(Updated - December 9, 2021 8:44 AM EST)

Earlier today, CSL Behring and uniQure N.V. (NASDAQ: QURE) announced that etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe to moderately severe hemophilia B, achieved the pre-specified primary endpoint of non-inferiority in annualized bleeding rate (ABR) 18-months following administration compared to baseline Factor IX (FIX) prophylactic therapy in the pivotal Phase III HOPE-B gene therapy trial. The study also successfully achieved a secondary endpoint demonstrating statistical superiority in reduction of ABR compared to baseline FIX prophylactic therapy.

Reacting to the news, Robyn Karnauskas of Truist Securities believes that the data is “in-line with expectations”, but highlights 2 key differentiators of the drug:

1. The factor levels stayed relatively stable at 18 months (36.9%) vs. 6 months (39%)

2. The data continue to be better than Pfizer’s (NYSE: PFE) that reported 22.8% ± 9.9% at 5x10^11 vg/kg of fidanacogene elaparvovec.

The analyst acknowledges that: “QURE faces competition from Freeline Therapeutics (Private) whose treated patients have reported FIX expression of 26% - 160% following treatment.”

Based on the data, Truist currently models $485M in peak royalties to QURE, based on $2.2B in peak end-user sales globally for Hem B gene therapy.

The report also lists several key near-term catalysts:

1) Preliminary HD from 4 patients expected YE2021;

2) Initiation Ph1b/2 European HD trial YE2021

3) 1H2022 Hem B BLA submission.

Trust maintains a “BUY” rating on the company’s shares with $90 Price Target.

Written by Vlad Schepkov | [email protected]