Quince Therapeutics Announces Frontiers in Neurology Publication of Long-Term Safety of EryDex in Pediatric Patients with Ataxia-Telangiectasia
Quince Therapeutics (NASDAQ: QNCX) announced the publication of safety data in Frontiers in Neurology regarding EryDex treatment in pediatric patients with Ataxia-Telangiectasia (A-T). The data comes from patients treated for a minimum of 24 months in Phase 3 clinical trials.
EryDex, which encapsulates dexamethasone sodium phosphate in patients' own red blood cells, demonstrated favorable safety profiles without the typical chronic side effects associated with standard corticosteroid treatment. The company has administered over 6,000 monthly doses to nearly 400 patients over 10+ years.
The most common treatment-related side effect was temporary infusion-related pruritus. The company is currently conducting the pivotal Phase 3 NEAT trial under a Special Protocol Assessment agreement with the FDA, with topline results expected in Q4 2025. Quince plans to submit New Drug Application to FDA and Marketing Authorization Application to EMA in 2026, pending positive results.
Quince Therapeutics (NASDAQ: QNCX) ha annunciato la pubblicazione di dati sulla sicurezza in Frontiers in Neurology riguardanti il trattamento con EryDex in pazienti pediatrici affetti da Atassia-Telangiectasia (A-T). I dati provengono da pazienti trattati per un minimo di 24 mesi in studi clinici di Fase 3.
EryDex, che incapsula il fosfato di sodio di desametasone nei globuli rossi dei pazienti, ha dimostrato profili di sicurezza favorevoli senza gli effetti collaterali cronici tipici associati al trattamento con corticosteroidi standard. L'azienda ha somministrato oltre 6.000 dosi mensili a quasi 400 pazienti in oltre 10 anni.
L'effetto collaterale più comune correlato al trattamento è stato il prurito temporaneo legato all'infusione. Attualmente, l'azienda sta conducendo il trial pivotale di Fase 3 NEAT ai sensi di un accordo di Valutazione del Protocollo Speciale con la FDA, con risultati preliminari previsti nel quarto trimestre del 2025. Quince prevede di presentare la Domanda di Nuovo Farmaco alla FDA e la Domanda di Autorizzazione alla Commercializzazione all'EMA nel 2026, in attesa di risultati positivi.
Quince Therapeutics (NASDAQ: QNCX) anunció la publicación de datos de seguridad en Frontiers in Neurology sobre el tratamiento con EryDex en pacientes pediátricos con Ataxia-Telangiectasia (A-T). Los datos provienen de pacientes tratados durante un mínimo de 24 meses en ensayos clínicos de Fase 3.
EryDex, que encapsula fosfato de sodio de dexametasona en los glóbulos rojos de los pacientes, mostró perfiles de seguridad favorables sin los efectos secundarios crónicos típicos asociados con el tratamiento estándar con corticosteroides. La empresa ha administrado más de 6,000 dosis mensuales a casi 400 pacientes durante más de 10 años.
El efecto secundario relacionado con el tratamiento más común fue el prurito temporal relacionado con la infusión. Actualmente, la empresa está llevando a cabo el ensayo pivotal de Fase 3 NEAT bajo un acuerdo de Evaluación de Protocolo Especial con la FDA, con resultados preliminares esperados en el cuarto trimestre de 2025. Quince planea presentar una Solicitud de Nuevo Medicamento a la FDA y una Solicitud de Autorización de Comercialización a la EMA en 2026, pendientes de resultados positivos.
퀸스 테라퓨틱스 (NASDAQ: QNCX)는 소아 환자에서의 에리덱스 치료와 관련된 안전성 데이터를 Frontiers in Neurology에 발표했습니다. 이 데이터는 3상 임상 시험에서 최소 24개월 동안 치료를 받은 환자들로부터 나왔습니다.
에리덱스는 환자의 적혈구에 디프로산 나트륨 포스페이트를 캡슐화하여 일반 스테로이드 치료와 관련된 전형적인 만성 부작용 없이 유리한 안전성 프로파일을 보여주었습니다. 이 회사는 10년 넘게 거의 400명 환자에게 매달 6,000회 이상의 용량을 투여해 왔습니다.
가장 흔한 치료 관련 부작용은 일시적인 주입 관련 가려움증이었습니다. 현재 이 회사는 FDA와의 특별 프로토콜 평가 계약 아래에서 주요 3상 NEAT 시험을 진행하고 있으며, 2025년 4분기에 주요 결과가 예상됩니다. 퀸스는 긍정적인 결과를 전제로 2026년에 FDA에 신규 약물 신청과 EMA에 마케팅 허가 신청을 제출할 계획입니다.
Quince Therapeutics (NASDAQ: QNCX) a annoncé la publication de données de sécurité dans Frontiers in Neurology concernant le traitement par EryDex chez des patients pédiatriques atteints d'Ataxie-Télangiectasie (A-T). Les données proviennent de patients traités pendant au moins 24 mois dans des essais cliniques de phase 3.
EryDex, qui encapsule le phosphate de dexaméthasone sodique dans les globules rouges des patients, a montré des profils de sécurité favorables sans les effets secondaires chroniques typiques associés au traitement standard par corticostéroïdes. L'entreprise a administré plus de 6 000 doses mensuelles à près de 400 patients au cours de plus de 10 ans.
L'effet secondaire lié au traitement le plus courant a été le prurit temporaire lié à l'infusion. L'entreprise mène actuellement l'essai pivot de phase 3 NEAT dans le cadre d'un accord d'évaluation de protocole spécial avec la FDA, avec des résultats préliminaires attendus au quatrième trimestre de 2025. Quince prévoit de soumettre une demande de nouveau médicament à la FDA et une demande d'autorisation de mise sur le marché à l'EMA en 2026, sous réserve de résultats positifs.
Quince Therapeutics (NASDAQ: QNCX) gab die Veröffentlichung von Sicherheitsdaten in Frontiers in Neurology bekannt, die die Behandlung mit EryDex bei pädiatrischen Patienten mit Ataxie-Telangiektasie (A-T) betreffen. Die Daten stammen von Patienten, die mindestens 24 Monate lang in Phase-3-Studien behandelt wurden.
EryDex, das Dexamethason-Natriumphosphat in den roten Blutkörperchen der Patienten kapselt, zeigte günstige Sicherheitsprofile ohne die typischen chronischen Nebenwirkungen, die mit der standardmäßigen Kortikosteroidbehandlung verbunden sind. Das Unternehmen hat über 6.000 monatliche Dosen an fast 400 Patienten über mehr als 10 Jahre verabreicht.
Die häufigsten behandlungsbedingten Nebenwirkungen waren vorübergehender infusionsbedingter Juckreiz. Das Unternehmen führt derzeit die entscheidende Phase-3-NEAT-Studie im Rahmen einer Vereinbarung zur besonderen Protokollbewertung mit der FDA durch, wobei die Hauptergebnisse für das vierte Quartal 2025 erwartet werden. Quince plant, 2026 einen Neuzulassungsantrag bei der FDA und einen Antrag auf Marktzulassung bei der EMA einzureichen, sofern die Ergebnisse positiv ausfallen.
- Received FDA Fast Track designation for A-T treatment
- Strong safety profile with over 6,000 doses administered to nearly 400 patients
- Minimal adverse effects compared to standard corticosteroid treatments
- Clear regulatory pathway with Special Protocol Assessment agreement with FDA
- Phase 3 trial results not expected until Q4 2025
- Observed decline in bone mineral density of 0.4 z scores over two years
Insights
The publication of long-term safety data for EryDex represents a significant milestone in addressing an unmet medical need in Ataxia-Telangiectasia treatment. The safety profile is particularly noteworthy as it demonstrates minimal steroid-related toxicities - a important factor for pediatric patients requiring long-term treatment. With over 6,000 monthly doses administered to nearly 400 patients over a decade, the robust safety database strengthens EryDex's regulatory position.
The ongoing Phase 3 NEAT trial's design under Special Protocol Assessment (SPA) agreement with FDA significantly de-risks the regulatory pathway. The trial's structure, targeting 86 patients aged 6-9 years for primary analysis, is well-positioned to demonstrate efficacy. The Fast Track designation further expedites the review process and highlights the FDA's recognition of the significant unmet need in A-T treatment.
The innovative approach of using autologous erythrocytes (patient's own red blood cells) to deliver dexamethasone represents a potential breakthrough in steroid delivery systems. This could create a new paradigm for treating rare pediatric diseases where long-term steroid use is necessary but by toxicity concerns. The planned regulatory submissions to both FDA and EMA in 2026 position Quince to potentially capture first-mover advantage in the A-T market.
“A-T is a rare pediatric disease with unmet medical need and no approved therapeutic treatment. This post-hoc analysis of patients with A-T treated for a minimum of 24 months in a Phase 3 clinical trial (ATTeST) and an open label extension study, provides data supporting the potential to deliver dexamethasone sodium phosphate encapsulated in a patient’s own red blood cells using our EryDex System without the chronic debilitating toxicities observed with standard corticosteroid treatment,” said Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer. “Adverse events typically observed with prolonged corticosteroid use such as Cushingoid features, weight gain, hypertension, hirsutism, diabetes, or stunted growth were rarely reported in these studies. With more than 6,000 monthly doses administered to nearly 400 patients over the last 10-plus years, we are building a strong safety database as our pivotal Phase 3 clinical trial (NEAT) and its open label extension study continue.”
Frontiers in Neurology Publication Highlights
The Frontiers in Neurology publication entitled Long-term safety of dexamethasone sodium phosphate encapsulated in autologous erythrocytes in pediatric patients with Ataxia-Telangiectasia describes the safety of long-term use of EryDex in treatment of pediatric patients with A-T.
Highlights include:
- EryDex is dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes that has been developed in an effort to address chronic steroid toxicity while maintaining efficacy.
- Results include analyses of data from patients with A-T treated with EryDex for a minimum of 24 months.
- Analyses included treatment-emergent and treatment-related adverse events, serious adverse events, and adverse events of special interest.
- The most common treatment-related adverse event was transient infusion-related pruritus, which is the most common steroid related side effect, and low serum iron levels, which are not associated with negative clinical outcomes.
- Adverse events typically observed with prolonged glucocorticoid use such as Cushingoid features, weight gain, hypertension, hirsutism, diabetes, or stunted growth were infrequently reported over 24 to 61 months of dosing.
- There was a decline in bone mineral density (BMD) of 0.4 z scores over the two-year period – which could not be distinguished from the natural course of disease in patients with A-T.
- There were no reported adverse effects on height, weight and body mass index as supported by stable z-scores throughout the two years of treatment.
- Values for glucose, HbA1c, cortisol, and CD4+ lymphocyte counts did not show clinically significant changes during prolonged treatment with EryDex.
Quince is currently enrolling the pivotal Phase 3 NEAT clinical trial (#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.
The Phase 3 NEAT trial is being conducted under a Special Protocol Assessment (SPA) agreement with the
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
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. The EryDex 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.
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, 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 current and future clinical development of EryDex, including for the potential treatment of Ataxia-Telangiectasia (A-T), Duchenne muscular dystrophy (DMD), and other potential indications, related development and 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, success, and reporting of results of the clinical trials and related data, including plans and the ability to initiate, fund, enroll, 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 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 November 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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20250127038684/en/
Media & Investor Contact:
Stacy Roughan
Quince Therapeutics, Inc.
Vice President, Corporate Communications & Investor Relations
ir@quincetx.com
Source: Quince Therapeutics, Inc.
FAQ
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