Azura Ophthalmics Announces Positive Topline Results From Phase 2 Program of Investigational Treatment for MGD

Azura Ophthalmics announced topline results from a phase 2 program evaluating the company’s investigational therapy for the treatment of Meibomian gland dysfunction (MGD). AZR-MD-001 met its primary endpoints showing improvements in signs and symptoms of MGD, reaching statistical significance compared to control. Full data will be presented at upcoming scientific meetings.

“We are thrilled by the positive results showing a statistically significant and clinically meaningful improvement in the signs and symptoms of Meibomian gland dysfunction, which validates our multi-mechanism of action and suggests that AZR-MD-001 has the potential to be a first-in-class treatment option for patients with Meibomian gland dysfunction,” Marc Gleeson, CEO of Azura, said in a company news release. “The study findings also provide insight into the target populations, appropriate dosing and endpoints for our phase 3 program.”

Research shows that MGD is the main cause of dry eye disease (DED). Current treatment options for DED are primarily focused on treating inflammation, which only accounts for a minority of the DED population. Azura’s lead compound, AZR-MD-001, is designed to restore Meibomian gland function by addressing the abnormal hyperkeratinization that blocks the glands, alters the quality of the oil and prevents the secretion of lipids into the tears. There are currently no approved medicines for the treatment of MGD.

“Though there are approved treatments for dry eye disease, they are only appropriate for the patients where inflammation is the cause of the disease,” Edward Holland, MD, Director, Cornea Services/Professor of Clinical Ophthalmology, Cincinnati Eye Institute/University of Cincinnati, said in the news release. “Research shows that 86% of people with dry eye disease exhibit signs of Meibomian gland dysfunction[1.] By addressing the underlying cause of Meibomian gland dysfunction that leads to dry eye, Azura is taking an entirely different approach to the disease that has the potential to benefit a much broader population of patients.”

About the Phase 2 Program

The program was a multicenter, double-masked, vehicle-controlled integrated analyses of four phase 2 studies that evaluated the safety and efficacy of AZR-MD-001 (0.1%, 0.5% and 1.0%) in 95 patients with MGD. Patients were dosed twice weekly at night time. Primary endpoints included patient-reported symptoms as measured by the Ocular Surface Disease Index (OSDI) score, the quality of fluid secretion as measured by the Meibomian Gland Score (MGS) and the number of glands secreting meibum as measured by the Meibomian Glands Yielding Liquid Secretion (MGYLS) score.

There was a statistically significant and clinically meaningful reduction of symptoms as measured by a reduction in OSDI score in the two highest doses tested (0.5% and 1.0%) compared to baseline and control:

• Statistically significant improvements from baseline were observed in both the 0.5% and 1.0% dose groups (P<0.01) with significant improvements over control observed by Month 3 in the 0.5% group (P<0.05)
• Up to 58% of patients became non-symptomatic (score of <13) after 3 months of treatment compared to 16% of patients in the control arm (P<0.05)

Similar trends were observed in the secondary endpoint of patient-reported symptoms as measured by the Standardized Patient Evaluation of Eye Dryness (SPEED) Questionnaire. Results demonstrated a statistically significant difference from baseline and control in the 1.0% dose group (P<0.05) and a statistically significant change from baseline in the 0.5% dose group (P<0.05) and compared to control (P<0.08).

Results also indicated a clear dose-response in the number of glands secreting meibum as measured by a statistically significant improvement in MGYLS score in the two highest doses tested (0.5%, 1.0%) compared to baseline and control:

• Statistically significant increases from baseline were observed in both the 0.5% and 1.0% dose groups (P<0.01) with significant improvements over control observed as early as Month 1 with 1.0% (P<0.01) and continuing to Month 3 (p<0.01)
• Up to 46% of patients achieved a clinically meaningful increase in MGYLS compared to 8% of patients in the control arm (P<0.01)

Additionally, results further indicated a clear dose-response in the quality of meibum secreted as measured by a statistically significant improvement in MGS in both dose groups (0.5%, 1.0%) compared to baseline and in the highest dose tested (1.0%) compared to control:

• Statistically significant increases from baseline were observed in both the 0.5% and 1.0% dose groups (P<0.01) with significant improvements over control observed as early as Month 2 with 1.0% (P<0.05) and continuing to Month 3 (P<0.01)
• Up to 59% of patients returned to a normal range (score of >12) after 3 months of treatment compared to 31% of patients on control (P<0.05)

AZR-MD-001 was well tolerated with no serious ocular treatment emergent adverse events (TEAEs). The most common TEAEs were application site irritation, stinging upon application and watery eyes, which only occurred in patients in the 1.0% dose group.