Solid phase II data on Ascendis Pharma’s TransCon PTH in hypoparathyroidism position the therapy to go into phase III studies by the end of the year – something that cannot happen too soon as far as patients are concerned, since the only other therapy for this rare condition, Takeda’s Natpara, was pulled from the US market last year. After the four-week fixed dose portion of Ascendis’s Path Forward trial, half of the TransCon PTH-treated patients hit all four of the components of the primary endpoint, compared with 15% of placebo subjects; the difference was statistically significant. 82% of patients across all doses of TransCon PTH, and 100% of those given the highest dose, were able to eliminate standard of care – vitamin D and calcium supplements. Leerink analysts believe that, Covid-19 permitting, an expedited regulatory path could be possible for the therapy, and put the drug’s peak sales at $1bn in 2030, with a 75% probability of success. While investors wait for that, Ascendis’s lead product, a formulation of growth hormone, could reach the market; according to consensus forecasts compiled by EvaluatePharma, the sellside sees this product becoming a blockbuster in 2024, presumably contributing to Ascendis’s vast valuation of $6.2bn.
|Annual WW sales ($m)|
|Phase III||TransCon Growth Hormone||-||82||310||715||1,139|
|Phase II||TransCon PTH||-||-||-||56||210|
|Phase II||TransCon CNP||-||-||-||2||29|