Type 1 Diabetes (T1D): Enrolment of the First Patient Kicks off International Phase III Trial on Reparixin, the Investigational Drug Developed by Dompé R&D

  • T1D affects some 20 million people worldwide, accounting for 10 percent of all diabetic cases
  • Reparixin is the investigational drug developed by Dompé to improve efficacy of the experimental pancreatic islet transplantation procedure
  • After being granted Orphan Drug Designation by the FDA and the EMA, with the enrolment of the first patient Reparixin enters a Phase III trial investigating it in allogeneic pancreatic islet transplantation for the first time ever in the world
  • The Phase III trial will be conducted in 7 countries in Europe and in the United States
     

MILAN, Italy I October 23, 2012 I Dompé, one of Italy’s leading biopharmaceutical companies, today announced the enrolment of the first patient into a Phase III trial on Reparixin, the compound that has shown to improve the efficacy of transplantation of insulin-producing pancreatic islets, which is the new frontier in type 1 diabetes (T1D) treatment. This comes on the heels of the promising results obtained by Reparixin in previous stages of clinical development. The Orphan Drug Designation given by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to Reparixin marked a further milestone in the development of this novel investigational drug.

Reparixin is a potent selective chemokine interleukin-8 inhibitor developed by Dompè Italian R&D laboratories. It selectively inhibits the body’s anti-inflammatory response preserving islet function and thus contributing to improve the efficacy of pancreatic islet transplantation.

The randomized double-blind multicenter Phase III trial will be conducted in 10 centers of 7 countries in Europe and the United States on some 60 patients, i.e., half the total number of patients undergoing allogeneic pancreatic islet transplantation worldwide. The study will assess the efficacy of Reparixin in improving the efficiency of pancreatic islet transplantation, but also its efficacy in increasing patient insulin independence and blood glucose control.

T1D is the most common chronic disease in children and occurs when the body's immune system wrongly attacks and destroys the insulin-producing cells in the pancreas (beta cells) so patients need insulin injections to keep their blood sugar under control.

“Type 1 Diabetes is a disease which is on the rise globally with significant impact also on the young population” – said Eugenio Aringhieri, CEO, Dompé Group. “Together with the Orphan Drug Designation granted by the FDA and the EMA, the beginning of the Phase III trial marks a key milestone in the clinical development of Reparixin in view of improving the efficacy of pancreatic islet transplantation and help it become a standard procedure which provides a valuable option for juvenile diabetes patients.”

In a randomized multicenter Phase II trial Reparixin has shown to significantly improve the efficiency of pancreatic islet transplantation, preventing the death of beta-cells after transplantation, ensuring long term graft survival and thus increasing the percentage of patients who maintain insulin independence one year after the transplantation.

Thanks to the efforts of the international scientific community, in recent years pancreatic islet transplantation has become an increasingly used highly promising option. As pancreatic islet cells maintain their function when transplanted into other organs, the islets taken from a donor pancreas can be infused through the portal vein into the liver of the recipient with a simple injection. Once implanted, the beta cells in the islets begin to make and release insulin, thus keeping blood glucose under control.

“The idea that a targeted anti-inflammatory treatment could help pancreatic islet transplantation become a standard procedure has been confirmed in earlier clinical trials on this investigational drug” - said Lorenzo Piemonti, Principal Investigator, Head of the Pancreatic Islet Transplantation Program, San Raffaele Hospital IRCCS, Milan, Italy. “We expect major insight confirming the potential of this investigational drug from the international multicenter Phase III trial. Our hope and goal is significant improvement of patient’s quality of life and lower immune suppression-related risk.”

The benefits of pancreatic islet transplantation, which is still an experimental procedure, are apparent. Yet, there have been factors which progressively reduce the function of transplanted islets and have limited the procedure so far. A major factor has been the patient’s anti-inflammatory response in the days following islet infusion that adversely impacts cell survival with 50% decline in islet function in the first 7 days after transplantation. By inhibiting the anti-inflammatory response Reparixin may improve the efficacy of pancreatic cell transplantation.

About Reparixin
Reparixin is an inhibitor of the CXCR1 receptor which in the body is activated by chemokine interleukin-8 that plays a key role in anti-inflammatory response.
It is the first in a novel class of low-molecular weight inhibitors that can selectively modulate the receptor activity via an allosteric mechanism of action. An allosteric inhibitor can freeze the receptor in an inactive position binding it to a different site than the site taken by the natural ligand (IL-8).
Made in collaboration with the research team led by Prof. Alberto Mantovani, world leading expert in chemokine research, the characterization of the action mechanism of Reparixin is a mainstay in the research of drugs capable of modulating the activity of this important receptor family.

Type 1 Diabetes (T1D) and Pancreatic Islet Transplantation
It is estimated that in Italy about 250,000 people have Type 1 diabetes mellitus. Every year 84 new T1D cases are diagnosed every 1,000,000 inhabitants (about 5,000 cases yearly). Incidence varies significantly from region to region. Currently it stands around 12-13 cases every 100,000 people yearly but it is worryingly on the rise making Type 1 Diabetes one of the next health challenges.
Cure for type 1 diabetes and many cases of type 2 diabetes entails replacing impaired beta cells with healthy cells that can perform two key actions, i.e., gauge blood glucose levels and release the right quantity of insulin into the vascular bed. In fact, both type 1 and type 2 diabetes have in common impaired beta cell function although due to two different causes (auto-immune response and insulin resistance, respectively). At present, the only way to replace beta cells in diabetic patients is either pancreatic islet transplantation or pancreas transplantation. In the past decade the efficacy of pancreatic islet transplantation has impressively improved and this procedure is now a widely recognised treatment option for specific subgroups of patients. It is likely that in the near future its efficacy will improve further and hence be more extensively used in patients with uncontrolled blood glucose and recurring hypoglycemia. This procedure is also extraordinarily valuable as a model to study the processes that regulate autoimmunity. Experience gained over these years will lay the groundwork for future clinical development of any cellular/tissue or bio-artificial treatment solutions for replacement of impaired beta cells in patients with diabetes.

About Dompé
A leading pharmaceutical company in Italy Dompé develops innovative treatment solutions for diseases that have a high social impact, which are often orphan diseasées. Based in Italy with HQ in Milan Dompé focuses on Research in areas where there are still unmet treatment needs such as juvenile diabetes, ophthalmology and oncology. Its industrial site in L’Aquila (Abruzzi) has biotechnology facilities for the production of monoclonal antibodies and the development of Primary Care drugs that are sold internationally in over 60 countries. 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

SOURCE: Dompé

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