Azura Ophthalmics Corporate Update: AZR-MD-001 NDA Submission Expected in 2H 2026

—Following a Positive U.S. Food and Drug Administration (FDA) pre-New Drug Application (NDA) Meeting for AZR-MD-001, Company Plans NDA Submission with Completed Efficacy Studies—

—12-month Long Term Safety Data from the ASTRO Study Expected to Read-out in Q1 2026—

—AZR-MD-001 is a Potential First-in-Class Therapy Targeting Meibomian Gland Dysfunction (MGD), the Root Cause of Dry Eye Disease (DED) and Contact Lens Discomfort (CLD)—

TEL AVIV, Israel--(BUSINESS WIRE)--Azura Ophthalmics Ltd., a clinical-stage biopharmaceutical company developing AZR-MD-001, an investigational, potentially first-in-class, Ophthalmic Keratolytic targeting Meibomian Gland Dysfunction (MGD), today announced that it has received positive feedback from a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) confirming that the company’s existing clinical development program for AZR-MD-001 is sufficient to support its planned NDA submission.

“We are very encouraged by the FDA’s feedback and pleased by their determination that our existing clinical data package satisfies the requirements for a sign and symptom in two adequate and well-controlled confirmatory efficacy studies,” said Marc Gleeson, CEO of Azura Ophthalmics. “We believe AZR-MD-001 has the potential to redefine how patients with Meibomian Gland Dysfunction and Dry Eye Disease are treated by directly targeting gland function with a novel, easy-to-administer, twice-weekly ophthalmic ointment. As a company, we are focused on submitting our NDA package in the second half of 2026.”

The FDA confirmed the CELESTIAL and ARIES study data fulfill the regulatory efficacy requirements for approvability of AZR-MD-001 for the treatment of the signs and symptoms of Dry Eye Disease (DED). The results from these two studies demonstrate statistically significant improvement in a sign, Meibomian Glands Yielding Liquid Secretion (MGYLS), and symptomatic benefit in Ocular Surface Disease Index (OSDI^©) in CELESTIAL and increased comfortable contact lens wear time in ARIES.

The FDA indicated that the safety database across CELESTIAL, ARIES, and ASTRO is sufficient for submission, with long-term safety data from the ongoing ASTRO study expected to be shared in Q1 2026, further supporting the application.

The company believes that AZR-MD-001’s robust product profile, including its unique triple mechanism of action, keratolytic, keratostatic, and lipogenic, supports its differentiation from existing DED therapies. AZR-MD-001 is administered directly to the lower eyelid margin twice weekly at bedtime. It is designed to restore meibomian gland function, improve tear stability, and reduce symptoms associated with DED, including Contact Lens Discomfort (CLD).

“We continue to see growing recognition among physicians that AZR-MD-001 addresses meibomian gland obstruction, an important and previously untreated root cause of Dry Eye Disease,” said Dr. Francis Mah, Director of Cornea and External Disease and Co-Director, Refractive Surgery at Scripps Clinic Medical Group, La Jolla, Calif. “A product that can open and restore gland function with a convenient, twice-weekly dose could be practice-changing for millions of patients.”

AZR-MD-001 is an investigational drug candidate and has not been approved by the U.S. FDA. To date, over 500 patients have been treated with AZR-MD-001 in clinical trials, and the product appears to be safe and well-tolerated.

About Meibomian Gland Dysfunction (MGD)

Meibomian Gland Dysfunction (MGD) is a chronic and progressive condition that affects the quality and flow of meibum from the meibomian glands. MGD is a leading cause of Dry Eye Disease (DED). Without treatment, MGD may lead to glandular dropout and increased ocular surface damage, which reduces quality of vision and decreases quality of life. Current therapies for DED primarily focus on inflammation or tear production and do not address the underlying glandular dysfunction that characterizes MGD.

About CELESTIAL^1,2

CELESTIAL, a multicenter, vehicle-controlled, randomized study to evaluate the safety, tolerability, systemic pharmacokinetics, and pharmacodynamics of AZR-MD-001 in patients with Meibomian Gland Dysfunction (MGD) and evaporative Dry Eye Disease (DED) was a parallel group study of AZR-MD-001 (0.5% and 1.0%) and vehicle in a 1:1:1 ratio, dosed twice-weekly in the evening (at bedtime). Patients were followed for a total of 6 months. The pre-specified primary efficacy endpoints were Meibomian Glands Yielding Liquid Secretion (MGYLS) and total Ocular Surface Disease Index (OSDI©) change from baseline to Month 3.

About ARIES³

ARIES was a multi-center, vehicle-controlled study to evaluate the safety, tolerability, and pharmacodynamics of AZR-MD-001 and to determine common symptoms in patients with Dry Eye Disease (DED) characterized by signs of Meibomian Gland Dysfunction (MGD) and an inability to wear their contact lenses as desired. Patients were treated with either AZR-MD-001 0.5% and vehicle in a 1:1 ratio, dosed twice-weekly in the evening (at bedtime). Patients with DED were challenged with Contact Lens Wear and were followed for a total of 3 months. The primary efficacy sign endpoint was Meibomian Glands Yielding Liquid Secretion (MGYLS), and the symptom endpoint was Comfortable Contact Lens Wear Time in hours.

About ASTRO

ASTRO, a phase 3, multicenter, vehicle-controlled, randomized study to evaluate the efficacy, safety, and tolerability of AZR-MD-001 in patients with abnormal meibomian gland function and associated symptoms of Dry Eye Disease (DED), is an ongoing parallel group study of AZR-MD-001 0.5% and vehicle randomized in a 1:1 ratio. The study drug is dosed twice-weekly to the lower eyelid in the evening (at bedtime). The study objective supporting the New Drug Application (NDA) is to evaluate the safety and tolerability of AZR-MD-001 sterile ophthalmic ointment 0.5% or vehicle dosed twice-weekly through Month 12.

About AZR-MD-001

AZR-MD-001 is the first and only Ophthalmic Keratolytic investigational therapy in development as a foundational treatment for Meibomian Gland Dysfunction (MGD), a leading cause of Dry Eye Disease (DED). Delivered as a twice-weekly selenium sulfide ointment applied to the lower eyelid margin, AZR-MD-001 is designed to restore meibomian gland function by improving the quality and quantity of meibum, rejuvenating the lid margins, and alleviating evaporative dry eye symptoms. The therapy’s unique triple mechanism of action, keratolytic, keratostatic, and lipogenic, targets the root cause of MGD.

About Azura Ophthalmics

Azura Ophthalmics (“Azura”) is utilizing its deep understanding of ocular surface diseases and drug development to deliver a potential new therapeutic class of Ophthalmic Keratolytics to treat underserved ophthalmic conditions. The company’s differentiated approach combines ophthalmologic and dermatologic science to harness the unique properties of keratolytics for the treatment of Dry Eye Disease (DED) by targeting Meibomian Gland Dysfunction (MGD) and other ocular diseases. Azura’s pipeline of internally discovered new chemical entities represents a portfolio of potentially first-in-class ophthalmic therapeutics for significant unmet needs. For more information, visit www.azuraophthalmics.com and follow Azura on LinkedIn.

References

1. Watson SL, Jones LW, Stapleton F, Hinds M, Ng A, Tan J, et al. Efficacy and safety of AZR-MD-001 selenium sulfide ophthalmic ointment in adults with meibomian gland dysfunction: A vehicle-controlled, randomized clinical trial. Ocul Surf. 2023 Jul;29:537-46.
2. Downie LE, Craig JP, Stapleton F, Tan J, Jones LW, Ng A, et al. Efficacy and safety of AZR-MD-001 selenium sulfide ophthalmic ointment in adults with meibomian gland dysfunction over six months of treatment: A Phase 2, vehicle-controlled, randomized extension trial. Ocul Surf. 2025 Jan:35:15-24.
3. Stapleton F, Hinds M, Tan J, Jones L, Chalmers R, Bosworth C, Alster Y. AZR-MD-001 0.5% selenium sulfide ophthalmic ointment for the treatment of contact lens discomfort: A vehicle-controlled, randomized, clinical trial. Ocul Surf. 2024 Dec 28:S1542-0124(24)00144-7.