Quince’s Phase 3 lead asset, EryDex, targets the treatment of a rare neurodegenerative disease, Ataxia-Telangiectasia (A-T). Currently, there are no approved treatments for A-T globally.
EryDex utilizes a highly differentiated technology platform for autologous intracellular drug encapsulation (AIDE). Our proprietary AIDE technology is designed to optimize the biodistribution of dexamethasone sodium phosphate (DSP; a pro-drug) by using an A-T patient’s own red blood cells to deliver the sustained therapeutic over a once monthly treatment period.
By leveraging a patient’s own biology to deliver the therapeutic encapsulated in autologous red blood cells, our AIDE technology is believed to fundamentally alter a drug’s pharmacokinetics –allowing for the chronic administration of the therapy.
The market for A-T represents a $1+ billion estimated peak sales opportunity globally.
Quince plans to advance a single global Phase 3 clinical trial entitled NEAT (#IDEAT-04-2022 – Neurologic Effects of EryDex on Subjects with A–T), which is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the neurological effects of EryDex on patients with A-T. Enrollment for the Phase 3 NEAT trial is anticipated to begin in the second quarter of 2024. We plan to enroll a minimum of 86 A-T patients aged six to nine years-old and approximately 20 additional patients aged 10 years or older.
This pivotal clinical trial will be conducted under a Special Protocol Assessment (SPA) that has been agreed with the U.S. Food & Drug Administration (FDA), which should allow for the submission of a New Drug Application (NDA) following completion of this single study, assuming positive results.
EryDex also has received orphan drug designation for the treatment of A-T from both the U.S. Food & Drug Administration and the European Medicines Agency.
Quince plans to investigate additional indications for EryDex where chronic dexamethasone sodium phosphate treatment is – or has the potential to be – a standard of care. To support this expansion potential, we intend to evaluate clinical trial opportunities for potential additional rare disease indications.
Our flexible and expandable AIDE technology platform is designed to deliver a variety of therapeutics, ranging from small to large molecules, as well as biologics. Quince intends to investigate additional potential applications of the AIDE technology platform as it provides us with opportunities for future indication expansion.