It is in Mustang Bio Inc (NASDAQ:MBIO), where a culture of innovation thrives, thanks to a world-class team in-licenses and the development of next-generation therapies. By partnering with some of the leading academic institutions, the company has been advancing its portfolio to focus on glioblastoma, hematologic cancers, and rare genetic diseases.
Today, the biopharma has accomplished yet another medical breakthrough in cell and gene therapies with an Orphan Drug Designation of its MB-107 from the FDA. The lentiviral gene therapy is a treatment for X-linked severe combined immunodeficiency (“XSCID”) disease. Earlier on, MB-107 had received Regenerative Medicine Advanced Therapy and Rare Pediatric Disease Designations.
The Assessment of MB-107 in a Phase 1/2 Clinical Trial
Before the FDA can grant drugs and biologics Orphan Drug Designation, they must have the intended safety and effectiveness for treating rare diseases. This means having undergone various trials. A few months ago, Mustang filed a new drug application (“IND”) to initiate a multicenter Phase 2 clinical trial of MB-107. The subjects of the trial will enroll 10 subjects between two months to two years of age.
As that is expected to happen, MB-107 is currently undergoing a Phase 1/2 clinical evaluation for XSCID in newly diagnosed infants. This is taking place in three hospital sites with 15 patients and is being led by St. Jude Children’s Research Hospital. Another Phase 1/2 clinical trial for XSCID is also happening at the National Institutes of Health, and there is an anticipation of filing an IND to the FDA to start a multicenter Phase 2 clinical trial.
Mustang has had a Fruitful Quarter on the Regulatory Front
‘’…We are very pleased to achieve another significant milestone and receive Orphan Drug Designation for MB-107 for the treatment of XSCID in newly diagnosed patients…’’ Mustang’s CEO Manuel Litchman reported
The company has been a strong advocate for a much-needed treatment option to help children and their caregivers address XSCID, such a rare but devastating disease. Following the FDA’s approval, MB-107 is now entitled to seven years of market exclusivity and tax credits towards the cost of clinical trials.