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Rocket Pharmaceuticals Reports Second Quarter 2023 Financial Results and Highlights Recent Progress

Initiating enrollment of two-patient pediatric safety run-in for pivotal single-arm Phase 2 study of RP-A501 (Danon Disease); finalizing primary composite endpoint with FDA to support accelerated approval

RP-L201 (LAD-I) BLA submitted to FDA; represents Rocket’s first product filing

RP-A601 (PKP2-ACM) advancing towards first patient treatment in Phase 1 study following FDA clearance of IND; received FDA Fast Track and Orphan Drug designations

RP-L102 (Fanconi Anemia) U.S. pivotal Phase 2 study completed; product filing on track

RP-L301 (PKD) granted EMA PRIME and FDA RMAT designations

Cash, cash equivalents and investments of approximately $307M; expected operational runway into the first half of 2025

CRANBURY, N.J.–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial results for the quarter ending June 30, 2023, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.


“The second quarter of 2023 marked a period of strong forward momentum across our cardiovascular AAV and hematology LV portfolios highlighted by the BLA submission for our LAD-I program, Rocket’s first product filing. In addition, we are in advanced discussions with the FDA to finalize the components of the primary composite endpoint for the single-arm, pivotal Phase 2 Danon Disease study. In parallel, we are initiating enrollment in the trial following recent alignment with the FDA on our proposed two-patient pediatric safety run-in,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “We appreciate the strong and positive collaboration with the FDA afforded by our RMAT designation, which we believe will lead to the most optimal global development path forward for the first pivotal gene therapy trial for a cardiac condition. Simultaneously, in our second cardiac program, RP-A601 for the treatment of PKP2-ACM, we are rapidly moving towards first patient treatment following IND clearance and have also received FDA Fast Track and Orphan Drug designations.”

Dr. Shah continued, “Building on the BLA submission for LAD-I, we remain on track to submit the BLA for our LV-based Fanconi Anemia program in the fourth quarter. Taken together, I am very pleased with our progress across our entire pipeline of AAV and LV assets and look forward to continuing our momentum through the remainder of the year and beyond.”

Key Pipeline and Operational Updates

AAV Cardiovascular Portfolio

Danon Disease

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

LV Hematology Portfolio

Leukocyte Adhesion Deficiency-I (LAD-I)

Fanconi Anemia (FA)

Pyruvate Kinase Deficiency (PKD)

Operational Update

Second Quarter Financial Results

Financial Guidance

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket’s clinical programs using lentiviral vector (LV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure 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 rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating pediatric heart failure condition. Rocket also has received IND clearance for the AAV-based gene therapy program for PKP2-arrhythmogenic cardiomyopathy (ACM), a heart failure condition associated with life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death, and is advancing a preclinical program for BAG3-associated dilated cardiomyopathy (DCM). For more information about Rocket, please visit www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket’s future expectations, plans and prospects, including without limitation, 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), PKP2-ACM 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, Rocket’s plans for the advancement of its Danon Disease program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as “aim,” “anticipate,” “believe,” “can,” “continue,” “design,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “will give,” “seek,” “will,” “may,” “suggest” or similar terms, variations of such terms or the negative of those terms. 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 ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, our ability to submit regulatory filings with the U.S. Food and Drug Administration (FDA) and to obtain and maintain FDA or other regulatory authority approval of our product candidates, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, our competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, our integration of an acquired business, which involves a number of risks, including the possibility that the integration process could result in the loss of key employees, the disruption of our ongoing business, or inconsistencies in standards, controls, procedures, or policies, our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire and any unexpected expenditures, 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, 2022, filed February 28, 2023 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 June 30, Six Months Ended June 30,

 

2023

 

 

 

2022

 

 

 

2023

 

 

 

2022

 

Operating expenses:
Research and development

$

51,383

 

$

41,356

 

$

97,754

 

$

72,150

 

General and administrative

 

17,374

 

 

12,854

 

 

33,197

 

 

24,624

 

Total operating expenses

 

68,757

 

 

54,210

 

 

130,951

 

 

96,774

 

Loss from operations

 

(68,757

)

 

(54,210

)

 

(130,951

)

 

(96,774

)

Interest expense

 

(468

)

 

(465

)

 

(936

)

 

(928

)

Interest and other income, net

 

846

 

 

669

 

 

2,754

 

 

1,291

 

Accretion of discount and amortization of premium on investments, net

 

2,678

 

 

(396

)

 

5,097

 

 

(973

)

Net loss

$

(65,701

)

$

(54,402

)

$

(124,036

)

$

(97,384

)

Net loss per share – basic and diluted

$

(0.82

)

$

(0.83

)

$

(1.55

)

$

(1.50

)

Weighted-average common shares outstanding – basic and diluted

 

80,472,362

 

 

65,476,531

 

 

79,965,755

 

 

64,995,797

 

 
June 30, December 31,

 

2023

 

 

 

2022

 

Cash, cash equivalents, and investments

$

307,040

 

$

399,670

 

Total assets

 

461,781

 

 

551,807

 

Total liabilities

 

58,811

 

 

62,121

 

Total stockholders’ equity

 

402,970

 

 

489,686

 

 

Contacts

Media
Kevin Giordano

media@rocketpharma.com

Investors
Brooks Rahmer

investors@rocketpharma.com

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