Applied Therapeutics(APLT) - 2025 Q2 - Quarterly Results

Applied Therapeutics Second Quarter 2025 Results Management Commentary The interim CEO highlighted strong execution in H1 2025, focusing on clinical operations, pipeline advancement, and regulatory alignment for rare disease programs - The company is preparing for a potential New Drug Application (NDA) submission for govorestat for the treatment of CMT-SORD, with a meeting scheduled with the FDA in Q3 2025[3] - A new sponsored sorbitol assay has been launched at no cost to patients to remove barriers to diagnosis and treatment for the CMT-SORD community[3] - The company's primary focus for the remainder of the year is on regulatory alignment across its pipeline for rare diseases, including CMT-SORD, Classic Galactosemia, and PMM2-CDG[3] Recent Highlights & Pipeline Update Applied Therapeutics reported significant progress across its clinical pipeline, advancing CMT-SORD, addressing Classic Galactosemia CRL, presenting PMM2-CDG data, and completing corporate developments CMT-SORD Program The govorestat program for CMT-SORD is advancing toward a potential NDA submission, supported by positive 24-month MRI data and a newly launched no-cost Urine Sorbitol Assay - A meeting with the FDA is scheduled for Q3 2025 to align on the planned NDA submission strategy for govorestat in treating CMT-SORD[6] - Full 12-month results and 24-month MRI data from the INSPIRE Phase 2/3 trial were presented, demonstrating that govorestat slowed the progression of the disease and was generally safe and well-tolerated[7] - In July 2025, the company launched a sponsored, no-cost Urine Sorbitol Assay to help healthcare providers identify patients with suspected CMT-SORD[6][7] Classic Galactosemia Program The company is actively evaluating its response to the FDA's Complete Response Letter (CRL) for govorestat in Classic Galactosemia, with the review progressing as planned - The company is actively evaluating its response to the FDA's Complete Response Letter (CRL) for govorestat in Classic Galactosemia, with the process progressing as planned[8] PMM2-CDG Program Results from an investigator-initiated trial of govorestat for PMM2-CDG are scheduled for presentation, with govorestat having received Orphan Drug and Rare Pediatric Disease designations - Data from an ongoing trial of govorestat for PMM2-CDG will be presented at the ASHG Annual Meeting in October 2025[9] - Govorestat has received Orphan Drug Designation and Rare Pediatric Disease designation from the FDA for the treatment of PMM2-CDG, an ultra-rare and severe metabolic disorder[9] Corporate Developments The company entered an out-licensing agreement for AT-001 for Diabetic Cardiomyopathy and made key leadership promotions in July 2025 - Entered into an out-licensing agreement with Biossil, Inc. for AT-001, granting Biossil exclusive worldwide rights to develop and commercialize the drug for Diabetic Cardiomyopathy (DbCM), with Applied Therapeutics receiving an upfront payment and eligibility for future royalties and milestones[10] - Promoted Evan Bailey, M.D., to Chief Medical Officer and Dottie Caplan to Executive Vice President, Patient Advocacy and Government Affairs[10] Second Quarter 2025 Financial Results Applied Therapeutics reported a net loss of $21.3 million for Q2 2025, a significant shift from a net income of $2.9 million in Q2 2024, primarily due to changes in warrant liabilities and increased G&A expenses Financial Metrics | Financial Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Net Income (Loss) | $(21.3) million | $2.9 million | Decreased significantly | | Net Loss per Share (basic) | $(0.15) | $0.02 (income) | N/A | | R&D Expenses | $9.9 million | $10.0 million | -1.0% | | G&A Expenses | $13.2 million | $10.6 million | +24.5% | | Cash & Cash Equivalents | $30.4 million (as of Jun 30, 2025) | $79.4 million (as of Dec 31, 2024) | -61.7% YTD | - The increase in General and administrative expenses was primarily driven by higher legal, professional, personnel, and data storage costs[16] Financial Statements The condensed financial statements detail the company's financial position and operational results, reflecting a significant reduction in cash and total assets and a net loss for the quarter due to non-operational changes Condensed Balance Sheets As of June 30, 2025, total assets decreased to $37.3 million from $86.7 million at year-end 2024, primarily due to a decline in cash and cash equivalents Balance Sheet Summary (in thousands) | (in thousands) | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $30,421 | $79,398 | | Total current assets | $34,511 | $83,646 | | Total Assets | $37,342 | $86,691 | | Liabilities & Equity | | | | Total current liabilities | $17,749 | $27,296 | | Total liabilities | $19,904 | $29,685 | | Total stockholders' equity | $17,438 | $57,006 | | Total Liabilities and Stockholders' Equity | $37,342 | $86,691 | Condensed Statements of Operations For Q2 2025, the company reported no revenue and a net loss of $21.3 million, contrasting with a $2.9 million net income in Q2 2024, largely due to changes in the fair value of warrant liabilities Statements of Operations Summary (in thousands, except per share data) | (in thousands, except per share data) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Total revenue | $— | $144 | | Research and development | $9,923 | $10,004 | | General and administrative | $13,175 | $10,580 | | Loss from operations | $(23,098) | $(20,440) | | Change in fair value of warrant liabilities | $1,437 | $22,744 | | Net income (loss) | $(21,330) | $2,898 | | Net income (loss) per share - basic | $(0.15) | $0.02 |