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

Disease background and unmet need

• Ataxia-telangiectasia (AT) is a severe, inherited neurodegenerative disorder affecting about 10,000 patients in the US and Europe, with no approved therapies and a life expectancy into the mid-20s.

• Diagnosis is often delayed due to lack of standard prenatal or early childhood testing, leading to years of undetected progression.

• Current management is limited to supportive care, with no disease-modifying treatments available.

Technology 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 in 384 patients, including long-term use up to 13 years, have shown no steroid toxicity.

• The approach aims to slow neurological decline without the immunosuppressive risks of conventional steroids.

Clinical development and data

• The prior Phase 3 ATTeST study narrowly missed statistical significance overall but showed strong efficacy in patients aged 6–9, leading to the design of the NEAT study focused on this group.

• The NEAT Phase 3 trial is ongoing, with enrollment expected to complete by Q2 and top-line data anticipated by year-end.

• The primary endpoint is the Rescored Modified ICARS, a 29-point subset focused on gait and posture, agreed upon with the FDA under a special protocol assessment.