Regenxbio (NasdaqGS:RGNX) Q4 2025 Earnings call March 05, 2026 08:00 AM ET Company ParticipantsCurran Simpson - President and CEODaniel Connell - Account ManagerMitchell Chan - EVP and CFOPatrick Christmas II - Chief Legal OfficerSteve Pakola - Chief Medical OfficerConference Call ParticipantsAlec Stranahan - VP and Senior Equity Research AnalystAnnabel Samimy - Md and Senior Equity Research AnalystBrian Skorney - Md and Senior Research AnalystEduardo Martinez-Montes - Equity Research AnalystJudah Frommer - ...

Financial Data and Key Metrics Changes - REGENXBIO ended the quarter on December 31, 2025, with cash equivalents and marketable securities of $241 million, a slight decrease from $245 million as of December 31, 2024 [18] - R&D expenses increased to $228 million for the year ended December 31, 2025, compared to $209 million in 2024, primarily due to pivotal trial execution and manufacturing costs [19] - Total annual revenue was reported at $170 million, which includes upfront license revenue and an increase in royalty revenue [19][20] Business Line Data and Key Metrics Changes - The RGX-202 program for Duchenne muscular dystrophy (DMD) showed strong enrollment and positive functional outcomes, with an average improvement of 7.4 compared to the recognized CTAP model at 18 months [11][12] - The RGX-314 franchise for wet AMD and diabetic retinopathy is advancing, with enrollment completed in pivotal studies ATMOSPHERE and ASCENT, and a 93% reduction in annualized anti-VEGF injection need reported [14][15] - The NAVIGATE pivotal study for diabetic retinopathy is set to begin patient dosing next quarter, triggering a $100 million milestone from AbbVie [9][15] Market Data and Key Metrics Changes - The company is focusing on large indications such as Duchenne, wet AMD, and diabetic retinopathy, which represent significant commercial opportunities [22] - The unmet need in the Duchenne community remains significant, with less than 1% of the global population having received an approved gene therapy [7] Company Strategy and Development Direction - REGENXBIO aims to transition from a late-stage development organization to a commercial entity by advancing its late-stage pipeline and focusing on execution [6][22] - The company is committed to addressing clinical holds for RGX-111 and RGX-121 while continuing to support the MPS community [10][21] - The strategy includes leveraging in-house manufacturing and partnerships to enhance the treatment landscape [22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming pivotal readouts and the potential for accelerated approval pathways for RGX-202 [5][26] - The company is optimistic about its safety profile and the compelling data from its clinical trials, which are expected to support its regulatory submissions [28][37] - Management acknowledged the importance of ongoing dialogue with the FDA to ensure alignment on data analysis methods and submission expectations [90] Other Important Information - The company has received clinical hold letters for RGX-111 and RGX-121 but believes the requirements to remove the holds are addressable [9] - REGENXBIO's cash runway is expected to extend into early 2027, not including anticipated milestones and additional funds from collaborations [20][21] Q&A Session Summary Question: Concerns about control trials for DMD - Management expressed confidence in the design of the confirmatory study and the compelling data supporting their approach to accelerated approval [25][26] Question: Potential longer duration follow-up for FDA - Management indicated that they would have 12-month data on the pivotal dataset and that the confirmatory study would provide additional safety data [35][36] Question: Update on RGX-202 data presentation - Management confirmed that the upcoming presentation would include additional functional data and safety outcomes [60][61] Question: Safety profile for DMD - Management reported no cases of liver injury or thrombocytopenia in the RGX-202 study, highlighting a differentiated safety profile compared to existing therapies [69] Question: Heparan sulfate data in MPS I - Management acknowledged the importance of heparan sulfate measurements and indicated that they would provide more detailed results in response to the CRL [81][82]

Financial Data and Key Metrics Changes - REGENXBIO ended Q4 2025 with cash equivalents and marketable securities of $241 million, a slight decrease from $245 million at the end of 2024, reflecting cash used for operating activities despite significant upfront payments and royalty monetization [17] - R&D expenses increased to $228 million for the year ended December 31, 2025, compared to $209 million in 2024, primarily due to pivotal trial execution and manufacturing costs [18] - Total annual revenue for 2025 was $170 million, including upfront license revenue and increased royalty revenue from Zolgensma and Evrysdi [18] Business Line Data and Key Metrics Changes - The RGX-202 program for Duchenne muscular dystrophy (DMD) showed robust enrollment and positive safety and efficacy data, with an average improvement of 7.4 points in the NSAA compared to the CTAP model at 18 months [10][11] - The RGX-314 franchise for wet AMD and diabetic retinopathy is advancing, with enrollment completed in pivotal studies ATMOSPHERE and ASCENT, and a 93% reduction in annualized anti-VEGF injection need reported [12][13] - The company is addressing clinical holds for RGX-111 and RGX-121, with plans to resubmit the BLA after addressing the requirements [8][9] Market Data and Key Metrics Changes - The company is entering pivotal phase IIb/III programs for diabetic retinopathy in collaboration with AbbVie, with first patient dosing expected next quarter [8] - Top-line data for subretinal Surrovac in wet AMD is anticipated in Q4 2026, which would mark the first gene therapy for this indication if approved [12] Company Strategy and Development Direction - REGENXBIO aims to transition from a late-stage development organization to a commercial entity, focusing on executing key milestones and advancing its late-stage pipeline [4][20] - The company is committed to addressing unmet needs in rare diseases, particularly in the Duchenne and MPS communities, and is investing in in-house manufacturing capabilities [20][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming pivotal readouts and the potential to enhance treatment landscapes for DMD, wet AMD, and diabetic retinopathy, which represent significant commercial opportunities [20] - The company is preparing for discussions with the FDA regarding its BLA submission strategy, emphasizing the importance of functional data in supporting its case [30][31] Other Important Information - The company received a $100 million milestone payment from AbbVie upon first patient dosing in the NAVIGATE study, which is expected to extend its cash runway into the second half of 2027 [19] - Management highlighted the importance of ongoing dialogue with the FDA to ensure alignment on data analysis methods and regulatory expectations [87] Q&A Session Summary Question: Concerns about controlled trials for DMD - Management expressed confidence in the study design and FDA engagement, noting that the protocol was reviewed and approved by the FDA, and emphasized the compelling data from older patients [25][26] Question: Duration follow-up for safety in DMD - Management indicated that the confirmatory study would provide additional safety data, and they are prepared to discuss this with the FDA [34] Question: Moving forward with RGX-314 - Management clarified that the decision to proceed with Dose Level 3 was based on compelling results and safety data, and they are continuing to evaluate higher doses for wet AMD [41][44] Question: Functional data expectations - Management stated that they expect to provide top-line functional data for approximately seven patients at the 12-month mark, emphasizing the importance of this data for regulatory discussions [51][52] Question: Heparan sulfate data in MPS I - Management confirmed that they have been measuring heparan sulfate and will provide detailed results in response to the CRL, emphasizing the importance of this biomarker in their discussions with the FDA [78][79]