HemaQuest has recently completed Phase I clinical trials of HQK-1001 in healthy subjects and plans to begin clinical studies of this compound in both sickle cell anemia and beta thalassemia in the near future.
The compound’s therapeutic potential was discovered by Susan Perrine, the company’s chief scientific officer and vice president of clinical affairs, and her colleagues at Boston University.
Ronald Berenson, president and CEO of HemaQuest, said: “Orphan drug designations confirm the urgent medical need to develop new therapies to treat these two serious and life-threatening hemoglobin disorders. These designations by the FDA also provide us with strong incentives for our novel proprietary drug candidate, HQK-1001, which initially is being developed to treat both sickle cell anemia and beta thalassemia.”