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

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

IEDAT-02: EDS in Ataxia Telangiectasia Patients (clinicaltrials.gov) - P3; N=180; Not yet recruiting; Sponsor: Erydel

New P3 trial • Biosimilar

Aplidin - dexamethasone in relapsed/refractory myeloma (ADMYRE) (clinicaltrials.gov) - P3, N=250; Recruiting -> Active, not recruiting

Enrollment closed • Hematological Malignancies • Multiple Myeloma

ATM splicing variants as biomarkers for low dose dexamethasone treatment of A-T. (PubMed, Orphanet J Rare Dis) - "For the first time, the expression of ATM splicing variants, similar to those previously observed in vitro, has been found in the PBMCs of patients treated with EryDex. These findings show a correlation between the expression of ATMdexa1 transcripts and the clinical response to low dose dexamethasone administration."

Journal • Biosimilar