Latest news with #RGNX
Yahoo
13-05-2025
- Business
- Yahoo
Regenxbio Inc (RGNX) Q1 2025 Earnings Call Highlights: Strategic Partnerships and Financial ...
Cash, Cash Equivalents, and Marketable Securities: $272 million as of March 31, 2025, compared to $245 million as of December 31, 2024. Upfront Payment: $110 million received under the Nippon Shinyaku collaboration. R&D Expenses: $53 million for the quarter ended March 31, 2025, compared to $54.8 million for the quarter ended March 31, 2024. Cash Runway Guidance: Expected to fund operations into the second half of 2026, excluding potential future royalty income. Warning! GuruFocus has detected 4 Warning Signs with RGNX. Release Date: May 12, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Regenxbio Inc (NASDAQ:RGNX) has made significant progress in its late-stage clinical programs, particularly with RGX-121 for MPS II, which is on track for potential FDA approval in the second half of 2025. The company has established a strategic partnership with Nippon Shinyaku to commercialize its neurodegenerative franchise, enhancing its commercial readiness. RGX-202, a next-generation candidate for Duchenne muscular dystrophy, has surpassed 50% enrollment in its pivotal study, with strong interest from the patient community. Regenxbio Inc (NASDAQ:RGNX) has a state-of-the-art manufacturing facility capable of producing up to 2,500 doses of RGX-202 annually, ensuring readiness for commercial launch. The company's financial position is strong, with $272 million in cash and marketable securities, bolstered by a $110 million upfront payment from Nippon Shinyaku, providing a cash runway into the second half of 2026. The acceptance of the BLA for RGX-121 by the FDA is still pending, creating uncertainty around the timing of regulatory approval. There are concerns about potential changes in FDA requirements for safety profiles, especially in light of recent safety events in the gene therapy space. The company faces competition in the Duchenne muscular dystrophy market, with questions about the approvability of RGX-202 on an accelerated basis. Regenxbio Inc (NASDAQ:RGNX) is navigating a complex regulatory environment with multiple interactions with the FDA, which could impact timelines. The potential impact of recent announcements on drug pricing could affect the company's ability to price its gene therapies competitively. Q: Can you provide an update on the timing for the Hunter BLA acceptance? A: Curran Simpson, President and CEO, stated that the BLA acceptance is imminent. The review process is proceeding normally, with regular interactions and information requests from the FDA. They expect to announce acceptance soon. Q: How do you view the bar for approvability in Duchenne muscular dystrophy (DMD) on an accelerated basis, especially in light of recent industry developments? A: Curran Simpson explained that they have always aimed for an accelerated approval pathway, with discussions with the FDA being positive. They are confident in their data, including safety and initial functional data, and expect to provide further updates in the first half of the year. Q: Are there any expected changes from the FDA regarding safety profile requirements for your pivotal study, especially after recent safety events in the industry? A: Curran Simpson noted that their program is on track, with no anticipated changes in safety requirements. They are confident in their immune suppression regimen and safety profile, which have been well-received by investigators and the patient community. Q: Can you provide more details on the planning process with AbbVie for the diabetic retinopathy Phase 3 trial? A: Curran Simpson mentioned that they are finalizing feedback from regulatory agencies and expect to begin site activation this year. They are considering established endpoints for the pivotal protocol and will provide more specific timing as they approach the first patient dose. Q: What are the expectations for the RGX-202 commercial supply manufacturing process? A: Curran Simpson confirmed that the commercial production process will be the same as the current clinical trials. They plan to initiate commercial production this year, with the process being validated for the mid-2026 BLA submission. The manufacturing process is mature and produces high-purity capsids. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
Washington Post
12-05-2025
- Business
- Washington Post
Regenxbio: Q1 Earnings Snapshot
ROCKVILLE, Md. — ROCKVILLE, Md. — Regenxbio Inc. (RGNX) on Monday reported first-quarter earnings of $6.1 million. On a per-share basis, the Rockville, Maryland-based company said it had net income of 12 cents. The results did not meet Wall Street expectations. The average estimate of five analysts surveyed by Zacks Investment Research was for earnings of 41 cents per share.
Yahoo
14-03-2025
- Business
- Yahoo
Regenxbio Inc (RGNX) Q4 2024 Earnings Call Highlights: Strategic Advances and Financial Resilience
Cash Equivalents and Marketable Securities: $245 million as of December 31, 2024, down from $314 million as of December 31, 2023. R&D Expenses: $209 million for the year ended December 31, 2024, compared to $232 million in 2023. Net Proceeds from Public Offering: $131 million received from an upsized public offering of common stock and pre-funded warrants in March 2024. Cash Runway Guidance: Expected to fund operations into the second half of 2026, excluding potential commercial revenue from RGX-121. Warning! GuruFocus has detected 5 Warning Signs with RGNX. Release Date: March 13, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Regenxbio Inc (NASDAQ:RGNX) has submitted its first Biologics License Application (BLA) for RGX-121, targeting Hunter syndrome, with potential FDA approval expected in Q4 2025. The company is advancing its diabetic retinopathy program into a pivotal stage, with plans for future BLA filings for RGX-202 for Duchenne Muscular Dystrophy and ABBV-RGX-314 for Wet AMD. Regenxbio Inc (NASDAQ:RGNX) has a strong partnership with Nippon Shinyaku, enhancing its commercial capabilities and providing potential milestones and revenue. The company is well-positioned with robust commercial capabilities and global partners, aiming for sustainable profitability through its gene therapy leadership. Regenxbio Inc (NASDAQ:RGNX) has a solid financial position with $245 million in cash, cash equivalents, and marketable securities, expected to fund operations into the second half of 2026. Regenxbio Inc (NASDAQ:RGNX) experienced a decrease in cash and marketable securities from $314 million to $245 million over the year, primarily due to funding operating activities. The company faces risks and uncertainties associated with forward-looking statements, which may cause actual results to differ from forecasts. There is a potential regulatory approval risk for the priority review voucher (PRV) associated with RGX-121, which could impact non-dilutive financing options. The competitive landscape in the Wet AMD space poses challenges, with other long-acting TKI studies anticipated to read out in the same 2026 timeframe. Regenxbio Inc (NASDAQ:RGNX) has not yet initiated studies in non-ambulatory patients for its Duchenne Muscular Dystrophy program, focusing resources on current pivotal trials. Q: Can you delve deeper into the components of non-dilutive financing available and the probabilities around realizing those? Also, any expectations around potentially going to an ADCOM for RGX-202 once you have the full data set? A: Patrick Christmas, Executive Vice President, Chief Legal Officer, explained that non-dilutive options include the DR milestone expected in the second half of the year, the potential monetization of a priority review voucher upon regulatory approval, and the reversion of the Zolgensma royalty stream. Curran Simpson, Chief Operating Officer, added that the pre-BLA meeting with the FDA has de-risked the process, making approval and PRV high-probability events. Stephen Pakola, Chief Medical Officer, noted that while they can't predict an ADCOM with certainty, they are prepared if it occurs. Q: Regarding the DMD program, how is the pace of enrollment for the pivotal trial, and have you started enrolling younger patients? A: Curran Simpson stated that enrollment is encouraging, with significant interest from patients and families. They expect enrollment to accelerate as more sites are activated. Stephen Pakola added that they are enrolling patients across all age ranges, including younger patients, and these will count towards the pivotal trial. Q: Are you measuring cardiac endpoints in the AFFINITY study, and could this differentiate your program from others? A: Stephen Pakola confirmed that they are measuring cardiac endpoints like ejection fraction and troponin levels. While cardiac deterioration typically occurs in older patients, they are optimistic about the potential benefits of their therapy based on preclinical data. Q: On the regulatory discussions for diabetic retinopathy, are there differences between US and OUS regulators? A: Stephen Pakola explained that while the US has a clear path using the diabetic retinopathy severity scale, they are working with EMA and Japan to establish a similar path. They believe the case for using this scale is strong and are optimistic about global regulatory discussions. Q: What should we expect from the upcoming functional data update for the DMD program, and how will you analyze this data? A: Curran Simpson mentioned that the update will include new patients and longer-term data for previously reported patients. Stephen Pakola added that they will compare the data to matched external natural history controls, a method discussed with the FDA during the end of Phase 2 meeting. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data
