Opus Genetics Announces Financial Results for Second Quarter 2025 and Provides Corporate Update

Rhea-AI Summary

Opus Genetics (NASDAQ:IRD) reported Q2 2025 financial results and significant pipeline progress. The company achieved key milestones including FDA RMAT designation for OPGx-LCA5 gene therapy, supported by positive 12-month Phase 1/2 data showing sustained vision improvements in adult patients. Their Phentolamine program met primary endpoints in two Phase 3 trials for presbyopia and night vision disturbances.

Financial highlights include $32.4M cash position funding operations into H2 2026, $2.9M in revenue (vs $1.1M in Q2 2024), and a net loss of $7.4M ($0.12 per share). The company secured non-dilutive funding up to $3.6M from patient advocacy groups to advance early-stage gene therapy programs.

Positive

• FDA granted RMAT designation for OPGx-LCA5, enabling accelerated development
• Positive 12-month Phase 1/2 data showing sustained vision improvements for OPGx-LCA5
• Two Phase 3 trials (VEGA-3 and LYNX-2) met primary endpoints for Phentolamine
• Secured $3.6M in non-dilutive funding from patient advocacy groups
• Revenue increased to $2.9M from $1.1M year-over-year
• Cash runway extended into second half of 2026

Negative

• Increased G&A expenses to $5.8M from $3.4M year-over-year
• Net loss of $7.4M for Q2 2025

Insights

Ophthalmology & Gene Therapy Expert

Opus Genetics reports multiple clinical trial successes and FDA RMAT designation, bolstering their gene therapy and small molecule pipeline for eye diseases.

Opus Genetics has achieved several significant clinical and regulatory milestones that substantially strengthen their position in the inherited retinal disease (IRD) space. The FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for their lead program OPGx-LCA5 is particularly noteworthy, as this designation is reserved for therapies showing preliminary clinical evidence of addressing serious conditions with unmet medical needs. This regulatory pathway could accelerate approval and provides validation of their clinical data.

The 12-month data from their Phase 1/2 trial demonstrating sustained improvements in visual function in adult LCA5 patients represents compelling evidence of durable treatment effect – critical for gene therapies where single-administration efficacy is the goal. The early positive signals in the pediatric cohort are especially promising since treating patients earlier in disease progression typically yields better outcomes in genetic disorders.

Their small molecule asset, Phentolamine Ophthalmic Solution 0.75%, demonstrated statistical significance in two Phase 3 trials. In the VEGA-3 trial for presbyopia, 27.2% of treated patients achieved a ≥15-letter gain in near visual acuity versus 11.5% for placebo (p<0.0001), representing a meaningful functional improvement. The successful LYNX-2 results in patients with night vision disturbances after refractive surgery provides a second potential indication.

The non-dilutive funding from patient advocacy groups ($1.6 million from Global RDH12 Alliance and $2 million from Retinal Degeneration Fund) allows Opus to advance early-stage programs without diluting shareholders. This approach of leveraging foundation funding is an efficient capital allocation strategy for rare disease programs.

Financially, Opus reports $32.4 million in cash, providing runway into the second half of 2026. Their quarterly net loss of $7.4 million ($0.12 per share) represents an improvement from the $7.8 million loss ($0.30 per share) in Q2 2024, suggesting progress toward operational efficiency despite increased G&A expenses.

Biotech Financial Analyst

Opus Genetics shows clinical progress across multiple programs with strengthened financial position through non-dilutive funding and collaborative revenue streams.

Opus Genetics' financial performance shows meaningful improvements in capital efficiency while advancing multiple clinical programs. Quarterly revenue increased substantially to $2.9 million from $1.1 million year-over-year, driven by their Viatris collaboration. This 164% revenue growth demonstrates successful execution of their partnership strategy, creating value through external validation and non-dilutive funding.

The cash position of $32.4 million provides runway into H2 2026, giving them approximately 12+ months of operational capacity. This timeline aligns strategically with their expected clinical milestones, including potential regulatory submissions for Phentolamine and advancement of gene therapy programs. The decreased net loss per share from $0.30 to $0.12 reflects improved operational metrics, though this partially stems from an increased share count.

The company's strategy of securing non-dilutive funding is particularly astute in the current biotech financing environment. The combined $3.6 million from patient foundations offsets approximately half a quarter of R&D expenses, effectively extending their runway while advancing additional pipeline assets. This approach of foundation partnerships for rare disease programs represents an underappreciated aspect of their capital allocation strategy.

R&D expenses remained essentially flat at $6.0 million versus $6.1 million in the prior year, despite advancing multiple clinical programs, suggesting efficient resource deployment. The increase in G&A expenses to $5.8 million from $3.4 million (70.6% increase) warrants monitoring, though some of this likely relates to intellectual property protection and business development activities supporting future value creation.

The Viatris collaboration structure, where Phentolamine development costs are fully reimbursed, allows Opus to advance a late-stage asset with commercial potential while focusing internal capital on their gene therapy platform. With multiple value-creating catalysts expected in H2 2025, including an sNDA submission and additional clinical data readouts, Opus has positioned themselves for potential inflection points across both their small molecule and gene therapy portfolios.

- 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 -

- FDA grants Regenerative Medicine Advanced Therapy (RMAT) designation for OPGx-LCA5 -

- Positive topline results reported from VEGA-3 and LYNX-2 Phase 3 trials with Phentolamine Ophthalmic Solution 0.75% -

- OPGx-BEST1 on track to enter Phase 1/2 trial in H2 2025 for the treatment of bestrophin-1 related inherited retinal disease -

- Non-dilutive funding from patient advocacy groups secured to advance multiple early-stage gene therapy programs -

RESEARCH TRIANGLE PARK, N.C., Aug. 13, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders, today announced financial results for the second quarter ended June 30, 2025, and provided a corporate update.

“We’ve made significant progress across our pipeline, with multiple clinical and regulatory milestones achieved this quarter,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “Receiving RMAT designation for our OPGx-LCA5 program underscores the strength of our clinical data and the urgent need for effective gene therapies to treat inherited retinal diseases. We are encouraged by the sustained functional vision improvements observed in adult patients in our clinical trial to date and the early signs of efficacy in the pediatric cohort. In parallel, our advancement of OPGx-BEST1 toward the clinic and the nomination of two additional development candidates in partnership with the Retinal Degeneration Fund and the Global RDH12 Alliance highlight the breadth of our IRD pipeline.”

“Beyond gene therapy, the positive readouts from our two Phase 3 Phentolamine trials represent a major step toward our goal of bringing a new treatment option to millions of patients living with vision challenges. With several upcoming key milestones, including new clinical data, a supplemental New Drug Application (sNDA) submission, and the launch of a pivotal study, we remain focused on execution to deliver transformative treatments to patients with significant unmet needs,” Dr. Magrath concluded.

Pipeline Updates

OPGx-LCA5 – Gene Therapy for Leber Congenital Amaurosis (LCA)

• The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation for OPGx-LCA5, for the treatment of Leber Congenital Amaurosis (LCA) due to genetic variations in the LCA5 gene, signifying the program’s potential to address a serious condition and providing a pathway for accelerated development and review.
• 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. This data was presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in May.
• Initial pediatric data at one-month post-treatment showed vision improvement with no drug-related adverse events; three-month pediatric data is expected to be reported in Q3 2025.
  

OPGx-BEST1 – Gene Therapy for BEST1-Related IRD

• Preclinical data presented at the American Ophthalmological Society (AOS) in May demonstrated restoration of the retinal pigment epithelium-photoreceptor interface in a canine model of BEST1 using AAV-mediated gene delivery.
• Investigational New Drug (IND) submission and Phase 1/2 trial initiation remain on track for the second half of 2025.

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 up to $2 million received from the Retinal Degeneration Fund to advance OPGx-MERTK, targeting retinitis pigmentosa caused by MERTK mutations.
• Preclinical OPGx-MERTK data presented at the American Society of Gene & Cell Therapy (ASGCT) in May showed preservation of retinal function in animal models.
  

Phentolamine Ophthalmic Solution 0.75% – Advancing Toward sNDA Submissions

• 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) and favorable participant reported outcomes and safety profile.
• LYNX-2 Phase 3 trial met its primary and multiple secondary endpoints in keratorefractive patients with night vision disturbances. Patients showed statistically significant gains in mesopic low contrast vision and improvements in night-driving related symptoms.
• sNDA submission for presbyopia indication planned for the second half of 2025.
• LYNX-3 Phase 3 trial expected to initiate enrollment in the second half of 2025 targeting reduced low light vision and nighttime visual disturbances.

Additional Medical Meeting Presentations

• Virtual reality-guided functional testing to support meaningful clinical endpoints in IRD trials was presented at the Retinal Imaging Biomarkers Summit.
  

Upcoming Expected Data Readouts and Program Advancements

• Report three-month pediatric data from OPGx-LCA5 Phase 1/2 trial in Q3 2025.
• Initiate enrollment in Phase 1/2 trial for OPGx-BEST1 in H2 2025.
• Submit Phentolamine sNDA for presbyopia in H2 2025.
• Initiate enrollment in Phentolamine LYNX-3 Phase 3 trial in H2 2025.

Financial Results for the Second Quarter Ended June 30, 2025

Cash Position: As of June 30, 2025, Opus Genetics had cash and cash equivalents of $32.4 million. Based on current operating plans, the Company expects its existing cash resources will fund operations into the second half of 2026.

Revenue: License and collaboration revenue totaled $2.9 million for the second quarter of 2025, compared to $1.1 million in the same period in 2024. Revenue in both periods was driven by the Company’s collaboration with Viatris, Inc., primarily from reimbursement of R&D services.

General and Administrative (G&A) Expenses: G&A expenses were $5.8 million for the second quarter of 2025, compared to $3.4 million for the same period in 2024. The increase was mainly due to higher costs associated with legal and patent-related expenses, payroll, and business development activities. G&A expenses included $0.6 million and $0.5 million in stock-based compensation in the second quarters of 2025 and 2024, respectively.

Research and Development (R&D) Expenses: R&D expenses were $6.0 million for the second quarter of 2025, compared to $6.1 million in the prior-year period. The slight decrease was primarily due to lower manufacturing and consulting costs, partially offset by increased clinical trial, toxicology, and payroll-related expenses. R&D expenses related to Phentolamine Ophthalmic Solution 0.75% were fully reimbursed under the Viatris License Agreement. Stock-based compensation within R&D expenses was $0.3 million in both periods.

Net Loss: Net loss for the second quarter of 2025 was $7.4 million, or $(0.12) per basic and diluted share, compared to a net loss of $7.8 million, or $(0.30) per basic and diluted share, for the second quarter of 2024.

SEC Filing: Additional details on the Company’s financial results will be available in its Quarterly Report on Form 10-Q for the period ended June 30, 2025, to be filed with the U.S. Securities and Exchange Commission (SEC).

About Opus Genetics

Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders. The Company’s pipeline features AAV-based gene therapies targeting inherited retinal diseases including Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa. Its lead gene therapy candidates are OPGx-LCA5, which is in an ongoing Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1, a gene therapy targeting BEST1-related retinal degeneration. Opus is also advancing Phentolamine Ophthalmic Solution 0.75%, a partnered therapy currently approved in one indication and being studied in two Phase 3 programs for presbyopia and reduced low light vision and nighttime visual disturbances. The Company is based in Research Triangle Park, NC. For more information, visit www.opusgtx.com.

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 Annual Report on Form 10-K for the fiscal year ended December 31, 2024 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
Jenny Kobin
Remy Bernarda
IR Advisory Solutions
ir@opusgtx.com

Media
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com

Opus Genetics, Inc. Condensed Consolidated Balance Sheets (in thousands, except share amounts and par value)
		As of
		June 30,			December 31,
		2025			2024
Assets		(Unaudited)
Current assets:
Cash and cash equivalents		$	32,429			$	30,321
Accounts receivable			3,399				3,563
Contract assets and unbilled receivables			1,178				2,209
Prepaids and other current assets			1,433				515
Short-term investments			—				2
Total current assets			38,439				36,610
Property and equipment, net			226				252
Total assets		$	38,665			$	36,862
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable		$	1,465			$	3,148
Accrued expenses and other liabilities			6,927				8,147
Warrant liabilities			11,800				—
Total current liabilities			20,192				11,295
Long-term funding agreement, related party			1,000				—
Total liabilities			21,192				11,295
Commitments and contingencies
Series A preferred stock, par value $0.0001; 14,146 shares were designated as of June 30, 2025 and December 31, 2024; zero and 14,145.374 shares issued and outstanding at June 30, 2025 and December 31, 2024, respectively.			—				18,843
Stockholders’ equity:
Preferred stock, par value $0.0001; 9,985,854 shares authorized as of June 30, 2025 and December 31, 2024; no shares issued and outstanding at June 30, 2025 and December 31, 2024.			—				—
Common stock, par value $0.0001; 125,000,000 shares authorized as of June 30, 2025 and December 31, 2024; 59,908,055 and 31,574,657 shares issued and outstanding at June 30, 2025 and December 31, 2024, respectively.			6				3
Additional paid-in capital			172,079				145,719
Accumulated deficit			(154,612	)			(138,998	)
Total stockholders’ equity			17,473				6,724
Total liabilities, series A preferred stock and stockholders’ equity		$	38,665			$	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 June 30,								For the Six Months Ended June 30,
		2025				2024				2025				2024
License and collaborations revenue		$	2,882			$	1,112			$	7,252			$	2,823
Operating expenses:
General and administrative			5,766				3,354				12,112				8,024
Research and development			6,022				6,086				13,975				10,835
Total operating expenses			11,788				9,440				26,087				18,859
Loss from operations			(8,906	)			(8,328	)			(18,835	)			(16,036	)
Fair value change in warrant and other derivative liabilities			917				—				3,722				—
Financing costs			35				—				(1,337	)			—
Other income, net			534				563				836				1,165
Loss before income taxes			(7,420	)			(7,765	)			(15,614	)			(14,871	)
Benefit (provision) for income taxes			—				—				—				—
Net loss			(7,420	)			(7,765	)			(15,614	)			(14,871	)
Other comprehensive loss, net of tax							—								—
Comprehensive loss		$	(7,420	)		$	(7,765	)		$	(15,614	)		$	(14,871	)
Net loss per share:
Basic and diluted		$	(0.12	)		$	(0.30	)		$	(0.32	)		$	(0.59	)
Number of shares used in per share calculations:
Basic and diluted			63,376,392				25,827,265				48,712,124				25,175,596

Source: Opus Genetics, Inc.

FAQ

What were Opus Genetics (IRD) Q2 2025 financial results?

Opus reported $2.9M in revenue, $7.4M net loss ($0.12 per share), and had $32.4M in cash as of June 30, 2025, with runway into H2 2026.

What is the significance of RMAT designation for Opus Genetics' OPGx-LCA5?

The FDA's RMAT designation enables accelerated development and review of OPGx-LCA5, recognizing its potential to address serious conditions in Leber Congenital Amaurosis patients.

How did Opus Genetics' Phase 3 VEGA-3 trial perform?

VEGA-3 met primary endpoints with 27.2% of treated patients achieving ≥15-letter gain in near visual acuity vs 11.5% on placebo (p<0.0001) for presbyopia treatment.

What upcoming milestones does Opus Genetics (IRD) have in 2025?

Key milestones include 3-month pediatric data for OPGx-LCA5 in Q3, Phase 1/2 trial initiation for OPGx-BEST1, Phentolamine sNDA submission, and LYNX-3 Phase 3 trial initiation in H2 2025.

How much non-dilutive funding did Opus Genetics secure for gene therapy programs?

Opus secured up to $3.6M in total non-dilutive funding: $1.6M from Global RDH12 Alliance and $2M from Retinal Degeneration Fund.