Rocket Pharmaceuticals announces clearance to commence Phase 2 FANCOLEN-II trial
Rocket Pharmaceuticals announced clearance from the Spanish Agency for Medicines and Health Products to commence enrollment in the FANCOLEN-II Phase 2 registration-enabling study of RP-L102 for Fanconi Anemia, or FA, in Spain. The European Medicines Agency, or EMA, agreed to several key elements of the open-label Phase 2 trial, including endpoints that are designed to demonstrate reversion of the sensitivity of hematopoietic cells to DNA-damaging agents. The primary endpoint of the trial is the emergence of mitomycin-C, or MMC, resistance in bone marrow colony forming cells. A surrogate endpoint is a diepoxybutane, or DEB, chromosomal stability of peripheral blood T-lymphocytes. Sensitivity to DNA-damaging agents is a phenotypic hallmark of FA. Additional outcomes include stability or increase in blood counts with no significant worsening in anemia, neutropenia or thrombocytopenia and peripheral blood genetic correction, as demonstrated by vector copy number, or VCN, including progressive increases in peripheral blood VCN over the months subsequent to infusion. The open-label Phase 2 clinical trial will enroll up to five pediatric patients in Europe. The study will enroll younger patients who have not developed severe bone marrow failure. The patients will receive a single intravenous infusion of RP-L102 that utilizes "Process B" which incorporates enhanced stem cell enrichment, transduction enhancers and commercial-grade vector and manufacturing without the use of any conditioning treatment. The study is designed to assess the benefit/risk profile of RP-L102.