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Quince Therapeutics (Nasdaq: QNCX) announced the publication of Phase 3 ATTeST clinical trial data in The Lancet Neurology, evaluating EryDex for treating Ataxia-Telangiectasia (A-T). The study, involving 175 participants across 22 institutions in 12 countries, demonstrated a favorable safety profile and positive effects in patients aged 6-9 years.
Key findings include:
- No serious safety concerns typically associated with chronic corticosteroid use
- Statistically significant reduction in neurological symptoms in 6-9 year olds receiving high-dose EryDex
- Potential treatment delays due to COVID-19 pandemic affected overall results
Quince has initiated the pivotal Phase 3 NEAT study under FDA Special Protocol Assessment, with topline results expected in Q4 2025.
Quince Therapeutics (Nasdaq: QNCX) ha annunciato la pubblicazione dei dati del trial clinico di Fase 3 ATTeST su The Lancet Neurology, che valuta EryDex per il trattamento dell'Atassia-Telangiectasia (A-T). Lo studio, che ha coinvolto 175 partecipanti in 22 istituzioni in 12 paesi, ha dimostrato un profilo di sicurezza favorevole e effetti positivi nei pazienti di età compresa tra 6 e 9 anni.
I risultati chiave includono:
- Nessuna preoccupazione grave per la sicurezza comunemente associata all'uso cronico di corticosteroidi
- Riduzione statisticamente significativa dei sintomi neurologici nei bambini di 6-9 anni che ricevevano EryDex ad alte dosi
- I ritardi potenziali nel trattamento a causa della pandemia di COVID-19 hanno influenzato i risultati complessivi
Quince ha avviato il fondamentale studio di Fase 3 NEAT sotto la valutazione del protocollo speciale della FDA, con risultati preliminari attesi nel quarto trimestre del 2025.
Quince Therapeutics (Nasdaq: QNCX) anunció la publicación de los datos del ensayo clínico de Fase 3 ATTeST en The Lancet Neurology, evaluando EryDex para el tratamiento de la Ataxia-Telangiectasia (A-T). El estudio, que involucró a 175 participantes en 22 instituciones de 12 países, demostró un perfil de seguridad favorable y efectos positivos en pacientes de entre 6 y 9 años.
Los hallazgos clave incluyen:
- No hay preocupaciones serias de seguridad comúnmente asociadas con el uso crónico de corticosteroides
- Reducción estadísticamente significativa de los síntomas neurológicos en niños de 6 a 9 años que recibieron EryDex en altas dosis
- Los retrasos potenciales en el tratamiento debido a la pandemia de COVID-19 afectaron los resultados generales
Quince ha iniciado el estudio pivotal de Fase 3 NEAT bajo la Evaluación de Protocolo Especial de la FDA, con resultados preliminares esperados para el cuarto trimestre de 2025.
Quince Therapeutics (Nasdaq: QNCX)는 Ataxia-Telangiectasia (A-T) 치료를 위한 EryDex의 3상 ATTeST 임상 시험 데이터가 The Lancet Neurology에 발표되었다고 발표했습니다. 12개국 22개 기관에서 175명의 참가자가 참여한 이 연구는 6세에서 9세 사이의 환자들에게 유리한 안전성 프로필과 긍정적인 효과를 보여주었습니다.
주요 발견은 다음과 같습니다:
- 만성 corticosteroid 사용과 일반적으로 연관된 심각한 안전성 문제 없음
- 고용량 EryDex를 투여받은 6세에서 9세 아동의 신경학적 증상에서 통계적으로 유의미한 감소
- COVID-19 팬데믹으로 인한 치료 지연 가능성이 전체 결과에 영향을 미쳤음
Quince는 FDA 특별 프로토콜 평가에 따라 3상 NEAT 연구를 시작했으며, 2025년 4분기에 주요 결과를 발표할 예정입니다.
Quince Therapeutics (Nasdaq: QNCX) a annoncé la publication des données de l'essai clinique de phase 3 ATTeST dans The Lancet Neurology, évaluant EryDex pour le traitement de l'Ataxie-Télangiectasie (A-T). L'étude, impliquant 175 participants dans 22 institutions réparties sur 12 pays, a démontré un profil de sécurité favorable et des effets positifs chez les patients âgés de 6 à 9 ans.
Les principales conclusions comprennent :
- Aucune préoccupation grave pour la sécurité généralement associée à l'utilisation chronique de corticostéroïdes
- Réduction statistiquement significative des symptômes neurologiques chez les enfants de 6 à 9 ans recevant des doses élevées d'EryDex
- Des retards potentiels de traitement dus à la pandémie de COVID-19 ont eu un impact sur les résultats globaux
Quince a lancé l'étude pivot de phase 3 NEAT sous l'évaluation du protocole spécial de la FDA, avec des résultats préliminaires attendus au quatrième trimestre 2025.
Quince Therapeutics (Nasdaq: QNCX) hat die Veröffentlichung der Daten der klinischen Phase-3-Studie ATTeST in The Lancet Neurology bekannt gegeben, in der EryDex zur Behandlung von Ataxie-Telangiektasie (A-T) evaluiert wurde. Die Studie, an der 175 Teilnehmer in 22 Institutionen aus 12 Ländern beteiligt waren, zeigte ein günstiges Sicherheitsprofil und positive Effekte bei Patienten im Alter von 6 bis 9 Jahren.
Wichtige Ergebnisse sind:
- Keine schwerwiegenden Sicherheitsbedenken, die normalerweise mit der chronischen Verwendung von Kortikosteroiden verbunden sind
- Statistisch signifikante Reduktion neurologischer Symptome bei 6- bis 9-Jährigen, die hochdosiertes EryDex erhalten
- Potenzielle Behandlungsverzögerungen aufgrund der COVID-19-Pandemie beeinflussten die Gesamtergebnisse
Quince hat die entscheidende Phase-3-Studie NEAT unter der besonderen Protokollbewertung der FDA initiiert, mit Hauptresultaten, die im vierten Quartal 2025 erwartet werden.
Positive
- Favorable safety profile of EryDex with no serious concerns typically associated with chronic corticosteroid use
- Statistically significant reduction in neurological symptoms for 6-9 year old patients receiving high-dose EryDex
- FDA granted Fast Track designation for EryDex in A-T treatment
- Ongoing Phase 3 NEAT study being conducted under FDA Special Protocol Assessment agreement
- Potential NDA submission to FDA and MAA submission to EMA in 2026 if study results are positive
Negative
- Primary efficacy endpoint not met in overall study population
- COVID-19 pandemic disruptions caused treatment delays and missed doses, affecting 35% of the mITT population
- Inability to confirm treatment effect in the overall population
The publication of the ATTeST trial results in The Lancet Neurology marks a significant milestone in A-T research. The study, involving 175 participants across 22 institutions in 12 countries, is the largest completed trial for this rare disease. Key findings include:
- A favorable safety profile for EryDex, with no serious side effects typically associated with chronic corticosteroid use.
- A statistically significant reduction in neurological symptoms for the 6-9 age group receiving high-dose EryDex, with a
-2.8 change in mICARS score (p=0.019). - The per-protocol population showed more pronounced effects, suggesting that treatment adherence is crucial.
These results provide a strong foundation for the ongoing NEAT study, which focuses on the 6-9 age group where rapid clinical decline typically occurs. The FDA's Special Protocol Assessment and Fast Track designation further underscore the potential impact of this treatment for A-T patients.
Quince Therapeutics' EryDex represents a potentially groundbreaking treatment for Ataxia-Telangiectasia, a market with significant unmet need. Key points for investors:
- EryDex targets a rare pediatric disease with no approved treatments, potentially leading to orphan drug status and premium pricing.
- The Special Protocol Assessment agreement with the FDA reduces regulatory risk for the pivotal NEAT study.
- Fast Track designation could expedite the review process, potentially bringing EryDex to market sooner.
- The focused approach on the 6-9 age group in the NEAT study, based on ATTeST results, increases the likelihood of demonstrating efficacy.
If successful, EryDex could become a first-in-class therapy for A-T, potentially driving significant revenue growth for Quince. However, investors should note the long timeline, with topline results expected in Q4 2025 and potential regulatory submissions in 2026.
The ATTeST trial design and execution offer valuable insights for rare disease research:
- The large sample size (n=175) for a rare disease study enhances statistical power and reliability of results.
- The multinational approach (12 countries) increases generalizability and addresses potential genetic or environmental factors.
- The pre-specified subgroup analysis by age revealed important efficacy differences, highlighting the importance of age stratification in A-T trials.
- The impact of COVID-19 on treatment adherence (35% of mITT population affected) underscores the challenges in conducting long-term trials during global health crises.
The ongoing NEAT study appears well-designed, incorporating lessons from ATTeST. The focus on the 6-9 age group and inclusion of a smaller cohort of older patients aligns with observed age-related treatment effects. This strategic approach may increase the likelihood of demonstrating clinically meaningful outcomes in the primary analysis population.
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
“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
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. 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 pharmaco*kinetics, 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.
View source 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
Source: Quince Therapeutics, Inc.
FAQ
What were the main results of the Phase 3 ATTeST trial for Quince Therapeutics' EryDex (QNCX)?
The Phase 3 ATTeST trial showed a favorable safety profile for EryDex and a statistically significant reduction in neurological symptoms for A-T patients aged 6-9 years receiving high-dose treatment. However, the primary efficacy endpoint was not met in the overall study population.
When does Quince Therapeutics (QNCX) expect to report topline results from the Phase 3 NEAT study?
Quince Therapeutics expects to report topline results from the Phase 3 NEAT study in the fourth quarter of 2025.
What is the target age group for Quince Therapeutics' (QNCX) Phase 3 NEAT study of EryDex?
The primary analysis population for the Phase 3 NEAT study of EryDex is patients with A-T aged 6-9 years old, with an additional group of patients aged 10 years or older.
Has EryDex received any special designations from the FDA for Quince Therapeutics (QNCX)?
Yes, the FDA has granted Fast Track designation to Quince Therapeutics' EryDex System for the treatment of patients with Ataxia-Telangiectasia (A-T).
