The agreement with US-based biotech company EpiDestiny centres around EPI01 – a novel oral therapy for people living with sickle cell disease (SCD) and beta-thalassemia – two life-threatening genetic blood disorders.
“This is an important collaboration – not just for our R&D organisation and for the millions of patients living with these life-threatening conditions, but also in a commercial sense,” explained Christian Kanstrup, head of Novo Nordisk’s recently-established Biopharm Operations Unit.
“As we have already outlined in our leadership ambition for Biopharm, we have a strong focus on identifying acquisitions and bolt-on opportunities. Collaborations like this can deliver much-needed therapies to underserved patient populations while helping our Biopharm business return to growth.
“I’m confident that this deal is the first of many more to come as we seek to evolve the way we operate in the Biopharm space.”
SCD is an umbrella term for a group of life-threatening inherited blood disorders. It is one of the most common genetic diseases, and primarily affects people of Asian and African origin. There are approximately 20 million people living with the condition worldwide.
People with SCD have abnormal haemoglobin – a protein in red blood cells that carries oxygen throughout the body. This abnormality causes sickle-shaped red blood cells (pictured above), which are fragile and prone to rupture. Because of their unusual shape and rigidity, they are also liable to clump together and block small blood vessels, decreasing oxygen delivery and causing damage to multiple tissues and organs in the body.
Beta-thalassaemia, meanwhile, is a related group of red blood cell diseases. Instead of producing a mutated haemoglobin molecule, not enough haemoglobin is produced, leading to varying degrees of anaemia and decreased oxygen delivery.
EPI01 is designed to counteract the effects of both diseases by increasing levels of foetal haemoglobin (HbF) to ‘substitute’ for the defective haemoglobin in patients, thereby increasing the half-life of their red blood cells, reducing pain and improving life expectancy.
"I’m confident that this deal is the first of many more to come as we seek to evolve the way we operate in the Biopharm space," said Christian Kanstrup, SVP, Biopharm Operations.
According to Mads Krogsgaard Thomsen, EVP and Chief Science Officer, our collaboration with EpiDestiny will bring new hope to an underserved patient population.
“This is a great opportunity for Novo Nordisk to enter into a new therapeutic area closely related to our existing Biopharm business,” he said. “With our core R&D and commercial capabilities, we can make a significant difference for patients living with this 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.”
For more on this story, read the joint Novo Nordisk / EpiDestiny press release in full here.