- Favorable early safety and initial efficacy data from BEST1 program highlighted at premier gathering of global retinal experts with additional data expected mid-year 2026 -
- Reauthorization of FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program provides opportunity for Opus’ deep pipeline in rare inherited retinal diseases -
- FDA Prescription Drug User Fee Act (PDUFA) date in
- Funding from prominent healthcare investors expected to extend cash runway into 2028 -
“We are delivering a steady cadence of data and milestones across our pipeline,” said
Pipeline Updates
OPGx-BEST1 – Gene Therapy for BEST1-Related IRD
- Recruitment is ongoing at multiple
U.S. sites with two participants treated to date in the Phase 1/2 trial (BIRD-1) that includes patients with both dominant and recessive forms of BEST disease. - Positive initial three-month data from the first (sentinel) participant presented at
Macula Society - The data demonstrated that OPGx-BEST1 was well tolerated with no ocular inflammation, no ocular or treatment-related adverse events, and no dose limiting toxicities observed to date.
- Early signals of functional vision improvement were observed, including an equivalent 12-letter gain in Best Corrected Visual Acuity (BCVA) in the treated study eye.
- Structural improvement in central subfield thickness (CST) was observed with a 23% decrease in the study eye.
- Resolution of intraretinal fluid was also seen as early as 1-month in areas with less atrophy.
- Three-month results from the full Cohort 1 are expected in mid-year 2026.
OPGx-LCA5 – Gene Therapy for Leber Congenital Amaurosis (LCA5)
- Recruitment ongoing with multiple participants enrolled to date in a run-in period for the pivotal Phase 3 trial, which is targeted to enroll as few as eight participants in a single arm, 12-month study utilizing an adaptive design. Dosing with OPGx-LCA5 expected in the second half of 2026.
- Application expected to be submitted in Q1 2026 for Rare Disease Evidence Principles (RDEP) review process from the
U.S. Food and Drug Administration (FDA), designed to provide greater speed and predictability in the review of therapies intended to treat rare diseases with very small patient populations with significant unmet medical need and that are driven by a known genetic defect. - Presentation of Phase 1/2 six-month pediatric cohort data expected at the annual meeting of the
Association for Research in Vision and Ophthalmology (ARVO) conference inMay 2026 .
Preclinical Gene Therapy Pipeline
- Funding secured from Abu Dhabi’s
Healthcare Research and Innovation Fund to conduct a clinical trial evaluating OPGx-MERTK for MERTK-related retinitis pigmentosa (RP), a rare IRD that causes progressive vision loss and eventual blindness. Clinical development activities are underway. - Preclinical work ongoing for Opus’ broad IRD pipeline related to genetic mutations in RHO, CNGB1, RDH12-LCA, and NMNAT1, with one to two programs targeted to enter clinical testing this year.
Phentolamine Ophthalmic Solution 0.75% (PS)
- The FDA accepted the Company’s supplemental New Drug Application (sNDA) for Phentolamine Ophthalmic Solution 0.75% for the treatment of presbyopia and set a PDUFA action date of
October 17, 2026 . - LYNX-3, the second pivotal Phase 3 trial in keratorefractive participants with visual disturbances under mesopic, low-contrast conditions (dim light disturbances), is ongoing with topline results expected in the first half of 2026.
- Presentation at
The Macula Society Annual Meeting titled “Preliminary Results from Sentinel Patient in a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for the Treatment of BVMD and ARB Due to BEST1 Mutations.” - Presentation at the
Asia-Pacific Academy of Ophthalmology Congress (APAO) titled “Gene Therapy for BEST1 Inherited Retinal Disease.” - Presentation at the
Advanced Therapies Week Conference titled “Building Scalable Viral Vector Manufacturing Models.” - 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 2025 titled “LYNX-2: A Pivotal Phase 3 Trial of Phentolamine Ophthalmic Solution in Post-Keratorefractive Surgery Subjects with Decreased Mesopic Visual Acuity.” - Presentation at Eyecelerator at the
American Academy of Ophthalmology (AAO) Annual Meeting titled “Transformative Gene Therapies for the Treatment of Rare Inherited Retinal Diseases.”
Financial Results for the Year Ended
Cash Position: As of
Revenue: License and collaborations revenue totaled
Research and Development (R&D) Expenses: R&D expenses were
General and Administrative (G&A) Expenses: G&A expenses were
Net Loss: Net loss for the year ended
About
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 0.75%, OPGx-LCA5, OPGx-BEST1, RDH12 and earlier stage programs, and expectations regarding us, our business prospects and our results of operations, and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our most recent Annual Report on Form 10-K and in our other filings with the U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.
Contacts:
Investors
IR Advisory Solutions
ir@opusgtx.com
Media
917-291-5744
kimberly.ha@kkhadvisors.com
-Financial Tables Follow-
Consolidated Balance Sheets (in thousands, except share amounts and par value) | ||||||||
| As of | ||||||||
| 2025 | 2024 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 45,091 | $ | 30,321 | ||||
| Accounts receivable | 1,995 | 3,563 | ||||||
| Contract assets and unbilled receivables (Note 10) | 1,170 | 2,209 | ||||||
| Prepaids and other current assets | 1,788 | 515 | ||||||
| Short-term investments | — | 2 | ||||||
| Total current assets | 50,044 | 36,610 | ||||||
| Property and equipment, net | 199 | 252 | ||||||
| Total assets | $ | 50,243 | $ | 36,862 | ||||
| Liabilities, Series A preferred stock and stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 3,293 | $ | 3,148 | ||||
| Accrued expenses and other liabilities | 4,488 | 8,147 | ||||||
| Total current liabilities | 7,781 | 11,295 | ||||||
| Warrant liabilities | 25,985 | — | ||||||
| Funding agreement, related party | 1,129 | — | ||||||
| Total liabilities | 34,895 | 11,295 | ||||||
| Commitments and contingencies (Note 3 and Note 9) | ||||||||
| Series A preferred stock, par value | — | 18,843 | ||||||
| Stockholders’ equity: | ||||||||
| Preferred stock, par value | — | — | ||||||
| Common stock, par value | 7 | 3 | ||||||
| Additional paid-in capital | 203,930 | 145,719 | ||||||
| Accumulated deficit | (188,589 | ) | (138,998 | ) | ||||
| Total stockholders’ equity | 15,348 | 6,724 | ||||||
| Total liabilities, Series A preferred stock, and stockholders’ equity | $ | 50,243 | $ | 36,862 | ||||
Consolidated Statements of Comprehensive Loss (in thousands, except share and per share amounts) | ||||||||
| For the Year Ended | ||||||||
| 2025 | 2024 | |||||||
| License and collaborations revenue | $ | 14,196 | $ | 10,992 | ||||
| Operating expenses: | ||||||||
| Research and development | 30,812 | 26,851 | ||||||
| General and administrative | 21,983 | 18,215 | ||||||
| Acquired in-process research and development | — | 28,000 | ||||||
| Total operating expenses | 52,795 | 73,066 | ||||||
| Loss from operations | (38,599 | ) | (62,074 | ) | ||||
| Fair value change in warrant and other derivative liabilities | (11,515 | ) | 72 | |||||
| Financing costs | (1,337 | ) | — | |||||
| Interest expense | (129 | ) | — | |||||
| Other income, net | 1,989 | 4,470 | ||||||
| Loss before income taxes | (49,591 | ) | (57,532 | ) | ||||
| Provision for income taxes | — | — | ||||||
| Net loss | (49,591 | ) | (57,532 | ) | ||||
| Other comprehensive loss, net of tax | — | — | ||||||
| Comprehensive loss | $ | (49,591 | ) | $ | (57,532 | ) | ||
| Net loss per share | ||||||||
| Basic and diluted | $ | (0.80 | ) | $ | (2.15 | ) | ||
| Number of shares used in per share calculations: | ||||||||
| Basic and diluted | 62,221,901 | 26,715,526 | ||||||
Source:
