Regenxbio Stock Price, News & Analysis

REGENXBIO Inc. (Nasdaq: RGNX) has announced upcoming presentations of new interim data for ABBV-RGX-314 at the American Academy of Ophthalmology 2024 annual meeting. ABBV-RGX-314 is an investigational one-time AAV Therapeutic being developed in collaboration with AbbVie for treating wet age-related macular degeneration (wet AMD), diabetic retinopathy, and other chronic retinal conditions.

The presentations will include:

• New results from a bilateral dosing study using subretinal delivery for wet AMD
• Data from a Phase 2 dose-escalation study evaluating suprachoroidal delivery for diabetic retinopathy
• Long-term follow-up results from the Phase I/IIa subretinal delivery program in wet AMD

These presentations will provide insights into the potential efficacy and safety of ABBV-RGX-314 across different delivery methods and retinal conditions.

REGENXBIO Inc. (Nasdaq: RGNX) has announced its participation in Chardan's 8th Annual Genetic Medicines Conference on Monday, September 30, 2024. The event will take place in New York, NY, featuring a fireside chat at 8:30 a.m. ET.

Investors and interested parties can access a live webcast of the fireside chat through the Investors section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available for approximately 30 days following the presentation, allowing those unable to attend the live event to catch up on the discussion.

This conference participation highlights REGENXBIO's ongoing engagement with the investment community and its commitment to sharing insights into its work in the field of genetic medicines.

REGENXBIO Inc. (Nasdaq: RGNX) has appointed Mitchell Chan as Executive Vice President and Chief Financial Officer, effective September 17, 2024. Chan, a veteran finance executive with nearly 20 years of experience in the biotechnology industry, succeeds Vit Vasista, who served as CFO since 2009. Chan's extensive background includes key roles in capital raising, business development, and operations, most recently serving as Operating Partner at Catalio Capital Management. His appointment comes at a important time for REGENXBIO as the company advances its product pipeline towards commercialization.

Chan's previous experience includes serving as CFO of Viela Bio, where he oversaw a successful IPO and $3B acquisition by Horizon Therapeutics. He has also held leadership roles at AstraZeneca and Genentech-Roche. Vasista will remain as an advisor to REGENXBIO through January 3, 2025, to ensure a smooth transition.

REGENXBIO (Nasdaq: RGNX) announced positive results from the Phase I/II/III CAMPSIITE® trial of RGX-121 for treating Mucopolysaccharidosis Type II (MPS II), or Hunter syndrome. The pivotal dose level demonstrated an 85% median reduction in cerebrospinal fluid levels of heparan sulfate D2S6, a key biomarker of brain disease activity, sustained for up to two years. Notably, 80% of patients at the pivotal dose level were enzyme replacement therapy (ERT)-free at the last time point, up to 18 months post-dosing.

The trial met its primary endpoint with statistical significance, and patients exceeded expectations in neurodevelopmental function compared to natural history data. REGENXBIO plans to initiate a rolling Biologics License Application submission using the accelerated approval pathway in Q3 2024, potentially leading to a Priority Review Voucher in 2025.

REGENXBIO Inc. (Nasdaq: RGNX) has announced its participation in two upcoming investor conferences. The company will be present at the Morgan Stanley 22nd Annual Global Healthcare Conference on September 5, 2024, at 5:35 p.m. ET, and the Baird 2024 Global Healthcare Conference on September 11, 2024, at 9:40 a.m. ET. Both events will take place in New York, NY.

Investors can access live webcasts of the fireside chats from both conferences through the Investors section of REGENXBIO's website. Archived replays will be available for approximately 30 days after each presentation. This participation in high-profile healthcare conferences suggests REGENXBIO's commitment to engaging with investors and showcasing its developments in the biotechnology sector.

REGENXBIO Inc. (Nasdaq: RGNX) has announced that data from its RGX-121 program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be presented at the SSIEM 2024 Annual Symposium in Porto, Portugal from September 3-6, 2024. The presentation, titled 'CAMPSIITE™ phase I/II/III: Interim clinical update of RGX-121, an investigational gene therapy for treatment of neuronopathic mucopolysaccharidosis type II (MPS II),' will be given by Dr. Roberto Giugliani on Wednesday, September 4, 2024, at 6:15 p.m. WEST. This presentation suggests ongoing progress in REGENXBIO's gene therapy research for MPS II.

REGENXBIO Inc. (Nasdaq: RGNX) has announced its participation in the H.C. Wainwright 4th Annual Ophthalmology Virtual Conference on Thursday, August 15, 2024. The company will be part of a panel discussion titled 'The Evolving Therapeutic Landscape of AMD' scheduled for 9:00 a.m. EDT.

Interested parties can access a live webcast of the panel through the Investors section of REGENXBIO's website at www.regenxbio.com. An archived replay will be available for approximately 30 days following the presentation, providing an opportunity for those unable to attend the live event to catch up on the discussion.

REGENXBIO (Nasdaq: RGNX) reported its Q2 2024 financial results and recent operational highlights. The company is progressing its late-stage pipeline with the first Biologics License Application (BLA) expected in 2024. Key highlights include:

• Positive data from Phase II AFFINITY DUCHENNE trial of RGX-202 demonstrating high microdystrophin expression.
• End-of-Phase II meeting with FDA for RGX-202, supporting an accelerated approval pathway.
• End-of-Phase II meeting for ABBV-RGX-314 for diabetic retinopathy moved to Q4 2024.
• $327 million in cash and equivalents as of June 30, 2024, funding operations into 2026.
• Q2 revenues increased to $22.3 million, primarily due to Zolgensma royalties.
• R&D expenses decreased to $48.9 million, driven by reduced headcount and other costs.
• Net loss narrowed to $53 million, from $72.1 million in the same period last year.

REGENXBIO (RGNX) announced positive interim data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, a gene therapy for Duchenne muscular dystrophy. Key highlights include:

1. Robust microdystrophin expression observed in patients aged 5.8 and 8.5 years at 77.2% and 46.5% of control, respectively, at the pivotal dose.

2. Consistent high expression across all age groups supports plans for accelerated approval.

3. RGX-202 well-tolerated with no serious adverse events.

4. Meaningful increases in microdystrophin expression and reduction in serum creatinine kinase levels observed in all seven patients.

5. REGENXBIO plans to initiate pivotal trial in Q4 2024 and complete enrollment in dose level 2 expansion cohort in early Q3 2024.