- Novo Nordisk and Bluebird bio will collaborate to develop genome editing treatments for genetic diseases, citing specifically the bleeding disease hemophilia as a target disorder. The two companies did not disclose terms of their agreement.
- The three-year deal will focus initially on one-time treatments correcting mutations causing deficiencies in clotting Factor VIII, which causes hemophilia A. Novo Nordisk's marketed product NovoEight treats this condition by replacing Factor VIII with an infusion as often as every other day.
- Novo's portfolio of hemophilia treatments are under pressure as Roche's Hemlibra, which can be dosed monthly, takes market share in hemophilia A. Potentially one-time gene therapies using viral vectors, meanwhile, are advancing into late clinical development.
Recombinant factor replacement therapies have been used to treat hemophilia since the early 1990s. Newer products have been able to stretch the dosing frequency for bleeding prevention to at best once every four days.
Roche disrupted the market with the introduction of Hemlibra (emicizumab) in 2017, which can be dosed as seldom as once every four weeks in hemophilia A patients. Further disruption awaits in the form of gene therapies from BioMarin Pharmaceutical, Spark Therapeutics, Uniqure and Sangamo Therapeutics, which should require only a single treatment.
Novo has already felt the pressure from Hemlibra, as its 2018 sales of NovoSeven plummeted 14% to 7.9 billion Danish kroner, or about $1.2 billion. NovoSeven is used to treat patients who develop inhibitors to factor VIII replacement therapies, a setting where Hemlibra was first approved.
The deal with Bluebird will make use of a genome editing technology called MegaTAL, which the biotech says can be combined with viral or non-viral delivery methods.
Bluebird has already gained European approval of a gene therapy called Zynteglo, for the blood disease beta thalassemia. This treatment involves harvesting of stem cells and then modifying them outside the body using a virus encoding for production of a protein called beta-globin, before being re-infused into the patients.
The aim of the Novo agreement is to develop a one-time treatment that will alter genetic expression inside the body.
Without a product ready to be tested in humans, Novo and Bluebird remain well behind BioMarin, which plans on submitting an application for its hemophilia A gene therapy valoctocogene roxaparvovec, or valrox, to the Food and Drug Administration by the end of the year.
If that is successful, along with rivals from Spark or Sangamo and Pfizer, many eligible patients would likely have received gene therapy long before Novo and Bluebird can launch a product, leaving only new patients to be treated, wrote Cantor Fitzgerald analyst Elemer Piros.
"While the deal makes sense for both companies in our view, we believe other genetic therapies for hemophilia are progressing much faster, and could limit the market opportunity, should the program ever succeed," he wrote in an Oct. 9 note to clients.
Stifel analyst Benjamin Burnett wrote, however, that there may be room for later generation products because of questions over the durability and effectiveness of valrox.
"There does appear to be room for efficacy improvement as none of the clinical data generated from genetic medicines to date is a clear home run, in our opinion," he wrote.