Ascendis Soars on Encouraging Data From Dwarfism Drug Study
Shares of Ascendis Pharma ASND rose 17% on Monday after it reported top-line results from the pivotal ApproaCH study that evaluated investigational once-weekly administered TransCon CNP in children aged two to 11 years with achondroplasia, the most common form of dwarfism.
ASND Drug Shows AGV Benefit in Achondroplasia Patients
The ApproaCH study met its primary endpoint of annualized growth velocity (AGV) at 52 weeks — children treated with TransCon CNP achieved a mean AGV of 5.89 cm/year compared with 4.41 cm/year in the placebo group.
Management also provided sub-group analyses of this data by age groups — children aged two to less than five years and another group aged five to 11 years. Patients in the first group who received TransCon CNP achieved a mean AGV of 6.07 cm/year compared with 5.06 cm/year in placebo. Those who received the drug in the second age group achieved a mean AGV of 5.79 cm/year compared with 4.02 cm/year on placebo.
Per Ascendis, treatment with the drug also demonstrated statistically significant improvement in other secondary endpoints, including height Z-score and changing from baseline AGV.
But Why Did BioMarin Stock Dip on TransCon CNP Data
There is only one drug approved to treat achondroplasia in the United States and Europe — Voxzogo — which is marketed by BioMarin BMRN. Since its launch in 2021, BMRN has been experiencing a rapid uptake for Voxzogo driven by strong prescription demand. The drug added nearly $337 million to BioMarin’s topline in the first half of 2024, up 67% year over year.
While it is difficult to compare the results of two different clinical studies directly, investors were likely impressed with the once-weekly dosing advantage of TransCon CNP over Voxzogo, which requires daily dosing. This major factor was not only responsible for Ascendis’ share price soaring but also sent BioMarin stock down nearly 18% after the results were announced.
Ascendis’ shares have moved up 10.8% in the year compared with the industry’s 1.0% growth.
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Ascendis’ Future Plans for the Drug
Based on the ApproaCH study results, management intends to submit a regulatory filing seeking approval for TransCon CNP in achondroplasia with the FDA during first-quarter 2025. It also plans to submit a similar filing with the EMA by third-quarter 2025.
ASND’s Zacks Ranks
Ascendis currently carries a Zacks Rank #3 (Hold).
Ascendis Pharma A/S Price
Ascendis Pharma A/S price | Ascendis Pharma A/S Quote
Our Key Picks in the Biotech Sector
Some better-ranked stocks in the overall healthcare sector are Bioventus BVS and Illumina ILMN. Each stock presently sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Bioventus’ 2024 earnings per share (EPS) have risen from 27 cents to 40 cents. Estimates for 2025 have increased from 43 cents to 45 cents during the same period. Year to date, shares of Bioventus have surged 109.9%.
BVS’ earnings beat estimates in three of the last four quarters and missed the mark on one occasion. Bioventus delivered a four-quarter average earnings surprise of 102.86%.
In the past 60 days, estimates for Illumina’s 2024 EPS have risen from $1.84 to $3.62. Estimates for 2025 have increased from $3.22 to $4.43 during the same period. Year to date, Illumina’s shares have lost 7.0%.
Earnings of Illumina beat estimates in each of the last four quarters. Illumina delivered a four-quarter average earnings surprise of 463.46%.
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