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Core Insights - REGENXBIO Inc. reported positive interim data from the Phase I/II AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, presented at the 2025 MDA Clinical & Scientific Conference [1][2] Company Overview - REGENXBIO is a biotechnology company focused on gene therapy, with RGX-202 being the only next-generation gene therapy in pivotal trials for Duchenne muscular dystrophy [2][9] - The company aims to improve lives through the curative potential of gene therapy and has a late-stage pipeline for various rare diseases [12] Clinical Trial Updates - The AFFINITY DUCHENNE trial is progressing towards completing enrollment in 2025, with a Biologics License Application (BLA) submission anticipated by mid-2026 [2][7] - New biomarker data from patients aged 1-3 showed microdystrophin expression levels of 122.3% compared to control in a 3-year-old patient, indicating robust expression across all ages [4][7] - RGX-202 demonstrated the highest reported vector genome copies (4.9-55.4) among approved or investigational gene therapies [6] Safety and Efficacy - As of February 21, 2025, RGX-202 was well tolerated with no serious adverse events (SAEs) reported, and common drug-related adverse events included nausea, vomiting, and fatigue [7][8] - The treatment has shown functional improvements exceeding external natural history controls, with patients demonstrating stable or improved function on the North Star Ambulatory Assessment (NSAA) [8] Future Prospects - The company plans to share additional Phase I/II functional data in the first half of 2025, reinforcing the potential of RGX-202 to alter the course of Duchenne muscular dystrophy [2][8]

Financial Data and Key Metrics Changes - REGENXBIO ended Q4 2024 with cash, cash equivalents, and marketable securities of $245 million, down from $314 million as of December 31, 2023, primarily due to cash used for operating activities [38] - R&D expenses were $209 million for the year ended December 31, 2024, compared to $232 million in 2023, reflecting a decrease attributed to reduced headcount and preclinical activities [39] Business Line Data and Key Metrics Changes - The company submitted its first Biologics License Application (BLA) for RGX-121, a treatment for Hunter syndrome, under the accelerated approval pathway, with potential FDA approval expected in Q4 2025 [8][36] - The pivotal study for RGX-202, targeting Duchenne muscular dystrophy, is advancing rapidly, with nearly half of the enrollment completed and expected to submit a BLA by mid-2026 [15][17] - The diabetic retinopathy program is preparing for a pivotal study following a successful end of Phase 2 meeting with the FDA [20] Market Data and Key Metrics Changes - The company anticipates that more than half of the prevalent population for Duchenne muscular dystrophy will remain untreated in the coming years, indicating a significant market opportunity [16] - The partnership with AbbVie for RGX-314 is expected to yield additional milestone payments, enhancing the company's financial position [21] Company Strategy and Development Direction - REGENXBIO aims to launch multiple first or best-in-class gene therapies, focusing on sustainable profitability and leveraging over fifteen years of gene therapy leadership [9] - The company is strategically positioned to deliver on multiple late-stage opportunities, with a focus on differentiation against standard care treatments [45] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential approval of RGX-121 and the progress of RGX-202, highlighting strong momentum across the pipeline [42] - The company is optimistic about its partnerships and the potential for non-dilutive financing options to extend its cash runway beyond 2026 [40] Other Important Information - The company has a robust manufacturing capability, producing 2,500 doses of RGX-202 per year with industry-leading purity levels [17] - The BLA submission for RGX-121 is based on significant clinical data, including a high percentage of patients discontinuing enzyme replacement therapy [36] Q&A Session Summary Question: Can you delve deeper into the components of non-dilutive financing? - Management outlined three components: expected milestone payments from AbbVie, potential monetization of a priority review voucher upon regulatory approval, and the reversion of Zolgensma royalty income once a cap is reached [50][53] Question: What are the expectations around potentially going to an advisory committee for RGX-202? - Management indicated that while they do not foresee a significant issue that would necessitate an advisory committee, they are prepared for such a scenario if it arises [60] Question: What is the current pace of enrollment for the DMD pivotal trial? - Management reported encouraging enrollment and expects acceleration as new sites are activated, aiming to complete enrollment this year [76][78] Question: Are cardiac endpoints being measured in the DMD study? - Management confirmed that cardiac function will be monitored, including ejection fraction and troponin levels, but noted that significant changes may not be observable in younger patients [90][91] Question: Are there any key differences between US and OUS regulatory pathways for diabetic retinopathy? - Management highlighted that while the US pathway is clear, there are ongoing discussions with EMA and Japanese regulators to establish a solid case for the diabetic retinopathy severity scale [112][115]

Group 1 - Regenxbio reported a quarterly loss of $1.01 per share, better than the Zacks Consensus Estimate of a loss of $1.27, and an improvement from a loss of $1.43 per share a year ago, resulting in an earnings surprise of 20.47% [1] - The company posted revenues of $21.21 million for the quarter ended December 2024, missing the Zacks Consensus Estimate by 7.36%, and down from $22.21 million in the same quarter last year [2] - Over the last four quarters, Regenxbio has surpassed consensus EPS estimates two times and topped consensus revenue estimates two times [2] Group 2 - The stock has underperformed the market, losing about 13.7% since the beginning of the year compared to the S&P 500's decline of 4.8% [3] - The company's earnings outlook, including current consensus earnings expectations for upcoming quarters, will be crucial for investors [4] - The trend for estimate revisions for Regenxbio is currently favorable, leading to a Zacks Rank 2 (Buy) for the stock, indicating expected outperformance in the near future [6] Group 3 - The current consensus EPS estimate for the upcoming quarter is -$1.08 on revenues of $20.36 million, and for the current fiscal year, it is -$1.52 on revenues of $274.95 million [7] - The Medical - Biomedical and Genetics industry, to which Regenxbio belongs, is currently in the top 26% of Zacks industries, suggesting a positive outlook for the sector [8]

Financial Data and Key Metrics Changes - REGENXBIO ended the quarter on December 31, 2024, with cash, cash equivalents, and marketable securities of $245 million, down from $314 million as of December 31, 2023, primarily due to cash used for operating activities [26] - R&D expenses were $209 million for the year ended December 31, 2024, compared to $232 million in 2023, attributed to decreases in headcount and preclinical activities [26][27] - The company expects the cash runway to fund operations into the second half of 2026, with potential extensions through non-dilutive financing options [27] Business Line Data and Key Metrics Changes - The pivotal study for RGX-202 is advancing rapidly, with nearly half of the enrollment completed, and the company expects to submit a BLA under the accelerated approval pathway by mid-2026 [12][29] - The BLA for RGX-121 has been submitted, with potential FDA approval anticipated in the second half of 2025 [7][24] - The diabetic retinopathy program is moving towards a pivotal program with AbbVie, with successful end-of-Phase II meetings held with the FDA [14][20] Market Data and Key Metrics Changes - The partnership with Nippon Shinyaku for MPS programs is strategically significant, combining REGENXBIO's development expertise with Nippon's commercialization experience [9][10] - The company is preparing for potential approval of RGX-121 for MPS II in Q4 2025, which could enhance its market position [10][24] Company Strategy and Development Direction - REGENXBIO aims to launch multiple first or best-in-class gene therapies, focusing on sustainable profitability and leveraging over 15 years of gene therapy leadership [8][29] - The company is positioned to deliver on multiple late-stage opportunities, with a focus on differentiation against standard care and available treatments [29][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong momentum across the pipeline, with key milestones approaching [29] - The company believes it is well-positioned to deliver transformative treatments for patients in need of new options, particularly in the Duchenne muscular dystrophy and retinal disease markets [29][31] Other Important Information - The company has a robust commercial capability and global partnerships, which are expected to support the launch of its gene therapies [7][10] - REGENXBIO is actively working on non-dilutive financing options, including milestone payments and potential monetization of a priority review voucher [27] Q&A Session Summary Question: Can you delve deeper into the components of non-dilutive financing? - Management outlined three components: expected milestone payments from the diabetic retinopathy program, potential monetization of a priority review voucher upon regulatory approval, and the reversion of royalty income from Zolgensma once a cap is reached [36][39][40] Question: What are the expectations around potentially going to an advisory committee for RGX-202? - Management indicated that while they do not foresee a significant issue that would necessitate an advisory committee, they are prepared for it if required [46][47] Question: Can you provide an update on the NDA and patient data for the one to three-year-old cohort? - Management plans to update on one patient from the one to four cohort at the upcoming MDA meeting, with expectations of higher microdystrophin levels in younger patients [51][53] Question: What is the current pace of enrollment for the DMD pivotal trial? - Management reported encouraging enrollment and expects acceleration as more sites are activated, with a significant number of patients showing interest [58][59] Question: Are cardiac endpoints being measured in the DMD study? - Management confirmed that they are measuring cardiac endpoints, including ejection fraction and troponin levels, but noted that significant changes may not be observable in younger patients [67][72] Question: Will enrollment for the wet AMD study be cut short to accelerate timelines? - Management stated that they will not cut enrollment short, as they aim to fully characterize safety and efficacy in collaboration with AbbVie [75][76]

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, 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 number: 001-37553 REGENXBIO Inc. (Exact name of registrant as specified in its charter) (State or other jurisdiction of incorporation or organiz ...

EXHIBIT 99.1 REGENXBIO Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Operational Updates ROCKVILLE, Md., March 13, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and recent operational highlights for the fourth quarter and year ended December 31, 2024. "REGENXBIO is on the cusp of delivering potential first- or best-in-class gene therapies to market. In 2025, we have already submitted our first BLA and will accelerate late-stage development and a ...

Biologics Licensing Application (BLA) for clemidsogene lanparvovec (RGX-121) submitted and on track for potential FDA approval 2H 2025; strategic partnership with Nippon Shinyaku aims to expand potential access and commercial opportunity in MPS II and MPS I Pivotal trial of RGX-202 for Duchenne Muscular Dystrophy progressing rapidly; enrollment completion expected in 2025 with BLA filing in mid-2026 AbbVie-partnered retinal franchise continues advancing; pivotal data evaluating the safety and efficacy of ...

ROCKVILLE, Md., March 10, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced new interim biomarker data from the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202 for the treatment of Duchenne muscular dystrophy will be presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Dallas, TX, March 16-19, 2025. Title: RGX-202, an investigational gene therapy for the treatment of Duchenne Muscular Dystrophy: Interim clinical data (O75 ...

The market expects Regenxbio (RGNX) to deliver a year-over-year increase in earnings on higher revenues when it reports results for the quarter ended December 2024. This widely-known consensus outlook is important in assessing the company's earnings picture, but a powerful factor that might influence its near-term stock price is how the actual results compare to these estimates.The stock might move higher if these key numbers top expectations in the upcoming earnings report, which is expected to be released ...