Mustang Bio’s MB-102 Granted Orphan Drug Designation for AML

Jul 25, 2019 | Acute Lymphoblastic Leukemia, AML, Orphan Designation, Rare Diseases

Mustang Bio’s MB-102 Granted Orphan Drug Designation for AML

MB-102, a CAR T-cell therapy under development by Mustang Bio, Inc. has received orphan drug designation for Acute Myeloid Leukemia (AML). 

What is MB-102?

CD123 is over expressed in 75-89% of AML patients and over 90% in Blastic plasmacytoid dendritic cell neoplasm (BPDCN) patients. There were an estimated 19,520 new U.S. cases of AML in 2018 with an estimated 25% 5-year survival rate. BPDCN is a rare and aggressive blood cancer with 500 to 1,000 patients per year in the U.S. and a median overall survival rate of 9-12 months. MB-102 has shown promising response rates in early, small populations of these patients in a physician IND study out of City of Hope.

MB102 is a CAR T-cell therapy that is produced by engineering patient T-cells to recognize and eliminate CD123-expressing tumors. In the first-in-human study at City of Hope, MB-102 demonstrated complete responses at low doses in AML and BPDCN without dose-limiting toxicitie.

What is Orphan Drug Designation?

The FDA designates orphan status to drugs and biologics defined as “those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing the drug.”

Principal Investigator Comments

Elizabeth Lihua Budde, MD, PhD, City of Hope noted “There is an increased expression of CD123 on AML blasts, leukemic stem cells and BPDCN cells compared to normal hematopoietic stem cells, and it is therefore a promising target for cellular immunotherapy.  We remain encouraged by interim data showing MB-102’s potential to treat BPDCN and AML and continue to evaluate MB-102’s clinical benefits in our ongoing phase I clinical trial.”