— RP-L201 well-tolerated with durable CD18
expression for all patients in Phase 2 pivotal trial —
— 100% overall survival in patients at one year
after RP-L201 infusion based on Kaplan-Meier estimate —
— All patients showed clinical reversal of
disease course, including statistically significant reduction in
all-cause hospitalizations and incidence of severe infections —
— Initiating discussions with health
authorities on filing plans; regulatory filings anticipated in the
first half of 2023 —
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading
late-stage, clinical biotechnology company advancing an integrated
and sustainable pipeline of genetic therapies for rare childhood
disorders with high unmet need, today announces positive top-line
safety and efficacy data from its Phase 2 pivotal trial for severe
Leukocyte Adhesion Deficiency-I (LAD-I) at the 25th Annual Meeting
of the American Society of Gene and Cell Therapy (ASGCT).
“We are excited to present positive top-line safety and efficacy
data from our Phase 2 pivotal trial for LAD-I today at ASGCT,
representing a significant step forward in the development of
RP-L201 for the treatment of LAD-I, one of the most aggressive and
highly fatal immunodeficiencies ever characterized. Moreover, they
point to the great possibilities lentiviral-based gene therapies
can offer for patients with devastating diseases,” said Gaurav
Shah, M.D., Chief Executive Officer of Rocket Pharma. “At three to
24 months after RP-L201 infusion, all nine patients sustained
stable CD18 expression (median: 56%) with no therapy-related
serious adverse events.”
Dr. Shah continued, “We are also pleased to report 100% overall
survival at 12-months post-infusion via Kaplan Meier estimate and a
statistically significant reduction in all hospitalizations,
infection- and inflammatory-related hospitalizations and prolonged
hospitalizations for all nine LAD-I patients with three to 24
months of available follow-up. Data also shows evidence of
resolution of LAD-I-related skin rash and restoration of wound
repair capabilities.”
“While allogeneic transplant options are available, they
continue to be associated with considerable toxicity and today’s
top-line safety and efficacy data point to the potential of RP-L201
to change the treatment paradigm for patients living with severe
LAD-I,” said Dr. Shah. “These results are a testament to the LAD-I
patient community and focus of the Rocket team on steady, reliable
execution. Based on these results, we are initiating discussions
with health authorities on filing plans and anticipate filings in
the first half of 2023.”
Interim Results From an Ongoing Phase 1/2 Study of
Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients
with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
The oral presentation includes efficacy and safety data (cut-off
March 9, 2022) at three to 24 months of follow-up after RP-L201
infusion for all patients and overall survival data for seven
patients at 12 months or longer after infusion. RP-L201 is Rocket’s
ex-vivo lentiviral gene therapy candidate for severe LAD-I.
- All patients, aged five months to nine years, demonstrated
sustained CD18 restoration and expression on more than 10% of
neutrophils (range: 20%-87%, median: 56%).
- At one year, the overall survival without allogeneic
hematopoietic stem cell transplantation across the cohort is 100%
based on the Kaplan-Meier estimate.
- All patients demonstrated a statistically significant reduction
in the rate of all-cause hospitalizations and severe infections,
relative to pre-treatment.
- Evidence of resolution of LAD-I-related skin rash and
restoration of wound repair capabilities has been shown along with
sustained phenotypic correction.
- The safety profile of RP-L201 has been highly favorable in all
patients with no RP-L201-related serious adverse events to
date.
- Adverse events related to other study procedures, including
busulfan conditioning, have been previously disclosed and
consistent with the safety profiles of those agents and
procedures.
About RP-L201
RP-L201 is an investigational gene therapy product being
developed for severe Leukocyte Adhesion Deficiency-I (LAD-I). The
therapy consists of hematopoietic stem cells from the patient that
have been genetically modified with a lentiviral vector to contain
a functional copy of the ITGB2 gene. RP-L201 is currently being
evaluated in a Phase 1/2 clinical trial. The interim analysis of
the trial at three to 24 months of follow-up showed RP-L201 had a
favorable safety profile and evidence of efficacy with durable CD18
expression.
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, mortality in patients with severe LAD-I is
60-75% prior to the age of 2 and survival beyond the age of 5 is
uncommon. 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 advancing an
integrated and sustainable pipeline of genetic therapies that
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 that 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. 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 its guidance for 2022 in light of
COVID-19, 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),
and Danon Disease, the expected timing and data readouts of
Rocket’s ongoing and planned clinical trials, Rocket’s plans for
the advancement of its Danon Disease program following the lifting
of the FDA’s clinical hold and the safety, effectiveness and timing
of related pre-clinical studies and clinical trials, and Rocket’s
plans for the advancement of its LAD-I program based on the data
presented at ASGCT and the potential for therapeutic benefit
related thereto, 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 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, our expectations regarding the delays and impact of
COVID-19 on clinical sites, 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,
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, 2021, filed February 28, 2022 with the SEC.
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/20220519005420/en/
Media Kevin Giordano Director, Corporate Communications
kgiordano@rocketpharma.com
Investors Jessie Yeung, M.B.A. Vice President, Investor
Relations and Corporate Finance investors@rocketpharma.com
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