Azura Ophthalmics Announces Positive Results from Phase 2b Clinical Trial of AZR-MD-001 in Meibomian Gland Dysfunction

AZR-MD-001 0.5% meets co-primary efficacy endpoints, making it the first investigational drug to show significant improvements in both signs and symptoms of Meibomian Gland Dysfunction

Restoration of gland function shown to improve downstream response measures of ocular conditions associated with Meibomian Gland Dysfunction

Rapidly advancing AZR-MD-001 to Phase 3 in 2023

TEL AVIV, Israel & MELBOURNE, Australia–(BUSINESS WIRE)–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). 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^�1 (OSDI^©; improved symptoms).

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,” said Marc Gleeson, Chief Executive Officer of Azura. “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,” said Lisa Nijm, M.D., J.D., Founder & Medical Director of Warrenville EyeCare & LASIK. “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

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 Meibomian Gland Dysfunction

Meibomian Gland Dysfunction is a chronic and progressive condition associated with blockage of the meibomian glands and alteration in the quality of expressed meibum which can end in gland atrophy. It is the leading cause of Dry Eye Disease and Contact Lens Discomfort.^4,5 MGD is commonly characterized by terminal duct obstruction and/or qualitative/quantitative changes in the glandular secretion.⁶ There are no approved prescription pharmaceutical agents that specifically treat these glandular changes. If left untreated, MGD will alter the tear film, which can initiate or exacerbate additional ocular surface diseases such as Dry Eye Disease, resulting in corneal ulcers and ocular infections.

About AZR-MD-001

Azura’s lead clinical-stage drug candidate, AZR-MD-001, harnesses the power of selenium sulfide (SeS₂) in an easy-to-use 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 Meibomian Gland Dysfunction 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.

About Azura Ophthalmics, Ltd.

Azura Ophthalmics is utilizing our deep understanding of ocular surface diseases and drug development to deliver a new therapeutic class of Ophthalmic Keratolytics to treat underserved ophthalmic conditions. Our differentiated approach combines ophthalmologic and dermatologic solutions to harness the unique properties of keratolytics to treat the root cause of numerous underserved ocular indications. Our internally discovered pipeline of new chemical entities allows us to develop a portfolio of first-in-class ophthalmic therapeutics for significant unmet needs. For more information visit: www.azuraophthalmics.com and follow Azura on LinkedIn and Twitter.

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References

1. Ocular Surface Disease Index (OSDI) Version 1; © 1995 Allergan, All Rights Reserved.

2. MGD Prevalence: Molinari, J. F., & Stanek, S. (2000). Meibomian gland status comparison between active-duty personnel and US veterans. Military medicine, 165(8), 591-593. Hom, M.M. (1990) Prevalence of meibomian gland dysfunction. Optom Vis Sci, 67(9), 710-712. Blackie C. A. Prevalence of meibomian gland dysfunction – a systematic review and analysis of published evidence. (2019) Investigative Ophthalmology and Visual Science – ARVO Abstract Issue. 60(9).

3. MGD Screening: American Optometric Association; Azura Primary Research

4. Milner, M. S., Beckman, K. A., Luchs, J. I., Allen, Q. B., Awdeh, R. M., Berdahl, J., Boland, T. S., Buznego, C., Gira, J. P., Goldberg, D. F., Goldman, D., Goyal, R. K., Jackson, M. A., Katz, J., Kim, T., Majmudar, P. A., Malhotra, R. P., McDonald, M. B., Rajpal, R. K., Raviv, T., … Yeu, E. (2017). Dysfunctional tear syndrome: dry eye disease and associated tear film disorders – new strategies for diagnosis and treatment. Current opinion in ophthalmology, 27 Suppl 1(Suppl 1), 3–47. https://doi.org/10.1097/01.icu.0000512373.81749.b7.

5. Foulks GN, Bran AJ. Meibomian gland dysfunction: a clinical scheme for description, diagnosis, classification, and grading. Ocul Surf. 2003;1:107-126.

6. Efron N, Jones L, Bron AJ, et al. The TFOS International Workshop on Contact Lens Discomfort: Report of the Contact Lens Interactions With the Ocular Surface and Adnexa Subcommittee. Invest Ophthalmol Vis Sci. 2013;54:TFOS98–TFOS122.

Contacts

Investor:
Ashwin Agarwal

Chief Financial Officer

949-439-1865

Ashwin.agarwal@azuraophthalmics.com

Media:
Tara Mulloy

MacDougall Advisors

978-855-5219

tmulloy@macdougall.bio

Jenna Kane

Health & Commerce

480-388-9587

jennakane@healthandcommerce.com