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Mustang Bio down 18% on less effect of gene therapy on older XSCID patients

Dec. 09, 2019 4:08 PM ETMustang Bio, Inc. (MBIO) StockMBIOBy: Douglas W. House, SA News Editor
  • Thinly traded Mustang Bio (MBIO -17.7%) is down on almost 4x higher volume, albeit on turnover of less than 1M shares, in apparent reaction to updated data from ongoing Phase 1/2 clinical trials evaluating lentiviral gene therapy MB-107 in patients with X-linked severe combined immunodeficiency (XSCID), an inherited disorder also known as "Bubble Boy Disease" since it occurs almost exclusively in males who lack the necessary immune cells to fight infections. The results were presented as ASH in Orlando.
  • In the study being conducted at St. Jude Children's Hospital in Memphis, infants under the age of two showed "robust" stem cell recovery with 3-4 weeks post infusion without blood support. Nine participants who had been followed for more than three months achieved normal-for-age T-cell and NK-cell numbers within three-to-four months post treatment. Five were off IV immunoglobulin therapy and three of the five responded to vaccines.
  • Investors appear to be reacting to data from the second trial, being conducted at NIH in Bethesda, MD, in five older children and young adults with XSCID who received MB-107 as salvage therapy after haploidentical hematopoietic stem cell transplantation.
  • Patients required large quantities of vector due to the relative inefficient transduction of hematopoietic stem/progenitor cells which resulted in a relatively low vector copy number (VCN) in myeloid cells in some patients. This delayed immune cell recovery and failed to prevent persistent clinical disease, especially in the last patient treated. NIH refined the transduction procedure and incorporated enhancers which produced a more significant benefit by month 3.
  • Development is ongoing.
  • #ASH19

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