Quince Therapeutics (QNCX) Investor Update summary

Disease Overview and Unmet Need

• Ataxia telangiectasia (A-T) is a rare, progressive neurodegenerative disorder caused by ATM gene mutations, leading to severe motor impairment, immunodeficiency, and increased cancer risk, with most patients not surviving beyond 30 years.

• Approximately 4,600 diagnosed A-T patients are in the US, but the true number may be higher; current therapies are only supportive, with no disease-modifying or curative options.

• Rapid neurological progression occurs between ages 6-9, significantly impacting mobility and life expectancy.

Technology and Clinical Development

• EryDex, an autologous erythrocyte-encapsulated dexamethasone, uses AIDE technology to enable monthly infusions, optimizing drug delivery and reducing systemic steroid toxicity.

• Phase 3 ATHEST and NEAT trials showed statistically significant slowing of neurological decline in 6–9-year-old A-T patients, with a favorable safety profile and minimal steroid-related side effects.

• Long-term data from open-label extensions and expanded access programs show sustained benefit and lack of chronic steroid toxicity, even in patients treated for over a decade.

• The NEAT pivotal phase 3 trial is a global, multicenter, randomized, double-blind, placebo-controlled study under FDA Special Protocol Assessment, enrolling 6–9-year-olds as the primary population, with top-line data expected in Q4 2025.

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

Regulatory and Operational Updates

• The NEAT study uses the rescored modified ICARS (RmICARS) as the primary endpoint, focusing on posture and gait, and is powered based on prior ATHEST results.

• The FDA recommended including patients 10 years and older to potentially broaden the label, and a pediatric plan is in place to study children under six using a lower blood volume process.

• Cash runway extends into 2026, expected to cover completion of the pivotal trial.