Quince Therapeutics (QNCX) Piper Sandler 37th Annual Healthcare Conference summary

Key technology and therapy overview

• Acquired a drug-device technology that encapsulates dexamethasone in a patient's red blood cells, enabling chronic dosing with reduced toxicity.

• The process uses a bedside machine to process a small blood sample and re-infuse it with the drug after 90 minutes.

• Enables once-monthly dosing, aiming to maintain efficacy while minimizing steroid-related toxicities.

Clinical development and trial design

• Previous phase 3 (ATTEST) missed significance in the overall population but showed strong benefit in ages 6–9, leading to the NEAT study focusing on this group.

• Ataxia-telangiectasia patients experience rapid neurological decline between ages 2–10, making this age group optimal for clinical trials.

• The NEAT study uses the RmICARS scale (focused on gait and posture) as the primary endpoint, per FDA guidance.

• Special Protocol Assessment agreement with FDA allows a single pivotal trial for approval if successful.

Safety and efficacy monitoring

• Safety data monitoring committee (DSMB) has reviewed both blinded and unblinded data three times, finding no safety concerns.

• Extensive safety data from long-term open-label extension studies support a strong safety profile.

• Efficacy will be determined in Q1, with a 1.5-point change on the 29-point RmICARS considered statistically significant.

• The trial is powered to detect a 2.5-point difference, which could mean the difference between walking autonomously and with support.