REGENXBIO Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Operational Updates
-- Biologics Licensing Application (BLA) for clemidsogene lanparvovec
(RGX-121) submitted and on track for potential FDA approval 2H 2025;
strategic partnership with Nippon Shinyaku aims to expand potential
access and commercial opportunity in MPS II and MPS I
-- Pivotal trial of RGX-202 for Duchenne Muscular Dystrophy progressing
rapidly; enrollment completion expected in 2025 with BLA filing in
mid-2026
-- AbbVie-partnered retinal franchise continues advancing; pivotal data
evaluating the safety and efficacy of the subretinal delivery of
surabgene lomparvovec (ABBV-RGX-314) 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
ROCKVILLE, Md., March 13, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and recent operational highlights for the fourth quarter and year ended December 31, 2024.
"REGENXBIO is on the cusp of delivering potential first- or best-in-class gene therapies to market. In 2025, we have already submitted our first BLA and will accelerate late-stage development and advance commercial preparations for multiple potential product launches," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. "RGX-202 is progressing rapidly through pivotal study, continues to demonstrate a differentiated profile, and remains on track to be the next gene therapy in Duchenne. Our partnership with AbbVie continues to advance multiple large, global commercial opportunities. Now partnered with Nippon Shinyaku, RGX-121 is poised to bring another meaningful revenue stream to REGENXBIO if approved in the coming months. We remain focused on bringing multiple potentially transformative new medicines to patients."
PROGRAM HIGHLIGHTS AND MILESTONES
Neurodegenerative Disease: Clemidsogene lanparvovec (RGX-121) is a potentially first-in-class treatment for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, being developed and potentially commercialized in partnership with Nippon Shinyaku.
-- In March 2025, REGENXBIO completed its Biologics License Application
(BLA) submission to the U.S. Food and Drug Administration (FDA), seeking
accelerated approval of clemidsogene lanparvovec.
-- REGENXBIO expects potential approval of clemidsogene lanparvovec in the
second half of 2025.
-- FDA approval could result in receipt of a Priority Review Voucher (PRV).
REGENXBIO retains full rights to the PRV.
-- Clemidsogene lanparvovec remains on track to be the potential first gene
therapy and one-time treatment approved for MPS II.
Neuromuscular Disease: RGX-202 is designed to deliver a differentiated, novel microdystrophin gene for improved function and outcomes for patients living with Duchenne.
-- The pivotal Phase I/II/III AFFINITY DUCHENNE(®) trial is ongoing in
ambulatory patients and will enroll approximately 30 patients aged 1+ in
the U.S. and Canada. In November 2024, REGENXBIO announced the first
patient had been dosed in the pivotal phase.
-- The pivotal trial is nearly 50% enrolled, and REGENXBIO expects to
complete enrollment in the study in 2025, share top line data in the
first half of 2026, and submit a BLA under the accelerated approval
pathway in mid-2026.
-- Results to date from the ongoing Phase I/II trial demonstrate a
favorable safety profile with no SAEs or AEs of special interest, robust
microdystrophin expression, and improved functional outcomes at 9 and 12
months, supporting the potential of RGX-202 to be a differentiated gene
therapy in Duchenne. The latest results were published in November 2024
(press release).
-- REGENXBIO expects to share additional Phase I/II biomarker data at the
2025 Muscular Dystrophy Association (MDA) Clinical & Scientific
Conference, including the first biomarker data from the cohort of
patients aged 1-3. The company expects to share additional efficacy and
safety data, including additional functional data, in the first half of
2025.
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(TM) pivotal
trials. REGENXBIO and AbbVie expect to share topline results in 2026.
-- Enrollment in the Phase II fellow eye sub-study evaluating the
subretinal delivery of sura-vec in patients with bilateral wet AMD is
complete. Positive results from this study were presented at the 2024
American Academy of Ophthalmology annual meeting, supporting the
potential of sura-vec to treat bilateral disease at an expected
commercial launch (press release).
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
-- In March 2025, REGENXBIO announced the successful closing of its
strategic partnership with Nippon Shinyaku to develop and commercialize
RGX-121 for the treatment of MPS II and RGX-111 for MPS I in the United
States and Asia. Per the agreement, REGENXBIO will receive $110 million
up front and up to an additional $700 million if certain milestones are
achieved.
-- REGENXBIO announced the promotions of two key leaders:
-- Ram Palanki, PharmD, from Executive Vice President of Commercial
Strategy and Operations to Executive Vice President, Chief
Commercial Officer. Dr. Palanki joined the company in 2018.
-- Craig Malzahn from Senior Vice President, Technical Operations, to
Executive Vice President, Product Development and Chief Technology
Officer. Mr. Malzahn joined the company in 2019.
FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable securities were $244.9 million as of December 31, 2024, compared to $314.1 million as of December 31, 2023. The decrease was primarily driven by cash used to fund operating activities during the year ended December 31, 2024, and was partially offset by $131.1 million of aggregate net proceeds received from the follow-on public offering of the Company's common stock and pre-funded warrants completed in March 2024.
Revenues: Revenues were $21.2 million and $83.3 million for the three months and full year ended December 31, 2024, respectively, compared to $22.2 million and $90.2 million for the three months and full year ended December 31, 2023, respectively. The decreases were primarily attributable to Zolgensma royalty revenues, which decreased from $85.3 million in 2023 to $81.5 million in 2024.
Research and Development Expenses: Research and development expenses were $50.4 million and $208.5 million for the three months and full year ended December 31, 2024, respectively, compared to $55.7 million and $232.3 million for the three months and full year ended December 31, 2023, respectively. The decrease for the full year ended December 31, 2024 was primarily attributable to lower personnel-related costs, manufacturing and clinical supply costs for lead product candidates and early-stage research and development activities, and was partially offset by increases in clinical trial expenses for ABBV-RGX-314 and RGX-202. The decrease for the fourth quarter of 2024 was largely driven by personnel-related costs and clinical trial expenses as compared to the fourth quarter of 2023.
General and Administrative Expenses: General and administrative expenses were $20.1 million and $76.6 million for the three months and full year ended December 31, 2024, respectively, compared to $19.1 million and $88.5 million for the three months and full year ended December 31, 2023, respectively. The decrease for the full year ended December 31, 2024 was primarily attributable professional services, consulting fees and other corporate overhead expenses. The increase for the fourth quarter of 2024 was largely driven by stock-based compensation expense as compared to the fourth quarter of 2023.
Net Loss: Net loss was $51.2 million, or $1.01 basic and diluted net loss per share, for the three months ended December 31, 2024, compared to a net loss of $62.9 million, or $1.43 basic and diluted net loss per share, for the three months ended December 31, 2023. Net loss was $227.1 million, or $4.59 basic and diluted net loss per share, for the year ended December 31, 2024, compared to a net loss of $263.5 million, or $6.02 basic and diluted net loss per share, for the year ended December 31, 2023.
FINANCIAL GUIDANCE
REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $244.9 million as of December 31, 2024 and the $110.0 million upfront payment to be received under the Nippon Shinyaku collaboration to fund its operations into the second half of 2026. 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.
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, which will be filed with the U.S. Securities and Exchange Commission (SEC) in the first quarter of 2025, 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)
December 31, 2024 December 31, 2023
Assets
Current assets
Cash and cash equivalents $
57,526 $
34,522
Marketable securities 177,161 240,736
Accounts Receivable, net 20,473 24,790
Prepaid expenses 9,067 14,520
Other current assets 13,774 20,403
Total current assets 278,001 334,971
Marketable securities 10,179 38,871
Accounts receivable 474 701
Property and equipment, net 117,589 132,103
Operating lease right-of-use assets 53,716 60,487
Restricted cash 2,030 2,030
Other assets 4,000 4,807
Total assets $
465,989 $
573,970
Liabilities and Stockholders' Equity
Current liabilities
Accounts payable $
22,798 $
22,786
Accrued expenses and other current liabilities 38,070 49,703
Deferred revenue 115 148
Operating lease liabilities 7,902 7,068
Liability related to sale of future royalties 34,309 50,567
Total current liabilities 103,194 130,272
Operating lease liabilities 74,131 82,222
Liability related to sale of future royalties 25,378 43,485
Other liabilities 3,635 6,249
Total liabilities 206,338 262,228
Stockholders' equity
Preferred stock; no shares issued and outstanding
at December 31, 2024 and December 31, 2023
Common stock; 49,549 and 44,046 shares issued 5 4
and outstanding at December 31, 2024 and
December 31, 2023, respectively
Additional paid-in capital 1,192,536 1,021,214
Accumulated other comprehensive loss (741) (4,429)
Accumulated deficit (932,149) (705,047)
Total stockholders' equity 259,651 311,742
Total liabilities and stockholders' equity $
465,989 $
573,970
REGENXBIO INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
(in thousands, except per share data)
Three Months Years
Ended December 31, Ended December 31,
2024 2023 2024 2023
Revenues
License and royalty revenue $
21,214 $
22,213 $
83,328 $
90,242
Total revenues 21,214 22,213 83,328 90,242
Operating Expenses
Cost of revenues 6,318 11,238 33,567 37,213
Research and development 50,380 55,681 208,522 232,266
General and administrative 20,051 19,079 76,619 88,494
Credit losses (recoveries) (5,000) (5,000)
Impairment of long-lived assets 2,101
Other operating expenses 833 118 865 397
Total operating expenses 72,582 86,116 316,674 358,370
Loss from operations (51,368) (63,903) (233,346) (268,128)
Other Income (Expense)
Interest income from licensing 83 (141) 174 25
Investment income 9,516 2,366 18,729 11,319
Interest expense (9,417) (1,363) (12,659) (6,862)
Total other income 182 862 6,244 4,482
Loss before income taxes (51,186) (63,041) (227,102) (263,646)
Income Tax Benefit 152 152
Net loss $
(51,186) $
(62,889) $
(227,102) $
(263,494)
Other Comprehensive Income (Loss)
Unrealized gain (loss) on available-for-sale securities, net (159) 2,984 3,688 10,972
Total other comprehensive income (loss) (159) 2,984 3,688 10,972
Comprehensive loss $
(51,345) $
(59,905) $
(223,414) $
(252,522)
Net loss per share, basic and diluted $
(1.01) $
(1.43) $
(4.59) $
(6.02)
Weighted-average common shares outstanding, basic and diluted 50,871 44,001 49,509 43,734
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