The disease-modifying therapy, consisting of decitabine and tetrahydrouridine, is aimed at helping patients increase fetal hemoglobin levels to replace the hemoglobin that’s defective in the disease. The idea is to prevent red blood cells from becoming sickle-shaped, increasing oxygen levels in the blood.
Under their agreement, the companies will collaborate on the continued development of EPI01 for sickle cell disease and beta-thalassemia. EpiDestiny will obtain royalties on the therapy’s sales, once it’s approved. It can also earn more than $400 million in non-royalty income — up-front payments and rewards for achieving development and sales milestones.
Studies have shown that higher levels of fetal hemoglobin can improve sickle cell disease. It helps red blood cells last longer, reduces the number of patients’ pain crises, and increases their life expectancy, according to the research.
EpiDestiny recently completed a Phase 1 clinical trial (NCT01685515) to see if EPI01 could increase patients’ fetal hemoglobin levels and improve their sickle cell disease symptoms. Another objective was to see how patients’ bodies reacted to the treatment.
The team at EpiDestiny has been trying to develop a treatment for sickle cell disease that is less toxic than current therapies and that can generate higher levels of fetal hemoglobin.
Key findings of the Phase 1 trial were that EPI01 increased fetal hemoglobin levels in a small patient group treated for eight weeks, and was safer than other therapies.
“We are looking forward to working closely with EpiDestiny and their great network among sickle cell disease experts and the sickle cell community,” Mads Krogsgaard Thomsen, the executive vice president and chief science officer of Novo Nordisk, said in a press release. “We are confident that together we can make a significant difference for SCD [sickle cell disease] patients and their families globally.”
“We would like to extend our gratitude to all the dedicated patients, physicians and nurses who will participate in the clinical studies of EPI01 in SCD and beta-thalassaemia,” said Santhosh Vadivelu, the president and chief executive officer of EpiDestiny. “The support we receive from Novo Nordisk will allow EpiDestiny to invest and explore the full potential of EPI01 within oncology and to pursue our other pipeline compounds in oncology and other indications.”
EPI01 has already been granted rare pediatric disease status, orphan drug status, and fast track status in the United States, EpiDestiny said.