Quince Therapeutics (QNCX) LD Micro Main Event XVII summary

Clinical development and pipeline

• Pivotal Phase III study for EryDex in ataxia telangiectasia (A-T), a rare pediatric neurodegenerative disease with no approved treatments and a $1 billion+ market opportunity, is underway, with top-line results expected in Q4 2025.

• The study is risk-mitigated under FDA Special Protocol Assessment, with Fast Track and orphan drug designations from FDA and EMA.

• Prior phase III data showed significant improvement in 6–9-year-old A-T patients and no serious safety concerns over 3+ years, guiding current study design.

• Duchenne muscular dystrophy (DMD) selected as a second indication, with proof-of-concept studies planned for 2024–2025 and top-line data expected in Q4 2025.

• Open-label extension study initiated for NEAT participants, and prior study published in Lancet Neurology.

Technology and product profile

• AIDE technology enables autologous intracellular drug encapsulation, allowing drugs to be loaded into patients' red blood cells at the point of care.

• EryDex encapsulates dexamethasone, providing chronic steroid therapy without typical steroid-related toxicities.

• Over 6,000 doses administered and over 270 patients treated, with several hundred patient-years of data and a clean safety profile.

• IP exclusivity until 2034 globally and 2035 in the U.S.; CE marked in Europe.

Market opportunity and commercial strategy

• A-T affects about 10,000 patients across the U.S. and EU, with first-to-market potential and minimal cost of goods.

• Pricing strategy informed by recent rare disease drug approvals, with expected high reimbursement and minimal production costs.

• High physician interest and significant unmet need among pediatric neurologists, with high expected patient uptake upon approval.

• Manufacturing infrastructure is highly scalable, with low direct costs.

• Plans to expand into additional rare disease indications, including neuromuscular, hematology, and autoimmune disorders.