— New Long-Term Follow-Up Data Demonstrate
Durable CD18 Expression, Improved Skin Lesions, and Consistent
Peripheral Vector Copy Numbers Up to 18-Months Post-Treatment —
— Second Patient Nearing One-Year Primary
Endpoint for Phase 2 Portion of Study —
— Two Additional Patients with Shorter
Follow-Up Demonstrate Evidence of Engraftment —
— RP-L201 was Well Tolerated in All Patients
—
— More Comprehensive Phase 2 Results Expected
in Second Half of 2021 —
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage
company advancing an integrated and sustainable pipeline of genetic
therapies for rare childhood disorders, today announces positive
interim data from the Company’s Phase 1/2 clinical trial studying
RP-L201, its lentiviral-based gene therapy for the treatment of
severe Leukocyte Adhesion Deficiency-I (LAD-I). Severe LAD-I is a
rare pediatric disease that prevents patients from adequately
combating infections. LAD-I leads to recurrent life-threatening
bacterial and fungal infections that respond poorly to antibiotics,
require frequent hospitalizations and are ultimately fatal. These
results were presented in a virtual poster session at the Clinical
Immunology Society (CIS) 2021 Annual Meeting.
“Today’s positive updates on our LAD-I program add to the
growing body of encouraging evidence that RP-L201 may provide
durable clinical benefit for patients with severe LAD-I who face
recurrent, life-threatening infections from birth,” said Jonathan
Schwartz, M.D., Chief Medical Officer and Senior Vice President of
Rocket. “We are very pleased to report that a second patient is
nearing survival at one-year post-treatment, the primary outcome
measure for the Phase 2 portion of the study. In all patients
treated, CD18 expression has substantially exceeded the 4-10%
threshold associated with survival into adulthood, with consistent
peripheral blood vector copy number levels. Improved
disease-related skin lesions, absence of new infections
post-treatment, and no further requirements for prophylactic
anti-infectives were also observed in both Phase 1 patients with
prolonged follow-up. Initial evidence of engraftment and phenotypic
correction was observed in two additional patients with shorter
follow-up. These updates move us one step closer towards BLA/MAA
filings in the US and Europe and eventual commercialization of a
potentially curative option for the children facing this truly
devastating disease. We look forward to providing more
comprehensive Phase 2 results in the second half of 2021.”
The data reported in the poster presentation are from four
pediatric patients with severe LAD-I, as defined by CD18 expression
of less than 2%. The patients were treated with RP-L201, Rocket’s
ex-vivo lentiviral gene therapy candidate. Data were reported as of
the cutoff date of February 2021. Patient 1001 was 9 years-of-age
at enrollment and had been followed for 18-months after RP-L201
therapy. Patient 1004 was 3 years-of-age at enrollment and had been
followed for 9-months. Patients 2006 and 2005 were 7 months- and 2
years-of-age at enrollment and had been followed for 3-months. Key
highlights from the poster presentation include:
- RP-L201 was well tolerated, no safety issues reported with
infusion or treatment
- All patients achieved hematopoietic reconstitution within
5-weeks
- Neutrophil CD18-expression and peripheral blood vector copy
numbers (VCN) were assessed post-treatment to evaluate engraftment
and phenotypic correction:
- 18-months post-treatment, Patient 1001 demonstrated durable
CD18 expression of ~40% and resolution of skin lesions with no new
lesions reported; 12-months post-treatment, peripheral blood VCN
levels were 1.2
- 9-months post-treatment, Patient 1004 demonstrated CD18
expression of ~28%; 6-months post-treatment, peripheral blood VCN
levels were 0.75 with kinetics consistent with those of the first
patient
- 3-months post-treatment, Patient 2006 demonstrated CD18
expression of ~70%; 1.5-months post-treatment, peripheral blood VCN
kinetics were consistent with those of the first two patients
- 3-months post-treatment, Patient 2005 demonstrated CD18
expression of ~51%; 1.5-months post-treatment, peripheral blood VCN
kinetics were consistent with those of the first two patients
To access the poster, please visit: www.rocketpharma.com/CIS
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 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.
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 which are frequently fatal, Pyruvate Kinase Deficiency
(PKD), a rare, monogenic red blood cell disorder resulting in
increased red cell destruction and mild to life-threatening anemia,
and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived
disorder. 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 2021 in light of
COVID-19, 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 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, 2020, filed
March 1, 2021 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/20210414005251/en/
Claudine Prowse, Ph.D. SVP, Strategy & Corporate Development
investors@rocketpharma.com
Rocket Pharmaceuticals (NASDAQ:RCKT)
Historical Stock Chart
From Aug 2024 to Sep 2024
Rocket Pharmaceuticals (NASDAQ:RCKT)
Historical Stock Chart
From Sep 2023 to Sep 2024