On February 24, 2022 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported its financial and operational results for the fourth quarter and year ended December 31, 2021 (Press release, Rocket Pharmaceuticals, FEB 24, 2022, View Source [SID1234609007]).
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"2021 was a very productive year marked by clinical data supporting potential durable cures in three devastating bone marrow-derived diseases, and for the first time in history by the demonstration of potential durable clinical benefit in a cardiac disease using a gene therapy approach," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "The stage is set for 2022 to be an important year for Rocket in which we anticipate progression of the Danon Disease program toward a registrational trial, as well as top-line data from our pivotal LAD-I and Fanconi Anemia clinical trials that move us toward registration. I am growing increasingly confident about our unique gene therapy approach at Rocket focused on addressing specific rare diseases using highly targeted, enabled technologies with a proven track record. Additionally, we anticipate in-house AAV cGMP manufacturing readiness in the second quarter of this year, as we rapidly scale our capabilities and work towards commercial readiness."
Dr. Shah continued, "I am extremely proud of the positive momentum witnessed in 2021 and our steadfast focus on superior execution. Notably, we announced positive data from our Phase 1 trial in Danon Disease showing clinical, functional and biomarker improvements at one year or beyond, and we initiated treatment in the pediatric patient cohort at the low-dose, with pediatric data anticipated in the third quarter of 2022. In our pivotal Phase 2 trials in LAD-I and Fanconi Anemia, the favorable safety and efficacy observed are very encouraging as we approach top-line data readouts. We were also pleased with the progress of our PKD program demonstrating sustained normal-range hemoglobin through 12-months post-treatment in two adult patients in the Phase 1 trial. As we continue accelerating our current first wave of clinical programs, we are also poised to grow our Wave 2 pipeline based on our core R&D strategy from this year onward. We are excited by the momentum and look forward to continuing on our mission of seeking gene therapy cures for young patients and their families facing such devastating diseases."
Key Pipeline and Operational Updates
Announced positive clinical data from ongoing Phase 1 trial of RP-A501 for Danon Disease. In November 2021, the Company reported updated data from the Phase 1 trial evaluating a single intravenous infusion of RP-A501 for the treatment of Danon Disease, including interim safety and efficacy data from patients in the low-dose (6.7e13 vg/kg; n=3) and high-dose (1.1e14 vg/kg; n=2) adult and adolescent cohorts. Results demonstrated sustained benefit across clinical, functional and biomarker endpoints in all four patients with long-term follow up. RP-A501 was generally well tolerated at the low-dose. Data from the pediatric patient cohort of the Phase 1 trial are expected in the third quarter of 2022 and Phase 2 trial activities are expected to begin in the fourth quarter of 2022.
Announced positive clinical data from ongoing pivotal Phase 2 trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). The Company provided an update in its presentation at the 40th Annual J.P. Morgan Healthcare Conference, indicating engraftment in all nine severe LAD-I patients who have received RP-L201 in the pivotal Phase 2 trial; all patients had at least three months of follow-up and demonstrated CD18 expression ranging from 26% to 87% of normal. The most recently treated patient has engrafted with neutrophil CD18 expression of 61% at three months. The Company previously presented preliminary data from eight of nine severe LAD-I patients who received RP-L201 treatment at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Top-line data from the pivotal Phase 2 trial are expected in the second quarter of 2022.
Announced positive clinical data from ongoing pivotal Phase 2 trial of RP-L102 for Fanconi Anemia (FA). Data presented at the 63rd ASH (Free ASH Whitepaper) Annual Meeting included preliminary data from 11 pediatric FA patients who were treated with RP-L102 as of the Nov. 1, 2021, cut-off date. Evidence of engraftment was observed in six of eight patients with at least 12-months of follow-up with MMC resistance between 16% and 63% at a minimum of one timepoint. The tolerability profile of RP-L102 appeared favorable and all patients were treated without conditioning. Top-line data from the pivotal Phase 2 trial are expected in the third quarter of 2022.
Announced positive clinical data from ongoing Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD). Data presented at the 63rd ASH (Free ASH Whitepaper) Annual Meeting included preliminary data from two adult PKD patients with significant anemia and transfusion requirements who were treated with RP-L301 as of the Nov. 3, 2021, cut-off date. RP-L301 was well tolerated, with no drug product-related serious adverse events or infusion-related complications observed through 12-months post-treatment. Preliminary clinical activity was observed in both patients at 12-months post-RP-L301 infusion. Both patients reported improved quality of life following treatment. Preliminary Phase 1 data and Phase 2 trial initiation activities are expected in the fourth quarter of 2022.
Received Regenerative Medicine Advanced Therapy (RMAT) designation and Priority Medicines (PRIME) designation for RP-L201 gene therapy for the treatment of LAD-I. RMAT designation allows companies to work closely with the U.S. Food and Drug Administration (FDA) on a program’s development and includes all the benefits of the FDA’s Fast Track and Breakthrough Therapy designations. The European Medicine Agency’s (EMA) PRIME program aims to optimize development plans and speed up evaluation of medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. These medicines are considered priority medicines by the EMA and are intended to reach patients earlier. Rocket also holds Rare Pediatric (U.S.), Orphan Drug (U.S./EU), and Advanced Therapy Medicinal Product (EU) designations for the RP-L201 program.
Strengthened leadership team with new appointments. The Company announced the appointment of Isabel Carmona, J.D., as Chief Human Resources Officer in September 2021. She has more than 25 years of experience in human resources and organizational leadership, including the last 15 years within the life sciences industry. Ms. Carmona joined Rocket from Ichnos Sciences, where she was Chief Human Resources Officer. Martin L. Wilson joined Rocket as General Counsel and Chief Compliance Officer in December 2021. Mr. Wilson has nearly 20 years of legal, compliance and executive experience and accomplishment within the life sciences industry. Before Rocket, Mr. Wilson was General Counsel and Chief Corporate Officer at Ichnos Sciences.
Continued buildout of R&D and manufacturing facility. The Company expects to achieve in-house AAV current Good Manufacturing Practice (cGMP) manufacturing readiness in its approximately 100,000-square-foot facility in Cranbury, NJ in the second quarter of 2022.
Continued our commitment to the patient community. In February 2021, the Company recognized Rare Disease Day by leading the "Light Up for Rare" campaign and hosting a month-long series of virtual expert panels to provide educational resources for patients with rare diseases and their families. On February 28, 2021, global landmarks including the Empire State Building and Niagara Falls were lit to drive awareness and foster solidarity among the global rare disease community.
Closed $26.4 million private placement. In August 2021, the Company closed a private placement with a fund affiliated with RTW Investments, LP, the Company’s largest shareholder, for which the net proceeds are expected to be used to continue to advance and expand Rocket’s pipeline of product candidates, for research and development expenses and for working capital.
Anticipated 2022 Milestones
RP-A501 for Danon Disease (AAV)
Report data from pediatric patient cohort of Phase 1 trial – Q3 2022
Initiate pivotal Phase 2 trial activities – Q4 2022
RP-L201 for Leukocyte Adhesion Deficiency-I (LVV)
Report top-line data from pivotal Phase 2 trial – Q2 2022
RP-L102 for Fanconi Anemia (LVV)
Report top-line data from pivotal Phase 2 trial – Q3 2022
RP-L301 for Pyruvate Kinase Deficiency (LVV)
Report preliminary Phase 1 data – Q4 2022
Initiate pivotal Phase 2 trial activities – Q4 2022
Manufacturing Facility in Cranbury, New Jersey
Achieve in-house AAV current Good Manufacturing Practice (cGMP) manufacturing readiness – Q2 2022
Upcoming Investor Conferences
Cowen’s 42nd Annual Health Care Conference – March 7-9, 2022
Guggenheim Genomic Medicines & Rare Disease Day – March 31-April 1, 2022
Needham 21st Annual Virtual Healthcare Conference – April 11-14, 2022
Fourth Quarter and Full Year 2021 Financial Results
Cash position. Cash, cash equivalents and investments as of December 31, 2021, were $388.7 million.
R&D expenses. Research and development expenses were $32.2 million and $125.5 million for the three and twelve months ended December 31, 2021, compared to $50.1 million and $105.4 million for the three and twelve months ended December 31, 2020. The increase in research and development expenses for the three and twelve months ended December 31, 2021, was primarily driven by an increase in manufacturing and development costs, compensation and benefits expense due to increased R&D headcount and an increase in non-cash stock compensation expense, offset by a decrease in non-cash new research agreements expenses.
G&A expenses. General and administrative expenses were $12.2 million and $41.8 million for the three and twelve months ended December 31, 2021, compared to $9.1 million and $28.9 million for the three and twelve months ended December 31, 2020. The increase in general and administrative expenses for three and twelve months ended December 31, 2021, was primarily driven by an increase in non-cash stock compensation expense, an increase in compensation and benefits expense due to increased G&A headcount, an increase in office and administrative costs, and an increase in commercial preparation expenses, offset by a decrease in debt conversion expense.
Net loss. Net loss was $44.2 million and $169.1 million or $0.69 and $2.67 per share (basic and diluted) for the three and twelve months ended December 31, 2021, compared to $60.9 million and $139.7 million or $1.08 and $2.52 per share (basic and diluted) for the three and twelve months ended December 31, 2020.
Shares outstanding. 64,505,889 shares of common stock were outstanding as of December 31, 2021.
Financial Guidance
Cash position. As of December 31, 2021, we had cash, cash equivalents and investments of $388.7 million. Rocket expects such resources will be sufficient to fund its operations into the second half of 2023, including the continued buildout and initiation of AAV cGMP manufacturing capabilities at our Cranbury, New Jersey R&D and manufacturing facility and continued development of our four clinical programs.