Novo Nordisk is coming for Roche’s Hemlibra. A phase 3 trial of the Danish drugmaker’s challenger to the blockbuster hemophilia drug hit its primary endpoint, clearing the path for a filing for approval around the end of the year.
Roche’s hemophilia A treatment Hemlibra brought in (PDF) 4.1 billion Swiss francs ($4.5 billion) last year. Novo Nordisk is taking the same approach to preventing bleeds with Mim8, a bispecific antibody designed to bridge Factor IXa and X to replace missing Factor VIII. Roche’s Hemlibra showed (PDF) the approach can enable most people to be free from treated bleeds on its path to market.
Now, Novo Nordisk has shared phase 3 data on Mim8. The trial met its co-primary endpoints by linking once-weekly and once-monthly Mim8 to statistically significant reductions in treated bleeding episodes.
Novo Nordisk found the treatment safe and well tolerated, with no deaths or thromboembolic events reported in the trial. The combination of a primary endpoint hit and clean safety profile could be enough for Mim8 to get a favorable response when Novo Nordisk submits it to regulators late this year. The pharma will face a well-entrenched rival in Roche if it brings Mim8 to market, but analysts are upbeat.
“Positive headline phase 3 for Novo's Mim8 in hemophilia A may near our ‘best’ case scenario, with convenient once-monthly dosing seeming similarly effective to once-weekly, and efficacy data that may challenge Roche's incumbent Hemlibra,” Jefferies analysts wrote in a note to investors.
Efficacy is one area where Novo Nordisk predicted it may have an edge. Martin Lange, M.D., Ph.D., Novo Nordisk’s executive vice president of development, told attendees at the company’s Capital Markets Day in March that Mim8 has the potential to render more than 70% of patients free from treated bleeds.
In patients with no prior prophylaxis treatment, 86% of people treated with once-weekly Mim8 and 95% of those treated with once-monthly Mim8 had no treated bleeds. Jefferies analysts compared the results to the 80% reported by Roche's HAVEN-6 Hemlibra trial at its interim readout and the 56% to 60% range in Roche’s HAVEN-3.
Meanwhile, the zero treated bleed rates in people with prior coagulation factor prophylaxis were 66% in the once-weekly arm and 65% in the once-monthly cohort. The analysts compared the result to the 54% rate of zero treated bleeds seen in patients on prior coagulation factor prophylaxis in HAVEN-3.
The similarity of the once-weekly and once-monthly results supports Novo Nordisk’s attempt to establish convenience as a differentiator versus Hemlibra. Patients receive Roche’s therapy once a week for four weeks, at which point they start taking maintenance doses either every week, every two weeks or every four weeks. Lange said Mim8 was designed to have a “true monthly profile.”
Roche Pharmaceuticals CEO Teresa Graham sounded confident when asked about Mim8 on an earnings call in April, saying Hemlibra will remain the treatment of choice for patients switching to a prophylactic treatment. The confidence is built on real-world data showing 80% of patients have zero bleeds and a drug profile that means 60% of people take the medicine every two weeks or every four weeks.