Before discontinuing the asset, Ascendis Pharma was studying onvapegleukin alfa in advanced or metastatic solid tumors, demonstrating a three- to four-month overall survival advantage over historical controls.
Ascendis Pharma’s plans of expanding into cancer haven’t panned out, and the Danish biotech is now returning to its rare endocrinology roots.
Ascendis announced this strategy change Thursday alongside its Q1 earnings results, revealing that it will no longer invest in the development of onvapegleukin alfa, an IL-2 β/γ asset that was being tested in the Phase 1/2 IL-BELIEVE study, alone or in combination with other anti-cancer therapies, for advanced or metastatic solid tumors.
“Further internal oncology development does not align with our strategic focus,” Ascendis said by way of explaining onvapegleukin alfa’s discontinuation. The asset makes use of Ascendis’ TransCon technology, which allows for sustained and predictable release of a therapeutic agent, in turn enabling less frequent dosing but consistent drug exposure.
Before being axed, onvapegleukin alfa in IL-BELIEVE demonstrated a median overall survival of 10 months, Ascendis said Thursday, versus six to seven months in historical controls. The drug was “generally well-tolerated” in patients with late-stage platinum-resistant ovarian cancer.
While Ascendis is no longer putting money behind onvapegleukin alfa, the company said that it will “explore other ways to maximize the value of this asset.” There has been no word yet of what these alternatives might look like.
Onvapegleukin alfa still appears on Ascendis’ pipeline page, but the biotech lists no other cancer assets.
With cancer no longer a priority, Ascendis will return its focus to rare endocrinology portfolio. This includes its recently approved achondroplasia drug Yuviwel, indicated for children 2 years and older. The company is continuing to develop Yuviwel, with an ongoing study looking at its therapeutic value in infants. Enrollment for this trial is expected to wrap up in the third quarter, the company said.
Ascendis is also planning a Phase 3 trial for Yuviwel in hypochondroplasia, set to start in the second half of 2026. Since its approval in March, Yuviwel has reached more than 60 patients.
In the first quarter, Ascendis made €247 million (roughly $290 million), more than double its €101 million (around $118.55 million) revenue during the same period last year. The hormone replacement therapy Yorvipath, indicated for hypoparathyroidism, accounted for much of this growth, Ascendis said Thursday. Sales of the product jumped to €197 million (approximately $231.4 million) in the most recent quarter, from €44.7 million (around $52.5 million) in the first quarter of 2025.
