A 104 week dose finding open label trial followed by an optional 78 week open label extension of lonapegsomatropin, a long-acting growth hormone product, administered once-a-week versus daily somatropin product in prepubertal individuals with Turner syndrome. Approximately 48 individuals (12 individuals per arm) will be randomized to receive one of three doses of lonapegsomatropin or a daily injection of somatropin. This is a trial that will be conducted in the United States.
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.
Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.
Ascendis Pharma (ASND) is the sponsoring company for this trial. BiotechSign currently grades this company D (39/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.