Applied Therapeutics Stock Craters After FDA Rejects Rare Genetic Disorder Drug Candidate: Retail Mood Dented
By Stocktwits Inc | First Published Nov 29, 2024, 6:19 PM ISTA CRL is a communication from the FDA that the agency cannot approve a pending regulatory application before it in its present form.
Applied Therapeutics, Inc. ($APLT), a clinical-stage biopharma developing drugs against molecular targets in rare diseases, announced late-Wednesday an adverse regulatory outcome for its lead candidate.
The New York-based company said the FDA issues a complete response letter (CRL) for its new drug application (NDA) for govorestat, which is being evaluated as a treatment option for classic galactosemia.
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The drug regulator cited deficiencies in the clinical application for the action.
Govorestat in galactosemia had a FDA decision date, aka Prescription Drug User Fee Act (PDUFA) date, of Nov. 28.
A CRL is a communication from the FDA that the agency cannot approve a pending regulatory application before it in its present form but it does not mean an outright rejection.
Shoshana Shendelman, founder and CEO of Applied Therapeutics, said, “We are disappointed by the FDA’s decision today.”"
“Galactosemia is a progressive and debilitating disease without any existing treatment options and there remains a high unmet medical need for this community,” the executive said.
Data shared by the company showed that galactosemia incidence is about 3,300 patients in the U.S., with 80-100 new births per year, and 4,400 patients in the E.U., with 120 new births per year.
Govorestat, codenamed AT-007, is a central nervous system penetrant aldose reductant inhibitor (ARI), which is being developed for multiple rare diseases.
Shendelman said, “As we move forward, we plan to work with the FDA to address the concerns in the CRL and determine an expeditious path to bring this much needed treatment to patients.”
On Stocktwits, sentiment toward Applied Therapeutics stock remained ‘bearish’ (43/100), although a slight improvement from the ‘extremely bearish’ mood that prevailed a day ago, immediately after the announcement. Retail chatter remained brisk at ‘high’ levels
On Friday, in premarket-session, the stock was down a whopping 68.03% at $2.74. The stock has run up over 155% so far this year.
Retail lamented the fact that galactosemia is now a distant opportunity. Even if the biotech resubmits the application by early 2025, an approval is likely only by the year-end, a user said, adding the company might also be running low on cash.
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