REGENXBIO(RGNX) - 2025 Q2 - Quarterly Results

Executive Summary & Company Overview CEO Statement & Q2 2025 Highlights REGENXBIO's CEO, Curran M. Simpson, highlighted the acceleration of multiple first or best-in-class gene therapy programs in Q2 2025, including the potential FDA approval of clemidsogene lanparvovec (RGX-121) in November, rapid progress of RGX-202, and the advancement of a late-stage program for diabetic retinopathy - REGENXBIO is accelerating multiple first or best-in-class gene therapies for serious diseases like Duchenne, Hunter syndrome, and retinal diseases[3] - Potential FDA approval of clemidsogene lanparvovec (RGX-121) is on track for November 2025[3] - RGX-202 is progressing rapidly through pivotal study, and a new late-stage program for diabetic retinopathy is advancing[3] About REGENXBIO Inc. REGENXBIO is a biotechnology company focused on AAV gene therapy, advancing a late-stage pipeline for rare and retinal diseases, including RGX-202 for Duchenne, RGX-121 for MPS II, RGX-111 for MPS I (with Nippon Shinyaku), and surabgene lomparvovec (sura-vec) for wet AMD and diabetic retinopathy (with AbbVie), with their AAV platform utilized in treatments for thousands of patients, including Novartis' ZOLGENSMA® - REGENXBIO is a biotechnology company pioneering AAV gene therapy since its founding in 2009[23] - The company is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202, RGX-121, RGX-111, and surabgene lomparvovec[23] - Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®[23] Program Highlights and Milestones Neuromuscular Disease Programs REGENXBIO is making significant progress in its neuromuscular disease pipeline, with RGX-202 for Duchenne muscular dystrophy on track for accelerated pivotal trial enrollment and BLA submission, and clemidsogene lanparvovec (RGX-121) for MPS II nearing potential FDA approval following successful inspections RGX-202 for Duchenne Muscular Dystrophy RGX-202, a potential best-in-class gene therapy for Duchenne, is on track for topline results in 1H 2026 and BLA submission mid-2026, with pivotal trial enrollment accelerating and expected to complete in October 2025, ahead of previous guidance, and positive Phase I/II data demonstrating a favorable safety profile and robust microdystrophin expression - RGX-202 pivotal trial enrollment is expected to complete in October 2025, ahead of previous guidance[5][6] - Topline results for RGX-202 are expected in the first half of 2026, with a Biologics License Application (BLA) submission planned for mid-2026 under the accelerated approval pathway[5][10] - Phase I/II data showed a favorable safety profile with no serious adverse events or signs of liver injury, and all dose level 2 participants exceeded external natural history controls on functional measures[10] Clemidsogene lanparvovec (RGX-121) for MPS II (Hunter Syndrome) Clemidsogene lanparvovec (RGX-121) is on track to be the first gene therapy and one-time treatment for MPS II, with an FDA Prescription Drug User Fee Act (PDUFA) target action date of November 9, 2025, following successful FDA inspections of manufacturing, quality systems, and clinical data practices with no observations, and potential FDA approval could result in a Priority Review Voucher (PRV) - RGX-121 is on track for potential FDA approval as the first gene therapy for MPS II, with a PDUFA target action date of November 9, 2025[5][10] - FDA Pre-license inspection (PLI) of the in-house manufacturing facility and Bioresearch monitoring information (BIMO) inspection of laboratory and clinical data practices were successfully completed with no observations[5][10] - FDA approval could result in the receipt of a Priority Review Voucher (PRV), to which REGENXBIO has full rights[10] Retinal Disease Programs REGENXBIO's surabgene lomparvovec (sura-vec) program for retinal diseases is advancing, with positive Phase II data supporting a pivotal program for diabetic retinopathy (DR) via suprachoroidal delivery, and ongoing pivotal trials for wet age-related macular degeneration (wet AMD) via subretinal delivery, with topline results expected in 2026 Surabgene lomparvovec (sura-vec) for Diabetic Retinopathy (Suprachoroidal Delivery) Sura-vec for diabetic retinopathy (DR) is advancing to a global pivotal program, supported by positive Phase II ALTITUDE® trial data reported in June 2025, demonstrating a durable safety and efficacy profile through two years with a single, in-office injection and no drug-related serious adverse events, with a two-part placebo-controlled Phase IIb/III trial to be initiated with 2-step DRSS improvement as the primary endpoint - Sura-vec using suprachoroidal delivery for diabetic retinopathy is advancing to a global pivotal program, supported by positive Phase II trial data[5][10] - Phase II ALTITUDE® trial data demonstrated durable safety and efficacy in patients with non-proliferative DR through two years with a single, in-office injection, with no drug-related serious adverse events[10] - A two-part placebo-controlled Phase IIb/III trial will be initiated, with the primary endpoint being 2-step DRSS improvement[10] Surabgene lomparvovec (sura-vec) for Wet AMD (Subretinal Delivery) Enrollment is ongoing in the ATMOSPHERE® and ASCENT™ pivotal trials for the subretinal delivery of sura-vec in patients with wet AMD, with topline results expected in 2026, positioning sura-vec to be the first approved gene therapy for wet AMD - Enrollment is ongoing in the ATMOSPHERE® and ASCENT™ pivotal trials for subretinal delivery of sura-vec in patients with wet AMD[11] - Topline results for subretinal sura-vec in wet AMD are expected in 2026[5][11] - Sura-vec is on track to be the first approved gene therapy for wet AMD[5][11] Surabgene lomparvovec (sura-vec) for Wet AMD (Suprachoroidal Delivery) The Phase II AAVIATE® trial continues enrolling a new cohort to evaluate sura-vec at dose level 4 (1.5x10e12 GC/eye) for wet AMD using suprachoroidal delivery, with patients in this cohort also receiving short-course prophylactic steroid eye drops - The Phase II AAVIATE® trial is enrolling a new cohort to evaluate suprachoroidal sura-vec at dose level 4 (1.5x10e12 GC/eye) for wet AMD[12] - Patients in this cohort will receive short-course prophylactic steroid eye drops[12] Corporate Updates Royalty Monetization In May 2025, REGENXBIO closed a non-dilutive, limited recourse royalty bond agreement of up to $250 million with Healthcare Royalty (HCRx), receiving $150 million at closing and eligible for an additional $100 million in two separate $50 million tranches - Closed a non-dilutive, limited recourse royalty bond agreement of up to $250 million with Healthcare Royalty (HCRx) in May 2025[13] - Received $150 million at closing and is eligible for an additional $100 million in two $50 million tranches[13] AbbVie Eye Care Collaboration Amendment In August 2025, REGENXBIO and AbbVie amended their collaboration and license agreement, updating the milestone structure for the diabetic retinopathy (DR) program, with AbbVie paying REGENXBIO $100 million upon the first subject dosed in the Phase IIb/III trial and an additional $100 million for a second Phase III clinical trial, and AbbVie independently advancing and funding a new Phase III ACHIEVE trial for wet AMD - Amendment to the collaboration and license agreement with AbbVie includes an updated milestone structure for the DR program[17] - AbbVie will pay REGENXBIO $100 million upon first subject dosed in the Phase IIb/III trial and an additional $100 million upon first subject dosed in a second Phase III clinical trial[17] - AbbVie will independently advance and pay all costs for a new Phase III ACHIEVE trial in wet AMD[17] Financial Results (Q2 2025) Cash Position REGENXBIO's cash, cash equivalents, and marketable securities increased to $363.6 million as of June 30, 2025, from $244.9 million at December 31, 2024, primarily driven by a $110.0 million upfront payment from the Nippon Shinyaku partnership and $144.5 million in net proceeds from the HCRx royalty monetization, partially offset by cash used in operating activities Cash, Cash Equivalents and Marketable Securities | Metric | June 30, 2025 (in millions) | December 31, 2024 (in millions) | Change | | :-------------------------------- | :-------------------------- | :-------------------------- | :----- | | Cash, cash equivalents & marketable securities | $363.6 | $244.9 | +$118.7 | - The increase was primarily attributable to a $110.0 million upfront payment from the Nippon Shinyaku partnership (March 2025) and $144.5 million in net proceeds from the royalty monetization with HCRx (May 2025)[15] Revenues Total revenues for the three months ended June 30, 2025, decreased slightly to $21.4 million from $22.3 million in the prior year period, primarily due to lower Zolgensma royalties, partially offset by an increase in service revenues, mainly from the Nippon Shinyaku partnership Revenues (Three Months Ended June 30) | Revenue Type | Q2 2025 (in thousands) | Q2 2024 (in thousands) | YoY Change (in thousands) | YoY Change (%) | | :------------------------ | :--------------------- | :--------------------- | :------------------------ | :------------- | | License and royalty revenue | $18,465 | $21,846 | $(3,381) | -15.5% | | Service revenue | $2,894 | $449 | $2,445 | +544.5% | | Total revenues | $21,359 | $22,295 | $(936) | -4.2% | - The decrease in total revenues was primarily attributable to Zolgensma royalties, which decreased from $21.8 million in Q2 2024 to $18.4 million in Q2 2025[16] - The decrease was partially offset by an increase in service revenues, driven primarily by $2.7 million of development service revenue under the Nippon Shinyaku partnership in Q2 2025[16] Operating Expenses Operating expenses increased to $84.6 million in Q2 2025 from $78.3 million in Q2 2024, primarily driven by higher research and development expenses related to pivotal trials and increased general and administrative costs Research and Development Expenses Research and development expenses increased to $59.5 million for Q2 2025, up from $48.9 million for Q2 2024, primarily due to higher manufacturing-related expenses, other clinical supply costs, and clinical trial expenses for the sura-vec and RGX-202 pivotal trials Research and Development Expenses (Three Months Ended June 30) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | YoY Change (in thousands) | YoY Change (%) | | :------------------------ | :--------------------- | :--------------------- | :------------------------ | :------------- | | Research and development | $59,500 | $48,869 | $10,631 | +21.8% | - The increase was primarily attributable to manufacturing-related expenses and other clinical supply costs and clinical trial expenses for sura-vec and RGX-202 pivotal trials[18] General and Administrative Expenses General and administrative expenses increased slightly to $19.9 million for Q2 2025, compared to $18.9 million for Q2 2024, mainly due to increased personnel-related costs and expenses for consulting and professional services General and Administrative Expenses (Three Months Ended June 30) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | YoY Change (in thousands) | YoY Change (%) | | :-------------------------- | :--------------------- | :--------------------- | :------------------------ | :------------- | | General and administrative | $19,883 | $18,855 | $1,028 | +5.5% | - The increase was primarily attributable to personnel-related costs and expenses for consulting and professional services[19] Net Loss REGENXBIO reported a net loss of $70.9 million, or $1.38 basic and diluted net loss per share, for Q2 2025, widening from a net loss of $53.0 million, or $1.05 per share, in Q2 2024, primarily driven by higher operating expenses and a significant increase in interest expense Net Loss (Three Months Ended June 30) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | YoY Change (in thousands) | YoY Change (%) | | :------------------------ | :--------------------- | :--------------------- | :------------------------ | :------------- | | Net loss | $(70,871) | $(52,989) | $(17,882) | +33.7% | | Net loss per share (basic & diluted) | $(1.38) | $(1.05) | $(0.33) | +31.4% | - The increase in net loss was influenced by higher research and development expenses and a significant increase in interest expense[18][20][30] Financial Guidance Cash Runway Guidance REGENXBIO expects its balance of cash, cash equivalents, and marketable securities, totaling $363.6 million as of June 30, 2025, to fund its operations into early 2027, with this guidance based on current operational plans and excluding the impact of potential material payments from partners or licensees, as well as the potential monetization of a Priority Review Voucher (PRV) - Cash, cash equivalents, and marketable securities of $363.6 million as of June 30, 2025, are expected to fund operations into early 2027[21] - This cash runway guidance excludes the impact of any material payments from partners or licensees and potential monetization of a PRV[21] Financial Statements Consolidated Balance Sheets As of June 30, 2025, REGENXBIO's total assets increased to $581.0 million from $466.0 million at December 31, 2024, while total liabilities significantly increased to $367.3 million from $206.3 million, primarily driven by a substantial rise in royalty monetization liabilities and deferred revenue, consequently decreasing total stockholders' equity to $213.7 million from $259.7 million Consolidated Balance Sheet Highlights (in thousands) | Metric | June 30, 2025 | December 31, 2024 | Change | | :------------------------- | :-------------- | :---------------- | :----- | | Total assets | $581,027 | $465,989 | +$115,038 | | Total liabilities | $367,348 | $206,338 | +$161,010 | | Total stockholders' equity | $213,679 | $259,651 | $(45,972) | - Royalty monetization liabilities increased significantly from $25,378 thousand at December 31, 2024, to $153,693 thousand at June 30, 2025[29] - Deferred revenue (non-current) increased from $0 to $23,804 thousand[29] Consolidated Statements of Operations and Comprehensive Loss For the six months ended June 30, 2025, total revenues significantly increased to $110.4 million from $37.9 million in the prior year period, primarily due to a substantial rise in license and royalty revenue, and despite an increase in total operating expenses, the loss from operations improved, leading to a net loss of $64.8 million, a reduction from the $116.3 million net loss in the same period of 2024, though interest expense also saw a notable increase Consolidated Statements of Operations Highlights (Six Months Ended June 30, in thousands) | Metric | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | YoY Change (in thousands) | YoY Change (%) | | :-------------------------------- | :--------------------------- | :--------------------------- | :------------------------ | :------------- | | Total revenues | $110,371 | $37,917 | $72,454 | +191.1% | | Total operating expenses | $161,522 | $157,817 | $3,705 | +2.3% | | Loss from operations | $(51,151) | $(119,900) | $68,749 | -57.3% | | Total other income (expense) | $(13,637) | $3,581 | $(17,218) | -480.8% | | Net loss | $(64,788) | $(116,319) | $51,531 | -44.3% | | Net loss per share (basic & diluted) | $(1.26) | $(2.41) | $(1.15) | -47.7% | - The substantial increase in total revenues for the six-month period was primarily driven by license and royalty revenue, which rose from $37,190 thousand to $105,514 thousand[30] - Interest expense significantly increased from $2,422 thousand in the first six months of 2024 to $19,563 thousand in the same period of 2025[30] Additional Information Conference Call Details REGENXBIO hosted a conference call and webcast on August 7, 2025, at 8:00 a.m. ET to discuss the second quarter 2025 financial results and operational highlights, with details for webcast registration and dial-in for the Q&A session provided - REGENXBIO hosted a conference call and webcast at 8:00 a.m. ET on August 7, 2025[5][22] - Listeners could register for the webcast via a provided link, and analysts could access the live call by dialing specific numbers and entering a passcode[22] Forward-Looking Statements This press release contains forward-looking statements regarding REGENXBIO's future operations, clinical trials, costs, and cash flow, based on current expectations and assumptions, which are subject to various risks and uncertainties, including the timing and success of clinical trials, regulatory approvals, and market acceptance, as detailed in the company's Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the SEC - The press release includes forward-looking statements concerning REGENXBIO's future operations, clinical trials, costs, and cash flow[24][25] - These statements are subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials, regulatory approval, and market acceptance[25] - Readers are cautioned not to rely too heavily on these statements, and REGENXBIO does not undertake any obligation to update or revise them, except as required by law[25] Contacts Contact information for REGENXBIO's Corporate Communications (Dana Cormack) and Investor Relations (George E. MacDougall) is provided for inquiries - Contact information for Corporate Communications (Dana Cormack) and Investor Relations (George E. MacDougall) is provided[27]