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Opus Genetics Highlights 2025 Progress and Upcoming 2026 Catalysts

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- Multi-asset pipeline with significant targeted data readouts and milestones - - Two lead ophthalmi...

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Corporate Developments

The informative nature of upcoming milestones and financial stability heights the relevance of the article. Positive clinical trials are crucial for biotech companies, influencing future stock price significantly.

FAQ

Why Bullish?

The anticipated clinical data and regulatory designations can positively influence investor sentiment, potentially increasing IRD’s stock price. Past examples include companies like Spark Therapeutics, which saw share price boosts upon successful trial outcomes and regulatory progress.

How important is it?

The informative nature of upcoming milestones and financial stability heights the relevance of the article. Positive clinical trials are crucial for biotech companies, influencing future stock price significantly.

Why Short Term?

Pending clinical data from the BEST1 program and LCA5 trials are imminent; thus, the impact is likely to materialize quickly. Rapid reactions to announcements are common in biotech stocks.

Related Companies

Opus Genetics (IRD) Highlights 2025 Advancements and 2026 Projections

RESEARCH TRIANGLE PARK, N.C., January 8, 2026 (GLOBE NEWSWIRE) – Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biopharmaceutical company focused on gene therapies for inherited retinal diseases (IRDs), has announced significant achievements from 2025 and outlined exciting developments anticipated for 2026. The company is poised to share substantial clinical data and accelerate its pipeline towards FDA approval.

2025 Achievements and 2026 Expectations

Opus Genetics anticipates announcing vital clinical data from its BEST1 gene therapy program in 2026, while also preparing to move the LCA5 program into pivotal testing. This testing could lead to potential FDA approval. Most programs within Opus's pipeline are eligible for Rare Pediatric Disease designations, which may eventually result in Priority Review Vouchers (PRVs), offering non-dilutive capital for growth.

“2025 was a year defined by strong execution at Opus,” said George Magrath, M.D., Chief Executive Officer of Opus Genetics. “We achieved aggressive milestones across multiple ophthalmic programs…”

Key Clinical Programs

  • OPGx-BEST1: This gene therapy aims to treat both dominant and recessive forms of BEST1 disease. The first participant has been dosed in the Phase 1/2 trial (BIRD-1), with initial data expected at the Macula Society this quarter.
  • OPGx-LCA5: Focused on Leber Congenital Amaurosis (LCA), this program has received multiple regulatory designations including Rare Pediatric Disease and Orphan Drug. Positive outcomes have been reported in the Phase 1/2 trials, and the pivotal Phase 3 trial is expected to commence later in 2026.

Advancements in the Pipeline

Opus Genetics is cultivating partnerships with patient advocacy groups, enhancing awareness and funding for IRD issues. Prominent pre-clinical programs target genetic mutations including RHO, CNGB1, MERTK, RDH12-LCA, and NMNAT1, with plans to initiate clinical testing for one or two this year.

Regulatory Developments

The company has submitted a supplemental New Drug Application (sNDA) for the treatment of presbyopia, with a regulatory decision anticipated by the end of 2026. The ongoing LYNX-3 trial seeks to validate the efficacy of Phentolamine Ophthalmic Solution 0.75% in keratorefractive patients.

Financial Position and Future Outlook

As of September 30, 2025, Opus Genetics reported cash and cash equivalents totaling $30.8 million. Following a recent equity offering that raised approximately $23.0 million, the company’s cash position has improved to over $50 million. This capital is expected to support operational needs into the second half of 2027, absent any additional funding from warrants or milestone payments.

About Opus Genetics

Opus Genetics is dedicated to developing gene therapies that aim to restore vision and prevent blindness for patients suffering from inherited retinal diseases (IRDs). With a pipeline featuring seven AAV-based programs, the company’s focus includes therapies for LCA5-related mutations and BEST1-related retinal degeneration among others. Located in Research Triangle Park, NC, Opus Genetics is also investigating new indications for existing compounds, aiming to innovate in the field of ophthalmology.

Forward-Looking Statements

This press release contains forward-looking statements under the Private Securities Litigation Reform Act of 1995. These include projections related to clinical developments, cash resources, and regulatory outcomes.

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