REGENXBIO Reports Third Quarter 2025 Financial Results and Operational Highlights

ROCKVILLE, Md., Nov. 6, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and operational highlights for the third quarter ended September 30, 2025.

"The positive data and significant clinical milestones achieved across each of our late-stage programs underscore the meaningful progress we're making towards delivering potentially transformative gene therapies," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. "Our strong momentum is driven by our differentiated, in-house end-to-end capabilities, including commercial-ready manufacturing with capacity to seize blockbuster opportunities, and a deep commitment to improve the lives of patients with Duchenne, Hunter syndrome, wet AMD and diabetic retinopathy. With the rapid advancement of our programs and leading technology platform, we are well-positioned to become a commercial company early next year."

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, including the novel construct, a proactive immune suppression regimen, and suspension-based manufacturing process delivering industry-leading product purity levels, is designed for improved muscle function, durability and safety outcomes for patients.

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.

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 Wet AMD (Subretinal Delivery)

Sura-vec for the Treatment of DR (Suprachoroidal Delivery)

Sura-vec for the Treatment of Wet AMD (Suprachoroidal Delivery)

FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities were $302.0 million as of September 30, 2025, compared to $244.9 million as of December 31, 2024. The increase was primarily attributable to the $110.0 million upfront payment received under the Nippon Shinyaku partnership in March 2025 and $144.5 million in net proceeds received from the royalty monetization with HCRx in May 2025, and was partially offset by cash used to fund operating activities during the nine months ended September 30, 2025.

Revenues: Revenues were $29.7 million for the three months ended September 30, 2025, compared to $24.2 million for the three months ended September 30, 2024. The increase was primarily attributable to $5.9 million of development service revenue under the Nippon Shinyaku partnership in the third quarter of 2025.

Research and Development Expenses: Research and development expenses were $56.1 million for the three months ended September 30, 2025, compared to $54.4 million for the three months ended September 30, 2024. The increase was primarily attributable to personnel costs and manufacturing-related expenses for clemidsogene lanparvovec.

General and Administrative Expenses: General and administrative expenses were $20.3 million for the three months ended September 30, 2025, compared to $19.4 million for the three months ended September 30, 2024. The increase was largely driven by professional services, consulting and other corporate advisory services.

Net Income: Net loss was $61.9 million, or $1.20 basic and diluted net loss per share, for the three months ended September 30, 2025, compared to a net loss of $59.6 million, or $1.17 basic and diluted net loss per share, for the three months ended September 30, 2024.

FINANCIAL GUIDANCE

REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $302.0 million as of September 30, 2025 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 potential monetization of a PRV that would be received upon potential approval of clemidsogene lanparvovec.

CONFERENCE CALL

In connection with this announcement, REGENXBIO will host a conference call and webcast at 8:00 a.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)

September 30, 2025

December 31, 2024

Assets

Current assets

Cash and cash equivalents

58,802

57,526

Marketable securities

215,403

177,161

Accounts receivable

25,347

20,473

Prepaid expenses

12,943

9,067

Other current assets

19,472

13,774

Total current assets

331,967

278,001

Marketable securities

27,838

10,179

Accounts receivable

2,765

474

Property and equipment, net

107,515

117,589

Operating lease right-of-use assets

48,820

53,716

Restricted cash

2,030

Other assets

4,268

4,000

Total assets

525,203

465,989

Liabilities and Stockholders' Equity

Current liabilities

Accounts payable

22,266

22,798

Accrued expenses and other current liabilities

38,316

38,070

Deferred revenue

13,195

115

Operating lease liabilities

9,073

7,902

Royalty monetization liabilities

41,718

34,309

Total current liabilities

124,568

103,194

Deferred revenue

21,186

—

Operating lease liabilities

67,433

74,131

Royalty monetization liabilities

149,916

25,378

Other liabilities

648

3,635

Total liabilities

363,751

206,338

Stockholders' equity

Preferred stock; no shares issued and outstanding

at September 30, 2025 and December 31, 2024

—

Common stock; 50,619 and 49,549 shares issued

and outstanding at September 30, 2025 and

December 31, 2024, respectively

Additional paid-in capital

1,220,977

1,192,536

Accumulated other comprehensive loss

(652)

(741)

Accumulated deficit

(1,058,878)

(932,149)

Total stockholders' equity

161,452

259,651

Total liabilities and stockholders' equity

525,203

465,989

REGENXBIO INC.

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(unaudited)

(in thousands, except per share data)

Three Months

Nine Months

Ended September 30,

2025

2024

2025

2024

Revenues

License and royalty revenue

23,605

23,982

129,119

61,172

Service revenue

6,128

215

10,985

942

Total revenues

29,733

24,197

140,104

62,114

Operating Expenses

Cost of license and royalty revenues

5,725

12,387

14,370

27,249

Research and development

56,101

54,429

168,688

158,142

General and administrative

20,253

19,422

60,483

56,568

Impairment of long-lived assets

—

2,101

Other operating expenses

125

Total operating expenses

82,144

86,275

243,666

244,092

Loss from operations

(52,411)

(62,078)

(103,562)

(181,978)

Other Income (Expense)

Interest income from licensing

Investment income

3,620

3,276

9,500

9,213

Interest expense

(13,169)

(820)

(32,732)

(3,242)

Total other income (expense)

(9,530)

2,481

(23,167)

6,062

Net loss

(61,941)

(59,597)

(126,729)

(175,916)

Other Comprehensive Income

Unrealized gain on available-for-sale securities, net

1,684

3,847

Total other comprehensive income

1,684

3,847

Comprehensive loss

(61,843)

(57,913)

(126,640)

(172,069)

Net loss per share, basic and diluted

(1.20)

(1.17)

(2.46)

(3.59)

Weighted-average common shares outstanding, basic and diluted

51,689

50,800

51,513

49,051

SOURCE REGENXBIO Inc.