- Alnylam Pharmaceuticals, looking to shake off a damaging start to the fourth quarter, plans to highlight the growing momentum behind its experimental hemophilia treatment fitusiran at this weekend's annual meeting of the American Society of Hematology (ASH)
- "We feel that this could really be a disruptive agent in the hemophilia market," said company CEO John Maraganore, speaking at an annual healthcare conference hosted by investment firm Piper Jaffray.
- Alnylam will detail updated data from patients with hemophilia A and B, including those with inhibitors that interfere with management of bleeding episodes. Fitusiran is set to compete against Roche's experimental drug emicizumab.
Alnylam has had a tough fourth quarter.
In early October, the company shelved further development of its lead candidate revusiran after a safety review found an "imbalance of mortality" in the treatment arm of a Phase 3 study. The news triggered a sell-off in Alynlam stock, which shed nearly half its value in the days following.
With revusiran in the dustbin, Alnylam is looking to get back on track by highlighting its experimental RNA interference drugs patisiran and fitusiran.
Fitusiran, currently in Phase 1 and Phase 1/2 studies, promises to be more convenient and more consistent treatment in preventing bleeding than many current options in the hemophilia market.
Data from the Phase 1 study released in July showed treatment with fitusiran led to an annualized bleed rate of zero in 17 patients with both types of hemophilia. Early results from patients with inhibitors — antibodies that hinder treatment with replacement factor — appeared encouraging as well.
Sanofi Genzyme certainly thought so, picking up its option to co-develop and co-commercialize fitusiran in the U.S., Canada and Western Europe. Phase 3 studies are slated to start in early 2017.
Yet that won't be fast enough to match Roche, which hopes to submit an initial application for approval of its experimental hemophilia A drug emicizumab sometime in 2017.
Alnylam's Maraganore believes emicizumab, rather than existing hemophilia drugs, will be fitusiran's main rival. "I think emicizumab, which is the non-proprietary name for ACE910, is definitely the competition," Maraganore said at the conference.
He highlighted the potentially broader reach of fitusiran though, as well as its cleaner safety profile: "Our mechanism of action and our drug has a broader applicability across the space than does emicizumab."
"Now they have more patient's worth of data. They are a little bit further ahead. But we think we have a profile that is going to be very competitive," he added.