dexamethasone sodium phosphate-loaded RBC (EDS-EP) / Quince Therap - LARVOL DELTA

OLE_NEAT: Open-Label Extension of EryDex Study IEDAT-04-2022 (clinicaltrials.gov) - P3 | N=101 | Terminated | Sponsor: Quince Therapeutics S.p.A. | Trial completion date: Nov 2026 ➔ Jan 2026 | Recruiting ➔ Terminated | Trial primary completion date: Nov 2026 ➔ Jan 2026; eDSP development discontinued

Trial completion date • Trial primary completion date • Trial termination • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

NEAT: Evaluate the Neurological Effects of EryDex on Subjects With A-T (clinicaltrials.gov) - P3 | N=105 | Completed | Sponsor: Quince Therapeutics S.p.A. | Active, not recruiting ➔ Completed

Trial completion • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

Use of encapsulated dexamethasone sodium phosphate (eDSP) in chronic obstructive pulmonary disease, cystic fibrosis, and inflammatory bowel disorders. (PubMed, Front Drug Deliv) - "Early studies demonstrate that eDSP is a feasible and well-tolerated treatment in children and older patients, delivering low-dose corticosteroids with prolonged therapeutic levels. These findings support further development of erythrocyte-based drug delivery for chronic inflammatory diseases in patients with steroid sensitive or steroid-dependent disease."

Journal • Review • Chronic Obstructive Pulmonary Disease • Crohn's disease • Cystic Fibrosis • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Immunology • Infectious Disease • Inflammation • Inflammatory Bowel Disease • Pediatrics • Pulmonary Disease • Respiratory Diseases • Ulcerative Colitis

Quince Therapeutics Announces Publication of Use of eDSP in Early-Stage Clinical Studies in Pulmonary and Inflammatory Bowel Disorders (Businesswire) - "Highlights include: Early-stage clinical studies investigated eDSP in patients, whose age ranged from five to 83 years, with chronic obstructive pulmonary disease (COPD), cystic fibrosis (CF), Crohn’s disease (CD), and ulcerative colitis (UC)...eDSP resulted in improved FEV1 and reduced infections in CF patients and improved symptoms in COPD with markedly reduced corticosteroid doses. In IBD, eDSP at low doses enabled corticosteroid withdrawal in 60% to 78% of patients and achieved remission in pediatric and adult CD and UC...Early studies demonstrate that eDSP is a feasible and well-tolerated treatment in children and older patients, delivering low-dose corticosteroids with prolonged therapeutic levels."

Retrospective data • Ataxia • Chronic Obstructive Pulmonary Disease • Crohn's disease • Cystic Fibrosis • Ulcerative Colitis

Lets talk about ataxia-telangiectasia: Meeting report of the AT clinical research conference June 2025. (PubMed, DNA Repair (Amst)) - "The implications of these roles of ATM in protecting the genome/cell and the development of new technology, such as organoids, were widely discussed in the clinical setting of patients with A-T. An important contribution to the meeting was the description of pathways /mechanisms that have led to the development of therapeutic approaches for A-T including the use of specific antisense oligonucleotides to restore ATM function in patients; delivery of full-length ATM cDNA to A-T cells; eDSP (formerly EryDex) that encapsulates dexamethasone sodium phosphate in a patient's own red blood cells and the use of small molecules (triheptanoin, nicotinamide riboside and N-acetyl leucine) to correct mitochondrial and metabolic function."

Journal • Ataxia • CNS Disorders • Immunology • Metabolic Disorders • Movement Disorders • Primary Immunodeficiency

Population Pharmacokinetic Modeling and Pediatric Exposure of Dexamethasone Sodium Phosphate Encapsulated in Erythrocytes (eDSP) Administered Monthly for Treatment of Neurological Symptoms of Patients With Ataxia Telangiectasia. (PubMed, CPT Pharmacometrics Syst Pharmacol) - "The EryDex System (EDS) is a drug/device combination, which has been tested in clinical trials for ataxia telangiectasia (AT)...Monte Carlo simulations of eDSP showed a rapid peak at 0.67 h, followed by sustained dexamethasone release; faster in the first 24 h, then slower over 20-30 days. No accumulation occurred with once-monthly dosing."

Journal • PK/PD data • Ataxia • Immunology • Movement Disorders • Pediatrics • Primary Immunodeficiency

NEAT: Evaluate the Neurological Effects of EryDex on Subjects With A-T (clinicaltrials.gov) - P3 | N=105 | Active, not recruiting | Sponsor: Quince Therapeutics S.p.A. | Recruiting ➔ Active, not recruiting | Trial primary completion date: Aug 2025 ➔ Dec 2025

Enrollment closed • Trial primary completion date • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

Quince Therapeutics to Host Investor Webinar Today Focused on Addressing the High Unmet Need in Ataxia-Telangiectasia (Businesswire) - 'Quince also disclosed that the company has enrolled 46 participants to date in its pivotal Phase 3 NEAT...clinical trial to evaluate the neurological effects of EryDex in patients with A-T...'We continue to make significant progress with clinical site activations and enrollment of our pivotal Phase 3 NEAT clinical trial. With 61 participants screened to date, enrollment is nearing 50%...We anticipate completing enrollment during the second quarter of 2025 and reporting topline results before the end of 2025.'"

Enrollment status • P3 data: top line • Ataxia

Long-term safety of dexamethasone sodium phosphate encapsulated in autologous erythrocytes in pediatric patients with ataxia telangiectasia. (PubMed, Front Neurol) - P3 | "Dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes (EryDex) was developed as an alternative to standard glucocorticoids in an effort to eliminate chronic steroid toxicity while preserving efficacy...Adverse events typically observed with prolonged glucocorticoid use such as Cushingoid features, weight gain, hypertension, hirsutism, diabetes or stunted growth were rarely reported. ClinicalTrials.gov, identifiers: NCT02770807 and NCT03563053."

Journal • Ataxia • Cardiovascular • Dermatology • Diabetes • Hematological Disorders • Hypertension • Immunology • Metabolic Disorders • Movement Disorders • Pediatrics • Primary Immunodeficiency • Pruritus • CD4

Quince is currently enrolling the pivotal Phase 3 NEAT clinical trial… (Businesswire) - "The Phase 3 NEAT trial is being conducted under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA), and the company expects to report topline results in the fourth quarter of 2025 with a potential New Drug Application (NDA) submission to the FDA and a Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) in 2026, assuming positive study results."

EMA filing • FDA filing • P3 data: top line • Ataxia • Genetic Disorders

Quince Therapeutics Announces Frontiers in Neurology Publication of Long-Term Safety of EryDex in Pediatric Patients with Ataxia-Telangiectasia (Businesswire) - P3 | N=104 | OLE-IEDAT (NCT03563053) | P3 | N=176 | ATTeST (NCT02770807) | Sponsor: Quince Therapeutics S.p.A. | "Results include analyses of data from patients with A-T treated with EryDex for a minimum of 24 months...Adverse events typically observed with prolonged glucocorticoid use such as Cushingoid features, weight gain, hypertension, hirsutism, diabetes, or stunted growth were infrequently reported over 24 to 61 months of dosing. There was a decline in bone mineral density (BMD) of 0.4 z scores over the two-year period – which could not be distinguished from the natural course of disease in patients with A-T. There were no reported adverse effects on height, weight and body mass index as supported by stable z-scores throughout the two years of treatment. Values for glucose, HbA1c, cortisol, and CD4+ lymphocyte counts did not show clinically significant changes during prolonged treatment with EryDex."

P3 data • Ataxia • Genetic Disorders

OLE_NEAT: Open-Label Extension of EryDex Study IEDAT-04-2022 (clinicaltrials.gov) - P3 | N=106 | Recruiting | Sponsor: Quince Therapeutics S.p.A. | Not yet recruiting ➔ Recruiting

Enrollment open • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

OLE_NEAT: Open-Label Extension of EryDex Study IEDAT-04-2022 (clinicaltrials.gov) - P3 | N=106 | Not yet recruiting | Sponsor: Quince Therapeutics S.p.A.

New P3 trial • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

OLE-IEDAT: Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study (clinicaltrials.gov) - P3 | N=104 | Terminated | Sponsor: Quince Therapeutics S.p.A. | N=50 ➔ 104 | Recruiting ➔ Terminated; terminated by sponsor

Enrollment change • Trial termination • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

The Latest Developments for the Treatment of Ataxia Telangiectasia: A Narrative Review. (PubMed, Cerebellum) - "Most of the treatments under investigation are in the early stages, except for the EryDex System. It appears that the EryDex system and N-Acetyl-DL-Leucine may hold promise as potential treatment options."

Journal • Review • Ataxia • Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Immunology • Movement Disorders • Primary Immunodeficiency • Transplantation • ATM

Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia (ATTeST): a multicentre, randomised, double-blind, placebo-controlled phase 3 trial. (PubMed, Lancet Neurol) - P3 | "Although there were no safety concerns, the primary efficacy endpoint was not met, possibly related to delays in treatment reducing the number of participants who received treatment as outlined in the protocol, and potentially different treatment effects according to age. Studies of intra-erythrocyte delivery of dexamethasone sodium phosphate will continue in participants aged 6-9 years, on the basis of findings from subgroup analyses from this trial."

Clinical • Journal • P3 data • Ataxia • Cardiovascular • CNS Disorders • Diabetes • Hypertension • Immunology • Movement Disorders • Primary Immunodeficiency

NEAT: To Evaluate the Effects of EryDex in Patients With A-T (clinicaltrials.gov) - P3 | N=106 | Recruiting | Sponsor: Quince Therapeutics S.p.A. | Not yet recruiting ➔ Recruiting

Enrollment open • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

NEAT: To Evaluate the Effects of EryDex in Patients With A-T (clinicaltrials.gov) - P3 | N=106 | Not yet recruiting | Sponsor: Erydel

New P3 trial • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

New human ATM variants are able to regain ATM functions in ataxia telangiectasia disease. (PubMed, Cell Mol Life Sci) - "No cure is currently available for these patients but positive effects on neurologic features in AT patients have been achieved by dexamethasone administration through autologous erythrocytes (EryDex) in phase II and phase III clinical trials, leading us to explore the molecular mechanisms behind the drug action...These outcomes are triggered by the kinase and further functional domains of the tested ATM variants, that are useful for restoring cellular functionality. The in silico designed ATM variant eliciting most of the functionality recover may be exploited in gene therapy or gene delivery for the treatment of AT patients."

Journal • Ataxia • CNS Disorders • Gene Therapies • Immunology • Movement Disorders • Primary Immunodeficiency • ATM

OLE-IEDAT: Open-label, Long-term, Extension Treatment Using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients With Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study (clinicaltrials.gov) - P3; N=50; Recruiting; Sponsor: Erydel; Trial completion date: Aug 2020 ➔ Dec 2021; Trial primary completion date: Aug 2020 ➔ Dec 2021

Clinical • Trial completion date • Trial primary completion date • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency • CD4

ATTeST: EDS in Ataxia Telangiectasia Patients (clinicaltrials.gov) - P3; N=175; Completed; Sponsor: Erydel; Recruiting ➔ Completed

Trial completion • Ataxia • Immunology • Movement Disorders • Primary Immunodeficiency

Temsirolimus, dexamethasone, mitoxantrone hydrochloride, vincristine sulfate, and pegaspargase in treating young patients with relapsed acute lymphoblastic leukemia or non-Hodgkin lymphoma (clinicaltrials.gov) - P1, N=60; Not yet recruiting -> Recruiting; Start date: Aug '11 -> Oct '11

Enrollment open • Start date • Oncology

FUS1-nanoparticles and erlotinib in stage IV lung cancer (clinicaltrials.gov) - P1/2, N=51; Not yet recruiting; New P1/2 trial

New trial • Oncology

The In Vivo Recovery/Survival and Pharmacokinetic Properties of Dexamethasone Sodium Phosphate Encapsulated in Autologous Erythrocytes (ASH 2016) - "The EryDex System (EDS) is an automated system that loads RBC ex vivo using hypotonic opening of RBC followed by hypertonic resealing of the RBC and washing to prepare the DSP-RBC for infusion. The results for the mean RBC in vivo recovery for DSP-loaded EDS-processed cells meet the FDA criteria for 24-hour RBC recovery of ≥ 75%, without adverse impact on the survival of EDS-processed RBCs. Most of the dexamethasone was rapidly released from the RBCs in vivo with a maximum peak occurring 1 hour after the end of the intravenous infusion, independent of the dose administered, but sustained release of dexamethasone could be detected until 14 and 35 days post infusion for the low and high doses, respectively. DSP-loaded autologous RBCs prepared using the EDS delivered a sustained dose of dexamethasone in vivo."

Biosimilar • Crohn's Disease • Hematological Malignancies • Immunology • Inflammatory Bowel Disease • Oncology

ATTeST: EDS in Ataxia Telangiectasia Patients (clinicaltrials.gov) - P3; N=180; Recruiting; Sponsor: Erydel; Not yet recruiting ➔ Recruiting

Enrollment open • Biosimilar