California-based biotech Quince Therapeutics has won fast track designation from the US Food and Drug Administration (FDA) for its ataxia-telangiectasia (A-T) drug EryDex.

According to the 3 June announcement, fast track designation was awarded to address a high unmet medical need for patients with A-T. The status allows for earlier interactions with the FDA as Quince seeks accelerated approval, and also the possibility to undergo rolling reviews.

A-T is a rare disease of the nervous and immune systems, affecting motor movement and speech. EryDex utilises autologous intracellular drug encapsulation (AIDE) technology to administer dexamethasone sodium phosphate (DSP) into a patient’s red blood cells. Red blood cells filled with DSP are then reinfused into the patient, allowing for the slow release of steroids over several weeks, without the long-term toxicity commonly associated with chronic administration.

The FDA lifted a partial clinical hold that was on the drug in October 2023, advancing it into the Phase III NEAT study (NCT06193200). The FDA removed the partial clinical hold after requesting additional information on plastics used in the single-use EryKit, which is part of EryDex. Changes were made to comply with recent European regulations.

The NEAT study will enrol approximately 86 six- to nine-year-old patients with A-T as the primary analysis population, as well as 20 patients over the age of ten years. Participants will be randomised between EryDex or placebo and treatment will involve six infusions once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last visit completion in the rescored modified International Cooperative Ataxia Rating Scale (RmICARS).

Quince shared in an April 2024 business update that it anticipates topline results in H2 2025 with a potential NDA submission in 2026.

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Quince inherited EryDex in July 2023 when it acquired biotech EryDel in a $485m deal in October 2023. Formerly known as Cortexyme, Quince changed its name in 2022 and focused on rare diseases following several setbacks, including a clinical hold on its Alzheimer’s disease drug atuzaginstat.

In the announcement accompanying the fast track, Quince CEO Dirk Thye said: “The granting of fast track status for EryDex System marks another important milestone in our endeavour to identify a beneficial therapeutic solution for patients with A-T.

“We have initiated our pivotal Phase III NEAT clinical trial, which is being conducted in the US, UK, and the European Union.”