Italy’s Dompé has unveiled ambitious expansion plans, with chief executive Eugenio Aringhieri telling PharmaTimes that its rhNGF (recombinant human nerve growth factor) will be the springboard that will transform the company.
His confidence will have been boosted by preliminary results from clinical studies of rhNGF in treating neurotrophic keratitis, presented at the Congress of the European Society of Cornea and Ocular Surface Disease Specialists in London.
In 2012 Dompé acquired Italian biotech Anabasis and its in-house rhNGF expertise. Dompé have since mastered production of rhNGF and are now producing clinical grade material. According to Mr Aringhieri, further investment of 50 million euros, including a substantial grant from the Italian Government, will scale production to commercial grade.
Dompé’s rhNGF is the only recombinant human variant of the NGF molecule for ophthalmic use. The company has strong IP protection for both indication and manufacturing process, Mr Aringhieri said.
The US Food and Drug Administration has assigned rhNGF orphan status for neurotrophic keratitis, a degenerative corneal disease that affects around one in 5,000 people and for which there is currently no cure. Orphan drug designation for the same disorder is expected from the European Medicines Agency in early 2015; both the FDA and the EMA have granted rhNGF orphan drug status for the treatment of retinitis pigmentosa.
Looking at dry eye and glaucoma
With Phase II data expected early 2015, Mr Aringhieri said that the company will be meeting with EMA officials in the autumn, to explore accelerated access to market. Looking to the future, he said that the real promise for rhNGF lies in a step-wise transition to eye disorders that affect millions of patients, such as dry eye and glaucoma.
According to recent estimates, some five million Americans aged 50 years and older have dry eye and tens of millions more have less severe symptoms. It has been estimated that 80 million people world-wide will have glaucoma by 2020. Dompé has clinical trials of rhNGF underway in both eye disorders, including a US glaucoma study.
The neurotrophic keratitis results presented at EuCornea were preliminary findings from the Phase I REPARO Study. Although still blinded, the data confirms a good safety and tolerability profile with reductions in symptoms such as blurred vision (-24%), burning (-16%), foreign body sensation (-9%), itching (-8%), and photophobia (-14%).
In 73% of patients treated, complete closure of the corneal wound was observed, which in one out of three cases was associated with improvement of the sensitivity of the cornea, said Flavio Mentellis, medical director of ophthalmology at Dompé. The Phase II segment of the REPARO study is currently underway in 39 centres in nine European countries with a parallel Phase II study starting in the USA this December.
NGF was discovered by Rita Levi Montalcini, who was awarded the Nobel Prize for Medicine and Physiology in 1986 for her work. The protein stimulates the growth, maintenance, and survival of nerve cells, in addition to the cells of the cornea and the retina.
Headquartered in Milan, Dompé had 2013 revenues of 400 million Euro with 12% of turnover currently invested in R&D. Other therapeutic areas include diabetes, organ transplantation and oncology.