Latest news with #RGX-202
Business Insider
2 days ago
- Business
- Business Insider
Regenxbio's RGX-202 shows efficacy in Phase 1/2 Duchenne trial
Regenxbio (RGNX) announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial. Updates include positive functional, safety and biomarker data for RGX-202, Regenxbio's potential best-in-class, investigational gene therapy for Duchenne muscular dystrophy. The functional data demonstrate consistent benefit among dose level 2 participants at 9 and 12 months following treatment with RGX-202. The data further supports the planned Biologics License Application submission under accelerated approval in mid-2026. Regenxbio announced positive interim functional results from the first five participants, aged approximately 6 to 12 years at dosing, receiving RGX-202 at dose level 2. RGX-202 continues to demonstrate evidence of positively impacting disease trajectory. At 9 months, RGX-202 participants demonstrated improvement in function and exceeded external controls on all measures. Four out of the five participants reached 12-months post dosing. Results at 12 months are similar to those seen at 9 months. RGX-202 participants demonstrated improved performance on timed function tests and NSAA, exceeding external natural history controls at 12 months. All participants within this cohort demonstrated improvement on all timed function tests compared to baseline. RGX-202 was appropriately localized to the sarcolemma, demonstrating that the differentiated construct is appropriately targeting the muscle. RGX-202 was well tolerated with no serious adverse events and no AEs of special interest. Regenxbio is enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial of RGX-202. The pivotal trial is expected to support a Biologics License Application submission using the accelerated approval pathway in mid-2026. Regenxbio expects to share top-line data in the first half of 2026.
Yahoo
05-06-2025
- Business
- Yahoo
REGENXBIO REPORTS NEW POSITIVE FUNCTIONAL DATA FROM PHASE I/II AFFINITY DUCHENNE® TRIAL OF RGX-202
RGX-202 demonstrating consistent evidence of positively changing disease trajectory for Duchenne All dose level 2 participants exceeded external natural history controls on all functional measures Biomarker data demonstrate consistent, robust microdystrophin expression and transduction levels across all treated ages One new participant aged 2 years at dosing had expression level at 118.6% compared to control Favorable safety profile continues with no serious adverse events or adverse events of special interest observed Webcast to be held at 8:00 a.m. today ROCKVILLE, Md., June 5, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial. Updates include positive functional, safety and biomarker data for RGX-202, REGENXBIO's potential best-in-class, investigational gene therapy for Duchenne muscular dystrophy. The functional data demonstrate consistent benefit among dose level 2 participants at 9 and 12 months following treatment with RGX-202. "Today's findings support the potential of RGX-202 to positively change the disease course for Duchenne and meaningfully benefit patients living with this degenerative disease. At the same dose being used in the pivotal trial, RGX-202 participants exceeded natural history across all key measures, including the North Star Ambulatory Assessment, which is striking," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "We are particularly encouraged by the outperformance observed in older patients. The continued, positive data further strengthen our commitment to rapidly bring this potentially transformative therapy to market and support our planned Biologics License Application submission under accelerated approval in mid-2026." "These findings suggest that the microdystrophin expression observed with RGX-202 is leading to meaningful functional improvements, even in individuals with DMD who are expected to experience functional decline," said Aravindhan Veerapandiyan, M.D., of Arkansas Children's Hospital. "These Phase I/II results, demonstrating functional improvements and favorable safety profile, underscore the potential of RGX-202 as a treatment option for individuals with DMD. It is both encouraging and essential to have innovative therapies that can help preserve muscle integrity and substantially delay disease progression. I'm enthusiastic about the continued development of RGX-202 and the promise it holds for the Duchenne community." AFFINITY DUCHENNE Phase I/II Interim Data Updates (data cut: May 7, 2025) Functional DataToday, REGENXBIO announced positive interim functional results from the first five participants, aged approximately 6 to 12 years at dosing, receiving RGX-202 at dose level 2 (2x1014 GC/kg). Based on these patients' age at dosing and baseline function, four out of five patients are expected to be in the decline phase of their disease trajectory. Results were measured against external natural history controls that were strictly matched for age and baseline function1. RGX-202 continues to demonstrate evidence of positively impacting disease trajectory with dose level 2 participants demonstrating improved performance on North Star Ambulatory Assessment (NSAA) and timed function tests (Time to Stand, 10 Meter Walk-run, Time to Climb), exceeding external natural history controls. At 9 months, RGX-202 participants demonstrated improvement in function and exceeded external controls on all measures. On NSAA, RGX-202 recipients improved an average of 4 points from baseline and 4.8 points compared to natural history. [Figure 1] Four out of the five participants reached 12-months post dosing. Results at 12 months are similar to those seen at 9 months. RGX-202 participants demonstrated improved performance on timed function tests and NSAA, exceeding external natural history controls at 12 months. All participants within this cohort demonstrated improvement on all timed function tests compared to baseline. On NSAA, RGX-202 recipients improved an average of 4.5 points from baseline and 6.8 points compared to natural history. [Figure 2] Additionally, dose level 2 participants' timed task velocity changes exceeded minimal clinically important difference (MCID) benchmarks at 12 months, a measure referenced by the FDA in the approval of an available gene therapy. Biomarker DataBiomarker data from the Phase I/II study continues to support consistent, high expression and transduction of RGX-202 microdystrophin. New data from an additional patient, aged 2 at dosing, had a microdystrophin expression level of 118.6% compared to control. The primary endpoint in the pivotal phase of AFFINITY DUCHENNE is the proportion of participants whose RGX-202 microdystrophin expression is ≥10% at Week 12. RGX-202 was appropriately localized to the sarcolemma, demonstrating that the differentiated construct with the inclusion of the C-Terminal (CT) domain is appropriately targeting the muscle. Mean at 12 Weeks (min, max) Dose Level 1 1x1014 GC/kg Dose Level 2 2x1014 GC/kg Age range (number with data) 4-7 (2) 8-11 (1) 1-3 (2) 4-7 (2) 8-11 (5) RGX-202 Microdystrophin % normal control (Western Blot) 60.6 (37.8, 83.4) 10.4 120.5 (118.6, 122.3) 54.3 (31.5, 77.2) 39.7 (20.8, 75.7) VCN copies/nucleus (qPCR) 9.8 (7.4,12.1) 5.4 24.8 (20.4, 29.1) 30.1 (4.9, 55.4) 17.8 (12.0,30.7) Positive Fibers % (Immunofluorescence) 79.32 34.6 82.12 50.3 (29.4, 71.1) 45.7 (21.3,70.6) RGX-202 also continues to demonstrate the highest reported vector genome copies (4.9-55.4) measured by qPCR across approved or investigational gene therapies. Safety and Tolerability Data RGX-202 was well tolerated with no serious adverse events (SAEs) and no AEs of special interest (AESIs). Common drug-related AEs included nausea, vomiting and fatigue. All are typically anticipated with gene therapy administration. A proactive, short-course immune modulation regimen in combination with a differentiated construct and industry-leading product purity levels of more than 80% full capsids may contribute to a favorable safety profile for RGX-202. Phase I/II AFFINITY DUCHENNE Trial: RGX-202 Treatment Emergent Adverse Events Dose Level 1 Dose Evaluation (1x1014 GC/kg) Dose Level 2 Dose Younger Boys (2x1014 GC/kg) Dose Level 2 Evaluation / Expansion (2x1014 GC/kg) Total n=13 Age Range (number dosed) 4-11 (n=3) 1-3 (n=3) 4-11 (n=7) All Ages SAE 0 0 0 0 AESI Central or peripheral neurotoxicity 0 0 0 0 Drug-induced liver injury 0 0 0 0 Thrombocytopenia 0 0 0 0 Myocarditis 0 0 0 0 Myositis 0 0 0 0 AFFINITY DUCHENNE Pivotal TrialREGENXBIO is enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial of RGX-202. The trial is expected to enroll approximately 30 patients aged 1+ in the U.S. and Canada by 2025, with more than half already enrolled to support the pivotal dataset. The pivotal trial is expected to support a Biologics License Application (BLA) submission using the accelerated approval pathway in mid-2026. REGENXBIO expects to share top-line data in the first half of 2026 and plans to include biomarker, functional, and safety data in its submission. Webcast DetailsREGENXBIO will host a webcast featuring REGENXBIO management and Dr. Veerapandiyan to discuss today's developments at 8:00 a.m. EST. The live webcast can be accessed here and in the Investors section of REGENXBIO's website at An archived replay of the webcast will be available for approximately 30 days following the presentation. About RGX-202RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or in late-stage development for Duchenne with a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT) domain. In preclinical studies, the CT domain has been shown to protect the muscle from contraction-induced stress and improve its ability to repair itself. Additional design features may potentially improve gene expression, increase protein translation efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV® AAV8 vector and a well-characterized muscle-specific promoter (Spc5-12). RGX-202 is manufactured using REGENXBIO's proprietary, high-yielding NAVXpress™ suspension-based platform process. About Duchenne Muscular DystrophyDuchenne is a severe, progressive, degenerative muscle disease, affecting 1 in 3,500 to 5,000 boys born each year worldwide. Duchenne is caused by mutations in the Duchenne gene which encodes for dystrophin, a protein involved in muscle cell structure and signaling pathways. Without dystrophin, muscles throughout the body degenerate and become weak, eventually leading to loss of movement and independence, required support for breathing, cardiomyopathy and premature death. ABOUT REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit FORWARD-LOOKING STATEMENTSThis press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2024, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Zolgensma® is a registered trademark of Novartis AG. All other trademarks referenced herein are registered trademarks of REGENXBIO. Contacts:Dana CormackCorporate Communicationsdcormack@ Investors:George E. MacDougallInvestor RelationsIR@ ____________________________________ 1 For NSAA, the EC matched subjects of one treated subject did not have data at Month 9 or Month 12. The delta was based on the mean of RGX-202 participants' changes from baseline minus stratum-based mean change from baseline of EC matched participants. 2 One sample could not be evaluated View original content to download multimedia: SOURCE REGENXBIO Inc. Sign in to access your portfolio
Yahoo
02-06-2025
- Business
- Yahoo
REGENXBIO to Host Webcast Discussing Interim Functional Data from the Phase I/II AFFINITY DUCHENNE® Trial of RGX-202
Event will feature Aravindhan Veerapandiyan, M.D., principal investigator of the AFFINITY DUCHENNE® trial ROCKVILLE, Md., June 2, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast to discuss new interim functional data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, the company's next-generation investigational gene therapy for the treatment of Duchenne muscular dystrophy. The webcast will feature AFFINITY DUCHENNE principal investigator Aravindhan Veerapandiyan, M.D., Arkansas Children's Hospital. Webcast detailsTitle: AFFINITY DUCHENNE® Trial of RGX-202: Phase I/II Interim Functional DataDate/Time: Thursday, June 5, 2025, at 8:00 a.m. EDT Access: The live webcast can be accessed here and in the Investors section of REGENXBIO's website at An archived replay of the webcast will be available for approximately 30 days following the presentation. ABOUT REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit Contacts: Dana CormackCorporate Communicationsdcormack@ Investors:George E. MacDougallInvestor RelationsIR@ View original content to download multimedia: SOURCE REGENXBIO Inc.
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
