Opus Genetics (IRD) investor relations material

Company overview and gene therapy platform

• Focuses on gene therapies for inherited retinal diseases, with seven assets in the portfolio and two already in the clinic; three more are expected to enter the clinic within 12 months.

• Platform leverages structure-function dissociation, targeting diseases where tissue structure is preserved but function is lost, using well-characterized AAV capsids to minimize risk.

• Regulatory environment remains favorable for rare disease and gene therapy, with steady interactions at the project level despite leadership changes at the FDA and CBER.

Clinical program updates and trial design

• BEST1 program targets about 8,400 U.S. patients, with both recessive and dominant forms; phase I/II basket trial includes both types, with five patients per cohort and two dose levels.

• Sentinel patient in BEST1 trial, legally blind for decades, showed a 12-letter BCVA gain after low-dose treatment, attributed to reduction in central fluid pocket.

• September data readout will include BCVA, low luminance visual acuity, microperimetry, contrast sensitivity, and volumetric OCT analysis; primary endpoint may be microperimetry or OCT-based.

• LCA5 program addresses a population of about 170 in the U.S.; phase I/II data showed meaningful vision gains, leading to RMAT and RDEP status and a seamless phase I/II/III trial design.

• Phase III LCA5 trial uses a run-in natural history period, enrolling both pediatric and adult patients, with each patient serving as their own control.

Pipeline and financial outlook

• Three additional programs (RDH12, MERTK, RHO) are set to enter the clinic, with RDH12 and MERTK targeting pediatric populations and RHO primarily adult onset.

• MERTK trial will be conducted in Abu Dhabi due to higher prevalence and local support; RHO program targets retinitis pigmentosa with a dual construct approach.

• At least five out of seven programs are eligible for Rare Pediatric Disease Priority Review Vouchers, potentially generating significant non-dilutive capital.

• Presbyopia program, partnered with Viatris, has an October PDUFA date and could yield regulatory milestones and royalties.

• Cash position stands at $90 million, providing runway into 2029 and supporting advancement to the first PRV auction.