REGENXBIO Reports First Quarter 2026 Financial Results and Operational Highlights
ROCKVILLE, Md., May 14, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and operational highlights for the first quarter ended March 31, 2026.
"REGENXBIO enters a transformative year with positive momentum, reaching significant late-stage milestones to support our potential first- and best-in-class gene therapies," said Curran Simpson, President and Chief Executive Officer, REGENXBIO. "On our mission to bring meaningful new therapies to rare disease communities facing limited options, we remain focused on advancing RGX-202 for Duchenne toward potential BLA submission and are excited to share topline pivotal data in our webcast today; we also continue engaging with the FDA regarding a potential path forward for RGX-121 for Hunter syndrome. In chronic retinal diseases, we are executing against our multi-indication strategy and preparing for the Phase 3 wet AMD readout with partner AbbVie. Additionally, we continue harnessing internal manufacturing capabilities to support anticipated commercial needs. We remain well-positioned to capitalize on several near-term, high-value opportunities and bring these highly differentiated treatments to patients."
PROGRAM HIGHLIGHTS AND MILESTONES
Neuromuscular Disease: RGX-202 is a potential best-in-class gene therapy for Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to address the underlying cause of Duchenne by enabling targeted expression of a novel microdystrophin that is closest to naturally occurring dystrophin. It is the only microdystrophin that includes the C-Terminal domain, which has been shown to protect and preserve muscle function. The differentiated therapeutic approach behind RGX-202 includes a novel construct, a proactive immune suppression regimen, and a suspension-based manufacturing process that delivers industry-leading product purity levels. RGX-202 is designed for improved muscle function, durability and positive safety outcomes and is being evaluated in the Phase I/II/III AFFINITY DUCHENNE ® trial in ambulatory patients aged 1+.
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1 30 of 31 total participants have Week 12 biopsy available for evaluation; one participant refused muscle biopsy.
Retinal Disease: Surabgene lomparvovec (sura-vec, ABBV-RGX-314), developed in collaboration with AbbVie, is potentially the first-in-class gene therapy treatment for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).
Sura-vec for the Treatment of Wet AMD (Subretinal Delivery)
Sura-vec for the Treatment of DR (Suprachoroidal Delivery)
Sura-vec for the Treatment of Wet AMD (Suprachoroidal Delivery)
Neurodegenerative Disease: Clemidsogene lanparvovec (RGX-121) is a potential first-in-class treatment for Mucopolysaccharidosis (MPS) Type II, also known as Hunter syndrome. RGX-111 is an investigational one-time treatment for severe MPS I, also known as Hurler syndrome. These programs are partnered with Nippon Shinyaku.
FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable securities were $150.5 million as of March 31, 2026, compared to $240.9 million as of December 31, 2025. The decrease was primarily driven by cash used to fund operating activities during the first quarter of 2026. Cash used in operations in the first quarter of 2026 included a non-recurring payment of $10.0 million under a previously announced settlement agreement with GlaxoSmithKline in March 2026, and is otherwise generally consistent with historical first quarter cash spend.
Revenues: Revenues were $6.4 million for the three months ended March 31, 2026, compared to $89.0 million for the three months ended March 31, 2025. The decrease was primarily attributable to $70.0 million of upfront license recognized under the collaboration with Nippon Shinyaku in the first quarter of 2025, as well as a $12.2 million decrease in ZOLGENSMA ® royalty revenues due to the expiration of licensed patents in the U.S. in January 2026. Novartis launched U.S. sales of ITVISMA ® in the first quarter of 2026. REGENXBIO is entitled to royalties on certain net sales of ITVISMA in the U.S. and other jurisdictions with patents extending until 2037.
Research and Development (R&D) Expenses: R&D expenses were $57.3 million for the three months ended March 31, 2026 compared to $53.1 million for the three months ended March 31, 2025. The increase was primarily attributable to clinical trial expenses for RGX-202 and personnel-related costs.
General and Administrative Expenses: General and administrative expenses were $21.3 million for the three months ended March 31, 2026 compared to $20.3 million for the three months ended March 31, 2025. The increase was largely driven by personnel-related costs, consulting and corporate advisory services.
Net Income: Net loss was $90.1 million, or $1.72 basic and diluted net loss per share, for the three months ended March 31, 2026, compared to net income of $6.1 million, or $0.12 basic and diluted net income per share, for the three months ended March 31, 2025.
FINANCIAL GUIDANCE
REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $150.5 million as of March 31, 2026 to fund its operations into early 2027. This cash runway guidance is based on the Company's current operational plans and excludes the impact of any material payments that may potentially be received from partners or licensees upon the achievement of development or regulatory milestones, or upon the approval or commercialization of product candidates, and excludes any additional potential dilutive or non-dilutive funding opportunities.
CONFERENCE CALL
REGENXBIO will host a conference call and webcast at 8:00 a.m. ET today to discuss the RGX-202 topline data as well as first quarter 2026 updates. The live webcast can be accessed here and in the Investor section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.
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, 2025, filed with the U.S. Securities and Exchange Commission (SEC) 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 ® and Itvisma ® are registered trademarks of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.
CONTACTS:
Dana Cormack
Corporate Communications
[email protected]
George E. MacDougall
Investor Relations
[email protected]
REGENXBIO INC.
CONSOLIDATED BALANCE SHEETS
(unaudited)
(in thousands)
March 31, 2026
December 31, 2025
Assets
Current assets
Cash and cash equivalents
$
15,229
$
34,466
Marketable securities
135,262
195,604
Accounts receivable
10,038
26,379
Prepaid expenses
11,543
11,927
Other current assets
14,444
12,905
Total current assets
186,516
281,281
Marketable securities
—
10,785
Accounts receivable
420
2,312
Property and equipment, net
101,874
104,855
Operating lease right-of-use assets
45,541
47,156
Restricted cash
2,030
2,030
Other assets
5,513
4,613
Total assets
$
341,894
$
453,032
Liabilities and Stockholders' Equity
Current liabilities
Accounts payable
$
21,207
$
21,358
Accrued expenses and other current liabilities
21,892
38,390
Deferred revenue
5,919
10,452
Operating lease liabilities
7,867
8,286
Royalty monetization liabilities
14,225
39,609
Total current liabilities
71,110
118,095
Deferred revenue
22,776
18,943
Operating lease liabilities
63,199
65,215
Royalty monetization liabilities
163,105
147,408
Other liabilities
622
638
Total liabilities
320,812
350,299
Stockholders' equity
Preferred stock; no shares issued and outstanding
at March 31, 2026 and December 31, 2025
—
—
Common stock; 51,617 and 50,892 shares issued
and outstanding at March 31, 2026 and
December 31, 2025, respectively
5
5
Additional paid-in capital
1,238,025
1,229,442
Accumulated other comprehensive loss
(870)
(687)
Accumulated deficit
(1,216,078)
(1,126,027)
Total stockholders' equity
21,082
102,733
Total liabilities and stockholders' equity
$
341,894
$
453,032
REGENXBIO INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)
(unaudited)
(in thousands, except per share data)
Three Months Ended March 31,
2026
2025
Revenues
License and royalty revenue
$
5,090
$
87,049
Service revenue
1,303
1,963
Total revenues
6,393
89,012
Operating Expenses
Cost of license and royalty revenues
11,074
3,436
Research and development
57,339
53,087
General and administrative
21,306
20,347
Other operating expenses
36
15
Total operating expenses
89,755
76,885
Income (loss) from operations
(83,362)
12,127
Other Income (Expense)
Interest income from licensing
16
25
Investment income
2,003
2,501
Interest expense
(8,708)
(8,570)
Total other income (expense)
(6,689)
(6,044)
Net income (loss)
$
(90,051)
$
6,083
Other Comprehensive Loss
Unrealized loss on available-for-sale securities, net
(183)
(21)
Total other comprehensive loss
(183)
(21)
Comprehensive income (loss)
$
(90,234)
$
6,062
Net income (loss) per share:
Basic
$
(1.72)
$
0.12
Diluted
$
(1.72)
$
0.12
Weighted-average common shares outstanding:
Basic
52,428
51,362
Diluted
52,428
51,434
SOURCE REGENXBIO Inc.