On August 10, 2023 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, reported financial results for the quarter ending June 30, 2023, and updates from the Company’s key pipeline developments, business operations and upcoming milestones (Press release, Rocket Pharmaceuticals, AUG 10, 2023, View Source [SID1234634245]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"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
Received European designations including Priority Medicines (PRIME) and Advanced Therapy Medicinal Products (ATMP) for RP-A501. PRIME designation offers the benefits of early and enhanced support from the European Medicines Agency (EMA) and the opportunity for accelerated marketing application review. It is granted to medicines that target an unmet need for which no treatment option exists and with data showing a meaningful improvement of clinical outcomes. ATMP designation is intended for medicines that offer groundbreaking new opportunities and allows for a single evaluation and authorization procedure.
Positive clinical data from the Phase 1 trial of RP-A501 presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. As of May 2023, all six optimally enrolled patients in the Danon Disease Phase 1 trial continued to demonstrate improvement or stabilization in ongoing follow-up of six to 36 months. Patients would have typically experienced progressive disease or death without treatment.
Initiating two-patient pediatric safety run-in for RP-A501 pivotal study; approaching final alignment with FDA on primary composite endpoint to support accelerated approval. Rocket has obtained IRB approvals and begun screening activities. Initial clinical sites are the University of California, San Diego (UCSD) and Children’s Hospital of Philadelphia.
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Received FDA Fast Track and Orphan Drug designations for RP-A601. Fast Track designation facilitates the development and expedited review of medicines that treat serious conditions and fill an unmet medical need. It also enables increased communication with the FDA, offering the potential for accelerated approval and priority review if criteria are met, and permits a rolling Biologics License Application (BLA) review. Orphan Drug designation is granted to support the development of medicines for rare disorders and provides certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials.
Positive proof of concept from preclinical studies of RP-A601 presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. Data demonstrated decreased arrhythmias and increased survival in the PKP2 knockout mouse model. Based on these data and the completion of Investigational New Drug (IND)-enabling toxicology studies, Rocket received IND clearance from the FDA for a Phase 1 study of RP-A601 that will assess the impact of RP-A601 on PKP2 myocardial protein expression, cardiac biomarkers, and clinical predictors of life-threatening ventricular arrhythmias and sudden cardiac death.
Phase 1 start-up activities for RP-A601 are well progressed following IND clearance to allow for first patient treatment. Rocket has gained IRB approval at UCSD, initiated Phase 1 study start up activities and is rapidly advancing toward first patient treatment.
LV Hematology Portfolio
Leukocyte Adhesion Deficiency-I (LAD-I)
Positive clinical data from pivotal Phase 2 trial of RP-L201 presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. Positive, updated top-line data demonstrated 100% overall survival at 12 months post-infusion (and for entire duration of follow-up) via Kaplan Meier estimate for all nine LAD-I patients with 12 to 24 months of available follow-up. Data also showed evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities. The safety profile was highly favorable in all patients with no RP-L201-related serious adverse events.
BLA submitted to FDA for RP-L201. Based on the positive efficacy and safety data from the pivotal study of RP-L201, Rocket submitted the BLA to the FDA, as its first product filing.
Fanconi Anemia (FA)
Positive clinical data from pivotal Phase 2 trial of RP-L102 presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. Positive, updated top-line data showed sustained genetic correction in eight of 12 evaluable patients and comprehensive phenotypic correction in seven of 12 evaluable patients with ≥12 months of follow up as demonstrated by increased resistance to mitomycin-C (MMC) in bone marrow (BM)-derived colony forming cells and hematologic stabilization. The safety profile was highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated.
BLA submission for RP-L102 anticipated in Q4 2023. Based on the positive efficacy and safety data from the pivotal study of RP-L102, Rocket is on track to submit the BLA during the fourth quarter of 2023 and anticipates providing an update following FDA acceptance.
Pyruvate Kinase Deficiency (PKD)
Presented positive clinical data from Phase 1 trial of RP-L301 at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. Data showed robust and sustained efficacy in both adult patients for up to 30 months. Results from the first pediatric patient indicate preliminary efficacy and favorable safety.
Received EMA Priority Medicines (PRIME) designation for RP-L301 in July. PRIME designation offers the benefits of early and enhanced support from the European Medicines Agency (EMA) and the opportunity for accelerated marketing application review. It is granted to medicines that target an unmet need for which no treatment option exists and with data showing a meaningful improvement of clinical outcomes.
Received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for RP-L301. RMAT designation was granted based on robust safety and efficacy data from the ongoing Phase 1 study and its potential to cure a life-threatening disease for which no curative therapies currently exist. The designation will provide the benefits of added FDA guidance and expedited review through the program’s development. All four Rocket-sponsored programs with clinical data have received RMAT designation.
Adult and pediatric enrollment are completed in the Phase 1 study. Phase 2 pivotal trial initiation activities are anticipated in the fourth quarter of 2023.
Operational Update
Entered into data licensing agreement with Invitae to support timely access to genetic testing for patients who may have genetic forms of cardiomyopathy. The Invitae Unlock Cardiomyopathy and Arrhythmia genetic testing program evaluates causal mutations in approximately 170 genes, including LAMP2, PKP2 and BAG3, that result in Danon Disease, PKP2-ACM and BAG3-DCM, respectively. As part of the agreement, Rocket is raising awareness of this testing program and educating healthcare professionals across the U.S. on the benefits of screening for genetic forms of cardiomyopathy.
Second Quarter Financial Results
Cash position. Cash, cash equivalents and investments as of June 30, 2023, were $307.0 million.
R&D expenses. Research and development expenses were $51.4 million for the three months ended June 30, 2023, compared to $41.4 million for the three months ended June 30, 2022. The increase in R&D expenses was primarily driven by increases in compensation and benefits expense of $5.7 million due to increased R&D headcount, clinical trial expenses of $2.7 million, and non-cash stock-based compensation of $1.7 million.
G&A expenses. General and administrative expenses were $17.4 million for the three months ended June 30, 2023, compared to $12.9 million for the three months ended June 30, 2022. The increase in G&A expenses was primarily driven by increases in commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $1.3 million, compensation and benefits of $0.8 million due to increased G&A headcount and non-cash stock compensation expense of $1.1 million.
Net loss. Net loss was $65.7 million or $0.82 per share (basic and diluted) for the three months ended June 30, 2023, compared to $54.4 million or $0.83 (basic and diluted) for the three months ended June 30, 2022.
Shares outstanding. 80,521,415 shares of common stock were outstanding as of June 30, 2023.
Financial Guidance
Cash position. As of June 30, 2023, Rocket had cash, cash equivalents and investments of $307.0 million. Rocket expects such resources will be sufficient to fund its operations into the first half of 2025, 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.