Roche says its new interim update on hemophilia A drug is positive. But it’s hard to gauge.

Roche today sent out a small, blurry snapshot of the interim data from a Phase III study of emicizumab in children suffering from hemophilia A.

What did we find out?

Sandra Horning

The data are positive and “clinically meaningful” — though presumably not statistically significant, which is the phrase you look for in determining success or failure — in cutting the bleed rate among children 12 or younger with inhibitors to Factor VIII.

The most common adverse events: injection site reactions and nasopharyngitis.

Any serious adverse events, like the ones seen in HAVEN 1?

In a followup, a spokesperson tells me: “No thromboembolic (TE) events or cases of thrombotic microangiopathy (TMA) occurred in this study population (HAVEN 2).”

And as for the “clinically meaningful” bottom line, she adds: “(G)iven the study design is a single arm (no comparator), we can’t say “statistically significant” because there is no hypothesis testing, but the reduction is clinically meaningful to treaters and patients. This type of study design is standard for pediatric studies in hemophilia. We look forward to sharing details of the bleed rates when the data are presented at congress. We’ve submitted to ISTH in July.”

Fair enough.

Roche’s recent success in gaining an approval for Ocrevus in multiple sclerosis set them on a direct course to disrupting the multiple sclerosis field, with another likely blockbuster to add to a list that also includes the PD-L1 checkpoint Tecentriq. Emicizumab, better known as ACE910, is intended to be the next on the list with peak sales estimates from analysts hovering around $1.4 billion.

Two months ago Roche triggered fresh doubts about its drug after a patient in HAVEN 1 died following two serious adverse events. The death spurred fresh questions about the drug’s safety when investigators had to fend off persistent questions about 4 spontaneously reported SAEs after two patients had thromboembolic events and two patients developed thrombotic microangiopathy, or TMA. That news helped briefly buoy Shire and Novo Nordisk, which both see a big rival to their blockbuster hemophilia franchises in emicizumab.

Two more Phase III studies in the program are ongoing.

“Managing haemophilia A with inhibitors to factor VIII is especially challenging for children and their caregivers, because bleeding is difficult to control and current treatments require frequent intravenous infusions”, said Sandra Horning, Roche’s CMO and product development chief. “We are encouraged that once-weekly subcutaneous emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time in children and are pleased to share these results with the community as we join in celebrating World Hemophilia Day.”

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