REGENXBIO Reports Third Quarter 2026 Financial Results and Operational Highlights

Lower ZOLGENSMA royalties weighed on revenue, while a settlement gain helped narrow the quarterly loss.

Rhea-AI Summary

REGENXBIO (Nasdaq: RGNX) reported a narrower third-quarter net loss while advancing its gene therapy programs toward planned regulatory submissions. Revenue fell to $9.2 million from $29.7 million a year earlier, primarily reflecting lower ZOLGENSMA royalties after U.S. patent expiration. Net loss narrowed to $40.7 million, or $0.62 per share, from $61.9 million, or $1.20 per share; results included a $38.0 million settlement gain.

The company expects an RGX-202 application for accelerated approval in Q1 2027, with potential approval in 2H 2027, and pivotal wet AMD data with AbbVie in Q4 2026. The NAAVIGATE diabetic retinopathy trial is enrolling. RGX-121 remains on clinical hold following asymptomatic spine MRI findings in five participants. Cash, equivalents and marketable securities totaled $251.0 million; including October settlement proceeds, the company expects funding into late Q4 2027.

16 points · 2 major

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Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.

It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.

Rhea-AI Sentiment measures something else, the tone of the wording.

How the balance worksHow Sentiment works

1 major · 11 points

Hollow bars mark forward-looking points. How the balance works

Positive

• Major pointAbbVie milestone payment of $100.0 million received in July for dosing NAAVIGATE's first patient. 20% of market cap
• Major pointJuly public offering generated $107.8 million in net proceeds. 21% of market cap
• Moderate pointQ3 net loss narrowed to $40.7 million, or $0.62 per share, from $61.9 million, or $1.20.
• Moderate pointSarepta settlement payment of $39.0 million received in October, after a $38.0 million Q3 gain. 7.7% of market cap
• Moderate point. Forward-looking: it has not happened yet and may not happen.REGENXBIO expects funding into late Q4 2027 under current operational plans.
• Moderate point. Forward-looking: it has not happened yet and may not happen.RGX-202 accelerated-approval application remains planned for Q1 2027, supporting potential approval in 2H 2027.
• Moderate point. Forward-looking: it has not happened yet and may not happen.AFFINITY RISE study initiation expected in 1H 2027 to support RGX-202 global regulatory submissions.
• Moderate point. Forward-looking: it has not happened yet and may not happen.Sura-vec pivotal wet AMD data expected in Q4 2026, with global regulatory submissions expected in 2027.
• Moderate pointITVISMA generated $2.8 million in Q3 royalties; qualifying sales carry royalty rights with patents extending until 2037.
• Moderate pointQ3 cost of license and royalty revenues fell to $1.141 million from $5.725 million a year earlier.
• Moderate pointQ3 interest expense declined to $7.472 million from $13.169 million a year earlier.

5 minor points

• Minor pointCash, equivalents and marketable securities rose to $251.0 million at September 30 from $240.9 million at year-end.
• Minor pointRGX-202 intended commercial supply manufacturing continues at the company's in-house facility.
• Minor pointALTITUDE follow-up showed durable safety and efficacy through 3 years at the dose being evaluated in NAAVIGATE.
• Minor pointNAAVIGATE Phase IIb/III diabetic retinopathy trial is enrolling.
• Minor pointNine-month net loss narrowed to $108.080 million, or $1.90 per share, from $126.729 million, or $2.46.

Negative

• Major pointJuly offering issued common stock and pre-funded warrants, diluting existing holders.
• Moderate pointQ3 revenue declined to $9.2 million from $29.7 million a year earlier.
• Moderate pointRGX-121 remains under FDA clinical hold following asymptomatic spine MRI findings in five CAMPSIITE participants.
• Moderate pointQ3 R&D expenses increased to $61.0 million from $56.1 million, primarily for RGX-202 clinical trials.
• Minor pointZOLGENSMA royalties fell $21.2 million, primarily following U.S. licensed-patent expiration in January 2026.

6 minor points

• Minor pointQ3 G&A expenses were $20.6 million, versus $20.3 million a year earlier.
• Minor pointQ3 investment income declined to $2.252 million from $3.620 million a year earlier.
• Minor pointNine-month revenue declined to $123.574 million from $140.104 million a year earlier.
• Minor pointNine-month R&D expenses rose to $174.434 million from $168.688 million a year earlier.
• Minor pointNine-month G&A expenses rose to $63.501 million from $60.483 million a year earlier.
• Minor pointNine-month investment income fell to $5.477 million from $9.500 million a year earlier.

News Explained

The completed July underwritten offering raised net proceeds of $107.8 million through common stock and pre-funded warrants. The balance sheet lists 50,892 thousand shares outstanding at December 31, 2025 and 66,119 thousand at September 30, 2026, while exercised pre-funded warrants convert into shares and additional shares reduce existing holders’ ownership percentages.

Key Figures

Cash, cash equivalents and marketable securities: $251.0 million Sarepta settlement payment: $39.0 million Quarterly revenue: $9.2 million vs. $29.7 million +5 more

Cash, cash equivalents and marketable securities

$251.0 million

As of September 30, 2026

Sarepta settlement payment

$39.0 million

Received in October 2026

Quarterly revenue

$9.2 million vs. $29.7 million

Three months ended September 30, 2026 vs. 2025

R&D expenses

$61.0 million vs. $56.1 million

Three months ended September 30, 2026 vs. 2025

Net loss

$40.7 million vs. $61.9 million

Three months ended September 30, 2026 vs. 2025

Cash runway guidance

Into late Q4 2027

Based on September 30 cash and October settlement proceeds

RGX-202 BLA and potential approval timing

BLA submission in Q1 2027; potential accelerated approval in 2H 2027

Company-stated expected timeline

Sura-vec pivotal wet AMD topline data

Q4 2026

ATMOSPHERE and ASCENT trials using subretinal delivery

Previous Earnings Reports

3 past events · Latest: Aug 06

Same Type 3 events

1. Aug 06

   Quarterly earnings

   24h Move

   +7.4%

   Phase III AFFINITY DUCHENNE met its primary endpoint; cash runway extended into Q4 2027.

2. May 14

   Quarterly earnings

   24h Move

   -37.8%

   Phase III AFFINITY DUCHENNE met its primary endpoint, supporting a potential accelerated approval in 2027.

3. Nov 06

   Quarterly earnings

   24h Move

   -3.8%

   Q3 financial baseline and RGX-202 milestones were reported alongside FDA setbacks for RGX-121.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

clinical hold, npdr, ci-dme

3 terms

clinical hold regulatory

"the FDA placed a clinical hold on RGX-121"

A clinical hold is an order from a drug or medical-device regulator to stop or suspend a clinical trial or development activity because of safety concerns, inadequate study plans, or incomplete data. Think of it like a referee pausing a game until rules or safety issues are resolved; investors care because a hold can delay approval, increase costs, create uncertainty about a product’s future, and often affects a company’s valuation until the issues are addressed.

npdr medical

"subjects with non-proliferative DR (NPDR)"

Nonproliferative diabetic retinopathy (NPDR) is an early-to-moderate stage of diabetic eye disease where high blood sugar damages retinal blood vessels, causing leakage, swelling, or small hemorrhages but not new abnormal vessel growth. It matters to investors because NPDR defines a measurable patient group, clinical trial endpoints, and potential market for treatments—think of it as a warning stage that determines who needs monitoring, medical interventions, or later-stage therapies.

ci-dme medical

"without center-involved diabetic macular edema (CI-DME)"

Center-involving diabetic macular edema (CI-DME) is swelling of the macula caused by diabetes in which the fluid or retinal thickening affects the central subfield of the macula (the foveal center responsible for sharp central vision). Clinically it is identified by examination or retinal imaging (most commonly optical coherence tomography) showing intraretinal or subretinal fluid or increased thickness at the fovea; involvement of the center is the key distinction because it is more likely to reduce central visual acuity and is commonly used as an eligibility or endpoint criterion in clinical trials and treatment guidelines.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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• RGX-202 BLA submission for Duchenne muscular dystrophy on track for Q1 2027, with potential accelerated approval in 2H 2027
• Topline data from pivotal subretinal wet AMD studies, ATMOSPHERE^® and ASCENT^®, expected in Q4 2026
• Phase IIb/III NAAVIGATE trial for diabetic retinopathy ongoing
• Webcast today at 8:00 a.m. ET

ROCKVILLE, Md., Nov. 5, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and operational highlights for the third quarter ended September 30, 2026.

"We are continuing to build momentum as we prepare to launch potential first- or best-in-class gene therapies in 2027 and 2028," said Curran Simpson, President and Chief Executive Officer of REGENXBIO. "With topline pivotal data expected this quarter, sura-vec has the potential to transform the treatment paradigm for a broad spectrum of patients with wet AMD. We also remain on track for the RGX-202 BLA submission early next year as we work toward delivering this important therapy to Duchenne patients as quickly as possible. We remain focused on executing these near-term milestones and leveraging our fully integrated capabilities to deliver meaningful new treatment options for 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. The differentiated therapeutic approach behind RGX-202 includes a novel construct, with the C-Terminal domain, 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 and confirmatory trial in ambulatory patients aged 1+.

• The Company expects to have a pre-Biologics License Application (BLA) meeting with the FDA in Q4 2026 and is on track to submit a BLA in Q1 2027 under the accelerated approval pathway, supporting potential approval in 2H 2027.
• REGENXBIO continues to manufacture intended commercial supply of RGX-202 at its in-house Manufacturing Innovation Center.
• REGENXBIO expects to initiate AFFINITY^® RISE, an ex-U.S. randomized, placebo-controlled study to support RGX-202 global regulatory submissions, in 1H 2027.

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)

• REGENXBIO expects to announce topline data with AbbVie from the ATMOSPHERE^® and ASCENT^® pivotal trials of sura-vec using subretinal delivery in Q4 2026.
• Global regulatory submissions are expected in 2027.

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

• Long-term follow-up data from the ALTITUDE^® trial, presented at the Retina Society 59^th Annual Scientific Meeting in September 2026, showed a durable safety and efficacy profile through 3 years at the dose being evaluated in the Phase IIb/III NAAVIGATE study with short-course prophylactic topical steroids.
• NAAVIGATE, a multicenter, randomized, masked, sham-controlled study enrolling subjects with non-proliferative DR (NPDR) without center-involved diabetic macular edema (CI-DME) to evaluate the safety and efficacy of a one-time, in-office administration of sura-vec, is enrolling.

Neurodegenerative Disease: RGX-121 (clemidsogene lanparvovec) 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.

• In August 2026, REGENXBIO announced the FDA placed a clinical hold on RGX-121 following the discovery of asymptomatic spine MRI findings in five participants in the CAMPSIITE^® study. REGENXBIO continues to collect and evaluate additional patient imaging and long-term follow-up data.

FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable securities were $251.0 million as of September 30, 2026, compared to $240.9 million as of December 31, 2025. The increase was primarily attributable to a $100.0 million milestone payment received from AbbVie in July 2026 for the dosing of the first patient in the Phase IIb/III NAAVIGATE study and net proceeds of $107.8 million from an underwritten public offering of common stock and pre-funded warrants completed in July 2026, and was partially offset by cash used to fund operating activities during the nine months ended September 30, 2026. Cash, cash equivalents and marketable securities as of September 30, 2026 excludes a $39.0 million payment received in October 2026 pursuant to a settlement agreement with Sarepta Therapeutics.

Revenues: Revenues were $9.2 million for the three months ended September 30, 2026, compared to $29.7 million for the three months ended September 30, 2025. The decrease was primarily attributable to ZOLGENSMA^® royalty revenues, which decreased by $21.2 million due primarily to the expiration of licensed patents in the U.S. in January 2026. The decrease was partially offset by ITVISMA^® royalty revenues of $2.8 million for the third quarter of 2026. Novartis launched U.S. sales of ITVISMA in the first quarter of 2026. ITVISMA is now also approved in the EU, Japan and multiple Gulf countries. 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 $61.0 million for the three months ended September 30, 2026, compared to $56.1 million for the three months ended September 30, 2025. The increase was primarily attributable to clinical trial expenses for RGX-202.

General and Administrative (G&A) Expenses: G&A expenses were $20.6 million for the three months ended September 30, 2026, consistent with $20.3 million for the three months ended September 30, 2025.

Gain on Litigation Settlement: REGENXBIO recorded a gain of $38.0 million in the third quarter of 2026 related to a settlement agreement with Sarepta Therapeutics to resolve all patent litigation matters between the parties. The gain consisted of a $39.0 million settlement payment due from Sarepta, net of proceeds payable to certain licensors as a result of the settlement. The $39.0 million settlement payment was received from Sarepta in October 2026.

Net Loss: Net loss was $40.7 million, or $0.62 basic and diluted net loss per share, for the three months ended September 30, 2026, compared to a net loss of $61.9 million, or $1.20 basic and diluted net loss per share, for the three months ended September 30, 2025.

FINANCIAL GUIDANCE
REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $251.0 million as of September 30, 2026, along with the $39.0 million settlement proceeds received in October 2026, are sufficient to fund operations into late Q4 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
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; surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie, and RGX-121 (clemidsogene lanparvovec) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA^® and ITVISMA^®. 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. 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, 2026				December 31, 2025
Assets
Current assets
Cash and cash equivalents		$	95,795			$	34,466
Marketable securities			155,181				195,604
Accounts receivable			8,536				26,379
Prepaid expenses			13,009				11,927
Restricted cash			225				—
Other current assets			48,146				12,905
Total current assets			320,892				281,281
Marketable securities			—				10,785
Accounts receivable			450				2,312
Property and equipment, net			95,347				104,855
Operating lease right-of-use assets			42,246				47,156
Restricted cash			1,805				2,030
Other assets			5,300				4,613
Total assets		$	466,040			$	453,032
Liabilities and Stockholders' Equity
Current liabilities
Accounts payable		$	13,482			$	21,358
Accrued expenses and other current liabilities			29,611				38,390
Deferred revenue			5,985				10,452
Operating lease liabilities			6,492				8,286
Royalty monetization liabilities			12,114				39,609
Total current liabilities			67,684				118,095
Deferred revenue			16,221				18,943
Operating lease liabilities			60,054				65,215
Royalty monetization liabilities			173,564				147,408
Other liabilities			475				638
Total liabilities			317,998				350,299
Stockholders' equity
Preferred stock; no shares issued and outstanding    at September 30, 2026 and December 31, 2025			—				—
Common stock; 66,119 and 50,892 shares issued    and outstanding at September 30, 2026 and    December 31, 2025, respectively			7				5
Additional paid-in capital			1,383,187				1,229,442
Accumulated other comprehensive loss			(1,045)				(687)
Accumulated deficit			(1,234,107)				(1,126,027)
Total stockholders' equity			148,042				102,733
Total liabilities and stockholders' equity		$	466,040			$	453,032

 

REGENXBIO INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
(in thousands, except per share data)
		Three Months								Nine Months
		Ended September 30,								Ended September 30,
		2026				2025				2026				2025
Revenues
License and royalty revenue		$	5,244			$	23,605			$	114,175			$	129,119
Service revenue			3,918				6,128				9,399				10,985
Total revenues			9,162				29,733				123,574				140,104
Operating Expenses (Income)
Cost of license and royalty revenues			1,141				5,725				13,257				14,370
Research and development			61,009				56,101				174,434				168,688
General and administrative			20,579				20,253				63,501				60,483
Gain on litigation settlement			(38,026)				—				(38,026)				—
Other operating expenses (income)			(7)				65				41				125
Total operating expenses			44,696				82,144				213,207				243,666
Loss from operations			(35,534)				(52,411)				(89,633)				(103,562)
Other Income (Expense)
Interest income from licensing			16				19				49				65
Investment income			2,252				3,620				5,477				9,500
Interest expense			(7,472)				(13,169)				(23,973)				(32,732)
Total other income (expense)			(5,204)				(9,530)				(18,447)				(23,167)
Net loss		$	(40,738)			$	(61,941)			$	(108,080)			$	(126,729)
Other Comprehensive Income (Loss)
Unrealized gain (loss) on available-for-sale securities, net			(170)				98				(358)				89
Total other comprehensive income (loss)			(170)				98				(358)				89
Comprehensive loss		$	(40,908)			$	(61,843)			$	(108,438)			$	(126,640)
Net loss per share, basic and diluted		$	(0.62)			$	(1.20)			$	(1.90)			$	(2.46)
Weighted-average common shares outstanding, basic and diluted			65,263				51,689				56,884				51,513

 

 

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SOURCE REGENXBIO Inc.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

When does REGENXBIO expect to submit RGX-202 for FDA approval?

REGENXBIO expects to submit its RGX-202 biologics license application under the accelerated approval pathway in Q1 2027, supporting potential approval in 2H 2027. A pre-application meeting with the FDA is expected in Q4 2026.

What does REGENXBIO's cash runway guidance exclude?

REGENXBIO's guidance excludes potential material partner or licensee payments tied to development, regulatory, approval or commercialization milestones, and additional potential dilutive or non-dilutive funding. Its expected runway into late Q4 2027 is based on current operational plans.

What patients is REGENXBIO enrolling in the NAAVIGATE trial?

NAAVIGATE is enrolling patients with non-proliferative diabetic retinopathy without center-involved diabetic macular edema. The randomized, masked, sham-controlled Phase IIb/III study evaluates the safety and efficacy of a one-time, in-office administration of sura-vec.

What is REGENXBIO doing following the RGX-121 clinical hold?

REGENXBIO continues to collect and evaluate additional patient imaging and long-term follow-up data. The FDA placed RGX-121 on clinical hold following the discovery of asymptomatic spine MRI findings in five participants in the CAMPSIITE study.