Race Oncology (ASX:RAC) has announced a significant expansion of its clinical development strategy, unveiling two new programs for its lead asset, RC220, in acute myeloid leukaemia (AML) and EGFR-mutated non-small cell lung cancer (NSCLC).
The company described the move as a significant milestone in translating recent scientific advances into clinical opportunities across two major cancer markets.
The new programs complement Race’s existing clinical activities. They are designed to capture the value created by the discovery that (E,E)-bisantrene, which is the active ingredient in RC220, is a DNA and RNA G-quadruplex (G4) binder. This mechanism has opened new potential pathways for treating cancers driven by MYC and other oncogenic signalling.
The company has initiated a planned Phase 1a/b trial in EGFR-mutated NSCLC, one of the largest molecularly defined oncology markets, with global sales of EGFR tyrosine kinase inhibitors (TKIs) exceeding US$10 billion annually. Although third-generation EGFR TKIs, such as osimertinib, are highly effective as first-line treatments, patients almost universally develop resistance, leading to rapid disease progression and limited subsequent treatment options.
Preclinical studies conducted by Race have shown that RC220’s mechanism of action enables it to delay, or even prevent, the emergence of TKI resistance. The drug has demonstrated activity across multiple known resistance pathways, including MYC-driven signalling, Topo2a-associated resistance, and RNA methylation changes linked to treatment failure.
Planning for the NSCLC study is well advanced, the company said, with key opinion leaders, principal investigators, and five major clinical sites in Sydney, Melbourne, and Brisbane already recruited. The company has submitted the trial protocol and all required documentation to the Bellberry Human Research Ethics Committee.
CEO Dr Daniel Tillett said the clinical progress reflects a rapid translation of scientific insight into tangible development opportunities:
“The opportunity to use RC220 to delay, or even prevent, TKI resistance across a range of cancers is a compelling opportunity. The scale of the opportunity is enormous, with more than US$10 billion of EGFRm TKIs sold every year for lung cancer alone."
Race has also announced a pivotal Phase 3 trial in relapsed or refractory AML, representing a potential rapid pathway to regulatory approval for RC220. The program includes a bridging and dose-optimisation stage designed to demonstrate pharmacokinetic and pharmacodynamic equivalence between RC220 and the earlier RC110 formulation, as well as compliance with the US FDA’s Project Optimus requirements for dose selection.
The bridging stage will also generate in vivo biomarker data, including the effects of MYC gene silencing in cancer cells, providing further insight into the drug’s mechanism. A low-cost investigator-sponsored Phase 1b/2 trial will run in parallel to explore optimal combinations of RC220 with established AML treatments.
The company emphasised that RC110 has already demonstrated promising activity in heavily pre-treated AML patients, with overall response rates of 40 per cent both as monotherapy and in combination with nucleoside antimetabolites.
Race confirmed that its existing Phase 1a/b study of RC220 in combination with doxorubicin, focused on both cardioprotection and enhanced anticancer activity, continues unchanged. The company described the program as “a clear priority” due to its potential dual benefit for patients and its value for shareholders.
Race emphasised that while planning for the new trials is well advanced, significant spending will not begin until sufficient funds are secured. The company is relying on shareholder exercise of piggyback options issued in June 2024, which would fully fund the new programs. As of mid-November, 18 per cent of these options had been converted, with a further 12 per cent held by Dr Tillett.
Race remains funded for all existing trials until mid-2027 and continues to assess capital management and partnership opportunities.