Regenxbio

Four innovative therapies are advancing through clinical trials to address critical unmet needs in MPS I treatment, particularly targeting central nervous system complications that current therapy Aldurazyme cannot reach.

Satellos Bioscience's investigational treatment for Duchenne muscular dystrophy (DMD) has demonstrated encouraging efficacy signals in adult patients, offering potential new options in a challenging therapeutic area.

• REGENXBIO has closed a non-dilutive royalty bond agreement with Healthcare Royalty for up to $250 million, receiving $150 million upfront that extends its cash runway into early 2027. • The deal monetizes select royalties from ZOLGENSMA for SMA and payments from gene therapies for MPS disorders, while REGENXBIO retains other funding opportunities including a potential Priority Review Voucher. • This strategic financing supports REGENXBIO's late-stage pipeline development, including RGX-121 for MPS II, RGX-202 for Duchenne muscular dystrophy, and ABBV-RGX-314 for wet AMD.

• Phase I/II Deliver trial demonstrates sustained functional improvements in DMD patients treated with DYNE-251, showing mean absolute dystrophin expression of 8.72% above baseline at six months with the 20mg/kg dose. • The therapy, designed for patients amenable to exon 51 skipping, showed improvements across multiple functional endpoints including mobility assessments, positioning Dyne for potential accelerated FDA approval submission in early 2026. • If approved, DYNE-251 could generate significant market impact, with GlobalData projecting revenue growth from $5 million in 2025 to $129 million by 2030 in the competitive DMD treatment landscape.

REGENXBIO and Nippon Shinyaku have entered an exclusive partnership to develop and commercialize RGX-121 for MPS II and RGX-111 for MPS I.

• Phase 2 study of ABBV-RGX-314 shows a 97% reduction in annualized anti-VEGF treatment burden in patients with bilateral wet AMD. • 78% of patients were completely injection-free at 9 months post-administration of ABBV-RGX-314 in the fellow eye. • The study demonstrated sustained best-corrected visual acuity (BCVA) and central retinal thickness (CRT) at 9 months. • ABBV-RGX-314 was well-tolerated, with no drug-related serious adverse events observed in the treated fellow eye.

REGENXBIO has dosed the first patient in the Phase 3 AFFINITY DUCHENNE trial evaluating RGX-202 for Duchenne muscular dystrophy (DMD).

REGENXBIO's RGX-202, a gene therapy for Duchenne muscular dystrophy (DMD), has advanced to a pivotal Phase III trial after positive Phase I/II results.

• Regenxbio has secured an accelerated approval pathway from the FDA for its experimental gene therapy, RGX-202, targeting Duchenne muscular dystrophy. • The pivotal Phase 1/2 trial will assess RGX-202's ability to produce at least 10% of normal microdystrophin levels after three months in Duchenne patients. • Unlike previous trials, Regenxbio's study will not include a placebo comparator, focusing on microdystrophin production as the primary endpoint. • Early data suggests RGX-202 may offer enhanced benefits compared to existing Duchenne gene therapies like Sarepta's Elevidys, showing higher microdystrophin expression.

• Regenxbio's RGX-202, a gene therapy for Duchenne muscular dystrophy, has secured FDA agreement for an accelerated approval pathway based on an ongoing Phase 1/2 trial. • The pivotal trial will assess RGX-202 in approximately 30 Duchenne patients, focusing on achieving at least 10% of normal microdystrophin levels after three months. • Early data suggests RGX-202 may offer enhanced benefits compared to existing Duchenne gene therapies, showing higher microdystrophin expression levels in treated patients. • The FDA's decision signals continued openness to streamlined approvals for microdystrophin-based gene therapies, potentially benefiting other Duchenne gene therapy developers.