X4 Pharmaceuticals to Present Clinical Data from Phase 2 Study of X4P-001-RD in WHIM Syndrome

Study designed to evaluate X4P-001-RD in a rare genetic, primary
immunodeficiency disease to be presented at the Annual Congress
of the European Hematology Association

, a clinical stage biotechnology company developing
novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat
cancer and rare disease, today announced that an abstract highlighting X4P-001-RD,
the company’s CXCR4 antagonist, has been selected for poster
presentation at the 23rd Annual Congress of the European
Hematology Association (EHA), taking place June 14-17 in Stockholm,
Sweden. The presentation will describe interim clinical results from the
ongoing Phase 2/3 study of X4P-001-RD in patients with WHIM syndrome, a
rare genetic, primary immunodeficiency disease.

Details of the presentation on X4P-001-RD in WHIM syndrome are as


    Phase 2 Study of X4P-001: A Targeted Oral Therapy for Patients with
WHIM Syndrome


David Dale, M.D., University of Washington

Abstract #:


Poster Session:

Bone marrow failure syndromes incl. PNH – Clinical

Date and Time:

Saturday, June 16, 5:30-7:00 p.m. CEST

About WHIM Syndrome

WHIM syndrome is a primary immunodeficiency disease caused by genetic mutations
in the CXCR4 receptor gene
resulting in susceptibility to certain
types of infections. WHIM is an abbreviation for the characteristic
clinical symptoms of the syndrome: Warts, Hypogammaglobulinemia,
Infections, and Myelokathexis. Within the overall category of primary
immunodeficiencies, there are between 15,000 and 100,000 patients in the
US that are classified with primary immunodeficiency disease of unknown
origin – of which WHIM is one.1,2,3 WHIM syndrome is a rare
disorder and the precise prevalence or incidence of patients that have
the genetic mutation responsible for WHIM syndrome is unknown. Because
patients are highly susceptible to infections, WHIM syndrome is
associated with significant morbidity beginning in early childhood and
continuing throughout life. Current therapy is limited to treatment of
acute infections with antibiotics or prevention through the use of
intravenous immunoglobulin or G-CSF. There is no approved therapy for
the treatment of WHIM syndrome.

About X4 Pharmaceuticals

is developing novel therapeutics designed to improve
immune cell trafficking to treat cancer and rare diseases. The Company’s
oral small molecule drug candidates antagonize the CXCR4
pathway, which plays a central role in immune surveillance. X4’s most
advanced product candidate, X4P-001-RD, is in a Phase 2/3 study in
patients with WHIM syndrome, a rare genetic, primary immunodeficiency
disease. X4P-001-IO is currently under investigation in multiple
clinical studies in solid tumors. X4 was founded and is led by a team
with deep product development and commercialization expertise, including
several former members of the Genzyme leadership team, and is located in
Cambridge, MA. For more information, visit www.x4pharma.com.

1 Boyle JM, Buckley, RH, Population Prevalence of Diagnosed Primary
Immunodeficiency Diseases in the United States. J Clin Immunol

2 Gathmann B, Grimbacher B, et al. The European internet-based patient
and research database for primary immunodeficiencies: results 2006–2008.
Clin Exp Immunol. 2009 Sep;157 Suppl 1:3-11.

3 Modell V, Gee B, et al. Global study of primary immunodeficiency
diseases (PI) — diagnosis, treatment, and economic impact: an updated
report from the Jeffrey Modell Foundation. Immunol Res. 2011;51:61–70.


X4 Pharmaceuticals
Kathryn Morris, 914-204-6412