Regenxbio (RGNX) Stock News, Apr-Jul 2025

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.

REGENXBIO (RGNX) reported significant progress in Q1 2025, with a net income of $6.1 million ($0.12 per share) compared to a loss of $63.3 million in Q1 2024. Revenue surged to $89.0 million, up from $15.6 million year-over-year, largely due to the Nippon Shinyaku partnership. The company's cash position strengthened to $272.7 million, expected to fund operations into H2 2026. Key pipeline developments include: RGX-202 for Duchenne muscular dystrophy advancing toward BLA submission in mid-2026, clemidsogene lanparvovec's BLA acceptance expected May 2025, and surabgene lomparvovec's pivotal data for wet AMD expected in 2026. The Nippon Shinyaku partnership brought a $110 million upfront payment with potential for $700 million in additional milestone payments.

REGENXBIO (RGNX) will present multiple studies at the American Society of Gene & Cell Therapy 28th Annual Meeting in New Orleans from May 13-17, 2025. The presentations will showcase the company's gene therapy capabilities across research, development, and manufacturing. Key highlights include:

- Late-stage clinical trial data for RGX-121 (clemidsogene lanparvovec) for MPS II treatment and RGX-202 for Duchenne muscular dystrophy

The company will deliver three oral presentations focusing on: • Commercial manufacturing process for RGX-202 • RGX-121 gene therapy for neuronopathic mucopolysaccharidosis • RGX-202 interim clinical data for Duchenne muscular dystrophy

Additionally, five poster presentations will cover various aspects of AAV vector development, testing methods, and blood-brain barrier crossing vectors.

REGENXBIO (RGNX) has announced its participation in multiple upcoming investor conferences throughout May and June 2025. The company will be present at five major healthcare and biotech events:

- BofA Securities Health Care Conference in Las Vegas (May 13)
- RBC Global Healthcare Conference in New York (May 20)
- H.C. Wainwright BioConnect in New York (May 20)
- Stifel Virtual Ophthalmology Forum (May 27)
- UBS Spring Biotech Conference in New York (June 24)

Select presentations and fireside chats will be available via webcast on REGENXBIO's website, with recordings accessible for approximately 30 days after each event.

REGENXBIO (Nasdaq: RGNX) has scheduled a conference call for Monday, May 12, at 4:30 p.m. ET to present its financial performance for the first quarter ending March 31, 2025, along with recent operational updates.

The company has provided two access options for interested parties:

• General listeners can join via a webcast link
• Analysts can participate in the Q&A session through a separate dedicated link

A recording of the webcast will be made available on REGENXBIO's investor website approximately two hours after the call concludes. Participants are encouraged to connect 15 minutes before the scheduled start time.