Quince Therapeutics announced the publication of long-term safety data from Ataxia-Telangiectasia (A-T) patients treated with EryDex for at least two years. The data, published in •Frontiers in Neurology•, demonstrates the potential of EryDex to deliver dexamethasone sodium phosphate without the chronic toxicities typically associated with standard corticosteroid treatment. This is particularly important given A-T’s lack of approved therapies.
This research is crucial for A-T patients, who currently face a debilitating and life-shortening illness with no effective treatment options. The findings suggest that EryDex could offer a safer and more tolerable way to manage A-T symptoms, potentially improving their quality of life and extending their lifespan. The absence of commonly observed corticosteroid side effects like Cushingoid features, weight gain, and stunted growth in the EryDex studies signifies a substantial advancement in A-T treatment.
The published data underscores the long-term safety profile of EryDex in pediatric A-T patients. With over 6,000 monthly doses administered to nearly 400 patients over the past decade, a robust safety database is being established. Quince’s ongoing pivotal Phase 3 NEAT clinical trial, conducted under an FDA Special Protocol Assessment, aims to enroll approximately 106 patients. Topline results are anticipated in Q4 2025, with potential New Drug Application and Marketing Authorization Application submissions planned for 2026, pending positive outcomes. Quince has also received Fast Track designation from the FDA for EryDex in A-T.
This research suggests a promising future for A-T patients. If the Phase 3 trial confirms these positive safety findings and demonstrates clinical efficacy, EryDex could become the first approved therapy for this rare disease, significantly impacting patient lives and transforming the A-T treatment landscape. This also strengthens Quince’s position as a leader in developing innovative therapies for rare diseases.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
