The FDA granted Rare Pediatric Disease, Orphan Drug and Regenerative Medicine Advanced Therapy Designations to MB-107 for the treatment of XSCID in newly diagnosed infants
Mustang Bio and Minaris Regenerative Medicine have announced an agreement to enable technology transfer and GMP clinical manufacturing of Mustang’s MB-107 lentiviral gene therapy program for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease, in Europe.
Minaris will perform technology transfer of the manufacturing and analytical processes, as well as their adoption to the European regulatory environment, for the GMP-compliant manufacturing of the drug product at its site in Ottobrunn, Germany, with the goal of supplying clinical trials in Europe.
MB-107 is currently being assessed in a Phase I/II clinical trial for XSCID in newly diagnosed infants under the age of two at St. Jude Children’s Research Hospital, UCSF Benioff Children’s Hospital in San Francisco and Seattle Children’s Hospital. Mustang submitted an investigational new drug application (IND) to the FDA to initiate a pivotal multi-centre Phase 2 clinical trial of MB-107 in this same patient population.
The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multi-centre trial led by St. Jude, will be compared with 25 matched historical control patients who have undergone hematopoietic stem cell transplantation. The primary efficacy endpoint will be event-free survival. The initiation of this trial is expected soon, and Mustang is targeting topline data from this trial in the second half of 2022.
“We look forward to a productive and successful partnership with Mustang where Minaris will be able to support them with our extensive experience in the clinical and commercial manufacturing of autologous gene therapies,” said Dusan Kosijer, MD of Minaris. “We are eager to work together with Mustang in the fight against this devastating disease.”
“This agreement with Minaris is an important step in supporting expansion of our MB-107 pivotal clinical trial into Europe,” said Manuel Litchman, President and CEO of Mustang. “We look forward to working with Minaris to grow our geographic footprint and bring this potential life-saving therapy to XSCID patients in need internationally.”