Applied Therapeutics Advances Pipeline Amid Financial Challenges: Q2 Earnings Breakdown

Applied Therapeutics (APLT) has released Q2 earnings, highlighting advancements in its pipeline and financial challenges. The company is preparing for a meeting with the FDA to discuss govorestat for CMT-SORD treatment, has launched a sorbitol assay, and presented promising clinical trial data. Net loss increased to $21.3 million, while cash and cash equivalents decreased to $30.4 million. APLT has entered an out-licensing agreement with Biossil and made key leadership promotions to strengthen its clinical and advocacy efforts.

Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company focused on rare diseases, has reported its second-quarter 2025 financial results. The company highlighted significant advancements in its pipeline, including progress in the development of govorestat for the treatment of CMT-SORD, and presented promising clinical trial data. Despite these developments, the company faced financial challenges, with a net loss of $21.3 million and cash and cash equivalents decreasing to $30.4 million [1].

Key highlights from the quarter include:

1. CMT-SORD Meeting with FDA: Applied Therapeutics has been granted a meeting with the U.S. Food and Drug Administration (FDA) to discuss the potential New Drug Application (NDA) submission strategy for govorestat, expected to be held in the third quarter of 2025. The company plans to present comprehensive data on govorestat for the treatment of Sorbitol Dehydrogenase Deficiency (SORD), a subtype of Charcot-Marie-Tooth (CMT) disease [1].

2. Sorbitol Assay Launch: In July 2025, the company launched a sponsored Urine Sorbitol Assay to support the identification of patients suspected of having CMT-SORD. This assay is available to healthcare providers at no cost, aiming to remove potential barriers to diagnosis and treatment [1].

3. Clinical Trial Data: Full 12-month clinical results and new topline data from the INSPIRE Phase 2/3 trial of govorestat for CMT-SORD were presented at the Peripheral Nerve Society (PNS) 2025 Annual Meeting. The data showed statistically significant improvements across key secondary endpoints and demonstrated slowed progression of disease via MRI at 24 months [1].

4. Out-Licensing Agreement: Applied Therapeutics entered into an out-licensing agreement with Biossil, Inc. for AT-001, its investigational Aldose Reductase Inhibitor (ARI) developed for Diabetic Cardiomyopathy (DbCM). Biossil gained exclusive worldwide rights to develop and commercialize AT-001, excluding a select number of rare disease indications [1].

5. Leadership Promotions: The company promoted Evan Bailey, M.D., to Chief Medical Officer, and Dottie Caplan to Executive Vice President, Patient Advocacy and Government Affairs, to strengthen its clinical and advocacy efforts [1].

Financial Results:

- Cash and cash equivalents totaled $30.4 million as of June 30, 2025, compared to $79.4 million at December 31, 2024.
- Research and development expenses for the three months ended June 30, 2025, were $9.9 million, compared to $10.0 million for the same period in 2024.
- General and administrative expenses were $13.2 million for the three months ended June 30, 2025, compared to $10.6 million for the same period in 2024.
- Net loss for the second quarter of 2025 was $21.3 million, or $0.15 per basic and diluted common share, compared to a net income of $2.9 million, or $0.02 per basic common share, and a net loss of $0.13 per diluted common share, for the second quarter of 2024 [1].

References:
[1] https://www.biospace.com/press-releases/applied-therapeutics-reports-second-quarter-2025-financial-results