iOnctura at Campus Biotech: exploring new approaches to myelofibrosis

Founded in 2017, with offices at Campus Biotech, iOnctura is a clinical-stage biopharmaceutical company developing precision oral small molecules for rare and hard-to-treat cancers. Its work in myelofibrosis exemplifies a translational research approach closely connected to clinical practice, fully aligned with the missions of Campus Biotech.

A clinical-stage company focused on rare cancers 

iOnctura is dedicated to the development of first-in-class small molecules targeting biological mechanisms involved in cancers that remain inadequately treated. Its lead asset, roginolisib, is currently being evaluated in multiple randomized Phase II clinical trials across solid and hematological malignancies.

Headquartered in Amsterdam, with subsidiaries in Geneva and Cambridge, Massachusetts (USA), the company operates within an international network of researchers and clinicians. Its presence at Campus Biotech reflects this positioning, at the intersection of scientific research, clinical development and biomedical innovation.

Myelofibrosis: a significant unmet medical need

Myelofibrosis is a rare cancer of the bone marrow. It is characterized by progressive marrow fibrosis, leading to impaired blood cell production and severe clinical manifestations, including profound fatigue, anemia and splenomegaly.

For more than a decade, Janus kinase (JAK) inhibitors have been the standard of care. While they provide symptomatic relief, they do not alter the natural course of the disease. Approximately half of patients discontinue treatment within three years due to loss of efficacy, intolerance or adverse events.

When the disease adapts to treatment 

Myelofibrosis does not arise from a single biological mechanism. Although dysregulation of the JAK–STAT pathway plays a central role in disease pathophysiology, alternative signaling pathways can compensate when this pathway is inhibited by treatment.

One such pathway is PI3K signaling, particularly the PI3Kδ isoform, which is implicated in hematologic malignancies. Research indicates that when JAK activity is suppressed, malignant cells may activate the PI3Kδ pathway to sustain proliferation. This mechanism contributes to the non-response or loss of response observed in a proportion of patients treated with JAK inhibitors.

A combination strategy under clinical evaluation 

In myelofibrosis, iOnctura is evaluating the combination of roginolisib with ruxolitinib, a JAK inhibitor and standard-of-care therapy. This strategy is being investigated in the Phase I/II HEMA-MED clinical trial, enrolling patients who no longer respond to JAK inhibitor monotherapy.

Non-clinical data presented at the annual meeting of the American Society of Hematology demonstrate that this combination exerts a synergistic effect in cellular models of myelofibrosis.

As highlighted by Dr Michael Lahn, Chief Medical Officer of iOnctura:

“Based on non-clinical data being presented at ASH, we anticipate that targeting both the PI3Kδ and JAK pathways could have a synergistic effect in myelofibrosis — an approach that has previously eluded older-generation PI3Kδ inhibitors because of their unfavorable tolerability profiles.”

Research aligned with the missions of Campus Biotech 

By advancing targeted therapeutic approaches for rare diseases at the interface of science and clinical medicine, iOnctura exemplifies the mission of Campus Biotech: to foster translational research capable of transforming scientific advances into tangible solutions for patients.