Stargardt Disease Research Advances

BELITE BIO CONDUCTING PHASE 3 CLINICAL TRIAL OF TREATMENT TO SLOW DISEASE PROGRESSION

The San Diego-based biopharmaceutical company Belite Bio is enrolling adolescent patients (ages 12-18) with Stargardt disease in DRAGON, its Phase 3 clinical trial for Tinlarebant, an emerging oral medication designed to slow disease progression and vision loss. Tinlarebant is designed to inhibit a protein known as retinol binding protein 4 (RBP4) to reduce the uptake of vitamin A to the retina, thereby decreasing the production and accumulation of toxic vitamin A byproducts, which are the hallmark of Stargardt disease.

NANOSCOPE DOSES SIX PATIENTS IN PHASE 2 CLINICAL TRIAL OF OPTOGENETIC THERAPY FOR STARGARDT DISEASE

Nanoscope Therapeutics, a biotechnology company developing gene therapies for retinal degenerative diseases, has dosed six participants in the Phase 2 STARLIGHT clinical trial of its optogenetic therapy for people with advanced Stargardt disease. Some patients had rudimentary vision improvements after receiving the emerging treatment. Known as MCO-010, the optogenetic therapy is designed for people who have lost most or all of their photoreceptors, the cells that make vision possible. The treatment, a small drop of liquid delivered by an intravitreal injection, uses a human-engineered virus to deliver copies of the Multi-Characteristic Opsin (MCO) gene to bipolar cells — cells that don’t normally sense light but often survive after photoreceptors are lost to advanced retinal disease.

ALKEUS REPORTS ENCOURAGING RESULTS IN CLINICAL TRIAL FOR DRUG DESIGNED TO REDUCE TOXINS IN RETINA

The biotech company Alkeus is conducting a multi-center Phase 2 clinical trial for a drug (ALK-001) that targets the toxic build-up in the retina that is thought to cause degeneration and vision loss. The therapy slowed lesion growth by about 30 percent. The emerging therapy is a modified form of vitamin A, which when metabolized in the retina, results in much less waste. Scientists developed ALK-001 by replacing hydrogen atoms in vitamin A with deuterium. Known as deuterated vitamin A, it “burns cleaner” than the natural form. Deuterium is a safe, naturally occurring, stable form of hydrogen, which is present in the human body.

STARGARDT DISEASE NATURAL HISTORY STUDY HELPING PREPARE FOR FUTURE CLINICAL TRIALS

The Foundation Fighting Blindness completed a $6 million natural history study for people affected by Stargardt disease. Known as ProgSTAR, the study has three primary goals: 1) Determine the best outcome measures to accelerate evaluation of emerging treatments in clinical trials, 2) better understand disease progression for selecting future clinical trial participants, and 3) identify potential participants for forthcoming clinical trials. The study has enrolled 365 patients in 10 international clinical centers.

RESEARCH TEAM IS CREATING PATCH DERIVED FROM STEM CELLS TO RESTORE VISION

David Gamm, MD, PhD, at the University of Wisconsin–Madison, is leading a team of experts to create a patch composed of retinal cells derived from stem cells as a vision-restoring therapy for people with macular conditions such as Stargardt disease. The company Opsis Therapeutics was recently formed to advance the therapy into and through a clinical trial and out to the patients who need it. The patch consists of two layers. One layer will serve as a precursor to vision-enabling photoreceptors. Once transplanted, the stem cells of this layer will mature into photoreceptors. The other layer will consist of mature retinal pigment epithelial (RPE) cells, which provide waste disposal and nutrition for photoreceptors. A thin plastic film will serve as a structural backbone for the patch. A biodegradable gel will protect the cells and hold the layers together. The Foundation Fighting Blindness currently funds the project and has funded Dr. Gamm for more than a decade to advance this therapeutic approach.

ASTELLAS CONDUCTING CLINICAL TRIAL OF COMPLEMENT INHIBITOR FOR STARGARDT DISEASE

ASTELLAS is conducting a Phase 2 clinical trial its C5 complement inhibitor designed to slow the progression of Stargardt disease. In many retinal diseases, the complement system (part of the innate immune system) is overactive, thereby exacerbating retinal degeneration and accelerating vision loss.

JANUARY 2024