Core Viewpoint - REGENXBIO Inc. will present at the 44th Annual J.P. Morgan Healthcare Conference, highlighting its advancements in gene therapy and its late-stage pipeline of treatments for rare and retinal diseases [1][2]. Company Overview - REGENXBIO is a biotechnology company founded in 2009, focused on gene therapy with a pioneering role in AAV gene therapy [3]. - The company is advancing a late-stage pipeline that includes treatments for Duchenne (RGX-202), MPS II (clemidsogene lanparvovec, RGX-121), and MPS I (RGX-111), in partnership with Nippon Shinyaku [3]. - Additionally, REGENXBIO is collaborating with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [3]. - Thousands of patients have been treated using REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [3]. - The investigational gene therapies from REGENXBIO have the potential to significantly impact healthcare delivery for millions [3].

Summary of REGENXBIO FY Conference Call Company Overview - **Company**: REGENXBIO (NasdaqGS: RGNX) - **Industry**: Gene Therapy Key Milestones and Programs - **Hunter Program**: PDUFA date set for February 2026, marking a significant milestone after nine years of development [2][9] - **Duchenne Program**: Top-line data expected in early Q2 2026, with enrollment completed in October 2025 [2][9] - **Subretinal Program for Wet AMD**: Top-line data anticipated late next year, with the market size projected to exceed $10 billion by launch [3][28] Manufacturing Capabilities - REGENXBIO has invested significantly in its manufacturing facility in Rockville, designed to meet modern gene therapy standards [7][26] - The facility has been inspected with no observations, which is considered a de-risking factor for the company [10][26] Commercialization Strategy - Partnership with NS Pharma for commercialization of the Hunter program, allowing REGENXBIO to focus on manufacturing and supply chain control [12][26] - The company has a long-standing relationship with the MPS Society, aiding in patient identification for the ultra-rare disease [13] Market Position and Differentiation - REGENXBIO's one-time gene therapy approach is expected to significantly reduce the burden of care compared to traditional treatments, such as enzyme replacement therapy [14] - The company aims to be the first gene therapy to enter the Enzyme Replacement Therapy (ERT) market, which is a notable milestone [15] Data and Expectations - Upcoming data readouts are critical, with a focus on safety and efficacy in younger patient populations for the Duchenne program [20][21] - The company anticipates a successful BLA filing in mid-2026, contingent on positive data outcomes [24] Financial Position - Current cash balance is projected to sustain operations into early 2027, with additional non-dilutive financing expected [43] - Potential monetization of a Priority Review Voucher (PRV) associated with the Hunter program's approval could provide further financial support [43] Investor Insights - REGENXBIO has emphasized its long-term commitment to gene therapy, leveraging its extensive experience and knowledge in the field [45][46] - The company aims to maintain transparency with investors regarding data and safety as it transitions into a commercial entity [46] Conclusion - REGENXBIO is positioned for a pivotal year in 2026 with multiple key milestones, including PDUFA dates and data readouts that could significantly impact its market presence and stock performance [44]

Core Insights - REGENXBIO Inc. will participate in the Piper Sandler 37th Annual Healthcare Conference with a fireside chat scheduled for December 2, 2025, at 8:30 a.m. ET [1] - The company is focused on advancing gene therapy, particularly in treating rare and retinal diseases, with a late-stage pipeline that includes several investigational therapies [2] Company Overview - REGENXBIO, founded in 2009, specializes in AAV gene therapy and aims to improve lives through its curative potential [2] - The company has developed treatments such as RGX-202 for Duchenne muscular dystrophy, RGX-121 for MPS II, and RGX-111 for MPS I, in collaboration with Nippon Shinyaku [2] - REGENXBIO is also working with AbbVie on surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy [2] - Thousands of patients have been treated using REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® [2] Recent Developments - The company announced the completion of pivotal enrollment in the AFFINITY DUCHENNE® trial for RGX-202, marking a significant milestone in its Duchenne gene therapy program [5]

Summary of Regenxbio Conference Call Company Overview - **Company**: Regenxbio - **Industry**: Gene Therapy Key Points Industry Context - Gene therapy is regaining favor despite regulatory challenges from the FDA, leading to increased optimism in the field [1][2] - The FDA's evolving stance on gene therapy approvals is a significant factor affecting the industry [7][8] Company Differentiation - Regenxbio has 15 years of experience in gene therapy, focusing on immune suppression and careful study designs, which enhances the probability of success in clinical trials [2][4] - The company has established manufacturing capabilities, with a modern suspension bioreactor process that passed FDA inspection without observations, a rare achievement [4][5] - Regenxbio can produce 2,500 doses per year for Duchenne and approximately 100,000 doses for retina programs, indicating strong manufacturing capacity for the next four to five years [5][6] Clinical Programs - The company is involved in three Phase 3 programs, with commercial-level processes already in place for pivotal studies [6] - The Hunter program is progressing well, with a straightforward FDA review process and no design questions raised [9][10] - The Duchenne program utilizes external match controls to assess treatment effects, which is critical for understanding patient outcomes [10][11] Market Potential - The Hunter disease market is small, with about 500 prevalent patients and 50 new cases annually, but gene therapy offers a one-time treatment option that could significantly reduce the burden of care compared to enzyme replacement therapy [16][17] - The company has seen a high percentage (80%) of patients in pivotal studies able to avoid enzyme replacement therapy, indicating strong potential for market disruption [17] Safety and Efficacy - Regenxbio has implemented a comprehensive safety regimen in its trials, resulting in no serious adverse events and zero liver injuries reported in the Phase 1/2 study [24][25] - The company has achieved higher microdystrophin expression levels (averaging 40%) in older boys compared to competitors, which is crucial for functional benefits [26][27] - The upcoming pivotal top-line results are expected in early Q2, with a strong focus on safety and efficacy data [28][29] Financial Position - Regenxbio has a market cap of approximately $600 million, with $350 million in non-dilutive financing expected, providing a solid financial foundation for upcoming milestones and commercial launches [49] Investor Sentiment - Despite skepticism from the investor community regarding the wet AMD market, there is growing interest in gene therapy as a disruptive force in ocular treatments [35][36] - The company has conducted the largest gene therapy trials to date, which may enhance credibility and investor confidence [36][41] Future Outlook - The company is optimistic about its position in the gene therapy landscape, with strong advocacy support and a clear path to market for its products [19][20][49]

Summary of REGENXBIO FY Conference Call Company Overview - **Company**: REGENXBIO (NasdaqGS: RGNX) - **Event**: FY Conference on November 10, 2025 - **Speakers**: CEO Curran Simpson and CMO Steve Pakola Key Points Industry and Product Development - REGENXBIO has been working on AAV (Adeno-Associated Virus) delivery for over 15 years, contributing to significant products like Zolgensma [3][4] - The company is focused on late-stage assets, with a BLA (Biologics License Application) review for the Hunter program targeting MPS II disease, with a PDUFA date set for February [3][4] - The Duchenne program has completed enrollment for its pivotal cohort, with top-line data expected in Q2 next year [4][19] - REGENXBIO is also involved in a partnership with AbbVie for the RGX-314 program targeting wet AMD, with top-line data expected by the end of 2026 [4][7] Clinical Trials and Data Expectations - The RGX-314 program is noted as one of the largest gene therapy clinical trials ever conducted, focusing on non-inferiority against existing anti-VEGF treatments [5][6] - The company aims to demonstrate a decrease in treatment burden and improved vision outcomes through its gene therapy approach [7][16] - The Duchenne program aims to replicate natural dystrophin closely, with a focus on the C-terminal domain for better efficacy [19][20] Commercial Strategy - AbbVie will lead the commercialization of the RGX-314 program, with a 50/50 profit share agreement [11][12] - REGENXBIO does not intend to build its own sales force, relying on AbbVie’s extensive commercial infrastructure [12] Market Needs and Compliance - The primary issue addressed by the RGX-314 program is patient compliance, as many patients struggle with frequent injections [13][16] - The company highlights the significant unmet need for less frequent injections, which has been shown to improve patient outcomes [15][16] Manufacturing and Cost of Goods - REGENXBIO has invested heavily in manufacturing, aiming for cost of goods to align more with biologics than traditional gene therapy [18] - The company reports good yields from its bioreactor process, which is crucial for meeting the demands of the market [18] Regulatory Interactions - The company has maintained consistent interactions with the FDA, with a focus on the Hunter program and the Duchenne protocol [24][29] - A major amendment was triggered for the Hunter program, moving the PDUFA date from November to early February [29] Safety and Efficacy - The company has implemented a targeted immunosuppression regimen to improve safety outcomes, reporting no liver injuries in treated patients [22][23] - The results from the Duchenne program have shown unexpected improvements in older patients, which is a positive indicator for the therapy's efficacy [25][27] Future Expectations - REGENXBIO is optimistic about the potential for traditional approval for the Hunter program, given the robust clinical data provided [32] - The company is also considering pursuing a Priority Review Voucher (PRV) for the Hunter program if approved before September 2026 [35] Conclusion - REGENXBIO is positioned for a significant year ahead with multiple key data readouts and regulatory milestones, focusing on innovative gene therapies that address critical unmet needs in rare diseases [3][4][32]

Core Insights - REGENXBIO Inc. reported a GAAP EPS loss of -$1.20 for Q3 2025, which exceeded estimates by approximately $0.14 [1] - The company's revenue for the same quarter was $29.7 million, reflecting a year-over-year increase of 22.7% and surpassing expectations by about $5.29 million [1] Financial Performance - Q3 2025 GAAP EPS loss: -$1.20, beating estimates by $0.14 [1] - Revenue: $29.7 million, up 22.7% year-over-year, exceeding expectations by $5.29 million [1]

分组1 - Regenxbio reported a quarterly loss of $1.2 per share, which was better than the Zacks Consensus Estimate of a loss of $1.38, representing an earnings surprise of +13.04% [1] - The company posted revenues of $29.73 million for the quarter ended September 2025, exceeding the Zacks Consensus Estimate by 23.73%, compared to $24.2 million in the same quarter last year [2] - Over the last four quarters, Regenxbio has surpassed consensus EPS estimates two times and topped revenue estimates once [2] 分组2 - The stock has gained approximately 50.2% since the beginning of the year, outperforming the S&P 500's gain of 15.6% [3] - The company's earnings outlook is crucial for future stock performance, with current consensus EPS estimates at $0.27 on $108.94 million in revenues for the upcoming quarter and -$2.43 on $228 million in revenues for the current fiscal year [7] - The Medical - Biomedical and Genetics industry, to which Regenxbio belongs, is currently ranked in the top 41% of over 250 Zacks industries, indicating a favorable outlook [8] 分组3 - The estimate revisions trend for Regenxbio was favorable ahead of the earnings release, resulting in a Zacks Rank 2 (Buy) for the stock, suggesting it is expected to outperform the market [6] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors [5]

Financial Data and Key Metrics Changes - REGENXBIO ended Q3 2025 with cash, cash equivalents, and marketable securities of $302 million, an increase from $245 million as of December 31, 2024, primarily driven by a $110 million upfront payment from Nippon Shinyaku and $145 million in net proceeds from royalty monetization [17][18] - Revenues for Q3 2025 were $30 million, compared to $24 million in Q3 2024, mainly due to development service revenue under the Nippon Shinyaku partnership [17] Business Line Data and Key Metrics Changes - The RGX-202 program for Duchenne muscular dystrophy is progressing well, with enrollment completed in the Affinity Duchenne Pivotal Trial, and top-line pivotal data expected in early Q2 2026 [5][11] - RGX-121, a potential gene therapy for MPS II, is on track for FDA approval by early 2026, with positive 12-month data delivered to the FDA [8][14] - The retinal disease franchise, particularly the ABBV-RGX-314 program for wet AMD, has completed enrollment in two global phase 3 studies, representing the largest global gene therapy program ever conducted [9][10] Market Data and Key Metrics Changes - The anticipated market for RGX-202 is significant, with the ability to produce 2,500 doses per year, positioning the company for clinical and commercial success [7] - The prevalent market for Duchenne is expected to be around 14,000 patients by 2027, with approximately 3,000 eligible for gene therapy [82] Company Strategy and Development Direction - The company is focused on advancing its late-stage pipeline of gene therapies, with a commitment to bringing new medicines to patients in need [4] - REGENXBIO is preparing for a commercial launch of RGX-202 in 2027 and is exploring opportunities to expand the program outside the U.S. [6][14] - The company aims to leverage its manufacturing capabilities and innovative science to deliver best-in-class therapeutics [10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing BLA review for RGX-121, highlighting productive interactions with the FDA and a strong safety profile [8][32] - The company anticipates a transformational year ahead, with multiple product launches and significant unmet needs being addressed by its gene therapies [19] Other Important Information - The company has a strong financial position, with cash runway expected to extend into early 2027, not including potential non-dilutive financing opportunities [18] - The manufacturing facility in Rockville is capable of producing high-purity gene therapies, which is crucial for commercial readiness [7][35] Q&A Session Summary Question: Update on RGX-202 interactions with FDA - Management indicated that top-line data will be available in early Q2 2026, with a pre-BLA meeting expected around that time [21][23] Question: Cash runway and non-dilutive financing - The company expects non-dilutive financing options to extend cash runway well into 2027 or early 2028 [24][25] Question: Confirmatory trial enrollment for DMD - Enrollment for the confirmatory study has begun, and management expects substantial progress by mid-2026 [26][27] Question: Regulatory interactions for DMD and MPS II - Management confirmed a late cycle meeting with the FDA for RGX-121 and a pre-BLA meeting for RGX-202 is anticipated [30][32] Question: Manufacturing capacity and production volume - The Rockville site has a 2,000-liter bioreactor, capable of producing up to 2,500 doses of RGX-202 annually [34][35] Question: Use of FDA natural history as a control in DMD - Management confirmed that propensity score matching is prospectively specified in the protocol for RGX-202 [38][40] Question: Interest in gene therapy from retina specialists - There is significant excitement about gene therapy among retina specialists, with half of surveyed specialists expressing interest in gene therapy approaches [42][44] Question: Expectations for black box warnings - Management does not expect a black box warning for RGX-202 due to its strong safety profile [48][49] Question: EMA plans and requirements - The company is evaluating the EMA's requirements for RGX-121 and RGX-202, with ongoing discussions about potential placebo control needs [52][54] Question: Diabetic retinopathy study adjustments - The company is considering the ordinal two-step DRSS change as a potential primary endpoint for their pivotal studies [58][61] Question: Enrollment for suprachoroidal delivery program - The company is looking to enroll 20 patients in the suprachoroidal delivery arm, with expectations for increased enrollment speed following the completion of the subretinal program [63][66]

Financial Position - Cash, cash equivalents, and marketable securities were $302.0 million as of September 30, 2025, up from $244.9 million as of December 31, 2024, primarily due to a $110.0 million upfront payment from the Nippon Shinyaku partnership and $144.5 million in net proceeds from royalty monetization[12] - Total current assets increased to $331,967,000 as of September 30, 2025, compared to $278,001,000 at December 31, 2024, reflecting a growth of 19.3%[23] - Total liabilities increased to $363,751,000 as of September 30, 2025, from $206,338,000 at December 31, 2024, representing a substantial increase of 76.2%[23] - The company’s total stockholders' equity decreased to $161,452,000 as of September 30, 2025, down from $259,651,000 at December 31, 2024, indicating a decline of 37.8%[23] - The company’s cash and cash equivalents increased to $58,802,000 as of September 30, 2025, compared to $57,526,000 at December 31, 2024, showing a slight increase of 2.2%[23] Revenue Performance - Revenues for the third quarter of 2025 were $29.7 million, an increase of 22.8% compared to $24.2 million for the same period in 2024, driven by $5.9 million of development service revenue under the Nippon Shinyaku partnership[13] - Total revenues for the three months ended September 30, 2025, were $29,733,000, an increase from $24,197,000 in the same period of 2024, representing a growth of 22.5%[24] - License and royalty revenue for the nine months ended September 30, 2025, was $129,119,000, significantly higher than $61,172,000 for the same period in 2024, indicating a year-over-year increase of 111.5%[24] Expenses - Research and development expenses rose to $56.1 million in Q3 2025, compared to $54.4 million in Q3 2024, mainly due to increased personnel and manufacturing costs for clemidsogene lanparvovec[14] - General and administrative expenses increased to $20.3 million in Q3 2025 from $19.4 million in Q3 2024, largely due to higher professional services and consulting costs[15] - Research and development expenses for the nine months ended September 30, 2025, totaled $168,688,000, up from $158,142,000 in the same period of 2024, marking a rise of 6.5%[24] - The company reported total operating expenses of $82,144,000 for the three months ended September 30, 2025, a decrease from $86,275,000 in the same period of 2024, representing a reduction of 4.9%[24] Net Loss - The net loss for Q3 2025 was $61.9 million, or $1.20 per share, compared to a net loss of $59.6 million, or $1.17 per share, in Q3 2024[16] - The company reported a net loss of $61,941,000 for the three months ended September 30, 2025, compared to a net loss of $59,597,000 for the same period in 2024, showing an increase in loss of 3.9%[24] Product Development - The RGX-202 program for Duchenne muscular dystrophy is advancing rapidly, with topline results expected in early Q2 2026 and a Biologics License Application (BLA) submission planned for mid-2026[5] - Clemidsogene lanparvovec (RGX-121) is on track to be the first gene therapy for MPS II, with a PDUFA date set for February 8, 2026[6] - Enrollment in pivotal trials for surabgene lomparvovec (sura-vec) has been completed, with topline data expected in Q4 2026, positioning it to be the first approved gene therapy for wet AMD[9] - The pivotal program for sura-vec is supported by durable safety and efficacy data observed in patients with non-proliferative diabetic retinopathy through two years in the Phase II ALTITUDE trial[10] Cash Flow Outlook - REGENXBIO expects its cash position to fund operations into early 2027, excluding potential milestone payments from partners[17]

Financial Performance - Total revenues for Q3 2025 were $29.733 million, a 23.5% increase from $24.197 million in Q3 2024[18] - License and royalty revenue for Q3 2025 was $23.605 million, slightly down from $23.982 million in Q3 2024[18] - Net loss for Q3 2025 was $61.941 million, compared to a net loss of $59.597 million in Q3 2024, representing a 3.9% increase in loss[18] - The company reported a comprehensive loss of $61.843 million for Q3 2025, compared to a comprehensive loss of $57.913 million in Q3 2024[18] - The company generated a net cash used in operating activities of $71.7 million for the nine months ended September 30, 2025, an improvement from $141.5 million in the prior year[26] - The Company reported revenues of $29.7 million for the three months ended September 30, 2025, compared to $24.2 million for the same period in 2024, representing a 22.5% increase[139] Assets and Liabilities - Total assets increased to $525.203 million as of September 30, 2025, up from $465.989 million at the end of 2024, reflecting a 12.8% growth[17] - Total liabilities rose significantly to $363.751 million as of September 30, 2025, compared to $206.338 million at the end of 2024, marking a 76.2% increase[17] - The accumulated deficit as of September 30, 2025, was $1.058 billion, an increase from $932.149 million at the end of 2024[17] - Cash and cash equivalents increased to $58.802 million as of September 30, 2025, from $57.526 million at the end of 2024[17] - The company had cash, cash equivalents, and marketable securities totaling $302.0 million as of September 30, 2025, which is expected to fund operations for at least the next 12 months[30] Research and Development - Research and development expenses for the nine months ended September 30, 2025, were $168.688 million, up from $158.142 million in the same period of 2024[18] - Research and development expenses rose to $56.1 million in Q3 2025, up $1.7 million from $54.4 million in Q3 2024, despite a $3.7 million decrease in clinical trial costs[209] - The increase in research and development expenses was partially offset by a decrease of $4.6 million in costs associated with clinical trials and regulatory activities[215] Revenue Recognition and Agreements - Revenue is recognized in accordance with ASC 606, requiring the transfer of control of goods or services to customers[43] - The transaction price for license and collaboration agreements includes fixed and variable considerations, with milestone payments included if probable[52][54] - The Company received an up-front fee of $370.0 million from AbbVie under the collaboration agreement, with potential milestone payments of up to $1.38 billion[108] - The Company entered into a collaboration and license agreement with Nippon Shinyaku, receiving an up-front fee of $110.0 million and up to $700.0 million in milestone payments[116] - The Company recognized total revenues of $80.4 million under the Nippon Shinyaku Collaboration Agreement for the nine months ended September 30, 2025[123] Stock and Compensation - Stock-based compensation expense for the nine months ended September 30, 2025, was $26.2 million, slightly lower than the $28.9 million reported for the same period in 2024[26] - The total intrinsic value of restricted stock units vested during the nine months ended September 30, 2025, was $5.9 million[132] - The Company had $57.2 million of unrecognized stock-based compensation expense expected to be recognized over a weighted-average period of 2.3 years[128] Collaboration and Licensing - The NAV Technology Platform is being utilized by licensees in one commercial product, Zolgensma®, and in the development of several other licensed products[29] - The Company has selectively licensed its NAV Technology Platform to other biotechnology and pharmaceutical companies, indicating ongoing collaboration and market expansion efforts[29] - The Company retains all rights to any priority review vouchers that may be issued upon potential approvals of RGX-121 and RGX-111[116] Expenses - General and administrative expenses increased to $20.3 million in Q3 2025 from $19.4 million in Q3 2024, driven by higher professional services and consulting costs[210] - Interest expense surged to $13.2 million in Q3 2025, up from $0.8 million in Q3 2024, primarily due to increased interest under royalty monetization liabilities[212] Future Outlook - The company expects general and administrative expenses to increase as it prepares for the potential commercialization of product candidates, particularly RGX-202 and ABBV-RGX-314[186] - Future revenues from licensing agreements are uncertain and may fluctuate significantly due to the contingent nature of payments[175]