Applied Therapeutics Stock Skyrockets 121% on New Trial Plans
Surge follows announcement of a new confirmatory Phase 3 trial for its lead drug candidate, AT-007, for a rare neurodegenerative disease.
Shares of Applied Therapeutics (NASDAQ: APLT) in recent trading following a significant update on the clinical development of its leading drug candidate. The massive rally was triggered by the company's announcement of its intention to launch a new Phase 3 clinical trial for AT-007, also known as govorestat, a potential treatment for Charcot-Marie-Tooth (CMT) disease.
The update has ignited investor confidence, signaling a clear and potentially accelerated path toward regulatory approval. The company unveiled plans for a for AT-007. This new study is designed to affirm the long-term efficacy and clinical benefits of the drug for patients with CMT disease associated with Sorbitol Dehydrogenase (SORD) deficiency, a rare and progressive neurodegenerative condition.
Analysts noted that the description of the study as "confirmatory" suggests the company has had positive discussions with the FDA. This could place AT-007 on a Subpart H accelerated approval pathway, which allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need. This news builds on a foundation of encouraging clinical data, including from the ongoing INSPIRE Phase 3 trial, which demonstrated significant reductions in sorbitol levels, a key biomarker of the disease.
The upcoming trial will further evaluate the effects of govorestat on a range of critical outcomes, including functional and patient-reported measures like the CMT-Health Index and the 10-meter walk/run test. With a clear clinical path forward, Applied Therapeutics has indicated it for AT-007 in 2025, marking a pivotal moment for the company and for patients awaiting new treatment options.