Benzinga | Aug 2, 2021 08:18AM EDT

Mustang Bio Reports Received European Medicines Agency PRIME Designation For MB-107 To Treat X-Linked Severe Combined Immunodeficiency In Newly-Diagnosed Infants

Mustang Bio, Inc. ("Mustang") (NASDAQ:MBIO), 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, today announced that the European Medicines Agency ("EMA") has granted Priority Medicines ("PRIME") designation to MB-107, its lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("XSCID") in newly diagnosed infants, also known as bubble boy disease.

In addition to PRIME designation, the EMA granted Advanced Therapy Medicinal Product ("ATMP") classification to MB-107 in April 2020 and Orphan Drug designation in November 2020. MB-107 has also received Orphan Drug, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy ("RMAT") designations from the U.S. Food and Drug Administration ("FDA").

"We are very pleased that the EMA continues to acknowledge the potential for MB-107 to be a new treatment option for patients with XSCID, a devastating rare disease," said Manuel Litchman, M.D., President and Chief Executive Officer of Mustang. "Receiving PRIME designation is an exciting milestone for Mustang as we prepare to initiate a pivotal, multicenter Phase 2 clinical trial of MB-107 in newly diagnosed patients with XSCID at the end of this quarter. We look forward to having a kick-off meeting with the Committee on Advanced Therapies and its multidisciplinary group of experts to provide additional guidance on our overall MB-107 development plan and regulatory strategy to potentially further expedite clinical development and approval in Europe."

The PRIME program aims to optimize development plans and speed up evaluation of promising medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. To be eligible and accepted for PRIME deisgnation, a treatment has to demonstrate the potential to benefit patients with unmet medical needs based on early clinical data. Through the PRIME program, the EMA offers enhanced interactions and early dialogue, including the appointment of a Committee on Advanced Therapies ("CAT") rapporteur, to help build knowledge ahead of a Marketing Authorization Application ("MAA"). It also provides a pathway for accelerated assessment of an MAA under the centralized procedure so that these medicines may reach patients as early as possible. For more information on the PRIME program visit the EMA website.

The same lentiviral vector used in MB-107 is currently being assessed in an ongoing Phase 1/2 clinical trial for XSCID in newly diagnosed infants under 2 years of age at St. Jude Children's Research Hospital ("St. Jude"), UCSF Benioff Children's Hospital and Seattle Children's Hospital. Additionally, it is being assessed in a Phase 1/2 clinical trial at the National Institute of Allergy and Infectious Diseases ("NIAID"), part of the National Institutes of Health, for XSCID patients who have been previously treated with hematopoietic stem cell transplantation ("HSCT") and for whom re-treatment is indicated.