Novartis has taken an option to buy a novel drug for sickle cell disease, as well as the US biotech company that developed it, in a deal valued at up to $665m in upfront, acquisition and milestone payments.
Oklahoma-based Selexys Pharmaceuticals has just completed a $23m equity financing to help fund the next stage of development of the drug, which is an anti-P-selectin antibody called SelG1.
And Novartis has taken an exclusive option to acquire Selexys and SelG1 after the completion of a phase II study in patients with sickle cell disease, a condition in which red blood cells form an abnormal sickle or crescent shape.
The deformation of the red blood cells causes them to stiffen so they cannot pass through small blood vessels so easily, which in turn prevents oxygen from being delivered to tissues. In severe episodes - known as vaso-occlusive crises - the blood vessels become obstructed leading to ischaemia, pain and organ damage.
"Patients with sickle cell disease endure great suffering and frequent hospitalisation due to painful vaso-occlusive crises," said Selexys' president and chief executive Dr Scott Rollins.
The company developed SelG1 based on the idea that blocking P-selectin - an adhesion molecule that causes cells to clump together - may help avert vaso-occlusive crises in sickle cell patients.
A recently-completed phase I trial supported the antibody's safety in human volunteers and the company is now pressing ahead with a phase II evaluation in patients with the disease.
Rollins said the financing will help Selexys fund not only the SelG1 trial but also a phase I study of a second antibody candidate which targets PSGL-1 and could have activity in the treatment of inflammatory bowel diseases such as Crohn's.
Sickle cell disease is currently treated using a handful of pharmacological therapies. The only drug specifically approved to treat the underlying pathology in the disease is hydroxyurea, with other drugs used to treat symptoms such as pain.
Another drug in trials for sickle cell is Emmaus Medical's L-glutamine, which is currently in phase III testing and has been designated an orphan drug by the US FDA.