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 U.S. Food and Drug
Administration (FDA) has granted Regenerative Medicine Advanced
Therapy (RMAT) designation to RP-L301, the Company’s
investigational lentiviral-based gene therapy for Pyruvate Kinase
Deficiency (PKD), a rare blood disorder characterized by severe
anemia and excessive red blood cell breakdown. RMAT designation was
granted based on robust safety and efficacy data from the ongoing
Phase 1 RP-L301 clinical trial and its potential to cure a
life-threatening disease for which no curative therapies currently
exist. The designation will provide the benefits of added FDA
guidance and expedited review through the program’s
development.
“Receiving RMAT designation from the FDA for RP-L301 is a major
achievement in our pursuit to bring the first, potentially curative
gene therapy treatment to patients living with PKD who have high
unmet need. Notably, PKD has an estimated prevalence of up to 8,000
patients in the U.S. and Europe and represents one of the most
significant patient opportunities in our LV hematology portfolio,”
said Kinnari Patel, PharmD, MBA, President and Chief Operating
Officer, Rocket Pharma. “Further, all four Rocket-sponsored
programs with clinical data now have received RMAT designation from
the FDA across both platforms, a unique showcase of our team’s
ability both to select appropriate targets and develop gene
therapies for them.”
Dr. Patel continued, “Results from the RP-L301 program
demonstrate robust efficacy in both adult patients for up to 30
months with a highly favorable safety profile and were recently
presented at ASGCT. The first pediatric patient has shown promising
initial results similar to the adults, and enrollment has been
completed in the Phase 1 study. We look forward to initiating the
Phase 2 pivotal trial in the fourth quarter of 2023 as we continue
to advance our world-class pipeline for patients facing such rare
and devastating diseases.”
Established under the 21st Century Cures Act, RMAT designation
is a dedicated program designed to expedite the drug development
and review processes for promising pipeline products, including
gene therapies. A regenerative medicine therapy is eligible for
RMAT designation if it is intended to treat, modify, reverse or
cure a serious or life-threatening disease or condition, and
preliminary clinical evidence indicates that the drug or therapy
has the potential to address unmet medical needs for such disease
or condition.
Similar to Breakthrough Therapy designation, RMAT designation
provides the benefits of intensive FDA guidance on efficient drug
development, including the ability for early FDA interactions to
discuss surrogate or intermediate endpoints, potential ways to
support accelerated approval and satisfy post-approval
requirements, potential priority review of the biologics license
application (BLA) and other opportunities to expedite development
and review. In addition to RMAT, RP-L301 has also received Fast
Track and Orphan Drug Designation.
Results from the Phase 1 program presented recently at the 26th
Annual Meeting of the American Society of Gene and Cell Therapy
(ASGCT) demonstrate robust and sustained efficacy in both adult
patients up to 30 months post-infusion demonstrated by normalized
hemoglobin (from baseline levels in the 7.0-7.5 g/dL range),
improved hemolysis parameters, red blood cell transfusion
independence and improved quality of life with documented
improvements via formal quality of life assessments. The safety
profile appears highly favorable, with no RP-L301-related serious
adverse events in either of the adult patients.
The first pediatric patient results suggest efficacy similar to
the adult cohort with an initial greater than five-point increase
in hemoglobin (from median baseline level of 7.9 g/dL). The
infusion was well tolerated, with engraftment achieved at day +15,
hospital discharge less than one month following infusion, and no
RP-L301-related serious adverse events or red blood cell
transfusion requirements following engraftment.
About Pyruvate Kinase Deficiency
Pyruvate Kinase Deficiency (PKD) is a rare, monogenic red blood
cell disorder resulting from a mutation in the PKLR gene encoding
for the pyruvate kinase enzyme, a key component of the red blood
cell glycolytic pathway. Mutations in the PKLR gene result in
increased red blood cell destruction and the disorder ranges from
mild to life-threatening anemia. PKD has an estimated prevalence of
4,000 to 8,000 patients in the U.S. and Europe. Children are the
most commonly and severely affected subgroup of patients. Patients
with PKD have a high unmet medical need, as currently available
treatments include splenectomy and red blood cell transfusions,
which are associated with immune defects and chronic iron overload.
Recently, mitapivat, an oral enzyme activator, was approved for use
in adult patients, however its efficacy is limited in more
severely-afflicted patients, most notably in those who are
splenectomized, transfusion-dependent, or whose disease results
from deleterious mutations.
RP-L301 was in-licensed from the Centro de Investigaciones
Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de
Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and
Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz
(IIS-FJD).
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.
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version on businesswire.com: https://www.businesswire.com/news/home/20230523005548/en/
Media Kevin Giordano kgiordano@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
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