News Release Details
Rocket Pharmaceuticals Announces Preclinical Data for AAV-Based RP-A501, the First Investigational Gene Therapy Program for a Monogenic Heart Failure Syndrome
Nov 26, 2018
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- Preclinical Data Package Shows Dose-Dependent Survival, Functional, Structural, and Molecular Benefits with No Safety or Tolerability Issues Observed -
- RP-A501’s Market Opportunity: 15,000-30,000 Estimated Prevalence in the U.S. and E.U. -
- RP-A501 Anticipated to Enter the Clinic in the First Half of 2019; Strong Intellectual Property Protection in Place -
RP-A501 is in development under a collaboration led by Dr.
“Danon disease is a devastating multisystemic disorder that typically leads to death in patients from progressive heart failure in their teens and twenties. Along with severe cardiomyopathy, other Danon disease symptoms can include skeletal muscle weakness, liver disease, and intellectual impairment. Heart transplantation is an important treatment option but is not curative and is associated with approximately 50% ten-year survival post-transplant, considerable morbidity, and a very high cost to the medical system. Due to its nonspecific clinical presentation and the lack of standardized genetic testing, Danon disease is poorly recognized, under diagnosed, and often confused with other multisystemic disorders,” said Dr. Adler. “RP-A501 is the first investigational gene therapy for Danon disease and has the potential to prevent and treat heart failure and other sequelae for these patients. Preclinical studies show RP-A501 improves survival and corrects the disease phenotype with dose-dependent improvements in molecular, structural, and functional endpoints alongside a clean safety and tolerability profile. Rocket has exceptional core strength in clinical development, and I look forward to advancing the first gene therapy program for a monogenic heart failure syndrome to the finish line with them.”
“We are excited to announce this monogenic heart failure program as part
of our growing pipeline. Our goal at Rocket is to develop
paradigm-shifting medicines that meaningfully improve lives of patients
with devastating diseases where no therapies exist,” said
“We have adequate vector supply for the planned clinical trials with
RP-A501 and are on track to begin studies in early 2019. Finally,
through an agreement with
Summary Preclinical RP-A501 Data:
Preclinical efficacy studies were performed in LAMP-2 knockout (KO) mice. Four doses of vector were tested for optimal transduction of the heart, skeletal muscle, and liver. Toxicology studies were conducted in wild-type mice and non-human primates. The results from these studies are summarized as follows:
- Increased survival rates were observed at higher doses of RP-A501 along with dose-dependent improvements and restoration of cardiac function.
- RP-A501 elicited phenotypic reversals at a structural and molecular level in cardiac, liver, and skeletal muscle tissue.
- There were no treatment-related adverse events or deaths associated with RP-A501. All doses were observed to be well-tolerated in Good Laboratory Practice (GLP) biodistribution and toxicology studies in both wildtype mice and additional studies in non-human primates.
Based on the preclinical safety and efficacy data observed in mice and non-human primate studies, Rocket plans to initiate a Phase 1 dose-escalation study of RP-A501 in patients with Danon disease in the first half of 2019.
Full preclinical data have been submitted to a medical conference.
Intellectual Property:
Rocket’s intellectual property protection includes the exclusive
development and commercialization rights for an AAV-based gene therapy
treatment for Danon disease licensed from UCSD, as well as exclusive
worldwide rights to the AAV9 capsid for the treatment of the indication
through a license agreement with
A replay of the Company’s conference call and webcast to discuss these preclinical results will be available on the “Investors” section of the Company’s website at www.rocketpharma.com. The webcast replay will be available for a limited time.
About Danon Disease
Danon disease is a rare neuromuscular and cardiovascular disease
characterized by profound cardiomyopathy, skeletal myopathies, and mild
cognitive impairment. It is estimated to have a prevalence of 15,000 to
30,000 patients in the U.S. and the
About
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, including in collaboration
with academic partners, to treat 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 preclinical
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
View source version on businesswire.com: https://www.businesswire.com/news/home/20181126005168/en/
Source:
Claudine Prowse, Ph.D.
SVP, Corporate Strategy and IR Officer
Rocket
Pharma, Inc.
The Empire State Building, Suite 7530
New York,
NY 10118
www.rocketpharma.com
investors@rocketpharma.com