Rare Daily Staff

Novo Nordisk agreed to pay an upfront fee of $400 million for an exclusive worldwide license for EpiDestiny program EPI01 in rare, genetic blood disorders Sickle Cell Disease and beta-thalassemia.

Under the terms of the agreement, Novo Nordisk will collaborate to development the experimental therapy for the two indications while EpiDestiny will retains all rights to continue development of EPI01 in oncology. Novo Nordisk will also pay undisclosed development and sales milestone payments, as well as royalties on net sales.

Sickle cell disease is caused by mutations in the gene for the hemoglobin beta chain that carries oxygen in red blood cells. The resulting sickle hemoglobin molecule forms large fibrils in red blood cells, which renders red blood cells rigid and deformed. The sickle-shaped red blood cells tend to aggregate and can block small blood vessels, lead to chronic anemia, decreasing oxygen delivery and damaging multiple tissues and organs.

Damage to organs, such as the spleen, impairs immunity and causes high risk of infections such as pneumonia. Large vessels in the brain can be affected by the rigid red blood cells and anemia, leading to overt strokes in 10-15 percent of children and silent strokes in many more. Chronic anemia also affects the heart and lungs, contributing to a life expectancy which may be reduced by several decades, since there are limited medicines that address the root cause of the disease.

Beta-thalassemia is a related group of red blood cell diseases also caused by mutations in the gene for the hemoglobin beta chain. Instead of producing a mutated hemoglobin molecule, not enough of the hemoglobin chain is produced, leading to varying degrees of anemia and decreased oxygen delivery. The most severe cases of beta-thalassemia require regular blood transfusions. The blood transfusions, although initially life-saving, eventually cause too much iron to accumulate in the body, which affects the heart and eventually causes death.

EPI01 is an oral, fixed dose formulation that has potential to work by increasing the amount of fetal hemoglobin that can substitute the defective hemoglobin in sickle cell disease patients, thereby intending to prevent the deformation of the red blood cells and improving the oxygen level in the blood. The gene that produces fetal hemoglobin is usually silenced after birth. EpiDestiny has been granted Rare Pediatric Disease, Fast Track and Orphan Designations by the U.S. Food and Drug Administration for EPI01.

EpiDestiny recently completed a phase 1 trial with EPI01 in sickle cell disease patients demonstrating increased fetal hemoglobin expression and safety after eight weeks of administration in a small patient cohort. The study demonstrated the potential for EPI01 to serve as a safe and meaningful disease-modifying therapy for the disease, the company said.

“The collaboration with Novo Nordisk represents an important step in the continued development of EPI01 for patients with this underserved life-threatening disease. We are looking forward to sharing this program with Novo Nordisk, which has a long history of successfully developing and commercializing products within chronic specialty care across the globe,” said Santhosh Vadivelu, president and CEO 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.”

April 9, 2018
Photo: Santhosh Vadivelu, president and CEO of EpiDestiny

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