publication date: Sep. 13, 2019

Drugs & Targets

Adaptimmune receives Orphan Drug Designation for SPEAR T-cells targeting MAGE4-A in soft tissue sarcoma 

Adaptimmune Therapeutics plc said FDA has granted Orphan Drug Designation to SPEAR T-cells targeting MAGE-A4 (Adaptimmune’s ADP-A2M4 program) for the treatment of soft tissue sarcomas. 

Adaptimmune’s ADP-A2M4 (MAGE-A4) SPEAR T-cell therapy is directed to a member of the MAGE family of cancer testis antigens expressed in a number of solid tumor cell types. 

Adaptimmune is evaluating its affinity-enhanced SPEAR T-cells in synovial sarcoma and MRCLS in three trials: (1) the recently initiated phase II trial SPEARHEAD-1 in sarcoma; (2) a radiation sub-study to enhance antitumor activity; and, (3) the ongoing pilot trial. Both the radiation sub-study and pilot trial include sarcoma as well as multiple other solid tumor indications. 

Adaptimmune is also evaluating a next-generation SPEAR T-cell (ADP-A2M4CD8) in sarcoma as well as other solid tumor indications in the SURPASS trial.


BioXcel receives Orphan Drug Designation for BXCL701 in AML

BioXcel Therapeutics Inc. said FDA has granted Orphan Drug Designation for BXCL701, an investigational orally-available systemic innate immunity activator with dual mechanisms of action, for the treatment of acute myeloid leukemia.

BXCL701 is designed to activate innate immune cells, specifically macrophages, by inhibiting the dipeptidyl dipeptidases DPP8 and DPP9, a novel mechanism, and exerts immune stimulatory activity through a pro-inflammatory form of programmed cell death known as pyroptosis. The company is leveraging the pro-inflammatory mechanism of action of BXCL701 for treatment-Emergent Neuroendocrine Prostate cancer and Pancreatic cancer. 

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