Novo Nordisk Ties Up Sickle Cell Disease Treatment in $400M Deal

Danish pharma giant Novo Nordisk tied up a sickle cell and beta-thalassemia program in a $400 million deal with Ohio-based EpiDestiny.

Novo Nordisk will acquire the gene control therapy EPI01, which recently finished Phase I testing for sickle cell disease. Phase I testing of EPI01, which is a combination of tetrahydrouridine and decitabine, in a small group of patients showed that EPI01 increased levels of fetal hemoglobin. An increase in fetal hemoglobin is important to both sickle cell and beta-thalassemia patients. Elevated HbF correlated with increased red blood cell half-life, reduced number of pain crises and increased life expectancy, Novo Nordisk said in its announcement.

 "This is a great opportunity for Novo Nordisk to enter into a new therapeutic area closely related to our existing biopharmaceutical business and thereby utilize our core R&D and commercial capabilities to make a significant difference for patients living with a serious chronic disease. We are looking forward to working closely with EpiDestiny and their great network among sickle cell disease experts and the sickle cell community. We are confident that together we can make a significant difference for SCD patients and their families globally," Novo Nordisk Chief Science Officer Mads Krogsgaard Thomsen said in a statement.

Under terms of the deal, the $400 million paid to EpiDestiny will include upfront fees, as well as developmental and commercial milestone payments. The companies did not disclose a breakdown in the way the money will be paid out. The two companies will collaborate on the development of EPI01 for both sickle cell disease and beta-thalassemia.

With Phase I data in hand the Danish company is looking to take orally-administered EPI01 to the next stage of clinical testing. If the drug continues to impress it’s likely to be a contender with hydroxyurea, a standard sickle cell drug. That standard care though does have some toxicity issues. There is currently no treatment for sickle cell disease that targets the cause. Sickle cell disease can cause blood vessels to become blocked. That blockage can damage organs and cause death.

Following the small Phase I trial EpiDestiny said EPI01 provided an effective way of controlling gene expression, without causing the toxicity problems of hydroxyurea.

The acquisition of the EpiDestiny product comes at a time when Novo Nordisk, known for its diabetes products, is looking to boost its hematology program. Last year Novo snagged approval from the U.S. Food and Drug Administration for Rebinyn, a hemophilia B treatment for adults and children.

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