REGENXBIO Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Operational Highlights

    --  Focus on clinical stage AAV Therapeutic product candidates addressing
        large commercial opportunities and value generation
    --  Prioritized pipeline is expected to further progress to pivotal stage
        and first BLA filing in 2024
    --  New updates planned for Duchenne and in-office delivery retinal disease
        programs starting in March
    --  $314 million in cash, cash equivalents and marketable securities as of
        December 31, 2023, expected to fund operational runway into the second
        half of 2025
    --  Conference call Tuesday, February 27, at 4:30 p.m. ET

ROCKVILLE, Md., Feb. 27, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the fourth quarter and full year ended December 31, 2023, and recent operational highlights, including progress on the strategic pipeline prioritization and corporate restructuring intended to significantly reduce operating expenses and support meaningful value generation from the Company's strong pipeline of AAV Therapeutics.

"We started 2024 with amazing data from our AbbVie-partnered eye care programs and our treatments for Duchenne and Hunter syndrome. Our strategic pipeline prioritization at the end of 2023 created a sharpened focus for us and today we are rapidly advancing products through late-stage clinical trials. We believe this is the best way to support the creation of meaningful value," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We expect more important data readouts and milestones to be achieved across all programs and the initiation of new pivotal trials this year. 2024 will be a turning point in our journey to bring ground-breaking AAV Therapeutics to millions of patients."

PROGRAM HIGHLIGHTS AND MILESTONES

Retinal Disease: ABBV-RGX?314, in collaboration with AbbVie, is a potential one-time treatment for wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other chronic retinal disease that is designed to continually produce an anti-VEGF protein in the eye. ABBV-RGX-314 is currently being evaluated in patients with wet AMD and DR in nine ongoing clinical trials, including two pivotal trials. A single ABBV-RGX-314 treatment has the potential to become a new standard-of-care option among anti-VEGF treatments by sustaining vision health long term and overcoming the clinical challenges of managing retinal disease due to the treatment burden of chronic anti-VEGF injections.

    --  ABBV-RGX-314 Subretinal Delivery for the Treatment of Wet AMD
        --  Enrollment is on track in ATMOSPHERE(®) and ASCENT(TM) pivotal
            trials and these trials are expected to support global regulatory
            submissions with the U.S. Food and Drug Administration and the
            European Medicines Agency in late 2025 through the first half of
            2026.
    --  ABBV-RGX-314 Suprachoroidal Delivery for Treatment of Wet AMD
        --  REGENXBIO expects to share new program and data updates for the
            Phase II AAVIATE trial in mid-2024.
        --  In January 2024, REGENXBIO presented data from the AAVIATE(®) trial
            demonstrating that, at six months, patients treated with
            ABBV-RGX-314 continue to demonstrate stable vision and retinal
            anatomy while a meaningful reduction in anti-VEGF treatment burden
            was observed. The highest reduction was seen in dose level 3,
            demonstrating an 80% reduction in annualized injection rate with 50%
            of patients remaining injection-free.
    --  ABBV-RGX-314 Suprachoroidal Delivery for Treatment of DR
        --  REGENXBIO expects to share new program and data updates for the
            Phase II ALTITUDE(®) trial in Q2 2024.
        --  In November 2023, REGENXBIO presented data from the ALTITUDE trial
            showing that, at one year, dose level 2 in non-proliferative DR
            patients prevented disease progression as measured by the Early
            Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity
            Scale. Dose level 2 reduced the risk of patients developing
            vision-threatening events by 89%.

Neuromuscular Disease: RGX-202 is an investigational one-time AAV therapeutic designed to meaningfully impact disease by delivering a gene for a novel microdystrophin with important biology most similar to naturally occurring dystrophin that protects from the muscle degradation associated with Duchenne.

    --  REGENXBIO plans to share new updates from the Phase I/II AFFINITY
        DUCHENNE(® )trial at the Muscular Dystrophy Association Clinical and
        Scientific Meeting being held next week in Orlando, FL and virtually.
    --  REGENXBIO expects to make a pivotal dose determination in mid-2024. The
        Company also expects to share initial strength and functional assessment
        data for both dose levels and the initiation of a pivotal program in the
        second half of 2024.
    --  In February 2024, REGENXBIO reported interim data from the AFFINITY
        DUCHENNE trial, demonstrating that all three patients at dose level 1
        indicate encouraging increases in expression of RGX-202 microdystrophin
        at three months and reduction from baseline in serum creatinine kinase
        levels, supporting evidence of clinical improvement. In the third
        patient, RGX-202 microdystrophin expression was measured to be 83.4%.
    --  REGENXBIO plans to use RGX-202 microdystrophin expression as a surrogate
        endpoint to support a Biologics License Application (BLA) filing using
        the accelerated approval pathway.

Neurodegenerative Disease: RGX-121 is an investigational one-time AAV therapeutic designed to change the course of disease by restoring the gene missing in boys with MPS II.

    --  On track to file a BLA in 2024 using the accelerated approval pathway.
        Approval of the planned BLA could result in receipt of a Priority Review
        Voucher in 2025.
    --  In February 2024, REGENXBIO reported the pivotal phase of the
        CAMPSIITE(®) trial achieved its primary endpoint, as treated patients
        achieved decreased cerebrospinal fluid (CSF) levels of D2S6 below
        maximum attenuated disease levels at 16 weeks (p value of 0.00016).
        Patients treated with RGX-121 have showed continued improvement in
        neurodevelopmental skill acquisition up to four years and discontinued
        intravenous enzyme therapy.

NAV(®) TECHNOLOGY PLATFORM LICENSEE PROGRAM HIGHLIGHTS

Novartis AG reported fourth quarter and full year 2023 global sales of Zolgensma, for the treatment of spinal muscular atrophy, of $286 million and $1.21 billion, respectively. Novartis, Rocket Pharmaceuticals and Ultragenyx Pharmaceutical all have investigational AAV Therapeutics in pivotal phase that have multiple milestones expected throughout 2024. Eli Lilly is also developing several AAV Therapeutics in Phase II for neurodegenerative diseases using REGENXBIO NAV Technology.

FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities were $314.1 million as of December 31, 2023, compared to $565.2 million as of December 31, 2022. The decrease was primarily driven by cash used to fund operating activities during the year ended December 31, 2023.

Revenues: Revenues were $22.2 million and $90.2 million for the three months and full year ended December 31, 2023, respectively, compared to $31.3 million and $112.7 million for the three months and full year ended December 31, 2022, respectively. The decreases were primarily attributable to Zolgensma royalty revenues, which decreased from $101.9 million for the year ended December 31, 2022 to $85.3 million for the year ended December 31, 2023.

Research and Development Expenses: Research and development expenses were $55.7 million and $232.3 million for the three months and full year ended December 31, 2023, respectively, compared to $62.5 million and $242.5 million for the three months and full year ended December 31, 2022, respectively. The decreases were primarily attributable to clinical trial and manufacturing expenses for ABBV-RGX-314 resulting from an increase in development cost reimbursement from AbbVie under our eye care collaboration and were partially offset by increased clinical trial expenses for our other lead product candidates.

General and Administrative Expenses: General and administrative expenses were $19.1 million and $88.5 million for the three months and full year ended December 31, 2023, respectively, compared to $21.2 million and $85.3 million for the three months and full year ended December 31, 2022, respectively. The increase for the full year ended December 31, 2023 was primarily attributable to personnel-related costs, expenses for professional services and other corporate overhead costs, and was partially offset by a decrease in these costs in the fourth quarter of 2023 as compared to the fourth quarter of 2022.

Net Loss: Net loss was $62.9 million, or $1.43 basic and diluted net loss per share, for the three months ended December 31, 2023, compared to a net loss of $59.9 million, or $1.38 basic and diluted net loss per share, for the three months ended December 31, 2022. Net loss was $263.5 million, or $6.02 basic and diluted net loss per share, for the year ended December 31, 2023, compared to a net loss of $280.3 million, or $6.50 basic and diluted net loss per share, for the year ended December 31, 2022.

FINANCIAL GUIDANCE

REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $314.1 million as of December 31, 2023 to fund its operations into the second half of 2025. This cash runway guidance is based on the Company's current operational plans and excludes the impact of any payments that may be received from AbbVie upon the achievement of development or commercial milestones under our ABBV-RGX-314 collaboration.

CONFERENCE CALL

In connection with this announcement, REGENXBIO will host a conference call and webcast today at 4:30 p.m. ET. 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 leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics 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 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, 2023, which will be filed with the SEC in the first quarter of 2024 and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (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.


                                                              
       
             REGENXBIO INC.

                                                           
        
        CONSOLIDATED BALANCE SHEETS

                                                               
       
             (unaudited)

                                                              
       
             (in thousands)


                                                                                                           December 31, 2023                December 31, 2022



           
              Assets



           Current assets



           Cash and cash equivalents                                                             $
        34,522                $
         96,952



           Marketable securities                                                                      240,736                      267,690



           Accounts receivable, net                                                                    24,790                       28,082



           Prepaid expenses                                                                            14,520                       13,900



           Other current assets                                                                        20,403                        9,352



           Total current assets                                                                       334,971                      415,976



           Marketable securities                                                                       38,871                      200,560



           Accounts receivable, net                                                                       701                        1,504



           Property and equipment, net                                                                132,103                      141,685



           Operating lease right-of-use assets                                                         60,487                       65,116



           Restricted cash                                                                              2,030                        2,030



           Other assets                                                                                 4,807                        6,397



           Total assets                                                                         $
        573,970               $
         833,268



           
              Liabilities and Stockholders' Equity



           Current liabilities



           Accounts payable                                                                      $
        22,786                $
         27,213



           Accrued expenses and other current liabilities                                              49,703                       46,794



           Deferred revenue                                                                               148                        1,829



           Operating lease liabilities                                                                  7,068                        5,997



           Liability related to sale of future royalties                                               50,567                       48,601



           Total current liabilities                                                                  130,272                      130,434



           Operating lease liabilities                                                                 82,222                       88,802



           Liability related to sale of future royalties                                               43,485                       89,005



           Other liabilities                                                                            6,249                        8,832



           Total liabilities                                                                          262,228                      317,073



           Stockholders' equity



           Preferred stock; no shares issued and outstanding
      at December 31, 2023 and 2022



           Common stock; 44,046 and 43,299 shares issued                                                    4                            4
      and outstanding at December 31, 2023 and
      2022, respectively



           Additional paid-in capital                                                               1,021,214                      973,145



           Accumulated other comprehensive loss                                                       (4,429)                    (15,401)



           Accumulated deficit                                                                      (705,047)                   (441,553)



           Total stockholders' equity                                                                 311,742                      516,195



           Total liabilities and stockholders' equity                                           $
        573,970               $
         833,268


                                                                         
              
                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,


                                                                                                                              2023                           2022                2023                           2022



     
                Revenues



     License and royalty revenue                                                                                 $
              22,213                  $
           31,345      $
            90,242                $
            112,724



     Total revenues                                                                                                        22,213                           31,345                90,242                          112,724



     
                Operating Expenses



     Cost of revenues                                                                                                      11,238                           12,783                37,213                           54,545



     Research and development                                                                                              55,681                           62,505               232,266                          242,453



     General and administrative                                                                                            19,079                           21,210                88,494                           85,281



     Other operating expenses (income)                                                                                        118                          (7,382)                  397                          (6,679)



     Total operating expenses                                                                                              86,116                           89,116               358,370                          375,600



     Loss from operations                                                                                                (63,903)                        (57,771)            (268,128)                       (262,876)



     
                Other Income (Expense)



     Interest income from licensing                                                                                         (141)                              77                    25                              342



     Investment income                                                                                                      2,366                            2,026                11,319                            5,383



     Interest expense                                                                                                     (1,363)                         (4,310)              (6,862)                        (23,254)



     Total other income (expense)                                                                                             862                          (2,207)                4,482                         (17,529)



     Loss before income taxes                                                                                            (63,041)                        (59,978)            (263,646)                       (280,405)



     
                Income Tax Benefit                                                                                          152                               43                   152                               84



     Net loss                                                                                                  $
              (62,889)               $
           (59,935)  $
            (263,494)             $
            (280,321)



     
                Other Comprehensive Loss



     Unrealized gain (loss) on available-for-sale securities, net                                                           2,984                            2,855                10,972                         (12,832)



     Total other comprehensive income (loss)                                                                                2,984                            2,855                10,972                         (12,832)



     Comprehensive loss                                                                                        $
              (59,905)               $
           (57,080)  $
            (252,522)             $
            (293,153)





     Net loss per share, basic and diluted                                                                       $
              (1.43)                 $
           (1.38)     $
            (6.02)                $
            (6.50)



     Weighted-average common shares outstanding, basic and diluted                                                         44,001                           43,296                43,734                           43,152

CONTACTS:

Dana Cormack
Corporate Communications
Dcormack@regenxbio.com

Chris Brinzey, ICR Westwicke
339-970-2843
Chris.Brinzey@westwicke.com

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SOURCE REGENXBIO Inc.