Mustang Bio Inc. (NASDAQ: MBIO) has announced that the US FDA has imposed a hold, pending CMC (Chemistry, Manufacturing, and Controls)clearance on its investigational new drug application.
Mustang Bio evaluating safety and efficacy of MB-2017 in XSCID
The IND, which was submitted in December 2021, is for a crucial Phase two multi-site study to assess the tolerability, safety, and efficacy of MB-207 in patient populations with X-linked severe combined immunodeficiency (“XSCID”), also recognized as bubble boy disease, that have received prior hematopoietic stem cell transplantation (“HSCT”) and require re-treatment. In addition, MB-207 has previously received Rare Pediatric Disease and Orphan Drug Designations from the FDA. As a result, should the FDA approve an MB-207 Biologics License Application (“BLA”), MB-207 will qualify for a rare pediatric disease grant.
St. Jude, UCSF Benioff Children’s Hospital in San Francisco, and Seattle Children’s Hospital are all conducting a Phase 1/2 clinical research for XSCID in recently diagnosed infants under the age of two. MB-107 is the name of the drug candidate in the study.
Mustang studying MB-107 in infants with XSCID
Mustang plans to start a multicenter critical Phase II clinical study of MB-107 in recently diagnosed babies with XSCID aged two months to two years old in Q3 2022 under a Mustang-sponsored IND. The experiment is scheduled to enroll 10 individuals, who will be compared to 25 matching historical control subjects who have had HSCT, along with 15 subjects engaged in an ongoing multi-site trial conducted by St. Jude. Event-free survival will be the major efficacy outcome. If FDA authorizes an MB-107 BLA, it will be eligible for a rare pediatric disease grant.
For the treatment of XSCID in recently diagnosed newborns, the FDA previously granted MB-107 Orphan Drug, Rare Pediatric Disease, and Regenerative Medicine Advanced Therapy Designations. In addition, OrphanMB-107 has also been given Orphan Drug, Advanced Therapy Medicinal Product Classification, and PRIME designation by the EMA.
CEO Manuel Litchman commented, “We appreciate the FDA’s support and guidance as we pursue the advancement of MB-107 and MB-207 as potential treatment options for XSCID, a life-threatening, rare genetic disorder with limited treatment options available.”