Applied Therapeutics, Inc. (NASDAQ: APLT) witnessed a notable surge of 39.93% in its stock price on Thursday, culminating in a closing figure of $3.75. This significant uptick in APLT stock followed the release of encouraging interim results from a clinical trial.
Applied Therapeutics (APLT) disclosed positive interim findings from the ongoing Phase 3 INSPIRE trial, marking a milestone as the primary and several key secondary endpoints were successfully met. The INSPIRE trial, a Phase 3 double-blind placebo-controlled registrational study, is evaluating the efficacy of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency across the US and Europe.
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The primary goal of the pre-specified 12-month interim analysis was to discern early indications of govorestat’s therapeutic efficacy, aiming to inform future regulatory deliberations and bolster a potential New Drug Application (NDA) submission. Given the pressing demand for treatment options and the dearth thereof for patients grappling with SORD Deficiency, this interim assessment carried substantial weight.
The analysis encompassed both a clinical efficacy primary endpoint, revolving around the correlation of sorbitol with composite clinical outcome measures, and a pharmacodynamic (PD) biomarker primary endpoint, predicated on sorbitol reduction. The interim analysis yielded significant revelations, underscoring the potential of govorestat in ameliorating the plight of individuals grappling with SORD Deficiency.
Notably, there emerged a robust correlation between sorbitol levels and the designated clinical endpoint, namely the CMT-FOM composite. Patients subjected to govorestat treatment exhibited a sustained reduction in sorbitol levels over the 12-month period, a phenomenon conspicuously absent among their counterparts who received a placebo.
Buoyed by the affirmative outcomes of the 12-month interim analysis, Applied Therapeutics remains resolute in its conviction regarding the pivotal role of sorbitol in driving disease severity and progression. With the ongoing INSPIRE trial slated for reassessment at the 24-month mark, the Company anticipates a comprehensive evaluation of clinical outcomes, with the 10-meter walk-run test assuming precedence as the primary clinical efficacy endpoint.
Leveraging the amassed clinical data, Applied Therapeutics envisages engaging in deliberations with the U.S. Food and Drug Administration (FDA) regarding a potential NDA submission, underscoring its unwavering commitment to advancing therapeutic paradigms and addressing unmet medical needs.
