Opus Genetics Reports Progress in Gene Therapy Pipeline; Ultragenyx Announces Restructuring

Opus Genetics announced positive early clinical data from its gene therapy programs for inherited retinal diseases and provided financial results for the year ended December 31, 2025. The FDA set a PDUFA action date of October 17, 2026 for the company's Phentolamine Ophthalmic Solution 0.75% for the treatment of presbyopia.

The company's OPGx-BEST1 gene therapy program for BEST1-related inherited retinal disease showed favorable early safety and initial efficacy data. Positive initial three-month data from the first participant in the Phase 1/2 trial (BIRD-1) 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.

Recruitment is ongoing at multiple U.S. sites with two participants treated to date in the Phase 1/2 trial that includes patients with both dominant and recessive forms of BEST disease.

For the OPGx-LCA5 gene therapy program for Leber Congenital Amaurosis, recruitment is 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 is expected in the second half of 2026. An application is expected to be submitted in Q1 2026 for Rare Disease Evidence Principles (RDEP) review process from the 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 is expected at the Association for Research in Vision and Ophthalmology (ARVO) conference in May 2026.

Opus secured funding from Abu Dhabi's Healthcare Research and Innovation Fund to conduct a clinical trial evaluating OPGx-MERTK for MERTK-related retinitis pigmentosa, a rare inherited retinal disease that causes progressive vision loss and eventual blindness. Clinical development activities are underway. Preclinical work is ongoing for Opus' broad inherited retinal disease pipeline related to genetic mutations in RHO, CNGB1, RDH12-LCA, and NMNAT1, with one to two programs targeted to enter clinical testing this year.

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.

Funding from prominent healthcare investors is expected to extend Opus' cash runway into 2028.

Separately, Ultragenyx Pharmaceutical Inc. reported financial results for the quarter and year ended December 31, 2025. The company reported $207 million in total revenue for the fourth quarter 2025, which represents 25% growth compared to the same period in 2024. Total revenue for the year ended December 31, 2025 was $673 million, which represents 20% growth compared to the prior year.

Crysvita revenue in the fourth quarter 2025 was $145 million, which includes product sales of $40 million from Latin America and Türkiye. Full year 2025 Crysvita revenue was $481 million, which represents 17% growth compared to the prior year. This includes product sales of $177 million from Latin America and Türkiye, which represents 31% growth compared to the prior year.

Dojolvi revenue in the fourth quarter 2025 was $32 million. Dojolvi revenue in 2025 was $96 million, which represents 9% growth compared to the prior year. Evkeeza revenue in the fourth quarter 2025 was $17 million. Evkeeza revenue in 2025 was $59 million, which represents 84% growth compared to the prior year, as demand continues to build following launches in the company's territories outside of the United States. Mepsevii revenue in the fourth quarter 2025 was $13 million, and full year 2025 revenue was $37 million.

Total operating expenses for the fourth quarter 2025 were $321 million. Total operating expenses for the year ended December 31, 2025 were $1.2 billion, including $153 million of non-cash stock-based compensation. Research and development expenses for the year were $750 million, and selling, general and administrative expenses were $349 million.

Net loss for the fourth quarter 2025 was $129 million, or $1.29 per share basic and diluted, compared with a net loss for the fourth quarter 2024 of $133 million, or $1.39 per share basic and diluted. Net loss for the year ended December 31, 2025 was $575 million, or $5.83 per share basic and diluted, compared with a net loss the prior year of $569 million, or $6.29 per share, basic and diluted.

Cash, cash equivalents, and marketable securities were $737 million as of December 31, 2025. For the year ended December 31, 2025, net cash used in operations was $466 million.

Ultragenyx announced 2026 total revenue from current products is expected to be between $730 million to $760 million. The company initiated a strategic restructuring plan to significantly reduce and focus expenses and headcount, reiterating a path to profitability in 2027. The company stated that 2026 catalysts include two potential approvals and expected pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome.

On December 29, 2025, Ultragenyx announced that both its Phase III Orbit and Cosmic Studies had not achieved statistical significance against the primary endpoints of reduction in annualized clinical fracture rate compared to placebo or bisphosphonates, respectively. The company attributed the study failure to a low fracture rate in the placebo group of Orbit and a trend that fell shy of statistical significance in Cosmic. On July 9, 2025, Ultragenyx had revealed that the Phase III Orbit study failed to achieve statistical significance for the second interim analysis and that Phase III Orbit and Cosmic studies would be progressing toward final analysis.