Stem Cell Mobilizer

Burixafor

Overview

TaiGen's stem cell mobilizer Burixafor is the company's first fully in-house developed product, a First-in-Class drug with an IND under US FDA. With a variety of potential applications in a number of disease indications, if proven effective in clinical trials, Burixafor will be able to address several unmet medical needs. The molecule is a potent and selective chemokine receptor antagonist which can rapidly mobilize stem cells and progenitor cells from the bone marrow into peripheral circulation. Burixafor also has potential application in chemosensitization treatment of leukemia patients, delaying relapse after chemotherapy. In addition to hematological indications, Taigen and its collaborator have shown Burixafor’s efficacy in animal models of ischemic tissue repair such as strokes, myocardial infarction, and ischemic limb claudication. To this end, TaiGen plans to initiate clinical trials of Burixafor as a chemotherapy sensitizer in China shortly.

For patients undergoing leukemia stem cell transplantation, current conventional therapy is to inject granulocyte colony-stimulating factor (G-CSF) to stimulate the proliferation of hematopoietic stem cells, which are then collected for later transplantation. However, if administration of G-CSF does not bring about the desired result, current practice is to use Mozobil (Sanofi) to then be injected together with G-CSF. Burixafor in comparison could be used as a monotherapy in certain type of patients without combination with G-CSF. Phase II clinical trials are underway in the U.S., with Phase IIa already completed. In December 2013 preliminary data from these trials was presented at American Society of Hematology Annual Meeting in New Orleans, US.
  • Indications in Development
  • Hematopoietic stem cell transplantation
  • Chemosensitization in hematological and solid tumors
  • Stem cell collection in healthy individuals for personalized regenerative medicine

Superiority

  • Hematopoietic stem cell transplantation
  • Re-establish hematopoietic function in patients whose bone marrow or immune system was damaged or defective due to chemotherapeutic treatment of hematological cancers.
  • Can be used as a single agent or in combination with G-CSF to mobilize sufficient amount of hematopoietic stem cells for transplantation.
  • Results from the first US Phase 2 trial showed excellent stem cell mobilization activity and safety in hematological cancer patients. All patients reached the endpoint and successfully transplanted.
  • A second US Phase 2 study demonstrated Burixafor + G-CSF can mobilize sufficient stem cells in two collections, with 100% success rate. Burixafor + G-CSF greatly reduces the time, cost, and side effects of stem cell transplantation.
  • Chemosensitization
  • Conventional chemotherapy cannot eradicate hematological or solid tumors completely as some cancer cells are hiding in the bone marrow and insensitive to chemotherapeutic agents. Cancer cells can be mobilized into peripheral blood circulation and render them sensitive to chemotherapeutic agents.
  • A clinical trial in refractory and relapsed acute myeloid leukemia (AML) was completed in China.

Marketplace

Globally ~70,000 hematopoietic stem cell transplantations are performed per year.

Over 90% of transplantations use hematopoietic stem cells collected from peripheral blood. The collection procedure is lengthy, expensive and requires multiple administration of G-CSF. Use of G-CSF is often associated with side effects such as bone pain.

Development

  • Intellectual Property : Burixafor is protected globally by a portfolio of patents that cover composition of matters, use and manufacturing process up to 2029.
  • Completed phase I & II clinical trials for hematopoietic stem cell transplantation under the U.S. IND.
  • Completed phase I clinical study for chemsensitization in China.
  • Transferred worldwide rights of Burixafor to GPCR Therapeutics Inc., a leading Korean biotechnology company, for the continued development.

Honor

  • 2015
  • Silver Award for New Drug R&D
  • 2008
  • Taipei Biotech Award for R&D Innovational Award, Golden Medal Award
  • 2008
  • National Innovation Award

Mechanism