Ascendis Pharma’s Uncommon Dwarfism Candidate Exhibits Promise In Trial, FDA Submission Anticipated Subsequent Yr – Ascendis Pharma (NASDAQ:ASND)

Ascendis Pharma A/S ASND launched topline information on Monday from a trial that included 84 kids with achondroplasia, a genetic dysfunction that causes dwarfism and disproportionate quick stature.

TransCon CNP (navepegritide), an investigational prodrug of C-type natriuretic peptide (CNP) administered as soon as weekly and designed to supply sustained launch and steady publicity of lively CNP.

CNP regulates blood movement, blood strain, and the reactivity of platelets and leukocytes.

Additionally Learn: Ascendis Pharma Faces Challenges With Skytrofa And Yorvipath Pricing: Analysts Weigh In.

Within the trial, kids handled with once-weekly TransCon CNP demonstrated annualized progress velocity (AGV) superior to placebo.

• For the first endpoint of AGV at Week 52, kids handled with TransCon CNP (n=57) demonstrated an LS imply AGV of 5.89 cm/12 months in comparison with 4.41 cm/12 months within the placebo arm (n=27), an LS imply distinction of 1.49 cm/12 months (p<0.0001).

Sub-group analyses:

• Kids aged 2 to <5 years handled with TransCon CNP (n=21) demonstrated an LS imply AGV at Week 52 of 6.07 cm/12 months in comparison with 5.06 cm/12 months within the placebo arm.
• Kids aged 5-11 handled with TransCon CNP (n=36) demonstrated an LS imply AGV of 5.79 cm/12 months at Week 52, in comparison with 4.02 cm/12 months within the placebo arm.
• Kids aged 2 to <5 years handled with TransCon CNP (n=19) demonstrated a change from baseline AGV at Week 52 of 1.57 cm/12 months in comparison with 0.43 cm/12 months within the placebo arm.
• Kids aged 5-11 handled with TransCon CNP (n=35) demonstrated a 2.29 cm/12 months change from baseline AGV at Week 52 in comparison with 0.52 cm/12 months within the placebo arm.

For the secondary endpoint of change in ACH Peak Z-score, kids handled with TransCon CNP (n=57) demonstrated an LS imply change from baseline ACH Peak Z-score of 0.30 in comparison with 0.01 within the placebo arm.

For the secondary endpoint of change in CDC Peak Z-score, kids handled with TransCon CNP (n=55) demonstrated an LS imply change from baseline CDC Peak Z-score of 0.15 in comparison with -0.15 within the placebo arm.

Within the whole trial inhabitants, therapy with TransCon CNP resulted in numerical enhancements in health-related high quality of life in comparison with placebo as measured in a number of Achondroplasia Youngster Expertise Measure (ACEM) domains.

TransCon CNP continues to point out a security profile corresponding to placebo and was usually well-tolerated, with usually gentle treatment-emergent antagonistic occasions (TEAEs), no proof of hypotensive impact, and a low frequency of injection website reactions, all gentle.

Ascendis plans to submit an FDA advertising software for TransCon CNP for achondroplasia through the first quarter of 2025 and a European software through the third quarter of 2025.

Value Motion: ASND inventory is up 17.4% at $140 finally examine Monday.

Learn Subsequent: