vQuince Therapeutics Reports Topline Results from Phase 3 NEAT Trial of eDSP in Ataxia-Telangiectasia
Quince Therapeutics a late-stage biotechnology company focused on developing therapies for rare diseases by leveraging the biology of a patient’s own cells, has announced topline results from its pivotal Phase 3 NEAT clinical trial evaluating its lead candidate, eDSP, in patients with Ataxia-Telangiectasia (A-T). While the therapy demonstrated a favorable safety and tolerability profile, the study did not meet its primary or key secondary efficacy endpoints. As a result, the company plans to discontinue further clinical development of eDSP and shift its focus toward preserving capital and assessing strategic alternatives.
The announcement marks a significant development not only for Quince Therapeutics but also for the global A-T patient community, which continues to face limited treatment options for this rare, progressive neurodegenerative disorder.
Ataxia-Telangiectasia
Ataxia-Telangiectasia is a rare, inherited, multisystem disorder caused by mutations in the ATM gene, which plays a crucial role in DNA repair and cellular stress responses. The disease typically manifests in early childhood and is characterized by progressive loss of motor coordination (ataxia), immune system dysfunction, increased susceptibility to infections, and a heightened risk of certain cancers. Patients may also develop oculocutaneous telangiectasias, speech difficulties, and other neurological complications.
A-T remains an area of significant unmet medical need. There are currently no disease-modifying therapies approved specifically to halt or reverse neurological progression in A-T. Management largely focuses on supportive care, infection control, and monitoring for complications. Given the complexity of the disease and the vulnerability of the patient population, therapeutic development in A-T presents substantial scientific and clinical challenges.
Overview of eDSP and Its Mechanism
Quince’s investigational therapy, eDSP (dexamethasone sodium phosphate encapsulated in autologous erythrocytes), was designed as a novel drug delivery approach intended to provide sustained, low-dose exposure to dexamethasone while potentially minimizing systemic side effects commonly associated with chronic corticosteroid use.
The technology involves encapsulating dexamethasone sodium phosphate inside a patient’s own red blood cells (autologous erythrocytes). These modified cells are then reinfused into the patient, acting as a circulating depot that gradually releases the active drug over time. The strategy aimed to harness the anti-inflammatory and potential neuroprotective effects of corticosteroids while avoiding the peaks and troughs of conventional dosing.
Earlier-stage clinical work had suggested potential signals of benefit, supporting advancement into a global Phase 3 program. The NEAT trial was therefore designed to provide definitive evidence of efficacy and safety in a larger A-T population
Design of the Phase 3 NEAT Study
The NEAT study was an international, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial. A total of 105 patients with Ataxia-Telangiectasia were enrolled. Participants were randomized to receive either eDSP or placebo, with efficacy assessed over a six-month treatment period.
The primary endpoint of the trial was the change from baseline to the last efficacy visit at month six in the Rescored modified International Cooperative Ataxia Rating Scale (RmICARS), a widely used clinical scale that evaluates neurological function and ataxia severity. The RmICARS measures multiple domains of motor performance, including posture, gait, limb coordination, and speech.
A key secondary endpoint assessed changes in disease severity using the Clinical Global Impression of Severity (CGI-S) scale, a clinician-rated measure of overall illness severity.
Primary Endpoint Results
According to the topline data, the study did not achieve statistical significance for its primary endpoint. Patients in the active treatment arm experienced a mean change from baseline to month six of 0.94 points on the RmICARS scale, compared with a mean change of 2.24 points in the placebo arm. The calculated difference between the groups was –1.30 points in favor of eDSP.
However, this difference did not meet the predefined threshold for statistical significance, with a p-value of 0.0851. Although the numerical trend favored the active treatment, the results fell short of the level of certainty required to establish efficacy in a pivotal trial setting.
Key Secondary Endpoint Findings
The study also did not meet its key secondary endpoint related to the Clinical Global Impression of Severity (CGI-S). Changes from baseline to month six on the CGI-S scale did not demonstrate a statistically meaningful difference between the eDSP and placebo groups. The p-value associated with this comparison was 0.522, indicating no significant separation between treatment arms on this clinician-based global assessment.
Together, the primary and secondary endpoint results led to the overall conclusion that the NEAT trial did not provide sufficient evidence of clinical benefit for eDSP in A-T.
Safety and Tolerability Profile
Despite the lack of efficacy, eDSP was generally well tolerated in the study population. The safety data did not reveal any clinically meaningful concerns, an important consideration in a medically fragile group such as A-T patients.
The most commonly reported adverse events in the eDSP treatment arm were pruritis (itching) and pyrexia (fever). No new or unexpected safety signals emerged, and the overall tolerability profile was consistent with prior experience with the therapy.
These findings suggest that the erythrocyte-encapsulation approach did not introduce significant additional safety risks, even though the clinical benefit endpoints were not met.
Company Response and Strategic Direction
In light of the trial outcome, Quince Therapeutics has decided to cease further clinical development of eDSP. The company indicated that it will prioritize cash preservation and evaluate available strategic options moving forward. Such options may include restructuring, partnerships, licensing arrangements, or other corporate actions aimed at maximizing value.
Dr. Dirk Thye, Chief Executive Officer and Chief Medical Officer of Quince Therapeutics, acknowledged the disappointment associated with the results while expressing appreciation for the broader A-T community.
He stated that the company extends its compassion and hope for future therapeutic advances for individuals living with A-T. He also conveyed gratitude to the patients and families who participated in the study, as well as to academic investigators, clinical sites, and Quince employees who contributed to the program over many years.
Implications for the A-T Community
The outcome of the NEAT trial represents a setback in the search for effective treatments for Ataxia-Telangiectasia. However, clinical research in rare neurodegenerative diseases often involves significant uncertainty, and negative results can still provide valuable scientific insights. Data from the study may help inform future trial designs, endpoint selection, and therapeutic strategies in A-T and related conditions.
For patients and families affected by A-T, the need for disease-modifying therapies remains urgent. Continued collaboration among researchers, clinicians, advocacy groups, and biopharmaceutical companies will be critical to advancing new approaches.
While Quince Therapeutics shifts its strategic focus, the broader field of rare disease research continues to evolve. Advances in gene therapy, targeted molecular treatments, and innovative drug delivery systems are being explored across multiple neurogenetic disorders.
Although eDSP will not move forward, the knowledge gained from the NEAT study contributes to the collective understanding of A-T and underscores the importance of rigorous late-stage clinical testing. The hope remains that ongoing and future research efforts will ultimately yield effective therapies for patients living with this challenging disease.
About eDSP
eDSP is comprised of dexamethasone sodium phosphate (DSP) encapsulated in a patient’s own red blood cells (autologous erythrocytes). DSP is a corticosteroid well known for its anti-inflammatory properties as well as its dose-limiting toxicity due to adrenal suppression. The eDSP System is designed to provide the efficacy of corticosteroids and to reduce or eliminate the significant adverse effects that accompany chronic use of corticosteroid treatment.
eDSP leverages Quince’s proprietary Autologous Intracellular Drug Encapsulation, or AIDE, technology platform, which is a novel drug/device combination that uses an automated process designed to encapsulate a drug into the patient’s own red blood cells. Red blood cells have several characteristics that make them a potentially effective vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Quince’s AIDE technology is designed to harness these benefits to allow for the chronic administration of drugs that have limitations due to toxicity, poor biodistribution, suboptimal pharmacokinetics, or immune response.
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