Novo Nordisk gets PhIII hemophilia trials for a Roche rival back on track after completing work-around on blood clot threat
After abruptly shutting down three hemophilia studies back in March due to serious safety issues, Novo Nordisk is getting things going again.
The Danish pharma gave the go-ahead to resume Phase III trials for its concizumab candidate, which are investigating subcutaneous prophylaxis treatment in hemophilia A and B patients regardless of inhibitor status. Novo Nordisk had halted the studies after three patients experienced non-fatal blood clots.
In a statement, the company said it had found a “new path forward” for concizumab. The FDA has agreed to new safety measures and guidelines and lifted the clinical hold, Novo Nordisk said.
Asked to expand on these additional measures, a Novo spokesperson said they include “specific recommendations for the treatment of breakthrough bleeding episodes, both in terms of dosing and frequency, mandating the use of lower doses of additional hemostatic agents, as well as a new concizumab dosing regimen, with an initial daily dose of 0.20 mg/kg concizumab for all patients.”
These trials had initially begun in late 2019 after Novo had released positive proof-of-concept data. Concizumab is an anti-tissue factor pathway inhibitor that targets Factor Xa, attempting to limit its activation. If the candidate works, it could restore the deficiencies present in hemophilia A (factor VIII) and hemophilia B (factor IX).
Hemophilia drugs are designed to prevent uncontrolled bleeding in patients and the current market leader is Roche’s Hemlibra, which Novo had hoped to rival. So far in 2020, Hemlibra has netted Roche over $700 million in US sales and topped $1 billion globally. The drug accounts for 23% of US patient share, according to the pharma’s second quarter earnings report.
But the hemophilia arena has been trending toward newer gene therapy treatments for some time. Roche’s own therapy, coming out of the biotech Spark Therapeutics, ran into some bumps back in 2017 for hemophilia A and now expects to start a Phase III sometime in 2021. That put it behind frontrunner BioMarin, which expects an FDA decision on its hemophilia A program before the end of August.
Should BioMarin receive approval, it would be the world’s first gene therapy program approved for hemophilia A. But it’s shown signs of trouble as well, as mean Factor VIII activity levels have slid in each successive year. CEO JJ Bienaimé has indicated the biotech intends to charge somewhere in the $2 million to $3 million range, a price tag that could make the therapy the most expensive single-use therapy.
Sangamo and Pfizer are also in the hemophilia gene therapy game, and while they’re behind both BioMarin and and Spark, stronger data on durability could prevent competitors from capturing and keeping market share.