Sanofi (SNY +2.1%) announces preliminary results from a 34-subject Phase 2 open-label extension study
evaluating RNAi candidate fitusiran in patients with moderate-to-severe
hemophilia A or B with or without inhibitors. The data were presented
at the World Federation of Hemophilia Virtual Summit.
Patients receiving monthly subcutaneous doses of
fitusiran and followed for as long as 4.7 years (median exposure of
2.6years) experienced sustained lowering of antithrombin (~75% from
baseline) resulting in median peak thrombin values at the lower end of
the normal range. Thrombin
is an enzyme that causes the blood to clot. Antithrombin is an antibody
that binds to (inactivates) thrombin thereby interfering with clotting.
Median annualized bleed rate (ABR) overall was
0.84. Median ABR in the non-inhibitor subgroup was 1.01 and 0.44 in the
inhibitor subgroup.
Overall, the rate of spontaneous bleeds was a
median of 0.38. Median rates in the non-inhibitor and inhibitor groups
were 0.33 and 0.39, respectively.
No cases of anti-fitusiran antibody formation were observed.
On the safety front, the most common adverse
events were: increase in ALT (29%) (enzyme biomarker of liver
stress/damage), injection site erythema (reddening of the skin) (21%),
nasopharyngitis (21%), upper respiratory tract infection (18%), diarrhea
(18%), arthralgia (joint pain) (18%), back pain (18%) and increased
transaminases (liver enzymes) (15%).
Results from Phase 3 studies should be available in H1 2021.
https://seekingalpha.com/news/3584552-sanofi-rnai-drug-shows-sustained-benefit-in-hemophilia-patients