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]

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duche ...

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 ...

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 ...

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 ...

Company Overview - REGENXBIO Inc. is a clinical-stage biotechnology company focused on developing adeno-associated virus (AAV) gene therapies for both common and rare diseases, utilizing its proprietary NAV platform to create one-dose AAV treatments that target the root causes of diseases [9][24] - The company has a diverse pipeline that includes ABBV-RGX-314 for age-related macular degeneration (AMD) and diabetic retinopathy (DR), RGX-202 for Duchenne muscular dystrophy (DMD), and RGX-121 for Hunter syndrome [25][26][18] Market Potential - The global market potential for ABBV-RGX-314 is estimated to be approximately $17.0 billion over the next five years, with the US anti-VEGF market alone valued at $4.5 billion annually [6] - The US retinal disease market is projected to reach $11.5 billion, while the EU market is estimated at $4.5 billion and Japan at $1 billion [6] - The global market for DMD is estimated at $7.0 billion, and for Mucopolysaccharidosis Type II (MPS II), it is around $1.0 billion [6] Financial Position - RGNX's balance sheet shows $113.0 million in cash and equivalents, plus $225.7 million in marketable securities, totaling $338.7 million in liquidity after a recent $140.0 million equity raise [13] - The company's latest quarterly cash burn was $56.1 million, indicating a yearly cash burn rate of $224.4 million, which suggests a cash runway of about 1.5 years, sufficient to finance operations until late 2025 [13] Clinical Development - ABBV-RGX-314 is currently in phases 2 and 3 for wet AMD, with regulatory submissions planned for late 2025 and early 2026 [10] - RGX-202 is in phase 2 for DMD, utilizing an AAV8 vector to deliver a transgene that encodes a microdystrophin protein to restore muscle functionality [26] - RGX-121 is in a pivotal study for MPS II, with plans to file a Biologics License Application (BLA) in 2024 [18] Strategic Collaborations - RGNX has a strategic collaboration with AbbVie for the development of ABBV-RGX-314, which includes a $200.0 million milestone payment related to DR [8][15] - AbbVie has committed significant resources, including a $370 million upfront payment for the rights to ABBV-RGX-314, with potential additional development and milestone payments totaling up to $1.38 billion [27] Valuation and Investment Outlook - RGNX trades at a market cap of $693.5 million, with a forward price-to-sales (P/S) ratio of 2.8, which is considered cheap compared to the sector median of 3.7 [29] - The company is viewed as undervalued despite its promising pipeline and potential revenue streams from ongoing projects and collaborations [22][29]

Company Overview - REGENXBIO Inc. is a leading clinical-stage biotechnology company focused on gene therapy with a mission to improve lives through its curative potential [3] - The company has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines since its founding in 2009 [3] - REGENXBIO is advancing a pipeline of AAV Therapeutics targeting retinal and rare diseases, including collaborations with AbbVie for ABBV-RGX-314 [3] Upcoming Event - REGENXBIO will participate in the Goldman Sachs 45th Annual Global Healthcare Conference on June 12, 2024, at 8:00 a.m. EDT [2] - A live webcast of the event will be available on the Investors section of REGENXBIO's website, with an archived replay accessible for approximately 30 days post-presentation [2] Product Pipeline - The company is developing several AAV Therapeutics, including RGX-202 for Duchenne muscular dystrophy and RGX-121 for MPS II [3] - Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, which includes Novartis' ZOLGENSMA for spinal muscular atrophy [3] - AAV Therapeutics are designed as one-time treatments, potentially transforming healthcare delivery for millions [3]

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 ...