Pipeline

Our proprietary Autologous Intracellular Drug Encapsulation, or AIDE, technology platform is an innovative drug/device combination platform that uses an automated process to encapsulate a drug into a patient’s own red blood cells. Red blood cells have several characteristics that make them a potentially ideal vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Our AIDE technology is designed to harness many of these benefits to allow for new and improved therapeutic options for patients living with high unmet medical needs. The AIDE technology platform may confer benefits over conventional therapies and if so, AIDE has the potential to be applied to a broad range of small or large molecule drugs and biologics.

EryDex is the first product in development that leverages our AIDE technology and is composed of dexamethasone sodium phosphate (DSP) encapsulated in autologous red blood cells targeted to treat a rare pediatric neurodegenerative disease called Ataxia-Telangiectasia, or A-T. DSP is a corticosteroid well known for its anti-inflammatory properties, as well as its dose-limiting toxicity due to adrenal suppression. EryDex is designed to provide the efficacy of corticosteroids and to reduce or eliminate the significant adverse effects that accompany chronic corticosteroid treatment.

Our strategic focus is to complete the pivotal Phase 3 clinical trial (#IEDAT-04-2022/NCT06193200) of EryDex called NEAT (Neurologic Effects of EryDex on Subjects with A-T) to evaluate its safety and efficacy for the treatment of A-T, an inherited autosomal recessive neurodegenerative and immunodeficiency disorder with currently no approved therapeutic treatments in any global market for this rare pediatric disease.

Quince has dosed the first patient and is actively enrolling participants for our global the Phase 3 NEAT study. Patients with A-T interested in participating can learn more about the NEAT study criteria and locations by visiting Clinicaltrials.gov here and Clinical Trials Information System here.

NEAT is an international, multicenter, randomized, double-blind, placebo-controlled study to evaluate the neurological effects of EryDex in patients with A-T. We plan to enroll approximately 86 patients with A-T ages six to nine years old randomized (1:1) between EryDex or placebo, and approximately 20 patients with A-T ages 10 years or older. Participants who complete the full treatment period, complete the study assessments, and provide informed consent will be eligible to transition to an open label extension program after trial completion. The primary efficacy endpoint for the NEAT study will be measured by the change from baseline to last visit completion in Rescored modified International Cooperative Ataxia Rating Scale (RmICARS).

The pivotal Phase 3 NEAT clinical trial will be conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food ad Drug Administration (FDA), which should allow for the submission of a New Drug Application (NDA) and a Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) in 2026, assuming positive study results. The U.S. FDA also granted Fast Track designation for our EryDex System for the treatment of patients with A-T, underscoring the high unmet medical need as there are currently no approved therapeutic treatments for this rare pediatric disease A-T. Additionally, EryDex has received orphan drug designation for the treatment of A-T from the FDA and The European Commission.

We completed an initial patient sizing project with third-party analysis from IQVIA Medical Claims (Dx), PharmetricsPlus (P+), IQVIA Analytics, which confirmed that there are approximately 4,600 diagnosed patients with A-T in the U.S. Quince estimates that there are approximately 5,000 patients with A-T in the U.K. and EU4 countries. Currently, there are no approved treatments for A-T and the global market, based on our internal estimates and assumptions, represents a more than $1 billion peak commercial opportunity. We believe this makes EryDex an ideal lead asset to demonstrate the clinical and commercial potential of our AIDE technology.

• The Lancet Neurology: Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia-telangiectasia (ATTeST): a multicenter, randomized, double-blind, placebo-controlled phase 3 trial describes safety and efficacy results from Quince’s prior Phase 3 ATTeST clinical trial, which was the largest completed study in A-T.

• 53^rd Child Neurology Society (CNS) Annual Meeting: Treatment-Emergent Adverse Events (TEAEs) in Children With Ataxia-Telangiectasia Treated for One Year With Intra-Erythrocyte Dexamethasone Sodium Phosphate (EryDex) poster describes evaluation of treatment-emergent adverse events (TEAEs) in patients with A-T treated with EryDex for one year compared to placebo control.

• 2024 International Congress for Ataxia Research: Growth and Bone Mineral Density (BMD) in Children with Ataxia-Telangiectasia (A-T) Treated with Intra-Erythrocyte Dexamethasone (EryDex) for 24 months describes growth and bone mineral density in patients with A-T treated for 24 months with EryDex.

• 2024 International Congress for Ataxia Research: Cross-sectional Analysis of International Cooperative Ataxia Rating Scale (ICARS) Subcomponent Scores in Children with Ataxia-Telangiectasia (A-T) describes the baseline ICARS subcomponent scores by age in a cross-sectional analysis of treatment-naive patients from the ATTeST dataset and identifies ICARS subcomponents that best reflect progression of disease by age.

Quince also will investigate additional potential indications for EryDex where chronic corticosteroid treatment is – or has the potential to become – a standard of care if there were not corticosteroid-related safety concerns.

We selected Duchenne muscular dystrophy (DMD) as our second development program.

We consider DMD an ideal indication for EryDex as corticosteroids are the standard of care for this rare disease, but their utility is limited by significant chronic toxicity due to adrenal suppression. As a result, corticosteroid treatment in patients with DMD is commonly interrupted during adolescence due to interference with sexual maturation and delayed puberty.

As a next step, we intend to focus on a Phase 2 clinical trial study design to evaluate EryDex for the potential treatment of patients with DMD, including those with corticosteroid intolerance, who represent the majority of the DMD population.

We completed an evaluation process of other potential rare disease indications beyond A-T and Duchenne muscular dystrophy for EryDex where chronic corticosteroid treatment is – or has the potential to become – a standard of care, if there were not corticosteroid-related safety concerns. The prioritized list of other potential rare disease targets under consideration includes: 1) autoimmune hepatitis, 2) dermatomyositis, 3) pemphigus vulgaris, 4) Hashimoto’s encephalopathy, 5) Becker muscular dystrophy, 6) pediatric lupus, 7) juvenile idiopathic arthritis, 8) myasthenia gravis, 9) limb-girdle muscular dystrophy, 10) chronic inflammatory demyelinating polyradiculoneuropathy, and 11) pulmonary sarcoidosis. To support our drug development pipeline expansion in a capital efficient structure, we would prioritize an investigator initiated clinical trial approach to evaluate EryDex for other potential rare disease indications.

The flexibility of the AIDE platform has the potential to provide benefits over conventional therapies and if so, could be applied to a broad range of drugs ranging from small to large molecules, as well as biologics, targeted at rare and debilitating diseases to further expand our drug development pipeline. Potential benefits include better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life which we believe could be an advantage for treating patients.

Please contact bd@quincetx.com regarding licensing opportunities.