Dompé Receives Positive CHMP Opinion in Europe For Oxervate® (Cenegermin Eye Drops) For the Treatment of Adult Patients with Moderate or Severe Neurotrophic Keratitis

Today, the biopharmaceutical company Dompé has announced that the Committee for Human Medicinal Products (CHMP) of EMA (European Medicines Agency) has released a positive opinion, recommending the marketing authorization for Oxervate® (cenegermin eye drops), for the treatment of adult patients with moderate or severe neurotrophic keratitis. This is a rare and disabling eye condition that can lead to the loss of vision. As of today, no satisfactory treatment is available for this disease. Due to the risks related to this pathology and to the lack of viable therapeutic options, CHMP completed its review through an accelerated assessment. It began in November 2016 and ended with a positive opinion last week.

The origin of neurotrophic keratitis is related to a trigeminal nerve impairment (one of the nerves responsible for the eye anatomy and function), which can lead to the loss of corneal sensitivity. In its most serious forms, it can cause ulcers, melting and corneal perforation, affecting the visual ability of patients².

If confirmed by EMA’s Committee for Orphan Medicinal Products (COMP) and approved by the European Commission, Oxervate® would be the first biotechnology orphan drug in the world authorized for this indication.

Cenegermin, the name of the active agent, is the recombinant version of the human nerve growth factor (NGF) discovered by Nobel Laureate Rita Levi Montalcini. This protein is naturally produced by the human body and is implicated in the development, maintenance and survival of nerve cells³. Administered in the form of eye drops to patients with neurotrophic keratitis, Oxervate® helps restore the normal healing processes of the eye and repair the corneal damage.

Oxervate® is produced through the recombinant DNA technology, with the introduction of a gene (DNA) into bacteria to allow these latter to produce the human nerve growth factor.

CHMP conclusions are based on data from two phase II clinical trials involving 204 patients with moderate and severe neurotrophic keratitis. Both studies have shown that after eight weeks, a higher number of Oxervate® treated patients reached a complete corneal healing vs patients on placebo. The most common adverse reactions observed with Oxervate® include eye pain, eye inflammation, increased lacrimation (secretion of tears), eyelid pain and foreign body sensation in the eye.

"We welcome the CHMP opinion with great satisfaction. To us, making Oxervate® available to patients who live with this rare disease is a major achievement. It would be the first biotech treatment obtained thanks to our research efforts", said Eugenio Aringhieri, Dompé’s Chief Executive Officer. "This decision paves the way to the significant prospect of a therapy for those suffering from this disease. In the future, our goal is to keep investigating its potential in other pathologies, to allow more and more patients to benefit from this innovative therapeutic approach".

"Having been the first to turn the discovery of the Nerve Growth Factor into a potential therapy is a further confirmation of the value of ‘Made in Italy’ research", explained Sergio Dompé, Chairman of Dompé. "At this time my thoughts and gratitude go to the team of researchers who passionately developed Oxervate®, and especially to Professor Rita Levi Montalcini for her great intuition, from which this research project stems, namely the neuronal growth factor for which she received the Nobel Prize."

Until the European Commission’s final decision, Oxervate® remains an investigational therapy. Its marketing authorization has not been granted yet in any country.

About Dompé

Dompé is one of the leading biopharmaceutical companies in Italy. It focuses on the development of innovative therapeutic solutions for diseases with a high social impact for which therapeutic options are lacking. Based in Italy, Dompé has its headquarters in Milan. Its research efforts focus on unmet therapeutic needs such as diabetes, organ transplantation, ophthalmology and oncology. The industrial pole of L’ Aquila (Abruzzo) is home to a world class biotechnology plant developing drugs for Primary Care for the markets of about 40 countries worldwide. Dompé has its offices also in Albania, France, Germany, Great Britain, Spain and United States (New York).

For more information: www.dompe.com and www.dompetrials.com

Forward looking statements

This press release makes reference to certain information that may not coincide with expected future results. Dompé firmly believes in the soundness and reasonableness of the concepts expressed. However, some of the information is subject to a certain degree of indetermination in relation to its research and development activities and the necessary verifications to be performed by regulatory bodies. Therefore, as of today, Dompé cannot guarantee that the expected results will be consistent with the information provided above.

¹ M. Sacchetti, and A. Lambiase, Diagnosis and management of neurotrophic keratitis. Clin Ophthalmol 8 (2014) 571-9.
² Idem
³ R. Levi Montalcini, The nerve growth factor 35 years later, Science 1987

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Contact information

Dompé
Laura Sprea, +39 02 58383567 | +39 3355745188
Corporate Communications Senior Specialist
laura.sprea@dompe.com