Applied Therapeutics Eyes FDA NDA After CMT-SORD Trial Setback and $75M Stock Raise

Introduction

Applied Therapeutics Inc (NASDAQ: APLT) is a clinical-stage biopharmaceutical company headquartered in New York, New York. The firm utilizes biomarker guidance and molecular targeting to develop treatments for rare diseases with unmet medical needs. Its strategy includes accelerated clinical pathways and validated disease endpoints to expedite the delivery of therapies to patients.

Corporate Structure

Founded to focus on rare and metabolic disorders, Applied Therapeutics employs between 11 and 50 professionals, including leaders in corporate development, chemistry, manufacturing, and formulation. Key executives include Constantine Chinoporos (Corporate Development), Reena Thomas Colacot and Kimberly Gutek Cox (Operations), and Andrew Wasmuth (VP of Chemistry, Manufacturing, and Formulation). The compact team brings expertise in drug discovery, clinical operations, and regulatory affairs.

Biopharmaceuticals by little plant

Recent Developments and News

• August 13, 2025: In its second-quarter financial report, Applied Therapeutics announced a meeting with the U.S. Food and Drug Administration scheduled for Q3 2025 to discuss a New Drug Application (NDA) for govorestat for Charcot-Marie-Tooth disease type SORD (CMT-SORD). The company also launched a sponsored urine sorbitol assay to assist in the early diagnosis of CMT-SORD and presented 12-month results from the INSPIRE Phase 2/3 trial, highlighting statistical improvements on secondary endpoints.
• August 14, 2025: A Schedule 13G/A filing disclosed that T. Rowe Price Investment Management holds 18,683,990 shares (13.2% of outstanding stock), with sole voting power.
• August 29, 2025: Applied Therapeutics filed an 8-K reporting current operations and strategic initiatives; on the same day, it filed a prospectus supplement (Rule 424(b)(5)) authorizing the sale of up to $75 million of common stock through Cantor Fitzgerald & Co. as sales agent, with a fee of 3.0% of gross proceeds.
• August 2025: Biospace reported that govorestat did not meet its primary endpoint in the INSPIRE Phase 2/3 trial for sorbitol dehydrogenase deficiency, indicating no statistically significant difference versus placebo on the 10-meter walk-run test at 12 months. Applied noted positive outcomes on secondary measures and plans for additional FDA interactions.

Financial and Strategic Analysis

As of September 29, 2025, APLT shares traded at $0.5599, reflecting a 54.85% decline over the past year on a volume of 6.6 million shares. Key metrics from Yahoo Finance include:
• Market capitalization of $178.6 million and enterprise value of $150.8 million
• Trailing twelve-month revenue of $121,000 and a net loss of $67.7 million
• Total cash of $30.4 million against debt representing 14.9% of equity
• Price/Sales ratio of 1,480x and Price/Book ratio of 10.24x

The proposed $75 million equity offering under the 424B5 prospectus is intended to enhance cash reserves for ongoing clinical development and corporate activities. Significant shareholder T. Rowe Price may provide strategic support for advancing the company's pipeline.

Market Position and Industry Context

Applied Therapeutics operates within the biotechnology research sector, focusing on rare metabolic and genetic diseases. Its primary technology platform includes novel aldose reductase inhibitors (ARIs) being developed for galactosemia, SORD deficiency (CMT-SORD), PMM2-CDG, and diabetic retinopathy. The rare disease market offers expedited regulatory pathways due to unmet patient needs; however, competition from established biopharmaceutical firms and emerging gene therapy approaches presents various challenges. Successful outcomes depend on robust clinical data, favorable FDA interactions, and the ability to secure funding for ongoing needs.

tl;dr

• Applied Therapeutics is preparing for an NDA meeting with the FDA in Q3 2025 for govorestat in CMT-SORD in light of a recent Phase 2/3 trial failure regarding its primary endpoint.
• A $75 million equity offering and active involvement from T. Rowe Price aim to support continued advancement of the pipeline into late-stage trials and potential market approvals.