On November 3, 2022 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, reported that financial results for the quarter ending September 30, 2022, and updates from the Company’s key pipeline developments, business operations and upcoming milestones (Press release, Rocket Pharmaceuticals, NOV 3, 2022, View Source [SID1234622984]).
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"Rocket had a remarkable third quarter highlighted by significant progress in our Phase 1 clinical trial for Danon Disease, with positive early findings demonstrating RP-A501 was generally well tolerated with evidence of positive treatment effect and improvement of the natural course of the disease in pediatric patients with up to nine months follow-up. Additionally, we observed continued, durable robust activity in adults with up to 36 months follow-up," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "As previously announced, we reached an understanding with the FDA on chemistry, manufacturing and controls (CMC) requirements to start AAV cGMP manufacturing at our in-house facility as well as potency assay plans for a Phase 2 pivotal study. We are now laser focused on advancing Phase 2 pivotal study design and endpoint selection with FDA feedback expected this quarter."
Dr. Shah continued, "In the third quarter, we also announced the expected acquisition of Renovacor which aims to further solidify our leadership in AAV-based cardiac gene therapy. By combining Rocket’s clinical, regulatory, and chemistry, manufacturing and control expertise with Renovacor’s compelling preclinical research in BAG3-associated dilated cardiomyopathy and early assets, we look forward to advancing precision gene therapies even further to address the significant unmet need of patients with genetically driven cardiac diseases."
"Based on the strong results presented across our Leukocyte Adhesion Deficiency-I (LAD-I) and Fanconi Anemia (FA) lentiviral gene therapy programs to date, we remain on track towards our first regulatory filings in 2023 – LAD-I in the first half and FA in the second half – and expect to present data on both programs at ASH (Free ASH Whitepaper) in December," said Dr. Shah. "Regarding our Pyruvate Kinase Deficiency (PKD) program, we also expect to share updated data from both adult patients at ASH (Free ASH Whitepaper). Both pediatric patients in the study have been enrolled. We now anticipate a Phase 1 update in the first half of 2023 and initiation of the Phase 2 pivotal trial in 2023."
Dr. Shah continued, "Finally, our exceptional third quarter progress was capped off by an extension of our cash runway into the second half of 2024 following an equity raise with net proceeds of $108.2 million. Taken together, I am very proud of our continued progress at Rocket and look forward to the final push towards another successful year in our pursuit of gene therapy cures for patients and their loved ones facing these devastating, life-threatening genetic diseases."
Key Pipeline and Operational Updates
Announced positive clinical data from ongoing Phase 1 trial of RP-A501 for Danon Disease. Data presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022 included updated safety and efficacy data from patients in the pediatric and adult cohorts. Results showed RP-A501 was generally well tolerated with evidence of treatment effect and improvement of Danon Disease, including for both pediatric patients with up to nine months of follow-up and four adult patients with up to 36 months of follow-up. Efficacy data from the pediatric patients are following similar or more favorable positive trends as in the adults at a similar timeframe. Based on the data available, the Company expects feedback from the FDA on Phase 2 pivotal study design and endpoints this quarter. Previously disclosed data will be presented in an oral presentation at the American Heart Association (AHA) Scientific Sessions 2022 taking place in Chicago, Illinois from November 5-7.
Announced plans to acquire Renovacor, extending leadership in AAV-based cardiac gene therapy. The Company announced plans to acquire Renovacor in an all-stock transaction for an implied value of approximately $2.60 per share, based on the volume weighted average trading price of Rocket shares of $15.51 for the 30 trading days through and including Monday, September 19, 2022. The acquisition is expected to close by the first quarter of 2023.
Phase 2 pivotal studies of RP-L201 for LAD-I and RP-L102 for FA and first regulatory filings remain on track. Based on positive top-line safety and efficacy data from Phase 2 pivotal studies, regulatory filings are anticipated for RP-L201 for LAD-I in the first half of 2023 and for RP-L102 for FA in the second half of 2023.
Clinical data to be presented across all three LV-based programs at ASH (Free ASH Whitepaper) 2022. The Company will present posters from its LV-based gene therapy programs in LAD-I, FA and PKD at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place in New Orleans, Louisiana, from December 10-13, 2022. Updated safety and efficacy data will be presented from the Phase 2 pivotal trial of RP-L102 for FA and Phase 1 trial of RP-L301 for PKD. Previously disclosed data will be presented from the Phase 2 pivotal trial of RP-L201 for LAD-I.
Enrollment completed in Phase 1 trial of RP-L301 for PKD. Both pediatric patients in the Phase 1 study have been enrolled. Updated safety and efficacy data will be presented from the two adult patients at ASH (Free ASH Whitepaper) 2022. The Company now anticipates a Phase 1 update across both cohorts in the first half of 2023 and initiation of the Phase 2 pivotal trial is expected to occur in 2023.
Public offering. On October 6, 2022, the Company completed a public offering of 7,820,000 shares of common stock at a public offering price of $14.75 per share. The net proceeds to Rocket were $108.2 million. All shares in the offering were sold by Rocket. Morgan Stanley, J.P. Morgan and SVB Securities acted as joint book-running managers, and BTIG acted as the lead manager for the offering.
Upcoming Investor Conferences
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Stifel Healthcare Conference 2022, November 15-16, 2022
5th Annual Evercore ISI HealthCONx Conference 2022, November 29-December 1, 2022
Third Quarter Financial Results
Cash position. Cash, cash equivalents and investments as of September 30, 2022, were $306.5 million.
R&D expenses. Research and development expenses were $43.4 million for the three months ended September 30, 2022, compared to $39.6 million for the three months ended September 30, 2021. The increase in research and development expenses was primarily driven by increases in manufacturing and development costs, direct materials, laboratory supplies and compensation and benefits expenses due to increased R&D headcount.
G&A expenses. General and administrative expenses were $15.1 million for the three months ended September 30, 2022, compared to $10.0 million for the three months ended September 30, 2021. The increase in general and administrative expenses was primarily driven by increases in compensation and benefits expense due to increased G&A headcount, non-cash stock compensation expense, consulting expenses and pre-acquisition related expenses.
Net loss. Net loss was $57.8 million or $0.87 per share (basic and diluted) for the three months ended September 30, 2022, compared to $50.1 million or $0.79 per share (basic and diluted) for the three months ended September 30, 2021.
Shares outstanding. 67,838,803 shares of common stock were issued and outstanding as of September 30, 2022.
Financial Guidance
Cash position. As of September 30, 2022, the Company had cash, cash equivalents and investments of $306.5 million. Through September 30, 2022, the Company sold 3.3 million shares of common stock for net proceeds of $46.6 million under its at-the-market facility. With the at-the-market facility proceeds and the October 2022 public offering proceeds, the Company expects that such resources will be sufficient to fund our operating expenses and capital expenditure requirements into the second half of 2024, including the continued buildout and initiation of AAV cGMP manufacturing capabilities at our Cranbury, New Jersey R&D and manufacturing facility, and the continued development of our four clinical programs, as well as future pipeline programs.