Long-term safety and efficacy follow-up for participants with Hemophilia B who were previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation sub-study evaluating safety, tolerability, and kinetics of a higher dose with long-term safety and efficacy follow-up. Participants in the substudy do not need to have participated in C0371005.
Phase 2 trials begin testing efficacy in a larger patient group (100–300). This is often where biotech binary events occur — positive Phase 2 data can significantly advance a company's pipeline narrative, while failures can be terminal for a program.
This trial was terminated before completion. Termination can indicate safety signals, futility findings, or business/regulatory decisions by the sponsor.
Pfizer Inc (PFE) is the sponsoring company for this trial. BiotechSign currently grades this company C (52/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.