REGENXBIO Inc. Q4 FY2025 earnings call

2026 is a pivotal year for Regenexx Bio. Focus on advancing late-stage pipeline. RGX202, potential best-in-class gene therapy for Duchenne, completed dosing in pivotal study, strong enrollment in confirmatory trial, growing clinical data set. Cerevec franchise advancing one-time treatment for wet AMD and diabetic retinopathy. Partnership with AbbVie progresses, top line data for subretinal Cirovec in wet AMD expected in Q4. Site activation plans underway in Navigate Pivotal Study in Diabetic Retinopathy. Received CRL for RGX121 and clinical hold letters for RGX-111 and RGX-121, working to address holds and resubmit BLA.

Cash equivalent and marketable securities at year end 2025 were $241 million. Expect cash runway to fund operations into early 2027, and could extend with $100 million milestone from AVI and additional funds from royalty agreement. Guidance doesn't include revenue from MPS programs.

Forward-looking statements subject to risks and uncertainties. Risks described in Risk Factors and Management's Discussion and Analysis sections of annual and quarterly reports. For MPS programs, AAV vector integration event associated with tumor formation in a patient, but clinicians view benefit-risk as favorable given unmet need.

Q: As we get closer to data and regulatory clarity in terms of submission around DMD, concerns about controlled trial or appropriate control arm.

A: Confidence from protocol prospectively reviewed by FDA, compelling data on functional outcomes especially in older patients, confirmatory study enrollment increasing safety exposures.

Q: If FDA wants longer duration follow-up for safety and traditional approval, what would that entail?

A: Near-term 12-month functional data on full pivotal data set, confirmatory study enrollment providing more data.

Q: On AMD, why not go straight to phase three from dose level three?

A: Phase two-b three to expand data set, validate results, prudent risk management.

Q: On MPS1 neoplasm, how shifts benefit-risk discussion?

A: Clinicians view rare tumor risk vs inexorable decline and brain damage, benefit-risk favorable.

Q: On RGX202 data at MDA, patient numbers and FDA acceptance of evaluation framework?

A: Substantial update on functional data, CTAP analysis is supportive and FDA acceptance considered.