Final alignment reached on a 12 patient,
single-arm, open label study with a biomarker based co-primary
endpoint assessed at 12 months to support accelerated approval
Co-primary endpoint consisting of LAMP2 protein
expression and Left Ventricular (LV) Mass change from baseline
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 alignment has been reached
with the Food and Drug Administration (FDA) on the global Phase 2
pivotal trial of RP-A501 for Danon Disease. Danon Disease is a
uniformly fatal inherited cardiomyopathy that leads to mortality in
the majority of male patients at age ~20 and females at age ~40,
and for which there are no approved curative or disease-modifying
therapies. The disease affects an estimated 15,000 to 30,000
patients in the U.S. and Europe.
“I am very excited to announce our alignment with the FDA on our
pivotal study design for RP-A501 for Danon Disease, which reflects
the highly collaborative discussions with the review team and
senior management at FDA’s Center for Biologics Evaluation and
Research and marks the first-ever regulatory pathway to approval
for a genetic treatment for heart disease. We believe this
milestone sets us on the most efficient and rapid path to
delivering this potentially transformative therapy to Danon Disease
patients who would otherwise progress to heart transplantation or
death,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket
Pharma. “I would also like to highlight the work conducted by our
CMC team over the past several years to establish our in-house cGMP
manufacturing capabilities, which has already provided us with
sufficient material for the pivotal study and should support our
eventual commercialization efforts.”
Dr. Shah continued “As a one-time potentially curative infusion,
RP-A501 has the potential to restore normal cardiac function and
provide a lifetime of benefit to patients with Danon Disease who
have no other viable treatment options. With today’s progress in
our Danon Disease program, we believe we are forging a path to
bring curative gene therapies to patients affected by devastating
cardiovascular diseases and broadening the possibilities for
addressing the large array of inherited heart diseases through the
promise of cardiac gene therapy.”
Phase 2 Pivotal Trial of RP-A501 for Danon Disease The
global, single-arm, multi-center Phase 2 pivotal trial will
evaluate the efficacy and safety of RP-A501 in 12 patients with
Danon Disease, including a pediatric safety run-in (n=2), with a
natural history comparator and a dose level of 6.7 x 1013
GC/kg.
- To support accelerated approval, the study will assess the
efficacy of RP-A501 as measured by the biomarker-based co-primary
endpoint consisting of improvements in LAMP2 protein expression (≥
Grade 1, as measured by immunohistochemistry), and reductions in
left ventricular (LV) mass.
- Key secondary endpoint is change in troponin. Additional
secondary endpoints will include natriuretic peptides, Kansas City
Cardiomyopathy Questionnaire (KCCQ), New York Heart Association
(NYHA) class, event free survival to 24 months and treatment
emergent safety events. These endpoints could support full approval
with longer-term follow-up.
- A global natural history study will serve as an external
comparator and run concurrently to the Phase 2 pivotal trial.
- In-house manufacturing has been completed with sufficient
high-quality drug product produced to fully supply the Phase 2
pivotal study. Potency assays have been developed and qualified in
accordance with FDA guidance.
Filing of the Clinical Trial Application (CTA)/Investigational
Medicinal Product Dossier (IMPD) for RP-A501 to enable initiation
of EU study activities is anticipated in the third quarter of this
year. Additionally, Rocket has secured an ICD-10 code from CMS for
LAMP2 deficiency in Danon Disease
About RP-A501 RP-A501 is Rocket’s investigational gene
therapy product for the treatment of Danon Disease and the first
gene therapy for a cardiovascular condition to demonstrate safety
and efficacy in clinical studies. Danon Disease is caused by
mutations in the LAMP2 gene. RP-A501 consists of a recombinant
adeno-associated serotype 9 (AAV9) capsid containing a full-length,
wild-type version of the human LAMP2B transgene (AAV9.LAMP2B)
which, when inserted into heart cells harboring mutations in the
endogenous LAMP2 gene, has the potential to fully restore cardiac
function at its root. RP-A501 represents a single dose treatment
and is administered as an intravenous (IV) infusion. In preclinical
and clinical studies, AAV9.LAMP2B has been shown to target cardiac
cells (cardiomyocytes) and deliver the functional LAMP2B gene to
heart tissue, which ultimately leads to improved cardiac structure
and function in patients.
About Danon Disease Danon Disease is a rare X-linked
inherited disorder caused by mutations in the gene encoding
lysosome-associated membrane protein 2 (LAMP-2), an important
mediator of autophagy. This results in accumulation of
autophagosomes and glycogen, particularly in cardiac muscle and
other tissues, which ultimately leads to heart failure, and for
male patients, frequent death during adolescence or early
adulthood. It is estimated to have a prevalence of 15,000 to 30,000
patients in the U.S. and Europe. The only available treatment
option for Danon Disease is cardiac transplantation, which is
associated with substantial complications and is not considered
curative. There is a high unmet medical need for patients with
Danon Disease.
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 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, 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 received IND clearance for the
AAV-based gene therapy program for PKP2-arrhythmogenic
cardiomyopathy (ACM) and is advancing a preclinical program for
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 Danon Disease (DD), 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
"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, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230912416941/en/
Media Kevin Giordano kgiordano@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
Rocket Pharmaceuticals (NASDAQ:RCKT)
Gráfico Histórico do Ativo
De Abr 2024 até Mai 2024
Rocket Pharmaceuticals (NASDAQ:RCKT)
Gráfico Histórico do Ativo
De Mai 2023 até Mai 2024