Drug Research and Development

Drug Research And Development

PC-002

PC-002 is a novel small-molecule drug targeting the MYC gene mutation, which has been approved by the FDA for an MRCT phase 2 clinical study in high-grade B-cell lymphoma (HGBCL) and neuroendocrine prostate cancer (NEPC).

To develop a MYC targeting drug has been a challenge for many years
- MYC acts as a transcription factor and transcription signal amplifier to regulate the expression of hundreds of other genes. Direct inhibition of MYC will affect many physiological processes.
- MYC protein has a flexible structure. No active binding pocket of small molecules has been identified.
- MYC binds to DNA as part of a protein complex. It can also bind to promoters/enhancers directly. It is difficult to develop small molecule drugs that directly target MYC protein-protein and protein-DNA interactions.
Novel targeting mechanism
- The new direct target of PC-002 screened out by i-CR® platform is responsible for the selective degradation of MYC protein, thus turns Myc from “un-targetable” into “targetable”.
- Inhibition of this target protein can efficiently induce apoptosis of cancer cells with deregulated Myc gene.
Specifically effective for cancers with Myc gene alteration
- c-Myc amplification and translocation are correlated with PC-002 sensitivity. PC-002 displays strong inhibitory effects on diffused large B cell lymphomas (DLBCL) with c-Myc translocation.
- N-Myc amplification is correlated with PC-002 sensitivity. PC-002 displays strong inhibitory effects on neuroblastomas with N-MYC amplification.
- PC-002 demonstrates significant effect on various tumor models, in vitro and in vivo.
- Eleven clinical trials with good safety results.
- A phase II clinical trial is imminent. The application for the trial has been submitted to the FDA.