On February 26, 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 operational results for the fourth quarter and year ended December 31, 2023 (Press release, Rocket Pharmaceuticals, FEB 26, 2024, View Source [SID1234640460]).
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"I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spanning our AAV cardiovascular and LV hematology portfolios," said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. "We are well poised to make further strides in 2024, notably expanding our commercial and operational capabilities to support the anticipated launch of KRESLADI (marnetegragene autotemcel) for severe LAD-I, representing Rocket’s first step in making our gene therapies available to patients who need them most. At the same time, we are laser focused on regulatory filings in Fanconi Anemia and continuing to advance our clinical programs in Danon Disease, PKP2-ACM and PKD in the year ahead. As we close the quarter, Rocket continues to solidify our industry leadership in gene therapy across multiple therapeutic areas and platforms as we seek to meet the needs of patients living with rare and devastating diseases."
Recent Pipeline and Operational Updates
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Milestones remain on track for 2024 across pipeline of adeno-associated virus (AAV) cardiovascular and lentiviral (LV) vector hematology portfolios. The Marketing Authorisation Application (MAA) and Biologics License Application (BLA) for Fanconi Anemia remain on track for filing with the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA), respectively, in the first half of 2024. Rocket is also progressing its Phase 2 pivotal study of RP-A501 for Danon Disease, Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD), Phase 1 study of RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM), and IND-enabling studies for BAG3-associated dilated cardiomyopathy (DCM).
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KRESLADITM for severe Leukocyte Adhesion Deficiency-I (LAD-I) on track for PDUFA date of June 30, 2024. Based on the positive top-line efficacy and safety data from the global Phase 1/2 study and high unmet need in patients with severe LAD-I, the FDA accepted the BLA and granted Priority Review for KRESLADITM. On February 13, 2024, Rocket announced the FDA extended the Priority Review period by three months, to June 30, 2024, to allow additional time to review clarifying Chemistry, Manufacturing, and Controls (CMC) information submitted by Rocket in response to FDA information requests. The FDA further confirmed that an advisory committee meeting is not needed. In support of its first product launch, Rocket continues to expand its commercial and operational infrastructure, including Qualified Treatment Center initiation, channel strategy, disease education, and payer engagement.
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Published data from preclinical study of RP-A601 in PKP2-ACM. "AAV-Mediated Delivery of Plakophilin-2a Arrests Progression of Arrhythmogenic Right Ventricular Cardiomyopathy in Murine Hearts: Preclinical Evidence Supporting Gene Therapy in Humans" was published in Circulation: Genomic and Precision Medicine. Results from the study demonstrated the potential of RP-A601 as a gene therapy for patients with PKP2-ACM.
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Bolstered commercial and operational expertise of Board of Directors with appointment of R. Keith Woods. Mr. Woods is a seasoned executive with more than 30 years of experience spanning commercialization, global operations, business strategy and supply chain. He recently served as Chief Operating Officer of argenx, where he led its global commercial organization, including marketing, market access, medical affairs, program management, and supply chain, during the company’s successful transition to commercial stage. Mr. Woods launched Vyvgart, a treatment for a rare autoimmune condition that causes muscle weakness, generating more than $1 billion in sales.
Upcoming Investor Conferences
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Cowen’s 44th Annual Health Care Conference: March 5, 2024
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Leerink Partners Global Biopharma Conference: March 12, 2024
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Needham 23rd Annual Virtual Healthcare Conference: April 9, 2024
Fourth Quarter and Full Year 2023 Financial Results
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Cash position. Cash, cash equivalents and investments as of December 31, 2023, were $407.5 million.
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R&D expenses. Research and development expenses were $41.7 million and $186.3 million for the three and twelve months ended December 31, 2023, respectively, compared to $50.0 million and $165.6 million for the three and twelve months ended December 31, 2022, respectively. The increase in R&D expenses for the twelve months ended December 31, 2023, was primarily driven by increased compensation and benefits expense of $16.9 million due to increased R&D headcount, increased clinical trial costs of $14.5 million, and increased non-cash stock compensation expense of $5.0 million, offset by a decrease in manufacturing and development costs of $17.0 million.
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G&A expenses. General and administrative expenses were $21.5 million and $73.3 million for the three and twelve months ended December 31, 2023, respectively, compared to $19.0 million and $58.8 million for the three and twelve months ended December 31, 2022, respectively. The increase in G&A expenses for the twelve months ended December 31, 2023, was primarily driven by increases in commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $8.4 million and increased non-cash stock compensation expense of $3.4 million.
Net loss.
Net loss was $59.7 million and $245.6 million or $0.64 and $2.92 per share (basic and diluted) for the three and twelve months ended December 31, 2023, compared to $66.7 million and $221.9 million or $0.92 and $3.26 per share (basic and diluted) for the three and twelve months ended December 31, 2022.
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Shares outstanding.
90,282,267 shares of common stock were outstanding as of December 31, 2023.
Financial Guidance
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Cash position. As of December 31, 2023, Rocket had cash, cash equivalents and investments of $407.5 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.