New medicine for rare diseases! The FDA granted CD123 targeted CAR-T cell therapy MB-102 the status of orphan drugs for BPDCN

Mustang Bio is a clinical biopharmaceutical company focused on developing new immunotherapy and gene therapies based on proprietary chime antigen-engineered T-cell (CART) technology for the treatment of rare diseases. Recently, the company announced that the U.S. Food and Drug Administration (FDA) has awarded MB-102 (CD123 CAR-T) the orphan drug for the treatment of maternal plasma cell-like dendring cell tumors (BPDCN).
is used to prevent, treat and diagnose rare diseases, which are the general term for a very low incidence of diseases, also known as "orphan diseases". In the United States, rare diseases are the type of disease that affects fewer than 200,000 people. Pharmaceutical companies that develop rare disease drugs will receive incentives, including various clinical development incentives, such as tax credits related to clinical trial costs, FDA user fee waivers, FDA assistance in clinical trial design, and a seven-year market exclusive period after the drug is approved for market.
MB-102 is a CAR-T cell therapy targeted at CD123 that identifies and eliminates tumor cells that express CD123 by engineering patient T-cells. CD123 is widely expressed on the surface of myeloid cells in patients with myeloid hyperplus syndrome, as well as in a variety of hematopathic malignancies, including acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia, hair cell leukemia, BPDCN, chronic myeloid leukemia, Hodgkin's lymphoma, etc.
in the first human clinical study, City of Hope (NCT02159495), MB-102 showed complete relief with low-dose treatment in patients with AML and BPDCN, and no dose-limiting toxicity. At present, the increase in the dose of these two therapeutic adaptations is continuing. Mustang Bio has plans to launch a multicenter Phase I/II clinical study of MB-102 in 2019 to treat AML, BPDCN, and high-risk bone marrow growth syndrome.
BPDCN is an invasive, rare malignant tumor of the blood system with poor prognosmation, an area of treatment where medical needs are not met. BPDCN's characteristics may be similar to certain diseases and may be misdiagnosed as certain diseases, including acute myeloid leukemia (AML), non-Hodgkin's lymphoma (NHL), acute lymphoblastic leukemia (ALL), myeloid hyperplus syndrome, chronic granulocytic leukemia (CML), and other malignant tumors with skin manifestations. BPDCN is usually present in the bone marrow and/or skin and may also involve lymph nodes and internal organs. THE diagnosis of BPDCN is based on the immunodiagnosis triplets of CD123, CD4, and CD56. CD123 (IL-3R) is a key marker for identifying BPDCN and a rapidly emerging target in various cancer treatment studies.Elzonris: The first drug to treat BPDCN and the first drug to target CD123
It's worth noting that Elzonris, an immunotoxin drug from Stemline Therapeutics, is a drug that targets THED123 SL-401) is approved by the U.S. FDA for the treatment of BPDCN pediatric and adult patients 2 years and older, including those who have not been treated in the past (primary treatment) and those who have been treated in the past (treated). The approval makes Elzonris the first drug approved to treat BPDCN and the first approved CD123 targeted drug. Stemline has plans to bring Elzonris to market in early 2019.
Elzonris is a CD123-guided cytotoxin specifically designed for CD123 targets, the drug is composed of human IL-3 and cut diphtheria toxin (DT) recombination fusion, IL-3 domain can be cytotoxic DT fragments to express CD123 tumor cells. After internalizing tumor cells, Elzonris is able to irreversibly inhibit protein synthesis and induce target apoptosis.
, Elzonris has been granted breakthrough drug eligibility (BTD) and orphan drug eligibility (ODD) and approved through the FDA's priority vetting process. In the European Union, Elzonris has been granted accelerated approval, which is expected to be approved in the first quarter of 2019. Stemline is also evaluating Elzonris for other conditions, including chronic bone marrow monocytular leukemia (CMML), bone marrow fibrosis (MF) and other conditions. (Bio Valley)