Sepantronium Bromide (PC-002)

Cothera’s lead program PC-002 (also known as YM155, sepantronium bromide) is a first-in-class small molecule drug inhibitor of DUB, an enzyme family which removes ubiquitin from cellular proteins otherwise destined for degradation. With more than 50% of human cancers showing increased expression, Myc is regarded as one of the most important yet “undruggable” cancer targets. By inhibiting DUB that stabilizes the Myc protein in cancer cells, PC-002 selectively induces Myc protein degradation and cell apoptosis in Myc-dependent tumors. PC-002 may potentially target multiple cancer indications involving Myc dysregulation.

A paper1 describing PC-002’s mechanism-of-action has been accepted for publication on European Journal of Pharmaceutical Science.

1. Li et al. YM155 Inhibits Neuroblastoma Growth through Degradation of MYCN: A New Role as a USP7 Inhibitor, European Journal of Pharmaceutical Sciences (2022) doi: 

Cothera is currently conducting a global multi-center phase 2 clinical trial with PC-002 for the treatment of high-grade B-cell lymphoma with Myc rearrangement (NCT05263583). This is a single-arm, single-agent proof-of-concept trial designed to evaluate PC-002 in patients who are either resistant or refractory to prior treatments. Cothera is also evaluating PC-002 program for additional indications such as neuroendocrine prostate cancer in patients with n-MYC abnormality or pediatric sarcoma.

Cothera also has additional clinical stage programs for previous undruggable oncology targets with high unmet medical needs.