August 20, 2017 3:12 PM ET


Company Overview of X4 Pharmaceuticals Inc.

Company Overview

X4 Pharmaceuticals Inc., a clinical-stage oncology company, develops oral small molecule CXCR4 antagonists for the treatment of refractory solid tumors. It focuses on developing X4P-001, a drug candidate for refractory clear cell renal cell carcinoma and refractory epithelial ovarian cancer; and X4P-002, a series of late lead molecules that penetrate the blood brain barrier. The company was incorporated in 2012 and is based in Cambridge, Massachusetts.

784 Memorial Drive

Suite 140

Cambridge, MA 02139

United States

Founded in 2012



Key Executives for X4 Pharmaceuticals Inc.

Founder, Chief Executive Officer, President and Director
Founder and Member of Scientific Advisory Board
Chief Operating Officer
Senior Vice President of Clinical Development and Translational Research
Age: 69
Chief Medical Officer
Compensation as of Fiscal Year 2017.

X4 Pharmaceuticals Inc. Key Developments

X4 Pharmaceuticals Announces Rare Disease Research Collaboration with Yale University

X4 Pharmaceuticals announced the initiation of a multi-year sponsored research program with Yale University to develop and study a genetic model of WHIM syndrome, a rare genetic immunodeficiency disease which currently has no approved treatments. The multi-year research collaboration will investigate the fundamental mechanisms that result in chronic immune deficiency in a genetic preclinical model of WHIM syndrome. WHIM syndrome is a primary genetic immunodeficiency disease caused by mutations in the CXCR4 receptor gene resulting susceptibility to certain infections. WHIM is an abbreviation for the characteristic symptoms of the syndrome: Warts, Hypogammaglobulinemia, Infections, and Myelokathexis.

X4 Pharmaceuticals Announces Initiation of the Phase 2 Expansion of Phase 1/2 Study of X4P-001 in Patients with Advanced Clear Cell Renal Cell Carcinoma

X4 Pharmaceuticals announced dosing of the first patient in the Phase 2 portion of the ongoing Phase 1/2 study evaluating X4P-001, the company’s lead CXCR4 inhibitor, in combination with Inlyta® (axitinib) in patients with advanced clear cell renal cell carcinoma (ccRCC). Full results from the Phase 1 portion of the study including the selection of the once daily oral dose used in the expansion cohort will be presented at an upcoming medical meeting. In addition to safety and tolerability, the Phase 2 portion of the study will evaluate clinical efficacy as measured by objective response rate (ORR), duration of response (DOR), and progression free survival (PFS) and will explore the correlation of biomarkers with efficacy. Multiple cancer centers with renal cell carcinoma researchers in the U.S. and South Korea are participating in the study. In addition to this Phase 1/2 study of X4P-001 in combination with Inlyta®, a VEGF kinase inhibitor, X4 has additional oncology clinical studies ongoing, including a Phase 1/2 study in patients with advanced ccRCC to evaluate X4P-001 in combination with Opdivo® (nivolumab), and a Phase 1b biomarker study in patients with advanced melanoma to evaluate X4P-001 in combination with Keytruda® (pembrolizumab).

X4 Pharmaceuticals Inc. Presents at 16th Annual Needham Healthcare Conference, Apr-05-2017 10:40 AM

X4 Pharmaceuticals Inc. Presents at 16th Annual Needham Healthcare Conference, Apr-05-2017 10:40 AM. Venue: Westin Grand Central Hotel, New York, New York, United States. Speakers: Paula Ragan, Founder, Chief Executive Officer, President and Director.

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