— RP-A501 for
Danon Disease well-tolerated in two pediatric patients with no
complement-mediated toxicities reported; efficacy update remains on
track for Q3 —
— Sustained
genetic correction observed in six of nine evaluable Fanconi Anemia
patients at least 12 months after RP-L102 treatment; five reached
target MMC resistance at two time points with concomitant
hematologic stabilization —
— Robust and
sustained efficacy in both Pyruvate Kinase Deficiency patients at
18 months post-RP-L301 infusion demonstrated by normalized
hemoglobin and improved hemolysis parameters —
— Top-line
readout from Phase 2 pivotal trial of RP-L201 for Leukocyte
Adhesion Deficiency-I expected on Thursday, May 19 —
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 clinical
updates from its Phase 2 pivotal trial for Fanconi Anemia (FA) and
Phase 1 trials for Danon Disease and Pyruvate Kinase Deficiency
(PKD) at the 25th Annual Meeting of the American Society of Gene
and Cell Therapy (ASGCT).
“We are highly encouraged by the initial safety data from the
two pediatric patients in the Phase 1 Danon Disease trial that
suggest RP-A501 was well-tolerated,” said Gaurav Shah, M.D., Chief
Executive Officer of Rocket Pharma. “These two patients had
markedly reduced complement activation and no complement-related
adverse events. They received preventative therapy with a modified
immunosuppressive regimen. We believe today’s results represent an
important step forward in optimizing the safety of AAV9-based gene
therapy for Danon Disease and the Danon patient community. Updated
data are anticipated in the third quarter, and we believe that the
totality of data from seven patients treated in Phase 1 will
support a rapid advance toward a Phase 2 pivotal study.”
“In our Phase 2 pivotal study in Fanconi Anemia, five of nine
patients sustained increasing bone marrow cell resistance to
mitomycin-C (MMC) confirmed over two consecutive timepoints,” said
Dr. Shah. “In these patients, MMC-resistance increased to 51%-94%
at 18 to 21 months, up from 21%-42% at 12 to 18 months. The
increase in MMC resistance was accompanied by concomitant genetic
markings and hematologic stabilization. Addressing the hematologic
aspects of Fanconi Anemia is essential because this devastating
disorder leads to bone marrow failure in the first decade of life.
With these results, we have reached our primary endpoint as defined
in the trial protocol and are initiating dialogue with health
authorities on next steps forward.”
Dr. Shah continued, “Today’s positive updates from a subset of
our world-class gene therapy pipeline highlight the momentum of our
targeted and multi-platform approach toward developing potential
cures for these rare, devastating bone marrow-derived and
cardiovascular diseases for which viable treatment options are
extremely limited.”
Extended Results From First-In-Human Clinical Trial of
RP-A501 (AAV9:LAMP2B) Gene Therapy Treatment for Danon
Disease
The data described in this oral presentation are from the
ongoing first-in-human Phase 1 clinical trial evaluating a single
intravenous infusion of RP-A501, the Company’s investigational gene
therapy for the treatment of Danon Disease. The presentation
includes previously disclosed safety and efficacy data from
patients in the low-dose (6.7 x 1013 GC/kg; n=3) and high-dose (1.1
x 1014 GC/kg; n=2) young adult and adolescent cohort and new
initial safety data from the low-dose (6.7 x 1013GC/kg; n=2)
pediatric cohort.
- In the pediatric cohort (data cut-off April 30, 2022), RP-A501
was well-tolerated in both patients. The patients were observed to
have normal-range platelets, diminished complement activation and
no complement-related adverse events. The two patients received
preventative treatment with a modified immunosuppressive regimen.
No significant immediate or delayed toxicities have been observed
to date.
- As previously disclosed:
- In the young adult and adolescent low-dose cohort, RP-A501 was
generally well-tolerated.
- All young adult and adolescent patients with observed
immunosuppressive regimen compliance and appropriate long-term
follow-up demonstrated disease modification across molecular,
echocardiographic and patient functional parameters.
- These patients demonstrated evidence of cardiac LAMP2B
expression versus normal control by immunohistochemistry.
- Echocardiograms showed decreased cardiac wall thickness with
improved or stabilized ejection fraction.
- Patients demonstrated sustained improvement or stabilization in
Brain Natriuretic Peptide (BNP) and New York Heart Association
(NYHA) class, 6-minute walk test and reported increases in physical
activity.
- Adverse events were manageable with transient
immunosuppression.
- All adverse events in pediatric and adult cohorts were
reversible with no lasting renal, hepatic, or other sequelae.
Ex-Vivo Lentiviral-Mediated Gene Therapy for Patients with
Fanconi Anemia [Group A]: Updated Results From Global RP-L102
Clinical Trials
The oral presentation includes data (cut-off April 4, 2022) from
the initial nine of 12 patients who received RP-L102, Rocket’s
ex-vivo lentiviral gene therapy candidate for FA.
- Five of nine evaluable patients had increased resistance to MMC
in bone marrow-derived colony forming cells, ranging from 21% to
42% at 12 to 18 months, compared to 51% to 94% at 18 to 21 months.
The primary endpoint has been achieved, based on a trial protocol
in which statistical and clinical significance requires a minimum
of five patients to attain increased MMC resistance at least 10%
above baseline at two or more timepoints and concomitant evidence
of genetic correction and clinical stabilization.
- A sixth patient has displayed evidence of progressively
increasing genetic correction as evidenced by peripheral VCN. Two
additional patients are currently only 12 months post-treatment,
and one patient had progressive bone marrow failure and underwent
successful allogeneic transplant as previously disclosed.
- Three of twelve total treated patients have not yet reached the
12-month time point.
- The safety profile of RP-L102 appears favorable with no signs
of dysplasia, clonal dominance or oncogenic integrations; as
previously reported, one patient experienced a Grade 2 transient
infusion-related reaction, which resolved.
Changing the Treatment Paradigm for Pyruvate Kinase
Deficiency with Lentiviral-Mediated Gene Therapy: Interim Results
From an Ongoing Global Phase 1 Study
The poster presentation includes data (cut-off April 13, 2022)
from two adult patients with significant anemia who were treated
with RP-L301, Rocket’s ex-vivo lentiviral gene therapy candidate
for PKD.
- At 18 months post-infusion, both patients have sustained
transgene expression, normalized hemoglobin, improved hemolysis, no
red blood cell transfusion requirements post-engraftment and
improved quality of life both reported anecdotally and as
documented via formal quality of life assessments.
- The safety profile of RP-L301 appears favorable, with no
IP-related serious adverse events 18 months post-infusion.
Transient transaminase elevation was seen in both patients
post-therapy/conditioning, with no clinical stigmata of liver
injury and subsequent resolution without clinical sequelae.
- The pediatric cohort is currently enrolling.
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 are no specific therapies available for
the treatment of Danon Disease.
About Fanconi Anemia
Fanconi Anemia (FA) is a rare pediatric disease characterized by
bone marrow failure, malformations and cancer predisposition. The
primary cause of death among patients with FA is bone marrow
failure, which typically occurs during the first decade of life.
Allogeneic hematopoietic stem cell transplantation (HSCT), when
available, corrects the hematologic component of FA, but requires
myeloablative conditioning. Graft-versus-host disease, a known
complication of allogeneic HSCT, is associated with an increased
risk of solid tumors, mainly squamous cell carcinomas of the head
and neck region. Approximately 60-70% of patients with FA have a
Fanconi Anemia complementation group A (FANCA) gene mutation, which
encodes for a protein essential for DNA repair. Mutations in the
FANCA gene leads to chromosomal breakage and increased sensitivity
to oxidative and environmental stress. Increased sensitivity to
DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane
(DEB) is a ‘gold standard’ test for FA diagnosis. Somatic mosaicism
occurs when there is a spontaneous correction of the mutated gene
that can lead to stabilization or correction of a FA patient’s
blood counts in the absence of any administered therapy. Somatic
mosaicism, often referred to as ‘natural gene therapy’ provides a
strong rationale for the development of FA gene therapy because of
the selective growth advantage of gene-corrected hematopoietic stem
cells over FA cells.
About Pyruvate Kinase Deficiency
Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood
cell disorder resulting from a mutation in the PKLR gene encoding
for the pyruvate kinase enzyme, a key component of the red blood
cell glycolytic pathway. Mutations in the PKLR gene result in
increased red cell destruction and the disorder ranges from mild to
life-threatening anemia. PKD has an estimated prevalence of 3,000
to 8,000 patients in the United States and the European Union.
Children are the most commonly and severely affected subgroup of
patients. Currently available treatments include splenectomy and
red blood cell transfusions, which are associated with immune
defects and chronic iron overload.
RP-L301 was in-licensed from the Centro de Investigaciones
Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de
Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and
Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz
(IIS-FJD).
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 Danon Disease, FA and PKD programs
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/20220516005358/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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