Quince Therapeutics Announces Last Patient Last Visit in Phase 3 NEAT Clinical Trial in Patients with Ataxia-Telangiectasia

Key Terms

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.

special protocol assessment regulatory

A special protocol assessment is a formal, written agreement between a drug or device developer and a health regulator about the design, size and analysis plans of a pivotal clinical trial or study. It matters to investors because it reduces regulatory uncertainty—like getting a signed blueprint before building—by signaling that if the study follows the agreed plan and meets its goals, the regulator is unlikely to reject the results solely for design reasons, though it does not guarantee approval.

u.s. food and drug administration regulatory

The U.S. Food and Drug Administration is the federal agency that evaluates and enforces safety, effectiveness and labeling standards for medicines, medical devices, vaccines, food and related products before they reach consumers. For investors it matters because FDA approvals, warnings or recalls determine whether a product can be sold, how quickly it reaches the market and how costly compliance will be—changes that directly affect a company’s revenue, costs and stock value.

data safety monitoring board medical

A data safety monitoring board is a group of experts who regularly review information from a research or testing process to ensure it is safe and ethical. Think of them as watchdogs that watch over ongoing projects to protect participants and ensure everything is proceeding correctly. Their oversight helps maintain trust and safety, which is important for investors who want to see responsible management and reliable results.

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.

open label extension medical

An open-label extension is a follow-on phase of a clinical trial where participants keep receiving the experimental drug and both doctors and patients know what treatment is being given. It matters to investors because it produces longer-term safety and effectiveness information, helps regulators and companies assess ongoing benefits or risks, and can indicate whether a therapy has staying commercial value — like an extended test drive revealing durability and real-world performance.

rescorded modified international cooperative ataxia rating scale (rmicars) medical

A recorded modified International Cooperative Ataxia Rating Scale (rMICARS) is a standardized checklist used by clinicians to measure the severity and progression of ataxia, a movement and coordination disorder, where assessments are recorded (often on video) and adjusted from the original scale to improve consistency or suit a trial. For investors, rMICARS matters because it provides a repeatable, quantifiable trial endpoint—like a report card judges use—to show whether a drug or therapy produces meaningful clinical improvement, which can influence regulatory decisions, market expectations, and valuation.

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.

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 that the last patient has completed their last visit in the company’s pivotal Phase 3 NEAT (Neurological Effects of eDSP in Subjects with A-T; NCT06193200/IEDAT-04-2022) clinical trial of its lead asset, encapsulated dexamethasone sodium phosphate (eDSP), in patients with Ataxia-Telangiectasia (A-T). The company expects to report topline results from this study in the middle of the first quarter of 2026.

Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, said, “Completing our pivotal Phase 3 NEAT clinical trial marks a major milestone for Quince as we rapidly move toward reporting topline results in the middle of the first quarter of 2026. Conducted under a Special Protocol Assessment agreement with the U.S. Food and Drug Administration, the study was powered at approximately 90% for a treatment difference versus placebo associated with a statistically significant and clinically meaningful treatment difference in a previous trial. The study has been executed with low rates of discontinuations and missing visits – all of which we believe enhance our probability of success. In addition, an unblinded, independent data safety monitoring board has met three times during the course of the study with no safety concerns identified.

“Quince believes that eDSP has great potential to improve the lives of patients living with A-T, a devastating pediatric neurodegenerative rare disease with no currently approved treatments. We are grateful for the participation of patients and their families, as well as the close collaboration with principal investigators and patient advocacy organizations in the U.S. and Europe who made the NEAT study possible,” concluded Dr. Thye.

Quince’s pivotal Phase 3 NEAT study is an international, multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the neurological effects of eDSP in patients with A-T. This 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 will be measured by the change from baseline to last efficacy visit using the Rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo. 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, and all but one of the participants elected to transition to the open label extension (OLE) study (NCT06664853/IEDAT-04-2022).

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 results of clinical trials and related data, including for the company’s Phase 3 NEAT study; current and future clinical development of eDSP, including for the potential treatment of Ataxia-Telangiectasia (A-T); the strategic development path for eDSP; and the potential benefits of eDSP 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 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20251215328276/en/

Media & Investor Contact:

Stacy Roughan

Quince Therapeutics, Inc.

Vice President, Corporate Communications & Investor Relations

ir@quincetx.com

Source: Quince Therapeutics, Inc.