Quince Therapeutics has dosed the first subject in its Phase III NEAT clinical trial of EryDex for the treatment of Ataxia-Telangiectasia (A-T), an inherited neurodegenerative and immunodeficiency disorder.

EryDex consists of dexamethasone sodium phosphate (DSP) encapsulated within the red blood cells of the patient.

The global, multicentre, double-blind, randomised, placebo-controlled trial will analyse the neurological effects of EryDex in A-T patients.

Conducted under a Special Protocol Assessment (SPA) agreement with the US Food and Drug Administration (FDA), the trial aims to enrol approximately 86 subjects aged six to nine years (primary analysis population) and nearly 20 people aged ten years or above.

Trial subjects will be categorised into a 1:1 ratio to receive either EryDex or a placebo, with the treatment regimen involving six infusions given every 21 to 30 days.

The variation in the rescored modified International Cooperative Ataxia Rating Scale (RmICARS) from baseline to last visit is the trial’s primary efficacy endpoint.

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Those who conclude the full treatment duration and study assessments, and also give informed consent, can become part of the open label extension (OLE) study.

Quince anticipates reporting topline results from the Phase III NEAT trial in the second half of next year.

On receipt of positive data, the company will submit a new drug application (NDA) in 2026.

Following the enrolment of the first patient in this trial, Quince will issue a $5m cash milestone payment to previous shareholders of EryDel.

Quince CEO and chief medical officer Dirk Thye said: “The initiation of our pivotal Phase III NEAT study is a major milestone for Quince, and an opportunity for patients living with the devastating effects of Ataxia-Telangiectasia to participate in research seeking to identify a beneficial therapeutic solution.

“There are currently no approved therapeutic treatments in any global market for this rare paediatric disease, and our primary corporate objective is to change that for patients with A-T and their families.”