Roche’s hemophilia drug slashes bleeding rate, boosting blockbuster projections — but doubts on safety linger

Roche HQ in Basel on May 21, 2017 / John McCarter

Roche served up another portion of late-stage data on its hemophilia drug emicizumab (ACE910), underscoring why analysts believe the Swiss pharma giant has another blockbuster drug on its hands.

Prepping for an upcoming science conference, investigators reported that 62.9% of the hemophilia A patients who had developed inhibitors to standard therapies and were on their drug had zero bleeding episodes at 31 weeks, compared to only 5.6% of patients on bypassing agents. The patients saw bleeding rates slashed for all kinds of bleeds reviewed, from all bleeds to joint bleeds, with a clear win on a quality of life measurement as well. And in a small subset of patients provided a preemptive bypassing agent and then emicizumab, the risk of bleeding dropped 79%.

But the two Phase III studies Roche reported on were not without danger. There were 2 cases of thromboembolic events and 3 instances of thrombotic microangiopathy. Back in February Roche rattled its most bullish followers on this drug with the report that a patient died from a rectal hemorrhage after being treated with emicizumab and a bypassing agent during a breakthrough bleeding episode. The incidents were attributed to the BPAs.

As a result of the adverse events, analysts backing Shire and Novo Nordisk – both of which have hemophilia franchises threatened by Roche’s drug – have been speculating about a possible direct tie to the Roche drug. That will soon be handled by regulators. While Roche would appear to have the inside track among patients who had developed inhibitors to standard drugs, it could face resistance to its use among others without inhibitors, restricting its use and limiting its commercial appeal.

The drug remains one of Roche’s top Phase III prospects and one of the potentially most valuable orphan products now in the late-stage pipeline. EvaluatePharma has estimated 2022 sales at close to $1.5 billion with the most optimistic analysts ready to multiply that by at least two. With Roche’s PD-L1 checkpoint drug Tecentriq came under a cloud after failing a Phase III, success on this score is critical.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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