Ocuphire Pharma(OCUP) - 2025 Q2 - Quarterly Results

Executive Summary & Corporate Update Opus Genetics reported Q2 2025 financial results and provided a corporate update, highlighting significant clinical and regulatory milestones across its gene therapy and small molecule programs Introduction Opus Genetics announced its financial results for the second quarter ended June 30, 2025, and provided a corporate update, highlighting significant clinical and regulatory milestones across its gene therapy and small molecule programs - Positive 12-month Phase 1/2 clinical data in adult cohort and early pediatric clinical data support potential for meaningful vision restoration with OPGx-LCA5[2] - FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for OPGx-LCA5, providing a pathway for accelerated development and review[2] - Positive topline results reported from VEGA-3 and LYNX-2 Phase 3 trials with Phentolamine Ophthalmic Solution 0.75%[2] - OPGx-BEST1 is on track to enter Phase 1/2 trial in H2 2025 for the treatment of bestrophin-1 related inherited retinal disease[2] CEO Commentary CEO George Magrath highlighted significant progress across the pipeline, including the RMAT designation for OPGx-LCA5, sustained functional vision improvements in adult patients, and early efficacy signs in pediatric cohorts. He also noted the advancement of OPGx-BEST1 and the positive Phase 3 readouts for Phentolamine, emphasizing the company's focus on delivering transformative treatments - RMAT designation for OPGx-LCA5 program underscores the strength of clinical data and the urgent need for effective gene therapies[3] - Sustained functional vision improvements observed in adult patients and early signs of efficacy in the pediatric cohort for OPGx-LCA5[3] - Advancement of OPGx-BEST1 toward the clinic and nomination of two additional development candidates (RDH12, MERTK) highlight the breadth of the IRD pipeline[3] - Positive readouts from two Phase 3 Phentolamine trials represent a major step toward bringing a new treatment option to patients with vision challenges[3] Pipeline Updates Opus Genetics provided updates on its gene therapy programs, including RMAT designation for OPGx-LCA5, preclinical progress for OPGx-BEST1, and non-dilutive funding for OPGx-RDH12 and OPGx-MERTK, alongside positive Phase 3 results for Phentolamine Ophthalmic Solution OPGx-LCA5 Program The OPGx-LCA5 program received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, signifying its potential for accelerated development. Clinical data from the Phase 1/2 trial showed sustained visual function improvements in adult participants over 12 months, and initial one-month pediatric data indicated vision improvement with no drug-related adverse events - FDA granted RMAT designation for OPGx-LCA5 for the treatment of Leber Congenital Amaurosis (LCA) due to genetic variations in the LCA5 gene[6] - Twelve-month clinical data from adult participants in the Phase 1/2 trial demonstrated sustained improvements in visual function, including visual acuity gains and improved mobility testing scores[6] - Initial pediatric data at one-month post-treatment showed vision improvement with no drug-related adverse events[6] OPGx-BEST1 Program Preclinical data for OPGx-BEST1 demonstrated restoration of the retinal pigment epithelium-photoreceptor interface in a canine model. The program is on track for Investigational New Drug (IND) submission and initiation of a Phase 1/2 trial in the second half of 2025 - Preclinical data presented at the American Ophthalmological Society (AOS) demonstrated restoration of the retinal pigment epithelium-photoreceptor interface in a canine model of BEST1 using AAV-mediated gene delivery[6] - Investigational New Drug (IND) submission and Phase 1/2 trial initiation remain on track for the second half of 2025[6] OPGx-RDH12 and OPGx-MERTK Programs Opus Genetics secured non-dilutive funding to advance its OPGx-RDH12 and OPGx-MERTK programs. The OPGx-RDH12 program received up to $1.6 million from the Global RDH12 Alliance, while OPGx-MERTK received up to $2 million from the Retinal Degeneration Fund. Preclinical data for OPGx-MERTK showed preservation of retinal function in animal models - Partnership with the Global RDH12 Alliance provides up to $1.6 million in non-dilutive funding to accelerate development of OPGx-RDH12 for Leber congenital amaurosis (RDH12-LCA)[6] - Non-dilutive funding up to $2 million received from the Retinal Degeneration Fund to advance OPGx-MERTK, targeting retinitis pigmentosa caused by MERTK mutations[6] - Preclinical OPGx-MERTK data presented at the American Society of Gene & Cell Therapy (ASGCT) in May showed preservation of retinal function in animal models[6] Phentolamine Ophthalmic Solution 0.75% Program The VEGA-3 Phase 3 trial for presbyopia met its primary and multiple secondary endpoints, demonstrating significant gains in near visual acuity and a favorable safety profile. The LYNX-2 Phase 3 trial also met its endpoints for night vision disturbances in keratorefractive patients, showing improved mesopic low contrast vision and reduced night-driving symptoms. An sNDA submission for the presbyopia indication is planned for the second half of 2025 - VEGA-3 Phase 3 trial met its primary and multiple secondary endpoints in presbyopia, with 27.2% of treated patients achieving a ≥15-letter gain in near visual acuity (vs. 11.5% on placebo, p<0.0001)[12] - LYNX-2 Phase 3 trial met its primary and multiple secondary endpoints in keratorefractive patients with night vision disturbances, showing statistically significant gains in mesopic low contrast vision and improvements in night-driving related symptoms[12] - Supplemental New Drug Application (sNDA) submission for presbyopia indication planned for the second half of 2025[12] Additional Medical Meeting Presentations Opus Genetics presented on the use of virtual reality-guided functional testing to support meaningful clinical endpoints in inherited retinal disease (IRD) trials at the Retinal Imaging Biomarkers Summit - Virtual reality-guided functional testing to support meaningful clinical endpoints in IRD trials was presented at the Retinal Imaging Biomarkers Summit[8] Upcoming Milestones & Data Readouts Opus Genetics anticipates several key milestones in the second half of 2025, including reporting three-month pediatric data for OPGx-LCA5, initiating enrollment for the OPGx-BEST1 Phase 1/2 trial, submitting the Phentolamine sNDA for presbyopia, and initiating enrollment for the Phentolamine LYNX-3 Phase 3 trial - Report three-month pediatric data from OPGx-LCA5 Phase 1/2 trial in Q3 2025[12] - Initiate enrollment in Phase 1/2 trial for OPGx-BEST1 in H2 2025[12] - Submit Phentolamine sNDA for presbyopia in H2 2025[12] - Initiate enrollment in Phentolamine LYNX-3 Phase 3 trial in H2 2025, targeting reduced low light vision and nighttime visual disturbances[12] Financial Results for the Second Quarter Ended June 30, 2025 Opus Genetics reported its financial performance for Q2 2025, detailing cash position, revenue growth, operating expenses, and a reduced net loss, reflecting strategic investments and collaboration reimbursements Cash Position As of June 30, 2025, Opus Genetics held $32.4 million in cash and cash equivalents, which is projected to fund its operations into the second half of 2026 Cash and Cash Equivalents | Metric | June 30, 2025 (in thousands) | | :----- | :--------------------------- | | Cash and cash equivalents | $32,429 | - Based on current operating plans, the Company expects its existing cash resources will fund operations into the second half of 2026[9] Revenue License and collaboration revenue significantly increased to $2.9 million for Q2 2025, up from $1.1 million in the prior-year period. This growth was primarily driven by reimbursement for R&D services under the Company's collaboration with Viatris, Inc License and Collaboration Revenue (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY) | | :----- | :--------------------- | :--------------------- | :----------- | | Revenue | $2,882 | $1,112 | +159.2% | - Revenue in both periods was driven by the Company's collaboration with Viatris, Inc., primarily from reimbursement of R&D services[10] Operating Expenses Operating expenses for Q2 2025 saw an increase in G&A due to legal and business development costs, while R&D expenses remained stable, largely offset by reimbursements General and Administrative (G&A) Expenses G&A expenses increased to $5.8 million in Q2 2025 from $3.4 million in Q2 2024, primarily due to higher costs associated with legal and patent-related expenses, payroll, and business development activities. Stock-based compensation also saw a slight increase General and Administrative Expenses (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY) | | :----- | :--------------------- | :--------------------- | :----------- | | G&A Expenses | $5,766 | $3,354 | +71.9% | | Stock-based compensation | $600 | $500 | +20.0% | - The increase was mainly due to higher costs associated with legal and patent-related expenses, payroll, and business development activities[11] Research and Development (R&D) Expenses R&D expenses slightly decreased to $6.0 million for Q2 2025, compared to $6.1 million in the prior-year period. This minor reduction was mainly attributable to lower manufacturing and consulting costs, partially offset by increased clinical trial, toxicology, and payroll-related expenses. R&D expenses for Phentolamine Ophthalmic Solution 0.75% were fully reimbursed Research and Development Expenses (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY) | | :----- | :--------------------- | :--------------------- | :----------- | | R&D Expenses | $6,022 | $6,086 | -1.0% | | Stock-based compensation | $300 | $300 | 0.0% | - The slight decrease was primarily due to lower manufacturing and consulting costs, partially offset by increased clinical trial, toxicology, and payroll-related expenses[13] - R&D expenses related to Phentolamine Ophthalamine Solution 0.75% were fully reimbursed under the Viatris License Agreement[13] Net Loss and Earnings Per Share (EPS) Opus Genetics reported a net loss of $7.4 million, or $(0.12) per basic and diluted share, for Q2 2025, an improvement from a net loss of $7.8 million, or $(0.30) per share, in Q2 2024 Net Loss and EPS (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY) | | :----- | :--------------------- | :--------------------- | :----------- | | Net Loss | $(7,420) | $(7,765) | -4.4% | | Basic and Diluted EPS | $(0.12) | $(0.30) | +60.0% | - The number of shares used in per share calculations significantly increased from 25,827,265 in Q2 2024 to 63,376,392 in Q2 2025[21] Condensed Consolidated Financial Statements The condensed consolidated financial statements for Q2 2025 show an increase in total assets and stockholders' equity, alongside a significant rise in liabilities primarily due to warrant liabilities and a long-term funding agreement Condensed Consolidated Balance Sheets As of June 30, 2025, Opus Genetics reported total assets of $38.7 million, an increase from $36.9 million at December 31, 2024. Total liabilities significantly increased to $21.2 million from $11.3 million, primarily due to the introduction of warrant liabilities and a long-term funding agreement. Total stockholders' equity also increased to $17.5 million from $6.7 million Condensed Consolidated Balance Sheets (as of June 30, 2025 vs. December 31, 2024) | Metric (in thousands) | June 30, 2025 | December 31, 2024 | Change | | :-------------------- | :------------ | :---------------- | :----- | | Cash and cash equivalents | $32,429 | $30,321 | +$2,108 | | Total current assets | $38,439 | $36,610 | +$1,829 | | Total assets | $38,665 | $36,862 | +$1,803 | | Warrant liabilities | $11,800 | $— | +$11,800 | | Total current liabilities | $20,192 | $11,295 | +$8,897 | | Total liabilities | $21,192 | $11,295 | +$9,897 | | Total stockholders' equity | $17,473 | $6,724 | +$10,749 | - The significant increase in total liabilities was driven by the introduction of warrant liabilities ($11.8 million) and a new long-term funding agreement ($1.0 million) with a related party[19] Condensed Consolidated Statements of Comprehensive Loss For the three months ended June 30, 2025, Opus Genetics reported a comprehensive loss of $7.4 million, an improvement from $7.8 million in the prior-year period. This was influenced by increased license and collaboration revenue and a fair value change in warrant liabilities, despite higher operating expenses Condensed Consolidated Statements of Comprehensive Loss (Q2 2025 vs. Q2 2024) | Metric (in thousands) | Q2 2025 | Q2 2024 | Change | | :-------------------- | :------ | :------ | :----- | | License and collaborations revenue | $2,882 | $1,112 | +$1,770 | | Total operating expenses | $11,788 | $9,440 | +$2,348 | | Loss from operations | $(8,906) | $(8,328) | -$(578) | | Fair value change in warrant and other derivative liabilities | $917 | $— | +$917 | | Net loss | $(7,420) | $(7,765) | +$345 | | Comprehensive loss | $(7,420) | $(7,765) | +$345 | - For the six months ended June 30, 2025, the net loss was $(15,614) thousand, compared to $(14,871) thousand for the same period in 2024[21] About Opus Genetics Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders. Its pipeline includes AAV-based gene therapies like OPGx-LCA5 and OPGx-BEST1, and the partnered Phentolamine Ophthalmic Solution 0.75% for presbyopia and night vision disturbances - Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders[15] - The Company's pipeline features AAV-based gene therapies targeting inherited retinal diseases including Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa[15] - Lead gene therapy candidates are OPGx-LCA5 (in Phase 1/2 trial for LCA5-related mutations) and OPGx-BEST1 (targeting BEST1-related retinal degeneration)[15] - Opus is also advancing Phentolamine Ophthalmic Solution 0.75%, a partnered therapy being studied in two Phase 3 programs for presbyopia and reduced low light vision and nighttime visual disturbances[15] Forward-Looking Statements & Contacts This section provides a standard disclaimer regarding forward-looking statements, cautioning readers about inherent risks and uncertainties that could cause actual results to differ materially from projections. It also includes contact information for investor relations and media inquiries - This press release contains forward-looking statements subject to certain risks and uncertainties that could cause actual business, prospects, and results of operations to differ materially[16] - Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of the press release[16] - Contact information for investors (Jenny Kobin, Remy Bernarda) and media (Kimberly Ha) is provided[17]