Quince Therapeutics (QNCX) Investor Day 2025 summary

Strategic vision, technology platform, and future plans

• eDSP, an autologous red blood cell-encapsulated dexamethasone therapy, targets rare diseases starting with ataxia-telangiectasia (AT) and expanding to Duchenne Muscular Dystrophy (DMD) and other indications.

• The AIDE technology platform is fully automated, mobile, CE marked, and supported by a robust patent portfolio and regulatory compliance in the US and EU.

• Over 7,800 eDSP infusions have been administered to more than 425 participants, demonstrating a strong safety and efficacy profile.

• Commercial readiness includes a partnership with Option Care Health for scalable US delivery and a supply chain supporting future launches.

• Strong relationships with patient advocacy groups and a focus on early diagnosis and treatment aim to maximize patient benefit.

Clinical development, regulatory milestones, and business progress

• Pivotal phase III NEAT trial in AT completed enrollment (105 patients, 83 in primary analysis, ages 6–9), with topline results expected in Q1 2026.

• The NEAT trial is powered at 90%+ for statistical significance, with the FDA-mandated RMICARS endpoint for gait and posture.

• Previous ATTeST/ATTEST phase III trial showed a 24% difference vs. placebo in the target age group, with no serious safety concerns and no adrenal suppression.

• Orphan drug and Fast Track designations in the US and EU, with regulatory exclusivity and patent protection through at least 2036.

• NDA submission is planned for the second half of 2026, with supplemental pediatric data and a request for Priority Review.

Mechanism of action and biomarker insights

• eDSP enables sustained, monthly dexamethasone exposure below toxicity thresholds, avoiding chronic steroid side effects.

• RNA sequencing from the largest A-T trial to date revealed eDSP downregulates interferon-stimulated and pro-inflammatory genes, and upregulates neuroprotective and mitochondrial genes.

• High-dose eDSP showed a clear dose response in gene expression changes, supporting its selection for ongoing trials.

• Biomarker discovery efforts are ongoing, with plans to correlate gene expression with clinical outcomes in NEAT.