Azura Ophthalmics reveals positive results from phase 2b clinical trial of AZR-MD-001 for meibomian gland dysfunction

AZR-MD-001 is thought to have a multi-modal mechanism of action that treats the pathophysiology of MGD along with the resulting ocular surface symptoms.

Azura Ophthalmics Ltd. a clinical-stage biopharmaceutical company developing a new therapeutic class of ophthalmic keratolytics for ocular surface diseases, today announced positive 3-month efficacy and safety results from its Phase 2b study of AZR-MD-001 0.5% in meibomian gland dysfunction (MGD).

According to the company, the trial met its co-primary endpoints of improvements in Meibomian Glands Yielding Liquid Secretion (MGYLS; number of open glands) and Ocular Surface Disease Index (OSDI; improved symptoms).

The company noted in its news release that the trial also met additional clinically meaningful endpoints, including improvements in meibum quality (measured by MGS), improvements in tear stability (measured by tear break up time) and improvements across multiple patient-reported outcome measures (SPEED and average VAS). AZR-MD-001 was safe and well tolerated in this study.

“At Azura, we are taking a completely new approach to treating MGD which is the root cause of many downstream ocular surface conditions,” Marc Gleeson, CEO of Azura, said in the news release. “These data clearly demonstrate consistency in efficacy across multiple sign and symptom endpoints. With as little as four applications of AZR-MD-001, improvement in glandular function was observed and continued to improve over three months. We are thrilled to build upon these positive results by advancing AZR-MD-001 to a pivotal Phase 3 clinical trial for MGD in 2023.”

MGD is a chronic condition characterized by abnormal keratin production which leads to blocked glands and impacts the quality and quantity of meibum secretions in the upper and lower eyelids. It leads to inflammatory ocular surface conditions, ocular surface dryness, pain, irritation, and reduced quality of vision. Approximately 30-40 million people are diagnosed with MGD in the United States,2,3 with the total prevalent population estimated at 100 million Americans.2,3

“From a clinical perspective, it’s incredibly encouraging to see that AZR-MD-001 0.5% achieved improvements in both the signs and symptoms,” Lisa Nijm, MD, JD, Founder & Medical Director of Warrenville EyeCare & LASIK, said in the news release. “These positive data offer the opportunity for us as physicians to address MGD in a completely new way and bring relief to countless patients who are burdened by it and associated ocular surface conditions. I look forward to collaborating with the Azura team and my fellow scientific advisory board members to advance AZR-MD-001.”

About the Phase 2b 3-Month Results

According to the company, the Phase 2b trial was a multi-center, double-masked, vehicle-controlled, parallel group study that evaluated the safety and efficacy of AZR-MD-001 in 245 patients with MGD. Patients administered AZR-MD-001 twice weekly to the lower eyelid at bedtime. The prospectively defined co-primary efficacy endpoints included the number of glands secreting meibum as measured by the Meibomian Glands Yielding Liquid Secretion (MGYLS) score and patient-reported symptoms as measured by the Ocular Surface Disease Index (OSDI) score.

AZR-MD-001 0.5% achieved statistically significant differences compared to vehicle in both signs and symptoms at month 3:

• Co-primary
• Significant improvements in MGYLS score, with patients experiencing an average increase of 1.8 more open glands secreting meibum from baseline (p = 0.0004).
• Significant improvements in OSDI score, with patients reporting an average improvement of 3.5 from baseline (p = 0.0438).
• Key Secondary
• 46.9% of patients became asymptomatic as measured by Total OSDI© responder rate.
• 45.7% of patients had at least 5 more glands opened from baseline of 1.7, as measured by MGYLS responder rate.
• 68.7% of patients had their meibum quality return to normal levels, as measured by MGS responder rate.

Additional patient-reported outcomes, using well established questionnaires, also demonstrated statistically significant improvements for AZR-MD-001 0.5% from baseline:

• Significant improvements in SPEED (p<0.0001) and Average VAS (visual analogue scale) (p<0.0001).
• Significant improvements in Eye Discomfort (p<0.0001), Eye Dryness (p<0.0001) and Ocular Itch (p<0.0001).

The majority of adverse events (AEs) were mild and transient with no serious treatment-related AEs. The number of subjects with treatment emergent AEs leading to discontinuation was 2.4% for AZR-MD-001 0.5%.

About AZR-MD-001

Azura’s lead clinical-stage drug candidate, AZR-MD-001, harnesses the power of selenium sulfide (SeS2) in an ophthalmic ointment preparation applied directly to the meibomian glands in the lower eyelid. AZR-MD-001 is thought to have a multi-modal mechanism of action that treats the pathophysiology of MGD along with the resulting ocular surface symptoms. It breaks down the bonds between abnormal keratin proteins to soften the blockage, slows down the production of keratin to prevent future blockages and increases the quality and quantity of meibum produced by the meibomian glands.

AZR-MD-001 is currently being studied to evaluate the safety, efficacy, and tolerability of the study drug in patients with MGD. Azura expects to initiate a second pivotal multi-center clinical trial of AZR-MD-001 0.5% in 2023.