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Rocket Pharmaceuticals Reports Third Quarter 2024 Financial Results and Highlights Recent Progress

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Rocket Pharmaceuticals (NASDAQ: RCKT) reported Q3 2024 financial results and pipeline updates. Key developments include completed enrollment in Phase 2 pivotal study of RP-A501 for Danon disease and low-dose cohort completion for RP-A601 PKP2-ACM study. The company initiated rolling BLA submission for RP-L102 and expects KRESLADI approval in 2025. Financial highlights show cash position of $235.7M expected to fund operations into 2026. R&D expenses decreased to $133.9M from $144.6M year-over-year, while G&A expenses increased to $76.6M from $51.8M. Net loss was $198.4M or $2.11 per share.

Rocket Pharmaceuticals (NASDAQ: RCKT) ha riportato i risultati finanziari del terzo trimestre del 2024 e aggiornamenti sul pipeline. Gli sviluppi chiave includono il completamento dell'arruolamento nello studio pivotale di Fase 2 per RP-A501 per la malattia di Danon e il completamento del gruppo a basso dosaggio per lo studio RP-A601 PKP2-ACM. L'azienda ha avviato la presentazione progressiva della BLA per RP-L102 e si aspetta l'approvazione di KRESLADI nel 2025. I punti salienti finanziari mostrano una posizione di cassa di 235,7 milioni di dollari, prevista per finanziare le operazioni fino al 2026. Le spese per R&S sono diminuite a 133,9 milioni di dollari rispetto ai 144,6 milioni dell'anno precedente, mentre le spese generali e amministrative sono aumentate a 76,6 milioni di dollari rispetto ai 51,8 milioni. La perdita netta è stata di 198,4 milioni di dollari, ovvero 2,11 dollari per azione.

Rocket Pharmaceuticals (NASDAQ: RCKT) reportó los resultados financieros del tercer trimestre de 2024 y actualizaciones sobre su pipeline. Los desarrollos clave incluyen la finalización de la inscripción en el estudio pivotal de Fase 2 de RP-A501 para la enfermedad de Danon y la finalización del grupo de dosis bajas para el estudio RP-A601 PKP2-ACM. La compañía inició la presentación progresiva de la BLA para RP-L102 y espera la aprobación de KRESLADI en 2025. Los aspectos destacados financieros muestran una posición de efectivo de 235.7 millones de dólares, que se espera financie las operaciones hasta 2026. Los gastos de I+D disminuyeron a 133.9 millones de dólares desde 144.6 millones en comparación con el año anterior, mientras que los gastos generales y administrativos aumentaron a 76.6 millones de dólares desde 51.8 millones. La pérdida neta fue de 198.4 millones de dólares o 2.11 dólares por acción.

로켓 제약 (NASDAQ: RCKT)가 2024년 3분기 재무 결과 및 파이프라인 업데이트를 발표했습니다. 주요 개발 사항으로는 다논병을 위한 RP-A501의 2상 주요 연구에서 환자 등록 완료 및 RP-A601 PKP2-ACM 연구의 저용량 그룹 완료가 포함됩니다. 이 회사는 RP-L102의 점진적인 BLA 제출을 시작했으며 2025년 KRESLADI 승인을 기대하고 있습니다. 재무 하이라이트는 2,3570만 달러의 현금 보유액을 보여주며, 2026년까지 운영 자금으로 사용될 것으로 예상됩니다. 연구 개발 비용은 전년 대비 1억 3,390만 달러로 줄어들었고, 일반 관리 비용은 5,180만 달러에서 7,660만 달러로 증가했습니다. 순손실은 1억 9,840만 달러로, 주당 2.11달러입니다.

Rocket Pharmaceuticals (NASDAQ: RCKT) a publié ses résultats financiers pour le troisième trimestre 2024 ainsi que des mises à jour sur sa pipeline. Parmi les développements clés, on trouve l'achèvement de l'inscription à l'étude pivotale de Phase 2 de RP-A501 pour la maladie de Danon et la finalisation du groupe à faible dose de l'étude RP-A601 PKP2-ACM. La société a lancé la soumission progressive du BLA pour RP-L102 et s'attend à l'approbation de KRESLADI en 2025. Les faits saillants financiers montrent une position de trésorerie de 235,7 millions de dollars, censée financer les opérations jusqu'en 2026. Les dépenses en R&D ont diminué à 133,9 millions de dollars contre 144,6 millions de dollars l'année précédente, tandis que les dépenses générales et administratives ont augmenté à 76,6 millions de dollars contre 51,8 millions de dollars. La perte nette était de 198,4 millions de dollars, soit 2,11 dollars par action.

Rocket Pharmaceuticals (NASDAQ: RCKT) hat die Finanzergebnisse für das dritte Quartal 2024 und Aktualisierungen der Pipeline veröffentlicht. Zu den wichtigen Entwicklungen gehört der Abschluss der Rekrutierung in der Phase-2-Studie von RP-A501 zur Danon-Krankheit sowie der Abschluss der Niedrigdosisgruppe für die RP-A601 PKP2-ACM-Studie. Das Unternehmen hat die schrittweise BLA-Einreichung für RP-L102 eingeleitet und erwartet die Genehmigung von KRESLADI im Jahr 2025. Finanzielle Highlights zeigen eine Barreservenposition von 235,7 Millionen Dollar, die voraussichtlich die Betriebe bis 2026 finanzieren wird. Die F&E-Ausgaben sanken auf 133,9 Millionen Dollar von 144,6 Millionen im Vorjahr, während die allgemeinen und administrativen Ausgaben auf 76,6 Millionen Dollar von 51,8 Millionen anstiegen. Der Nettoverlust betrug 198,4 Millionen Dollar oder 2,11 Dollar pro Aktie.

Positive
  • Completed enrollment in Phase 2 pivotal study for Danon disease treatment
  • Strong cash position of $235.7M with runway into 2026
  • R&D expenses decreased by $10.7M year-over-year
  • Initiated rolling BLA submission for RP-L102
Negative
  • Net loss increased to $198.4M from $185.9M year-over-year
  • G&A expenses increased by $24.8M (48%) year-over-year
  • KRESLADI approval delayed to 2025 pending additional FDA review

Insights

The Q3 results reveal a mixed financial picture. With $235.7M in cash and equivalents, operational runway into 2026 provides solid financial stability. However, increased G&A expenses of $76.6M (up 48% YoY) due to commercial preparation costs signals significant pre-launch investments. The $198.4M net loss ($2.11 per share) shows continued high cash burn, though R&D expenses decreased by $10.7M YoY, indicating improved operational efficiency.

Key pipeline developments, particularly in Danon disease and PKP2-ACM programs, along with the rolling BLA submission for Fanconi Anemia treatment, demonstrate advancing commercialization potential. The anticipated 2025 approval for KRESLADI could mark first revenue generation, though substantial marketing investments will be needed.

The completion of enrollment in multiple clinical trials marks significant operational progress. The Phase 2 pivotal study for RP-A501 in Danon disease and Phase 1 low-dose cohort for RP-A601 in PKP2-ACM represent critical advancement in rare cardiovascular diseases. The PKP2-ACM program particularly stands out with an estimated 50,000 patients in the U.S. and Europe, representing substantial market potential.

The diverse pipeline spanning cardiovascular and hematological disorders demonstrates strong therapeutic diversification. The progression of BAG3-associated dilated cardiomyopathy program toward IND submission further strengthens the cardiovascular portfolio. The regulatory advancement of KRESLADI for LAD-I and RP-L102 for Fanconi Anemia indicates maturing pipeline with near-term approval potential.

Enrollment completed in September and dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease

Updated Phase 1 data from RP-A501 for Danon disease anticipated at American Heart Association’s 2024 Late-Breaking Scientific Sessions

Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Preliminary data expected in the first half of 2025

Rolling BLA submission initiated for RP-L102 for Fanconi Anemia

Progress on FDA-approval of KRESLADI for severe LAD-I; Approval anticipated in 2025

Appointed Mikael Dolsten, M.D., Ph.D., to Board of Directors

Cash, cash equivalents and investments of approximately $235.7M; expected operational runway into 2026

CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the third quarter ending September 30, 2024.

“Rocket made meaningful progress during the third quarter, notably with the completion of enrollment in the RP-A501 program for Danon disease, low dose cohort enrollment completion in the RP-A601 program for PKP2-ACM, and appointment of seasoned pharmaceutical executive, Mikael Dolsten to our Board of Directors,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. “As we continue to pursue our mission of seeking gene therapy cures for patients with rare and devastating diseases, we remain focused on expediently advancing our deep pipeline of cardiovascular and hematology programs.”

Recent Pipeline and Operational Updates

  • Continued advancement of Phase 2 pivotal study of RP-A501 for Danon Disease.
    • In September, Rocket announced completion of enrollment in the Phase 2 pivotal study of RP-A501 to treat Danon Disease.
    • Dosing in the Phase 2 pivotal study is ongoing.
    • Updated data from the Phase 1 study to be presented at the American Heart Association’s 2024 Late-Breaking Science sessions on November 18.
    • Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034.
  • Progressed the Phase 1 clinical study of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM).
    • Completed patient enrollment in the low dose cohort.
    • Preliminary data from the Phase 1 study is expected in the first half of 2025.
    • Ongoing internal estimates confirm that PKP2-ACM affects approximately 50,000 people in the U.S. and Europe, representing the largest market opportunity in Rocket’s pipeline of disclosed programs.
    • Details of the Phase 1 study can be found at www.ClinicalTrials.gov under the NCT identifier NCT05885412.
  • Progressed BAG3-associated dilated cardiomyopathy preclinical program.
    • Nonclinical, IND-enabling studies are ongoing.
    • Submission of the IND is anticipated in the first half of 2025.
  • Progressed RP-L102 investigational gene therapy for Fanconi Anemia (FA).
    • Initiated rolling Biologics License Application (BLA) for RP-L102.
    • Secured an ICD-10 code from the Centers for Medicare and Medicaid Services to document patients with FA.
    • Regulatory review for RP-L102 for the treatment of FA by the European Medicines Authority is underway.
  • U.S. Food and Drug Administration (FDA) review of limited additional Chemistry Manufacturing and Controls (CMC) information ongoing for KRESLADITM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).
    • Rocket previously disclosed that the FDA requested limited additional CMC information to complete its review of KRESLADI to treat severe LAD-I.
    • The Company continues to work with senior leaders and reviewers from the FDA’s Center for Biologics Evaluation and Research.
    • Approval of KRESLADI anticipated in 2025.
  • Initiated global Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency.
  • Appointed Mikael Dolsten, M.D., Ph.D., to Board of Directors.
    • Dr. Dolsten is an accomplished industry executive with extensive global pharmaceutical management experience. He currently serves as the Chief Scientific Officer, President, Research & Development at Pfizer Inc., focused on advancing gene therapies, small-molecule medicines, biotherapeutics, and vaccines.

Third Quarter Financial Results

  • Cash position. Cash, cash equivalents and investments as of September 30, 2024, were $235.7 million.
  • R&D expenses. Research and development expenses were $133.9 million for the nine months ended September 30, 2024, compared to $144.6 million for the nine months ended September 30, 2023. The decrease of $10.7 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $17.4 million and costs for research agreements of $1.2 million. The decreases were partially offset by increases in the costs for professional fees and consultants of $4.9 million, non-cash stock compensation expenses of $1.2 million, and compensation and benefit expenses of $1.0 million.
  • G&A expenses. General and administrative expenses were $76.6 million for the nine months ended September 30, 2024, compared to $51.8 million for the nine months ended September 30, 2023. The increase in G&A expenses was primarily driven by increased commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $15.7 million, legal expenses of $4.0 million, non-cash stock compensation expense of $2.2 million, and compensation and benefit expenses of $1.9 million.
  • Net loss. Net loss was $198.4 million or $2.11 per share (basic and diluted) for the nine months ended September 30, 2024, compared to $185.9 million or $2.30 (basic and diluted) for the nine months ended September 30, 2023.
  • Shares outstanding. 91,116,692 shares of common stock were outstanding as of September 30, 2024.

Financial Guidance

  • Cash position. As of September 30, 2024, Rocket had cash, cash equivalents and investments of $235.7 million. Rocket expects such resources will be sufficient to fund its operations into 2026, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of its six clinical and/or preclinical programs.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives.

Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia.

Rocket’s adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles.

For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn, YouTube, and X.

Rocket Cautionary Statement Regarding Forward-Looking Statements

This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as “could,” “believe,” “expect,” “anticipate,” “intend,” “plan,” “will give,” “estimate,” “seek,” “will,” “may,” “suggest” or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, including the timing and outcome of the FDA’s review of the additional CMC information that Rocket will provide in response to the FDA’s request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket’s ability to establish key collaborations and vendor relationships for its product candidates, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket’s ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket’s ability to transition to a commercial stage pharmaceutical company, and Rocket’s expectation that its cash, cash equivalents and investments will be sufficient to funds its operations into 2026. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket’s competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket’s ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket’s ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket’s ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled “Risk Factors” in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2023, filed February 27, 2024 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Three Months Ended September 30, Nine Months Ended September 30,

 

2024

 

 

2023

 

 

2024

 

 

2023

 

Operating expenses:
Research and development

$

42,315

 

$

46,844

 

$

133,887

 

$

144,598

 

General and administrative

 

27,109

 

 

18,585

 

 

76,624

 

 

51,782

 

Total operating expenses

 

69,424

 

 

65,429

 

 

210,511

 

 

196,380

 

Loss from operations

 

(69,424

)

 

(65,429

)

 

(210,511

)

 

(196,380

)

Interest expense

 

(471

)

 

(469

)

 

(1,413

)

 

(1,405

)

Interest and other income, net

 

1,327

 

 

1,720

 

 

6,650

 

 

4,474

 

Accretion of discount on investments, net

 

1,849

 

 

2,279

 

 

6,855

 

 

7,376

 

Net loss

$

(66,719

)

$

(61,899

)

$

(198,419

)

$

(185,935

)

Net loss per share - basic and diluted

$

(0.71

)

$

(0.75

)

$

(2.11

)

$

(2.30

)

Weighted-average common shares outstanding - basic and diluted

 

94,158,491

 

 

82,636,120

 

 

93,893,729

 

 

80,865,658

 

 
 
September 30, 2024 December 31, 2023
Cash, cash equivalents, and investments

$

235,662

 

$

407,495

 

Total assets

 

393,688

 

 

566,341

 

Total liabilities

 

63,917

 

 

73,767

 

Total stockholders' equity

 

329,771

 

 

492,574

 

 

 

Media & Investors

Meg Dodge

mdodge@rocketpharma.com



Media

Kevin Giordano

media@rocketpharma.com



Investors

Brooks Rahmer

investors@rocketpharma.com

Source: Rocket Pharmaceuticals, Inc.

FAQ

What was Rocket Pharmaceuticals (RCKT) Q3 2024 net loss per share?

Rocket Pharmaceuticals reported a net loss of $2.11 per share for the nine months ended September 30, 2024.

How much cash does RCKT have as of Q3 2024?

Rocket Pharmaceuticals had cash, cash equivalents and investments of $235.7 million as of September 30, 2024.

When does RCKT expect FDA approval for KRESLADI?

Rocket Pharmaceuticals anticipates FDA approval for KRESLADI in 2025, pending review of additional CMC information.

What is the status of RCKT's RP-A501 Danon disease program?

Enrollment was completed in September 2024 for the Phase 2 pivotal study of RP-A501, with dosing ongoing and updated Phase 1 data expected at AHA's 2024 Late-Breaking Scientific Sessions.

Rocket Pharmaceuticals, Inc.

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