Opus Genetics Announces Financial Results for First Quarter 2025 and Provides Corporate Update ARVO presentation highlights 12-month results from Phase 1/2 study that support potential of OPGx-LCA5 gene therapy to restor

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

ARVO presentation highlights 12-month results from Phase 1/2 study that support

potential of OPGx-LCA5 gene therapy to restore meaningful vision

Pediatric cohort of LCA5 study ongoing with initial multi-patient data anticipated in Q3 2025

OPGx-BEST1 on track for IND filing and initiation of Phase 1/2 trial with early data expected in Q1 2026

Leading healthcare investors provide funding to deliver on key milestones

Research Triangle Park, NC, May 15, 2025 (GLOBE NEWSWIRE) - Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biopharmaceutical company developing important

new therapies for the treatment of inherited retinal diseases (IRDs) and other ophthalmic disorders ("Opus" or the "Company"), today announced financial results for the first quarter ended March 31, 2025, and provided a corporate update.

"Opus is off to a strong start in 2025, with progress across our two product portfolios - the inherited retinal disease platform and the phentolamine

eye drop franchise," said George Magrath, M.D., Chief Executive Officer. "We reported positive 12-month data from the Phase 1/2 trial of OPGx-LCA5 for the treatment of Leber congenital amaurosis

5 (LCA5), an inherited disease which leads to severe vision loss from infancy. The evidence of durable efficacy in adult patients is very encouraging, with the treatment benefits previously seen

at 6 months now sustained out to one year. We are now enrolling pediatric patients and are encouraged by the meaningful improvement in visual function and early safety profile observed in the first of these patients at one month.

Dr. Magrath continued, "We believe that the initial success with our lead IRD program, OPGx-LCA5, has the potential to translate to the rest of our

pipeline, which includes gene therapy candidates for six additional IRDs. We plan to initiate a Phase 1/2 study with our BEST-1 program later this year with early data expected in the first

"In the phentolamine eye drop pipeline, we look forward to near-term clinical data readouts from the Phase 3 data trials in dim light vision

disturbances and in presbyopia. We ended the first quarter with a strong cash position, having completed a successful financing with leading institutional healthcare investors who share our confidence in the Company's strategic direction,"

concluded Dr. Magrath.

Strategic Highlights

Recent Business Highlights and Corporate Updates

Gene Therapy Programs

Phentolamine Ophthalmic Solution 0.75%

Expected Growth Drivers in 2025 and Beyond

Financial Highlights for the First Quarter Ended March 31, 2025

As of March 31, 2025, Opus had cash and cash equivalents of $41.8 million. Based on current projections, management believes that the cash on hand will be sufficient to

fund operations into the second quarter of 2026.

License and collaborations revenue was $4.4 million and $1.7 million during the three months ended March 31, 2025 and 2024, respectively. Revenue during both quarterly

periods was derived from the Company's license and collaboration agreement (the "Viatris License Agreement") with Viatris, Inc. ("Viatris") largely from the reimbursement of research and development services and to a much lesser degree from royalty

payments stemming from the sales of RYZUMVI by Viatris.

General and administrative ("G&A") expenses for the three months ended March 31, 2025 were $6.3 million compared to $4.7 million for the three months ended March 31,

2024. The increase was primarily attributable to professional services fees, corporate legal support, legal fees associated with intellectual property and public company costs, including filing fees and investor relations and governance costs, offset

in part by decreases in general operating and other costs on a net basis. G&A expenses included $0.6 million and $0.8 million in stock-based compensation expense during the three months ended March 31, 2025 and 2024, respectively.

Research and development ("R&D") expenses for the three months ended March 31, 2025 were $8.0 million compared to

$4.7 million for the three months ended March 31, 2024. The increase was primarily attributable to higher clinical costs and payroll related costs, offset partially by lower manufacturing expenses attributed to an activity reduction in the VEGA-2

trial and by lower regulatory and other costs on a net basis. Pursuant to the Viatris License Agreement, budgeted R&D expenses related to the development of the Phentolamine Ophthalmic Solution 0.75% products have been fully reimbursed by Viatris

to date. R&D expenses included $0.3 million and $0.2 million in stock-based compensation expense during the three months ended March 31, 2025 and 2024, respectively.

Net loss for the quarter ended March 31, 2025, was $8.2 million or $(0.24)

per basic and diluted share, as compared to net loss of $7.1 million, or $(0.29) per basic and diluted share, for the quarter ended March 31, 2024.

For further details on financial results, refer to the Company's Quarterly Report on Form 10-Q for the quarter ended March 31 2025, to be filed with the Securities and

Exchange Commission (the "SEC").

Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing therapies to treat patients with IRDs and other treatments for ophthalmic disorders. Our

pipeline includes adeno-associated virus (AAV)-based investigational gene therapies that address gene mutations responsible for different forms of Leber congenital amaurosis ("LCA"), bestrophinopathy and retinitis pigmentosa. Our most advanced

investigational gene therapy program is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein and is currently being evaluated in a Phase 1/2 open-label, dose-escalation trial, with encouraging early data. Our

pipeline also includes BEST1 investigational gene therapy, designed to address mutations in the BEST1 gene, which is associated with retinal degeneration. The pipeline also includes Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1

and alpha-2 adrenergic antagonist being investigated to reduce pupil size that is currently being evaluated in Phase 3 trials for presbyopia and mesopic (dim) light vision disturbances. For additional information, please visit www.opusgtx.com.

University of Pennsylvania ("Penn") Financial Disclosure: The

laboratory of Dr. Tomas Aleman has received clinical trial research funding from Opus Genetics. Penn and Dr. Aleman have either received, or may receive in the future, financial consideration related to the licensing of certain Penn intellectual

property to Opus Genetics.

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, expectations regarding our cash runway, expectations of potential growth, and statements concerning data from and future enrollment for our clinical trials and our pipeline of additional indications.

These forward-looking statements relate to 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, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, and in our other

filings with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. 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.

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, including, without limitation:

The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive. Readers are urged to carefully

review and consider the various disclosures made by us in this report and in our other reports filed with the SEC that advise interested parties of the risks and factors that may affect our business. All forward-looking statements contained in this

press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Condensed Consolidated Balance Sheets

(in thousands, except share amounts and par value)

Condensed Consolidated Statements of Comprehensive Loss

(in thousands, except share and per share amounts)