Welcome to our dedicated page for REGENXBIO news (Ticker: RGNX), a resource for investors and traders seeking the latest updates and insights on REGENXBIO stock.
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 and Nippon Shinyaku have announced a strategic partnership for developing and commercializing RGX-121 and RGX-111, gene therapies for MPS II (Hunter syndrome) and MPS I (Hurler syndrome) respectively. The deal includes a $110 million upfront payment to REGENXBIO, with potential milestone payments of up to $700 million ($40M in development/regulatory milestones and $660M in sales milestones).
REGENXBIO will receive double-digit royalties on net sales in the U.S. and Asia, lead manufacturing operations, and retain rights to the RGX-121 Priority Review Voucher. Nippon Shinyaku will lead commercialization efforts in these territories. RGX-121's FDA approval is anticipated by late 2025, with a rolling BLA submission currently underway. The transaction is expected to close by Q1 2025.
AbbVie (ABBV) and REGENXBIO (RGNX) have announced key updates for their ABBV-RGX-314 clinical program. The companies expect pivotal data on the safety and efficacy of subretinal delivery for wet age-related macular degeneration (wet AMD) patients in 2026 through the ATMOSPHERE® and ASCENT™ trials.
Additionally, both companies will plan a Phase 3 clinical program for diabetic retinopathy (DR) treatment using suprachoroidal delivery via the SCS Microinjector®. This gene therapy aims to address the treatment burden of chronic, frequent dosing in retinal diseases, which are among the leading causes of blindness. ABBV-RGX-314 is being developed as the first potential one-time gene therapy for both wet AMD and DR patients.
REGENXBIO has initiated the pivotal phase of AFFINITY DUCHENNE® trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, with FDA alignment on accelerated approval pathway and expected BLA in 2026. The trial reports positive functional data across both dose levels, with improvements in North Star Ambulatory Assessment scores and timed function tests. New biomarker data shows consistent robust expression of RGX-202 microdystrophin in muscle tissue, with the highest reported levels among gene therapies. The pivotal trial will evaluate approximately 30 ambulatory patients aged 1 and older, with no serious adverse events reported to date.
REGENXBIO (Nasdaq: RGNX) has announced a webcast to discuss the AFFINITY DUCHENNE® pivotal program and present first functional data from their Phase I/II study of RGX-202, a next-generation gene therapy for Duchenne muscular dystrophy. The webcast, scheduled for November 18, 2024, will feature principal investigator Dr. Aravindhan Veerapandiyan from Arkansas Children's Hospital and Dr. Michael Kelly, Chief Scientific Officer of CureDuchenne.
The findings will also be presented at the American Society of Gene and Cell Therapy and Muscular Dystrophy Association 2024 Breakthroughs conference in Chicago on November 19, 2024.
REGENXBIO reported Q3 2024 financial results with revenues of $24.2 million, down from $28.9 million in Q3 2023. The company posted a net loss of $59.6 million ($1.17 per share). Cash position stood at $278.6 million, expected to fund operations into 2026.
Key operational updates include advancement in Phase I/II AFFINITY DUCHENNE trial of RGX-202, BLA submission initiation for RGX-121 (expected completion in Q1 2025), and positive Phase II data for ABBV-RGX-314. The company accelerated End-of-Phase II meeting for ABBV-RGX-314 in diabetic retinopathy to Q4 2024, with global pivotal program initiation planned for H1 2025.
REGENXBIO (Nasdaq: RGNX) has announced its participation in three major healthcare investor conferences in late 2024. The company will present at the UBS Global Healthcare Conference on November 13 in Rancho Palos Verdes, the Stifel Healthcare Conference on November 19 in New York, and the Piper Sandler Annual Healthcare Conference on December 5 in New York. All presentations will be available via live webcast through REGENXBIO's website, with recordings accessible for approximately 30 days after each event.
REGENXBIO (Nasdaq: RGNX) has scheduled a conference call for Wednesday, November 6, at 4:30 p.m. ET to present its Q3 2024 financial results and recent operational highlights. The call will include a question-and-answer session for analysts, and participants are advised to join 15 minutes before the start time. A webcast replay will be available on the company's investor website approximately two hours after the call ends.
REGENXBIO (Nasdaq: RGNX) presented positive data from the Phase II fellow eye sub-study of subretinal ABBV-RGX-314 in patients with bilateral wet age-related macular degeneration (wet AMD) at the American Academy of Ophthalmology (AAO) meeting. Key findings at nine months post-administration include:
- 97% reduction in annualized anti-VEGF treatment burden
- 100% of patients required either zero or one supplemental injection
- 78% of patients were completely injection-free
The treatment was well-tolerated with no drug-related serious adverse events and no cases of intraocular inflammation. Patients demonstrated sustained best-corrected visual acuity (BCVA) and central retinal thickness (CRT). The data highlight the potential of ABBV-RGX-314 as a one-time effective treatment option for patients with bilateral wet AMD.
REGENXBIO Inc. (Nasdaq: RGNX) has announced upcoming presentations of new interim data for ABBV-RGX-314 at the American Academy of Ophthalmology 2024 annual meeting. ABBV-RGX-314 is an investigational one-time AAV Therapeutic being developed in collaboration with AbbVie for treating wet age-related macular degeneration (wet AMD), diabetic retinopathy, and other chronic retinal conditions.
The presentations will include:
- New results from a bilateral dosing study using subretinal delivery for wet AMD
- Data from a Phase 2 dose-escalation study evaluating suprachoroidal delivery for diabetic retinopathy
- Long-term follow-up results from the Phase I/IIa subretinal delivery program in wet AMD
These presentations will provide insights into the potential efficacy and safety of ABBV-RGX-314 across different delivery methods and retinal conditions.
REGENXBIO Inc. (Nasdaq: RGNX) has announced its participation in Chardan's 8th Annual Genetic Medicines Conference on Monday, September 30, 2024. The event will take place in New York, NY, featuring a fireside chat at 8:30 a.m. ET.
Investors and interested parties can access a live webcast of the fireside chat through the Investors section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available for approximately 30 days following the presentation, allowing those unable to attend the live event to catch up on the discussion.
This conference participation highlights REGENXBIO's ongoing engagement with the investment community and its commitment to sharing insights into its work in the field of genetic medicines.
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