Cothera Announces Dosing of First Patient in Phase 2 Clinical Trial to Test Its DUB Inhibitor PC-002 for the Treatment of High-Grade B-cell Lymphoma

San Francisco, December 19, 2022–Cothera Bioscience today announced that it has dosed the first patient with PC-002, a first-in-class deubiquitinase (DUB) inhibitor, in a global multi-center phase 2 clinical trial 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. The company also announced that a paper1 describing PC-002’s mechanism-of-action was accepted for publication in the European Journal of Pharmaceutical Science.

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

Dr. Alex Wu, Co-founder and CEO of Cothera Bioscience, commented: “We are excited to dose the first patient in this important proof-of-concept trial. DUBs are an emerging target family with broad potential in cancer and inflammation treatment. PC-002 is the most advanced clinical stage DUB inhibitor which works by accelerating the degradation of Myc oncoprotein and has great potential to treat cancers depending on high Myc activity. Myc-driven high-grade lymphoma is a highly malignant, life-threatening disease for which there is currently no good treatment. We look forward to validating the safety and efficacy of PC-002 through this trial, providing a new solution for improving the prognosis of patients with this devastating disease, and delivering a breakthrough in the treatment of Myc-driven tumors.”

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:

About Cothera Bioscience, Inc.

Cothera Bioscience is a clinical stage biotech company developing therapeutics for previously undruggable oncology targets with high unmet medical needs. The company’s strength is in its clinically validated translational platform exploiting synthetic lethality and protein degradation pathways for cancer killing. It was formed by the core founding members of Crown Bioscience, a leading contract research organization specializing in translational oncology, together with veteran serial entrepreneurs from Silicon Valley.

Proprietary i-CR® technology platform

Cothera Bioscience developed a patented proprietary i-CR® technology platform, a powerful method for individualized drug screening and new drug development. The platform combines a high content screening system with conditional reprogramming of primary cultured tumor cells, which enables efficient and undifferentiated expansion of primary patient-derived tumor cells in vitro while preserving the tumor’s heterogeneity. Through collaboration with leading oncology medical centers, Cothera Bioscience carried out prospective clinical trials and demonstrated that the i-CR® system can effectively predict the actual clinical response of a drug, with potential to significantly improve the clinical efficacy and success rate in anticancer drug development. (Transl Oncol. 2021 Jan;14(1):100935).

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