Regenxbio (NasdaqGS:RGNX) FY Conference Transcript

Summary of REGENXBIO FY Conference Call Company Overview - Company: REGENXBIO (NasdaqGS: RGNX) - Event: FY Conference on November 10, 2025 - Speakers: CEO Curran Simpson and CMO Steve Pakola Key Points Industry and Product Development - REGENXBIO has been working on AAV (Adeno-Associated Virus) delivery for over 15 years, contributing to significant products like Zolgensma [3][4] - The company is focused on late-stage assets, with a BLA (Biologics License Application) review for the Hunter program targeting MPS II disease, with a PDUFA date set for February [3][4] - The Duchenne program has completed enrollment for its pivotal cohort, with top-line data expected in Q2 next year [4][19] - REGENXBIO is also involved in a partnership with AbbVie for the RGX-314 program targeting wet AMD, with top-line data expected by the end of 2026 [4][7] Clinical Trials and Data Expectations - The RGX-314 program is noted as one of the largest gene therapy clinical trials ever conducted, focusing on non-inferiority against existing anti-VEGF treatments [5][6] - The company aims to demonstrate a decrease in treatment burden and improved vision outcomes through its gene therapy approach [7][16] - The Duchenne program aims to replicate natural dystrophin closely, with a focus on the C-terminal domain for better efficacy [19][20] Commercial Strategy - AbbVie will lead the commercialization of the RGX-314 program, with a 50/50 profit share agreement [11][12] - REGENXBIO does not intend to build its own sales force, relying on AbbVie’s extensive commercial infrastructure [12] Market Needs and Compliance - The primary issue addressed by the RGX-314 program is patient compliance, as many patients struggle with frequent injections [13][16] - The company highlights the significant unmet need for less frequent injections, which has been shown to improve patient outcomes [15][16] Manufacturing and Cost of Goods - REGENXBIO has invested heavily in manufacturing, aiming for cost of goods to align more with biologics than traditional gene therapy [18] - The company reports good yields from its bioreactor process, which is crucial for meeting the demands of the market [18] Regulatory Interactions - The company has maintained consistent interactions with the FDA, with a focus on the Hunter program and the Duchenne protocol [24][29] - A major amendment was triggered for the Hunter program, moving the PDUFA date from November to early February [29] Safety and Efficacy - The company has implemented a targeted immunosuppression regimen to improve safety outcomes, reporting no liver injuries in treated patients [22][23] - The results from the Duchenne program have shown unexpected improvements in older patients, which is a positive indicator for the therapy's efficacy [25][27] Future Expectations - REGENXBIO is optimistic about the potential for traditional approval for the Hunter program, given the robust clinical data provided [32] - The company is also considering pursuing a Priority Review Voucher (PRV) for the Hunter program if approved before September 2026 [35] Conclusion - REGENXBIO is positioned for a significant year ahead with multiple key data readouts and regulatory milestones, focusing on innovative gene therapies that address critical unmet needs in rare diseases [3][4][32]