X4 Pharmaceuticals initiates Phase II/III trial of X4P-001-LD to treat WHIM syndrome

30th January 2017 (Last Updated January 30th, 2017 18:30)

Clinical stage bio-technology firm X4 Pharmaceuticals has initiated its Phase II/III clinical trial of X4P-001-LD with the first patient dosing to treat warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome.

Clinical stage bio-technology firm X4 Pharmaceuticals has initiated its Phase II/III clinical trial of X4P-001-LD with the first patient dosing to treat warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome.

X4P-001-LD is a low-dose formulation of the firm's oral small molecule CXCR4 or C-X-C receptor type 4 inhibitor X4P-001.

WHIM is a genetic primary immunodeficiency disease caused due to mutations in the CXCR4 receptor gene, leading to suppression of the immune system, increased health complications along with infections such as pneumonia, meningitis, deep soft tissue abscesses and skin infections.

X4 Pharmaceuticals president and chief executive officer Paula Ragan said: “We are very pleased with the rapid progress across clinical, regulatory and formulation development that has resulted in X4 initiating this clinical study to evaluate the potential for our CXCR4 drug candidate to treat WHIM syndrome.

"The initiation of this Phase II/III study is a critical milestone in developing a potentially life-changing therapeutic for WHIM patients who otherwise have no therapeutic options."

“The initiation of this Phase II/III study is a critical milestone in developing a potentially life-changing therapeutic for WHIM patients who otherwise have no therapeutic options.”

The mutations to the CXCR4 receptor is reported to cause aberrant signalling, resulting in the retention of neutrophils and leukocytes in the bone marrow

X4P-001-LD has been developed to balance the signalling for the mutant CXCR4 receptor to improve the neutrophils and leukocytes release along with immune function, and blocks the binding of CXCL12 ligand to the CXCR4 receptor.

X4P-001 is currently in Phase I/II clinical development to evaluate its efficacy as a cancer therapy to treat patients with refractory clear-cell renal-cell carcinoma (ccRCC) and other solid tumour indications.