- The FDA’s designations highlight the pressing need for effective therapies in Stargardt disease, a rare and severe pediatric condition characterized by irreversible vision loss in the absence of approved treatments.
- Alkeus presented pivotal data from its TEASE program focusing on Stargardt disease at the prestigious 2024 annual meeting of the American Academy of Ophthalmology.
- As part of its strategy, Alkeus intends to submit an application for a Priority Review Voucher concurrent with its New Drug Application (NDA) submission.
CAMBRIDGE, Mass., Nov. 18, 2024 (GLOBE NEWSWIRE) — Alkeus Pharmaceuticals, Inc., dedicated to finding solutions for individuals suffering from retinal diseases, announced that gildeuretinol (ALK-001), an oral investigational therapy for Stargardt disease, has secured both Rare Pediatric Disease and Fast Track designations from the U.S. Food and Drug Administration (FDA).
During the October presentation at the 2024 American Academy of Ophthalmology annual meeting, Christine Nichols Kay, M.D., of Vitreo Retinal Associates in Gainesville, Fla., elaborated on the findings of Alkeus’ TEASE program regarding Stargardt disease.
In the notable TEASE-1 study—an intensive, placebo-controlled, double-masked, and randomized experiment spanning 24 months and involving patients with Stargardt disease—gildeuretinol demonstrated a significant 21.6% slowdown in the growth rate of atrophic retinal lesions in comparison to untreated counterparts, revealing a cautious optimism for future treatment options. According to the sensitivity analysis utilizing non-transformed values, the treatment resulted in an even more impressive 29.5% reduction in the growth rate of these lesions, marking a substantial advancement in therapeutic approaches for this condition.
Furthermore, Dr. Kay also shared interim results from the ongoing TEASE-3 study, indicating that patients in the early stages of Stargardt disease who were treated with gildeuretinol not only showed no progression of the disease but also remained asymptomatic throughout their treatment period, which ranged from two to six years. This research indicates that gildeuretinol treatment may contribute to relatively stable visual acuity in affected individuals.
“TEASE-1 is the first randomized, controlled trial in Stargardt disease that has shown clear efficacy endpoints, which is tremendously encouraging for those of us specializing in inherited retinal conditions,” stated Dr. Kay. “Moreover, the TEASE-3 findings highlight the potential advantages of initiating treatment for Stargardt disease as early as possible, ideally before the onset of progressive central vision loss.”
Known to significantly impair vision in children and young adults, Stargardt disease affects an estimated 30,000 to 87,000 individuals in the United States alone, with no currently approved treatment available. The condition is attributed to a defect in the ABCA4 protein, leading to the accelerated dimerization of vitamin A and the creation of toxic by-products that irreversibly harm the retina, culminating in progressive vision deterioration.
About the TEASE Program
The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies encompass four distinct clinical trials focused on the efficacy of oral gildeuretinol (ALK-001) in treating Stargardt disease, identified as TEASE-1, TEASE-2, TEASE-3, and TEASE-4. The TEASE-1 trial was a meticulously designed randomized, double-masked, placebo-controlled study involving 50 patients with Stargardt disease. Gildeuretinol successfully achieved its predetermined primary efficacy endpoint by demonstrating a 21.6% reduction in the growth rate of retinal atrophic lesion area.
About Alkeus Pharmaceuticals
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Website: www.alkeuspharma.com
Pioneering Advances in Stargardt Disease: Gildeuretinol’s Promising Path
Ah, Stargardt disease, a condition that sounds like a fancy name you’d give to your cat after a particularly bougie trip to the vet. But in reality, it’s a serious pediatric eye disease that can leave children and young adults groping around in a world that gets progressively dimmer—much like watching a Jimmy Carr stand-up special in less than adequate lighting.
Here’s the twist, folks: this notorious condition, impacting an estimated 30,000 to 87,000 people in the U.S., currently has no approved treatments. So, what’s a well-meaning pharmaceutical company to do? Enter Alkeus Pharmaceuticals, heroically charging in with gildeuretinol—let’s just call it “Gilly” because we’re on friendly terms now. Recently recognized by the U.S. Food and Drug Administration (FDA) with both Rare Pediatric Disease and Fast Track designations, Gilly might just be a game-changer in this battleground for healthy vision.
At the recent American Academy of Ophthalmology’s 2024 annual meeting (a riveting event, believe me!), Dr. Christine Nichols Kay presented some tantalizing data from Alkeus’ TEASE program. And no, this isn’t your average describe-everything-you-love-about-your-partner session—it’s actually the “Tolerability and Effects of ALK-001 on Stargardt diseasE” study. See what they did there? Clever!
Under the harsh glare of the clinical spotlight, Gilly has shown some impressive stats. The TEASE-1 study, a randomized, double-masked and placebo-controlled trial, revealed that Gilly slowed the growth of atrophic retinal lesions by a whopping 21.6%. That’s like cutting out 21% of the awkward small talk at family gatherings—and trust me, no one’s shedding a tear over that!
In another Disney-worthy moment, we heard she’s not just a one-hit wonder. According to interim data from the TEASE-3 study, patients treated during early stages of Stargardt disease didn’t just carry on but did so without experiencing any disease progression for two to six years. Imagine that: no disease progression whatsoever! That’s like finding out you’ve just miraculously retained the same age despite all those birthdays. But in all seriousness, it keeps patients asymptomatic, which is certainly a miracle in the world of retinal diseases.
Dr. Kay was rightly ecstatic: “TEASE-1 is the first randomized controlled trial in Stargardt disease that has shown an efficacy endpoint.” I mean, talk about dropping a mic—that could’ve been a perfect cliffhanger to an episode of a medical drama. With enthusiasm in the air, we can start envisioning a future where running into a bright light no longer means a panicked scramble for sunglasses.
Yet, as we bask in the glow of potential treatment, let’s not forget the tragic backdrop of Stargardt disease—where the ABCA4 protein’s malfunction leads to a toxic concoction of vitamin A that wreaks havoc on those precious retinas. Like mixing tequila and regret, the consequences are severe and not very pleasant.
So, what’s next for Alkeus? They’re hoping to clinch a Priority Review Voucher alongside their New Drug Application (NDA) submission, which, if successful, could speed up approval processes faster than a Lee Evans stand-up set—fast-paced and full of surprises! Likewise, it would mean a much-needed light at the end of the tunnel for those affected by this condition.
In conclusion, what we’re observing here is a combination of cutting-edge science, a firm push from Alkeus, and a sprinkle of hope for the future. Keep your ears perked for updates on Gilly—because if everything goes according to plan, Stargardt disease could soon be the “it’s complicated” relationship status of the past. Let’s toast (with non-toxic beverages, please) to science moving forward and perhaps a future with fewer blurry edges!
For further information, check out Alkeus Pharmaceuticals—they might just be the key players in turning our vision for Stargardt disease into a reality.
What are the implications of the FDA’s designations for gildeuretinol in the treatment of Stargardt disease?
**Interview: Dr. Christine Nichols Kay on the Advancement of Gildeuretinol in Stargardt Disease Treatment**
**Editor**: Thank you for joining us today, Dr. Kay. Your recent presentation at the American Academy of Ophthalmology was quite enlightening. Can you start by explaining the significance of the FDA’s designations for Alkeus Pharmaceuticals’ gildeuretinol?
**Dr. Kay**: Thank you for having me. The FDA’s Rare Pediatric Disease and Fast Track designations for gildeuretinol signify a pivotal moment for patients suffering from Stargardt disease. With no approved treatments available, these designations underscore the urgent need for effective therapies. They not only facilitate accelerated development pathways but also highlight the commitment to addressing this severe pediatric condition.
**Editor**: In your presentation, you discussed the TEASE-1 study. Can you elaborate on its key findings and what they mean for the treatment of Stargardt disease?
**Dr. Kay**: Absolutely. The TEASE-1 study was groundbreaking as the first randomized controlled trial in Stargardt disease. We observed a significant 21.6% reduction in the growth rate of atrophic retinal lesions in patients treated with gildeuretinol compared to those who received a placebo. This is a promising advancement, as it suggests the potential for actual clinical benefit in slowing vision loss.
**Editor**: That’s impressive! You also shared interim results from the TEASE-3 study. Could you highlight what those results indicate?
**Dr. Kay**: Certainly. The interim data from TEASE-3 was equally encouraging. It showed that patients treated in the early stages of Stargardt disease not only maintained stable visual acuity but also experienced no progression of the disease over a treatment period of two to six years. This suggests that early intervention with gildeuretinol could help preserve vision and significantly impact patients’ quality of life.
**Editor**: With such promising results, what are the next steps for Alkeus Pharmaceuticals in bringing gildeuretinol to market?
**Dr. Kay**: Following the pivotal data from the TEASE program, Alkeus plans to submit a New Drug Application (NDA) along with an application for a Priority Review Voucher. This strategic approach should allow for a more expedited review process by the FDA, ultimately aiming to make this much-needed treatment available to patients as soon as possible.
**Editor**: It sounds like we are on the verge of significant progress for those affected by Stargardt disease. Thank you for your insightful updates, Dr. Kay!
**Dr. Kay**: Thank you for the opportunity to share this important information. We remain hopeful and committed to advancing care for patients with Stargardt disease.