Phase 1 Trial: PMR-116, Targeting The Undruggable MYC Oncogene
Australian scientists are preparing to launch the world’s first human trial of a groundbreaking anti-cancer drug, PMR-116, developed to target one of the most elusive and dangerous cancer drivers: the MYC oncogene. The trial, set to begin in 2025, could mark a significant breakthrough in the treatment of aggressive, treatment-resistant cancers.
The MYC gene produces the MYC protein, which fuels the growth and spread of tumors while simultaneously rendering many traditional cancer therapies ineffective. Abnormal MYC activity is believed to play a role in approximately 70 percent of all cancers, making it one of the most widespread oncogenic forces in human disease. MYC overexpression is found in about one-third of prostate, pancreatic, liver, gastric, and breast cancers, and nearly two-thirds of ovarian cancers. Tumors driven by MYC are often among the most aggressive and hardest to treat. Despite its central role in cancer, MYC has long been considered “undruggable” due to the disordered structure of the MYC protein, which makes it nearly impossible to target directly with conventional drugs.
PMR-116, developed by scientists at The Australian National University (ANU) and the biotech company Pimera Therapeutics, takes a different approach. Instead of aiming at MYC itself, the drug disrupts a downstream pathway that MYC depends on to fuel tumor growth. Specifically, PMR-116 inhibits an enzyme critical for the synthesis of ribosomal RNA (rRNA), which is essential for protein production.
Early preclinical studies have shown remarkable promise. In a 2023 study conducted at The University of Melbourne, mice with MYC-driven prostate cancer treated with PMR-116 exhibited an 85 percent reduction in cancer lesions over a four-week period. Additionally, the drug was found to reduce the spread of MYC-driven cancer by 50 percent in just 12 hours, a result that raised hopes among researchers for its potential impact in humans.
For decades, MYC has been a symbol of cancer’s resilience, a gene too central and too slippery to pin down. PMR-116 may finally offer a way to disarm it—not by a frontal assault, but by shutting off the machinery it relies on to grow. If successful, this Australian-led effort could usher in a new era in the fight against some of the deadliest forms of cancer.