Reduce the bleeding rate of patients with hemophilia by about 90%. One-shot RNAi therapy per month reaches the phase 3 clinical endpoint

A few days ago, Sanofi announced that it will announce the hemophilia RNAi therapy fitusiran jointly developed with Alnylam at this year’s American Society of Hematology (ASH) annual meeting to treat both hemophilia A and hemophilia B.

Hemophilia A and hemophilia B are coagulation disorders caused by the patient's lack of coagulation factors VIII and IX, respectively

▲Fitusiran's mechanism of action (picture source: reference [2])

Previous ASH annual meeting abstracts showed that fitusiran showed good activity in hemophilia patients with coagulation factor inhibitors, and 65% of the treated patients had an annual bleeding rate of 0

Picture source: reference [1]

In terms of safety, 78.

The researchers pointed out in the abstract that fitusiran met all the primary and secondary endpoints of this phase 3 clinical trial

Reference materials:

[1] LBA-3 Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B).

[2] Development of a Pharmacokinetic-Pharmacodynamic (PK-PD) Model of Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia in Patients With and Without Inhibitors.