Dompé Farmaceutici and Dompé announced the first patient has been enrolled in Study NGF0118, a multicenter, randomized, double-masked, vehicle-controlled, parallel group study to evaluate the safety and efficacy of rhNGF eye solution vs. vehicle in patients with moderate to severe Dry Eye Disease (DED). The study will analyze approximately 300 patients at 11 different sites in the United States for a period beginning with four weeks of active treatment followed by 12 weeks of observation. In a separate clinical study, the company plans to collect a variety of biomarker information from DED patients as part of an initiative to develop tailored treatments.

"The effects of currently available treatments for chronic dry eye disease mostly address only inflammation and wear off soon after discontinuing therapy. Our researchers have decades of understanding of the mechanism of action of nerve growth factor, and this knowledge gives us hope that a new therapy, which includes rhNGF, could produce a more durable treatment effect. Our aim is to potentially free some patients from the burden of continuous treatment," said Flavio Mantelli, MD, PhD, a cornea specialist and Chief Medical Officer of Dompé.

"We are pleased to be part of the team involved in the NGF0118 Dry Eye Disease study," said Dr. Melissa Toyos, MD, Partner and Research Director at the Toyos Clinic and an investigator in the trial. "Dry eye can be challenging to diagnose and manage because it is multifactorial, and the signs and symptoms vary considerably from patient to patient. We are excited to be partnering with Dompé to explore a treatment that may address the underlying tear production deficiency and the neuro-sensory abnormalities, which could result in a disease modifying therapy."

This study will enroll eligible adult patients with moderate to severe DED, which is diagnosed through a variety of tests related to ocular surface health, objective measures of dryness, and patient reported ocular comfort.

The primary endpoint for the current study is the change from baseline in ocular surface tear wetting measured via Schirmer I test. Key secondary endpoints include signs and symptoms of DED, including ocular surface staining, which is an objective measure of ocular surface integrity and health.

The study design was informed by a Phase 2a, prospective open label, multiple-dose, efficacy and safety study of rhNGF eye drops in a cohort of patients with DED. The study was supported by the company and was conducted at the General Hospital of Vienna (Austria), and the results were recently published in the British Journal of Ophthalmology (BJO).

The National Eye Institute (NEI) estimates that DED affects millions of adults in the United States. The risk of developing DED increases with age and is shown to have a higher prevalence in women than in men.2 Current treatments for DED work to lubricate the exposed epithelium with tear substitutes and to control ocular surface inflammation. Recent studies have shown multifaceted degradation of patients' quality of life and visual performance in relation to DED. Patients with DED lack therapies that target the etiology and pathophysiology of DED.

Dry Eye Disease (DED) is a multifactorial disease that affects the ocular surface. Disruption of homeostasis is the unifying characteristic that describes the fundamental process in the development of DED.4 The tear film plays a vital role in providing lubrication and protection to the ocular surface, as well as maintaining a smooth, refractive surface for optimal visual performance. Corneal sensory nerves react to injuries at the ocular surface through producing pain and irritation and by triggering protective reflexes such as blinking and tearing. In addition to tear film, tears contribute to the maintenance of a healthy ocular surface, as they provide growth factors and other nutrients after stimulation by the neuro-secretory reflex. Over the last decade, there has been mounting evidence that neurosensory impairment may be an underlying cause of DED. The aim of DED management is to restore the homeostasis of the ocular surface and tear film, through breaking the vicious cycle of the disease.

Nerve growth factor (NGF), the first identified and most well-known member of the family of neurotrophins, is a key factor in the function of central and peripheral nervous systems, endocrine, immune and visual system. The neuroregenerative potential of NGF was discovered by Nobel-prize winning scientists in the 1950s, and since that time increasing experimental and clinical evidence has shown that NGF is an essential factor for the trophism, sensitivity and healing of the cornea and the conjunctiva.

The potential of the human NGF to become a drug was not fully realized until Dompé's research and development center in L'Aquila, Italy, created a recombinant version of this protein through a unique development process, and subsequent trials demonstrated its safety and effectiveness in neurotrophic keratitis (NK). Nerve growth factor (NGF) acts through specific high-affinity and low-affinity receptors on the lacrimal gland, epithelium, conjunctiva, endothelial cells and corneal nerves and is thought to work by supporting epithelial survival and maintenance, tear production, and corneal innervation.

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