Leveraging ARTEMIS, Alethio is building a pipeline of programmes aimed at addressing the underlying biology of MPNs and the persistent unmet needs of patients.
ATX- 011 is a first in class programme built around a novel target validated in tissue from more than 80 MPN patients, aiming to move treatment beyond symptom control toward true disease modification. This target is selectively expressed on cancerous disease driving stem and progenitor cells, making it ideal for an antibody approach that eliminates the root cause of disease.
ATX-011 is a novel monoclonal antibody being developed to reduce thrombotic risk and improve disease control in patients with chronic phase MPNs, predominantly essential thrombocythaemia (ET). These conditions are marked by excessively high platelet counts, where current treatments are blunt, broadly toxic, and slow to act.
In NHP studies, ATX-011 demonstrated rapid, controlled and reversible platelet reduction, with a favourable tolerability profile. Translational modelling supports convenient subcutaneous monthly dosing.
ATX-011 has a rapid path to clinical proof of concept with IND/CTA-enabling studies expected to begin in first half of 2027.
A mutant CALR (mutCALR)–targeted programme being developed for the ~30% of MPN patients who carry this driver mutation. The programme is designed to address a key biological challenge in ET and myelofibrosis and to expand the therapeutic potential of targeted approaches in these diseases
First in class ADC built on the same novel target as ATX-011, engineered to deliver potent, selective activity within the immunosuppressive and fibrotic MF bone marrow.
Preclinical studies show strong activity against patient derived mutant cells. In disease relevant models, our mutation-agnostic ADC has demonstrated compelling signs of disease modification, including reductions in spleen size and tumour burden, and a doubling of survival – highlighting its potential to deliver meaningful clinical benefit.
