- Preliminary Phase 1 Data Expected by the End
of 2019 -
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a
leading U.S.-based multi-platform clinical-stage gene therapy
company, today announces that the first patient received
investigational therapy in the open-label, Phase 1/2 clinical trial
of RP-L201. RP-L201 is the Company’s lentiviral vector (LVV)-based
gene therapy for the treatment of severe Leukocyte Adhesion
Deficiency-I (LAD-I) that was in-licensed from the Centro de
Investigaciones Energéticas, Medioambientales y Tecnológicas
(CIEMAT: Madrid, Spain). The lentiviral vector was developed in a
collaboration between The University College of London (UCL) and
CIEMAT. The University of California, Los Angeles (UCLA) Mattel
Children’s Hospital, is the lead U.S. clinical research center
under the leadership of Donald B. Kohn, M.D. Dr. Kohn is Professor
of Microbiology, Immunology and Molecular Genetics, Pediatrics
(Hematology/Oncology), Molecular and Medical Pharmacology, a member
of the Eli and Edythe Broad Center of Regenerative Medicine and
Stem Cell Research at UCLA, and is principal investigator of the
trial.
“We are excited to commence patient dosing in our Phase 1/2
trial of RP-L201 in support of registration, as it not only offers
hope for very young patients and families affected by this
devastating disease, but also a potential first path to approval
for Rocket’s gene therapy platform,” said Gaurav Shah, M.D., Chief
Executive Officer and President of Rocket. “LAD-I represents a
major area of unmet medical need as most severely-afflicted
patients die before the age of 2 in the absence of a hematopoietic
stem cell transplant, which is associated with graft-versus-host
disease. Our hope is that patients would benefit from a one-time
administration of autologous gene therapy facilitated with busulfan
conditioning. This process does not require immunosuppression and
eliminates the risk of graft-versus-host disease.”
The non-randomized, open-label Phase 1/2 trial is expected to
enroll nine pediatric patients globally. The Phase 1 portion of the
trial is expected to enroll two patients and will assess the safety
and tolerability of RP-L201. The Phase 2 portion of the trial will
evaluate overall survival at multiple sites globally (U.S. and
E.U.). Further information about the clinical program is available
here.
About Leukocyte Adhesion Deficiency-I
Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare,
autosomal recessive pediatric disease caused by a mutation of the
ITGB2 gene that encodes 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 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, mortality in patients with severe LAD-I is
60-75% prior to the age of 2 and survival beyond the age of 5 is
exceedingly rare. The unmet medical need for patients with severe
LAD-I is therefore significant.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) is an
emerging, clinical-stage biotechnology company focused on
developing first-in-class gene therapy treatment options for rare,
devastating diseases. Rocket’s multi-platform development approach
applies the well-established lentiviral vector (LVV) and
adeno-associated viral vector (AAV) gene therapy platforms.
Rocket's first two clinical programs using 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, and Leukocyte Adhesion Deficiency-I (LAD-I), a severe
pediatric genetic disorder that causes recurrent and
life-threatening infections which are frequently fatal. Rocket’s
first clinical program using AAV-based gene therapy is for Danon
disease, a devastating, pediatric heart failure condition. Rocket’s
pre-clinical pipeline programs for bone marrow-derived disorders
are for Pyruvate Kinase Deficiency (PKD) and Infantile Malignant
Osteopetrosis (IMO). 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, effectiveness and
timing of product candidates that Rocket may develop, to treat
Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I),
Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis
(IMO) and Danon disease, 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 "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. 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 successfully demonstrate the efficacy and safety of such
products and pre-clinical studies and clinical trials, its gene
therapy programs, the pre-clinical and clinical results for its
product candidates, which may not support further development and
marketing approval, the potential advantages of Rocket's product
candidates, actions of regulatory agencies, which may affect the
initiation, timing and progress of pre-clinical studies and
clinical trials of its product candidates, Rocket's and its
licensors’ ability to obtain, maintain and protect its and their
respective intellectual property, the timing, cost or other aspects
of a potential commercial launch of Rocket's product candidates,
Rocket's ability to manage operating expenses, Rocket's ability to
obtain additional funding to support its business activities and
establish and maintain strategic business alliances and new
business initiatives, Rocket's dependence on third parties for
development, manufacture, marketing, sales and distribution of
product candidates, the outcome of litigation, and 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, 2018. 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/20190905005285/en/
Claudine Prowse, Ph.D. SVP, Strategy & Corporate Development
Rocket Pharma, Inc. The Empire State Building, Suite 7530 New York,
NY 10118 www.rocketpharma.com investors@rocketpharma.com
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