FDA removes partial clinical hold on IND for rare pediatric disease drug-device combo

A Bay Area biotech company announced the FDA has lifted the partial clinical hold on EryDel SpA’s investigational new drug application for EryDex to treat ataxia-telangiectasia, a rare pediatric neurological disorder.

According to a press release, pending the closing of its acquisition of EryDel, Quince Therapeutics intends to commence the phase 3 Neurologic Effects of EryDex on Subjects with AT (NEAT) clinical trial to evaluate safety and efficacy of EryDex, a drug-device combination that allows for the autologous intracellular drug encapsulation of dexamethasone sodium phosphate into a patient’s red blood cells, intended to provide efficacious dosing without the toxicity inherent with chronic steroid administration. Currently, there are no approved treatments for patients with the disorder.

The FDA has requested additional information on extractables and leachables related to a change in plastics utilized in the EryKit, as the company adjusted its plastic bags and tubing to comply with European regulations regarding the type of plastics used in various products.

The phase 3 NEAT study is expected to be a double-blind, randomized, placebo-controlled, global trial including approximately 86 children aged 6 to 9 years with AT, with another cohort of 20 enrollees aged 10 years and older. The study’s primary endpoint will measure neurological function based on a rescored modified International Cooperative Ataxia Rating Scale from baseline to month 6 of treatment.

“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,” Quince CEO Dirk Thye, MD, said in the release. “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 following completion of this single study, assuming positive results.”

Quince’s acquisition of EryDel is expected to close in the fourth quarter of 2023, while its new drug application submission is expected by the end of 2025.