News Release Details
Rocket Pharmaceuticals Announces FDA Clearance of IND for Clinical Trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy (ACM)
May 9, 2023
First clinical gene therapy program for PKP2-ACM, a devastating inherited heart disease affecting approximately 50,000 people in the
Robust preclinical proof of concept studies showed RP-A601 decreased arrhythmias and increased survival in the PKP2 knockout mouse model
Initiating Phase 1 trial start-up activities and rapidly working towards first patient treatment
“Today’s news is a significant milestone, as we build on our leading cardiovascular gene therapy expertise to advance a historic second program targeting the heart into clinical development, this time for patients with PKP2-ACM who have an urgent need for improved treatment options,” said
The multi-center Phase 1, dose escalation trial will evaluate the safety and preliminary efficacy of RP-A601 in at least six adult PKP2-ACM patients with ICDs and overall high risk for arrhythmias. The study 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. Patients in the dose-escalation trial will receive a single dose of RP-A601. The starting dose will be 8 x 1013 GC/kg. The rh74 serotype used in RP-A601 was selected based on a favorable safety profile in gene therapy clinical development programs for other diseases and overall favorable benefit-risk profile in extensive preclinical RP-A601 studies.
RP-A601 Development Highlights
A partnership between Rocket and leading scientific collaborators at
NYU Grossman School of Medicineresulted in vigorous and translationally relevant animal models and preclinical studies. These studies showed that RP-A601 decreased arrhythmias, improved right ventricular morphology and function, and increased survival in PKP2 knockout mice. Scientific collaborators include Mario Delmar, M.D., Ph.D., Patricia and Robert Martinsen Professorof Cardiology and Marina Cerrone, M.D., Research Associate Professor, Co-Director, Inherited Arrhythmia Clinic.
- GMP drug product manufacturing has been completed, and a potency assay has been both developed and qualified.
- A robust clinical protocol has been developed, vetted by an advisory board comprised of experts in PKP2-ACM and gene therapy clinical development and informed by insights from the patient community.
- Natural history studies are planned to provide additional context for the Phase 1 trial.
Initial clinical sites for the Phase 1 trial have been selected. Principal Investigators include
Barry Greenberg, M.D., FHFSA, Director of the Advanced Heart Failure Treatment Program at University of California, San Diego Medical Centerand Joseph Rossano, M.D., M.S., FAAP, FACC, Co-Director of the Cardiac Center and Chief of the Division of Cardiologyat Children's Hospital of Philadelphia.
- Rocket is initiating Phase 1 study start-up activities and rapidly working towards first patient treatment.
Preclinical data from the RP-A601 program will be presented as a late-breaking abstract at the 26th Annual Meeting of the
About PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
PKP2-ACM is an inherited heart disease caused by mutations in the PKP2 gene and characterized by life-threatening ventricular arrhythmias, cardiac structural abnormalities, and sudden cardiac death. PKP2-ACM affects approximately 50,000 adults and children in the
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Various statements in this release concerning Rocket’s future expectations, plans and prospects, including without limitation, Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, Rocket’s plans for the advancement of its Danon Disease program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as “aim,” “anticipate,” "believe," “can,” “continue,” “design,” “estimate,” "expect," "intend," “may,” "plan," “potential,” "will give," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, our ability to submit regulatory filings with the