Alkeus Pharmaceuticals announced positive interim data from its TEASE-3 study. The data demonstrated that additional early-stage Stargardt disease patients treated with oral gildeuretinol acetate showed no disease progression, including stable visual acuity, over multiple years, the company said in a press release.
According to Seemi Khan, MD, MBA, chief medical officer at Alkeus Pharmaceuticals, two additional patients have completed 24 months of treatment in the TEASE-3 study, and the results demonstrated that once-daily oral gildeuretinol prevented progression and vision remained stable with a consistent, well-tolerated safety profile.
“In addition, the initial three participants who completed the study and remained on treatment for multiple years have continued to show no progression with the longest duration of therapy to date of more than 7 years,” said Dr. Khan in the press release. “These are extremely encouraging results that provide increased understanding of gildeuretinol’s potential to preserve sight. It is challenging to identify and enroll early-stage patients before they develop symptoms, and we are grateful to these patients and their families for participating in this study.”
According to the company, TEASE-3, the first clinical trial in early-stage Stargardt disease, is an open-label study of gildeuretinol. Participants have early signs of disease visible on retinal imaging but have not begun experiencing symptoms of vision loss. Fundus autofluorescence imaging and other outcome measures are used to assess the extent to which gildeuretinol affects disease progression. Year-over-year progression is assessed, and the primary endpoint is a measure of progression after the first 2 years of treatment. Following the initial 2-year treatment, patients can continue to receive gildeuretinol as part of an extension study. TEASE-3 has enrolled a total of seven patients to date, the company said.
According to Alkeus Pharmaceuticals, the Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol (ALK-001) in Stargardt disease, denoted as TEASE-1, TEASE-2, TEASE-3 and TEASE-4. The TEASE-1 study was a randomized, double-masked, placebo-controlled trial in 50 patients with Stargardt disease. Gildeuretinol met its prespecified primary efficacy endpoint showing a 21.6% reduction in the growth rate of retinal atrophic lesions area (square root) (p<0.001), and a 29.5% reduction for untransformed areas of retinal atrophic lesions against untreated patients. Gildeuretinol was well-tolerated.
The TEASE-2 trial is an ongoing, fully enrolled, randomized, double-masked, placebo-controlled trial in 80 patients with moderate Stargardt disease, expected to read out topline data in 2025, the company said. TEASE-3 is an open-label study of gildeuretinol in genetically confirmed patients with early signs of disease visible on retinal imaging, but who have not begun experiencing symptoms of vision loss. TEASE-4 is an open-label extension study.