Opus Genetics Highlights 2025 Progress and Upcoming 2026 Catalysts

Rhea-AI Summary

Opus Genetics (Nasdaq: IRD) reviewed 2025 progress and outlined 2026 catalysts across its ophthalmic gene therapy pipeline. Key near-term milestones include 3-month cohort data from the Phase 1/2 BEST1 trial expected mid-2026, initiation of pivotal Phase 3 dosing for LCA5 in the second half of 2026 after positive Phase 1/2 results, and an sNDA submission for a partnered presbyopia treatment with a regulatory decision anticipated by end of 2026. The company reported cash and cash equivalents of $30.8M as of Sept 30, 2025 and raised ~$23.0M in a subsequent registered direct offering, giving a cash runway into the second half of 2027 based on current plans. Multiple programs may qualify for Rare Pediatric Disease designation and other expedited pathways.

Positive

• Cash position increased to over $50M after $23M offering
• LCA5 showed positive Phase 1/2 safety and efficacy data
• Pivotal Phase 3 LCA5 dosing planned H2 2026
• BEST1 Phase 1/2 cohort data expected mid-2026
• sNDA filed for presbyopia with decision expected end-2026
• Multiple regulatory designations granted (Rare Pediatric, Orphan, RMAT)

Negative

• Cash runway projected only into the second half of 2027
• Raised $23M via equity offering, which may dilute shareholders

Key Figures

Cash & equivalents $30.8 million As of September 30, 2025

Registered direct proceeds $23.0 million Gross proceeds from equity offering after Q3 2025

Cash runway Into second half of 2027 Based on current operating plans

PS concentration 0.75% Phentolamine Ophthalmic Solution for presbyopia

OPGx-BEST1 stage Phase 1/2 trial BEST1-related inherited retinal disease program

OPGx-LCA5 stage Pivotal Phase 3 planned Adaptive trial with dosing expected 2H 2026

New clinical programs 1–2 programs Pre-clinical IRD candidates targeted to enter trials in 2026

Presbyopia decision timing By end of 2026 sNDA for phentolamine ophthalmic solution

Market Reality Check

$1.93 Last Close

Volume Volume 351,526 is below 20-day average 597,904 ahead of this catalyst update. low

Technical Shares at $2.11 are trading above the 200-day MA of $1.41 and 18.55% below the 52-week high.

Peers on Argus

IRD gained 1.44% while peers showed mixed moves: ANEB +3.42%, IMUX +7.23%, OKYO +0.45%, OVID +0.56%, ATRA -1.45%. With no peers in the momentum scanner and mixed directions, trading appeared more company-specific than sector-driven.

Historical Context

Date	Event	Sentiment	Move	Catalyst
Dec 16	Inducement equity grant	Neutral	-5.6%	Inducement stock options for a new hire under the 2021 plan.
Dec 11	Conference presentation	Neutral	-2.4%	Announcement of J.P. Morgan Healthcare Conference presentation by the CEO.
Dec 09	Clinical safety update	Positive	+4.4%	IDMC positive recommendation to continue the BEST1 Phase 1/2 BIRD-1 trial.
Dec 02	Inducement equity grants	Neutral	-0.5%	Equity option grants to two new non-executive employees under inducement plan.
Nov 20	Investor conferences	Neutral	+3.8%	Participation in BTIG Ophthalmology Day and Piper Sandler healthcare conference.

Pattern Detected

Limited history shows a positive clinical safety update for BEST1 coinciding with a 4.37% gain, while routine governance and conference items saw relatively modest, mixed reactions.

Recent Company History

Over late 2025, Opus mainly reported corporate housekeeping and investor-relations items, such as inducement grants and conference participation, which produced small, mixed price moves. One clear clinical catalyst was the Dec 9, 2025 positive IDMC recommendation for the BEST1 Phase 1/2 trial, which aligned with a 4.37% gain. Today’s broad 2025 progress and 2026 catalyst update ties directly to those BEST1 and LCA5 programs and to the capital raised in November 2025 to support their advancement.

Market Pulse Summary

The stock moved -5.2% in the session following this news. A negative reaction despite the multi-asset progress update could fit a pattern where routine or financing-linked news occasionally coincided with weakness, such as the -5.61% move after a December inducement grant. Investors may reassess dilution and long timelines to catalysts like 2026 BEST1 data and LCA5 Phase 3 dosing. If confidence in execution or funding durability fades, earlier positive responses to BEST1 safety data might not provide support.

Key Terms

gene therapy medical

"a clinical-stage biopharmaceutical company developing gene therapies to restore vision"

Gene therapy is a medical technique that involves altering or replacing faulty genes in a person's cells to treat or prevent disease. It is considered a promising area of innovation because it has the potential to provide long-term or even permanent solutions to genetic conditions. For investors, advancements in gene therapy can signal opportunities in biotech companies and emerging treatments with significant growth potential.

inherited retinal diseases medical

"prevent blindness in patients with inherited retinal diseases (IRDs)"

Inherited retinal diseases are a group of genetic conditions in which errors in the genes that build and maintain the light-sensing cells at the back of the eye cause progressive vision loss or blindness; think of it as faulty wiring in a camera that gradually degrades the picture. They matter to investors because they define a clear patient population with high unmet need, driving demand for diagnostics and novel treatments (gene, cell or drug therapies) that can produce long-term revenue, regulatory milestones and intellectual property value.

supplemental new drug application regulatory

"Supplemental New Drug Application (sNDA) submitted for partnered presbyopia treatment"

A supplemental new drug application is a request submitted to regulatory authorities to make changes to an existing approved medication, such as adding new uses, strengths, or formulations. For investors, it signals that a pharmaceutical company is seeking approval for new product developments or expanded applications, which can impact the company's future sales, market potential, and stock value.

rare pediatric disease regulatory

"qualify for Rare Pediatric Disease designation, which could result in Priority Review Vouchers"

A rare pediatric disease is a serious medical condition that primarily affects children and occurs so infrequently that only a small number of patients exist. Investors care because treatments for such conditions often get special regulatory incentives—think of government fast lanes and rewards for developers—making smaller markets potentially profitable due to pricing power, shorter development timelines, and reduced competition, much like a niche product that receives government-backed advantages.

priority review vouchers regulatory

"could result in Priority Review Vouchers (PRV), providing future non-dilutive capital"

A priority review voucher is an official token issued by a drug regulator that lets a company move a future drug application to the front of the review line, cutting the time regulators take to decide. It matters to investors because the voucher is often transferable or sellable and can be worth hundreds of millions, acting like a "fast-pass" that speeds a product to market or becomes a one-time cash asset on a company’s balance sheet.

regenerative medicine advanced therapy regulatory

"designations granted including Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy (RMAT)"

Regenerative Medicine Advanced Therapy (RMAT) is a U.S. regulatory designation for cell, gene, and tissue‑based therapies intended to treat serious or life‑threatening conditions; it gives developers a “fast lane” with more frequent agency interaction and eligibility for accelerated review pathways. For investors, an RMAT label signals that a therapy may reach market faster and face less regulatory uncertainty than a standard program, which can raise the potential value and reduce timeline risk—though it is not a guarantee of approval.

phase 1/2 trial medical

"First participant dosed in Phase 1/2 trial (BIRD-1) with enrollment ongoing"

A phase 1/2 trial combines the earliest human safety testing with an initial look at whether a treatment works, typically starting by checking tolerability and side effects and then expanding to measure early signs of benefit and the best dose. For investors, results from these trials are an early indicator of a drug’s clinical promise and regulatory path: positive data can materially increase a company’s value and reduce development risk, while negative data can sharply lower expectations.

pivotal phase 3 trial medical

"run-in period for planned, adaptive pivotal Phase 3 trial"

A pivotal phase 3 trial is the late-stage clinical study designed to provide the decisive evidence regulators need to approve a new drug or medical treatment. Think of it as the final exam where the treatment must prove it works and is safe in a large group of people; investors watch the results closely because a positive outcome can unlock regulatory approval, sales, milestone payments and big changes in a company’s valuation.

AI-generated analysis. Not financial advice.

- Multi-asset pipeline with significant targeted data readouts and milestones -

- Two lead ophthalmic gene therapy programs in clinical trials with new data anticipated in 2026 -

- Supplemental New Drug Application (sNDA) submitted for partnered presbyopia treatment -

- Corporate presentation at J.P. Morgan Healthcare Conference on Thursday, January 15, at 8:15 a.m. PT -

RESEARCH TRIANGLE PARK, N.C., Jan. 08, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the “Company,” “Opus,” 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 highlighted its progress achieved during 2025 and upcoming catalysts in 2026.

In 2026, Opus is expected to announce clinical data from its BEST1 program and accelerate its LCA5 program into pivotal testing and toward potential U.S. Food and Drug Administration (FDA) approval. Additional programs are also expected to advance into the clinic. Most of Opus’ pipeline programs have the potential to qualify for Rare Pediatric Disease designation, which could result in Priority Review Vouchers (PRV), providing future non-dilutive capital.

“2025 was a year defined by strong execution at Opus,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “We achieved aggressive milestones across multiple ophthalmic programs – advancing two gene therapies in clinical trials and submitting an application to the FDA for approval of our second partnered commercial product, which could potentially provide a large new market opportunity. We secured capital from leading institutional investors and through non-dilutive sources such as patient advocacy grant funding. We believe that the progress we’ve made with our LCA5 and BEST1 programs is proof of what’s possible and our broader pipeline is poised to follow. We enter 2026 with confidence, momentum, and an unwavering commitment to patients who inspire everything we do.”

Corporate Updates

OPGx-BEST1 – Gene Therapy for BEST1-Related IRD

• Potential treatment for both the dominant and recessive forms of BEST1 disease.
• First participant dosed in Phase 1/2 trial (BIRD-1) with enrollment ongoing.
• Initial data expected this quarter at Macula Society with 3-month results from the entire Cohort 1 expected in mid-2026.
• OPGx-BEST1 is potentially eligible for multiple regulatory designations which the Company expects to file for in 2026.

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

• Multiple regulatory designations granted including Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy (RMAT) with potential eligibility for Priority Review Voucher upon approval.
• Positive Phase 1/2 safety and efficacy data reported in adults and pediatric participants.
• Enrollment ongoing in run-in period for planned, adaptive pivotal Phase 3 trial.
• Dosing with OPGx-LCA5 in the Phase 3 trial expected in the second half of 2026.

Pre-Clinical Gene Therapy Pipeline

• Multiple partnerships with patient advocacy organizations are increasing awareness in the IRD community and providing non-dilutive funding for pre-clinical testing of Opus Genetics gene therapy pipeline.
• Promising programs for IRDs related to genetic mutations in RHO, CNGB1, MERTK, RDH12-LCA, and NMNAT1 with one to two programs targeted to enter clinical testing this year.

Phentolamine Ophthalmic Solution 0.75% (PS)

• Supplemental New Drug Application (sNDA) submitted for the treatment of presbyopia, with an anticipated regulatory decision by the end of 2026.
• LYNX-3, the second pivotal Phase 3 trial in keratorefractive participants with visual disturbances under mesopic, low-contrast conditions, is ongoing with topline results expected in the first half of 2026.

Financial Outlook 

As of September 30, 2025, Opus Genetics had cash and cash equivalents of $30.8 million. Subsequent to the end of the third quarter of 2025, the Company raised approximately $23.0 million in gross proceeds through a registered direct offering of equity securities, resulting in a total cash position of over $50 million. Based on current operating plans, the Company expects its existing cash resources will fund operations into the second half of 2027, excluding any potential proceeds from callable warrants or future milestone payments.

About OPGx-BEST1 and the Phase 1/2 Trial

OPGx-BEST1 leverages Opus Genetics’ proprietary AAV-based gene therapy platform, designed to deliver a functional copy of the BEST1 gene directly to the retinal pigment epithelium (RPE) cells where the defective gene resides. The program builds on extensive preclinical work demonstrating restoration of BEST1 protein expression and improved retinal function in relevant disease models. The multi-center, adaptive, open-label, dose-exploring Phase 1/2 trial, known as BIRD-1, will evaluate the safety, tolerability, and preliminary efficacy of OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB). Treatment will be administered via a single subretinal injection in one eye of each participant with two dosing cohorts. The trial will also explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure.

About OPGx-LCA5

OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being advanced into a pivotal Phase 3 trial. Data from pediatric participants demonstrated large gains in cone-mediated vision, and the therapy remains well tolerated with no ocular serious adverse events or dose-limiting toxicities. The adult cohort showed durable improvements in cone sensitivity and visual function out to 18 months. OPGx-LCA5 has received Rare Pediatric Disease, Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA.

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, CNGB1, RDH12, NMNAT1, and MERTK. Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75%, an approved small-molecule therapy for pharmacologically induced mydriasis, with additional potential indications in presbyopia and low-light visual disturbances following keratorefractive surgery. 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, our subsequent Quarterly Report on Form 10-Q, and 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

Source: Opus Genetics, Inc.

FAQ

What clinical data will Opus Genetics (IRD) report in mid-2026?

Opus expects 3-month results from Cohort 1 of the Phase 1/2 BEST1 trial in mid-2026.

When will Opus (IRD) begin pivotal Phase 3 dosing for LCA5?

Opus plans to start dosing in the pivotal Phase 3 LCA5 trial in the second half of 2026.

What is the status of the presbyopia approval effort for Opus (IRD)?

A supplemental NDA for the partnered presbyopia treatment was submitted, with a regulatory decision anticipated by end of 2026.

How much cash does Opus Genetics (IRD) have and how long will it fund operations?

Opus reported $30.8M as of Sept 30, 2025 and raised ~$23.0M, giving cash to fund operations into the second half of 2027 per current plans.

Which regulatory designations has Opus (IRD) received for LCA5?

LCA5 has received Rare Pediatric Disease, Orphan Drug, and RMAT designations.