U.S. FDA Partial Clinical Hold Lifted on IND for EryDel’s Lead Phase 3 Asset EryDex for the Treatment of Ataxia-Telangiectasia
28 September 2023 - 10:05PM
Business Wire
Quince Therapeutics, Inc. (Nasdaq: QNCX), a biotechnology
company focused on acquiring, developing, and commercializing
innovative therapeutics that transform patients’ lives, today
announced that the U.S. Food and Drug Administration (FDA or the
“Agency”) has lifted the partial clinical hold on EryDel S.p.A’s
Investigational New Drug (IND) 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
(Neurologic Effects of EryDex on Subjects with A-T) clinical trial
evaluating the safety and efficacy of EryDex for the potential
treatment of a 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.
Dirk Thye, M.D., Quince’s Chief Executive Officer, said, “We are
pleased with the FDA’s decision to lift the partial clinical hold
related to EryDel’s lead asset, EryDex. 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.
Notably, this pivotal trial will be conducted under a Special
Protocol Assessment (SPA) 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.
About EryDex and the Phase 3 NEAT Trial
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 (AIDE) of dexamethasone
sodium phosphate (DSP; 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 (EMA).
About Quince Therapeutics
Quince Therapeutics is a biotechnology company focused on
acquiring, developing, and commercializing innovative therapeutics
that transform the lives of patients suffering from debilitating
and rare diseases. For more information, visit www.quincetx.com and
follow Quince Therapeutics on LinkedIn and @Quince_Tx on
Twitter.
Forward-looking Statements
Statements in this news release contain “forward-looking
statements” within the meaning of the Private Securities Litigation
Reform Act of 1995 as contained in Section 27A of the Securities
Act of 1933, as amended, and Section 21E of the Securities Exchange
Act of 1934, as amended, which are subject to the “safe harbor”
created by those sections. All statements, other than statements of
historical facts, may be forward-looking statements.
Forward-looking statements contained in this news release may be
identified by the use of words such as “believe,” “may,” “should,”
“expect,” “anticipate,” “plan,” “believe,” “estimated,”
“potential,” “intend,” “will,” “can,” “seek,” or other similar
words. Examples of forward-looking statements include, among
others, statements relating to the continued current and future
clinical development of EryDel assets, related platform, and
related timing; the strategic development path for EryDex; planned
regulatory submissions and clinical trials and timeline, prospects,
and milestone expectations; the timing and success of the clinical
trials and related data, including plans and the ability to conduct
and/or complete the Phase 3 NEAT clinical trial; the potential
therapeutic benefits, safety, and efficacy of EryDex; statements
about its ability to obtain, and the timing relating to, further
development of EryDex, regulatory submissions and interactions with
regulators; therapeutic and commercial potential; Quince’s future
development plans and related timing; the company’s focus,
objectives, plans, and strategies; and the anticipated closing and
related timing of Quince’s acquisition of EryDel. Forward-looking
statements are based on Quince’s current expectations and are
subject to inherent uncertainties, risks, and assumptions that are
difficult to predict and could cause actual results to differ
materially from what the company expects. Further, certain
forward-looking statements are based on assumptions as to future
events that may not prove to be accurate. Factors that could cause
actual results to differ include, but are not limited to, the risks
and uncertainties described in the section titled “Risk Factors” in
the company’s Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission (SEC) on August 3, 2023, and
other reports as filed with the SEC. Forward-looking statements
contained in this news release are made as of this date, and Quince
undertakes no duty to update such information except as required
under applicable law.
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version on businesswire.com: https://www.businesswire.com/news/home/20230928420547/en/
Stacy Roughan Quince Therapeutics, Inc. Vice President,
Corporate Communications & Investor Relations
ir@quincetx.com
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