Quince Therapeutics Announces Topline Results from Pivotal Phase 3 NEAT Clinical Trial of eDSP in Ataxia-Telangiectasia

Key Terms

ataxia-telangiectasia medical

Ataxia-telangiectasia is a rare inherited disorder that damages the parts of the body that control movement and balance, causes small visible blood vessels to appear on the skin and eyes, and weakens the immune system, raising the risk of serious infections and cancer. For investors, it matters because the condition defines a small, high-need patient population that shapes the size of potential markets, the complexity and length of clinical trials, and regulatory and pricing dynamics for therapies under development — like a niche but urgent problem that often attracts specialized drug programs.

dexamethasone sodium phosphate medical

A water-soluble form of the corticosteroid drug dexamethasone used mainly for injections or intravenous delivery to rapidly reduce inflammation and suppress the immune response. Think of it as a fast-acting fire extinguisher for harmful inflammation in the body; its availability, dosing, and regulatory status matter to investors because they affect hospital purchasing, treatment choices, clinical trial results, and potential revenue for companies involved in manufacture or distribution.

autologous erythrocytes medical

Autologous erythrocytes are a patient’s own red blood cells used for medical treatments, storage, or laboratory procedures rather than cells from another person. Because they come from the same body, they greatly reduce the risk of immune reaction and disease transmission, much like using your own spare parts instead of someone else’s; for investors, this affects product safety profiles, regulatory pathways, manufacturing complexity, and potential costs or scalability of therapies that rely on personalized blood components.

phase 3 medical

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

randomized, double-blind, placebo-controlled medical

A "randomized, double-blind, placebo-controlled" process is a method used to test the effectiveness of a new treatment or intervention. Participants are randomly assigned to different groups, with one receiving the real treatment and the other a fake version, called a placebo. Neither the participants nor the researchers know who is receiving which, which helps ensure unbiased results. For investors, this rigorous approach increases confidence that the findings are accurate and not influenced by guesswork or bias.

rescored modified international cooperative ataxia rating scale medical

A rescored modified International Cooperative Ataxia Rating Scale is a clinical measurement that tracks changes in coordination, balance and movement problems caused by neurological conditions. It takes an existing standardized test, adjusts some items or scoring rules, and then rescales results so different visits or studies can be compared reliably—think of regrading and normalizing scores on a school exam. For investors, this matters because such a scale is used as a trial endpoint to show whether a drug or device meaningfully improves patient function, which directly affects regulatory decisions, market potential and valuation.

clinical global impression of severity medical

A clinician global impression of severity is a doctor’s overall rating of how serious a patient’s illness is at a given time, usually on a simple scale from 'normal' to 'extremely ill.' Think of it like a single snapshot score a clinician gives after seeing all symptoms and functioning, similar to a teacher giving an overall performance grade. Investors watch this measure because changes can signal whether a treatment is meaningfully improving patients, influence regulatory decisions and market potential, and help predict commercial demand.

p-value technical

A p-value is a number that helps determine how likely it is that a result or pattern happened by chance rather than because of a real effect. For investors, a low p-value suggests that the findings in a study or analysis are probably meaningful and not just random noise—like noticing a pattern in coin flips that’s unlikely to occur by chance. This helps in assessing the reliability of information used to make financial decisions.

Primary and key secondary endpoint did not achieve statistical significance

eDSP was generally well tolerated with no clinically meaningful safety concerns identified

Company to cease clinical development of eDSP; Intends to preserve cash and explore available options

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- 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 topline results from its pivotal Phase 3 NEAT clinical trial evaluating its lead asset, dexamethasone sodium phosphate encapsulated in autologous erythrocytes (eDSP), in patients with Ataxia-Telangiectasia (A-T).

In the NEAT study, the company’s pivotal Phase 3 international, multicenter, randomized, double-blind, placebo-controlled study (n=105), the primary endpoint, which measured the change from baseline to last efficacy visit at month six using the Rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo, did not reach statistical significance. The mean change from baseline to month six was 0.94 in the active arm compared to 2.24 in the placebo arm (difference -1.30) with a p-value of 0.0851. Furthermore, the study did not meet its key secondary endpoint of improvement in Clinical Global Impression of Severity (CGI-S) measured from baseline to month six with a p-value of 0.522.

eDSP was generally well tolerated and there were no clinically meaningful safety concerns identified. The most common adverse events reported in the eDSP arm included pruritis and pyrexia.

Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, said, “We express our compassion and hope for future therapeutic options to the A-T community. We have tremendous gratitude toward the patients, their families, academic investigators and study sites, as well as all Quince employees, who worked so diligently over many years on this program.”

About Pivotal Phase 3 NEAT Clinical Trial

NEAT (Neurological Effects of eDSP in Subjects with A-T; NCT06193200/IEDAT-04-2022) was an international, multicenter, randomized, double-blind, placebo-controlled clinical trial evaluating the neurological effects of eDSP in patients with A-T. A total of 105 participants were randomized across leading academic centers, including four clinical sites in the U.S. and 18 in the U.K. and Europe. The study consisted of two cohorts randomized (1:1) between eDSP or placebo and the treatment included six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint was measured by the change from baseline to last efficacy visit at month six using RmICARS compared to placebo. RmICARS is a refined version of the International Cooperative Ataxia Rating Scale (ICARS), a standard, clinician-administered tool used to quantify ataxia severity, which places increased focus on posture and gait disturbance with higher sensitivity in children ages six to 10 years old.

Quince enrolled a total of 105 participants in the NEAT study, including 83 participants in the six to nine year-old primary analysis population and 22 participants aged 10 years and older. Participants who completed the full treatment period, completed study assessments, and provided informed consent were eligible to transition to the company’s open label extension (OLE) study (NCT06664853/IEDAT-05-2024). All but one of the participants elected to transition to the 24-month OLE.

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 eDSP

eDSP 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. The eDSP System is designed to provide the efficacy of corticosteroids and to reduce or eliminate the significant adverse effects that accompany chronic use of corticosteroid treatment.

eDSP 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, Inc. (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 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 the Company’s intentions to preserve cash and explore available options. 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 March 24, 2025, Quarterly Report on Form 10-Q filed with the SEC on November 12, 2025, 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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Media & Investor Contact:

Stacy Roughan

Quince Therapeutics, Inc.

Vice President, Corporate Communications & Investor Relations

ir@quincetx.com

Source: Quince Therapeutics, Inc.