Rocket Pharmaceuticals (RCKT) Highlights Promising Preliminary Results from Phase 1 Trial of Commercial-Grade RP-L102 'Process B' for Fanconi Anemia
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) today announces encouraging preliminary results from its Phase 1 trial of commercial-grade RP-L102 “Process B” for Fanconi Anemia (FA) at the 61st American Society of Hematology (ASH) Annual Meeting. The data are highlighted in a poster presentation by Agnieszka Czechowicz M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine at the Stanford University School of Medicine, entitled “Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells.”
“The preliminary data presented at ASH provide evidence regarding the potential of our commercial-grade ‘Process B’ product in treating FA,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “Treatment with ‘Process B’ RP-L102 at four to six months post-infusion showed early signs of engraftment and bone marrow restoration. Further, previously declining blood cell counts appear to have been stabilized or even increased within six months of therapy, providing evidence regarding the potential benefits of treatment with a consistent, commercial-grade product. We are looking forward to presenting additional long-term follow-up data from these patients in the first half of 2020.”
Results presented in the poster highlight preliminary Phase 1 data from two pediatric patients (age 5 and 6 years) who were treated with “Process B” RP-L102 prior to the development of severe bone marrow failure and are in ongoing follow-up. Drug product was successfully manufactured using “Process B” optimization, including transduction enhancers, commercial-grade vector and modified cell processing. “Process B” drug product is manufactured to commercial grade standards, allowing for consistent drug product across patients and a vector copy number (VCN) two to three fold higher than that administered to optimally-treated “Process A” patients. Once transduced, drug product was infused fresh into patients without any prior conditioning regimen. To evaluate transduction efficiency, an analysis of the proportion of the MMC-resistant colony forming cells (CFC’s) was conducted. Both patients exhibited early signs of engraftment based on peripheral blood, VCN and/or MMC-resistance. Preliminary phenotypic correction was also apparent in both patients, as evidenced by stabilization or increases in blood cell lineages. No safety or tolerability issues have been reported.
“These preliminary results, notably achieved without the use of a conditioning regimen, underscore the exciting potential of RP-L102 as a therapy that, if approved, could be implemented early in life as a preventative measure for bone marrow failure,” said Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. “Treating FA patients early, before the need for a bone marrow transplant, would transform the lives of these patients. The present data suggest that RP-L102 has the potential to make this goal a reality.”
The global registrational Phase 2 study of “Process B” RP-L102 for Fanconi Anemia (NCT04069533) is currently underway, with primary endpoint of bone marrow MMC-resistance. The trial is expected to enroll five patients in the U.S. and five patients in Europe.
A copy of the poster can be accessed by visiting: https://www.rocketpharma.com/ash-presentations/
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 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. 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 ‘nature’s 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 cells1.
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