Unlocking the full potential of LSD1
EpiAxis is developing a new class of peptide inhibitors for patients with aggressive solid tumours.
Our mission
Rewriting the story for patients with solid tumours
Our mission is to bring impactful new medicines to patients whose cancer has stopped responding, and to extend what is possible for them.
EpiAxis Therapeutics is an Australian biotechnology company developing non-natural peptide inhibitors of LSD1. Our work is focused on advanced prostate cancer and small cell lung cancer.
What's known about LSD1
A promising target held back by one problem
LSD1 in solid tumours LSD1 is an epigenetic target that regulates the expression of gene programmes to control the identity and behaviour of cells. It is implicated across a range of blood and solid cancers. In advanced prostate and small cell lung cancer it helps drive the shift to more aggressive, less treatable forms of disease.
Drugs blocking LSD1 could offer a new option for patients with treatment-resistant disease.
LSD1 in the blood LSD1 is also required to make platelets, the cells in your blood that clot to stop bleeding. Megakaryocytes, the cells in bone marrow that produce platelets need LSD1 to mature. Blocking LSD1 and that maturation stalls, meaning fewer platelets are made. Eventually these leads to insufficient platelets in the blood, a potentially condition known as thrombocytopenia.
The challenge - Thrombocytopenia
Thrombocytopenia has set the dose for every LSD1 inhibitor to report data from cancer trials. Platelet counts have fallen to unsafe levels short of the dose needed for maximum antitumour effect, and that has happened with irreversible and reversible inhibitors alike. This toxicity also limits what these drugs can be combined with, since many of the treatments these patients receive carry their own blood toxicity.
An inhibitor without this toxicity could be dosed for full effect, and reach more patients.
Our approach
Short, non-natural peptides
Our leads fall into three chemically distinct series, two linear and one cyclic. They are selective inhibitors of LSD1, active at mid-picomolar to single-digit nanomolar concentrations and are stable in mouse plasma.
Our results
Tumour efficacy without platelet toxicity
We ran a 28-day study in a PC3 prostate xenograft, a model of AR-negative prostate cancer with few treatment options. A lead from each series slowed tumour growth as well as two clinical-stage LSD1 comparators, one reversible (Phase II) and one irreversible (Phase III). Platelet counts for our lead peptides showed no decrease from vehicle. The reversible comparator lowered platelets by 54%, the irreversible by 75%.
Roadmap
From lead peptide to first patients
- Now
Lead optimisation
PK/PD, MoA and preliminary tox studies to select the development candidate.
- Q1 2027
Development candidate nominated
- Q4 2027
IND-enabling studies begin
- Q3 GLP toxicology complete
First in human
Phase 1 dose escalation in patients.
- Q1 2029
Phase 1, first-in-human
Team
Re-energised under new leadership to deliver on LSD1's therapeutic promise
EpiAxis is run by a small executive team, with a board and advisory group drawn from biopharma development, peptide medicinal chemistry and life-sciences licensing. Between them, our advisors have contributed to eight approved medicines.
The company was founded in 2015 and recapitalised under new leadership in 2025. All of the chemistry and data behind the current programme has been generated since.
Research network
Collaborating institutions
