On November 1, 2018 4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 antagonist drugs to improve immune cell trafficking to treat rare disease and cancer, reported that an abstract highlighting X4P-001-RD, the company’s CXCR4 antagonist, has been selected for poster presentation at the 60th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) meeting, taking place Dec. 1-4 in San Diego (Press release, X4 Pharmaceuticals, NOV 1, 2018, View Source [SID1234530651]). The presentation will describe Phase 2 results used to determine the Phase 3 dose in the ongoing Phase 2/3 study of X4P-001-RD in patients with WHIM syndrome, a rare genetic, primary immunodeficiency disease.
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At ASH (Free ASH Whitepaper): X4 Pharmaceuticals to present clinical data used to determine Phase 3 Dose from Phase 2/3 Study of X4P-001-RD in WHIM Syndrome.
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Details of the presentation on X4P-001-RD in WHIM syndrome are as follows:
Title: Determination of Phase 3 Dose for X4P-001 in Patients with WHIM Syndrome
Date & Time: Saturday, December 1, 6:15 PM – 8:15 PM
Location: San Diego Convention Center, Hall GH
Publication #: 1102
Poster Session: 201. Granulocytes, Monocytes, and Macrophages: Poster I
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 U.S. 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. Individuals with WHIM syndrome are more susceptible to potentially life-threatening bacterial infections4. Additionally, 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.