Pfizer Inc (PFE)

[DewDiligence]

PFE GT for Hemophilia-A meets phase-3 endpoints:

https://finance.yahoo.com/news/pfizer-announces-positive-topline-results-104500301.html

The AFFINE study achieved its primary objective of non-inferiority, as well as superiority, of total annualized bleeding rate (ABR) from Week 12 through at least 15 months of follow up post-infusion compared with routine Factor VIII (FVIII) replacement prophylaxis treatment. Following a single 3e13 vg/kg dose, giroctocogene fitelparvovec demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period (1.24 vs 4.73; one-sided p-value=0.0040).

Key secondary endpoints as defined by the trial protocol were met and also demonstrated superiority compared to prophylaxis. 84% of participants maintained FVIII activity >5% at 15 months post-infusion (one-sided p-value = 0.0086) with the majority of participants having FVIII activity ≥15%, and the mean treated ABR showed a statistically significant 98.3% reduction from 4.08 in the pre-infusion period to 0.07 post-infusion (from Week 12 up to at least 15 months [15-44 months]; one-sided p-value < 0.0001). Throughout the study, among all dosed participants, one participant (1.3%) returned to prophylaxis post-infusion.

BMRN has an FDA-approved GT for hemophilia-A called Roctavian (#msg-172243819), but the sales to date have been de minimis (#msg-173910310).

Note: PFE’s giroctocogene fitelparvovec for hemophilia-A should not be confused with Beqvez, PFE’s FDA-approved GT for hemophilia-B (#msg-174309616).