Dompé Announces First Patient Enrollment in Phase 2b Clinical Trial Investigating Novel Mechanism of Action in Moderate to Severe Dry Eye Disease

-- Following the successful launch of Oxervate, the first-in-class recombinant human nerve growth factor (rhNGF) for treatment of neurotrophic keratitis (NK), Dompé embarks on a new clinical trial program to further investigate the relevance of treatment with rhNGF in Dry Eye Disease (DED).

-- The Dry Eye Disease study will evaluate rhNGF in select sub-populations of DED, consistent with the company's commitment to personalized medicine and addressing unmet medical needs of patients.

MILAN, Italy and SAN MATEO, CA, USA I June 25, 2019 I Dompé Farmaceutici S.p.A and Dompé U.S. Inc., (collectively Dompé) announced today that 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).1

For additional information about the NGF0118 study and to learn more about eligibility, find more information at ClinialTrials.gov on the study page (NCT03982368).

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.3 Patients with DED lack therapies that target the etiology and pathophysiology of DED.

About Dompé 
Dompé is a private, rapidly scaling global biopharmaceutical company founded in Milan, Italy, with a 165-year legacy of medical innovation. Today, Dompé employs more than 700 employees worldwide and maintains a U.S. commercial operations hub in the San Francisco Bay Area as well as an R&D presence in Boston.

About Dry Eye Disease
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.5 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.6,7 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.7 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.4

About Recombinant Human Nerve Growth Factor (rhNGF)
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. 1 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.1

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.7,8,9

Important Safety Information

Oxervate is a recombinant human nerve growth factor indicated for the treatment of neurotrophic keratitis.

Contact lenses should be removed before applying OXERVATE because the presence of a contact lens (either therapeutic or corrective) could theoretically limit the distribution of cenegermin-bkbj onto the area of the corneal lesion. Lenses may be reinserted 15 minutes after administration.

Oxervate may cause mild to moderate eye discomfort such as eye pain during treatment. The patient should be advised to contact their doctor if a more serious eye reaction occurs.

Other adverse reactions occurring in 1-10% of Oxervate patients and more frequently than in the vehicle-treated patients included corneal deposits, foreign body sensation, ocular hyperemia, ocular inflammation and tearing.

The full prescribing information can be found at www.oxervate.com

Forward Looking Statements 
This press release refers 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.

References

1 Sacchetti M, Lambiase A, Schmidl D, et al Effect of recombinant human nerve growth factor eye drops in patients with dry eye: a phase IIa, open label, multiple-dose study British Journal of Ophthalmology Published Online First: 03 April 2019. doi: 10.1136/bjophthalmol-2018-312470.

2 https://nei.nih.gov/health/dryeye/dryeye

3 Uchino M, Schaumberg DA. Dry Eye Disease: Impact on Quality of Life and Vision. Curr Ophthalmol Rep.; 1(2):51–57. doi:10.1007/s40135-013-0009-1.

4 Craig JP, et al., TFOS DEWS II Report Executive Summary, The Ocular Surface (2017), http://dx.doi.org/10.1016/ j.jtos.2017.08.003.

5 Willcox MDP, Argüeso P, Georgiev G, Holopainen J, Laurie G, Millar T, et al. TFOS DEWS II Tear Film report. Ocul Surf 2017;15:366e403.

6 Pellegrini JJ, Horn AK, Evinger C. The trigeminally evoked blink reflex. I. Neuronal circuits. Exp Brain Res. 1995;107(2):166-80.

7 Mastropasqua L, Massaro-Giordano G, Nubile M, Sacchetti M. Understanding the Pathogenesis of Neurotrophic Keratitis: The Role of Corneal Nerves. J Cell Physiol. 2017 Apr;232(4):717-724. doi: 10.1002/jcp.25623.

8 Sacchetti M, Lambiase A. Diagnosis and management of neurotrophic keratitis. Clin Ophthalm. 2014 Mar;8:571-579.

9 Müller LJ, Marfurt CF, Kruse F, et al. Corneal nerves: structure, contents and function. Exp Eye Res. 2003 May;76(5):521-542.

SOURCE: Dompé

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