Preliminary Phase 2 Results Demonstrate Engraftment with Minimal Neutropenia with MGTA-456, a CD34+ Expanded Cord Blood (CB) Product in Patients Transplanted for Inherited Metabolic Disorders (IMD)

Conclusions: Preliminary results of transplantation in IMD patients with MGTA-456, containing highly expanded CD34+ cell doses, demonstrated early and robust engraftment in all patients with marked reduction in days of neutropenia (to 0-4 days or mean 1.25 days) in comparison to a historical cohort. MGTA-456 was well tolerated with one infusion-related event of grade 3 nausea. These data, in combination with the previous 27 hematologic malignancy patients treated, suggest that MGTA-456 substantially enhances the engraftment potential of CB. Based on these promising data, MGTA-456 has potential to improve transplant-related outcomes in patients undergoing HSCT and increasing the availability of well-matched CB units that may have been previously excluded due to inadequate CD34+ dose.DisclosuresOrchard: Magenta Therapeutics: Research Funding. Raffel: Magenta Therapeutics: Employment, Equity Ownership. Condon: Magenta Therapeutics: Employment, Equity Ownership. Monaghan: Magenta Therapeutics: Employment, Equity Ownership. Vernet: Magenta Therapeutics: Employment. Sheirr: Magenta Therapeutics: Employment, Equity Ownership. Braun: Magenta Therapeutics: Research Funding. Shanley: Magenta Therapeutics: Research Funding. Lund: Magenta Therapeutics: Research Funding. Boitano: Magenta Therapeutics: Employment, Equity Ownership, Patents & Royalties. Cooke: Magenta Therapeutics: Employment, Equity Ownership, Patents & Royalties. Davis: Magenta Therapeutics: Employment, Equity Own...

Source: Blood - November 21, 2018 Category: Hematology Authors: Orchard, P., Raffel, G. D., Condon, C. H., Monaghan, C. A., Vernet, D., Sheirr, S. T., Braun, J., Shanley, R., Lund, T. C., Boitano, A. E., Cooke, M. P., Davis, J. C., Wagner, J. E. Tags: 732. Clinical Allogeneic Transplantation: Results: Poster II Source Type: research