Dompé: The first international clinical ophthalmological trial concerning treatment of neurotrophic keratitis (NK), an ocular pathology with no cure, takes place in Italy

MILAN--(BUSINESS WIRE)--Dompé, one of the leading biopharmaceutical companies in Italy, announced today that it has initiated a Phase I clinical trial on the use of recombinant human Nerve Growth Factor (rhNGF), the discovery of which earned Professor Rita Levi Montalcini a Nobel Prize, for treating neurotrophic keratitis, a rare ocular pathology which still has no cure, and affects one person in 10,000 worldwide.

“Our goal is to ensure responsible assistance to patients by developing new drugs that will mark a significant step forward compared to the therapeutic solutions available today.”

NGF is a soluble protein that stimulates the growth, maintenance and survival of neurons. Researchers from Anabasis, a company recently acquired by Dompé, have formulated the hypothesis of the effectiveness of the topical application of NGF, bearing in mind that NK is a neurodegenerative disease. It is a well-known fact that the integrity of the cornea - by far the most innervated organ of the human body (nearly 400 times more than the skin) - is maintained by innervation rather than vascularity. On the basis of this biological observation, over 100 patients with NK in several European countries have been treated with NGF of murine origin, and clinical trials have produced extremely promising data, published in leading international journals.

"Neurotrophic keratitis is a severe degenerative eye disease, caused by reduced innervation of the cornea as a result of a variety of pathological conditions (such as diabetes, cold sores, surgery) that can lead to debilitating consequences, like ulceration and perforation of the cornea, resulting in loss of visual function,” states Prof. Stefano Bonini, Head of the Department of Ophthalmology at Campus BioMedico in Rome, who is among the first researchers in the world to conduct studies on the use of NGF in the field of ophthalmology. "For these reasons, the clinical world's attention has long been focused on the findings of our experiments on NGF’s therapeutic potential. And today, after 10 years of study, I’m particularly proud to see the fruits of our research and to be able to follow the launch of the Phase I clinical trial, with the recruitment of the first volunteer patients. It’s a promising result for an unmet healthcare medical need, thanks to the dedication of all those who were involved in these trials and of my Research Group. Indispensable is the contribution of the industry which, through its expertise and vision, will ensure the development and supply of a new medicine for the treatment of neurotrophic keratitis.”

"We’re delighted with the results achieved so far, which, in view of the clinical pathway yet to be taken, testify to our company’s commitment to safeguarding health” states Sergio Dompé, chairman of the company. “Our goal is to ensure responsible assistance to patients by developing new drugs that will mark a significant step forward compared to the therapeutic solutions available today."

NGF’s ophthalmological potential was first analyzed by Anabasis researchers, as illustrated by the extremely promising data published in leading international journals, supporting the effectiveness of topical application of NGF for the treatment of diseases both of the front and back of the eye.

“NGF represents a fundamental milestone for our research strategy, focused on therapeutic areas with significant health requirements at global level,” says Chief Executive Officer of the Dompé Group Eugenio Aringhieri. “In a changing pharmaceutical scenario marked by several factors – such as average population ageing - awareness of eye diseases is constantly growing, and there is a need for more targeted and customized therapies, which like NGF can offer opportunities for care that were unthinkable until recently.”

In the light of these achievements, Dompé and Anabasis plan to continue their trials in a clinical setting to ensure biotechnological production of a medicine based on recombinant human NGF, with the potential of providing NK-afflicted patients with a real and effective therapeutic reaction.

The trial
Phase I trial, randomized, double-blind, placebo-controlled, combining single and repeated ascending dosage to assess the safety, tolerability and pharmacokinetics of recombinant human Nerve Growth Factor (rhNGF) in eye drops, on healthy male and female volunteers.

About Dompé
Dompé is one of the leading biopharmaceutical companies in Italy, focusing on the development of innovative therapeutic solutions for diseases with high social impact, often with no cure. Based in Italy, Dompé has its headquarters in Milan and focuses its efforts on research into areas of as yet unmet medical needs such as juvenile diabetes, ophthalmology, and oncology. Its industrial hub in L'Aquila (Abruzzo region) houses a biotechnology plant producing monoclonal antibodies, and develops medicines for primary care on the international markets of over 20 countries worldwide. In 2012, Dompé acquired Anabasis, an Italian biotech firm which develops innovative drugs based on rhNGF (the discovery of which earned Professor Rita Levi Montalcini a Nobel Prize) for the treatment of serious eye diseases which currently have no effective treatments.
For more information, please visit: www.dompe.com

Forward-looking statements
This press release refers to some information that may not correspond to future results. Dompé firmly believes in the soundness and reasonableness of the concepts expressed, but some of the information is subject to a margin of uncertainty, regarding research and development and appropriate inspections by the relevant regulatory bodies. Therefore, for the moment, Dompé cannot guarantee the consistency of expected results in relation to the above.