Alkeus Pharmaceuticals Announces Presentation of a TEASE-3 Study Update Showing Progression Stalled in Early-Stage Stargardt Disease Patients Treated with Gildeuretinol
2024年7月18日 - 11:20PM
Alkeus Pharmaceuticals, Inc. today announced the presentation of
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 while on
therapy ranging between two and six years. The study update was
presented during the 42nd American Society of Retina Specialists
(ASRS) Annual Scientific Meeting, being held July 17-20 in
Stockholm, Sweden.
“Interim results from the TEASE-3 study of gildeuretinol in
early-stage Stargardt patients indicate the potential value of
treating patients with confirmed disease-causing ABCA4 genetic
mutations as early as possible before the disease causes
progressive loss of central vision,” said Seemi Kahn, M.D., M.P.H.,
M.B.A., Chief Medical Officer of Alkeus Pharmaceuticals. “There is
high unmet need in this disease area with no existing treatment,
and we look forward to sharing additional results from this
encouraging study in the future as we work to advance our
development program, which has the potential to be the first
therapy for Stargardt disease.”
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. Each TEASE-3 study participant has a sibling who was
previously diagnosed with Stargardt disease, has identical gene
mutations and has experienced irreversible vision loss. The primary
endpoint of disease progression over two years is assessed by
retinal imaging and functional outcome measures. After the initial
two-year treatment period, patients continue to receive
gildeuretinol while enrolled in an open label long-term extension
study. The TEASE-3 study has enrolled a total of six patients, who
receive gildeuretinol as a once-a-day pill.
“The TEASE-3 study has been a unique opportunity to evaluate
gildeuretinol in a small group of presymptomatic children for whom
we had clinical history information regarding the course of
Stargardt disease in older siblings,” 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. “We were able to
assess the ability of the medication to stall vision loss in these
children as compared to the age-matched data of their
genetically-matched Stargardt siblings. The dramatic delay of
vision and photoreceptor loss in this small cohort is an exciting
advancement in the therapy for ABCA4-related Stargardt disease and
suggests that the clinical benefits may be greater at the earliest
stages of this condition.”
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 Relationsmedia@alkeuspharma.comWebsite:
www.alkeuspharma.com