REGENXBIO Reports First Quarter 2025 Financial Results and Recent Operational Highlights
- Net income of $6.1 million in Q1 2025, compared to $63.3 million loss in Q1 2024
- Revenue increased significantly to $89.0 million from $15.6 million YoY
- Strong cash position of $272.7 million, funding operations into H2 2026
- Strategic partnership with Nippon Shinyaku secured $110 million upfront with potential $700 million in milestone payments
- RGX-202 showing robust microdystrophin expression (up to 122.3% compared to control)
- Research and development expenses remain high at $53.1 million
- Increased general and administrative expenses to $20.3 million from $18.3 million YoY
Insights
REGENXBIO's gene therapy pipeline shows solid advancement with multiple catalysts ahead, now profitable with strong cash runway into H2 2026.
REGENXBIO's Q1 results reveal notable momentum across their gene therapy pipeline with three late-stage programs targeting commercial launches between 2025-2027. Their lead candidate RGX-202 for Duchenne muscular dystrophy (DMD) is progressing well with the pivotal AFFINITY DUCHENNE trial more than 50% enrolled and on track for completion in 2025. The microdystrophin expression data is particularly encouraging, showing 122.3% expression versus control in a 3-year-old patient and 31.5% in a 7-year-old patient, suggesting robust gene transduction.
The regulatory pathway is crystallizing with a BLA submission planned for mid-2026 under the accelerated approval pathway, positioning RGX-202 for potential 2027 launch when "the majority of the prevalent market will still be available." Commercial manufacturing preparations are underway with production starting in Q3 2025.
Their partnership with Nippon Shinyaku for clemidsogene lanparvovec (RGX-121) for MPS II is progressing toward a near-term catalyst with FDA BLA acceptance expected this month (May 2025) and potential approval in H2 2025. This represents a potential first-in-class gene therapy for Hunter syndrome and could yield a valuable Priority Review Voucher.
The AbbVie collaboration for surabgene lomparvovec (ABBV-RGX-314) continues advancing in both wet AMD and diabetic retinopathy indications. The subretinal delivery program for wet AMD has pivotal trials ongoing with data expected in 2026, while the diabetic retinopathy program is moving toward Phase III planning.
Financially, REGENXBIO showed considerable improvement, with $89 million in revenue (versus $15.6M in Q1 2024), driven by $71.8 million from the Nippon Shinyaku partnership. The company achieved profitability with $6.1 million in net income ($0.12 per share) compared to a $63.3 million loss in the year-ago period. Cash position strengthened to $272.7 million, extending runway into H2 2026, providing sufficient capital to reach multiple value-inflection points.
The company's internal manufacturing capabilities represent a strategic advantage for maintaining control over production quality and reducing reliance on third parties, particularly crucial for complex gene therapies where manufacturing consistency is critical for regulatory approval.
- RGX-202 in Duchenne muscular dystrophy on track for BLA submission mid-2026
- Pivotal trial more than half enrolled, with completion expected in 2025
- Additional Phase I/II functional data expected 1H 2025
- Initiating commercial supply manufacturing in Q3 2025
- FDA acceptance of Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121, partnered with Nippon Shinyaku) expected in May 2025
- Pivotal data evaluating the safety and efficacy of the subretinal delivery of surabgene lomparvovec (ABBV-RGX-314, partnered with AbbVie) in patients with wet age-related macular degeneration are expected in 2026 and planning of diabetic retinopathy pivotal study continues
- Conference call today at 4:30 p.m. ET
"We have made tremendous progress towards delivering multiple commercial gene therapies, starting this year," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. "Each of our late-stage assets is demonstrating differentiation against standard of care or available treatments, giving us a pipeline of potential first- or best-in-class gene therapies for rare and retinal diseases. With advanced clinical programs, commercial-ready manufacturing in-house at our
PROGRAM HIGHLIGHTS AND MILESTONES
Neuromuscular Disease: RGX-202 is designed to use the NAV® AAV8 vector to deliver a differentiated, novel microdystrophin gene for improved muscle function and outcomes for patients living with Duchenne. It is the only investigational or approved microdystrophin gene therapy construct to include the C-Terminal (CT) domain found in naturally occurring dystrophin.
- The pivotal Phase I/II/III AFFINITY DUCHENNE® trial of RGX-202 is ongoing in ambulatory patients and is expected to enroll approximately 30 patients aged 1+ in the
U.S. andCanada by 2025, with more than half already enrolled to support the pivotal dataset. - REGENXBIO expects to share topline data in the first half of 2026 and submit a Biologics License Application (BLA) under the accelerated approval pathway in mid-2026.
- Commercial readiness activities are underway to support an expected launch in 2027, when the majority of the prevalent market will still be available. REGENXBIO has manufactured RGX-202 supply for both clinical and confirmatory trials with the highest product purity levels in Duchenne gene therapies and expects to initiate commercial supply manufacturing in Q3 2025, with all production sourced in the
U.S. - Data continue to show consistent, robust expression and transduction of RGX-202 microdystrophin across all ages, supported by new biomarker data from two patients who received the pivotal dose of RGX-202 in the Phase I/II portion of the trial, which were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in March.
- In a patient aged 3 years at dosing, microdystrophin expression was measured to be
122.3% compared to control. In a patient aged 7 years old, RGX-202 microdystrophin expression was measured to be31.5% compared to control.
- In a patient aged 3 years at dosing, microdystrophin expression was measured to be
- REGENXBIO expects to share additional functional, efficacy, and safety data in the first half of 2025.
Neurodegenerative Disease: Clemidsogene lanparvovec (RGX-121) is a potential first-in-class treatment for MPS II, also known as Hunter syndrome, being developed and potentially commercialized in partnership with Nippon Shinyaku.
- This month, REGENXBIO expects the
U.S. Food and Drug Administration (FDA) to accept the BLA submitted in March 2025, and potential approval of clemidsogene lanparvovec in the second half of 2025. FDA approval could result in receipt of a Priority Review Voucher (PRV), to which REGENXBIO retains full rights. - Clemidsogene lanparvovec remains on track to be the potential first gene therapy and one-time treatment approved for MPS II.
Retinal Disease: Surabgene lomparvovec (sura-vec, ABBV-RGX-314), developed in collaboration with AbbVie, is potentially the first-in-class treatment for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).
Sura-vec for the Treatment of DR (Suprachoroidal Delivery)
- AbbVie and REGENXBIO announced in January 2025 that they will plan a Phase III clinical program. The program is expected to support global regulatory submissions.
- The Phase II ALTITUDE® trial is enrolling a cohort of patients with center-involved diabetic macular edema (DME). Patients will receive a one-time, in-office injection of sura-vec at dose level 4 (1.5x10e12 GC/eye) with short-course prophylactic steroid eye drops.
Sura-vec for the Treatment of Wet AMD (Subretinal Delivery)
- Enrollment is ongoing in the ATMOSPHERE® and ASCENT™ pivotal trials. REGENXBIO and AbbVie expect to share topline results in 2026. Sura-vec is on track to be the first approved gene therapy for wet AMD.
Sura-vec for the Treatment of 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). Patients in this cohort will also receive short-course prophylactic steroid eye drops.
CORPORATE UPDATES
Partnership with Nippon Shinyaku
- In March 2025, REGENXBIO announced the successful closing of its strategic partnership with Nippon Shinyaku to develop and commercialize clemidsogene lanparvovec for the treatment of MPS II and RGX-111 for the treatment of MPS I in
the United States andAsia . Per the agreement, REGENXBIO received$110 million $700 million
FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable securities were
Revenues: Revenues were
Research and Development Expenses: Research and development expenses were
General and Administrative Expenses: General and administrative expenses were
Net Income: Net income was
FINANCIAL GUIDANCE
REGENXBIO expects its balance in cash, cash equivalents and marketable securities of
CONFERENCE CALL
In connection with this announcement, REGENXBIO will host a conference call and webcast at 4:30 p.m. ET today. Listeners can register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time.
ABOUT REGENXBIO Inc.
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 www.REGENXBIO.com.
FORWARD-LOOKING STATEMENTS
This 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, clinical trials, costs and cash flow. 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 timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timing or likelihood of payments from AbbVie or Nippon Shinyaku, the monetization of any priority review voucher, 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 WWW.SEC.GOV. 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 Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.
CONTACTS:
Dana Cormack
Corporate Communications
Dcormack@regenxbio.com
George E. MacDougall
Investor Relations
IR@regenxbio.com
REGENXBIO INC. | ||||||||
CONSOLIDATED BALANCE SHEETS | ||||||||
(unaudited) | ||||||||
(in thousands) | ||||||||
March 31, 2025 | December 31, 2024 | |||||||
Assets | ||||||||
Current assets | ||||||||
Cash and cash equivalents | $ | 130,104 | $ | 57,526 | ||||
Marketable securities | 137,765 | 177,161 | ||||||
Accounts receivable | 18,192 | 20,473 | ||||||
Prepaid expenses | 11,094 | 9,067 | ||||||
Other current assets | 16,082 | 13,774 | ||||||
Total current assets | 313,237 | 278,001 | ||||||
Marketable securities | 4,858 | 10,179 | ||||||
Accounts receivable | 1,208 | 474 | ||||||
Property and equipment, net | 114,497 | 117,589 | ||||||
Operating lease right-of-use assets | 52,112 | 53,716 | ||||||
Restricted cash | 2,030 | 2,030 | ||||||
Other assets | 2,987 | 4,000 | ||||||
Total assets | $ | 490,929 | $ | 465,989 | ||||
Liabilities and Stockholders' Equity | ||||||||
Current liabilities | ||||||||
Accounts payable | $ | 19,044 | $ | 22,798 | ||||
Accrued expenses and other current liabilities | 23,597 | 38,070 | ||||||
Deferred revenue | 13,588 | 115 | ||||||
Operating lease liabilities | 7,942 | 7,902 | ||||||
Liability related to sale of future royalties | 42,560 | 34,309 | ||||||
Total current liabilities | 106,731 | 103,194 | ||||||
Deferred revenue | 26,297 | — | ||||||
Operating lease liabilities | 71,906 | 74,131 | ||||||
Liability related to sale of future royalties | 11,118 | 25,378 | ||||||
Other liabilities | 680 | 3,635 | ||||||
Total liabilities | 216,732 | 206,338 | ||||||
Stockholders' equity | ||||||||
Preferred stock; no shares issued and outstanding | — | — | ||||||
Common stock; 50,117 and 49,549 shares issued | 5 | 5 | ||||||
Additional paid-in capital | 1,201,020 | 1,192,536 | ||||||
Accumulated other comprehensive loss | (762) | (741) | ||||||
Accumulated deficit | (926,066) | (932,149) | ||||||
Total stockholders' equity | 274,197 | 259,651 | ||||||
Total liabilities and stockholders' equity | $ | 490,929 | $ | 465,989 | ||||
REGENXBIO INC. | ||||||||
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) | ||||||||
(unaudited) | ||||||||
(in thousands, except per share data) | ||||||||
Three Months Ended March 31, | ||||||||
2025 | 2024 | |||||||
Revenues | ||||||||
License and royalty revenue | $ | 87,049 | $ | 15,344 | ||||
Service revenue | 1,963 | 278 | ||||||
Total revenues | 89,012 | 15,622 | ||||||
Operating Expenses | ||||||||
Cost of license and royalty revenues | 3,436 | 4,283 | ||||||
Research and development | 53,087 | 54,844 | ||||||
General and administrative | 20,347 | 18,291 | ||||||
Impairment of long-lived assets | — | 2,101 | ||||||
Other operating expenses (income) | 15 | (34) | ||||||
Total operating expenses | 76,885 | 79,485 | ||||||
Income (loss) from operations | 12,127 | (63,863) | ||||||
Other Income (Expense) | ||||||||
Interest income from licensing | 25 | 37 | ||||||
Investment income | 2,501 | 2,469 | ||||||
Interest expense | (8,570) | (1,973) | ||||||
Total other income (expense) | (6,044) | 533 | ||||||
Net income (loss) | $ | 6,083 | $ | (63,330) | ||||
Other Comprehensive Income (Loss) | ||||||||
Unrealized gain (loss) on available-for-sale securities, net | (21) | 1,200 | ||||||
Total other comprehensive income (loss) | (21) | 1,200 | ||||||
Comprehensive income (loss) | $ | 6,062 | $ | (62,130) | ||||
Net income (loss) per share: | ||||||||
Basic | $ | 0.12 | $ | (1.38) | ||||
Diluted | $ | 0.12 | $ | (1.38) | ||||
Weighted-average common shares outstanding: | ||||||||
Basic | 51,362 | 45,733 | ||||||
Diluted | 51,434 | 45,733 | ||||||
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SOURCE REGENXBIO Inc.
FAQ
What were REGENXBIO's (RGNX) Q1 2025 financial results?
When is REGENXBIO (RGNX) expecting BLA submission for RGX-202 in Duchenne muscular dystrophy?
What is the status of REGENXBIO's partnership with Nippon Shinyaku?
When will REGENXBIO (RGNX) share pivotal data for surabgene lomparvovec in wet AMD?
How long will REGENXBIO's current cash position fund operations?
