On May 6, 2024 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, reported financial and recent operational results for the quarter ending March 31, 2024 (Press release, Rocket Pharmaceuticals, MAY 6, 2024, View Source [SID1234642685]).

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"Rocket has had a strong start to 2024. This quarter has been marked by meaningful progress across all our clinical programs, most notably with the EMA’s review of RP-L102 for Fanconi Anemia, commercial preparations for the potential approval of KRESLADI for severe LAD-I, and ongoing enrollment in the Phase 2, pivotal study of RP-A501 for Danon Disease and the Phase 1 study of RP-A601 for PKP2-ACM," said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. "As we build on the advancements made this quarter, we remain focused on execution for patients with rare and devastating diseases with limited treatment options."

Recent Pipeline and Operational Updates

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Advanced Fanconi Anemia (FA) program through key regulatory milestones.

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In April, Rocket announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102 for the treatment of FA. MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial.

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The Company remains on track to submit the Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the first half of 2024.

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KRESLADI on track for June 30, 2024, PDUFA date.

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Rocket continues to ramp up enabling activities to support the launch of its lentiviral (LV) vector portfolio beginning with KRESLADI for severe Leukocyte Adhesion Deficiency-I (LAD-I). Qualified Treatment Center initiation, disease education, payer engagement, and field team build-out are all underway.

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Bolstered finance, investor relations, and corporate communications expertise of company leadership to support evolution towards commercial stage.

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Rocket appointed Aaron Ondrey as Chief Financial Officer (CFO). Mr. Ondrey brings over 20 years of experience leading commercial-stage financial management, strategic planning, and capital allocation. Mr. Ondrey was previously the CFO at Mirati Therapeutics and has held multiple senior finance leadership positions at Arena Pharmaceuticals, Alexion Pharmaceuticals, and Regeneron Pharmaceuticals.

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In addition, Meg Dodge was appointed Vice President to lead Investor Relations & Corporate Communications. Ms. Dodge is experienced with engaging stakeholders across investors, media, and other communities in the biotech and financial sectors. Prior to joining Rocket, Ms. Dodge was Head of Investor Relations and Corporate Communications at Krystal Biotech.

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Celebrated annual Rare Disease Day with multi-faceted awareness campaign.

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On February 29, 2024, Rocket hosted its annual Rare Disease Day recognition program highlighting the theme, "Leap into Action for Rare." Several hundred attendees gathered at the Liberty Science Center and virtually to hear inspirational stories from the community. Rocket also continued its Light Up for Rare initiative in collaboration with global partners to light up buildings and landmarks in Rare Disease Day colors, including the Empire State Building.

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Rocket remains highly committed to supporting the rare disease community through patient-focused events, education and advancing science to bring potential treatments to patients with unmet needs.

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Milestones in 2024 are on track across its pipeline of gene therapies for rare and devastating diseases.

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Rocket continues to advance three disclosed programs from its adeno-associated virus (AAV) cardiovascular portfolio, including:

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Phase 2 pivotal study of RP-A501 for Danon Disease,

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Phase 1 study of RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM), and

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IND-enabling studies for BAG3-associated dilated cardiomyopathy (DCM)

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In its late-stage LV portfolio, Rocket is working towards initiation of the Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD).

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Data from Rocket’s LV hematology portfolio to be presented at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting.

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Updated data across Rocket’s LV hematology programs will be highlighted as oral presentations at the ASGCT (Free ASGCT Whitepaper) 27th Annual Meeting taking place May 7-11, 2024, in Baltimore, MD

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Incremental updates include longer-term data demonstrating the safety and efficacy of Rocket’s Phase 1/2 pivotal studies of KRESLADI for severe LAD-I and RP-L102 for FA, in addition to the Phase 1 study of RP-L301 for PKD.

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Details for oral presentations are as follows:

Title: Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301
Session: Clinical Trials Spotlight Symposium
Presenter: Julián Sevilla, M.D., Ph.D., Clinical Investigator, Hematología y Hemoterapia, Hematología y Oncología Pediátricas, Hospital Infantil Universitario Niño Jesús
Presentation date and time: Wednesday, May 8, 2024, 8:00 a.m. – 8:15 a.m. ET
Location: Ballroom 1
Presentation number: 4

Title: Lentiviral-Mediated Gene Therapy (RP-L102) for Fanconi Anemia [Group A] is Associated with Polyclonal Integration Patterns in the Absence of Conditioning
Session: Cell Therapy and Cell-Based Gene Therapy Trials
Presenter: Agnieszka Czechowicz, M.D., Ph.D., Center for Definitive and Curative Medicine, Department of Pediatrics, Division of Hematology/ Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Lucile Packard Children’s Hospital Stanford
Presentation date and time: Friday, May 10, 2024, 2:45 p.m. – 3:00 p.m. ET
Location: Ballroom 1
Presentation number: 245

Title: Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I Provides Sustained Efficacy with a Favorable Safety Profile
Session: Cell Therapy and Cell-Based Gene Therapy Trials
Presenter: Donald B. Kohn, M.D., Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy Program, University of California, Los Angeles
Presentation date and time: Friday, May 10, 2024, 3:00 p.m. – 3:15 p.m. ET
Location: Ballroom 1
Presentation number: 246

Upcoming Investor Conference

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Bank of America Global Healthcare Conference 2024: May 16, 2024

First Quarter Financial Results

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Cash position. Cash, cash equivalents and investments as of March 31, 2024, were $330.3 million.

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R&D expenses. Research and development expenses were $45.2 million for the three months ended March 31, 2024, compared to $46.4 million for the three months ended March 31, 2023. The decrease in R&D expenses was primarily driven by decreases in manufacturing and development costs and direct materials of $5.8 million. Decreases were partially offset by increases in costs for compensation and benefits expense of $1.4 million due to increased R&D headcount, professional fees of $1.1 million, laboratory supplies of $0.9 million, non-cash stock compensation expense of $0.8 million, and clinical trial costs of $0.6 million.

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G&A expenses. General and administrative expenses were $22.1 million for the three months ended March 31, 2024, compared to $15.8 million for the three months ended March 31, 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 $3.3 million, legal expenses of $1.5 million, and non-cash stock compensation expense of $0.5 million.

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Net loss. Net loss was $62.1 million or $0.66 per share (basic and diluted) for the three months ended March 31, 2024, compared to $58.3 million or $0.73 (basic and diluted) for the three months ended March 31, 2023.

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Shares outstanding. 90,646,590 shares of common stock were outstanding as of March 31, 2024.

Financial Guidance

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Cash position. As of March 31, 2024, Rocket had cash, cash equivalents and investments of $330.3 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.