Blood clots hit Roche hemophilia trial

Just a few months after Roche ($RHHBY) posted what it deemed a “promising safety and efficacy profile” from a long-term test of its experimental hemophilia med, the Swiss major is now said to have suffered a setback after a number of patients had blood clots.

According to a report from Reuters, quoting Roche, the study of what is touted as the “game-changing” emicizumab (a.k.a. ACE910) saw two thromboembolic events and two cases of thrombotic microangiopathy in patients.

Roche told Reuters that “additional monitoring had now been instigated in the study,” and there has been no word from the FDA or any other body that they are concerned about the safety aspects of the test.


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Deutsche Bank analyst Richard Parkes said: “We view the news as the first blemish on ACE910’s profile,” in a note to clients.

A Roche spokesman told Reuters that the cases involved patients with breakthrough bleeding who were treated using one of two so-called bypassing agents.

Back in July, Roche posted Phase I/II data for its med from the World Federation of Hemophilia 2016 World Congress showing that emicizumab given once-weekly subcutaneously showed “promising safety and prophylactic efficacy for people with severe haemophilia A.”

The hemophilia market, worth around $10 billion, is well catered for, with meds from Shire ($SHPG) Baxter (now Baxalta), Novo Nordisk ($NVO), Bayer and CSL.

Massachusetts-based Alnylam ($ALNY) has also been working on its own gene therapy that aims to cure the disease altogether.


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