Quince Therapeutics (QNCX) Oppenheimer 35th Annual Healthcare Life Sciences Conference (Virtual) 2025 summary

Disease background and unmet need

• Ataxia-telangiectasia (AT) affects about 10,000 patients in the US and Europe, with no approved therapies and a severe, progressive course leading to early disability and reduced lifespan.

• Diagnosis is often delayed due to lack of standard prenatal or early childhood testing, with children typically identified after years of developmental issues.

• Current management is limited to supportive care, as steroids show some neurological benefit but are limited by significant toxicity, especially in this immunosuppressed population.

Technology platform and therapeutic approach

• The platform encapsulates dexamethasone in patients' red blood cells, enabling monthly dosing that achieves both peak and sustained steroid exposure while minimizing toxicity.

• Over 6,000 doses have been administered to 384 patients, including long-term use up to 13 years, with no observed steroid toxicity.

• The approach aims to slow neurological decline in AT without the safety liabilities of conventional steroids.

Clinical development and trial updates

• The prior Phase 3 ATTeST study showed a strong efficacy signal in children aged 6–9, though the overall population narrowly missed statistical significance on the primary endpoint.

• The ongoing NEAT Phase 3 study focuses on the younger age group with a validated primary endpoint (Rescored Modified ICARS), under FDA special protocol assessment.

• Enrollment is expected to complete by Q2, with top-line data anticipated by year-end.