News Release Details
Rocket Pharmaceuticals Presents Updated Long-Term Phase 1/2 Clinical Data of RP-L102 in Fanconi Anemia Patients at the 2018 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT)
Oct 18, 2018
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- RP-L102 Continues to Demonstrate Increasing and Durable Engraftment (Peripheral Vector Copy Number) In Fanconi Anemia Patients for Up to Thirty Months with First-Generation Process –
Title: Gene Therapy Trial in Non-Conditioned Fanconi Anemia
Patients
Session: Blood Disorders I
Presenter:
Four patients have met the requisite 12-month follow-up time for long-term stem-cell engraftment analysis. As evidenced by progressively increasing vector copy number (VCN) in peripheral blood mononuclear cells, RP-L102 continues to demonstrate durable engraftment in all four patients, ranging from 18 to 30 months since gene therapy administration. Patients also demonstrated improved resistance of bone marrow CD34+ cells in the presence of mitomycin-C (MMC) and durable increases in peripheral T-lymphocyte chromosomal stability in the presence of diepoxybutane (DEB). MMC- and DEB-resistance are two key diagnostic measures of functional and phenotypic correction in FA. Trends for continued stabilization of previously-declining blood counts and the progressive increase of corrected versus non-corrected peripheral blood leukocytes continued for all patients. These patients were treated with “Process A”—the first one developed for RP-L102.
Of note, the first patient in this cohort (Patient 2002)—whose stem cells were collected at an early age and is representative of the target demographic—experienced improvements in peripheral VCN from 0 at month 0, to 0.43 at month 24, and to 0.55 at month 30. Patient 2002 also demonstrated improvements in bone marrow MMC-resistance from 0% to more than 70% at month 24, approaching the normal range.
“These data are very encouraging,” said
In the first half of 2019, Rocket plans to treat patients in the U.S.—under a Rocket-sponsored Investigational New Drug (IND) application—and in the EU with “Process B” RP-L102. This new optimized process incorporates higher cell doses, transduction enhancers, and commercial-grade vector.
Data from this presentation will be available in the Company’s corporate presentation at: www.rocketpharma.com/pipeline/.
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
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 many solid organ malignancies. 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
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
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"expect", "anticipate", "intend", "plan", "will give", "estimate",
"seek", "will", "may", "suggest" or similar terms, variations of such
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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
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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, 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