Regenxbio Stock Price, News & Analysis

REGENXBIO has announced positive interim data from the ongoing Phase I/II trial of RGX-121, a gene therapy for Mucopolysaccharidosis Type II (MPS II). Key findings include no serious drug-related adverse events and evidence of CNS activity in patients up to two years post-administration. Biomarkers indicate strong I2S enzyme activity, and most patients showed continued neurocognitive development. The company has initiated dosing in Cohort 3 at an increased dosage, further enhancing prospects for RGX-121 as a treatment option for MPS II.

REGENXBIO Inc. (Nasdaq: RGNX) will present at three upcoming virtual investor conferences in May 2021. The events include:

• Bank of America Securities 2021 Virtual Health Care Conference on May 12, 2021, at 12:30 p.m. E.T.
• RBC Capital Markets 2021 Global Healthcare Virtual Conference on May 18, 2021, at 10:55 a.m. E.T.
• UBS Global Healthcare Virtual Conference on May 25, 2021, at 11:00 a.m. E.T.

Live webcasts will be accessible on the REGENXBIO website, with replays available for 30 days.

REGENXBIO reported Q1 2021 financial results with revenues increasing to $18.9 million from $17.6 million year-over-year, primarily driven by a rise in Zolgensma royalties. The company holds $657 million in cash and equivalents, sufficient for operations into late 2023. Progress in clinical trials includes ongoing enrollment in pivotal trials for RGX-314 for wet AMD and advancements in RGX-121 and RGX-111 programs. Net loss widened to $50.1 million compared to $40.0 million in Q1 2020, reflecting increased R&D investments.

REGENXBIO Inc. (Nasdaq: RGNX) will host a conference call on May 5, 2021 at 4:30 p.m. ET to discuss its financial results for the quarter ended March 31, 2021, along with recent operational highlights. Investors can join the call by dialing (855) 422-8964 or (210) 229-8819 for international access, with the passcode 7044809. A live or recorded webcast will also be available on their website for about 30 days after the call. REGENXBIO focuses on gene therapy using its proprietary NAV^® Technology Platform with over 100 AAV vectors.

REGENXBIO Inc. (Nasdaq: RGNX) announced that it will present four oral and nine poster presentations at the American Society of Gene and Cell Therapy's 24th Annual Meeting, scheduled for May 11-14, 2021. Oral presentations feature topics such as AAV tissue transduction and the RGX-121 gene therapy for mucopolysaccharidosis type II. The poster presentations cover a range of subjects including AAV vector evaluation and gene therapy methodologies. Detailed information about the presentations can be found on the ASGCT website.

REGENXBIO Inc. (Nasdaq: RGNX) announced that Curran M. Simpson, Chief Operations and Technology Officer, will present at the Chardan 5th Annual Genetic Medicines Manufacturing Summit on April 27, 2021, at 3:15 p.m. ET. The event will be held virtually. A webcast of the presentation will be available in the Investors section of REGENXBIO's website and can be accessed at www.regenxbio.com. An archived replay will be accessible for 30 days post-event.

REGENXBIO is focused on advancing gene therapy using its NAV® Technology Platform.

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.