13 Biotech Stocks with Huge Upside Potential

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10. Ascendis Pharma A/S (NASDAQ:ASND)

Analysts’ Upside Potential as of June 9: 24.42%

Ascendis Pharma A/S (NASDAQ:ASND) reported interim Week 26 data from its Phase 2 COACH Trial, confirming that combining TransCon hGH (lonapegsomatropin) with TransCon CNP (navepegritide) raised annualized growth velocity (AGV) and height Z-scores in children with achondroplasia.

A mean AGV of 9.14 cm/year and a +0.53 Z-score rise were attained by treatment-naïve youngsters (N=12). Children (N=9) treated with TransCon CNP in the past achieved an AGV of 8.25 cm/year with a +0.44 Z-score improvement. The majority of side effects were modest, and safety remained consistent with monotherapies.

The COACH Trial is the first to assess this combination in kids between the ages of two and eleven. While TransCon CNP is undergoing FDA priority review, TransCon hGH is approved as SKYTROFA® for juvenile growth hormone insufficiency. The treatment focuses on the FGFR3-CNP signaling imbalance that is at the heart of achondroplasia, a disorder that affects more than 250,000 people worldwide.

It is anticipated that the results of week 52 and a scheduled Phase 3 trial in Q4 2025 will further clarify efficacy and long-term benefits.

The biopharmaceutical business Ascendis Pharma A/S (NASDAQ:ASND) uses its TransCon technology platform to significantly improve patient outcomes. It is one of the stocks with the biggest upside.

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