The First Patient Has Been Enrolled in the First International Clinical Trial of rhNGF for the Treatment of Neurotrophic Keratitis

MILAN--(BUSINESS WIRE)--The first patient has been enrolled in the phase I/II REPARO study, the first international clinical trial evaluating the ophthalmological use of a topical solution of recombinant human Nerve Growth Factor (rhNGF) for the treatment of moderate to severe neurotrophic keratitis (NK). The drug was developed on the basis of the experimental studies conducted by the late Professor Rita Levi Montalcini, who was awarded the Nobel Prize for her discovery of this signalling protein involved in the development of the nervous system.

“There is an increasing international awareness of ophthalmological diseases and the need to find new approaches to treating them. Our commitment to research in this area has the aim of making a significant step forward in comparison with the currently available therapeutic solutions.”

The first patient with this rare, sight-threatening degenerative disease of the cornea, which afflicts one in 10,000 people throughout the world, was recruited at San Raffaele Hospital in Milan. The clinical trial is sponsored by Dompé, one of Italy’s leading biopharmaceutical companies, dedicated to finding innovative therapeutic solutions for diseases that have a high social impact in the areas of diabetes, ophthalmology and oncology.

The REPARO Study is randomised, double-masked, placebo-controlled trial that will enrol patients with grade 2 (persistent epithelial defect) or grade 3 lesions (corneal ulcer) NK who do not respond to currently available medications in nine countries. The primary objective of the trial is to evaluate the safety and efficacy of two doses of a rhNGF (10 µg/ml and 20 µg/ml) solution administered topically in the form of eye drops. It is planned to enrol at least 174 patients, who will be randomized to one of the two doses or placebo during the 8 week controlled treatment period. There after follow-up will last at least 48 weeks. The secondary objectives of the trial are to evaluate the duration of corneal healing, improvement in visual acuity and corneal sensitivity.

“There is an increasing international awareness of ophthalmological diseases and the need to find new approaches to treating them. Our commitment to research in this area has the aim of making a significant step forward in comparison with the currently available therapeutic solutions.” - declared Eugenio Aringhieri, CEO of the Dompé Group. “To this end, our R&D centre has developed the human variant of NGF, and this trial will open the way to the development of an innovative drug for the treatment of severe neurotrophic keratitis. We believe that the results of the trial will allow us to offer a proven therapy to patients throughout the world, who run the risk of losing their eyesight due to the lack of effective treatment options and, more generally, will further clarify the potential use of NGF in ophthalmology."

"Neurotrophic keratitis is a severe degenerative eye disease caused by reduced corneal innervation that can lead to the ulceration and perforation of the cornea, and a consequent loss of visual function,” said Prof. Stefano Bonini, Director of the Department of Ophthalmology at Campus BioMedico in Rome, and one of the first researchers in the world to study the ophthalmological use of NGF. “It can be caused by various clinical conditions such as diabetes or herpetic infections, or previous surgery. As a result of the commitment of Dompé, we have been able to start an international research programme that has the aim of providing what will be the very first therapy for Neurotrophic keratitis directly targeting the underlying cause of this serious disease, reduced corneal innervation. It is a further demonstration of the importance of a continuous exchange of information between basic researchers and the highest levels of industrial capacity.”

The REPARO Study
REPARO is a phase I/II randomised, double-masked, placebo-controlled trial combining increasing single and repeated doses of rhNGF in eye drops in order to assess safety, tolerability and pharmacokinetics of the drug in adult patients affected by moderate to severe, unilateral grade 2 (persistent epithelial defect) or grade 3 (corneal ulcer) neurotrophic keratitis who have failed to respond to the currently available treatments. The planned treatment period is eight weeks, after which the patients will be followed up for 48 or 56 weeks. Eligible patients will be enrolled at participating centres located in nine different countries.

About Dompé
A leading biopharmaceutical company in Italy Dompé develops innovative treatment solutions for diseases that have a high social impact, which are often orphan diseases. 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 (Abruzzo, Italy) employs 230 people, of whom 70 in R&D, develops drugs that are sold in over 60 countries around the world.

In 2012, the Dompé Group has ploughed back over 10% of sales into R&D, focusing its commitment on therapeutic areas with unmet medical needs, such as diabetes, ophthalmology and oncology.
For more information: 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.