Opus Genetics, Inc.(IRD) - 2025 Q2 - Quarterly Results

Corporate Update and Pipeline Highlights The company received key regulatory designations, reported positive clinical trial results, and secured non-dilutive funding - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for OPGx-LCA5, a gene therapy for Leber Congenital Amaurosis (LCA), signaling potential for accelerated development[2] - Positive 12-month Phase 1/2 clinical data for OPGx-LCA5 in adults and encouraging early pediatric data support its potential for vision restoration[2] - Reported positive topline results from two Phase 3 trials (VEGA-3 and LYNX-2) for Phentolamine Ophthalmic Solution 0.75% in presbyopia and night vision disturbances[2] - The OPGx-BEST1 program for inherited retinal disease is on track to enter a Phase 1/2 trial in the second half of 2025[2] - Secured non-dilutive funding from patient advocacy groups to advance multiple early-stage gene therapy programs, including OPGx-RDH12 and OPGx-MERTK[2][6] Pipeline Updates This section details clinical and preclinical progress across the company's key therapeutic programs OPGx-LCA5 – Gene Therapy for Leber Congenital Amaurosis (LCA) The OPGx-LCA5 program received RMAT designation and demonstrated sustained vision improvements in clinical trials - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation, providing a pathway for accelerated development and review[6] - Twelve-month clinical data from the adult cohort in the Phase 1/2 trial showed sustained improvements in visual function, including gains in visual acuity and better mobility testing scores[6] - Initial one-month post-treatment data in the pediatric cohort indicated vision improvement with no drug-related adverse events[6] OPGx-BEST1 – Gene Therapy for BEST1-Related IRD The OPGx-BEST1 program is advancing toward a Phase 1/2 trial supported by positive preclinical data - Preclinical data presented in May showed restoration of the retinal pigment epithelium-photoreceptor interface in a canine model using AAV-mediated gene delivery[6] - The company remains on track to submit an IND application and initiate a Phase 1/2 trial in the second half of 2025[6] OPGx-RDH12 and OPGx-MERTK – Advancing with Non-Dilutive Support Early-stage gene therapies OPGx-RDH12 and OPGx-MERTK are advancing with non-dilutive funding from patient groups - A partnership with the Global RDH12 Alliance provides up to $1.6 million in non-dilutive funding to advance OPGx-RDH12 for RDH12-LCA[6] - Received up to $2 million in non-dilutive funding from the Retinal Degeneration Fund to advance OPGx-MERTK for retinitis pigmentosa caused by MERTK mutations[6] Phentolamine Ophthalmic Solution 0.75% – Advancing Toward sNDA Submissions Positive Phase 3 trial results for Phentolamine Ophthalmic Solution support planned sNDA submissions - The VEGA-3 Phase 3 trial for presbyopia met its primary endpoint, with 27.2% of treated patients achieving a ≥15-letter gain in near visual acuity compared to 11.5% on placebo (p<0.0001)[12] - The LYNX-2 Phase 3 trial for keratorefractive patients with night vision disturbances also met its primary and secondary endpoints, showing significant gains in mesopic low contrast vision[12] Outlook and Upcoming Milestones The company outlines key clinical and regulatory milestones expected in the second half of 2025 - Key upcoming milestones for the second half of 2025 include: - Reporting three-month pediatric data from the OPGx-LCA5 Phase 1/2 trial (Q3 2025) - Initiating enrollment in the Phase 1/2 trial for OPGx-BEST1 (H2 2025) - Submitting the Phentolamine sNDA for presbyopia (H2 2025) - Initiating enrollment in the Phentolamine LYNX-3 Phase 3 trial (H2 2025)[12] Second Quarter 2025 Financial Results The company reports Q2 2025 financial performance, including revenue growth and an updated cash runway Financial Performance Summary The company reported a net loss of $7.4 million on revenue of $2.9 million, with a cash runway into H2 2026 - As of June 30, 2025, the company had cash and cash equivalents of $32.4 million, which is expected to fund operations into the second half of 2026[9] Q2 2025 Key Financial Metrics (vs. Q2 2024) | Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | License & Collaboration Revenue | $2.9 million | $1.1 million | +163.6% | | G&A Expenses | $5.8 million | $3.4 million | +70.6% | | R&D Expenses | $6.0 million | $6.1 million | -1.6% | | Net Loss | $7.4 million | $7.8 million | Improved | | Net Loss per Share | $(0.12) | $(0.30) | Improved | Condensed Consolidated Balance Sheets Total assets grew to $38.7 million, while liabilities increased to $21.2 million as of June 30, 2025 Balance Sheet Summary (in thousands) | Balance Sheet Item | June 30, 2025 | Dec 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $32,429 | $30,321 | | Total assets | $38,665 | $36,862 | | Total liabilities | $21,192 | $11,295 | | Total stockholders' equity | $17,473 | $6,724 | Condensed Consolidated Statements of Comprehensive Loss The company's net loss improved to $7.4 million for Q2 2025, compared to $7.8 million in the prior-year period Statement of Comprehensive Loss (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | License and collaborations revenue | $2,882 | $1,112 | | Total operating expenses | $11,788 | $9,440 | | Loss from operations | $(8,906) | $(8,328) | | Net loss | $(7,420) | $(7,765) | | Net loss per share (basic and diluted) | $(0.12) | $(0.30) | | Weighted avg. shares outstanding | 63,376,392 | 25,827,265 | Company Overview Opus Genetics is a clinical-stage biopharmaceutical company focused on gene therapies for inherited retinal diseases - Opus Genetics is a clinical-stage biopharmaceutical company specializing in gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other eye disorders[15] - The company's lead gene therapy candidates are OPGx-LCA5 for Leber congenital amaurosis (LCA) and OPGx-BEST1 for BEST1-related retinal degeneration[15] - Opus is also advancing a partnered therapy, Phentolamine Ophthalmic Solution 0.75%, which is being studied in Phase 3 programs for presbyopia and nighttime visual disturbances[15]