The company announced that updated Phase 1/2 clinical data for MB-107 was presented at the 61st American Society of Hematology Annual Meeting.
Mustang Bio (NASDAQ:MBIO) has announced updated Phase 1/2 clinical date for MB-107 was presented at the National Institutes of Health at the 61st American Society of Hematology (ASH) meeting.
As quoted in the press release:
MB-107 is currently being assessed in two Phase 1/2 clinical trials for XSCID: the first in newly diagnosed infants under the age of two at St. Jude, and the second in patients over the age of two who have received prior hematopoietic stem cell transplantation at the National Institutes of Health. Under a licensing partnership with St. Jude, Mustang intends to develop the lentiviral gene therapy for commercial use as MB-107. The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to MB-107 for the treatment of XSCID in August 2019.
Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “The updated clinical data presented at the 2019 ASH Annual Meeting underscore the curative potential of MB-107 for newly diagnosed infants with XSCID, as well as its meaningful impact on older XSCID patients who received prior hematopoietic stem cell transplantation. St. Jude recently received the 2019 Smithsonian Magazine American Ingenuity Award for development of the lentiviral gene therapy, highlighting its potential to have an impact on this devastating disease. We are excited to be working with St. Jude and NIH to advance MB-107 and look forward to transferring the IND from St. Jude to Mustang in the first quarter of 2020.”
Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study (Abstract Number: 2058)
Poster presentation: Ewelina Mamcarz, M.D., Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children’s Research Hospital, Memphis, TN, USA
Interim data from the multicenter Phase 1/2 clinical trial for infants under the age of two treated with the lentiviral gene therapy preceded by low exposure-targeted busulfan conditioning were published in the New England Journal of Medicine. Updated data presented at the 2019 ASH Annual Meeting include three more patients (n=11), 8 months additional median follow up (23.6 months; range: 1.5 to 33.9 months), more extensive analysis of T and B cell functional recovery, and detailed vector integration site studies.