Shares of Ascendis Pharma (NASDAQ:ASND), a biopharmaceutical company committed to extended-release technology, are soaring after the company released pivotal trial results for its lead candidate. Investors expecting blockbuster sales have pushed the stock 58.8% higher as of 12:04 p.m. EST on Monday.
Annual sales of Pfizer's (NYSE:PFE) Genotropin brand of human growth hormone (HGH) replacement therapy peaked near $900 million around a decade ago, and Ascendis Pharma is probably right to suggest a longer-lasting version could perform much better. Ascendis Pharma stock is on the move today because pivotal trial results comparing a weekly injection of the company's lead candidate, TransCon HGH, suggest it's as good or better than daily injections of Pfizer's HGH.
Ascendis set out to prove its weekly injection was just as good, but today's results were a big surprise. After 52 weeks, children injected weekly with TransCon HGH were 0.86 cm taller than those given a daily HGH dose, a statistically significant difference.
Getting young children ready for each day is tough without adding a needle to their daily routine, and adults aren't too fond of stabbing themselves each day if they can help it. With this in mind, Ascendis thinks the global market opportunity for TransCon HGH can drive blockbuster sales if expanded to include the same patients as gonadotropin.
Growth hormone deficiency isn't the only condition treated with frequent injections of replacement hormones. Ascendis also has two more TransCon extended treatments in clinical-stage testing and enough cash to last a couple more years.
That should be a long enough runway to get its treatment to a commercial stage. The company expects results from a second pivotal study in the second quarter. If successful, as expected, Ascendis will use both to support an application the FDA could see in the first half of 2019.