FDA feedback supports Azura’s Ophthalmic's planned NDA for AZR-MD-001 in MGD

Azura Ophthalmics announced it has received positive feedback from a pre-New Drug Application (NDA) meeting with the US Food and Drug Administration (FDA) surrounding AZR-MD-001, the company’s ophthalmic keratolytic investigational therapy for meibomian gland dysfunction (MGD).

AZR-MD-001 is administered directly to the lower eyelid margin twice weekly at bedtime and is designed to restore meibomian gland function, improve tear stability, and reduce symptoms associated with dry eye disease (DED), including contact lens discomfort (CLD).

According to the company, feedback from the FDA indicated that the current clinical development program for AZR-MD-001 is sufficient to support the company’s planned NDA submission. The FDA confirmed that both 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 DED.

Marc Gleeson, CEO of Azura Ophthalmics, commented in a press release, saying, “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.”

Additionally, the FDA noted the safety database across CELESTIAL, ARIES, and ASTRO is sufficient for submission. Azura intends to release long-term safety data from the ongoing ASTRO study in Q1 2026.

Data from CELESTIAL²

CELESTIAL (NCT03652051) was a multicenter, vehicle-controlled, randomized study to evaluate the safety, tolerability, systemic pharmacokinetics, and pharmacodynamics of AZR-MD-001 in patients with MGD and evaporative DED. The trial 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 at bedtime.

Data showed that AZR-MD-001 0.5% (n = 82 patients) treatment resulted in significant improvements in Meibomian Glands Yielding Liquid Secretion (MGYLS) score. Patients in the trial experienced an average increase from baseline of 4.2 and 2.4 open glands secreting meibum for the drug and vehicle, respectively (p < 0.001). Additionally, a mean OSDI total score improvement from baseline of 7.3 and 3.8 for the drug and vehicle, respectively (p < 0.05), was seen.

Data from ARES³

ARIES (NCT05548491) was a 2-stage, multi-center, vehicle-controlled study to determine common symptoms in patients with CLD in stage 1 and to evaluate the safety, tolerability, and pharmacodynamics of AZR-MD-001 in stage 2.

In stage 1, up to 12 participants who experienced symptoms of CLD and experienced concomitant meibomian gland dysfunction completed a screening visit followed by a hybrid concept elicitation and cognitive debriefing interview, occurring at the end of the screening visit or up to 14 days later. While stage 2 was a multicenter, single-masked, vehicle-controlled, randomized, parallel group study. Patients were randomly assigned 1:1 to receive either active AZR-MD-001 ointment/semi-solid drug (1.0%) or AZR-MD-001 vehicle for 3 months.

Results from the trial showed that AZR-MD-001 0.5% achieved statistically significant differences compared to the vehicle in both signs and symptoms at month 3. According to results, patients experienced significantly more open glands on AZR-MD-001 than vehicle (5.0 glands vs 1.6 glands, p<0.0001). Additionally, significantly more patients treated with AZR-MD-001 had at least 5 more glands opened, as measured by MGYLS responder rate, when compared to the vehicle (58.2% vs 6.1%, p<0.001). Significantly more patients treated with AZR-MD-001 had their meibum quality return to normal levels as well, as measured by MGS responder rate, when compared to vehicle (97.1% vs. 33.6%, p<0.0001).

ASTRO trial

ASTRO (NCT06329791) is a multicenter, double-masked, vehicle-controlled, randomized, parallel-group study designed to evaluate the treatment of abnormal meibomian gland function and associated symptoms of DED using either AZR-MD-001 0.5% ophthalmic ointment or vehicle. Patients are treated twice weekly at bedtime for up to 12 months. The primary endpoint of the trial is the change from baseline in MGYLS and the change from baseline in total OSDI score.

Approximately 500 patients will be dosed twice weekly at their bedtime for up to 12 months after a screening qualification period and meeting inclusion criteria. Patients will be randomized centrally for treatment in a 1:1 ratio. Study follow-up visits will occur on day 14 and in months 1.5, 3, 4.5, 6, 9, and 12. Patients will then exit the study around 13 months after the baseline visit, with ongoing treatment-emergent adverse events at month 12 being revisited for another 30 days after.⁵

References:

1. Azura Ophthalmics Corporate Update: AZR-MD-001 NDA Submission Expected in 2H 2026. Published December 10, 2025. Accessed December 10, 2025. https://www.businesswire.com/news/home/20251210553083/en/Azura-Ophthalmics-Corporate-Update-AZR-MD-001-NDA-Submission-Expected-in-2H-2026

2. Watson SL, Jones LW, Stapleton F, Hinds M, Ng A, Tan J, Alster Y, Bosworth C, Rafaeli O, DePuy V; CELESTIAL STUDY Group. 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;29:537-546. doi: 10.1016/j.jtos.2023.07.002. Epub 2023 Jul 20. PMID: 37478969.

3. Azura Ophthalmics Announces Positive Results from Phase 2 Clinical Trial of AZR-MD-001 in Patients with Contact Lens Discomfort. Published December 18, 2023. Accessed December 12, 2025. https://www.businesswire.com/news/home/20231218472244/en/Azura-Ophthalmics-Announces-Positive-Results-from-Phase-2-Clinical-Trial-of-AZR-MD-001-in-Patients-with-Contact-Lens-Discomfort

4. Hutton D. Azura Ophthalmics announces positive results from Phase 2 clinical trial of AZR-MD-001 in patients with contact lens discomfort. Published December 18, 2023. Accessed December 12, 2025. https://www.ophthalmologytimes.com/view/azura-ophthalmics-announces-positive-results-from-phase-2-clinical-trial-of-azr-md-001-in-patients-with-contact-lens-discomfort

5. Joy J. Azura Ophthalmics enrolls first patient in Phase 3 trial evaluating AZR-MD-001 in patients with MGD. Published June 9, 2024. Accessed December 12, 2025. https://www.ophthalmologytimes.com/view/azura-ophthalmics-enrolls-first-patient-in-phase-3-trial-evaluating-azr-md-001-in-patients-with-mgd