First clinical gene therapy program for
PKP2-ACM, a devastating inherited heart disease affecting
approximately 50,000 people in the U.S. and EU
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
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, today announced that it has received clearance
from the U.S. Food and Drug Administration (FDA) for the Company’s
Investigational New Drug (IND) application for RP-A601, an
AAV.rh74-based gene therapy candidate for the treatment of
arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic
variants (PKP2-ACM) a devastating inherited heart disease that can
lead to life-threatening arrhythmias, cardiac structural
abnormalities, and sudden cardiac death. The current standard of
care for patients with PKP2-ACM consists of medical therapy,
implantable cardioverter defibrillators (ICDs), and ablations,
which are not curative. Even with treatment, life-threatening
arrhythmias and progression of disease may still occur. PKP2-ACM
affects approximately 50,000 people in the U.S. and Europe.
“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 Kinnari Patel, PharmD, MBA,
President and Chief Operating Officer, Rocket Pharma. “RP-A601
offers the potential of a one-time, curative alternative to medical
therapy, ICDs, and ablations which are associated with adverse
effects, complications, and recurrence of arrhythmias and do not
halt the progression of disease. Robust preclinical proof of
concept has demonstrated decreased arrhythmias and increased
survival. With IND clearance in hand, we are rapidly advancing the
first investigational gene therapy for PKP2-ACM into the
clinic.”
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 Medicine resulted 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 Professor
of 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 Center and Joseph
Rossano, M.D., M.S., FAAP, FACC, Co-Director of the Cardiac Center
and Chief of the Division of Cardiology at 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 American
Society of Gene and Cell Therapy (ASGCT) on Friday, May 19, 2023,
8:00 a.m. – 9:45 a.m. PT. More information can be found here.
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 U.S. and Europe. Patients living with PKP2-ACM have an urgent
unmet medical need, as current medical, implantable cardioverter
defibrillator (ICD), and ablation therapies do not consistently
prevent disease progression or arrhythmia recurrence, are
associated with significant morbidity including inappropriate
shocks and device and procedure-related complications, and do not
address the underlying pathophysiology or genetic mutation. RP-A601
is being investigated as a one-time, potentially curative gene
therapy treatment that may improve survival and quality of life for
patients affected by this devastating disease.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an
integrated and sustainable pipeline of investigational genetic
therapies designed to correct the root cause of complex and rare
disorders. The Company’s platform-agnostic approach enables it to
design the best therapy for each indication, creating potentially
transformative options for patients afflicted with rare genetic
diseases. Rocket's clinical programs using lentiviral vector (LV)
based gene therapy are for the treatment of Fanconi Anemia (FA), a
difficult to treat genetic disease that leads to bone marrow
failure and potentially cancer, Leukocyte Adhesion Deficiency-I
(LAD-I), a severe pediatric genetic disorder that causes recurrent
and life-threatening infections which are frequently fatal, and
Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell
disorder resulting in increased red cell destruction and mild to
life-threatening anemia. Rocket’s first clinical program using
adeno-associated virus (AAV)-based gene therapy is for Danon
Disease, a devastating, pediatric heart failure condition. Rocket
also has preclinical AAV-based gene therapy programs in
PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated
dilated cardiomyopathy (DCM). For more information about Rocket,
please visit www.rocketpharma.com.
Rocket Cautionary Statement Regarding Forward-Looking
Statements
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 U.S. Food and Drug Administration (FDA) and to obtain and
maintain FDA or other regulatory authority approval of our product
candidates, Rocket’s dependence on third parties for development,
manufacture, marketing, sales and distribution of product
candidates, the outcome of litigation, our competitors’ activities,
including decisions as to the timing of competing product launches,
pricing and discounting, our integration of an acquired business,
which involves a number of risks, including the possibility that
the integration process could result in the loss of key employees,
the disruption of our ongoing business, or inconsistencies in
standards, controls, procedures, or policies, our ability to
successfully develop and commercialize any technology that we may
in-license or products we may acquire and any unexpected
expenditures, as well as those risks more fully discussed in the
section entitled "Risk Factors" in Rocket’s Annual Report on Form
10-K for the year ended December 31, 2022, filed February 28, 2023
with the SEC and subsequent filings with the SEC including our
Quarterly Reports on Form 10-Q. Accordingly, you should not place
undue reliance on these forward-looking statements. All such
statements speak only as of the date made, and Rocket undertakes no
obligation to update or revise publicly any forward-looking
statements, whether as a result of new information, future events
or otherwise.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230509005540/en/
Media Kevin Giordano kgiordano@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
Rocket Pharmaceuticals (NASDAQ:RCKT)
Gráfico Histórico do Ativo
De Abr 2024 até Mai 2024
Rocket Pharmaceuticals (NASDAQ:RCKT)
Gráfico Histórico do Ativo
De Mai 2023 até Mai 2024