Rocket Pharmaceuticals Announces FDA Clearance of IND Application for RP-L102 Gene Therapy for Fanconi
Anemia
- Patients to Be Treated with RP-L102 under “Process B” Incorporating Higher Cell Doses, Transduction
Enhancers, and Commercial-grade Vector Manufacturing and Cell Processing –
- U.S. Trial to Commence Early 2019; No Conditioning Required -
- Center for Definitive and Curative Medicine at Stanford University School of Medicine to Lead U.S.
Clinical Studies -
Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, announces the
clearance of the Company’s Investigational New Drug (IND) application for RP-L102, the Company’s lentiviral vector (LVV)-based gene
therapy for the treatment of Fanconi Anemia (FA), by the U.S. Food and Drug Administration (FDA). The clinical trial will evaluate
“Process B” which incorporates higher cell doses, transduction enhancers, and commercial-grade vector manufacturing and cell
processing. This process improves upon the first-generation process developed in partnership with the Centro de Investigaciones
Energéticas, Medioambientales y Tecnológicas (CIEMAT) )/Ciber of Rare Diseases/Fundación Jiménez Díaz in Madrid, Spain.
“Today marks Rocket’s first IND clearance by the FDA and represents an important step forward for the patients and families
suffering from FA. Our team worked expeditiously to file this application ahead of schedule so that we may bring RP-L102 forward as
quickly as possible," said Kinnari Patel, Pharm.D., MBA, Chief Operating Officer and Head of Development of Rocket. “The U.S.
clinical trial will begin in early 2019. The goal of the trial is to evaluate the safety, tolerability, and efficacy of ‘Process B’
RP-L102 in patients with FA. No conditioning is required due to the selective advantage that is present in FA. We look forward to
engaging with regulatory authorities on a final registration path in the second half of 2019 after initial patients have been
treated.”
The planned clinical trial of “Process B” RP-L102 is expected to enroll approximately 12 FA patients at the Center for
Definitive and Curative Medicine at Stanford University School of Medicine, Hospital Niño Jesús/CIEMAT, and other leading centers
in the U.S. and in the EU.
About RP-L102 (LVV-based gene therapy for Fanconi Anemia)
RP-L102 is Rocket’s lentiviral vector (LVV)-based gene therapy in development for patients with FA with Rocket’s collaboration
partners at Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain, CIBER-Rare Diseases and
IIS-Fundación Jiménez Díaz. The International Fanconi Anemia Gene Therapy Working Group helped the development of new generation of
FA gene therapy programs, which began with a HIV-1-derived, self-inactivating lentiviral vector. RP-L102’s lentiviral vector
carries the FANC-A gene as part of the PGK-FANCA-WPRE expression cassette which includes a phosphoglycerate kinase (PKG) promoter
and an optimized woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The ex vivo administration process begins
with the removal and isolation of hematopoietic stem cells using a CD34+ selection process. Autologous genetically modified CD34+
enriched hematopoietic cells (fresh or cryopreserved) are infused back into patients to restore function. RP-L102 is currently
being studied in a Phase 1/2 clinical trial in the European Union with an Investigational Medicinal Product Dossier (IMPD) in place
with the Spanish Agency for Medicines and Health Products. The U.S. Food and Administration accepted the Company’s Investigational
New Drug (IND) application for RP-L102 utilizing “Process B” which incorporates higher cell doses, transduction enhancers, and
commercial-grade vector. RP-L102 has been granted Orphan Drug designation for the treatment of Fanconi Anemia type A in the United
States and in Europe.
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, which is highly toxic for the patient HSCT is frequently complicated by graft versus host disease and
also increases the risk of solid tumors, mainly squamous cell carcinomas. Approximately 60-70% of patients with FA have a FANC-A
gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and
increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as
mitomycin-C (MMC) or diepoxybutane (DEB) is the ‘gold standard’ test for FA diagnosis. The DEB assay can further differentiate FA
patients from somatic mosaic patients. Somatic mosaicism occurs when there is a spontaneous reversion mutation that can lead to a
mixed chimerism of corrected and uncorrected bone marrow cells leading to stabilization or correction of an FA patient’s blood
counts in the absence of any administered therapy. Somatic mosaicism provides strong rationale for the development of FA gene
therapy and demonstrates the selective advantage of gene-corrected hematopoietic cells in FA1.
1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis
of the FA/BRCA pathway. Blood 105: 1329-1336
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) is an emerging, clinical-stage biotechnology company focused on
developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket’s multi-platform development
approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms.
Rocket's lead clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic
disease that leads to bone marrow failure and potentially cancer. Preclinical studies of additional bone marrow-derived disorders
are ongoing and target Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant
Osteopetrosis (IMO). Rocket is also developing an AAV-based gene therapy program for an undisclosed rare pediatric disease. 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, effectiveness and timing of product candidates that Rocket may develop, including in
collaboration with academic partners, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase
Deficiency (PKD) and Infantile Malignant Osteopetrosis (IMO), 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, Rocket's ability to commence a registrational study in FA within the projected time periods, 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 December 31, 2017. 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.
Claudine Prowse, Ph.D.
SVP, Corporate Strategy and IRO
Rocket Pharma, Inc.
The Empire State Building, Suite 7530
New York, NY 10118
cp@rocketpharma.com
www.rocketpharma.com
investors@rocketpharma.com
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