Ocuphire Pharma(US:OCUP)2025-05-15 12:27markdown [First Quarter 2025 Corporate Update](index=1&type=section&id=1.%20First%20Quarter%202025%20Corporate%20Update) Opus Genetics announced its Q1 2025 financial results and provided a corporate update, highlighting positive 12-month data from the OPGx-LCA5 Phase 1/2 study, ongoing pediatric enrollment, and the anticipated clinical entry of OPGx-BEST1 [Overview and Key Announcements](index=1&type=section&id=1.1.%20Overview%20and%20Key%20Announcements) Opus Genetics announced its Q1 2025 financial results and provided a corporate update, highlighting positive 12-month data from the OPGx-LCA5 Phase 1/2 study, ongoing pediatric enrollment, and the anticipated clinical entry of OPGx-BEST1 - Opus Genetics announced **Q1 2025 financial results and corporate update**[1](index=1&type=chunk)[2](index=2&type=chunk) - ARVO presentation highlighted **12-month results from Phase 1/2 study** supporting **OPGx-LCA5 gene therapy** potential to restore meaningful vision[1](index=1&type=chunk) - **Pediatric cohort of LCA5 study** ongoing with initial multi-patient data anticipated in **Q3 2025**[1](index=1&type=chunk) - **OPGx-BEST1** is on track for **IND filing** and initiation of **Phase 1/2 trial**, with early data expected in **Q1 2026**[1](index=1&type=chunk) [CEO Commentary](index=1&type=section&id=1.2.%20CEO%20Commentary) CEO George Magrath emphasized a strong start to 2025 with significant progress across both the inherited retinal disease (IRD) gene therapy platform and the phentolamine eye drop franchise, noting durable efficacy for OPGx-LCA5 and a strengthened cash position from recent financing - **CEO George Magrath** noted a **strong start in 2025** with progress in **IRD platform** and **phentolamine eye drop franchise**[3](index=3&type=chunk) - **Positive 12-month data** from **OPGx-LCA5 Phase 1/2 trial** showed **durable efficacy** in adult patients[3](index=3&type=chunk) - Initial success with **OPGx-LCA5** has the potential to translate to the rest of the pipeline, including **six additional IRD gene therapy candidates**[4](index=4&type=chunk) - Company ended Q1 with a **strong cash position** after completing a successful financing with leading institutional healthcare investors[4](index=4&type=chunk) [Strategic Highlights and Pipeline Progress](index=2&type=section&id=2.%20Strategic%20Highlights%20and%20Pipeline%20Progress) Opus Genetics is advancing its gene therapy and ophthalmic solution pipelines, with significant clinical progress, regulatory designations, and upcoming data readouts across multiple programs [Gene Therapy Programs](index=2&type=section&id=2.1.%20Gene%20Therapy%20Programs) Opus Genetics is advancing its AAV-based gene therapy pipeline, with OPGx-LCA5 showing promising 12-month clinical data in adults, initiating a pediatric cohort, and receiving RMAT designation. OPGx-BEST1 is on track for IND filing and Phase 1/2 trial initiation by Q4 2025 - Opus's pipeline includes a portfolio of **seven adeno-associated virus (AAV)-based gene therapy assets**, each targeting a specific IRD[7](index=7&type=chunk) - Emerging clinical data on **OPGx-LCA5** provide evidence of **clinical proof of concept** and support its potential to restore meaningful vision[7](index=7&type=chunk) - **OPGx-BEST1** is on track to enter the clinic by **Q4 2025**, with preliminary data expected in **Q1 2026**[7](index=7&type=chunk) - **OPGx-LCA5** received **Regenerative Medicine Advanced Therapy (RMAT) designation** from the FDA, offering potential for expedited development and review[7](index=7&type=chunk) [OPGx-LCA5 Program](index=2&type=section&id=2.1.1.%20OPGx-LCA5%20Program) The OPGx-LCA5 program demonstrated sustained efficacy in adult patients, initiated pediatric enrollment with encouraging early safety, and discussed regulatory pathways with the FDA - **One-year open-label data** on adult patients in the **Phase 1/2 clinical trial of OPGx-LCA5** showed **sustained improvements** in subjective and objective measures of efficacy[7](index=7&type=chunk) - 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](index=7&type=chunk) - The pediatric cohort is expected to complete enrollment in **Q2 2025**, with initial data from all three patients anticipated in **Q3 2025**[7](index=7&type=chunk) - 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](index=8&type=chunk) [OPGx-BEST1 Program](index=3&type=section&id=2.1.2.%20OPGx-BEST1%20Program) The OPGx-BEST1 program is advancing towards an IND filing and Phase 1/2 trial initiation by Q4 2025, supported by positive IND-enabling study data - **OPGx-BEST1** is an investigational **Phase 1/2-ready asset** in development for IRDs associated with mutations in the **BEST1 gene**[11](index=11&type=chunk) - **IND-enabling studies of OPGx-BEST1** provided **safety and efficacy data** in support of a first-in-human clinical trial[11](index=11&type=chunk) - Opus plans to file an **IND** and begin a **Phase 1/2 trial by Q4 2025**, with preliminary data expected in **Q1 2026**[11](index=11&type=chunk) [Phentolamine Ophthalamine Solution 0.75% Program](index=3&type=section&id=2.2.%20Phentolamine%20Ophthalmic%20Solution%200.75%25%20Program) The Phentolamine Ophthalmic Solution 0.75% program is progressing with two pivotal Phase 3 trials, LYNX-2 (for dim light vision disturbances) and VEGA-3 (for presbyopia), both having completed enrollment in Q1 2025 with topline data expected mid-year 2025 and H1 2025, respectively. The program has FDA Fast Track designation for LYNX-2 and is funded by Viatris Inc - The **LYNX-2 pivotal Phase 3 trial** for visual loss in low light conditions completed enrollment in **Q1 2025**, with topline data expected **mid-year 2025**[11](index=11&type=chunk) - The **LYNX-2 trial** is covered by a **Special Protocol Assessment (SPA) agreement** with the FDA[11](index=11&type=chunk) - The **FDA** granted **Fast Track designation** for **Phentolamine Ophthalmic Solution 0.75%** for treatment of significant chronic night driving impairment[11](index=11&type=chunk) - 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](index=11&type=chunk) - The development portfolio related to **Phentolamine Ophthalmic Solution 0.75%** is being funded by the Company's partner, **Viatris Inc.**[11](index=11&type=chunk) [Expected Milestones and Growth Drivers](index=3&type=section&id=2.3.%20Expected%20Milestones%20and%20Growth%20Drivers) Key upcoming milestones include initial data from pediatric OPGx-LCA5 patients in Q3 2025, IND filing and Phase 1/2 initiation for OPGx-BEST1 in 2025 with Q1 2026 data, and topline data from both LYNX-2 and VEGA-3 Phase 3 trials for Phentolamine Ophthalmic Solution 0.75% in mid-2025 and H1 2025, respectively - **Initial data** from **three pediatric patients** treated with **OPGx-LCA5** anticipated in **Q3 2025**[11](index=11&type=chunk) - **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](index=11&type=chunk) - **Topline data** from the **LYNX-2 pivotal Phase 3 trial** evaluating **Phentolamine Ophthalmic Solution 0.75%** for visual loss in low light conditions associated with keratorefractive surgery are expected **mid-year 2025**[11](index=11&type=chunk) - **Topline data** from the **VEGA-3 pivotal Phase 3 clinical trial** evaluating **Phentolamine Ophthalmic Solution 0.75%** for the treatment of presbyopia are expected in the **first half of 2025**[11](index=11&type=chunk) [Financing and Funding](index=2&type=section&id=2.4.%20Financing%20and%20Funding) In March 2025, Opus completed a successful public offering and private placement, raising approximately $21.5 million in gross proceeds, with potential for an additional $21.4 million from warrant exercises tied to the BEST1 program, significantly strengthening the company's cash position - In **March 2025**, Opus completed a **successful underwritten public offering** and concurrent **private placement** anchored by leading healthcare investors[7](index=7&type=chunk) - The transaction raised approximately **$21.5 million in gross proceeds**[7](index=7&type=chunk) - Potential for up to **$21.4 million in additional proceeds** upon exercise of warrants that are tied to data release from the **BEST1 program**[7](index=7&type=chunk) [Financial Results for Q1 2025](index=4&type=section&id=3.%20Financial%20Results%20for%20Q1%202025) Opus Genetics reported its Q1 2025 financial performance, highlighting increased cash and cash equivalents, significant growth in license revenue, and higher operating expenses leading to an increased net loss [Financial Highlights](index=4&type=section&id=3.1.%20Financial%20Highlights) Opus Genetics reported $41.8 million in cash and cash equivalents as of March 31, 2025, providing a funding runway into Q2 2026. License and collaborations revenue increased significantly by 158.8% YoY, while both G&A and R&D expenses rose by 34.0% and 69.6% respectively, leading to an increased net loss of $8.2 million for the quarter Cash and Cash Equivalents | Metric | March 31, 2025 | | :---------------------- | :------------- | | Cash and cash equivalents | $41.8 million | | Funding runway | Into Q2 2026 | License and Collaborations Revenue | Metric | Q1 2025 | Q1 2024 | YoY Change | | :------ | :------------- | :------------- | :--------- | | Revenue | $4.4 million | $1.7 million | +158.8% | Operating Expenses (in millions) | Metric | Q1 2025 | Q1 2024 | YoY Change | | :----------- | :------ | :------ | :--------- | | G&A Expenses | $6.3 | $4.7 | +34.0% | | R&D Expenses | $8.0 | $4.7 | +69.6% | Net Loss and EPS | Metric | Q1 2025 | Q1 2024 | | :---------------- | :------------- | :------------- | | Net Loss | $(8.2) million | $(7.1) million | | Basic & Diluted EPS | $(0.24) | $(0.29) | [Condensed Consolidated Balance Sheets](index=9&type=section&id=3.2.%20Condensed%20Consolidated%20Balance%20Sheets) As of March 31, 2025, total assets increased to $48.167 million from $36.862 million at December 31, 2024, primarily driven by higher cash and cash equivalents. Total current liabilities significantly increased to $24.251 million due to the introduction of $12.715 million in warrant liabilities Condensed Consolidated Balance Sheets (in thousands) | | 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](index=10&type=section&id=3.3.%20Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) For Q1 2025, Opus Genetics reported a net loss of $8.194 million, an increase from $7.106 million in Q1 2024. This was primarily due to higher operating expenses (G&A and R&D) and new financing costs, partially offset by a fair value change in warrant liabilities and increased license revenue 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 | | Total operating expenses | $14,299 | $9,419 | | Loss from operations | $(9,929) | $(7,708) | | Financing costs | $(1,372) | $— | | Fair value change in warrant liabilities | $2,805 | $— | | Net loss | $(8,194) | $(7,106) | | Basic and diluted EPS | $(0.24) | $(0.29) | [Additional Information](index=5&type=section&id=4.%20Additional%20Information) This section provides an overview of Opus Genetics' mission and pipeline, outlines forward-looking statements and associated risk factors, and lists key contact information [About Opus Genetics](index=5&type=section&id=4.1.%20About%20Opus%20Genetics) Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company focused on developing AAV-based gene therapies for inherited retinal diseases (IRDs) such as LCA, bestrophinopathy, and retinitis pigmentosa, alongside Phentolamine Ophthalmic Solution 0.75% for presbyopia and dim light vision disturbances, currently in Phase 3 trials - **Opus Genetics** is a **clinical-stage ophthalmic biopharmaceutical company** developing therapies to treat patients with IRDs and other ophthalmic disorders[17](index=17&type=chunk) - Pipeline includes **AAV-based investigational gene therapies** addressing gene mutations for different forms of **Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa**[17](index=17&type=chunk) - The pipeline also includes **Phentolamine Ophthalmic Solution 0.75%**, being investigated in **Phase 3 trials** for **presbyopia** and **mesopic (dim) light vision disturbances**[17](index=17&type=chunk) [Forward-Looking Statements and Risk Factors](index=5&type=section&id=4.2.%20Forward-Looking%20Statements%20and%20Risk%20Factors) The report contains forward-looking statements regarding future operations and financial performance, which are subject to various risks and uncertainties. Key risks include the preliminary nature of clinical data, integration challenges from the Opus Acquisition, difficulties in gene therapy development, potential clinical trial delays, regulatory changes, dependence on pipeline success and third parties, intellectual property issues, and the need for additional capital - This press release contains **forward-looking statements** regarding cash runway, potential growth, and clinical trial data, subject to certain risks and uncertainties[19](index=19&type=chunk)[20](index=20&type=chunk) - **Key risks** include **preliminary clinical data**, failure to successfully integrate businesses after the **Opus Acquisition**, difficulties in developing and manufacturing **novel gene therapies**, and potential **clinical trial delays or failures**[21](index=21&type=chunk) - Other risks involve **changes in regulatory requirements**, heavy dependence on **product pipeline**, **intellectual property protection**, and the future need for **substantial additional capital**[21](index=21&type=chunk)[22](index=22&type=chunk)[25](index=25&type=chunk) - Readers are cautioned not to place undue reliance on these **forward-looking statements** and to review detailed **risk factors in SEC filings**[20](index=20&type=chunk)[23](index=23&type=chunk) [Contacts](index=8&type=section&id=4.3.%20Contacts) Contact information for corporate inquiries (Nirav Jhaveri, CFO) and investor relations (Corey Davis, Ph.D. at LifeSci Advisors) is provided Contacts | Role | Name | Email | | :--------------- | :-------------------- | :---------------------- | | Corporate (CFO) | Nirav Jhaveri | ir@ocuphire.com | | Investor Relations | Corey Davis, Ph.D. | cdavis@lifesciadvisors.com |