Quince Therapeutics has received positive safety review results from an independent data and safety monitoring board (iDSMB) for its pivotal Phase 3 NEAT clinical trial evaluating encapsulated dexamethasone sodium phosphate (eDSP) in patients with Ataxia-Telangiectasia (A-T). The monitoring board identified no safety concerns and recommended that the study continue without any modifications.
The announcement represents a significant milestone for what could become the first approved treatment for A-T, a rare genetic disorder. "The positive outcome from our recent iDSMB review of eDSP reaffirms our confidence in the favorable safety profile of eDSP to date," said Dirk Thye, M.D., Quince's Chief Executive Officer and Chief Medical Officer. "As we continue to advance eDSP as a potential first-to-market A-T treatment, we are pleased that no safety concerns have been identified as our pivotal Phase 3 NEAT study progresses."
Phase 3 NEAT Trial Design
The ongoing Phase 3 NEAT study (Neurological Effects of eDSP in Subjects with A-T; NCT06193200/IEDAT-04-2022) is an international, multicenter, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the neurological effects of eDSP in A-T patients. The study consists of two cohorts randomized 1:1 between eDSP or placebo, with treatment including six infusions scheduled once every 21 to 30 days.
The primary efficacy endpoint will be measured by the change from baseline to last efficacy visit using the Rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo. Quince remains on track to report topline results from the study in the first quarter of 2026.
Novel Drug Delivery Technology
eDSP represents an innovative approach to corticosteroid delivery, comprised of dexamethasone sodium phosphate encapsulated in a patient's own red blood cells (autologous erythrocytes). Dexamethasone sodium phosphate is a corticosteroid well known for its anti-inflammatory properties as well as its dose-limiting toxicity due to adrenal suppression. The eDSP System is designed to provide the efficacy of corticosteroids while reducing or eliminating the significant adverse effects that accompany chronic corticosteroid treatment.
The therapy leverages Quince's proprietary Autologous Intracellular Drug Encapsulation (AIDE) technology platform, which is a novel drug/device combination that uses an automated process designed to encapsulate drugs into patients' own red blood cells. Red blood cells have several characteristics that make them a potentially effective vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life.
Quince's AIDE technology is designed to harness these benefits to allow for the chronic administration of drugs that have limitations due to toxicity, poor biodistribution, suboptimal pharmacokinetics, or immune response. This approach could address the significant challenge of providing effective long-term corticosteroid treatment while minimizing the well-known adverse effects associated with chronic corticosteroid use.
