X4 Pharmaceuticals to Present Trial-in-Progress Poster on Phase 3 Study of Mavorixafor in Chronic Neutropenia at 66th ASH Annual Meeting & Exposition
X4 Pharmaceuticals (XFOR) announced upcoming presentations at the 66th ASH Annual Meeting, featuring a trial-in-progress poster on their Phase 3 4WARD clinical trial of mavorixafor for chronic neutropenia (CN). The trial is evaluating the oral CXCR4 antagonist as both monotherapy and combined with injectable G-CSF.
The 52-week study aims to enroll 150 participants across 90-110 sites in 20-25 countries. It's designed as a randomized, double-blind, placebo-controlled trial for patients with congenital, acquired primary autoimmune, or idiopathic CN experiencing recurrent infections.
The company will also present preclinical data on CXCR4 antagonism's potential to correct neutropenia and bone marrow neutrophil accumulation.
X4 Pharmaceuticals (XFOR) ha annunciato prossime presentazioni alla 66ª Riunione Annuale ASH, con un poster su uno studio in corso riguardante il loro trial clinico di fase 3 4WARD per il mavorixafor nella neutropenia cronica (CN). Lo studio sta valutando l'antagonista orale del CXCR4 sia come monoterapia che in combinazione con G-CSF iniettabile.
Lo studio di 52 settimane mira a reclutare 150 partecipanti in 90-110 centri in 20-25 paesi. È progettato come uno studio randomizzato, in doppio cieco e controllato con placebo per pazienti con CN congenita, acquisita primaria autoimmune o idiopatica che presentano infezioni ricorrenti.
La società presenterà anche dati preclinici sul potenziale dell'antagonismo del CXCR4 di correggere la neutropenia e l'accumulo di neutrofili nel midollo osseo.
X4 Pharmaceuticals (XFOR) anunció presentaciones próximas en la 66ª Reunión Anual de la ASH, destacando un póster sobre un ensayo en progreso de su ensayo clínico de fase 3 4WARD del mavorixafor para la neutropenia crónica (CN). El ensayo evalúa el antagonista oral CXCR4 tanto como monoterapia como en combinación con G-CSF inyectable.
El estudio de 52 semanas tiene como objetivo inscribir a 150 participantes en 90-110 sitios en 20-25 países. Está diseñado como un ensayo aleatorio, doble ciego y controlado por placebo para pacientes con CN congénita, adquirida primaria autoinmune o idiopática que experimentan infecciones recurrentes.
La compañía también presentará datos preclínicos sobre el potencial del antagonismo de CXCR4 para corregir la neutropenia y la acumulación de neutrófilos en la médula ósea.
X4 제약 (XFOR)는 66회 ASH 연례 회의에서 발표할 예정이며, 만성 신생아 백혈구 감소증 (CN)을 위한 그들의 3상 4WARD 임상 시험에 대한 진행 중인 시험 포스터를 소개할 것입니다. 이 시험은 경구적으로 복용하는 CXCR4 길항제를 단독 요법 및 주사형 G-CSF와 병행하여 평가하고 있습니다.
52주 연구는 20-25개 국가의 90-110개 사이트에서 150명의 참가자를 모집할 계획입니다. 이 연구는 선천성, 후천성 1차 자가 면역 또는 원인 불명의 CN으로 재발 감염이 발생하는 환자들을 위한 무작위 이중 맹검 위약 대조 임상 시험으로 설계되었습니다.
회사는 또한 CXCR4 길항 요법이 백혈구 감소증을 교정하고 골수의 호중구 축적을 개선할 수 있는 잠재력에 대한 전임상 데이터를 발표할 것입니다.
X4 Pharmaceuticals (XFOR) a annoncé de prochaines présentations lors de la 66e Réunion Annuelle ASH, présentant un poster sur un essai en cours concernant leur essai clinique de phase 3 4WARD du mavorixafor pour la neutropénie chronique (CN). L'essai évalue l'antagoniste oral CXCR4 à la fois en monothérapie et en combinaison avec le G-CSF injectable.
Cette étude de 52 semaines vise à recruter 150 participants dans 90-110 sites dans 20-25 pays. Elle est conçue comme un essai randomisé, en double aveugle et contrôlé par placebo pour des patients présentant une CN congénitale, acquise, primaire auto-immune ou idiopathique et souffrant d'infections récurrentes.
La société présentera également des données précliniques sur le potentiel de l'antagonisme de CXCR4 à corriger la neutropénie et l'accumulation de neutrophiles dans la moelle osseuse.
X4 Pharmaceuticals (XFOR) hat bevorstehende Präsentationen auf dem 66. ASH-Jahrestreffen angekündigt, darunter ein Poster zu einem in Arbeit befindlichen Versuch zu ihrem Phase 3 4WARD-Studie von Mavorixafor zur chronischen Neutropenie (CN). Die Studie bewertet den oralen CXCR4-Antagonisten sowohl als Monotherapie als auch in Kombination mit injizierbarem G-CSF.
Die 52-wöchige Studie zielt darauf ab, 150 Teilnehmer in 90-110 Standorten in 20-25 Ländern zu rekrutieren. Sie ist als randomisierte, doppelblinde, placebokontrollierte Studie für Patienten mit kongenitaler, erworbener primärer Autoimmun- oder idiopathischer CN konzipiert, die an wiederkehrenden Infektionen leiden.
Das Unternehmen wird auch präklinische Daten zum Potenzial der CXCR4-Antagonisierung vorstellen, die Neutropenie zu korrigieren und die Ansammlung von Neutrophilen im Knochenmark zu verringern.
- First potential treatment innovation for chronic neutropenia in 30 years
- Large-scale Phase 3 trial with 150 participants across multiple countries
- Potential to reduce or replace injectable treatments with oral medication
- Still in clinical trial phase with no guaranteed success
- Extended timeline with 52-week trial duration
Pivotal Phase 3 4WARD clinical trial is evaluating use of oral mavorixafor as monotherapy and in combination with injectable G-CSF
BOSTON, Dec. 04, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced its upcoming presentations at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, including a trial-in-progress poster on the ongoing pivotal Phase 3 clinical trial exploring the use of mavorixafor, an oral CXCR4 antagonist, for the treatment of people with chronic neutropenia (CN). The meeting will take place December 7-10, 2024, in San Diego, CA.
“We’re pleased to be sharing details of our ongoing Phase 3 clinical trial at ASH as we continue to recruit patients and activate study sites across the globe. The initiation of this trial was a significant milestone for the chronic neutropenia community that – unfortunately – hasn’t seen any treatment innovation in nearly 30 years,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “We look forward to connecting with clinicians at the ASH meeting and sharing more about our vision for mavorixafor as an oral option for the treatment of CN that could potentially reduce or replace the use of injectable G-CSF.”
The Phase 3 4WARD trial is evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without stable doses of injectable G-CSF) in people with congenital, acquired primary autoimmune or acquired primary idiopathic CN who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants across 90 to 110 sites in 20 to 25 countries.
X4 will also be hosting a booth (#2106) at the conference to share information about the ongoing 4WARD clinical trial.
Additionally, in an oral presentation at the meeting, the company will present preclinical data on the potential for CXCR4 antagonism to correct peripheral blood neutropenia and bone marrow neutrophil accumulation caused by a loss-of-function in the CXCR2 pathway.
| Poster Presentation Details: | |
| Title: “Trials in Progress: A Phase 3 Study to Investigate Efficacy, Safety, and Tolerability of Mavorixafor in Participants with Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders” | |
| Number: 3924.1 | |
| Date and Time: Monday, December 9, 2024: 6:00-8:00 p.m. PST | |
| Location: Manchester Grand Hyatt San Diego, Halls G-H | |
| Oral Presentation Details: | |
| Title: “CXCR4 Antagonism Corrects Peripheral Neutropenia and Mature Neutrophil Accumulation in Bone Marrow in a Pharmacological Mouse Model of CXCR2 Loss-of-Function” | |
| Number: 418 | |
| Date and Time: Sunday, December 8, 2024: 10:15 a.m. PST | |
| Location: Manchester Grand Hyatt San Diego, Grand Hall D | |
About the 4WARD Global, Pivotal, Phase 3 Clinical Trial
The 4WARD trial is a global, pivotal Phase 3 clinical trial (NCT06056297) evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in people with congenital, acquired primary autoimmune or acquired primary idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants with confirmed trough ANC levels less than 1,500 cells per microliter at baseline screening and histories of two or more serious and/or recurrent infections in the prior year. The primary endpoint of the trial is based on two outcome measures: annualized infection rate and positive ANC response.
About X4 Pharmaceuticals
X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging our expertise in CXCR4 and immune system biology, we have successfully developed mavorixafor, which has received U.S. approval as XOLREMDI® (mavorixafor) capsules in its first indication. We are also evaluating the use of mavorixafor in additional potential indications. X4 corporate headquarters are in Boston, Massachusetts and our research center of excellence is in Vienna, Austria. For more information, please visit our website at www.x4pharma.com.
Company Contact:
José Juves
Head of Corporate & Patient Affairs
jose.juves@x4pharma.com
Investor Contact:
Daniel Ferry
Managing Director, LifeSci Advisors
daniel@lifesciadvisors.com
(617) 430-7576
FAQ
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