Exicure Soars On Burixafor Data In Multiple Myeloma Trial

(RTTNews) - Shares of Exicure Inc. (XCUR) were up over 65% at $8.69 in overnight trading, following the presentation of positive topline phase 2 data for Burixafor in multiple myeloma at the ASH Annual Meeting yesterday.

Burixafor is an investigational small molecule that blocks CXCL12 binding to CXCR4 receptors on hematopoietic progenitor cells (HPCs), rapidly mobilizing these cells from the bone marrow into the peripheral blood.

The phase 2 trial is assessing whether Burixafor, in combination with propranolol and granulocyte colony-stimulating factor, can improve CD34+ hematopoietic stem cell mobilisation from the bone marrow into the peripheral blood, where they can be collected via leukapheresis for use in patients with multiple myeloma undergoing autologous hematopoietic cell transplantation (AHCT).

Multiple myeloma, also called Kahler's disease, is a rare blood cancer that develops in the plasma cells of the bone marrow. It disrupts normal blood formation, weakens the immune system, and often causes complications such as bone damage and kidney problems.

The standard of care treatment for newly diagnosed multiple myeloma in the US involves autologous hematopoietic cell transplantation (AHCT) following high-dose chemotherapy (HDT). In the autologous stem cell transplant procedure, patients' own stem cells are collected from the blood after giving them a mobilizing agent to help move stem cells from the bone marrow into the bloodstream. Effective mobilization and harvesting of hematopoietic progenitor cells are crucial for multiple myeloma patients undergoing autologous stem cell transplant.

The primary endpoint of the study was to determine the proportion of patients who would collect at least 2 × 106 CD34+ cells/kg within two leukapheresis sessions after receiving treatment.

According to the trial results, 17 out of 19 participants (89.5%) reached the primary endpoint of collecting at least 2 × 106 CD34+ cells per kilogram of body weight within the first two leukapheresis sessions, and 2 out of 19 participants needed a third leukapheresis to reach the required number of stem cells.

Among participants who proceeded to transplant, the median time to neutrophil engraftment or neutrophil counts recovering to a safe level was 13 days, and the median time to platelet engraftment or platelet levels recovering to a safe level was 17.5 days, noted the company.

Burixafor also showed a differentiated and rapid mobilization kinetics from other CXCR4 inhibitors, with peak peripheral levels of CD34+ cells observed within one hour of administration, making same-day Burixafor administration and apheresis feasible. The current FDA-approved CXCR4 antagonist drugs used to mobilize hematopoietic stem cells for transplantation, like plerixafor and motixafortide, require overnight pre-treatment.

On Monday, XCUR closed trading at $5.33, up 3.50%.