The next big thing on Roche’s list of would-be blockbusters—the hemophilia A drug emicizumab (or ACE910)—came through in a Phase III study, setting up a quick lob to regulators around the world.
Long one of Roche’s top prospects, as laid out by pharma chief Daniel O’Day, Genentech researchers say that the drug hit the primary as well as all the secondary endpoints in their late-stage test. The big goal was a statistically significant drop in the number of bleeds among patients with inhibitors to factor VIII. And one of the secondaries was a reduction in bleeds recorded in an “intra-patient comparison in people who had received prior bypassing agent prophylaxis treatment.”
Roche raised a red safety flag on the program back in early November when it recorded several cases of blood clots — two patients had thromboembolic events and two patients developed thrombotic microangiopathy, or TMA — among patients taking the drug. The news helped briefly buoy Shire and Novo Nordisk, which both see a big rival to their blockbuster hemophilia franchises in emicizumab.
But Roche says these patients were also being treated with agents for breakthrough bleeds, adding today that “neither thromboembolic event required anti-coagulation therapy and one patient restarted emicizumab. Both cases of TMA have completely resolved, and one patient restarted emicizumab.”
Roche’s looming rivalry with Shire and Novo has attracted considerable comment among analysts, who are still waiting for the actual Phase III data to help determine just how big an impact this drug can have in the market.
“In our view, the non-disclosures increase the importance of the mid-year updates that will include Phase 3 top-line ACE910 in the larger hemophilia non-inhibitor population and a likely presentation of the HAVEN results at ISTH medical meeting in July,” notes Leerink’s Jason Gerberry. “Shire shares are slightly down, but in line with FTSE 100 this morning which is not surprising as the ACE910 debate will linger into next year.”
Genentech recorded a big win with the approval of the first PD-L1 drug Tecentriq earlier in the year. And Roche was likely going to add another big nod for the MS drug Ocrevus, though they had to put that victory party on hold for the FDA to take more time on the manufacturing review. Another win with emicizumab will help position Roche for biosimilar competition to three big franchises: Avastin, Herceptin and Rituxan.
“The development of inhibitors that render factor VIII replacement less effective, or ineffective, is one of the greatest challenges in the treatment of hemophilia A today, putting patients at high risk for life-threatening bleeds and repeated bleeds that may cause long-term joint damage,” said Sandra Horning, MD, chief medical officer and head of Global Product Development. “We are pleased to see that, in our first pivotal trial, emicizumab prophylaxis significantly reduced the number of bleeds over time in people in this difficult-to-treat setting. We look forward to working with health authorities to bring this treatment to the hemophilia community as soon as possible.”
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