FDA Lifts Partial Clinical Hold on IND for EryDel’s Phase 3 Therapy for Ataxia-Telangiectasia
September 29, 2023
Rare Daily Staff
Quince Therapeutics said that The U.S. Food and Drug Administration has lifted the partial clinical hold on EryDel’s Investigational New Drug application for its lead phase 3 asset, EryDex.
Pending the closing of Quince’s acquisition of EryDel, Quince intends to advance the global phase 3 NEAT clinical trial evaluating the safety and efficacy of EryDex for the potential treatment of the rare, fatal pediatric neurological disease, ataxia-telangiectasia (A-T). Currently, there are no approved treatments for patients with A-T and the market represents a $1+ billion estimated peak sales opportunity.
“We look forward to completing the clinical and regulatory activities necessary to advance EryDex into the phase 3 NEAT study – with patient enrollment beginning as soon as the second quarter of 2024,” said Dirk Thye, CEO of Quince. “Notably, this pivotal trial will be conducted under a Special Protocol Assessment that has already been reviewed with the FDA, which should allow for the submission of a New Drug Application (NDA) following completion of this single study, assuming positive results.”
The agency had requested additional information on extractables and leachables related to a change in plastics utilized in the EryKit. The change in plastic bags and tubing in the EryKit were implemented in order to be compliant with recent European regulations regarding the type of plastics used in various products. The commercial version of the EryKit treatment consumables is already approved for clinical trial use in Europe.
Quince’s acquisition of EryDel is subject to certain regulatory approvals, including the foreign direct investment screening clearance in Italy, and other closing conditions and is expected to close in the fourth quarter of 2023.
EryDex utilizes a unique drug/device combination that enables a fully automated process at the point of patient care for the autologous intracellular drug encapsulation of dexamethasone sodium phosphate, a pro-drug, into a patient’s red blood cells. Dexamethasone-loaded red blood cells are then re-infused into the patient, resulting in the circulation of controlled, slow release, low doses of dexamethasone, intended to provide efficacious dosing while avoiding the long-term toxicity typically associated with chronic steroid administration.
The phase 3 NEAT clinical trial is a double blind, randomized, placebo-controlled, global study in approximately 86 A-T patients aged six to nine years-old – with up to an additional 20 patients aged 10 years or older to potentially expand the label. The study’s primary endpoint will measure neurological function based on a rescored modified International Cooperative Ataxia Rating Scale (RmICARS) from baseline to month six of treatment. NDA submission is currently targeted for the end of 2025, assuming positive phase 3 study results. EryDex has received orphan drug designation for the treatment of A-T from both the FDA and the European Medicines Agency.
Photo: Dirk Thye, CEO of Quince
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