Quince Therapeutics (QNCX) Investor Update summary

Technology Overview and Clinical Development

• EryDex is an autologous erythrocyte-encapsulated dexamethasone therapy using AIDE technology to optimize delivery and reduce steroid toxicity, enabling monthly infusions for ataxia telangiectasia (AT).

• The Phase 3 NEAT trial is ongoing, focusing on children aged 6–9, with topline data expected in Q4 2025; cash runway extends into 2026.

• The NEAT trial is a global, multicenter, randomized, double-blind, placebo-controlled study under FDA Special Protocol Assessment, supporting NDA submission upon positive results.

• Open-label extension has begun, with 17 participants transitioned in the US, UK, and EU.

• NDA and MAA submissions are planned for 2026, pending positive results.

Disease Background and Unmet Need

• AT is a rare, autosomal recessive neurodegenerative disorder caused by ATM gene mutations, with early childhood onset, progressive ataxia, immunodeficiency, and high cancer risk.

• No curative or disease-modifying therapies exist; current treatments are supportive, with systemic steroids limited by severe side effects.

• The most rapid neurological decline occurs between ages 6–9, making this group a key target for intervention.

• Approximately 4,600 diagnosed U.S. patients; high unmet medical need persists.

Clinical Trial Results and Endpoints

• The ATHEST/ATTEST Phase 3 trials showed statistically significant slowing of neurological decline in 6–9-year-olds using EryDex, with a five-point difference in modified ICARS scores versus placebo over six months.

• EryDex slowed neurological deterioration in all ages, with significant improvement in the 6–9 year-old subgroup across multiple endpoints.

• The NEAT trial uses the rescored modified ICARS (RmICARS), focusing on posture and gait, as the primary endpoint, per FDA guidance.

• Treatment involves six infusions every 21–30 days, randomized 1:1 between EryDex and placebo.