Hailshadow/iStock via Getty Images
Danish biotech Ascendis Pharma A/S (ASND) dropped ~15% early Monday as Wall Street reacted to Phase 2 data the company posted for TransCon CNP, an investigational therapy for bone disorder achondroplasia.
Ascendis (ASND) said that TransCon CNP, a long-acting form of C-type natriuretic peptide (CNP) met the primary objectives in the Phase 2 trial outperforming the placebo in children with achondroplasia aged 2 to 10 years old.
The topline data indicated the superiority of TransCon CNP at 100 µg/kg/week, as the 57-subject study met the primary efficacy endpoint of annualized height velocity (AHV) at 52 weeks, the company said.
The investigational therapy was safe and well tolerated, with no discontinuations in the trial.
Meanwhile, the data readout prompted Wal Street to compare TransCon CNP with Voxzogo, an FDA-approved therapy for achondroplasia developed by BioMarin Pharmaceutical (BMRN).
The results seem “competitive but not superior” to Voxzogo, Cowen analyst Yaron Werber said, with a Market Perform rating on ASND. “The benefit appears primarily driven by 2-5-year-olds vs 5-10-year-olds, which is surprising as Voxzogo’s data in younger patients has been lackluster and its data in 5-11-year-olds was robust,” Werber added.
However, the analyst acknowledged “data is difficult to interpret apples-to-apples given the lack of baseline (AHV) and the difference in endpoints.”
“BMRN’s dominance in achondroplasia is likely to continue,” Truist analyst Robyn Karnauskas with a Buy rating on BMRN, said, adding that data from the competitor “falls short” of the threshold expected by the key opinion leader.
Stifel analyst Paul Matteis who has a Buy rating on BMRN, agrees. “The Voxzogo launch has been one of the better orphan drug commercial rollouts ever, and we think the ASND data should now enable BMRN shares to price-in full credit for the asset’s success,” he added.
Monday’s selloff of Ascendis (ASND) shares comes even after the company announced the submission of a marketing application for TransCon PTH (palopegteriparatide) in Europe for adults with the rare hormone disorder hypoparathyroidism.