Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated,
late-stage biotechnology company advancing a sustainable pipeline
of genetic therapies for rare disorders with high unmet need, today
announced that the European Commission (EC), based on a positive
opinion issued by the Committee for Orphan Medicinal Products
(COMP) of the European Medicines Agency (EMA), has granted orphan
medicinal product designation for RP-A601, the Company’s
adeno-associated virus (AAV.rh74)-based gene therapy candidate for
the treatment of plakophilin-2 related arrhythmogenic
cardiomyopathy (PKP2-ACM).
PKP2-ACM is a devastating, inherited heart disease associated
with life-threatening arrhythmias, cardiac structural
abnormalities, and sudden cardiac death. Presently, there are no
curative treatment options available for PKP2-ACM. The current
standard of care consists of medical therapy, implantable
cardioverter defibrillators (ICDs), and ablation procedures. Even
with treatment, life-threatening arrhythmias and progression of
disease can still occur. PKP2-ACM affects approximately 50,000
people in the U.S. and Europe.
Orphan medicinal product designation by the EC is available to
novel therapeutics that prevent or treat life-threatening or
chronically debilitating conditions that affect fewer than five in
10,000 persons in the European Union (EU). The designation
qualifies for financial and regulatory benefits including protocol
assistance from the EMA during clinical development, access to
centralized marketing authorization, and a 10-year period of
marketing exclusivity after product approval.
The Company is enrolling patients in a Phase 1, dose escalation
trial evaluating the safety and preliminary efficacy of RP-A601 in
at least six adult PKP2-ACM patients with ICDs and who have an
overall high risk for life-threatening arrhythmias. The study is
assessing the impact of RP-A601 on PKP2 myocardial protein
expression, cardiac biomarkers, clinical predictors of
life-threatening ventricular arrhythmias, and sudden cardiac
death.
About RP-A601
RP-A601 is an investigational gene therapy for the treatment of
plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).
RP-A601 consists of a recombinant adeno-associated serotype rh74
(AAVrh74) capsid containing a functional version of the human PKP2
transgene (AAVrh74.PKP2) which is administered as a single
intravenous (IV) infusion. 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
PKP2-ACM. Rocket holds Fast Track designation in the U.S. and
Orphan Drug designation in the U.S. and Europe for the program.
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.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully
integrated, late-stage biotechnology company advancing a
sustainable pipeline of investigational genetic therapies designed
to correct the root cause of complex and rare disorders. Rocket’s
innovative multi-platform approach allows us to design the optimal
gene therapy for each indication, creating potentially
transformative options that enable people living with devastating
rare diseases to experience long and full lives.
Rocket’s lentiviral (LV) vector-based hematology portfolio
consists of late-stage programs for Fanconi Anemia (FA), a
difficult-to-treat genetic disease that leads to bone marrow
failure (BMF) 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 monogenic
red blood cell disorder resulting in increased red cell destruction
and mild to life-threatening anemia.
Rocket’s adeno-associated viral (AAV) vector-based
cardiovascular portfolio includes a late-stage program for Danon
Disease, a devastating heart failure condition resulting in
thickening of the heart, an early-stage program in clinical trials
for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening
heart failure disease causing ventricular arrhythmias and sudden
cardiac death, and a pre-clinical program targeting BAG3-associated
dilated cardiomyopathy (DCM), a heart failure condition that causes
enlarged ventricles.
For more information about Rocket, please visit
www.rocketpharma.com and follow us on LinkedIn, YouTube, and X.
Rocket Cautionary Statement Regarding Forward-Looking
Statements
This press release contains forward-looking statements
concerning Rocket’s future expectations, plans and prospects that
involve risks and uncertainties, as well as assumptions that, if
they do not materialize or prove incorrect, could cause our results
to differ materially from those expressed or implied by such
forward-looking statements. We make such forward-looking statements
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995 and other federal securities laws.
All statements other than statements of historical facts contained
in this release are forward-looking statements. You should not
place reliance on these forward-looking statements, which often
include words such as “believe,” “expect,” “anticipate,” “intend,”
“plan,” “will give,” “estimate,” “seek,” “will,” “may,” “suggest”
or similar terms, variations of such terms or the negative of those
terms. These forward-looking statements include, but are not
limited to, statements concerning 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 DD
program, including its planned pivotal trial, and the safety,
effectiveness and timing of related pre-clinical studies and
clinical trials, Rocket’s ability to establish key collaborations
and vendor relationships for its product candidates, Rocket’s
ability to develop sales and marketing capabilities or enter into
agreements with third parties to sell and market its product
candidates, Rocket’s ability to expand its pipeline to target
additional indications that are compatible with its gene therapy
technologies and Rocket’s ability to transition to a commercial
stage pharmaceutical company. 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 dependence on third parties for
development, manufacture, marketing, sales and distribution of
product candidates, the outcome of litigation, unexpected
expenditures, Rocket’s competitors’ activities, including decisions
as to the timing of competing product launches, pricing and
discounting, Rocket’s ability to develop, acquire and advance
product candidates into, enroll a sufficient number of patients
into, and successfully complete, clinical studies, the integration
of new executive team members and the effectiveness of the newly
configured corporate leadership team, Rocket’s ability to acquire
additional businesses, form strategic alliances or create joint
ventures and its ability to realize the benefit of such
acquisitions, alliances or joint ventures, Rocket’s ability to
obtain and enforce patents to protect its product candidates, and
its ability to successfully defend against unforeseen third-party
infringement claims, 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, 2023, filed February 27,
2024 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240529093516/en/
Media & Investors Meg Dodge
Media Kevin Giordano media@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
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