Accessibility StatementSkip Navigation ROCKVILLE, Md., Nov. 25, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following investor conference: Piper Sandler 37 Annual Healthcare ConferenceFireside Chat: Tuesday, December 2 at 8:30 a.m. ETLocation:Â New York, NY A live webcast of the fireside chat can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available for approximately 30 days foll ...

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]

Core Insights - REGENXBIO Inc. reported significant progress in its late-stage gene therapy programs, highlighting advancements in treatments for Duchenne muscular dystrophy, Hunter syndrome, wet AMD, and diabetic retinopathy [2][3]. Financial Performance - Cash, cash equivalents, and marketable securities totaled $302.0 million as of September 30, 2025, an increase from $244.9 million at the end of 2024, primarily due to a $110.0 million upfront payment from Nippon Shinyaku and $144.5 million from royalty monetization [7]. - Revenues for Q3 2025 were $29.7 million, up from $24.2 million in Q3 2024, driven by $5.9 million in development service revenue from the Nippon Shinyaku partnership [8]. - Research and development expenses rose to $56.1 million in Q3 2025 from $54.4 million in Q3 2024, mainly due to increased personnel and manufacturing costs [9]. - General and administrative expenses increased to $20.3 million in Q3 2025 from $19.4 million in Q3 2024, attributed to professional services and consulting [10]. - The net loss for Q3 2025 was $61.9 million, compared to a net loss of $59.6 million in Q3 2024, resulting in a basic and diluted net loss per share of $1.20 [11]. Program Highlights - RGX-202, a gene therapy 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][6]. - Clemidsogene lanparvovec (RGX-121) is on track to be the first gene therapy for Hunter syndrome, with a PDUFA date set for February 8, 2026 [4][12]. - Surabgene lomparvovec (sura-vec) is positioned to be the first gene therapy for chronic retinal diseases, with pivotal trial enrollment completed and topline data expected in Q4 2026 [5][7]. Operational Developments - Enrollment in the pivotal trial for RGX-202 was completed in October 2025, with ongoing confirmatory studies for ambulatory patients [6]. - REGENXBIO has begun manufacturing RGX-202 for commercial supply, anticipating a launch in 2027 [6]. - The company presented positive 12-month data for RGX-121, showing significant biomarker reductions and correlations with neurodevelopmental outcomes [12]. Future Outlook - REGENXBIO expects its cash position to fund operations into early 2027, excluding potential milestone payments from partners [13]. - The company is preparing for a conference call to discuss these results and operational highlights [14].

Core Insights - REGENXBIO Inc. has completed enrollment in the pivotal AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, and has successfully produced initial batches for commercial supply [1][2][5] Trial Details - The AFFINITY DUCHENNE trial has enrolled 30 participants, focusing on the primary endpoint of achieving 10% microdystrophin expression at Week 12, with secondary endpoints assessing functional improvements [3][4] - In the Phase I/II portion, microdystrophin levels in participants ranged from 20% to 122%, with no serious adverse events reported, indicating a positive safety profile [4][8] Commercial Readiness - REGENXBIO has manufactured the first commercial supply batches of RGX-202, anticipating approval and launch in 2027, coinciding with market availability [5][6] - The company can produce up to 2,500 doses of RGX-202 annually using its proprietary NAVXpress manufacturing process, which achieves over 80% product purity [6][8] Product Overview - RGX-202 is positioned as a potential best-in-class gene therapy, utilizing a differentiated microdystrophin construct that encodes essential regions of dystrophin, including the C-Terminal domain [7][8] - Additional design features aim to enhance gene expression and reduce immunogenicity, supporting targeted delivery throughout skeletal and heart muscle [8]