- Data from pivotal dose level demonstrates long-term, sustained reductions in CSF levels of HS D2S6, a key biomarker of brain disease in MPS II - 80% of patients who received the pivotal dose discontinued intravenous enzyme replacement therapy or remained treatment-naïve - Submission of a rolling BLA using the accelerated approval pathway on track for Q3 2024 ROCKVILLE, Md., Sept. 3, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced positive results from the Phase I/II/III CAMPSIITE® trial ...

ROCKVILLE, Md., Aug. 29, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate the following upcoming investor conferences: Morgan Stanley 22nd Annual Global Healthcare Conference Fireside Chat: Thursday, September 5, 2024 at 5:35 p.m. ET Location: New York, NY Baird 2024 Global Healthcare Conference Fireside Chat: Wednesday, September 11, 2024 at 9:40 a.m. ET Location: New York, NY Live webcasts of the fireside chats at the Morgan Stanley and Baird conferences can be access ...

ROCKVILLE, Md., Aug. 8, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the H.C. Wainwright 4th Annual Ophthalmology Virtual Conference on Thursday, August 15, 2024. H.C. Wainwright 4th Annual Ophthalmology Virtual Conference Panel: The Evolving Therapeutic Landscape of AMD Date/Time: Thursday, August 15, 2024 at 9:00 a.m. EDT Location: Virtual A live webcast of the panel can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com. An archiv ...

Shares of Regenxbio (RGNX) have gained 17.7% over the past four weeks to close the last trading session at $12.51, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $41.83 indicates a potential upside of 234.4%. The average comprises 12 short-term price targets ranging from a low of $28 to a high of $61, with a standard deviation of $9.47. While the lowest estimate indicates an in ...

Regenxbio (RGNX) came out with a quarterly loss of $1.05 per share versus the Zacks Consensus Estimate of a loss of $1.29. This compares to loss of $1.66 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of 18.60%. A quarter ago, it was expected that this biotechnology company would post a loss of $1.28 per share when it actually produced a loss of $1.38, delivering a surprise of -7.81%. Over the last four quarters, the company ha ...

Core Insights - REGENXBIO Inc. reported positive interim safety and efficacy data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202 for Duchenne muscular dystrophy, showing robust microdystrophin expression levels in treated patients [1][3][5] - The company plans to initiate a pivotal trial in Q4 2024 and expects to complete enrollment in the dose level 2 expansion cohort early in Q3 2024 [1][7] Company Overview - REGENXBIO is a clinical-stage biotechnology company focused on gene therapy, with a proprietary NAVXpress™ platform for manufacturing [10][12] - RGX-202 is a one-time AAV therapeutic designed to deliver a novel microdystrophin gene, which is the only gene therapy in development for Duchenne that encodes for the C-Terminal domain [2][10] Clinical Trial Details - The AFFINITY DUCHENNE trial is a multicenter, open-label study evaluating the safety, tolerability, and clinical efficacy of RGX-202 in patients aged 1-11 years [8][9] - Initial results showed microdystrophin expression levels of 77.2% and 46.5% in patients aged 5.8 and 8.5 years, respectively, compared to control [4][5] Manufacturing and Capacity - RGX-202 is manufactured at the REGENXBIO Manufacturing Innovation Center, which has the capacity to produce up to 2,500 doses per year using a scalable suspension-based process [6][10] Future Expectations - The company anticipates sharing initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial in the second half of 2024 [7]

Company Overview - REGENXBIO is a leading clinical-stage biotechnology company focused on gene therapy with a mission to improve lives through its innovative AAV Therapeutics [1] - The company was founded in 2009 and has developed a pipeline targeting retinal and rare diseases, including collaborations with AbbVie for ABBV-RGX-314 [1] Product Pipeline - REGENXBIO is advancing several AAV Therapeutics, including: - ABBV-RGX-314 for wet AMD and diabetic retinopathy [1] - RGX-202 for Duchenne muscular dystrophy [1] - RGX-121 for MPS II [1] - Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, which includes Novartis' ZOLGENSMA for spinal muscular atrophy [1] Treatment Potential - AAV Therapeutics are designed as one-time treatments, offering the potential to transform healthcare delivery for millions of patients [1]

Regenxbio (RGNX) shares rallied 18.3% in the last trading session to close at $12.71. This move can be attributable to notable volume with a higher number of shares being traded than in a typical session. This compares to the stock's 18.3% loss over the past four weeks. For Regenxbio, the consensus EPS estimate for the quarter has remained unchanged over the last 30 days. And a stock's price usually doesn't keep moving higher in the absence of any trend in earnings estimate revisions. So, make sure to keep ...

Company Overview - Regenxbio (RGNX) shares increased by 6.4% to $12.79 in the last trading session, following a significant trading volume, contrasting with a 19.4% decline over the past four weeks [1] Recent Developments - The company successfully completed a pre-biologics license application (BLA) meeting with the FDA for its candidate RGX-121, aimed at treating Mucopolysaccharidosis Type II (MPS II), and plans to initiate a rolling BLA submission in Q3 2024, which may have contributed to the recent share price increase [2] Financial Expectations - Regenxbio is projected to report a quarterly loss of $1.29 per share, reflecting a year-over-year increase of 22.3%, with expected revenues of $21.73 million, an 8.8% rise from the previous year [3] - The consensus EPS estimate for the upcoming quarter has been revised down by 2.7% over the last 30 days, indicating a negative trend in earnings estimate revisions, which typically does not lead to price appreciation [4] Industry Context - Regenxbio operates within the Zacks Medical - Biomedical and Genetics industry, where ProQR (PRQR) also resides, having closed 3.5% lower at $1.65, with a -9% return over the past month [4] - ProQR's consensus EPS estimate remains unchanged at -$0.12, representing a 9.1% decline compared to the previous year's EPS, and it also holds a Zacks Rank of 3 (Hold) [5]

"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 ...