Capricor Therapeutics(CAPR) - 2025 Q2 - Quarterly Results

Corporate Update and Recent Highlights The company advanced its Deramiocel regulatory path, initiated clinical trials for its StealthX™ platform, and achieved commercial manufacturing readiness Deramiocel Program Update The company is advancing the regulatory pathway for Deramiocel with the FDA while reporting positive long-term clinical data - A Type A meeting with the U.S. FDA is scheduled for August 2025 to discuss the Deramiocel BLA path forward after receiving a Complete Response Letter (CRL)[3][5][6] - The company aims to resubmit the BLA based on HOPE-2 data, with HOPE-3 topline data expected in Q4 2025 as potential support[3][5][6] - Four-year HOPE-2 extension data showed continued preservation of cardiac function (LVEF) and slowed skeletal muscle decline in patients treated with Deramiocel[7] - The FDA granted Orphan Drug Designation for Deramiocel in Becker muscular dystrophy (BMD)[13] StealthX™ Platform Update The StealthX™ exosome platform entered the clinical stage with FDA clearance and the initiation of a Phase 1 vaccine trial - The FDA cleared the IND for the StealthX™ exosome-based vaccine, marking its first-in-human use[3][5][13] - A Phase 1 clinical trial was initiated in August 2025, conducted and funded by NIAID as part of the Project NextGen initiative[3][13] Manufacturing and Commercial Readiness Capricor successfully resolved all FDA Pre-License Inspection observations, confirming its commercial manufacturing readiness - All 483 Pre-License Inspection observations have been resolved and accepted by the FDA, a key manufacturing milestone[3][5][6] Second Quarter 2025 Financial Results The company reported zero revenue and a wider net loss for Q2 2025, while maintaining a cash runway into late 2026 Key Financial Metrics The company reported zero revenue and a wider net loss in Q2 2025 due to increased operating expenses and prior milestone recognition Quarterly Financial Performance | Financial Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Revenues | $0 | $4.0M | -100% | | Total Operating Expenses | $27.7M | $15.6M | +77.8% | | Net Loss | ($25.9M) | ($11.0M) | +135.5% | | Net Loss Per Share | ($0.57) | ($0.35) | +62.9% | Half-Year Financial Performance | Financial Metric | H1 2025 | H1 2024 | Change | | :--- | :--- | :--- | :--- | | Revenues | $0 | $8.9M | -100% | | Total Operating Expenses | $52.7M | $30.7M | +71.6% | | Net Loss | ($50.3M) | ($20.8M) | +141.9% | | Net Loss Per Share | ($1.10) | ($0.66) | +66.7% | Financial Position and Outlook The company holds a strong cash position of $122.8 million, sufficient to fund operations into the fourth quarter of 2026 - Cash and equivalents totaled $122.8 million as of June 30, 2025, compared to $151.5 million at year-end 2024[8] - The current cash balance is expected to fund operations into the fourth quarter of 2026[5][12] Company and Product Information This section details the company's focus on rare disease therapeutics, its lead candidate Deramiocel, and its StealthX™ platform About Deramiocel (CAP-1002) Deramiocel is an allogeneic cell therapy for dystrophinopathies that has received multiple special regulatory designations - Deramiocel is an allogeneic cardiosphere-derived cell (CDC) therapy with immunomodulatory and anti-fibrotic actions[17] - The therapy holds multiple key regulatory designations, including Orphan Drug, RMAT, and Rare Pediatric Disease designations[18] About Capricor Therapeutics Capricor Therapeutics is a biotechnology company developing cell and exosome-based therapies for rare diseases - Capricor is a biotechnology company focused on cell and exosome-based therapeutics for rare diseases[19] - The company's pipeline is led by Deramiocel for DMD and the StealthX™ exosome platform for vaccines and targeted delivery[19] Financial Statements This section presents the company's unaudited consolidated statements of operations and summary balance sheets for the period Consolidated Statements of Operations and Comprehensive Loss The statement of operations shows zero revenue and a higher net loss for the three and six months ended June 30, 2025 Unaudited Consolidated Statements of Operations and Comprehensive Loss | (In thousands, except per share data) | Three Months Ended June 30, | Six Months Ended June 30, | | :--- | :--- | :--- | | | 2025 | 2024 | 2025 | 2024 | | Revenue | $0 | $3,971 | $0 | $8,878 | | Operating Expenses | | | | | | Research and development | $22,047 | $12,505 | $40,963 | $23,606 | | General and administrative | $5,672 | $3,058 | $11,739 | $7,130 | | Total Operating Expenses | $27,719 | $15,563 | $52,702 | $30,735 | | Loss from Operations | ($27,719) | ($11,591) | ($52,702) | ($21,857) | | Net Loss | ($25,911) | ($11,000) | ($50,302) | ($20,794) | | Net Loss Per Share | ($0.57) | ($0.35) | ($1.10) | ($0.66) | Summary Balance Sheets The balance sheet reflects a decrease in cash and total assets, resulting in lower stockholders' equity as of June 30, 2025 Unaudited Summary Balance Sheets | (In thousands) | June 30, 2025 (unaudited) | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $122,801 | $151,516 | | Total Assets | $133,569 | $170,481 | | Total Liabilities | $28,592 | $25,019 | | Total Stockholders' Equity | $104,977 | $145,462 | | Total Liabilities and Stockholders' Equity | $133,569 | $170,481 |