REGENXBIO(RGNX)

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REGENXBIO Announces Expansion of AFFINITY DUCHENNE® Trial to Include a New Cohort of Younger Patients
Prnewswire· 2024-06-24 11:05
"We believe RGX-202 has unique, differentiating features that support its potential to be a best-in-class product and we are pleased to expand its clinical development to reach a wider range of boys with Duchenne in need of treatment options," said Curran Simpson, Chief Operating Officer of REGENXBIO and President and CEOelect. "Supported by the strong safety profile and positive microdystrophin data demonstrated in the AFFINITY DUCHENNE trial, today's news marks significant steps in rapidly accelerating RG ...
REGENXBIO Announces Successful Pre-BLA Meeting with FDA to Support Accelerated Approval Pathway for RGX-121 for the Treatment of MPS II
Prnewswire· 2024-06-18 11:05
Aligned with FDA on content of BLA and plans for submission: Submission of a rolling BLA using the accelerated approval pathway expected to start in Q3 2024 Confirmatory trial expected to begin in H2 2025 FDA confirmed RGX-121 commercial bulk drug is comparable to clinical material Positive biomarker, neurocognitive and systemic data will be part of BLA submission The FDA continues to be aligned with REGENXBIO's plan to use cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6, a key biomarker of br ...
REGENXBIO Announces Leadership Transition
Prnewswire· 2024-06-12 11:05
Curran Simpson, Chief Operating Officer, Appointed as President and CEO Co-Founder Kenneth T. Mills to Step Down as President and CEO; Named Chairman of the Board Mr. Simpson is a seasoned biopharmaceuticals leader with over 35 years of industry experience. As the Chief Operating Officer (COO) at REGENXBIO since January 2023, he has been directly responsible for key business functions including Research & Clinical Development, Corporate Strategy, Manufacturing & Quality, Regulatory, and Commercial Operation ...
Regenxbio: Transforming Gene Therapy With Innovative AAV Treatments, Strong Buy
Seeking Alpha· 2024-06-11 03:55
REGENXBIO Inc. (NASDAQ:RGNX) is a biotech company developing AAV therapies for common and rare diseases. Its proprietary NAV platform creates one-dose AAV treatments that offer lasting therapeutic effects by targeting the root cause of diseases. AAV vectors deliver gene therapies to specific cells, maximizing efficacy and avoiding adverse immune responses. RGNX's pipeline includes promising candidates such as ABBV-RGX-314 for age-related AMD and DR, RGX-202 for DMD, and RGX-121 for Hunter syndrome. RGXN's s ...
REGENXBIO to Participate in the Goldman Sachs 45th Annual Global Healthcare Conference
Prnewswire· 2024-06-05 11:05
ROCKVILLE, Md., June 5, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the Goldman Sachs 45th Annual Global Healthcare Conference on Wednesday, June 12, 2024. Goldman Sachs 45th Annual Global Healthcare Conference Fireside Chat: Wednesday, June 12, 2024 at 8:00 a.m. EDT Location: Miami, FL A live webcast of the fireside chat can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available for ...
REGENXBIO(RGNX) - 2024 Q1 - Earnings Call Transcript
2024-05-09 01:42
REGENXBIO Inc. (NASDAQ:RGNX) Q1 2024 Earnings Conference Call May 8, 2024 4:30 PM ET Company Participants Patrick Christmas – Chief Legal Officer Ken Mills – Chief Executive Officer Curran Simpson – Chief Operating Officer Vit Vasista – Chief Financial Officer Conference Call Participants Gena Wang – Barclays Annabel Samimy – Stifel Brian Skorney – Baird Luca Issi – RBC Capital Daniil Gataulin – Chardan Operator Good day and thank you for standing by. Welcome to REGENXBIO's First Quarter 2024 Earnings Confe ...
REGENXBIO(RGNX) - 2024 Q1 - Quarterly Report
2024-05-08 20:16
Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) (Address of principal executive offices) (Zip Code) Delaware 47-1851754 (I.R.S. Employer Identification No.) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___ to ___ Commission File Numb ...
REGENXBIO(RGNX) - 2024 Q1 - Quarterly Results
2024-05-08 20:10
REGENXBIO Reports First Quarter 2024 Financial Results and Recent Operational Highlights ROCKVILLE, Md., May 8, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the first quarter ending March 31, 2024. Recent operational highlights, including acceleration of the prioritized pipeline supports meaningful value generation from the Company's strong portfolio of AAV Therapeutics. "Exciting program and data updates continue in 2024 and demonstrate remarkable progress on how ...
REGENXBIO(RGNX) - 2023 Q4 - Earnings Call Transcript
2024-02-28 02:45
Financial Data and Key Metrics Changes - REGENXBIO ended the year with cash, cash equivalents, and marketable securities totaling $314 million, a decrease from $565 million as of December 31, 2022, primarily due to cash used for operating activities in 2023 [136][45] - R&D expenses were $232 million for the year ended December 31, 2023, compared to $242 million in 2022, mainly due to clinical trial and manufacturing expenses for ABBV-RGX-314 [9] Business Line Data and Key Metrics Changes - RGX-314 is in pivotal trials for the treatment of wet AMD and diabetic retinopathy, with expectations for global regulatory submission by the end of 2025 through the first half of 2026 [121][10] - RGX-202 demonstrated strong microdystrophin expression, with the third patient at dose level 1 showing 83.4% expression compared to normal control at three months [124] Market Data and Key Metrics Changes - The anti-VEGF market opportunity is expected to grow significantly as the population ages, with RGX-314 being evaluated in two ongoing pivotal trials expected to enroll a total of 1,200 patients across the U.S., Europe, and Japan [6] Company Strategy and Development Direction - The company is focusing on a pipeline prioritization plan to concentrate resources on large commercial opportunities, particularly in gene therapies for wet AMD, diabetic retinopathy, Duchenne, and MPS II [4][5] - The operational goals with AbbVie include completing pivotal trials for RGX-314 and supporting the initiation of new trials for RGX-202 [10][46] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's positioning for success in 2024 and beyond, citing recent milestones and upcoming data updates as key drivers [21][32] - The company is optimistic about the potential of RGX-202 to address a significant unmet need in Duchenne, with plans to initiate a pivotal trial this year [46][127] Other Important Information - The company plans to share new data updates for RGX-202 at the Muscular Dystrophy Association Conference and for RGX-314 in Q2 and mid-2024 [5][48] - Management highlighted the importance of long-term vision health and the differentiation of RGX-314 in the competitive landscape [56][54] Q&A Session Summary Question: What competitive moat does RGX-314 have if approved? - Management emphasized the durability of treatment outcomes and the partnership with AbbVie as key competitive advantages, along with a large database of patient data [27][29] Question: What are the expectations for initial strength and functional data from RGX-202? - Management indicated that they expect to share strength and motor function data for both dose levels throughout 2024, with a focus on microdystrophin expression as a key indicator [33][34] Question: Will longer-term data from the suprachoroidal RGX-314 program be required before starting Phase 3? - Management stated that while longer-term data is valuable, they are on track to initiate Phase 3 based on current data and endpoints [63][64] Question: How does the company view the regulatory landscape for RGX-121? - Management expressed confidence in the use of biomarkers for accelerated approval and highlighted ongoing discussions with the FDA regarding the use of well-characterized biochemical markers [104][106]
REGENXBIO(RGNX) - 2023 Q4 - Annual Report
2024-02-27 21:15
Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-37553 Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 1 ...