Mustang Bio and St. Jude Children’s Research Hospital Receives RMAT Designation from FDA

Mustang Bio (NASDAQ:MBIO) and St. Jude Children’s Research hospital have announced that MB-107, a gene therapy to treat X-linked severe combined immunodeficiency, called bubble boy disease, has been awarded Regenerative Medicine Advanced Therapy (RMAT) designation by the US Food and Drug Administration (FDA).

As quoted in the press release:

Mustang, a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, and St. Jude, the nation’s leading hospital dedicated to understanding, treating and curing childhood cancer and other life-threatening diseases, established a partnership to continue development of the lentiviral gene therapy in August 2018.

Under the terms of the RMAT designation, the FDA will help facilitate the program’s expedited development and review and provide guidance on generating the evidence needed to support the approval of MB-107 for XSCID. RMAT designation was granted to MB-107 based on positive Phase 1/2 clinical data for infants with XSCID under the age of 2 (ClinicalTrials.gov Identifier: NCT01512888), which were published in the New England Journal of Medicine in April 2019.

“We are very pleased that the FDA has granted RMAT designation to MB-107,” said Manuel Litchman, M.D., president and chief executive officer of Mustang. “This exciting milestone further validates the compelling data generated to date and affirms the potential of MB-107 to address a typically fatal condition with few effective treatment options. We look forward to working with the FDA and our partners at St. Jude to potentially expedite the development and approval of this critically needed treatment option. In particular, we are on track to accept the Investigational New Drug (IND) transfer from St. Jude to Mustang in the fourth quarter of this year.”

Drugs eligible for RMAT designation are those intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and that present preliminary clinical evidence indicating the drug has the potential to address unmet medical needs for such disease or condition. The FDA has determined that MB-107 for the treatment of XSCID meets the criteria for RMAT designation.

Click here to read the full press release.