On May 6, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders, reported financial results for the quarter that ended March 31, 2020, along with an update on the Company’s key pipeline developments, business operations and upcoming milestones (Press release, Rocket Pharmaceuticals, MAY 6, 2020, View Source [SID1234557156]).
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"During the first quarter, we continued to advance five programs across both the AAV and Lenti platforms," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "Notably this quarter, we completed patient treatment in the low dose cohort for RP-A501 in the Phase 1 Danon Disease trial with no dose-limiting toxicities, and are now pleased to report FDA and IDSMC clearance to advance to a higher dose cohort. This has been an important priority for us as Danon Disease affects so many young patients with devastating heart, muscle and CNS manifestations. We also continued to progress our clinical programs for FA, LAD-I, and PKD. Rocket is at an exciting stage of growth, with a rich pipeline of development-stage opportunities and the potential to transform the lives of patients afflicted with rare disease. In the coming months, we look forward to presenting updated FA ‘Process A’ and LAD-I ‘Process B’ data at ASGCT (Free ASGCT Whitepaper), treating our first patient in a higher dose cohort for RP-A501, and bringing our fifth program, IMO, to the clinic."
Dr. Shah continued, "With regard to the COVID-19 pandemic, we are well-positioned overall to continue our progress while we monitor and adjust to this challenging global crisis. We have experienced modest COVID-19-related pauses in patient enrollment and follow-up that are being managed on a patient-by-patient basis, along with some delays in data collection. Nonetheless, we remain on track for providing updates on our lentiviral pipeline. Assuming that COVID-19 associated delays have shorter-term impact, we remain committed to providing data for Danon by the end of the year. Lastly, we continue to actively monitor the pandemic and refine operations to support the complete safety and well-being of our patients, employees and community."
Key Pipelines and Operational Updates
First cohort of patients completed in Phase 1 trial of RP-A501 for Danon Disease, with U.S. Food and Drug Administration (FDA) and Independent Data Safety Monitoring Committee (IDSMC) clearance to initiate a higher dose cohort. Rocket has treated the first three patients at the low dose of 6.7×1013 vector genomes (vg)/kilogram (kg). No investigational product (IP)-related dose-limiting safety concerns have been observed in any of the three patients treated. The low dose cohort demonstrates a favorable safety profile, and the Company has received clearance from the IDSMC and the FDA to move to a higher dose cohort. Patient treatment in this cohort is anticipated in the third quarter, with preliminary Danon data readout in the fourth quarter.
The Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) clinical studies have continued to progress. Updated data from FANCOLEN-I (utilizing "Process A") will be presented at ASGCT (Free ASGCT Whitepaper), as will updates from the Phase 1 LAD-I study utilizing "Process B." Updates on FA "Process B" data will be presented in the fourth quarter.
The Research & Development (R&D) and Chemistry, Manufacturing and Controls (CMC) facility in Cranbury, New Jersey resumes construction after COVID-19 associated delays. Build-out of the New Jersey facility experienced some construction delays associated with the COVID-19 pandemic, but resumed on May 4, 2020. Occupancy in the new facility is still anticipated in the first half of 2020. Approximately half of the newly constructed 103,720 square foot facility will be dedicated to adeno-associated virus (AAV) Current Good Manufacturing Practice (cGMP) manufacturing. As previously guided, the first cGMP clinical product release is expected in 2021.
Recent publications for FA and Danon Disease provide greater insight into Rocket’s approach for ongoing clinical trials. In February, the peer-reviewed journal Annals of Hematology published Rocket’s comprehensive review of somatic mosaicism in Fanconi Anemia (FA), written in collaboration with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and other international research partners. The article, entitled, "Mosaicism in Fanconi Anemia: Concise review and evaluation of published cases with focus on clinical course of blood count normalization," highlights research describing mosaicism in FA and supports Rocket’s approach of treating patients with RP-L102 without any pre-treatment conditioning measures. In March, Science Translational Medicine published positive preclinical data on Rocket’s Danon program, demonstrating that vector-mediated transfer of LAMP2B to deficient mice improved heart function and survival. The article, "AAV9.LAMP2B Reverses Metabolic and Physiologic Multiorgan Dysfunction in a Murine Model of Danon Disease," underscores the promise of Rocket’s AAV-based gene therapy candidate for Danon, RP-A501.
Rocket recognizes Rare Disease Day with an event at New York City’s Carnegie Hall. On February 29, 2020, Rocket hosted its second annual Rare Disease Day event highlighting the theme "I Am Rare, Hear Me Roar." Over 200 members of the rare disease and broader New York City biotech communities heard from patients and their families, learning more about the impact of rare disease and how to become partners in the fight to find new treatment options. The event culminated with the lighting of the Empire State Building in Rare Disease Day colors, honoring all those affected by rare disease.
Anticipated Milestones
FA (RP-L102)
Additional "Process A" data update (2Q)
Preliminary "Process B" data (4Q)
Danon Disease (RP-A501)
First patient treatment in higher dose (3Q)
Preliminary Phase 1 data (4Q)
LAD-I (RP-L201)
Phase 1 data update on first patient (2Q)
Initiate Phase 2 study (4Q)
Phase 1 data update (4Q)
PKD (RP-L301)
First patient treatment (3Q, previously 2Q)
Preliminary Phase 1 data (4Q)
IMO (RP-L401)
Initiation of clinical study (4Q)
Upcoming Investor Conferences
BofA Securities 2020 Virtual Health Care Conference, May 13, 2020
Jefferies Virtual Healthcare Conference, June 2-4, 2020
Third Quarter Financial Results
Cash position. Cash, cash equivalents and investments as of March 31, 2020, were $275.9 million.
Debt. Our balance sheet includes $52.0 million of fully convertible notes.
R&D expenses. Research and development expenses were $17.0 million for the three months ended March 31, 2020, compared to $15.1 million for the three months ended March 31, 2019. The increase was primarily driven by an increase in clinical trial expenses of $1.5 million.
G&A expenses. General and administrative expenses were $7.2 million for the three months ended March 31, 2020, compared to $3.8 million for the three months ended March 31, 2019. The increase was primarily driven by fees incurred for the convertible notes exchange of $1.6 million, and an increase in compensation expense of $1.3 million due to increased headcount.
Net loss. Net loss was $24.7 million or $0.45 per share (basic and diluted) for the three months ended March 31, 2020, compared to $19.5 million or $0.43 per share (basic and diluted) for the three months ended March 31, 2019.
Shares outstanding. 55,126,474 shares of common stock were outstanding as of March 31, 2020.
Financial Guidance
Cash position. As of March 31, 2020, we had cash, cash equivalents and investments of $275.9 million. Rocket expects such resources will be sufficient to fund its operations into 2022.