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 the European Medicines Agency
(EMA) has granted Priority Medicines (PRIME) designation to
RP-A501, the Company’s investigational adeno-associated virus
(AAV)-based gene therapy for the treatment of Danon Disease, a
devastating and fatal inherited cardiomyopathy for which there are
no curative therapies. PRIME designation was granted based on
positive safety and efficacy data from the Phase 1 clinical trial
of RP-A501 in patients with Danon Disease and the potential of
RP-A501 to meet the high unmet medical need in this population.
PRIME designation offers the benefits of early and enhanced
support from the EMA for the development of medicines that target
unmet medical needs, as well as the opportunity for an accelerated
review of the marketing application. Rocket was also recently
granted Regenerative Medicine Advanced Therapy (RMAT) designation
for its RP-A501 program, which also holds Fast Track, Orphan Drug
(U.S.) and Rare Pediatric designations.
“PRIME designation from the EMA further highlights the positive
benefit/risk profile of RP-A501 in addressing the critical unmet
need of patients facing Danon Disease,” said Kinnari Patel,
Pharm.D., MBA, President and Chief Operating Officer, Rocket
Pharma. “We are thrilled by the opportunity that PRIME grants us,
so that we may collaborate with our European Regulatory partners on
the development of RP-A501 in the most expedient and efficient path
forward.”
Results from the Phase 1 trial represent one of the most
comprehensive investigational gene therapy datasets for any cardiac
condition. RP-A501 was associated with a favorable safety profile.
The data demonstrated consistent and robust improvements in
multiple clinical and highly relevant laboratory parameters
including LAMP-2 protein expression, reduced autophagic vacuoles,
brain natriuretic peptide (BNP), high sensitivity troponin I, and
left ventricular mass and wall thickness. In addition, there was
improvement in symptoms, as assessed by New York Heart Association
class and quality of life, as measured by the Kansas City
Cardiomyopathy Questionnaire. Notably, the improvements and
stabilization of BNP in Phase 1 patients were in direct contrast to
worsening patterns observed in patients enrolled in a concurrent,
prospective natural history study. The results demonstrated
improvements and/or normalization across multiple quantifiable
parameters that cardiologists use in clinical practice to enable
risk assessment and treatment decisions.
About EMA PRIME Designation
Priority Medicines (PRIME) designation was created by the
European Medicines Agency (EMA) to enhance support for the
development of innovative medicines that target an unmet medical
need and demonstrate the potential to achieve relevant clinical
outcomes on morbidity, mortality or underlying disease progression.
The PRIME designation offers enhanced early interaction with
companies developing promising medicines, to optimize development
plans and speed up evaluation. PRIME focuses on medicines that may
offer a major therapeutic advantage over existing treatments, or
that benefit patients without treatment options.
About Danon Disease
Danon Disease is a rare X-linked inherited disorder caused by
mutations in the gene encoding lysosome-associated membrane protein
2 (LAMP-2), an important mediator of autophagy. This results in
accumulation of autophagosomes and glycogen, particularly in
cardiac muscle and other tissues, which ultimately leads to heart
failure, and for male patients, frequent death during adolescence
or early adulthood. It is estimated to have a prevalence of 15,000
to 30,000 patients in the U.S. and Europe. The only available
treatment option for Danon Disease is cardiac transplantation,
which is associated with substantial complications and is not
considered curative. There is a high unmet medical need for
patients with Danon 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
childhood 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 (LVV)-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.
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version on businesswire.com: https://www.businesswire.com/news/home/20230531005442/en/
Media Kevin Giordano kgiordano@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
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