Benzinga | Jan 25, 2022 07:14AM EST

FDA Holds Mustang Bio's Gene Therapy IND For Compromised Immune System Disorder

The FDA has issued a hold, pending Chemistry, Manufacturing & Controls (CMC) clearance, on Mustang Bio Inc's (NASDAQ:MBIO) Investigational New Drug (IND) application for MB-207 in patients with X-linked severe combined immunodeficiency (XSCID).

* The application was submitted to initiate a pivotal Phase 2 study to assess MB-207 (lentiviral gene therapy) in patients who have been previously treated with a hematopoietic stem cell transplantation, and for whom re-treatment is indicated.

* The FDA has previously granted MB-207 Orphan Drug and Rare Pediatric Disease Designations.

* Related content: Benzinga's Full FDA Calendar.

* An additional Phase 1/2 trial for XSCID in newly diagnosed infants is ongoing.

* Mustang expects to initiate a pivotal Phase 2 trial of MB-107 in newly diagnosed infants with XSCID who are between two months to two years in Q3 of 2022.

* The primary efficacy endpoint will be event-free survival.

* XSCID, also known as bubble boy disease, is a rare genetic disorder characterized by the absence or lack of function of key immune cells, resulting in a severely compromised immune system.

* Mustang was founded by Fortress Biotech Inc (NASDAQ:FBIO).

* Price Action: MBIO shares are closed at $1.30 on Monday.