Regenxbio Stock Price, News & Analysis

REGENXBIO (Nasdaq: RGNX) reported Q4 and full-year 2022 results, showing revenues of $31.3 million for Q4 and $112.7 million for the year, a decrease from $398.7 million and $470.3 million in 2021, primarily due to reduced collaboration revenues from AbbVie. The net loss for Q4 was $59.9 million, compared to a net income of $294.0 million in Q4 2021. The cash position stood at $565.2 million, funding operations into 2025. Several pivotal trials, including RGX-314 for wet AMD and diabetic retinopathy, remain ongoing, with the anticipated BLA filing by 2024. The company aims to advance five AAV Therapeutics by 2025 under its '5x'25 strategy.

REGENXBIO (RGNX) announced positive interim data for RGX-111, a one-time gene therapy targeting severe Mucopolysaccharidosis Type I (MPS I), at the WORLD Symposium. The Phase I/II trial shows RGX-111 is well-tolerated with no serious adverse events. Key findings include positive CNS biomarker activity and encouraging neurodevelopmental gains among participants. RGX-111 is designed to deliver the IDUA gene via AAV9 vector, aiming for long-term treatment effects. The company expects to manufacture commercial-scale material for further clinical development in 2023, aligning with its '5x'25 strategy to advance multiple gene therapies.

REGENXBIO announced promising interim data from the Phase I/II/III CAMPSIITE trial for RGX-121, a potential one-time gene therapy for Mucopolysaccharidosis Type II (MPS II). Results show RGX-121's excellent tolerability with no serious adverse events reported across three dosage levels. Notably, patients in Cohort 3 exhibited significant reductions in CSF GAGs, nearing normal levels at 48 weeks. Furthermore, improvements in neurodevelopmental functions were observed up to three years post-treatment. REGENXBIO plans to file for Biologics License Application in 2024, using the accelerated approval pathway.

REGENXBIO (NASDAQ: RGNX) announced a conference call scheduled for February 28, 2023, at 4:30 p.m. ET. This call will cover the company’s financial results for Q4 and the full year ended December 31, 2022, along with recent operational highlights.

REGENXBIO, a clinical-stage biotechnology company, focuses on gene therapy through its proprietary NAV Technology Platform, which comprises over 100 novel AAV vectors. The company aims to advance five AAV Therapeutics into pivotal-stage or commercial products by 2025.

REGENXBIO Inc. (RGNX) announced new interim data from trials of its gene therapies RGX-121 and RGX-111 for MPS II and MPS I, respectively. These results will be showcased at the 19th Annual WORLD Symposium™ from February 22-26, 2023, in Orlando, Florida. The company will present three oral and eight poster presentations, including interim analyses from both clinical trials. Additionally, REGENXBIO will host a symposium session on February 24, discussing AAV gene therapy for neuronopathic MPS II. The event underscores REGENXBIO's commitment to advancing gene therapy solutions and enhancing treatment options for rare diseases.

REGENXBIO (RGNX) announced promising interim results from a Phase II bridging study of RGX-314, a gene therapy for wet AMD, using its NAVXpress manufacturing platform. The study involved 60 patients and highlighted that RGX-314 produced via this new process showed similar safety and efficacy profiles to prior methods. The company aims for these results to support Biologics License Application (BLA) submission in 2024. With no severe adverse events linked to RGX-314, the therapy may significantly reduce treatment burdens for patients. A live webcast discussing these findings occurred on February 11, 2023, showcasing advancements toward RGX-314's commercialization.

REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in the SVB Securities Global Biopharma Conference on February 16, 2023, at 12:00 p.m. ET. A live webcast will be available on the company's website, with a replay archived for 30 days. REGENXBIO is a biotechnology company focused on gene therapy, utilizing its proprietary NAV Technology Platform, which includes over 100 AAV vectors for therapeutic development. The company aims to advance five AAV Therapeutics into pivotal or commercial stages by 2025, under its "strategy.

This engagement highlights the company's commitment to innovation and progress in biotechnology.

REGENXBIO Inc. (NASDAQ: RGNX) announced three key presentations on RGX-314 at the Angiogenesis, Exudation, and Degeneration 2023 Conference, scheduled for February 10-11, 2023. These presentations will showcase data from a Phase II bridging study assessing RGX-314's pharmacodynamics, safety, and efficacy for treating neovascular age-related macular degeneration (AMD), utilizing cGMP material from the NAVXpress™ platform. Presenters include Dr. Charles Wykoff, Dr. Allen Ho, and Dr. Peter Campochiaro, highlighting long-term follow-up and new delivery methods for AMD and diabetic retinopathy, which could support RGX-314's future commercialization.

REGENXBIO has commenced the Phase I/II AFFINITY DUCHENNE trial for RGX-202 to treat Duchenne muscular dystrophy. This innovative gene therapy utilizes a novel microdystrophin and the NAV® AAV8 vector, aiming to improve muscle strength in affected boys. The trial will evaluate safety, tolerability, and efficacy with a focus on pediatric patients aged 4 to 11. Additionally, REGENXBIO is enrolling patients for the observational AFFINITY BEYOND study, which aims to assess AAV8 antibody prevalence. This initiative aligns with the company's '5x'25' strategy to advance five gene therapies by 2025, highlighting a commitment to addressing unmet medical needs in the Duchenne community.