Regenxbio Stock Price, News & Analysis

REGENXBIO (NASDAQ:RGNX) reported Q2 2025 financial results and significant progress across its gene therapy pipeline. The company's cash position strengthened to $363.6 million, expected to fund operations into early 2027. Key developments include accelerated enrollment in the RGX-202 Duchenne muscular dystrophy pivotal trial, expected to complete by October 2025, with BLA submission planned for mid-2026.

The company reported Q2 2025 revenues of $21.4 million and a net loss of $70.9 million ($1.38 per share). Notable achievements include successful FDA inspections for clemidsogene lanparvovec (RGX-121) for MPS II, with a PDUFA date of November 9, 2025, and advancement of surabgene lomparvovec (sura-vec) for retinal diseases. The company secured a $250 million royalty agreement with Healthcare Royalty and amended its AbbVie collaboration with potential milestone payments of $200 million.

REGENXBIO (NASDAQ:RGNX) announced the advancement of surabgene lomparvovec (sura-vec) into a pivotal Phase IIb/III clinical trial for diabetic retinopathy (DR) using suprachoroidal delivery. The decision follows positive two-year data from the Phase II ALTITUDE® trial, demonstrating durable safety and efficacy with a single injection.

Under an amended collaboration with AbbVie, REGENXBIO will receive $100 million upon first subject dosing in the Phase IIb/III trial and another $100 million for the first subject in a second Phase III trial. The Phase II ALTITUDE trial showed favorable safety results, with no drug-related serious adverse events or intraocular inflammation observed through two years at dose level 3.

REGENXBIO (Nasdaq: RGNX) has scheduled a conference call for Thursday, August 7, 2025, at 8:00 a.m. ET to discuss its second quarter 2025 financial results and recent operational developments. The company will host a webcast that can be accessed through a registration link.

Analysts can participate in the Q&A session by dialing (646) 307-1963 (domestic) or (800) 715-9871 (international) with passcode 9571992. A replay will be available on REGENXBIO's investor website approximately two hours after the call concludes. Participants are encouraged to join 15 minutes before the start time.

REGENXBIO (NASDAQ:RGNX) has published significant preclinical results for RGX-202, their novel gene therapy for Duchenne Muscular Dystrophy. The research, published in Molecular Therapy Methods and Clinical Development, demonstrates that their unique microdystrophin construct containing the C-terminal (CT) domain shows superior performance compared to versions without it.

The studies in mdx mice revealed that the CT domain-containing construct achieved higher protein levels, increased muscle force, and improved resistance to damage. RGX-202 is currently the only microdystrophin gene therapy candidate that includes the CT domain, making it closest to natural dystrophin. These findings support positive interim results from the ongoing Phase I/II AFFINITY DUCHENNE® trial, with REGENXBIO planning a BLA submission in mid-2026 using the accelerated approval pathway.

REGENXBIO (RGNX) reported positive interim data from its Phase I/II AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy. At dose level 2, patients showed significant functional improvements, exceeding natural history controls across all measures. At 12 months, patients improved by 4.5 points on NSAA from baseline, outperforming natural history by 6.8 points. A new 2-year-old patient demonstrated 118.6% microdystrophin expression compared to control. The therapy maintained a favorable safety profile with no serious adverse events. The pivotal trial is enrolling approximately 30 patients, with over half already enrolled, targeting a BLA submission in mid-2026 under accelerated approval.

REGENXBIO (RGNX) has announced a webcast scheduled for June 5, 2025, at 8:00 a.m. EDT to present new interim functional data from their Phase I/II AFFINITY DUCHENNE trial of RGX-202, a next-generation gene therapy being developed for Duchenne muscular dystrophy. The presentation will feature principal investigator Dr. Aravindhan Veerapandiyan from Arkansas Children's Hospital. The webcast will be accessible through REGENXBIO's website, with a replay available for approximately 30 days after the event.

REGENXBIO (NASDAQ: RGNX) has secured a strategic royalty monetization agreement with Healthcare Royalty (HCRx) worth up to $250 million. The company received $150 million upfront, extending its cash runway into early 2027. The non-dilutive agreement includes rights to royalties from ZOLGENSMA sales and milestone payments from various partnerships.

Additional funding includes $50 million tied to ZOLGENSMA sales milestones (by April 2027) and another $50 million upon mutual agreement. HCRx will receive quarterly interest payments from royalty revenue and warrants to purchase 268,096 shares at $14.92. REGENXBIO retains rights to other funding sources, including a potential Priority Review Voucher for RGX-121 and milestones from AbbVie and Nippon Shinyaku.

The FDA has accepted REGENXBIO's (NASDAQ: RGNX) Biologics License Application for RGX-121, a pioneering gene therapy targeting Mucopolysaccharidosis II (MPS II). The application received Priority Review status with a PDUFA target date of November 9, 2025. This development follows a strategic partnership formed in January 2025 between REGENXBIO and Nippon Shinyaku, with NS Pharma (Nippon Shinyaku's subsidiary) set to handle U.S. commercialization upon approval. The partnership also includes RGX-111, a treatment for MPS I. If approved, RGX-121 would represent a first-in-class gene therapy option for MPS II patients.

The FDA has accepted and granted Priority Review for REGENXBIO's (NASDAQ: RGNX) Biologics License Application (BLA) for RGX-121, a potential first-in-class gene therapy for Hunter syndrome (MPS II). The FDA has set a PDUFA target action date of November 9, 2025.

RGX-121 has received multiple FDA designations including Orphan Drug Product, Rare Pediatric Disease, Fast Track, and RMAT. Under a partnership announced in January 2025, NS Pharma (Nippon Shinyaku subsidiary) will lead U.S. commercialization post-approval, while REGENXBIO maintains commercial manufacturing and supply chain responsibilities. If approved, REGENXBIO would retain rights to a potential Priority Review Voucher.

The one-time treatment aims to address both neurodevelopmental and systemic effects of Hunter syndrome, potentially replacing current weekly enzyme replacement therapy.