Findings from largest completed study in A-T
demonstrated favorable safety profile
Study showed positive effect of EryDex
treatment in a subset of patients with A-T ages six to nine – the
age range that typically experiences rapid clinical decline
Quince recently initiated pivotal Phase 3 NEAT
study, which is currently enrolling and being conducted under U.S.
FDA Special Protocol Assessment agreement
Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage
biotechnology company dedicated to unlocking the power of a
patient’s own biology for the treatment of rare diseases, today
announced the online publication of data in The Lancet Neurology
from its Phase 3 ATTeST (Ataxia-Telangiectasia
Trial with the EryDex SysTem;
#IEDAT-02-2015/NCT02770807) clinical trial evaluating the safety
and efficacy of its lead asset, EryDex (dexamethasone sodium
phosphate encapsulated in autologous erythrocytes), for the
treatment of A-T.
“The results of this study demonstrate the encouraging efficacy
and safety profile of our lead asset, EryDex, for the treatment of
patients with A-T, a rare pediatric disease with vast unmet need
and no approved treatments,” said Dirk Thye, M.D., Quince’s Chief
Executive Officer and Chief Medical Officer. “These findings
provide us with additional confidence in our pivotal Phase 3 study
of EryDex now underway with a primary analysis population comprised
of patients with A-T experiencing the most rapid clinical decline –
children ages six to nine – in addition to including participants
who are ten years of age or older.”
The Lancet Neurology Publication Highlights
The Lancet Neurology publication entitled Safety and efficacy
of intra-erythrocyte dexamethasone sodium phosphate in children
with ataxia-telangiectasia (ATTeST): a multicenter, randomized,
double-blind, placebo-controlled phase 3 trial describes safety
and efficacy results from the Phase 3 ATTeST clinical trial.
Highlights include:
- ATTeST was the largest completed study in A-T, with 175
participants conducted at 22 academic institutions and medical
centers on five continents in 12 different countries.
- ATTeST was a randomized, double-blind, placebo-controlled Phase
3 clinical trial to evaluate the safety and efficacy of two dose
levels of EryDex compared to placebo on neurological symptoms of
patients with A-T.
- In the ATTeST study, following six-months of EryDex treatment,
none of the serious safety concerns typically associated with
chronic corticosteroid administration were observed. There were no
reports of hyperglycemia, hypertension, hirsutism, or cushingoid
appearance in any of the treatment groups.
- The primary efficacy endpoint was measured by the change in
modified International Cooperative Ataxia Rating Scale (mICARS),
from baseline to month six, comparing results from the active and
placebo control groups. mICARS included assessment of the
participant’s posture and gait, kinetic functions, and speech. The
higher score denotes higher burden of neurological symptoms.
- In the ATTeST study, patients receiving high-dose EryDex had
reduced neurological symptoms with a -1.40 change in mICARS
observed in the treated group compared to placebo. This result was
not statistically significant (a nominal p-value =0.077). However,
patients in the per-protocol population who received high-dose
EryDex demonstrated statistical significance with a -2.2 change in
mICARS and a nominal p-value of 0.019.
- In a pre-specified subgroup analysis by age, a statistically
significant reduction in neurological symptoms observed in patients
ages six to nine years old receiving high-dose EryDex, notably, a
-2.8 and -4.4 change in mICARS was observed in the modified
intent-to-treat (mITT) and per-protocol populations compared to
placebo, with nominal p-values of 0.019 and 0.002,
respectively.
- Inability to confirm the effect of treatment in the overall
population might be related to delays and omissions in treatments,
as indicated by positive efficacy results in the per-protocol
population, which excluded participants with delayed and missed
treatments, and potential differences in treatment effect in
patients ages six to nine, compared with patients 10 years or
older.
- Contributors to delayed treatments included difficulties with
travel to treatment centers and disruptions caused by the COVID-19
pandemic. Pandemic control guidelines, including travel
restrictions, which varied among countries and institutions,
resulted in missed and delayed treatments. In some instances,
treatment was delayed because investigators delayed visits to
hospital to protect immunocompromised participants in the peak of
the pandemic. Because of delays in treatment and missed doses, 35%
of the mITT population did not receive treatment as planned.
“The publication of the ATTeST trial results in The Lancet
Neurology highlights promising clinical data that demonstrate
progress being made toward a potential treatment option for
patients with A-T, a rare pediatric disorder characterized by
progressive neurological decline, impaired motor control, speech
difficulties, and a life expectancy typically in the twenties or
thirties,” said Dr. William Whitehouse, Honorary Clinical Associate
Professor of the School of Medicine at the University of
Nottingham, and Consultant Paediatric Neurologist at Nottingham
Children’s Hospital, Nottingham University Hospitals NHS Trust.
“Natural history studies have shown that children between age six
and 10 with classic A-T experience rapid clinical decline, after
neurological symptoms worsen and patients with A-T frequently
become wheelchair-bound by adolescence. The differential response
in mICARS score by age seen in subgroup analyses from the ATTeST
study emphasizes the need to stratify future A-T clinical trials by
age.”
Quince is currently enrolling the pivotal Phase 3 NEAT study
(#IEDAT-04-2022/NCT06193200), which is an international,
multi-center, randomized, double-blind, placebo-controlled clinical
trial evaluating the neurological effects of EryDex in patients
with A-T. Quince 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. Seven
patients with A-T have enrolled in the Phase 3 NEAT study to
date.
The Phase 3 NEAT trial is being conducted under a Special
Protocol Assessment (SPA) agreement with the U.S. Food and Drug
Administration (FDA), and the company expects to report topline
results in the fourth quarter of 2025 with a potential New Drug
Application (NDA) submission to the FDA and a Marketing
Authorization Application (MAA) submission to the European
Medicines Agency (EMA) in 2026, assuming positive study results.
Additionally, Quince was granted Fast Track designation by the FDA
for the company’s EryDex System for the treatment of patients with
A-T based on the potential for EryDex to address a high unmet
medical need in A-T.
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 (autologous
erythrocytes). 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 power of a patient’s own biology
for the treatment of 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,
X, and YouTube.
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 for the potential treatment of
Ataxia-Telangiectasia (A-T) and other potential indications,
related development and commercial-stage inflection point for
EryDex, 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, fund, enroll, conduct, and/or complete current
and additional studies; research and development costs; the
company’s future development plans and related timing; cash
position and projected cash runway; the company’s focus,
objectives, plans, and strategies; and the potential benefits of
EryDex, AIDE technology 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 Quarterly Report on
Form 10-Q filed with the Securities and Exchange Commission (SEC)
on August 13, 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/20240815940181/en/
Media & Investor Contact: Stacy Roughan Quince
Therapeutics, Inc. Vice President, Corporate Communications &
Investor Relations ir@quincetx.com
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