General AML

The EMA grants GMI-1271 Orphan Drug Designation for AML

On 25th May 2017, the European Medicines Agency’s  (EMAs) Committee for Orphan Medicinal Products (COMP) granted Orphan Drug Designation for GMI-1271, a novel E-selectin (E-sel) antagonist , for the treatment of patients with Acute Myeloid Leukemia (AML).1  This comes after GMI-1271 was granted an orphan drug designation for the treatment of Relapsed or Refractory (R/R) AML by the U.S. Food and Drug Administration (FDA) earlier this month.

GMI-1271 is a novel antagonist of E-sel, (a cell adhesion molecule that contributes to leukemic cell survival pathways) and acts by disrupting leukemia cell survival pathways thus enhancing chemotherapy response.2,3

GMI-1271 is currently being investigated in the phase 2 portion of the phase 1/2 study (NCT02306291), which is aiming to assess the safety, efficacy and pharmacokinetics of this agent in combination with standard chemotherapy in patients with R/R AML and also in AML patients 60 years of age and older with newly diagnosed disease. Results from this study will be presented during this year’s American Society of Clinical Oncology Annual Meeting, taking place in Chicago between 2nd–6th June and also at the European Hematology Association (EHA) congress taking place in Madrid between 22nd–26th June.

References
  1. GlycoMimetics: GlycoMimetics’ GMI-1271 Receives EU Orphan Drug Designation for Acute Myeloid Leukemia 2017 May 17. http://www.businesswire.com/news/home/20170525005579/en/GlycoMimetics%E2%80%99-GMI-1271-Receives-EU-Orphan-Drug-Designation [Accessed 2017 May 30].
  2. National Cancer Institute: E-selectin antagonist GMI-1271. https://www.cancer.gov/publications/dictionaries/cancer-drug?cdrid=767121. [Accessed 2017 May 18].
  3. DeAngelo D. J. et al. A Phase I/II Study of GMI-1271, a Novel E-Selectin Antagonist, in Combination with Induction Chemotherapy in Relapsed/Refractory and Elderly Previously Untreated Acute Myeloid Leukemia; Results to Date. 2016 Dec 5. Abstract 2029. 58th Annual Meeting of the American Society of Hematology, San Diego, Ca.