Billionaire David E. Shaw’s 10 Small-Cap Stock Picks with Huge Upside Potential

3. PTC Therapeutics, Inc. (NASDAQ:PTCT)

D. E. Shaw’s Equity Stake: $69.62 Million

Market Capitalization as of May 9: $3.52 Billion

Stock Upside Potential as of May 9: 47.21%

Number of Hedge Fund Holders: 31

PTC Therapeutics, Inc. (NASDAQ:PTCT) is a biotechnology company that focuses on discovering, developing, and commercializing medicines for rare disorders. It offers Translarna and Emflaza for treating Duchenne muscular dystrophy; Upstaza for aromatic l-amino acid decarboxylase (AADC) deficiency, a central nervous system disorder. It is one of billionaire David E. Shaw’s 10 small-cap stock picks with huge upside potential as it continues to outperform the overall market.

Bank of America has already upgraded the stock to a Buy from Neutral and increased the price target to $68 from $55. The bullish stance comes as PTC Therapeutics, Inc. (NASDAQ:PTCT) reported a robust start to 2025, marked by significant progress in product launches and clinical trials. Revenue in the first quarter of 2025 totaled $190 million, with a significant portion coming from the Duchenne Muscular Dystrophy (DMD) franchise at $134 million.

PTC Therapeutics, Inc. (NASDAQ:PTCT) is on the cusp of diversifying its revenue base with the launch of SUFIANCE following a positive opinion from the CHMP. The company is targeting over $1 billion in revenue from the treatment. It’s also working on the PTC518 program for Huntington’s disease, which has already shown promising results in Phase 2 trials. The company remains in a solid cash position with over $2 billion that should support commercial and R&D activities.