REGENXBIO Inc. reports news on its AAV gene therapy pipeline and manufacturing platform for rare, neuromuscular and retinal diseases. Recurring updates cover RGX-202 for Duchenne muscular dystrophy; clemidsogene lanparvovec (RGX-121) for MPS II; RGX-111 for MPS I; and surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy.
Company announcements also include FDA regulatory communications, clinical and preclinical data presentations, financial results, operational highlights, and collaboration activity with partners including Nippon Shinyaku and AbbVie. News on the NAV and NAVXpress platforms addresses AAV vector technology, product quality, and manufacturing capabilities used across REGENXBIO's investigational programs.
REGENXBIO announced the expansion of its Phase I/II AFFINITY DUCHENNE trial to include a new cohort of patients aged 1-3 years to evaluate the safety and efficacy of RGX-202 for Duchenne muscular dystrophy (Duchenne). The company aims to use this data in its pivotal plans and a Biologics License Application (BLA) filing.
An end-of-Phase II meeting with the FDA is scheduled for late July to finalize the pivotal program design. The company remains on track to start the pivotal trial in late Q3 to early Q4 2024.
RGX-202 is a one-time AAV Therapeutic designed to deliver a novel microdystrophin gene. It is the only gene therapy in development for Duchenne that includes the C-Terminal domain, closely mimicking the naturally occurring dystrophin gene.
No drug-related serious adverse events were observed in five patients in the ongoing trial, with positive preliminary data supporting further development. Initial data from both dose levels of the trial are expected in the second half of 2024.
REGENXBIO (Nasdaq: RGNX) has completed a successful Pre-BLA meeting with the FDA for RGX-121, targeting MPS II. The FDA is aligned on using CSF levels of heparan sulfate D2S6 as a surrogate endpoint for accelerated approval. The company plans to start a rolling BLA submission in Q3 2024, with potential FDA approval in 2025. A confirmatory trial will begin in H2 2025. The FDA confirmed the commercial bulk drug and clinical material are comparable. Positive biomarker, neurocognitive, and systemic data will support the BLA. REGENXBIO expects an FDA inspection of its Manufacturing Innovation Center in H1 2025 and aims to receive a Rare Pediatric Disease Priority Review Voucher in 2025.
REGENXBIO (Nasdaq: RGNX) announced a leadership transition effective July 1, 2024. Curran Simpson, currently COO, will become President, CEO, and Board member. Co-Founder Kenneth T. Mills steps down as CEO but will assume the Chairman role. Daniel Tassé is appointed Lead Independent Director. Simpson, a veteran executive, has been with REGENXBIO since 2015, overseeing key business functions. The company reiterated its financial guidance, expecting $380.5 million in cash to fund operations into 2026, excluding potential milestone payments from AbbVie and monetization of a review voucher.
REGENXBIO (Nasdaq: RGNX) announced its participation in the Goldman Sachs 45th Annual Global Healthcare Conference, scheduled for June 12, 2024, at 8:00 a.m. EDT in Miami, FL. The event will feature a fireside chat, accessible via live webcast on REGENXBIO's website, with an archived replay available for 30 days post-event.
REGENXBIO Inc. (Nasdaq: RGNX) will participate in upcoming investor conferences including the BofA Securities Health Care Conference and the Stifel Tailoring Genes Genetic Medicines Forum. The events will feature fireside chats and webcasts accessible through the company's website.
REGENXBIO reported its first quarter 2024 financial results and recent operational highlights, showcasing progress towards BLA filing in 2024 and pivotal trial initiations for Duchenne and diabetic retinopathy. The company announced positive interim efficacy and safety data, cash reserves of $381 million, and planned meetings with the FDA. Program highlights include advancements in neuromuscular and retinal diseases. Financially, revenues decreased to $15.6 million, with increased cash position but higher R&D and G&A expenses leading to a net loss of $63.3 million.
REGENXBIO Inc. (Nasdaq: RGNX) will host a conference call on May 8, 2024, to discuss its first quarter 2024 financial results and operational highlights. Listeners can register for the webcast to participate in the Q&A session. A replay will be available on the investor website.
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