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 needs,
today announced a regulatory update for KRESLADI™ (marnetegragene
autotemcel; marne-cel), a lentiviral (LV) vector-based gene therapy
to treat severe leukocyte adhesion deficiency-I (LAD-I). The U.S.
Food and Drug Administration (FDA) has issued a Complete Response
Letter (CRL) in response to Rocket’s Biologics License Application
for KRESLADI™ wherein the FDA requested limited additional
Chemistry Manufacturing and Controls (CMC) information to complete
its review.
Rocket met with FDA senior leaders from Center for Biologics
Evaluation and Research (CBER) to align on the limited scope of
additional CMC information needed to support the approval of
KRESLADI™ as quickly as possible. “It is reassuring to have the FDA
as a close collaborator who understands the high unmet medical
need, clear clinical benefit and importance of timely patient
access,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket
Pharma. “CBER leadership’s direct involvement and commitment to
working expeditiously to deliver this therapy to patients gives us
great hope on behalf of the primary immunodeficiency
community.”
Positive top-line data from the global Phase 1/2 study of
KRESLADI™ demonstrated 100% overall survival at 12 months
post-infusion (and for the entire duration of follow-up) for all
nine LAD-I patients with 18 to 42 months of available follow-up.
Data also showed large decreases compared with pre-treatment
history in the incidences of significant infections, combined with
evidence of resolution of LAD-I-related skin lesions and
restoration of wound repair capabilities. All primary and secondary
endpoints were met, and KRESLADI™ was well tolerated in all
patients with no treatment-related serious adverse events.
About KRESLADI™ (marnetegragene autotemcel; marne-cel)
KRESLADI™ is an investigational gene therapy that contains
autologous (patient-derived) hematopoietic stem cells that have
been genetically modified with a lentiviral (LV) vector to deliver
a functional copy of the ITGB2 gene, which encodes for the beta-2
integrin component CD18, a key protein that facilitates leukocyte
adhesion and enables their extravasation from blood vessels to
fight infection.
Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT),
Rare Pediatric, and Fast Track designations in the U.S., PRIME and
Advanced Therapy Medicinal Product (ATMP) designations in the EU,
and Orphan Drug designations in both regions for the program.
KRESLADI™ was in-licensed from the Centro de Investigaciones
Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de
Investigación Biomédica en Red de Enfermedades Raras and Instituto
de Investigación Sanitaria Fundación Jiménez Díaz. The lentiviral
vector was developed in a collaboration between University College
London and CIEMAT.
About Leukocyte Adhesion Deficiency-I Severe Leukocyte
Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive
pediatric disease caused by mutations in the ITGB2 gene encoding
for the beta-2 integrin component CD18. CD18 is a key protein that
facilitates leukocyte adhesion and extravasation from blood vessels
to combat infections. As a result, children with severe LAD-I are
often affected immediately after birth. During infancy, they suffer
from recurrent life-threatening bacterial and fungal infections
that respond poorly to antibiotics and require frequent
hospitalizations. Children who survive infancy experience recurrent
severe infections including pneumonia, gingival ulcers, necrotic
skin ulcers, and septicemia. Without a successful bone marrow
transplant, survival beyond childhood is rare. LAD-I is estimated
to impact an estimated 800 to 1,000 individuals in the U.S. and
Europe. Currently the only potential curative treatment is an
allogeneic hematopoietic stem cell transplant, which may not be
available in time for these children and itself has substantial
morbidity and mortality. There is a high unmet medical need for
patients with severe LAD-I. Rocket’s LAD-I research is made
possible by a grant from the California Institute for Regenerative
Medicine (Grant Number CLIN2-11480). The contents of this press
release are solely the responsibility of Rocket and do not
necessarily represent the official views of CIRM or any other
agency of the State of California.
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 “could,” “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, including the
timing and outcome of the FDA’s review of the additional CMC
information that Rocket will provide in response to the FDA’s
request, the safety, effectiveness and timing of 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/20240628434643/en/
Media & Investors Meg Dodge
mdodge@rocketpharma.com
Media Kevin Giordano media@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
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