory trial for govorestat.
BioSpace has reached out to Applied Therapeutics to clarify the status of govorestat.
The study posting, according to William Blair, suggests that the company has reached alignment with the FDA. Still, without official word from Applied, the analysts remain “hesitant to draw conclusions” about govorestat’s development.
William Blair also flagged Applied Therapeutics’ financial situation. As of June 30, the biotech had $30.4 million in cash and cash equivalents, which the analysts said could pose “increased financial risk” for the company, given its “limited cash runway.”
SORD is a rare genetic condition characterized by the accumulation of the buildup of sorbitol sugars in nerves, leading to sensory loss and muscle weakness. Govorestat disables the aldose reductase enzyme, in turn disrupting the pathway that culminates in the production of sorbitol.
