News Release Details
Rocket Pharmaceuticals Announces IMPD Clearance of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency
Sep 23, 2019
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–Rocket’s Largest Lentiviral Pipeline Opportunity with an Estimated Prevalence of 3,000 to 8,000 Patients in the U.S. and EU–
–Phase 1 Clinical Trial to Commence in the Fourth Quarter–
“We are excited to advance the first gene therapy for PKD to the clinic following the clearance of the IMPD from the AEMPS for RP-L301,” said
“In preclinical models, RP-L301 reverses the hemolytic phenotype, including normalization of splenomegaly and reduction of anemia and reticulocytosis, when at least 20-30% of bone marrow progenitor cells are genetically corrected,” said Dr. José-
The planned open-label, single-arm, Phase 1 clinical trial of RP-L301 will enroll a total of six adult and pediatric transfusion-dependent PKD patients in
The Patient Focused Drug Development (PFDD) Meeting on PKD last week provided patients, families and caregivers the opportunity to share their personal experiences living with the disease and their familiarities with currently available treatments. This event was organized by the
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
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, 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 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
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Source:
Claudine Prowse, Ph.D.
SVP, Strategy & Corporate Development
Rocket Pharma, Inc.
The Empire State Building, Suite 7530
New York, NY 10118
www.rocketpharma.com
investors@rocketpharma.com