Credit: Vitreo Retinal Associates
Gildeuretinol (ALK-001), an innovative investigational oral therapy developed by Alkeus Pharmaceuticals, has attained prestigious Rare Pediatric Disease and Fast Track designations from the US Food and Drug Administration (FDA), paving the way for its use in treating the debilitating Stargardt disease, a genetic disorder that significantly affects vision in children and young adults.1
On November 18, 2024, Alkeus Pharmaceuticals made headlines by announcing their intention to pursue a Priority Review voucher in conjunction with the submission of their New Drug Application (NDA) for gildeuretinol, specifically targeting patients suffering from Stargardt disease.
Gildeuretinol functions by specifically aiming to reduce the dimerization of vitamin A without interfering with the crucial visual cycle, thereby offering a potential new avenue for treating this challenging condition.2 Recent findings from the randomized, placebo-controlled, double-masked TEASE-1 trial revealed a clinically and statistically significant slowing in the growth of retinal lesions after two years of treatment with gildeuretinol in patients with late-stage Stargardt disease.
During the 128th Annual American Academy of Ophthalmology (AAO) Meeting, data from the TEASE-1 trial highlighted that gildeuretinol effectively reduced the growth rate of atrophic retinal lesions by an impressive 21.6% when compared to individuals who received no treatment.3 Specifically, the growth rates of atrophic retinal lesions were measured at 0.18 mm/year in patients treated with gildeuretinol versus 0.23 mm/year in the untreated group, representing a mean difference of 0.05 mm/year (95% CI, 0.03 to 0.07, P).
According to interim data from the TEASE-3 study, which included patients with early-stage Stargardt disease, those treated with gildeuretinol exhibited no progression of their condition and remained symptom-free over a period of 2 to 6 years.1 Notably, visual acuity remained stable among early-stage Stargardt patients receiving gildeuretinol, indicating the treatment’s promising potential in early intervention.
“TEASE-1 is the first randomized, controlled trial in Stargardt disease that has shown an efficacy endpoint, which is very exciting,” said Dr. Christine N. Kay, an investigator affiliated with Vitreo Retinal Associates, expressing her enthusiasm for the findings in light of the challenges faced by patients with this inherited retinal disease.1 “In addition, the TEASE-3 data underscore the importance of early treatment in preventing the onset of progressive central vision loss.”
“Together, these achievements recognize the significant unmet medical need in Stargardt disease and the overwhelming burden on patients as well as their families and caregivers,” emphasized Dahan, highlighting the profound impact of the condition.1
For more insight into the TEASE trials, watch a full HCPLive interview featuring Kay, where she discusses the promising impact of gildeuretinol for Stargardt at the AAO 2024 event.3
“It was exciting to be able to present the first randomized, controlled trial demonstrating efficacy in Stargardt,” Kay remarked to HCPLive.3 “This drug has orphan drug status and breakthrough designation from the FDA, and the company is moving forward with the FDA pathway shortly. We’re eager to follow along and see how things progress.”
References
- Alkeus Pharmaceuticals receives FDA rare pediatric disease and fast track designations for Gildeuretinol as a treatment for stargardt disease. Alkeus Pharmaceuticals Inc. November 18, 2024. Accessed November 22, 2024. https://alkeuspharma.com/alkeus-pharmaceuticals-receives-fda-rare-pediatric-disease-and-fast-track-designations-for-gildeuretinol-as-a-treatment-for-stargardt-disease/.
- Iapoce C. Gildeuretinol shows promise in slowing geographic atrophy progression. HCP Live. October 21, 2024. Accessed November 22, 2024. https://www.hcplive.com/view/gildeuretinol-shows-promise-in-slowing-geographic-atrophy-progression.
- Iapoce C. Christine N. Kay, MD: Tease trials show promise of gildeuretinol for stargardt. HCP Live. October 28, 2024. Accessed November 22, 2024. https://www.hcplive.com/view/christine-n-kay-md-tease-trials-show-promise-of-gildeuretinol-for-stargardt.
- Kohli P, Tripathy K, Kaur K. Stargardt Disease. [Updated 2024 Jan 8]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from:
### Interview with Dr. Christine N. Kay on Gildeuretinol and its Impact on Stargardt Disease
### Interview with Dr. Christine N. Kay on Gildeuretinol and its Impact on Stargardt Disease
**Interviewer:** Thank you for joining us today, Dr. Kay. We are excited to discuss the latest findings regarding gildeuretinol (ALK-001) and its development for treating Stargardt disease. Can you start by telling us what gildeuretinol is and why it’s significant for patients with this condition?
**Dr. Kay:** Thank you for having me! Gildeuretinol is an innovative oral therapy developed by Alkeus Pharmaceuticals that targets Stargardt disease, a genetic disorder that leads to vision loss in children and young adults. It has recently received both Rare Pediatric Disease and Fast Track designations from the FDA, which highlight its potential importance in addressing a significant unmet medical need. Gildeuretinol works by reducing the dimerization of vitamin A, which is crucial for the visual cycle, thereby aiming to slow down retinal damage without adversely affecting vision.
**Interviewer:** Very interesting! The recent TEASE-1 trial showed promising results regarding the slowing of retinal lesion growth. Could you elaborate on those findings?
**Dr. Kay:** Absolutely! The TEASE-1 trial, which is a randomized, placebo-controlled study, revealed that gildeuretinol led to a significant reduction in the growth of atrophic retinal lesions in patients with late-stage Stargardt disease. Specifically, we observed a reduction in the growth rate by 21.6% compared to those who did not receive treatment. This was measured over two years, with treated patients showing an average growth rate of 0.18 mm/year versus 0.23 mm/year in the untreated cohort.
**Interviewer:** That’s a notable difference. How does this relate to the interim findings from the TEASE-3 trial, especially with early-stage patients?
**Dr. Kay:** The TEASE-3 trial had very encouraging interim results as well. It included patients with early-stage Stargardt disease, and we found that those treated with gildeuretinol showed no progression of their condition over 2 to 6 years, maintaining stable visual acuity. This highlights the potential for early intervention to prevent the onset of progressive vision loss, which is crucial in managing this devastating disease.
**Interviewer:** What does the future hold for gildeuretinol and the prospects for patients suffering from Stargardt disease?
**Dr. Kay:** We are optimistic about the future. Alkeus Pharmaceuticals is actively working on submitting a New Drug Application (NDA) along with a Priority Review voucher to expedite the approval process. Achievements like the ones from the TEASE trials not only underscore the serious burden of Stargardt disease but also signal hope for patients and their families. The goal is to provide a viable treatment option that could significantly improve the quality of life for those affected.
**Interviewer:** Dr. Kay, what message would you like to send to families affected by Stargardt disease?
**Dr. Kay:** I understand how difficult it can be for families affected by Stargardt disease. It’s crucial to remain hopeful as new research and treatments are on the horizon. Gildeuretinol represents a promising advance, and we are committed to finding solutions for this community. I encourage families to stay engaged with their healthcare providers for current information and support.
**Interviewer:** Thank you, Dr. Kay, for sharing this insightful information with us today. We look forward to seeing how gildeuretinol progresses through the regulatory process.
**Dr. Kay:** Thank you! It has been a pleasure discussing this important topic with you.
