MONTEREY, Calif.--(BUSINESS WIRE)--
Italian biopharmaceutical company Dompé today announced the launch of REP0112, a randomized double-blind multicenter Phase II/III trial to assess the efficacy and safety of Reparixin in autologous islet cell transplantation, a procedure where, unlike transplantation from a donor, pancreatic cells are taken from the patient's own pancreas and surgically removed for pancreatic pathology. Launch of the trial was announced at the 14th World Congress of the International Pancreas and Islet Transplant Association (IPITA 2013) in Monterey, California.
“Dompé is committed to identifying treatment solutions for rare, often orphan, diseases. For this reason we are particularly proud of the launch of this Phase II/III trial in the US, a step that brings us closer to providing patients with another effective and safe treatment that satisfies medical needs that are still unmet,” said Eugenio Aringhieri, CEO, Dompé. “The trial we are announcing today and Reparixin, one of the drugs developed by our company, produced at our bio-technology sites and supplied globally, are a testament to our commitment in the industry. This also marks a milestone in our expansion into the US, a country where we recently established a Group subsidiary.”
Enrollment of the first patient is scheduled by the end of 2013 and results are expected in early 2016. The REP0112 trial will enroll 100 islet transplantation-naïve adult patients with iatrogenic diabetes who have undergone total pancreatectomy (total pancreas removal) and are candidates for intrahepatic islet cell transplantation. Patients will be randomized into two groups to receive either Reparixin administered by continuous intravenous infusion for seven days or placebo. The primary objective of the trial is to assess whether Reparixin contributes to improve islet cell transplantation outcomes by measuring the percentage of patients who attain insulin independence after the procedure. The trial will also assess specific treatment safety parameters.
“This is the first time the efficacy and safety of a drug specifically developed to improve the outcomes of autologous islet cell transplantation is investigated in a trial in the US,” explains Dr. Melena Bellin, Schulze Diabetes Institute, University of Minnesota Medical School, and Primary Investigator of the trial. “The trial will be conducted in 100 patients in six US states. Its primary efficacy endpoint will be the percentage of patients who attain insulin independence one year after the procedure. Blood sugar levels and glycosylated hemoglobin (HbA1c) will be measured too, together with the need for insulin and specific inflammatory response parameters.”
Reparixin acts directly on the body’s anti-inflammatory response that occurs in the first days following autologous islet cell transplantation, specifically the response of white blood cells known as polymorphonuclear leukocytes. CXCL8 (or interleukin-8) plays a key role in the anti-inflammatory response triggered right after pancreatic cell infusion, and is therefore a key target in drug development to help facilitate successful implantation. Reparixin has shown to improve graft survival rates and graft function in both in vitro and in animal models by acting on CXCL8.
“The initiation of the US trial complements the Phase III trial underway in Europe. The European study will assess the efficacy and safety of Reparixin in allogeneic islet cell transplantation and involves nearly half of patients who undergo this procedure annually,” explains Prof. Lorenzo Piemonti, Deputy Director of the San Raffaele Diabetes Research Institute and Director of the Islet Cell Transplantation Program. “The patient’s anti-inflammatory response in the days following islet infusion adversely impacts cell survival with a 50% decline in islet function in the first 7 days after the procedure. Reparixin is being studied to assess its role in inhibiting the body’s inflammatory response and its potential to improve the efficacy of islet cell transplantation and insulin independence.”
Results from a Phase II trial of patients who have undergone islet cell transplantation from donors (allogeneic transplantation) and received Reparixin in combination with immunosuppressive drugs were also presented at IPITA 2013. The data demonstrated that treatment with Reparixin promotes graft function after transplantation, results in a significant improvement of blood glucose control compared to the pre-transplant condition and delivers insulin independence in three out of four patients.
Reparixin is a potent selective chemokine interleukin-8 inhibitor developed for the purpose of selectively inhibiting the body’s anti-inflammatory response and preserving islet function, thereby helping improve the efficacy of islet cell transplantation. Clinical data show that Reparixin can act both on inflammation (which initiates destruction of implanted cells) and subsequent rejection (of allografts). The goals of treatment with Reparixin include blocking the trigger of the anti-inflammatory immune response, preventing rejection of transplanted cells and increasing the probability of restoring the ability to keep blood sugar levels under control. Reparixin was granted Orphan Medicinal Product Designation in Europe in 2011 and in the United States in 2012 for the prevention of graft rejection in pancreatic islet transplantation.
A leading pharmaceutical company in Italy, Dompé develops innovative treatment solutions for diseases that have a high social impact and are often orphan diseases. Based in Italy with HQ in Milan, Dompé focuses on research in areas where an unmet treatment need still exists, such as juvenile diabetes, ophthalmology and oncology. Its industrial site in L’Aquila (Abruzzo) has biotechnology facilities for the production of monoclonal antibodies and the development of Primary Care drugs that are sold internationally. In 2012, Dompé acquired Anabasis, an Italian biotech company that develops innovative drugs based on rhNGF (whose discovery earned Professor Rita Levi Montalcini the Nobel Prize for Medicine) for serious eye diseases for which there are no effective treatments available.
For more information: www.dompe.com
Forward looking statements
This press release may contain forward looking statements. Dompé firmly believes the expectations reflected in forward looking statements are reasonable and built on sound assumptions. However, by their very nature, forward looking statements are subject to uncertainties including those inherent in R&D and those associated with decisions made by regulatory authorities. Hence, forward looking statements are not a guarantee of future results which may be different.
1) Dati su Trapianto isole pancreatiche: European survey conducted by IMS Consulting Group, 2012
2) P. Maffi et al., Islet allotransplantation in type 1 diabetes: phase 2 pilot study with CXCL8 inhibitor (reparixin), https://tech4pco.com/ipita2013/user_agendas/open_view/191
3) Data on insulin independence elaborated from Sutherland et al., 2008 and 2012
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