Quince Therapeutics Announces First Patient Dosed in Phase 3 Clinical Trial of EryDex for the Treatment of Ataxia-Telangiectasia
25 Junio 2024 - 6:00AM
Business Wire
Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage
biotechnology company developing an innovative drug delivery
technology designed to leverage a patient’s own biology to deliver
rare disease therapeutics, today announced that the first patient
has been dosed in the company’s Phase 3 NEAT (Neurologic
Effects of EryDex on Subjects with A-T)
clinical trial to evaluate the neurological effects of EryDex in
patients with Ataxia-Telangiectasia (A-T). This pivotal Phase 3
study will be conducted under a Special Protocol Assessment (SPA)
agreement with the U.S. Food & Drug Administration (FDA).
“The initiation of our pivotal Phase 3 NEAT study is a major
milestone for Quince, and an opportunity for patients living with
the devastating effects of Ataxia-Telangiectasia to participate in
research seeking to identify a beneficial therapeutic solution,”
said Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief
Medical Officer. “There are currently no approved therapeutic
treatments in any global market for this rare pediatric disease,
and our primary corporate objective is to change that for patients
with A-T and their families.”
The Phase 3 NEAT clinical trial (#IEDAT-04-2022/NCT06193200) is
an international, multi-center, randomized, double-blind,
placebo-controlled clinical trial to evaluate the neurological
effects of EryDex in patients with A-T. The study plans to enroll
approximately 86 patients with A-T ages six to nine years old
(primary analysis population) and approximately 20 patients with
A-T ages 10 years or older. Participants will be randomized (1:1)
between EryDex or placebo and treatment will consist of six
infusions scheduled every 21 to 30 days. The primary efficacy
endpoint will be measured by the change from baseline to last visit
completion in rescored modified International Cooperative Ataxia
Rating Scale (RmICARS). Participants who complete the full
treatment period, complete the study assessments, and provide
informed consent will be eligible to transition to an open label
extension (OLE) study. Quince expects to report Phase 3 NEAT
topline results in the second half of 2025 with a potential New
Drug Application (NDA) submission in 2026, assuming positive study
results.
With the achievement of the first patient enrolled in this
study, Quince will make a cash milestone payment of $5 million to
the former EryDel shareholders. The company owes no further
development related milestones to EryDel shareholders.
About Ataxia-Telangiectasia
A-T is an inherited autosomal recessive neurodegenerative and
immunodeficiency disorder caused by mutations in the ATM gene,
which is responsible for cell homeostatic and cell division
functions including but not limited to double-stranded DNA repair.
Typically, A-T is first diagnosed before the age of five as
children begin to develop an altered gait and fall with greater
frequency. Neurological symptoms worsen and patients with A-T
frequently become wheelchair-bound by adolescence. Teenage years
for patients with A-T are typically marked by repeated infections,
pulmonary impairment, and malignancies. The median lifespan is
approximately 25 to 30 years old with mortality due to infections
and malignancy. Based on IQVIA Medical Claims (Dx), PharmetricsPlus
(P+), and IQVIA Analytics information, there are approximately
4,600 diagnosed patients with A-T in the U.S. Quince estimates that
there are approximately 5,000 patients with A-T in the U.K. and EU4
countries. There are currently no approved therapeutic treatments
in any global market for A-T.
About EryDex for A-T
EryDex is comprised of dexamethasone sodium phosphate (DSP)
encapsulated in a patient’s own red blood cells. DSP is a
corticosteroid well known for its anti-inflammatory properties as
well as its dose-limiting toxicity due to adrenal suppression.
EryDex is designed to provide the efficacy of corticosteroids and
to reduce or eliminate the significant adverse effects that
accompany chronic use of corticosteroid treatment.
EryDex leverages Quince’s proprietary Autologous Intracellular
Drug Encapsulation, or AIDE, technology platform, which is a novel
drug/device combination that uses an automated process designed to
encapsulate a drug into the patient’s own red blood cells. Red
blood cells have several characteristics that make them a
potentially effective vehicle for drug delivery, including
potentially better tolerability, enhanced tissue distribution,
reduced immunogenicity, and prolongation of circulating half-life.
Quince’s AIDE technology is designed to harness these benefits to
allow for the chronic administration of drugs that have limitations
due to toxicity, poor biodistribution, suboptimal pharmacokinetics,
or immune response.
About Quince Therapeutics
Quince Therapeutics (Nasdaq: QNCX) is a late-stage biotechnology
company dedicated to unlocking the potential of a patient’s own
biology to deliver innovative and life-changing therapeutics to
those living with rare diseases. For more information on the
company and its latest news, visit www.quincetx.com and follow
Quince Therapeutics on social media platforms LinkedIn, Facebook,
and Twitter/X.
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 current and future clinical
development of EryDex, including a pivotal trial for
Ataxia-Telangiectasia, potential commercial-stage inflection point
for EryDex, and expansion of the company’s proprietary Autologous
Intracellular Drug Encapsulation (AIDE) technology for treatment of
other rare diseases; the strategic development path for EryDex;
planned regulatory agency 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 initiate, conduct, and/or complete current and
additional studies; the company’s future development plans and
related timing; the company’s focus, objectives, plans, and
strategies; and the company’s market opportunity. 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 Annual Report on Form 10-K filed with the Securities
and Exchange Commission (SEC) on April 1, 2024, 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/20240625494765/en/
Media & Investor Contact: Stacy Roughan Quince
Therapeutics, Inc. Vice President, Corporate Communications &
Investor Relations ir@quincetx.com
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