Opus Genetics Announces Financial Results for Third Quarter 2025 and Provides Corporate Update
Rhea-AI Summary
Opus Genetics (Nasdaq: IRD) reported Q3 2025 results and a corporate update on Nov 12, 2025. Key clinical highlights include positive 3-month pediatric and 18-month adult data from the OPGx-LCA5 Phase 1/2 trial and a successful FDA RMAT meeting that could enable an accelerated pathway. The company opened enrollment for OPGx-BEST1 and plans dosing for a Phase 3 LCA5 portion in H2 2026 with topline ~one year later.
Financially, cash was $30.8M at Sept 30, 2025 plus ~$23.0M raised post-quarter (cash >$50M) expected to fund operations into H2 2027; Q3 revenue was $3.1M and net loss was $17.5M (Q3 2025).
Positive
- OPGx-LCA5: positive pediatric 3-month and adult 18-month data
- FDA RMAT: Type B meeting outcome enables potential accelerated approval
- Cash position >$50.0M after $23.0M registered direct raise
- sNDA for Phentolamine Ophthalmic Solution planned by year-end 2025
Negative
- Net loss widened to $17.5M in Q3 2025 from $7.5M prior-year
- Q3 2025 revenue declined to $3.1M from $3.9M in Q3 2024
- G&A expenses rose to $5.0M in Q3 2025 from $2.9M in Q3 2024
- Equity raise of $23.0M may imply shareholder dilution
News Market Reaction
On the day this news was published, IRD declined 2.54%, reflecting a moderate negative market reaction. This price movement removed approximately $3M from the company's valuation, bringing the market cap to $111M at that time.
Data tracked by StockTitan Argus on the day of publication.
- Positive 3-month pediatric and 18-month adult clinical data from OPGx-LCA5 Phase 1/2 trial support the potential for restoring cone-mediated vision -
- Successful FDA RMAT meeting provides the potential for an accelerated regulatory pathway to approval for OPGx-LCA5 -
- OPGx-BEST1 gene therapy program underway with recruitment ongoing in Phase 1/2 trial for the treatment of BEST1 disease -
- Supplemental New Drug Application submission planned by year-end 2025 for Phentolamine Ophthalmic Solution
- Strengthened capital position from recent equity offering and non-dilutive funding from patient advocacy groups -
RESEARCH TRIANGLE PARK, N.C., Nov. 12, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced financial results for the third quarter ended September 30, 2025, and provided a corporate update.
“We have taken critical steps in advancing our pipeline, including the positive data and successful outcome of our recent FDA meeting regarding our LCA5 program and the opening of recruitment in our BEST1 Phase 1/2 clinical trial,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “We look forward to continuing this momentum into next year, when we expect multiple data readouts across several programs. The financing we recently completed reinforces the strong support from existing shareholders and new prominent healthcare investors and provides us with financial resources to expedite the path toward multiple approvals.”
Pipeline Updates
OPGx-LCA5 – Gene Therapy for Leber Congenital Amaurosis (LCA)
- In September 2025, positive data was reported from the six participants treated to date in an open-label, Phase 1/2 clinical trial. Large gains in cone-mediated vision with improvements across multiple measures of visual function were observed in the three pediatric participants treated over three months. In the three adult participants, responses have been observed out to 18 months, underscoring the potential durability of the treatment response. OPGx-LCA5 has been well tolerated with no ocular serious adverse events or dose-limiting toxicities to date.
- Successful completion of a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the U.S. Food and Drug Administration (FDA) provides the potential for an accelerated regulatory pathway to approval of OPGx-LCA5.
- The Company plans to advance its ongoing trial into a Phase 3 portion which is expected to enroll as few as 8 participants in a single arm, 12-month study utilizing an adaptive design, which provides flexibility on endpoints and number of participants, reflective of LCA5 as a rare condition with an urgent medical need.
- The first participant was enrolled in the planned run-in period of the Phase 3 portion of the study to evaluate the natural history of each participant to serve as their own control.
- Dosing with OPGx-LCA5 is anticipated in the second half of 2026 following availability of validated clinical drug supply manufactured with the intended commercial processes, with topline clinical data expected approximately one year later.
- LCA5 program featured on Good Morning America in honor of World Blindness Awareness Month.
OPGx-BEST1 – Gene Therapy for BEST1-Related IRD
- In August 2025, the FDA accepted the Company’s Investigational New Drug (IND) application for OPGx-BEST1 to initiate a clinical trial.
- The Company is currently recruiting participants in an adaptive, open-label, dose-exploring Phase 1/2 trial known as BIRD1 to evaluate the safety and tolerability of subretinally injected OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB).
- Initial data is expected in the first quarter of 2026.
OPGx-RDH12 and OPGx-MERTK – Advancing with Non-Dilutive Support
- 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). - Non-dilutive funding of up to
$2 million received from the Retinal Degeneration Fund to advance OPGx-MERTK, targeting retinitis pigmentosa caused by pathogenic variants in the Mer proto-oncogene tyrosine kinase (MERTK) gene.
Phentolamine Ophthalmic Solution
- Based on positive clinical data from the VEGA-3 Phase 3 trial to treat presbyopia, Opus Genetics plans to submit an sNDA to the FDA by year-end 2025.
- Full recruitment has been completed in LYNX-3, the second pivotal Phase 3 trial in keratorefractive participants with visual disturbances under mesopic, low-contrast conditions, with topline results expected in the first half of 2026.
- The program is being conducted under a Special Protocol Assessment (SPA) and has received Fast Track Designation from the FDA.
Medical Publications and Presentations
- Peer-reviewed publication of LCA5 Phase 1/2 trial data in Molecular Therapy: “Recovery of Cone-Mediated Vision in a Severe Ciliopathy after Gene Augmentation: One-Year Results of a Phase I/II Trial for LCA5-LCA,” authored
by Tomas S. Aleman, M.D., et al. - Presentation at Eyecelerator at the American Academy of Ophthalmology (AAO) Annual Meeting titled “Transformative Gene Therapies for the Treatment of Rare Inherited Retinal Diseases.”
- Presentation at the Cell and Gene Meeting on the Mesa titled: “Transformative Gene Therapies for the Treatment of Rare Inherited Retinal Diseases.”
- Poster presentation at the American Academy of Optometry Annual Meeting titled: “LYNX-2: A Pivotal Phase 3 Trial of Phentolamine Ophthalmic Solution in Post-Keratorefractive Surgery Subjects with Decreased Mesopic Visual Acuity.”
Financial Results for the Third Quarter Ended September 30, 2025
Cash Position: As of September 30, 2025, Opus Genetics had cash and cash equivalents of
Revenue: License and collaborations revenue totaled
General and Administrative (G&A) Expenses: G&A expenses were
Research and Development (R&D) Expenses: R&D expenses were
Net Loss: Net loss for the third quarter of 2025 was
About Opus Genetics
Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs). The Company is developing durable, one-time treatments designed to address the underlying genetic causes of severe retinal disorders. The Company’s pipeline includes seven AAV-based programs, led by OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration, with additional candidates targeting RHO, RDH12, and MERTK. Opus Genetics is also advancing Phentolamine Ophthalmic Solution
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements related to cash runway, the clinical development, clinical results, preclinical data, and future plans for Phentolamine Ophthalmic Solution
Contacts:
Investors
Jenny Kobin
Remy Bernarda
IR Advisory Solutions
ir@opusgtx.com
Media
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com
-Financial Tables Follow-
| Opus Genetics, Inc. Condensed Consolidated Balance Sheets (in thousands, except share amounts and par value) | |||||||
| As of | |||||||
| September 30, | December 31, | ||||||
| 2025 | 2024 | ||||||
| Assets | (Unaudited) | ||||||
| Current assets: | |||||||
| Cash and cash equivalents | $ | 30,815 | $ | 30,321 | |||
| Accounts receivable | 2,916 | 3,563 | |||||
| Contract assets and unbilled receivables (Note 11) | 1,364 | 2,209 | |||||
| Prepaids and other current assets | 815 | 515 | |||||
| Short-term investments | — | 2 | |||||
| Total current assets | 35,910 | 36,610 | |||||
| Property and equipment, net | 212 | 252 | |||||
| Total assets | $ | 36,122 | $ | 36,862 | |||
| Liabilities and stockholders’ equity | |||||||
| Current liabilities: | |||||||
| Accounts payable | $ | 2,395 | $ | 3,148 | |||
| Accrued expenses and other liabilities | 5,367 | 8,147 | |||||
| Warrant liabilities | 21,325 | — | |||||
| Total current liabilities | 29,087 | 11,295 | |||||
| Long-term funding agreement, related party | 1,068 | — | |||||
| Total liabilities | 30,155 | 11,295 | |||||
| Commitments and contingencies (Note 4 and Note 10) | |||||||
| Series A preferred stock, par value | — | 18,843 | |||||
| Stockholders’ equity: | |||||||
| Preferred stock, par value | — | — | |||||
| Common stock, par value | 6 | 3 | |||||
| Additional paid-in capital | 178,027 | 145,719 | |||||
| Accumulated deficit | (172,066 | ) | (138,998 | ) | |||
| Total stockholders’ equity | 5,967 | 6,724 | |||||
| Total liabilities, Series A preferred stock and stockholders’ equity | $ | 36,122 | $ | 36,862 | |||
| Opus Genetics, Inc. Condensed Consolidated Statements of Comprehensive Loss (in thousands, except share and per share amounts) (Unaudited) | ||||||||||||||||
| For the Three Months Ended September 30, | For the Nine Months Ended September 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| License and collaborations revenue | $ | 3,079 | $ | 3,867 | $ | 10,331 | $ | 6,690 | ||||||||
| Operating expenses: | ||||||||||||||||
| General and administrative | 4,981 | 2,894 | 17,093 | 10,918 | ||||||||||||
| Research and development | 6,409 | 8,982 | 20,384 | 19,817 | ||||||||||||
| Total operating expenses | 11,390 | 11,876 | 37,477 | 30,735 | ||||||||||||
| Loss from operations | (8,311 | ) | (8,009 | ) | (27,146 | ) | (24,045 | ) | ||||||||
| Fair value change in warrant and other derivative liabilities | (9,525 | ) | — | (5,803 | ) | — | ||||||||||
| Financing costs | — | — | (1,337 | ) | — | |||||||||||
| Interest expense | (68 | ) | — | (68 | ) | — | ||||||||||
| Other income, net | 450 | 483 | 1,286 | 1,648 | ||||||||||||
| Loss before income taxes | (17,454 | ) | (7,526 | ) | (33,068 | ) | (22,397 | ) | ||||||||
| Benefit (provision) for income taxes | — | — | — | — | ||||||||||||
| Net loss | (17,454 | ) | (7,526 | ) | (33,068 | ) | (22,397 | ) | ||||||||
| Other comprehensive loss, net of tax | — | — | — | — | ||||||||||||
| Comprehensive loss | $ | (17,454 | ) | $ | (7,526 | ) | $ | (33,068 | ) | $ | (22,397 | ) | ||||
| Net loss per share: | ||||||||||||||||
| Basic and diluted | $ | (0.25 | ) | $ | (0.29 | ) | $ | (0.59 | ) | $ | (0.88 | ) | ||||
| Number of shares used in per share calculations: | ||||||||||||||||
| Basic and diluted | 70,636,887 | 26,145,080 | 56,100,689 | 25,501,117 | ||||||||||||
Source: Opus Genetics, Inc.
FAQ
What did Opus Genetics (IRD) report for cash and runway on Nov 12, 2025?
What were the Q3 2025 revenue and net loss for Opus Genetics (IRD)?
What does the FDA RMAT outcome mean for Opus Genetics' OPGx-LCA5 (IRD)?
When is Opus Genetics expecting Phase 3 dosing and topline data for OPGx-LCA5 (IRD)?
When will initial data from the OPGx-BEST1 (IRD) trial be available?
What regulatory filing is Opus Genetics planning for Phentolamine Ophthalmic Solution (IRD)?
