Business Details
12 Years
experience
Innovative Rare Disease Therapies | X4 Pharmaceuticals
At X4 Pharmaceuticals, we are dedicated to advancing innovative therapies for individuals living with rare immune disorders. Our expertise in CXCR4 biology has enabled the successful development of mavorixafor, now marketed as XOLREMDI, the first FDA-approved treatment for WHIM syndrome. We are committed to addressing significant unmet medical needs through rigorous scientific research and patient-centric care. Our global partnerships and clinical trial initiatives reflect our mission to expand access to life-changing therapies. With a leadership team boasting decades of experience in rare disease research and drug development, we maintain a steadfast focus on quality, integrity, and patient outcomes.Our operations span the United States, European Union, and MENA region, supported by a dedicated commercial team and strategic distribution agreements. Through programs like X4Connect, we provide comprehensive support to patients and caregivers throughout their treatment journey. Our core values of excellence, accountability, and urgency drive our efforts to alleviate human suffering and cure disease. As a publicly traded company on the Nasdaq, we remain focused on long-term value creation while maintaining transparency and ethical standards in all our operations.
Services
Rare disease therapies
WHIM syndrome treatment
Clinical trial enrollment
Patient support programs
Global drug distribution
Details
- Year established: 2014
Highlights and features
- We developed the first FDA-approved therapy for WHIM syndrome
- Our team has decades of experience in rare disease research
- We conduct pivotal global Phase 3 clinical trials
- We partner with Norgine for EU distribution of XOLREMDI
- Our X4Connect program provides comprehensive patient support
Services
WHIM Syndrome Treatment
X4 Pharmaceuticals offers XOLREMDI (mavorixafor), the first FDA-approved oral therapy for WHIM syndrome. This groundbreaking treatment increases circulating white blood cells in patients aged 12+. Our development leveraged 25+ years of CXCR4 biology expertise, with global distribution partnerships established for EU and MENA regions following regulatory approvals.
Chronic Neutropenia Research
We conduct pivotal Phase 3 4WARD trials evaluating mavorixafor for chronic neutropenic disorders. This research addresses a 30-year treatment gap for patients experiencing recurrent infections. The global trial assesses oral therapy with/without G-CSF, offering potential alternatives to injectable treatments through rigorous clinical investigation.
Patient Support Programs
X4Connect provides dedicated support for XOLREMDI patients, including insurance navigation and copay assistance. Our program operates Monday-Friday 8am-8pm ET, offering disease education and treatment resources. This comprehensive support system ensures patients maintain consistent access to critical therapies throughout their care journey.
Global Drug Commercialization
We've established strategic partnerships with Norgine for EU distribution and taiba rare for MENA region commercialization of XOLREMDI. These collaborations leverage regional expertise while maintaining quality control, ensuring regulatory compliance across international markets. Our commercial team manages US operations directly since the drug's 2024 launch.
Expanded Access Programs
We provide expanded access to mavorixafor for WHIM syndrome patients meeting specific criteria outside clinical trials. This program requires physician-initiated applications and considers factors like treatment availability, patient eligibility, and supply constraints. Our medical team evaluates each request while maintaining development program integrity.
People
Adam Craig
Executive Chair
Dr. Craig brings extensive life science leadership experience, including former CEO roles at Stratus Therapeutics and CTI BioPharma. He holds medical and scientific degrees from the University of London and Leeds University, with an MBA from the Open Business School.
John Volpone
Chief Operating Officer
Mr. Volpone has over two decades of drug development leadership in oncology and rare diseases. He previously served as Executive Vice President at CTI BioPharma and holds a Biochemistry degree from University of Washington and an MBA from Seattle University.
David Kirske
Chief Financial Officer
Mr. Kirske has two decades of financial leadership experience, including CFO roles at Stratus Therapeutics and CTI BioPharma. He holds a B.A. in Business Administration from the University of Puget Sound and has managed financial operations through major corporate acquisitions.
Natasha Thoren
Chief Legal & Compliance Officer
Ms. Thoren has extensive pharmaceutical compliance experience, previously serving as Associate General Counsel at Albireo Pharma. She holds legal degrees from Western New England University and Saint Louis University, with bar admissions in multiple jurisdictions.
Christophe Arbet-Engels
Chief Medical Officer
Dr. Arbet-Engels has 25+ years of global drug development experience, including leadership roles at Biogen and Boehringer-Ingelheim. He received his MD and PhD from the University of Paris and has contributed to multiple blockbuster drug developments including LANTUS and JARDIANCE.
Questions
What rare diseases does X4 Pharmaceuticals focus on?
We specialize in rare immune disorders, particularly WHIM syndrome and chronic neutropenic disorders, through our CXCR4 pathway research and mavorixafor development.
What is XOLREMDI and when was it approved?
XOLREMDI (mavorixafor) is our FDA-approved oral therapy for WHIM syndrome, approved in April 2024 as the first treatment for this rare condition.
Are there clinical trials available for chronic neutropenia?
Yes, our global Phase 3 4WARD trial (NCT06056297) is evaluating mavorixafor for chronic neutropenic disorders. Patients can find details at 4WARDstudy.com.
How can patients access XOLREMDI?
Patients can access XOLREMDI through our X4Connect program offering insurance support and copay assistance, available Monday-Friday 8am-8pm ET via phone or website.
Does X4 offer international treatment access?
We have distribution partnerships in the EU with Norgine and in MENA region with taiba rare, while our expanded access program considers WHIM syndrome patients meeting specific criteria.