Our Focus
We focus on the areas that others shy away from; pioneering treatments for hard to treat, often neglected cancers.
Our commitment is unwavering: tackling the most challenging cancers that others overlook. We specialise in pioneering treatments for hard to treat, often neglected cancers, dedicating our resources and expertise to these critical areas. Our mission is driven by the belief that every patient deserves innovative, effective care — regardless of their diagnosis.
By focusing on cancers with high unmet need, we aim to make a profound impact on patient outcomes, offering hope and new possibilities where few exist. Our dedication to addressing these critical gaps in cancer care underscores our commitment to transforming the landscape of oncology and delivering tangible benefits to those who need it most.
Changing Lives
More time
for the moments that matter
We are setting new standards for treatment efficacy and safety, whilst significantly reducing negative side effects. As a leader in producing oral small molecules, our medicines can be taken independently at home, minimising disruption to day-to-day living.
Improving clinical profiles and extending quality of life, we enable patients to spend more quality time with the people that matter most to them.
“The impact of roginolisib on my life has been immeasurable. Not only am I still here, but I’m still able to enjoy all of the things I love doing, like being active with my friends and family.”
– Clinical trial patient, Europe
“Patients tolerated roginolisib very well and allowed the drug to be given for several months resulting in longer than historically expected overall survival.”
– Professor Michele Maio, Università Degli Studi Di Siena
“I have been very impressed with the safety profile of roginolisib and patients taking this drug have been able to live longer than expected.”
– Professor Dr Armando Santoro, Humanitas Milan
Clinical Trials
Interested in participating in groundbreaking research? Here you can find out more about our trials and how you may be able to play your part in the advancement of patient-first oncology.
Uveal melanoma is a rare cancer arising within the uveal tract of the eye. When the cancer metastasizes, which it does in approximately 50% of patients, there are limited treatment options and projected overall survival is only a year.
PI3Kδ over-expression stimulates multiple cancer mechanisms and has an oncogenic role in many tumor types, including uveal melanoma. Allosteric modulation of PI3Kδ by roginolisib boosts antitumor immunity by directly modulating regulatory and cytotoxic T-cells, reducing immunosuppression and also inhibits cancer cell survival directly. Compelling data from the Phase I/II DIONE-01 study supported the rational to expand the clinical program to the randomized controlled Phase II trial, OCULE-01, which started recruiting patients in March 2025.
OCULE-01 STUDY NCT06717126
OCULE-01 is a Phase II open-label, randomised, parallel-arm study, which is assessing the clinical efficacy of oral roginolisib as monotherapy against a control of Investigator’s treatment choice in patients with advanced or metastatic uveal melanoma (UM).
Additional study information can be found by visiting:
Pancreatic cancer is one of the most lethal cancers and is associated with devastating impact on the quality of life of patients. Current treatments are largely ineffective and there is a need for game-changing therapies. Inhibition of autotaxin is a novel treatment strategy that offers a three-pronged attack on the tumor through direct cancer cell inhibition, immune effector stimulation and inhibition of fibrotic processes, giving drugs and immune cells better access to the tumor.
Translational research showing the potential of cambritaxestat in multiple cancer models, including pancreatic cancer, has recently been published in the ESMO journal Immuno-Oncology and Technology (IOTECH), Cancer Research, the Journal of Experimental & Clinical Cancer Research, and Cancers. Across these publications, cambritaxestat showed strong reduction of metastasis and tumor outgrowth in preclinical models, as well as safe and tolerable dosing in healthy volunteers.
The Phase I AION-02 study reported results in October 2025.
The results showed showed the IOA-289-102 Phase 1 study met its primary endpoint to assess safety and antitumour responses in patients with metastatic pancreatic cancer.
Combining cambritaxestat with gemcitabine/nab-paclitaxel (GnP) was tolerable
and associated with anti-tumor responses.Cambritaxestat administration combined with GnP demonstrated reduction in
pharmacodynamic markers associated with fibrosis, immune regulation and
tumor progression.Additional information on the study can be found by visiting:
NSCLC is the most common type of lung cancer. Lung cancer is the leading cause of cancer death globally, accounting for approximately 1 in 5 of all cancer deaths.
Anti-programmed death-ligand 1 (PD-L1) or anti-programmed cell death protein 1 (PD1) immunotherapy is a standard therapy for NSCLC patients with no actionable mutations in the first or second lines of therapy, with or without chemotherapy. However, treatment is often of limited duration as the cancer cells develop resistance to the treatment.
Emerging clinical and translational biomarker data supports the hypothesis that combining roginolisib with an anti-PD-L1/PD1 agent, with or without docetaxel, may prevent or reverse drug resistance in NSCLC and may show synergistic anti-tumor immune activity without significant addition of toxicity.
This evidence supported the rational to expand the clinical program to the randomized controlled Phase I/II trial, PULMO-01, which started recruiting patients in May 2025.
PULMO-01 STUDY NCT06879717
PULMO-01 is an open-label, randomized, parallel-arm Phase I/II study assessing roginolisib in combination with dostarlimab, with or without docetaxel in patients with NSCLC resistant to first-line checkpoint inhibitor therapy.
Additional study information can be found by visiting:
MF is a rare myeloproliferative neoplasm marked by activation and growth of mutated cells in the bone marrow, with approximately 0.5 cases per 100,000 individuals diagnosed globally a year[1].
JAK inhibitors are a key treatment option for myelofibrosis patients and improve symptoms and quality of life, but approximately half of patients discontinue therapy within 30 months[2]. Reasons for discontinuation include tolerability and disease progression resulting from resistance to the JAK therapy2,[3],[4].
PI3K-Akt pathway activation is a well described mechanism of resistance in response to JAK inhibition[5]. Inhibition of this pathway using roginolisib may overcome resistance mechanisms and lead to a beneficial therapeutic effect.
HEMA-MED STUDY NCT06887803
The HEMA-MED study (NCT06887803) is a Phase I/II open label, multi-center study assessing the safety and tolerability of the PI3Kδ inhibitor roginolisib in combination with the JAK inhibitor ruxolitinib, in patients with MF who are unresponsive to JAK therapy.
Approximately 26 patients who no longer respond adequately to ruxolitinib will be enrolled at multiple sites across Europe. In addition to safety, the HEMA-MED study will determine biomarker responses, spleen reduction responses, preliminary signs of clinical efficacy, improvements of MF related symptoms and pharmacokinetic (PK) parameters.
The trial is currently recruiting patients.
[1] Titmarsh et al., Am J Hematol. 2014;89(6):581-587
[2] Verstovsek, Haematologica. 2015;100:479
[3] Kuykendall. Ann Hem. 2018; 97:435
[4] Newberry.Blood. 2017;130:1225
[5] Moyo et al., Clin Cancer Res (2023) 29 (13): 2375–2384
Chronic lymphocytic leukemia (CLL) is a slow-growing cancer of the blood and bone marrow, caused by the accumulation of dysfunctional B lymphocytes.
The US Department of Defense (DoD) awarded funding to Dr. Jennifer Brown at Dana-Farber Cancer Institute in Boston, MA, to explore the potential of roginolisib in relapsed / refractory CLL in a Phase I / II study.
This study (NCT06644183) will test the safety and anticancer activity of the combination of roginolisib, venetoclax and rituximab for R/R CLL after prior Brutons tyrosine kinase inhibitor therapy.
Preclinical evidence by Sasi, Tarantelli et al., published in mid-2025 supports the synergistic potential of roginolisib and venetoclax in the treatment of CLL.
Additional study information can be found by visiting:
Peripheral T-cell lymphomas (PTCLs) are a heterogeneous group of aggressive malignancies with poor responses to conventional therapies and dismal long-term outcomes.
Roginolisib is being investigated in the Phase I / II innovative platform study, PlaTform, in collaboration with LYSARC (NCT07018752). The efficacy and safety of roginolisib in relapsed/refractory PTCL is being investigated.
Additional study information can be found by visiting:
Useful Resources
Battling cancer or supporting somebody who is, can make you feel like you have a million questions and no answers. Below are some resources that may help.
Not sure where to turn?
Get support.