Regenxbio updates on Duchenne muscular dystrophy, MPS II, and retinal disease gene therapy trials.

Regenerative Medicine Group is on track to report topline results and submit a BLA for RGX-202 in Duchenne muscular dystrophy by 1H 2026. Enrollment for the pivotal trial is expected to complete in October 2025, ahead of previous guidance. Clemidsogene lanparvovec (RGX-121) is on track to be the first gene therapy for MPS II, with successful FDA inspections of the company's manufacturing facility and laboratory practices. Surabgene lomparvovec (sura-vec, ABBV-RGX-314) is also advancing, with pivotal data expected in 2026 for wet AMD and a global pivotal program for diabetic retinopathy.

Regenerative Medicine Group (RGNX), a leader in gene therapy, has made significant strides in its pipeline, positioning itself for key regulatory milestones. The company recently reported its second-quarter 2025 financial results, highlighting progress in its gene therapy programs for Duchenne muscular dystrophy (DMD), MPS II, and retinal diseases.

Duchenne Muscular Dystrophy (DMD)

RGNX's RGX-202, a potential best-in-class gene therapy for DMD, is on track to report topline results in the first half of 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026. Enrollment for the pivotal AFFINITY DUCHENNE trial is accelerating, expected to complete in October 2025, ahead of previous guidance. Positive Phase I/II data demonstrated the potential of RGX-202, with all dose level 2 participants exceeding natural history controls on functional measures and microdystrophin expression above the 10% threshold compared to normal [1].

MPS II

Clemidsogene lanparvovec (RGX-121), a potential first-in-class treatment for MPS II, also known as Hunter syndrome, is on track to be the first gene therapy for this condition. The U.S. Food and Drug Administration (FDA) granted priority review of the BLA seeking accelerated approval for RGX-121 for MPS II, with a Prescription Drug User Fee Act (PDUFA) target action date of November 9, 2025. Successful FDA inspections of the company's manufacturing facility and laboratory practices have been completed with no observations [2].

Retinal Diseases

Surabgene lomparvovec (sura-vec, ABBV-RGX-314), developed in collaboration with AbbVie, is potentially the first-in-class treatment for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR). Pivotal data evaluating the safety and efficacy of the subretinal delivery of sura-vec in patients with wet AMD are expected in 2026. The company is also advancing a global pivotal program for diabetic retinopathy, supported by positive Phase II trial data [2].

Financial Highlights

Financially, RGNX reported a net loss of $70.9 million ($1.38 per share) compared to $53.0 million ($1.05 per share) in Q2 2024. The company strengthened its balance sheet through a $250 million royalty bond agreement with Healthcare Royalty, receiving $150 million upfront. Cash position improved to $363.6 million from $244.9 million at year-end 2024, extending runway into early 2027 [1].

References

[1] https://www.stocktitan.net/news/RGNX/regenxbio-reports-second-quarter-2025-financial-results-and-nsiviizq1oqn.html
[2] https://www.prnewswire.com/news-releases/regenxbio-reports-second-quarter-2025-financial-results-and-operational-highlights-302523955.html