REGENXBIO Inc. (Nasdaq: RGNX) has announced advancements in its gene therapy programs for Duchenne Muscular Dystrophy, Mucopolysaccharidosis Type II (MPS II), and retinal diseases as of November 6, 2024. The company is progressing towards pivotal trials and regulatory submissions, marking a significant year for its pipeline.
RGX-202 for Duchenne Muscular Dystrophy
RGX-202, a gene therapy designed to deliver a novel microdystrophin gene, is currently in a Phase I/II AFFINITY DUCHENNE trial. The last patient in the dose level 2 expansion cohort (ages 4-11) has been dosed, and the first patient in the cohort for ages 1-3 has also been dosed. REGENXBIO is the only gene therapy sponsor recruiting patients under 4 years old in the U.S.
A clinical trials application (CTA) for RGX-202 has been authorized by Health Canada, with sites expected to be initiated in Canada in H1 2025. A full program update, including the pivotal trial design, plans for accelerated approval, and initial strength and functional assessment data, is expected this month.
RGX-121 for MPS II
REGENXBIO has initiated a rolling BLA submission for RGX-121, a potential first-in-class treatment for MPS II, utilizing the accelerated approval pathway. The submission is expected to be completed in Q1 2025. Approval of RGX-121 could result in the receipt of a Priority Review Voucher in 2025.
ABBV-RGX-314 for Retinal Diseases
ABBV-RGX-314, being developed in collaboration with AbbVie, targets wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).
Diabetic Retinopathy
Based on positive interim results from the Phase II ALTITUDE trial, AbbVie and REGENXBIO have accelerated the End-of-Phase II meeting with the FDA, expected this quarter. The first global pivotal trial is anticipated to begin in H1 2025. The ALTITUDE trial is enrolling a new cohort of patients with center-involved diabetic macular edema (DME), a vision-threatening complication of DR affecting an estimated 34 million people globally. Patients will receive a one-time injection of ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye) with short course prophylactic steroid eye drops.
Wet AMD (Subretinal Delivery)
Enrollment is on track in the ATMOSPHERE and ASCENT pivotal trials, expected to support global regulatory submissions with the FDA and European Medicines Agency in H1 2026. Phase II data presented at the American Academy of Ophthalmology meeting showed that ABBV-RGX-314 was well-tolerated in the treated fellow eye, with no drug-related serious adverse events and no cases of intraocular inflammation observed. At nine months post-administration, patients saw a 97% reduction in anti-VEGF treatment burden, with sustained vision and anatomy.
Wet AMD (Suprachoroidal Delivery)
Based on a favorable safety profile, the Phase II AAVIATE trial is enrolling a new cohort to evaluate ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye) to evaluate dose levels for a planned pivotal program. Patients in this cohort will also receive short course prophylactic steroid eye drops. At dose level 3, patients receiving ABBV-RGX-314 demonstrated an 80% reduction in annualized injection rate, with 50% of patients remaining injection-free at six months. Patients also demonstrated stable Best Corrected Visual Acuity and central retinal thickness.
Financial Position
REGENXBIO reported cash, cash equivalents, and marketable securities of $278.6 million as of September 30, 2024, expected to fund operations into 2026.
