REGENXBIO(US:RGNX)2025-11-06 14:00Financial Data and Key Metrics Changes - REGENXBIO ended Q3 2025 with cash, cash equivalents, and marketable securities of $302 million, an increase from $245 million as of December 31, 2024, primarily driven by a $110 million upfront payment from Nippon Shinyaku and $145 million in net proceeds from royalty monetization [17][18] - Revenues for Q3 2025 were $30 million, compared to $24 million in Q3 2024, mainly due to development service revenue under the Nippon Shinyaku partnership [17] Business Line Data and Key Metrics Changes - The RGX-202 program for Duchenne muscular dystrophy is progressing well, with enrollment completed in the Affinity Duchenne Pivotal Trial, and top-line pivotal data expected in early Q2 2026 [5][11] - RGX-121, a potential gene therapy for MPS II, is on track for FDA approval by early 2026, with positive 12-month data delivered to the FDA [8][14] - The retinal disease franchise, particularly the ABBV-RGX-314 program for wet AMD, has completed enrollment in two global phase 3 studies, representing the largest global gene therapy program ever conducted [9][10] Market Data and Key Metrics Changes - The anticipated market for RGX-202 is significant, with the ability to produce 2,500 doses per year, positioning the company for clinical and commercial success [7] - The prevalent market for Duchenne is expected to be around 14,000 patients by 2027, with approximately 3,000 eligible for gene therapy [82] Company Strategy and Development Direction - The company is focused on advancing its late-stage pipeline of gene therapies, with a commitment to bringing new medicines to patients in need [4] - REGENXBIO is preparing for a commercial launch of RGX-202 in 2027 and is exploring opportunities to expand the program outside the U.S. [6][14] - The company aims to leverage its manufacturing capabilities and innovative science to deliver best-in-class therapeutics [10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing BLA review for RGX-121, highlighting productive interactions with the FDA and a strong safety profile [8][32] - The company anticipates a transformational year ahead, with multiple product launches and significant unmet needs being addressed by its gene therapies [19] Other Important Information - The company has a strong financial position, with cash runway expected to extend into early 2027, not including potential non-dilutive financing opportunities [18] - The manufacturing facility in Rockville is capable of producing high-purity gene therapies, which is crucial for commercial readiness [7][35] Q&A Session Summary Question: Update on RGX-202 interactions with FDA - Management indicated that top-line data will be available in early Q2 2026, with a pre-BLA meeting expected around that time [21][23] Question: Cash runway and non-dilutive financing - The company expects non-dilutive financing options to extend cash runway well into 2027 or early 2028 [24][25] Question: Confirmatory trial enrollment for DMD - Enrollment for the confirmatory study has begun, and management expects substantial progress by mid-2026 [26][27] Question: Regulatory interactions for DMD and MPS II - Management confirmed a late cycle meeting with the FDA for RGX-121 and a pre-BLA meeting for RGX-202 is anticipated [30][32] Question: Manufacturing capacity and production volume - The Rockville site has a 2,000-liter bioreactor, capable of producing up to 2,500 doses of RGX-202 annually [34][35] Question: Use of FDA natural history as a control in DMD - Management confirmed that propensity score matching is prospectively specified in the protocol for RGX-202 [38][40] Question: Interest in gene therapy from retina specialists - There is significant excitement about gene therapy among retina specialists, with half of surveyed specialists expressing interest in gene therapy approaches [42][44] Question: Expectations for black box warnings - Management does not expect a black box warning for RGX-202 due to its strong safety profile [48][49] Question: EMA plans and requirements - The company is evaluating the EMA's requirements for RGX-121 and RGX-202, with ongoing discussions about potential placebo control needs [52][54] Question: Diabetic retinopathy study adjustments - The company is considering the ordinal two-step DRSS change as a potential primary endpoint for their pivotal studies [58][61] Question: Enrollment for suprachoroidal delivery program - The company is looking to enroll 20 patients in the suprachoroidal delivery arm, with expectations for increased enrollment speed following the completion of the subretinal program [63][66]