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REGENXBIO Reports Third Quarter 2024 Financial Results and Recent Operational Updates

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REGENXBIO reported Q3 2024 financial results with revenues of $24.2 million, down from $28.9 million in Q3 2023. The company posted a net loss of $59.6 million ($1.17 per share). Cash position stood at $278.6 million, expected to fund operations into 2026.

Key operational updates include advancement in Phase I/II AFFINITY DUCHENNE trial of RGX-202, BLA submission initiation for RGX-121 (expected completion in Q1 2025), and positive Phase II data for ABBV-RGX-314. The company accelerated End-of-Phase II meeting for ABBV-RGX-314 in diabetic retinopathy to Q4 2024, with global pivotal program initiation planned for H1 2025.

REGENXBIO ha riportato i risultati finanziari del terzo trimestre 2024 con ricavi di 24,2 milioni di dollari, in calo rispetto ai 28,9 milioni di dollari del terzo trimestre 2023. L'azienda ha registrato una perdita netta di 59,6 milioni di dollari (1,17 dollari per azione). La posizione di liquidità si attesta a 278,6 milioni di dollari, prevista per finanziare le operazioni fino al 2026.

I principali aggiornamenti operativi includono l'avanzamento nella fase I/II della sperimentazione AFFINITY DUCHENNE di RGX-202, l'inizio della presentazione della BLA per RGX-121 (completamento previsto nel primo trimestre 2025) e dati positivi della fase II per ABBV-RGX-314. L'azienda ha accelerato l'incontro di fine fase II per ABBV-RGX-314 nella retinopatia diabetica al quarto trimestre 2024, con avvio del programma globale pivottale previsto per il primo semestre 2025.

REGENXBIO reportó los resultados financieros del tercer trimestre de 2024 con ingresos de 24,2 millones de dólares, en comparación con 28,9 millones de dólares en el tercer trimestre de 2023. La empresa tuvo una pérdida neta de 59,6 millones de dólares (1,17 dólares por acción). La posición de efectivo se situaba en 278,6 millones de dólares, se espera que financie las operaciones hasta 2026.

Las actualizaciones operativas clave incluyen el avance en el ensayo AFFINITY DUCHENNE de fase I/II de RGX-202, el inicio de la presentación de la BLA para RGX-121 (se espera que se complete en el primer trimestre de 2025) y datos positivos de fase II para ABBV-RGX-314. La empresa aceleró la reunión de fin de fase II para ABBV-RGX-314 en retinopatía diabética al cuarto trimestre de 2024, con el inicio del programa pivotal global previsto para el primer semestre de 2025.

REGENXBIO는 2024년 3분기 재무 실적을 보고하며 매출이 2420만 달러로, 2023년 3분기의 2890만 달러에서 감소했다고 발표했습니다. 회사는 5960만 달러의 순손실(주당 1.17달러)을 기록했습니다. 현금 보유액은 2억 7860만 달러로 2026년까지 운영 자금을 지원할 것으로 예상됩니다.

주요 운영 업데이트로는 RGX-202의 AFFINITY DUCHENNE 1/2상 시험의 진전, RGX-121에 대한 BLA 제출 시작(2025년 1분기 완료 예상), 그리고 ABBV-RGX-314에 대한 긍정적인 2상 데이터가 포함됩니다. 회사는 당뇨병성 망막병증에 대한 ABBV-RGX-314의 종말 단계 II 미팅을 2024년 4분기로 앞당기고, 글로벌 중추 프로그램 시작을 2025년 상반기로 계획하고 있습니다.

REGENXBIO a annoncé ses résultats financiers pour le troisième trimestre 2024, avec des revenus de 24,2 millions de dollars, en baisse par rapport à 28,9 millions de dollars au troisième trimestre 2023. La société a enregistré une perte nette de 59,6 millions de dollars (1,17 dollar par action). La position de liquidité s'établissait à 278,6 millions de dollars, prévue pour financer les opérations jusqu'en 2026.

Les principales mises à jour opérationnelles incluent les avancées de l'essai de phase I/II AFFINITY DUCHENNE de RGX-202, le lancement de la soumission de BLA pour RGX-121 (achèvement prévu au premier trimestre 2025), et des données positives de phase II pour ABBV-RGX-314. L'entreprise a accéléré la réunion de fin de phase II pour ABBV-RGX-314 dans la rétinopathie diabétique au quatrième trimestre 2024, avec un lancement du programme mondial pivot prévu pour le premier semestre 2025.

REGENXBIO berichtete über die finanziellen Ergebnisse für das dritte Quartal 2024 mit Einnahmen von 24,2 Millionen US-Dollar, ein Rückgang gegenüber 28,9 Millionen US-Dollar im dritten Quartal 2023. Das Unternehmen verzeichnete einen Nettverlust von 59,6 Millionen US-Dollar (1,17 US-Dollar pro Aktie). Die liquiden Mittel lagen bei 278,6 Millionen US-Dollar, die voraussichtlich bis 2026 für laufende Geschäfte verwendet werden sollen.

Zu den wichtigen operativen Updates gehören der Fortschritt der Phase I/II AFFINITY DUCHENNE Studie von RGX-202, der Beginn der BLA-Einreichung für RGX-121 (Fertigstellung wird im ersten Quartal 2025 erwartet) und positive Phase-II-Daten für ABBV-RGX-314. Das Unternehmen hat das Treffen zum Ende der Phase II für ABBV-RGX-314 bei diabetischer Retinopathie auf das vierte Quartal 2024 vorverlegt, mit einer geplanten Einleitung des globalen Pivotal-Programms im ersten Halbjahr 2025.

Positive
  • Cash runway extended into 2026 with $278.6 million in cash and equivalents
  • BLA submission for RGX-121 initiated, potential Priority Review Voucher in 2025
  • Positive Phase II data for ABBV-RGX-314 showing 97% reduction in anti-VEGF treatment burden
Negative
  • Revenue decreased to $24.2M from $28.9M YoY in Q3
  • Net loss of $59.6M in Q3 2024
  • Zolgensma royalty revenues declined to $23.9M from $28.4M YoY

Insights

The Q3 2024 results reveal significant financial and operational developments. $278.6M cash position provides runway into 2026, though down from $314.1M in December 2023. Revenue declined to $24.2M from $28.9M YoY, primarily due to lower Zolgensma royalties. Operating expenses showed improvement with R&D at $54.4M (down $3.8M) and G&A at $19.4M (down $3.7M).

The pipeline progress is particularly noteworthy with three potential near-term catalysts: RGX-202's pivotal trial initiation, RGX-121's BLA completion in Q1 2025 and ABBV-RGX-314's accelerated regulatory pathway. These developments could significantly impact the company's market position and future revenue streams.

The clinical progress across multiple programs demonstrates strong execution. RGX-202 for Duchenne is expanding into younger patients (ages 1-3), addressing an unmet need as the only U.S. gene therapy recruiting this age group. The 97% reduction in anti-VEGF treatment burden for ABBV-RGX-314 in wet AMD is particularly impressive, suggesting strong commercial potential.

The acceleration of the End-of-Phase II meeting for diabetic retinopathy indicates confidence in the data and could expedite market entry. The BLA submission for RGX-121 in MPS II positions the company to potentially secure the first gene therapy approval in this indication, with a possible Priority Review Voucher adding significant value.

  • Advancement in Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for Duchenne Muscular Dystrophy; pivotal trial initiation and first functional data expected this month
  • BLA submission for RGX-121 initiated and expected to complete in Q1 2025
  • Positive Phase II data support bilateral administration of subretinal ABBV-RGX-314; data consistent with that from multiple previous studies demonstrating favorable safety and efficacy profile
  • End-of-Phase II meeting for ABBV-RGX-314 in diabetic retinopathy accelerated to Q4 2024 to support global pivotal program initiation in H1 2025
  • Conference call today at 4:30 p.m. ET

ROCKVILLE, Md., Nov. 6, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and recent operational highlights for the third quarter ended September 30, 2024.

"It has been a turning point year for REGENXBIO, as we are on the cusp of advancing AFFINITY DUCHENNE® to pivotal phase, completing our first BLA for MPS II and entering pivotal phase in a second indication within our global eyecare collaboration with AbbVie," said Curran M. Simpson, President and Chief Executive Officer of REGENXBIO. "The rapid progress we are making in RGX-202, which continues to demonstrate its potential to be a best-in-class gene therapy for Duchenne, is highly encouraging, and the near-term filing of our BLA for RGX-121 represents a major milestone for the patient community in need of a treatment to address both the neurocognitive and systemic effects of MPS II. The recent fellow eye data presented at the American Academy of Ophthalmology meeting demonstrate the potential of ABBV-RGX-314 to preserve vision long-term for patients with wet AMD as a one-time treatment for both eyes. Each of these programs represent one-time treatments with the potential to transform the trajectory and management of disease for patients in need of new and better options, and we look forward to continued momentum and important milestones in the last quarter of the year."

PROGRAM HIGHLIGHTS AND MILESTONES

Neuromuscular Disease: RGX-202 is a potential best-in-class gene therapy designed to deliver a differentiated, novel microdystrophin gene for improved function and outcomes for patients living with Duchenne.

  • In the Phase I/II AFFINITY DUCHENNE trial of RGX-202, the last patient has been dosed in the dose level 2 (pivotal dose) expansion cohort for ages 4-11 and the first patient has been dosed in the cohort for ages 1-3. Patients with Duchenne under 4 years old have no access to gene therapy, and REGENXBIO is the only gene therapy sponsor recruiting patients in this age group in the U.S.
  • A clinical trials application (CTA) for RGX-202 has been authorized by Health Canada. REGENXBIO expects to initiate sites in Canada in H1 2025.
  • REGENXBIO plans to share a full program update this month, including pivotal trial design and plans for accelerated approval, as well as initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial.

Retinal Disease: ABBV-RGX‑314, being developed in collaboration with AbbVie, is potentially the first-in-class treatment for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).

ABBV-RGX-314 for Treatment of DR (Suprachoroidal Delivery)

  • Based on positive interim results to date from the Phase II ALTITUDE® trial, AbbVie and REGENXBIO have accelerated a planned End-of-Phase II meeting with the U.S. Food and Drug Administration (FDA) expected this quarter. The Company expects to initiate the first global pivotal trial in H1 2025.
  • The ALTITUDE trial is enrolling a new cohort of patients with center-involved diabetic macular edema (DME). DME is a vision-threatening complication of DR; an estimated 34 million people globally have DME. Patients will receive a one-time, in-office injection of ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye) with short course prophylactic steroid eye drops.

ABBV-RGX-314 for the Treatment of Wet AMD (Subretinal Delivery)

  • Enrollment is on track in the ATMOSPHERE® and ASCENT™ pivotal trials and these trials are expected to support global regulatory submissions with the FDA and European Medicines Agency in H1 2026.
  • Positive data from the Phase II fellow eye sub-study evaluating the subretinal delivery of ABBV-RGX-314 in patients with bilateral wet AMD were recently presented at the American Academy of Ophthalmology annual meeting. As of September 11, 2024, ABBV-RGX-314 was well tolerated in the treated fellow eye, with no drug-related serious adverse events and no cases of intraocular inflammation observed. At nine months post-administration, patients saw a 97% reduction in anti-VEGF treatment burden and sustained vision and anatomy. These results support the potential of ABBV-RGX-314 to treat bilateral disease at an expected commercial launch.

ABBV-RGX-314 for the Treatment of Wet AMD (Suprachoroidal Delivery)

  • Based on a favorable safety profile and to evaluate dose levels for a planned pivotal program, the Phase II AAVIATE® trial is enrolling a new cohort to evaluate ABBV-RGX-314 at dose level 4 (1.5x10e12 GC/eye). Patients in this cohort will also receive short course prophylactic steroid eye drops. At dose level 3, patients receiving ABBV-RGX-314 demonstrated an 80% reduction in annualized injection rate, with 50% of patients remaining injection-free at six months. Patients also demonstrated stable Best Corrected Visual Acuity and central retinal thickness.

Neurodegenerative Disease: RGX-121 is a potential first-in-class treatment for MPS II.

  • REGENXBIO initiated a rolling BLA submission for RGX-121 using the accelerated approval pathway in Q3 2024. The BLA submission is expected to be complete in Q1 2025.
  • RGX-121 is on track to be the potential first gene therapy and one-time treatment approved for MPS II. Approval of RGX-121 could result in receipt of a Priority Review Voucher in 2025.

FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable securities were $278.6 million as of September 30, 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 nine months ended September 30, 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 $24.2 million for the three months ended September 30, 2024, compared to $28.9 million for the three months ended September 30, 2023. The decrease was primarily attributable to Zolgensma royalty revenues, which decreased from $28.4 million for the third quarter of 2023 to $23.9 million for the third quarter of 2024.

Research and Development Expenses: Research and development expenses were $54.4 million for the three months ended September 30, 2024, compared to $58.2 million for the three months ended September 30, 2023. The decrease was largely driven by lower personnel-related costs and early-stage research and development activities, and was partially offset by increases in clinical trial expenses for ABBV-RGX-314 and RGX-202.

General and Administrative Expenses: General and administrative expenses were $19.4 million for the three months ended September 30, 2024, compared to $23.1 million for the three months ended September 30, 2023. The decrease was primarily attributable to expenses for professional services and other corporate overhead costs.

Net Loss: Net loss was $59.6 million, or $1.17 basic and diluted net loss per share, for the three months ended September 30, 2024, compared to a net loss of $61.9 million, or $1.41 basic and diluted net loss per share, for the three months ended September 30, 2023.

FINANCIAL GUIDANCE
REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $278.6 million as of September 30, 2024 to fund its operations into 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. 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 rare and retinal diseases, including RGX-202 for the treatment of Duchenne, ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, 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 timing or likelihood of payments from AbbVie, 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, 2023, 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 AG. All other trademarks referenced herein are registered trademarks of REGENXBIO.

 

REGENXBIO INC.

CONSOLIDATED BALANCE SHEETS

(unaudited)

(in thousands)






September 30, 2024



December 31, 2023


Assets







Current assets







Cash and cash equivalents


$

56,617



$

34,522


Marketable securities



198,843




240,736


Accounts receivable, net



23,604




24,790


Prepaid expenses



11,002




14,520


Other current assets



23,330




20,403


Total current assets



313,396




334,971


Marketable securities



23,108




38,871


Accounts receivable



404




701


Property and equipment, net



120,551




132,103


Operating lease right-of-use assets



55,293




60,487


Restricted cash



2,030




2,030


Other assets



4,332




4,807


Total assets


$

519,114



$

573,970


Liabilities and Stockholders' Equity







Current liabilities







Accounts payable


$

19,522



$

22,786


Accrued expenses and other current liabilities



48,642




49,703


Deferred revenue



144




148


Operating lease liabilities



7,720




7,068


Liability related to sale of future royalties



26,697




50,567


Total current liabilities



102,725




130,272


Operating lease liabilities



76,342




82,222


Liability related to sale of future royalties



35,052




43,485


Other liabilities



3,579




6,249


Total liabilities



217,698




262,228


Stockholders' equity







Preferred stock; no shares issued and outstanding
   at September 30, 2024 and December 31, 2023







Common stock; 49,534 and 44,046 shares issued
   and outstanding at September 30, 2024 and
   December 31, 2023, respectively



5




4


Additional paid-in capital



1,182,956




1,021,214


Accumulated other comprehensive loss



(582)




(4,429)


Accumulated deficit



(880,963)




(705,047)


Total stockholders' equity



301,416




311,742


Total liabilities and stockholders' equity


$

519,114



$

573,970


 

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,



2024



2023



2024



2023


Revenues












License and royalty revenue

$

24,197



$

28,914



$

62,114



$

68,029


Total revenues


24,197




28,914




62,114




68,029


Operating Expenses












Cost of revenues


12,387




12,388




27,249




25,975


Research and development


54,429




58,183




158,142




176,585


General and administrative


19,422




23,083




56,568




69,415


Impairment of long-lived assets








2,101





Other operating expenses


37




220




32




279


Total operating expenses


86,275




93,874




244,092




272,254


Loss from operations


(62,078)




(64,960)




(181,978)




(204,225)


Other Income (Expense)












Interest income from licensing


25




56




91




166


Investment income


3,276




4,660




9,213




8,953


Interest expense


(820)




(1,624)




(3,242)




(5,499)


Total other income


2,481




3,092




6,062




3,620


Net loss

$

(59,597)



$

(61,868)



$

(175,916)



$

(200,605)


Other Comprehensive Income












Unrealized gain on available-for-sale securities, net


1,684




2,685




3,847




7,988


Total other comprehensive income


1,684




2,685




3,847




7,988


Comprehensive loss

$

(57,913)



$

(59,183)



$

(172,069)



$

(192,617)














Net loss per share, basic and diluted

$

(1.17)



$

(1.41)



$

(3.59)



$

(4.60)


Weighted-average common shares outstanding, basic and diluted


50,800




43,945




49,051




43,644


CONTACTS:

Dana Cormack
Corporate Communications
Dcormack@regenxbio.com

George E. MacDougall
Investor Relations
IR@regenxbio.com 

(PRNewsfoto/REGENXBIO Inc.)

 

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SOURCE REGENXBIO Inc.

FAQ

What were REGENXBIO's (RGNX) Q3 2024 financial results?

REGENXBIO reported Q3 2024 revenues of $24.2 million and a net loss of $59.6 million ($1.17 per share), with cash position at $278.6 million as of September 30, 2024.

When will REGENXBIO (RGNX) complete its BLA submission for RGX-121?

REGENXBIO expects to complete the BLA submission for RGX-121 in Q1 2025.

What is the cash runway projection for REGENXBIO (RGNX)?

REGENXBIO expects its current cash position of $278.6 million to fund operations into 2026.

What were the Phase II results for ABBV-RGX-314 in wet AMD?

Phase II results showed a 97% reduction in anti-VEGF treatment burden at nine months post-administration, with sustained vision and anatomy, and no drug-related serious adverse events.

REGENXBIO Inc.

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Biotechnology
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