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Therapeutic Areas

Our commitment to the patient


Dompé is currently involved in the Research & Development of innovative therapeutic solutions in ophthalmology. A journey that starts with the Italian research - born from the studies of the Nobel Prize winner Rita Levi Montalcini - for the development and production of potential biotechnological drugs in response to eye diseases, some of which are rare.

The Nerve Growth Factor (NGF) is a soluble protein that stimulates the growth, retention and survival of neurons. Discovered in the 1950s by Nobel Prize winner Rita Levi Montalcini, NGF belongs to the neurotrophins, a family of proteins that can play a crucial role in regulating the survival, development and functions of neurons, both at a central and peripheral level. Today the molecule is the basis of a solution for the treatment of a rare disease of the cornea, neurotrophic keratitis, and is also being studied for other diseases affecting the eye.

Click here to learn more about ophthalmology studies.


In the field of Oncology, Dompé is developing molecules that target specific and innovative cellular or biological mechanisms that can open up new perspectives in the treatment of some of the most common forms of cancer. As such, Reparixin, a product of Dompé’s research, is being tested in patients with triple negative metastatic breast cancer.

Breast cancer is the second most common cancer in the world and the most common among women, both in developed and developing countries. The only treatment currently available for the triple metastatic subtype is chemotherapy, with somewhat unsatisfactory results, as it seems to promote the development of cancer stem cells (CSC), which, given that they contain the instructions for producing new neoplastic units, are the basis for the progression of the disease, the development of metastasis and relapse after treatment. Cancer stem cells represent a small fraction of the total cancer cells and, biologically, they are able to support tumour growth thanks to their capacity for self-renewal, completely independent from the body’s control systems.

The objective of the Dompé Research is to identify therapeutic solutions that can combat this particularly aggressive form of tumour by inhibiting CSCs.

Preclinical testing of Reparixin has shown it to decrease the population of CSC both in vitro and in vivo1. The action of the molecule in animal models induces a delay in the development of the tumour and slows down the development of metastases.

The molecule was subsequently evaluated in 3 international trial clinical studies.

  • Phase Ib clinical study for the evaluation of the safety and pharmacokinetic profile of reparixin, administered orally in combination with Paclitaxel in women with metastatic breast cancer (completed at 5 US sites2).
  • Pilot clinical study to evaluate the effects of reparixin taken as oral monotherapy on CSC and on the tumour microenvironment in women with post-surgery early stage breast cancer (completed at 9 US sites).
  • Phase II randomised clinical trial (fRida) with the aim of evaluating progression-free survival after combination treatment of oral reparixin and standard therapy with Paclitaxel compared to placebo-associated paclitaxel treatment in patients with non-pre-treated triple-negative metastatic cancer. Enrolment took place at 65 sites in the US, Belgium, France, Italy, Poland, the Czech Republic and Spain.

The original mechanism of action of reparixin could find future application in the study of other neoplasms, such as pancreatic cancer3. Furthermore, it has been shown that silencing the CXCR1 and CXCR2 receptors results in inhibition of tumour and melanoma growth and the capacity for cell dissemination4.

Click here to learn more about oncology studies.

Diabetology and Transplantation


Diabetes is characterised by a rise in blood sugar or increased glucose levels in the blood. There are different forms of diabetes. Type 1 diabetes is an autoimmune disease and is the most widespread chronic disease in the paediatric age group, but the disease can also occur in adulthood. Type 1 diabetes is caused by the autoimmune destruction of insulin-producing Langerhans islet cells in the pancreas.

Dompé is involved in the field of type 1 diabetes, with two molecules resulting from its research, Reparixin and Ladarixin, able to act on the inhibition of Interleukin-8 (IL-8), a molecule involved in the pathogenesis of type 1 diabetes and in the inflammatory reaction immediately following pancreatic islet transplantation, which limits the efficacy of this therapeutic approach aimed at a group of patients with advanced type 1 diabetes.

Transplantation of Pancreatic Islets

The transplantation of donor or allogeneic pancreatic islets is a therapeutic option for patients with advanced type 1 diabetes who are unable to administer insulin to adequately control blood sugar. A second form of pancreatic islet transplantation (autologous transplantation) involves patients undergoing the surgical removal of the pancreas, which can prevent or reduce the severity of diabetes following surgery by receiving the pancreatic islets taken from the removed organ. Independence from insulin administration can be achieved in a proportion of patients up to 40-45% in the case of donor transplantation1 and 40% following autologous transplantation2.

Based on preclinical results in murine models3 and a first Phase II randomised clinical trial conducted on 9 patients who indicated that treatment with Reparixin given as a continuous intravenous infusion in the days immediately following transplantation could significantly improve the efficacy of pancreatic islet transplantation by donor, two randomised clinical trials were conducted to evaluate the efficacy and safety of the drug in the donor transplant, most developed in Italy and in Europe, and in the autologous one, a common practice in the United States after a pancreatectomy.

  • Donor transplantation. A Phase III registry study took place involving 8 sites in 5 countries between Europe and the US with the enrolment of around 50 patients. The primary objective of the study was the evaluation of the efficacy of Reparixin in the improvement of pancreatic islet transplantation, while the secondary objectives include measuring the percentage of patients who, after pancreatic islet transplantation and treatment with the drug, achieved independence from insulin and optimal control of blood sugar.
  • Autologous transplantation. In the US, a Phase II/III study was completed to assess the efficacy and safety profile of Reparixin in autologous pancreatic islet transplantation. The study involved the participation of about 100 adult patients for pancreatic islet transplantation and undergoing a total pancreatectomy who had not previously received an islet transplant. The aim of the study was to evaluate the efficacy of Reparixin in improving the function of transplanted cells by measuring the percentage of patients who become independent of insulin following islet transplantation.

Ladarixin and type 1 diabetes

Ladarixin is a drug discovered as a result of Dompé’s research. It acts as an inhibitor of IL-8, the main mediator of inflammation in the processes leading to the progressive autoimmune destruction of Langerhans islet cells in type 1 diabetes. The drug therefore aims to provide an innovative contrast to the inflammatory reactions that characterise the initial phase of the disease when the chances of modifying its clinical history are greater. After the positive results obtained in animal models that spontaneously develop type 1 diabetes1, a randomised phase 2 clinical trial is underway that has enrolled 76 patients at 8 specialist sites in Italy, Belgium and Germany, and will end in 2019.

Click here to learn more about diabetology and transplantation studies.


  1. C. Ginestier et al., CXCR1 blockade selectively targets human breast cancer stem cells in vitro and in xenografts, J Clin Invest. 2010;120:485–497. doi:10.1172/JCI39397
  2. AF. Schott et al., Phase Ib pilot study to evaluate reparixin in combination with weekly paclitaxel in patients with HER2 negative metastatic breast cancer. Clin Cancer Res 2017; 23:5358-65. doi:
  3. L. Chen et al., The IL-8/CXCR1 axis is associated with cancer stem cell-like properties and correlates with clinical prognosis in human pancreatic cancer cases. Sci Rep. 2014; 4: 5911. doi: 10.1038/srep05911
  4. Kemp DM et al.,Ladarixin, a dual CXCR1/2 inhibitor, attenuates experimental melanomas harboring different molecular defects by affecting malignant cells and tumor microenvironment. Oncotarget. 2017 Feb 28;8(9):14428-14442. doi: 10.18632/oncotarget.14803.
  5. Barton FB et al., Improvement in outcomes of clinical islet transplantation: 1999-2010, Diabetes Care 2012, 35(7): 1436-1445
  6. Sutherland DE et al., Total Pancreatectomy and Islet Autotransplantation for Chronic Pancreatitis, J Am Coll Surg., 2012, 12(5): 580–586
  7. Citro A et al., CXCR1/2 inhibition enhances pancreatic islet survival after transplantation, J Clin Invest 2012; 122:3647-51. doi 10.1172/JCI63089
  8. Citro A et al., CXCR1/2 inhibition blocks and reverses type 1 diabetes in mice. Diabetes 2015; 64:1329-40. doi: 10.2337/db14-0443