Rocket Pharmaceuticals Q1 2024 Financial Results and Recent Progress

Rocket Pharmaceuticals, Inc., a late-stage biotechnology company focused on genetic therapies for rare disorders, reported financial and operational results for the quarter ending March 31, 2024. The company has shown significant progress across its clinical programs, with key regulatory milestones for its Advanced RP-L102 for Fanconi Anemia and the anticipated launch of KRESLADI™ for severe LAD-I.

In April, Rocket announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102, based on positive data from its Phase 1/2 clinical trial. A Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) is expected in the first half of 2024. This marks a crucial step for Rocket in addressing Fanconi Anemia, a genetic disorder leading to bone marrow failure and potentially cancer.

Rocket is also preparing for the potential approval and launch of KRESLADI™ for severe Leukocyte Adhesion Deficiency-I (LAD-I), with a PDUFA date set for June 30, 2024. The company has initiated activities such as disease education, payer engagement, and building out a field team to support the launch of its lentiviral (LV) vector portfolio.

The company further strengthened its leadership by appointing Aaron Ondrey as Chief Financial Officer. Ondrey brings over 20 years of experience in commercial-stage financial management and strategic planning. Additionally, Meg Dodge was named Vice President to lead Investor Relations & Corporate Communications, bringing her expertise in stakeholder engagement from her previous role at Krystal Biotech.

Rocket hosted its annual Rare Disease Day on February 29, 2024, with a theme of “Leap into Action for Rare.” The event featured inspirational stories from the rare disease community and included the "Light Up for Rare" initiative, which lit up buildings and landmarks in Rare Disease Day colors.

In its pipeline, Rocket is advancing three programs from its adeno-associated virus (AAV) cardiovascular portfolio: RP-A501 for Danon Disease, RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM), and preclinical studies for BAG3-associated dilated cardiomyopathy (DCM). The company is also preparing for the Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder.

Rocket plans to present updated data from its LV hematology programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting in May 2024. Presentations will include longer-term data on the safety and efficacy of KRESLADI™ for severe LAD-I and RP-L102 for Fanconi Anemia, as well as RP-L301 for PKD.

Financially, Rocket reported cash, cash equivalents, and investments of $330.3 million as of March 31, 2024. Research and development expenses for the quarter were $45.2 million, a slight decrease from the previous year, while general and administrative expenses rose to $22.1 million. The company’s net loss was $62.1 million, or $0.66 per share, compared to $58.3 million, or $0.73 per share, for the same period in 2023.

Rocket expects its current resources to fund operations into 2026, which includes the production of AAV cGMP batches at its Cranbury, N.J. facility. The company remains committed to advancing its gene therapy programs for rare and devastating diseases, with a focus on execution and supporting the rare disease community through various patient-focused initiatives and educational campaigns.