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REGENXBIO Inc. (Nasdaq: RGNX) is a pioneering biotechnology company dedicated to transforming patient lives through the development and commercialization of innovative gene therapies. The company's core focus lies in the use of recombinant adeno-associated virus (AAV) gene therapy, leveraging its proprietary NAV® Technology Platform. This platform includes exclusive rights to over 100 novel AAV vectors, such as AAV7, AAV8, AAV9, and AAVrh10, which are crucial for delivering therapeutic genes. REGENXBIO aims to address serious diseases with significant unmet medical needs through both in-house development and third-party licensing agreements.
REGENXBIO's mission is realized through its robust pipeline, which targets various challenging conditions:
- Metabolic Diseases: The company is working on therapies for homozygous familial hypercholesterolemia.
- Neurodegenerative Conditions: Their efforts include treatments for mucopolysaccharidosis (MPS), specifically Type II (Hunter syndrome).
- Retinal Diseases: Projects include therapies for wet age-related macular degeneration (wet AMD) and X-linked retinitis pigmentosa.
REGENXBIO has several high-profile partnerships. Collaborations with AbbVie are advancing ABBV-RGX-314, a potential one-time treatment for wet AMD and diabetic retinopathy. They also have licensed their technology to renowned firms like Novartis and Eli Lilly, which are using it to develop therapies for spinal muscular atrophy and various neurodegenerative diseases, respectively.
Recent Achievements and Financial Highlights:
- Completion of enrollment at dose level 2 in the Phase I/II AFFINITY DUCHENNE® trial for RGX-202, aimed at treating Duchenne muscular dystrophy.
- Topline results from the CAMPSIITE® trial for RGX-121, targeting MPS II, showing significant improvement in brain function biomarkers.
- Publication of ABBV-RGX-314 trial results in The Lancet, demonstrating long-term efficacy for wet AMD.
Financial Condition: As of March 31, 2024, REGENXBIO has a strong cash position, with $380.5 million in cash, cash equivalents, and marketable securities. Despite a net loss of $63.3 million for Q1 2024, the company expects its cash reserves to fund operations into 2026, bolstered by proceeds from a recent public offering and potential milestone payments from partners.
REGENXBIO continues to make significant strides in its
REGENXBIO Inc. (Nasdaq: RGNX) has announced a conference call scheduled for Thursday, August 1, 2024, at 4:30 p.m. ET. The purpose of this call is to discuss the company's financial results for the second quarter ended June 30, 2024, along with recent operational highlights. Interested parties can access the live call by phone or via webcast on the REGENXBIO website. For phone access, dial (646) 307-1963 (domestic) or (800) 715-9871 (international) with passcode 4849384. The recorded webcast will be available for approximately 30 days after the call. Participants are advised to join 15 minutes before the start time.
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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