Dompé Doses First Patients in Europe and US in Phase 3 Studyof Isocyclosporin for Atopic Keratoconjunctivitis

Atopic keratoconjunctivitis(AKC) is a chronic inflammatory disease of the eye that is frequently associated with atopic conditions, and limited disease awareness often leads to delayed or missed diagnosis. Isocyclosporin is a novel, dual inhibitor oftwo critical immunepathways in ocular inflammation like AKC.

Dompé, a leading biopharmaceutical company with operations in Italy and the U.S., announced the first patients have been enrolled in a Phase 3 study of Isocyclosporin for the treatment of atopic keratoconjunctivitis (AKC). Isocyclosporin is a dual inhibitor of transient receptor potential (TRP) cation channel, mucolipin subfamily, member 2 (TRPML2) and calcineurin, key regulators of immune and inflammatory response in chronic inflammatory eye diseases. The Phase 3 trial is a multicenter, randomized, double-masked study comparing isocyclosporin against vehicle control in improving ocular signs and symptoms after six weeks of treatment.

“This milestone underscores Dompé’s commitment to groundbreaking science with the potential for life-changing patient impact,” said Marcello Allegretti, Chief Scientific Officer at Dompé. “The dual mechanism of isocyclosporin has the potential to redefine treatment and quality of life for patients living with poorly managed AKC. It also marks an important moment for Dompé as we advance a new asset into late-stage development for a disease with significant unmet need.”

AKC is a severe chronic inflammatory eye disease with no FDA-approved treatment options. If AKC is not properly managed or treated, it can cause scarring or thinning of the cornea, which may lead to blurry vision or even permanent vision loss.¹ In the absence of an FDA-approved treatment, therapies such as topical antihistamines, corticosteroids, immunomodulators, and emerging biologics are used.¹

“Today, patients with AKC are faced with limited treatment options often intended for short-term use due to severe side effects,” said Ahmed Enayetallah, Chief Development Officer at Dompé. “The inability to achieve long-term disease control diminishes day-to-day quality of life and exposes patients to potential vision loss from complications like scarring and ulceration. Isocyclosporin is designed to break this cycle by targeting two key disease pathways for more comprehensive long-term control.”

Previously, in a pivotal Phase 2 study (ISAAK, EU_CT#: 2023-508907-19-00), isocyclosporin provided statistically significant improvements in measures of AKC-associated signs and symptoms after four weeks of treatment in comparison to vehicle control. In the study, there were no discontinuations due to isocyclosporin-related adverse events and no difference in post-installation comfort scores in comparison with vehicle control. A publication detailing the results of this study is currently under development.

About Dompé
Dompé farmaceutici S.p.A. is a privately held, global biopharmaceutical company on a mission to bring the full potential of nerve growth factor (NGF) to patients. As the first company to unlock the therapeutic potential of NGF, Dompé developed the first FDA-approved NGF treatment. Today, our clinical pipeline reflects our commitment to redefining treatments across ophthalmic, neurological and pain-related conditions. Building on 130 years of independence, we are embracing the challenge to transform our science and ourselves by delivering first-in-class, disease-modifying therapies through NGF and other breakthrough molecules to help people improve their sight, support neurological recovery and manage pain effectively. Today, Dompé employs more than 950 employees worldwide and maintains a US commercial operations hub in the San Francisco Bay Area.

Learn more at https://www.dompe.com/us/.

About Isocyclosporin
Isocyclosporin is a novel, dual inhibitor of TRPML2, a key mediator of immune cell activation, and calcineurin, which regulates T-cell mediated immune responses. Both pathways play key roles in the immune and inflammatory response in eye disease. Long-term treatment with isocyclosporin in AKC is intended to control the inflammatory reaction, reducing symptoms and complications and sparing topical steroid use, which carries the risk of toxic side effects like glaucoma and cataracts. Additionally, isocyclosporin is designed for enhanced ocular solubility, to improve bioavailability, distribution and tolerability.

About Atopic Keratoconjunctivitis
AKC is typically caused by an overactive, chronic, inflammatory reaction in the eyes. It often occurs alongside conditions like eczema or asthma (atopy). In people with AKC the body’s immune system becomes overly sensitive to everyday allergens – like dust or pollen – leading to long-term inflammation and irritation of the eyes. Hallmark symptoms of AKC include severe itching, redness, and eye discomfort. It can also make the eyes more vulnerable to infections and chronic pain or discomfort. The persistent and debilitating symptoms of AKC can significantly disrupt daily activities and lead to emotional distress, reducing overall quality of life. The risk of vision loss and infection makes proper diagnosis and treatment crucial. Due to limited awareness, AKC is often misdiagnosed as other forms of allergic eye disease or less severe diseases that affect the surface of the eye, which can lead to delayed or improper treatment.

References:

1. Chen JJ, Applebaum DS, Sun GS, Pflugfelder SC. Atopic keratoconjunctivitis: A review. J Am Acad Dermatol. 2014 Mar;70(3):569-575. doi:10.1016/j.jaad.2013.10.036

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