A milestone in Stargardt juvenile macular degeneration treatment

Denver, Colo.—Stargardt disease is a rare inherited eye disease that affects over 40,000 Americans.  Usually, those afflicted with Stargardt disease start experiencing vision loss in childhood and those that don’t undergo any treatment become blind by age 50. One study presented this week at the 2022 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Denver, Colo. examines the safety and effectiveness of ALK-001 (C20-D3-Vitamin A) in its capability to delay or avert vision loss in Stargardt patients.

Hendrik P. Scholl, MD, MA, FARVO, and researchers from the Universität Basel Medizinische Fakultät and Institute of Molecular and Clinical Ophthalmology Basel in Switzerland and the Alkeus Pharmaceuticals and Columbia University Irving Medical Center in the U.S., were part of the Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE) study. This was a two-year multicenter Phase 2 double-masked, randomized, and placebo-controlled clinical trial studying the investigational drug, ALK-001, and its effect on 50 patients with Stargardt Disease (STGD1) and “a well-delineated area of” retinal pigment epithelium (RPE). ALK-001 is a chemically altered vitamin A that blocks “the formation of toxic vitamin A clumps in the eye” and is taken as a daily pill. Vitamin A is an essential nutrient for vision, but during its processing by the eye, it can accumulate into toxic deposits. These toxic clumps build up rapidly in patients with STGD1, leading to retinal atrophy and eventually vision loss. ALK-001 might be a vitamin A replacement, but functions differently because it does not form clumps.

The 50 patients from seven centers in the U.S., age ranges from 18-60, were randomized and given a placebo during the first year then 50% of the placebo patients were randomly crossed over to ALK-001 in the second year of treatment. 74% of the cases had bilateral atrophic lesions with ~5 mm² median area. Scholl and his team found that ALK-001 slowed the growth rate of retinal atrophy by approximately 30% per year. Patients also reported back that they tolerated ALK-001 well and with no harsh reactions.

“The study showed that ALK-001 slowed the progression of Stargardt disease in its advanced stage, where many believed any slowing was nearly impossible,” said Dr. Scholl. “We are extremely excited to see such a positive effect in advanced-stage patients and look forward to studying the drug’s effects in earlier stages of Stargardt, where an even larger treatment effect could be expected." In addition, “the TEASE study is the only trail with both a statistically significant and clinically meaningful effect on the progression of Stargardt disease.”

• Abstract title: ALK-001 (C20-D3-Vitamin A) slows the growth of atrophic lesions in ABCA4-related Stargardt Disease: Results of a Phase 2 placebo-controlled clinical trial (TEASE study)
• Presentation start/end time: Sunday, May 1, 12:49 – 1:06pm MT
• Location: Four Seasons Blrm 1 (Denver Convention Center)
• Also available on the virtual meeting site at https://arvo2022.arvo.org/ beginning May 11
• Abstract Number: 38

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The Association for Research in Vision and Ophthalmology (ARVO) is the largest eye and vision research organization in the world. Members include approximately 10,000 eye and vision researchers from over 75 countries. ARVO advances research worldwide into understanding the visual system and preventing, treating and curing its disorders. Learn more at ARVO.org.

The 2022 ARVO Annual Meeting will take place in Denver, Colo. from May 1 – 4 and virtually May 11 - 12. The Meeting is the premiere gathering of nearly 10,000 eye and vision researchers from around the world. During the Meeting, 4,800 abstracts will be presented on the latest basic and translational research in eye and vision science.

All abstracts accepted for presentation at the ARVO Annual Meeting represent previously unpublished data and conclusions. This research may be proprietary or may have been submitted for journal publication. Embargo policy: Journalists must seek approval from the presenter(s) before reporting data from paper or poster presentations. Press releases or stories on information presented at the ARVO Annual Meeting may not be released or published until the following embargo dates:

• May 1: Official launch of presentations of all posters (both presented in-person and virtually)
• Rolling basis: Paper session, Symposia, Minisymposia, Cross-sectional Groups, and invited speaker sessions that have specific presentation times will be embargoed until the end of those individual time slots.

Media contact:
Jenniffer Scherhaufer
1.240.221.2923
media@arvo.org