Ascendis Pharma's Rare Dwarfism Candidate Shows Promise In Trial, FDA Submission Expected Next Year

Ascendis Pharma A/S ASND released topline data on Monday from a trial that included 84 children with achondroplasia, a genetic disorder that causes dwarfism and disproportionate short stature.

TransCon CNP (navepegritide), an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to provide sustained release and continuous exposure of active CNP.

CNP regulates blood flow, blood pressure, and the reactivity of platelets and leukocytes.

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In the trial, children treated with once-weekly TransCon CNP demonstrated annualized growth velocity (AGV) superior to placebo.

• For the primary endpoint of AGV at Week 52, children treated with TransCon CNP (n=57) demonstrated an LS mean AGV of 5.89 cm/year compared to 4.41 cm/year in the placebo arm (n=27), an LS mean difference of 1.49 cm/year (p<0.0001).

Sub-group analyses:

• Children aged 2 to <5 years treated with TransCon CNP (n=21) demonstrated an LS mean AGV at Week 52 of 6.07 cm/year compared to 5.06 cm/year in the placebo arm.
• Children aged 5-11 treated with TransCon CNP (n=36) demonstrated an LS mean AGV of 5.79 cm/year at Week 52, compared to 4.02 cm/year in the placebo arm.
• Children aged 2 to <5 years treated with TransCon CNP (n=19) demonstrated a change from baseline AGV at Week 52 of 1.57 cm/year compared to 0.43 cm/year in the placebo arm.
• Children aged 5-11 treated with TransCon CNP (n=35) demonstrated a 2.29 cm/year change from baseline AGV at Week 52 compared to 0.52 cm/year in the placebo arm.

For the secondary endpoint of change in ACH Height Z-score, children treated with TransCon CNP (n=57) demonstrated an LS mean change from baseline ACH Height Z-score of 0.30 compared to 0.01 in the placebo arm.

For the secondary endpoint of change in CDC Height Z-score, children treated with TransCon CNP (n=55) demonstrated an LS mean change from baseline CDC Height Z-score of 0.15 compared to -0.15 in the placebo arm.

In the total trial population, treatment with TransCon CNP resulted in numerical improvements in health-related quality of life compared to placebo as measured in several Achondroplasia Child Experience Measure (ACEM) domains.

TransCon CNP continues to show a safety profile comparable to placebo and was generally well-tolerated, with generally mild treatment-emergent adverse events (TEAEs), no evidence of hypotensive effect, and a low frequency of injection site reactions, all mild.

Ascendis plans to submit an FDA marketing application for TransCon CNP for achondroplasia during the first quarter of 2025 and a European application during the third quarter of 2025.

Price Action: ASND stock is up 17.4% at $140 at last check Monday.

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