Regenxbio Stock Price, News & Analysis

REGENXBIO Inc. (Nasdaq: RGNX) announced positive interim data from the Phase II AAVIATE trial of RGX-314 for wet AMD, showing significant reductions in treatment burden. The trial reported an 85% reduction at the third dose level, with 67% of patients in Cohort 4 injection-free. Safety profiles were favorable with no serious drug-related adverse events. Positive long-term data from Phase I/IIa studies showed RGX-314 remains well tolerated and supports a BLA submission expected in 2024. Upcoming data from the ALTITUDE trial for diabetic retinopathy is anticipated at the Retina Society meeting in November.

REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in Chardan's 6th Annual Genetic Medicines Conference on October 4, 2022, at 1:30 p.m. ET in New York, NY. The company will conduct a fireside chat, accessible via a live webcast on its website, with an archived version available for about 30 days post-event. REGENXBIO is recognized for its gene therapy advancements, utilizing its proprietary NAV Technology Platform comprising over 100 AAV vectors. The firm aims to progress five gene therapy programs into pivotal stages or commercial products by 2025.

REGENXBIO Inc. (Nasdaq: RGNX) will present at the American Academy of Ophthalmology 2022 Annual Meeting from September 30 to October 3 in Chicago, IL. Key presentations include updates on RGX-314, an investigational gene therapy for chronic retinal conditions, including wet age-related macular degeneration and diabetic retinopathy. The company will also host a conference call on October 3 at 8:30 a.m. ET to discuss interim data from its ongoing Phase II AAVIATE trial.

REGENXBIO Inc. (Nasdaq: RGNX) has announced its participation in two key investor conferences:

• Morgan Stanley 20th Annual Global Healthcare Conference on September 14, 2022, at 2:55 p.m. ET in New York.
• UBS Biotech South Beach Conference on September 29, 2022, in Miami.

A live webcast of the Morgan Stanley conference will be available on REGENXBIO's website with a 30-day archived replay. The company is committed to advancing gene therapy through its proprietary NAV Technology Platform and aims to progress five AAV Therapeutics by 2025.

REGENXBIO (Nasdaq: RGNX) announced positive interim data for RGX-121, a gene therapy for Mucopolysaccharidosis Type II (MPS II), at the SSIEM Annual Symposium. The Phase I/II/III CAMPSIITE trial revealed significant median reductions in cerebrospinal fluid (CSF) glycosaminoglycans (GAGs) at 48 weeks, demonstrating a favorable safety profile with no serious drug-related adverse events. The company plans to file a Biologics License Application in 2024 via the accelerated approval pathway, aiming to address significant unmet needs in MPS II treatment.

REGENXBIO Inc. (Nasdaq: RGNX) announced key presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium from August 30 to September 2, 2022, in Freiburg, Germany. The focus will be on new data from the Phase I/II/III CAMPSIITE™ trial of RGX-121, a gene therapy for Mucopolysaccharidosis Type II, and RGX-111 for Mucopolysaccharidosis Type I. Notable presentations include interim analyses by Dr. Roberto Giugliani and Dr. Raymond Wang, as well as a discussion on neurodevelopment in MPS II by Dr. Maria Escolar.

REGENXBIO Inc. (Nasdaq: RGNX) will present at the H.C. Wainwright 2nd Annual Ophthalmology Virtual Conference on August 17, 2022, at 11:00 a.m. ET. The panel will focus on Unique Delivery Technology Platforms for Better Compliance and Efficacy. A live webcast will be accessible on the company's website, followed by an archived replay for 30 days. REGENXBIO is a clinical-stage biotechnology company harnessing gene therapy through its proprietary NAV Technology Platform, which includes over 100 exclusive AAV vectors, supporting its goal of advancing five AAV Therapeutics by 2025.

REGENXBIO announced its Q2 2022 financial results, reporting revenues of $32.6 million, driven by Zolgensma royalty revenues, up from $22.0 million in Q2 2021. The net loss increased to $68.2 million, or $1.58 per share, compared to $57.6 million a year ago. Key developments include progress in the RGX-314 program for wet AMD and diabetic retinopathy, with BLA filings anticipated in 2024. The company holds $682 million in cash, providing operational runway into 2025, supporting its '5x'25 strategy to have five gene therapies on the market or in late-stage development by 2025.

REGENXBIO (Nasdaq: RGNX) announced plans to file a Biologics License Application (BLA) for RGX-121 in 2024, seeking FDA's accelerated approval for treating Mucopolysaccharidosis Type II (MPS II). The pivotal CAMPSIITE trial is actively enrolling patients aged 4 months to 5 years. RGX-121, a one-time gene therapy, aims to provide a new treatment option as current therapies are inadequate. Preliminary trial results show a well-tolerated treatment with potential positive effects on neurodevelopmental function and significant reductions in glycosaminoglycans (GAGs) in cerebrospinal fluid.