Regenxbio Stock Price, News & Analysis

REGENXBIO has announced the dosing of the first patient in Cohort 3 of its Phase I/II trial for RGX-121, a one-time gene therapy targeting MPS II (Hunter Syndrome). This trial, involving a total of nine patients across the U.S. and Brazil, aims to assess safety and efficacy. Previous cohorts indicated positive interim results with reductions in CNS biomarkers and neurocognitive development. RGX-121 leverages the AAV9 vector for gene delivery, with potential systemic benefits. The trial's primary endpoint is establishing safety while evaluating enzyme activity and neurocognitive outcomes.

REGENXBIO reported Q4 and full-year 2020 financial results with revenues of $154.6 million, up from $35.2 million in 2019, primarily due to Zolgensma royalties. The company holds $523 million in cash and equivalents, enhanced by a recent $230 million stock offering. Key developments include active patient enrollment in pivotal trials for RGX-314 targeting wet AMD, with a BLA filing anticipated in 2024. Positive interim data from RGX-121's Phase I/II trial showcases reduced CNS biomarkers. Research on RGX-202 for Duchenne Muscular Dystrophy and other therapies continues with IND applications expected soon.

REGENXBIO Inc. (Nasdaq: RGNX) will participate in several upcoming virtual investor conferences in March 2021. The events include:

• Raymond James 42nd Annual Institutional Investors Conference: March 3, 2021, at 10:50 a.m. ET
• Barclays Global Healthcare Conference: March 9, 2021, at 4:10 p.m. ET
• Morgan Stanley Virtual Healthcare Corporate Access Day: March 16, 2021

Live webcasts for the Raymond James and Barclays events will be available on REGENXBIO's website, with archived versions accessible for 30 days post-event.

REGENXBIO Inc. (RGNX) announced a conference call scheduled for March 1, 2021, at 4:30 p.m. ET, to discuss its financial results for the fourth quarter and full year 2020, along with operational highlights. The call can be accessed via phone or through a webcast on the company's website. REGENXBIO is recognized for its innovative gene therapy solutions using its NAV Technology Platform, which includes a range of adeno-associated virus vectors aimed at therapeutic advancements across various medical fields.

On February 16, 2021, REGENXBIO announced positive interim data from the Phase I/IIa trial of RGX-314, a gene therapy for wet age-related macular degeneration (AMD). The treatment demonstrated durability over three years, with significant reductions in anti-VEGF injection burdens. Cohorts 4 and 5 showed stable visual acuity and decreased retinal thickness. Notably, 67% of patients were anti-VEGF injection-free for up to three years. The ATMOSPHERE trial, aimed at validating these findings, is currently enrolling patients. RGX-314's safety profile remains generally well-tolerated.

REGENXBIO Inc. (Nasdaq: RGNX) announced positive interim data from its Phase I/II trial of RGX-121 for treating Mucopolysaccharidosis Type II (MPS II, Hunter Syndrome) in children. Presented at the 17th Annual WORLD Symposium, the results from eight patients show a favorable safety profile and improvements in biomarker levels, indicating enhanced enzyme activity. RGX-121 aims to deliver the I2S enzyme gene via an AAV9 vector, with plans to commence patient enrollment for Cohort 3 at a higher dose in Q1 2021.

REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in the Angiogenesis, Exudation, and Degeneration 2021 conference on February 12-13, 2021. Two oral presentations will showcase advancements in gene therapy for eye diseases:

• Subretinal Gene Therapy for Exudative AMD by Allen C. Ho, M.D. on February 13 at 9:15 a.m. ET
• Suprachoroidal Gene Therapy for Exudative AMD and Diabetic Retinopathy by Peter A. Campochiaro, M.D. on February 13 at 9:45 a.m. ET

These presentations highlight the company's innovative approaches using its NAV® Technology Platform.

REGENXBIO Inc. (Nasdaq: RGNX) has announced three oral and nine poster presentations for the 17th Annual WORLD Symposium™ from February 8-12, 2021. Key presentations include interim results from the Phase I/II trial of RGX-121 targeting mucopolysaccharidosis type II (MPS II). Notable presentations cover comparative effectiveness of gene therapies and clinical updates on RGX-121. REGENXBIO is focused on advancing its NAV Technology Platform for gene therapy applications, with exclusive rights to over 100 AAV vectors.

REGENXBIO Inc. (Nasdaq: RGNX) has successfully closed its public offering of 4,260,000 common stock shares at $47.00 each, yielding approximately $230.3 million in gross proceeds after the underwriters' full option exercise for an additional 639,000 shares. This offering was managed by BofA Securities, Morgan Stanley, and Barclays. The shares were offered under a shelf registration statement effective since August 8, 2018. The funds raised will support REGENXBIO's ongoing gene therapy developments utilizing its proprietary NAV Technology Platform.