Edgewise Therapeutics(EWTX) - 2025 Q2 - Quarterly Results

Executive Summary & Q2 2025 Highlights Q2 2025 Highlights and CEO Commentary Edgewise Therapeutics reported Q2 2025 results, highlighting positive sevasemten data and EDG-7500 progress - Announced positive top-line data from the open-label extension MESA trial of sevasemten in participants with Becker muscular dystrophy[1] - Announced encouraging top-line data from the Phase 2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy[1] - Advanced the Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy (HCM)[1] - Company is advancing skeletal and cardiac muscle programs and building commercial infrastructure for a potential U.S. launch of sevasemten in Becker, while exploring Phase 3 trial designs in HCM and Duchenne[2] Recent Business Highlights Muscular Dystrophy Program Edgewise advanced its muscular dystrophy program with positive sevasemten trial results for Becker and Duchenne Sevasemten and Becker Muscular Dystrophy Sevasemten showed sustained disease stabilization in Becker muscular dystrophy, with GRAND CANYON data expected Q4 2026 - Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect against contraction-induced muscle damage in muscular dystrophies[3] - Becker muscular dystrophy is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder with no currently approved therapies[4] - Positive data from the MESA open-label extension trial showed sustained disease stabilization in NSAA scores for Becker participants, with a favorable safety profile after up to three years of treatment[5][6] - The global pivotal placebo-controlled GRAND CANYON trial completed enrollment of 175 adults in February 2025 and is on track for top-line data in Q4 2026[7][8] - The Company completed a successful Type C meeting with the FDA, providing a clear path to registration of sevasemten as the first therapy for Becker[8] Sevasemten and Duchenne Muscular Dystrophy Sevasemten Phase 2 trials in Duchenne showed encouraging results, identifying a 10 mg dose for Phase 3 - Duchenne is the most common type of muscular dystrophy, a severe degenerative muscle disorder with high unmet medical need despite existing therapies[9] - LYNX Phase 2 trial showed encouraging observations across functional measures (SV95C, NSAA, 4 stair-climb) and identified a 10 mg dose for Phase 3[10] - FOX Phase 2 trial in Duchenne boys previously treated with gene therapy supports the potential of sevasemten 10 mg to reduce the rate of functional decline[11] - The Company plans to meet with the FDA in Q4 2025 to discuss Phase 3 design and initiate a pivotal study in 2026[12] Cardiovascular and Cardiometabolic Programs Edgewise advances cardiovascular programs with EDG-7500 in Phase 2 for HCM and EDG-15400 entering Phase 1 EDG-7500 and Hypertrophic Cardiomyopathy (HCM) The CIRRUS-HCM Phase 2 trial for EDG-7500 is recruiting for Part D, with an update expected in Q4 2025 - EDG-7500 is a novel oral, selective, cardiac sarcomere modulator designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM[14] - HCM is the most common form of genetic heart disease, affecting approximately one in 500 people, with significant unmet needs for additional therapeutic approaches, particularly for nonobstructive HCM (nHCM)[14] - The multi-part, open-label CIRRUS-HCM Phase 2 trial is actively recruiting participants for Part D (12-week study in oHCM and nHCM), with an update expected in Q4 2025[15] Heart Failure and Preclinical Programs Edgewise plans to initiate a Phase 1 trial for EDG-15400 in heart failure in Q3 2025 - The Company expects to begin dosing in a first-in-human Phase 1 trial of EDG-15400 for heart failure during Q3 2025[16] - Edgewise continues to advance its preclinical cardiometabolic programs[16] Strengthened Engagement with the Scientific and Patient Communities Edgewise continued engagement with muscular dystrophy and HCM communities through educational outreach and conferences - The Company participated in the Parent Project Muscular Dystrophy annual conference, CureDuchenne Futures conference, and the Annual Congress of the Heart Failure Association of the ESC[17] - Edgewise continues to sponsor and participate in numerous other clinician and patient-focused events[17] Second Quarter Financial Results Financial Position (Cash, Cash Equivalents, Marketable Securities) Edgewise Therapeutics maintained a strong liquidity position with $594.0 million in cash, cash equivalents, and marketable securities Cash, Cash Equivalents and Marketable Securities | Metric | June 30, 2025 | | :----- | :------------ | | Cash, cash equivalents and marketable securities | $594.0 million | Operating Expenses Total operating expenses for Q2 2025 decreased to $42.6 million, primarily due to reduced R&D activities Total Operating Expenses (QoQ) | Metric | Q2 2025 | Q1 2025 | Change | | :----- | :------ | :------ | :----- | | Total operating expenses | $42.610 million | $45.959 million | -$3.349 million | Research and Development (R&D) Expenses R&D expenses decreased by $3.2 million to $33.6 million in Q2 2025, driven by reduced clinical development and personnel costs Research and Development Expenses (QoQ) | Metric | Q2 2025 | Q1 2025 | Change | | :----- | :------ | :------ | :----- | | R&D expenses | $33.6 million | $36.8 million | -$3.2 million | - Decrease primarily driven by a $2.6 million decrease in sevasemten clinical development, a $0.9 million decrease in EDG-7500 clinical development, and a $1.1 million decrease in personnel-related costs, offset by a $1.7 million increase in manufacturing expenses[19] General and Administrative (G&A) Expenses G&A expenses remained stable at $9.1 million for Q2 2025 General and Administrative Expenses (QoQ) | Metric | Q2 2025 | Q1 2025 | Change | | :----- | :------ | :------ | :----- | | G&A expenses | $9.1 million | $9.2 million | -$0.1 million | Net Loss and Net Loss Per Share Edgewise reported a net loss of $36.1 million (or $0.34 per share) for Q2 2025, an improvement Net Loss and Net Loss Per Share (QoQ) | Metric | Q2 2025 | Q1 2025 | Change | | :----- | :------ | :------ | :----- | | Net loss | $(36.1) million | $(40.8) million | +$4.7 million | | Net loss per share | $(0.34) | $(0.43) | +$0.09 | Company Overview Company Mission and Product Pipeline Edgewise Therapeutics develops novel therapeutics for muscular dystrophies and cardiac conditions, with sevasemten and EDG-7500 as key candidates - Edgewise Therapeutics is a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions[21] - Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor in late-stage clinical trials for Becker and Duchenne muscular dystrophies[21] - EDG-7500 is a novel cardiac sarcomere modulator for hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development[21] Financial Statements Condensed Statement of Operations The condensed statement of operations shows a net loss of $36.1 million for Q2 2025, an improvement driven by reduced R&D expenses Condensed Statement of Operations (Three months ended, in thousands) | Operating expenses: | June 30, 2025 | March 31, 2025 | | :------------------ | :------------ | :------------- | | Research and development | $33,558 | $36,757 | | General and administrative | $9,052 | $9,202 | | Total operating expenses | $42,610 | $45,959 | | Loss from operations | $(42,610) | $(45,959) | | Interest income | $6,495 | $5,161 | | Net loss | $(36,115) | $(40,798) | | Net loss per share - basic and diluted | $(0.34) | $(0.43) | | Weighted-average shares outstanding, basic and diluted | 104,940,493 | 95,130,053 | Condensed Balance Sheet Data Total assets increased to $613.3 million as of June 30, 2025, driven by increased cash and marketable securities Condensed Balance Sheet Data (in thousands) | Assets | June 30, 2025 | December 31, 2024 | | :----- | :------------ | :---------------- | | Cash, cash equivalents and marketable securities | $593,992 | $470,170 | | Other assets | $19,342 | $16,647 | | Total assets | $613,334 | $486,817 | | Liabilities | $24,263 | $27,601 | | Stockholders' equity | $589,071 | $459,216 | | Total liabilities and stockholders' equity | $613,334 | $486,817 | Additional Information Cautionary Note Regarding Forward-Looking Statements This section outlines forward-looking statements, emphasizing inherent risks and uncertainties that could cause actual results to differ - The press release contains forward-looking statements regarding product candidates, clinical trials, potential launches, market opportunities, and financial expectations[22] - Actual results could differ materially due to numerous risks and uncertainties, including those related to early development, lack of approved products, need for capital, dependence on product candidates, clinical trial outcomes, regulatory approvals, and intellectual property[22] Company Contacts Contact information is provided for investor relations and media inquiries - Investors can contact Michael Carruthers, Chief Financial Officer, at ir@edgewisetx.com[28] - Media inquiries can be directed to Maureen Franco, VP Corporate Communications, at media@edgewisetx.com[28]