New Insight: A Look at Gildeuretinol (ALK-001) for Stargardt Disease w/ Seemi Khan, MD


In this episode, Veeral Sheth, MD speaks with Alkeus Pharmaceutical's CMO on the company's lead candidate for Stargardt disease and the TEASE clinical trial program.

Episode Highlights

0:07 Episode Intro

0:57 Seemi Khan's background

4:38 Mechanism of action of gildeuretinol

7:22 Patient populations

9:00 TEASE clinical trial program

14:37 Gildeuretinol in GA

16:32 Safety data

18:47 Next steps for Alkeus

22:28 Conclusion

Recent years have proven the robustness of the pipeline in ophthalmology, and what’s coming down the pike could be some of the most impressive agents ever introduced to eye care.

In the latest episode of New Insight with Veeral Sheth, MD, host Veeral Sheth, MD, director of clinical research at University Retina, speaks with Seemi Khan, chief medical officer at Alkeus Pharmaceuticals, on the company’s lead candidate being evaluated in clinical trials for the treatment of Stargardt disease and geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

Stargardt disease affects nearly 30,000 people in the US and more than 150,000 worldwide, with no currently approved therapy. The ABCA4 protein is defective in Stargardt disease and leads to the dimerization of vitamin A, forming toxic byproducts that damage the retina.

Gildeuretinol is a novel molecule designed as a specialized form of deuterated vitamin A to reduce the dimerization of vitamin A without disrupting vision. The therapy has received Breakthrough and Orphan Drug designations from the US Food and Drug Administration (FDA).

The TEASE clinical trial program evaluating gildeuretinol consists of four clinical trials: TEASE-1, TEASE-2, TEASE-3, and TEASE-4. In the randomized, double-masked, placebo-controlled TEASE-1 trial, gildeuretinol was well-tolerated and achieved a 21% reduction in the growth of retinal atrophic lesions (P <.001).

Meanwhile, interim data from the open-label TEASE-3 study revealed that 50 patients with early-stage Stargardt disease exhibited no progression and remained asymptomatic after gildeuretinol treatment between two and six years.

The ongoing, randomized TEASE-2 trial is fully enrolled with 80 patients and is expected to read out topline data in 2025. TEASE-4 is an open-label extension study. A Phase 3 study of gildeuretinol in 200 patients with GA secondary to AMD is also expected to read out topline data in 2024.

Listen to the conversation between experts on Khan’s background in the pharmaceutical industry, including time spent at Abbott, Abbvie, and Quark Pharmaceuticals, her experience bringing treatments to patients with rare diseases, and the impressive data from gildeuretinol in the TEASE clinical trial program for a currently unmet need.

For more information on Stargardt disease, including patient support resources, visit Subscribe to the HCPLive YouTube channel and find us on Spotify and Apple Podcasts for more podcast content.

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