Opus Genetics, Inc.(IRD) - 2025 Q1 - Quarterly Results

Executive Summary Opus Genetics reported Q1 2025 financial results and corporate updates, highlighting progress in gene therapy programs and a strong financial position Q1 2025 Performance Overview Opus Genetics reported Q1 2025 financial results, highlighting progress in IRD gene therapy, positive OPGx-LCA5 data, and successful financing - Opus Genetics announced financial results for Q1 2025 and provided a corporate update[2] - Reported positive 12-month data from the Phase 1/2 trial of OPGx-LCA5, showing durable efficacy in adult patients[3] - Pediatric patient enrollment for LCA5 study is ongoing, with initial multi-patient data anticipated in Q3 2025[1][3] - OPGx-BEST1 program is on track for IND filing and initiation of Phase 1/2 trial, with early data expected in Q1 2026[1][4] - Completed a successful financing round with leading institutional healthcare investors[1][4] CEO Commentary CEO George Magrath emphasized a strong start to 2025, citing progress across the IRD platform and phentolamine eye drop franchise, with encouraging OPGx-LCA5 efficacy and upcoming clinical data readouts - CEO George Magrath stated Opus is off to a strong start in 2025 with progress across two product portfolios: IRD platform and phentolamine eye drop franchise[3] - Expressed belief that initial success with OPGx-LCA5 has the potential to translate to the rest of the pipeline, including six additional IRD gene therapy candidates[4] - Anticipates near-term clinical data readouts from Phase 3 trials for the phentolamine eye drop pipeline in dim light vision disturbances and presbyopia[4] - Concluded the quarter with a strong cash position following a successful financing round[4] Strategic Highlights and Corporate Updates This section details Opus Genetics' advancements in gene therapy programs, including OPGx-LCA5 and OPGx-BEST1, and the phentolamine eye drop franchise, alongside key upcoming milestones Gene Therapy Programs Opus Genetics is advancing its pipeline of adeno-associated virus (AAV)-based gene therapies for inherited retinal diseases (IRDs), with significant clinical and regulatory progress for OPGx-LCA5 and OPGx-BEST1, and a successful financing round raising approximately $21.5 million - Opus's pipeline includes a portfolio of seven adeno-associated virus (AAV)-based gene therapy assets, each targeting a specific IRD[7] - In March 2025, Opus completed a successful underwritten public offering and concurrent private placement, raising approximately $21.5 million in gross proceeds[7] OPGx-LCA5 Program Update OPGx-LCA5, targeting severe vision loss from LCA5 gene mutations, demonstrated durable efficacy in adult patients over one year in its Phase 1/2 trial, with pediatric enrollment underway and RMAT designation from the FDA - Emerging clinical data on OPGx-LCA5 provide evidence of clinical proof of concept and support its potential to restore meaningful vision for individuals with LCA5 gene mutations[7] - One-year open-label data on adult patients in the Phase 1/2 trial showed sustained improvements in subjective and objective measures of efficacy[7] - Enrollment in a cohort of three pediatric patients began in February 2025, with preliminary data on the first patient showing an encouraging early safety profile and meaningful improvement in visual function at one month[7] - The U.S. FDA granted a Regenerative Medicine Advanced Therapy (RMAT) designation to OPGx-LCA5, offering potential for expedited development and review[7] - A Type D meeting was held with the FDA in March 2025 to discuss the potential regulatory path for OPGx-LCA5, including the design of a potential registrational study[8] OPGx-BEST1 Program Update OPGx-BEST1, an investigational gene therapy for IRDs associated with BEST1 gene mutations, is Phase 1/2-ready, with IND-enabling studies supporting a first-in-human trial planned for Q4 2025 and preliminary data anticipated in Q1 2026 - OPGx-BEST1 is an investigational Phase 1/2-ready asset in development for IRDs associated with mutations in the BEST1 gene[11] - IND-enabling studies provided safety and efficacy data supporting a first-in-human clinical trial[11] - Opus plans to file an IND and begin a Phase 1/2 trial by the fourth quarter of 2025, with preliminary data expected in the first quarter of 2026[11] Phentolamine Ophthalmic Solution 0.75% Program Update Phentolamine Ophthalmic Solution 0.75% is rapidly progressing through pivotal Phase 3 trials for dim light vision disturbances (LYNX-2) and presbyopia (VEGA-3), with enrollment completed in Q1 2025 and topline data expected mid-year and in the first half of 2025, respectively, with funding from Viatris Inc - The LYNX-2 pivotal Phase 3 trial for visual loss in low light conditions associated with keratorefractive surgery completed enrollment in Q1 2025, with topline data expected mid-year 2025[11] - LYNX-2 trial is covered by a Special Protocol Assessment (SPA) agreement with the FDA and received Fast Track designation[11] - The VEGA-3 pivotal Phase 3 trial for presbyopia completed enrollment in Q1 2025, with topline data expected in the first half of 2025[11] - The development portfolio related to Phentolamine Ophthalmic Solution 0.75% is being funded by the Company's partner, Viatris Inc[11] Expected Growth Drivers and Milestones Key upcoming milestones include initial data from pediatric OPGx-LCA5 patients in Q3 2025, IND filing and Phase 1/2 trial initiation for OPGx-BEST1 in 2025 with preliminary data in Q1 2026, and topline data from both LYNX-2 and VEGA-3 Phase 3 trials in mid-2025 and H1 2025, respectively - Initial data from three pediatric patients treated with OPGx-LCA5 anticipated in Q3 2025[11] - IND filing and initiation of a Phase 1/2 clinical trial for OPGx-BEST1 is planned for 2025, with preliminary data expected in Q1 2026[11] - Topline data from the LYNX-2 pivotal Phase 3 trial for visual loss in low light conditions associated with keratorefractive surgery are expected mid-year 2025[11] - Topline data from the VEGA-3 pivotal Phase 3 clinical trial for the treatment of presbyopia are expected in the first half of 2025[11] Financial Highlights for the First Quarter Ended March 31, 2025 This section provides a detailed overview of Opus Genetics' financial performance for Q1 2025, covering cash position, revenue, operating expenses, and net loss Cash Position and Runway As of March 31, 2025, Opus Genetics reported a cash and cash equivalents balance of $41.8 million, projected to fund operations into the second quarter of 2026 Cash and Cash Equivalents | Metric | As of March 31, 2025 | As of December 31, 2024 | | :----- | :------------------- | :---------------------- | | Cash and cash equivalents | $41.8 million | $30.3 million | - Management believes current cash on hand is sufficient to fund operations into the second quarter of 2026[12] Revenue Performance License and collaborations revenue significantly increased to $4.4 million in Q1 2025 from $1.7 million in Q1 2024, primarily driven by reimbursement of research and development services under the Viatris License Agreement, with a smaller contribution from RYZUMVI™ royalties License and Collaborations Revenue (Three Months Ended March 31) | Metric | 2025 (in thousands) | 2024 (in thousands) | Change (YoY) | | :----- | :------------------ | :------------------ | :----------- | | Revenue | $4,370 | $1,711 | +155.4% | - Revenue was derived from the Viatris License Agreement, largely from reimbursement of R&D services and to a lesser degree from royalty payments from RYZUMVI™ sales[13] Operating Expenses Both General and Administrative (G&A) and Research and Development (R&D) expenses increased in Q1 2025 compared to Q1 2024, with G&A rising due to professional services and public company costs, and R&D increasing due to higher clinical and payroll costs, partially offset by lower manufacturing expenses, with R&D expenses for Phentolamine Ophthalmic Solution 0.75% fully reimbursed by Viatris Operating Expenses (Three Months Ended March 31) | Expense Category | 2025 (in thousands) | 2024 (in thousands) | Change (YoY) | | :--------------- | :------------------ | :------------------ | :----------- | | General and administrative | $6,346 | $4,670 | +35.9% |\n| Research and development | $7,953 | $4,749 | +67.5% |\n| Total operating expenses | $14,299 | $9,419 | +51.8% | - G&A increase primarily attributable to professional services fees, corporate legal support, intellectual property legal fees, and public company costs[14] - R&D increase primarily attributable to higher clinical costs and payroll-related costs, partially offset by lower manufacturing expenses and regulatory costs[15] - Budgeted R&D expenses related to Phentolamine Ophthalmic Solution 0.75% have been fully reimbursed by Viatris[15] Net Loss and EPS Opus Genetics reported an increased net loss of $8.2 million for Q1 2025, compared to $7.1 million in Q1 2024, though the net loss per share improved to $(0.24) from $(0.29) due to an increase in the number of shares outstanding Net Loss and EPS (Three Months Ended March 31) | Metric | 2025 | 2024 | Change (YoY) | | :----- | :--- | :--- | :----------- | | Net loss | $(8.2) million | $(7.1) million | +15.5% | | Basic and diluted EPS | $(0.24) | $(0.29) | +17.2% | Shares Used in Per Share Calculations (Three Months Ended March 31) | Metric | 2025 | 2024 | | :----- | :--- | :--- | | Basic and diluted shares | 33,884,920 | 24,520,475 | About Opus Genetics Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company focused on developing AAV-based gene therapies for inherited retinal diseases (IRDs) such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa, with lead gene therapy OPGx-LCA5 in a Phase 1/2 trial and other programs in Phase 3 - Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing therapies for IRDs and other ophthalmic disorders[17] - Pipeline includes AAV-based investigational gene therapies for gene mutations responsible for different forms of LCA, bestrophinopathy, and retinitis pigmentosa[17] - Most advanced investigational gene therapy program is OPGx-LCA5, currently in a Phase 1/2 open-label, dose-escalation trial[17] - Pipeline also includes BEST1 investigational gene therapy and Phentolamine Ophthalmic Solution 0.75% in Phase 3 trials for presbyopia and mesopic (dim) light vision disturbances[17] Forward-Looking Statements and Risk Factors This section contains forward-looking statements regarding Opus Genetics' future expectations, including cash runway, growth potential, and clinical trial outcomes, which are subject to various risks and uncertainties, cautioning readers to review detailed risk factors in SEC filings - The press release contains forward-looking statements regarding cash runway, potential growth, and clinical trial data/enrollment[19] - These statements are subject to risks and uncertainties that could cause actual results to differ materially, as described in the Annual Report on Form 10-K and Quarterly Report on Form 10-Q[20] - Key risks include preliminary clinical data being revised or invalidated, difficulties in developing and manufacturing novel gene therapies, potential delays or failures in clinical trials, changes in regulatory requirements, dependence on product pipeline and strategic partners, competition, and challenges in obtaining regulatory approval[21] - Additional risks involve lack of sales/marketing infrastructure, uncertain commercial success due to third-party reimbursement and competitors, product liability, compliance with health and safety laws, future capital needs and dilution, highly regulated industry, intellectual property protection, dependence on key personnel, and stock market volatility[21][22][25] Financial Statements Condensed Consolidated Balance Sheets The condensed consolidated balance sheets show Opus Genetics' financial position as of March 31, 2025, compared to December 31, 2024, with total assets increasing to $48.2 million primarily due to higher cash and cash equivalents, and total liabilities significantly rising from warrant liabilities Condensed Consolidated Balance Sheets (in thousands) | Metric | As of March 31, 2025 (Unaudited) | As of December 31, 2024 | | :-------------------------------- | :------------------------------- | :---------------------- | | Assets | | | | Cash and cash equivalents | $41,792 | $30,321 | | Total current assets | $47,928 | $36,610 | | Total assets | $48,167 | $36,862 | | Liabilities and Stockholders' Equity | | | | Warrant liabilities | $12,715 | — | | Total current liabilities | $24,251 | $11,295 | | Total liabilities | $24,251 | $11,295 | | Total stockholders' equity | $5,073 | $6,724 | Condensed Consolidated Statements of Comprehensive Loss The condensed consolidated statements of comprehensive loss for the three months ended March 31, 2025, show a net loss of $8.2 million, an increase from $7.1 million in the prior year, influenced by higher operating expenses, partially offset by increased license and collaborations revenue and a fair value change in warrant liabilities Condensed Consolidated Statements of Comprehensive Loss (in thousands) | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | License and collaborations revenue | $4,370 | $1,711 | | General and administrative | $6,346 | $4,670 | | Research and development | $7,953 | $4,749 | | Total operating expenses | $14,299 | $9,419 | | Loss from operations | $(9,929) | $(7,708) | | Fair value change in warrant liabilities | $2,805 | — | | Net loss | $(8,194) | $(7,106) | | Basic and diluted net loss per share | $(0.24) | $(0.29) | Contacts This section provides contact information for corporate inquiries and investor relations, including the CFO and LifeSci Advisors Contacts | Corporate | Investor Relations | | :-------- | :----------------- | | Nirav Jhaveri, CFO | Corey Davis, Ph.D. | | ir@ocuphire.com | LifeSci Advisors | | | cdavis@lifesciadvisors.com |