Alkeus Pharmaceuticals Announces Positive Interim Results Demonstrating No Signs of Disease Progression in Early-Stage Stargardt Disease Patients Treated with Gildeuretinol
May 08 2024 - 9:00AM
Alkeus Pharmaceuticals, Inc. today announced the presentation of
positive interim data from its TEASE-3 study demonstrating that
early-stage Stargardt disease patients treated with gildeuretinol
acetate showed no disease progression and remained asymptomatic for
the duration of therapy ranging between two and six years. Results
were presented during the Association for Research in Vision and
Ophthalmology (ARVO) 2024 Annual Meeting being held May 5-9 in
Seattle.
“These exciting results demonstrate the potential of
gildeuretinol acetate to prevent vision loss in early-stage
Stargardt patients,” said Leonide Saad, Ph.D., President and CEO of
Alkeus Pharmaceuticals. “We projected that without treatment,
patients in the TEASE-3 study would lose vision, following a
similar trajectory of vision loss as their previously-diagnosed
siblings with identical ABCA4 genetic mutations. All of us working
in our growing Alkeus team are committed to advancing research and
having an impact on patients living with vision loss. We look
forward to submitting our gildeuretinol data for regulatory review
later this year.”
TEASE-3, the first clinical trial in early-stage Stargardt
disease, 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. Retinal imaging and functional outcome measures were used to
assess disease progression. To date the TEASE-3 study has enrolled
a total of five patients, who receive gildeuretinol as a once-a-day
pill.
“Interim results of the TEASE-3 trial demonstrating clinical
benefit of gildeuretinol in early-stage Stargardt disease patients
are extremely promising,” said Michael B. Gorin, M.D., Ph.D., of
the Departments of Ophthalmology and of Human Genetics at the David
Geffen School of Medicine at UCLA. “The potential of gildeuretinol
to significantly slow disease progression and delay vision loss in
young patients is an exciting advancement.”
Stargardt disease is a serious cause of blindness in children
and young adults, with an estimated 30,000 people affected in the
U.S. and more than 150,000 worldwide. There is no approved
treatment. In individuals with Stargardt disease, the ABCA4 protein
is defective. This defect in the protein results in the accelerated
dimerization of vitamin A, forming toxic by-products that
irreversibly damage the retina, resulting in progressive vision
loss.
About the TEASE Trials
The TEASE trials consist of four clinical studies of
gildeuretinol acetate (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% reduction in the growth rate of
retinal atrophic lesions (p<0.001, square root units, 28%
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
Stargardt disease, expected to read out topline data in 2025.
TEASE-3 is an open-label study designed to assess gildeuretinol in
early-stage Stargardt patients. TEASE-4 is an open-label extension
study.
About Gildeuretinol Acetate (ALK-001)
Gildeuretinol acetate (ALK-001) is a novel molecule created as a
specialized form of deuterated vitamin A designed to reduce the
dimerization of vitamin A without disrupting vision. In preclinical
studies, gildeuretinol decreased vitamin A dimerization to the
normal rate seen in unaffected individuals and prevented retinal
degeneration and loss of visual function in animals with Stargardt
disease. Gildeuretinol has received breakthrough therapy
designation and orphan drug designation from the U.S. Food and Drug
Administration. In addition to the TEASE trials, a Phase 3 (SAGA)
study of gildeuretinol in 200 patients with geographic atrophy (GA)
secondary to age-related macular degeneration (AMD) is expected to
read out topline data in 2024.
About Alkeus Pharmaceuticals
Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical
company with headquarters in Cambridge, Mass., backed by
institutional investors led by Bain Capital Life Sciences. Founded
in 2010, Alkeus is developing therapies for serious diseases of the
eye with high unmet need. Alkeus’ breakthrough-designated lead
candidate, gildeuretinol acetate (ALK-001), is currently being
evaluated in clinical trials for the treatment of Stargardt disease
and for geographic atrophy (GA) secondary to age-related macular
degeneration (AMD).
For further information, please contact:
Media RelationsMedia01@alkeuspharma.comWebsite:
www.alkeuspharma.com