5 Small Cap Stocks with the Highest Upside Potential

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1. Annexon Inc. (NASDAQ:ANNX)

Average Upside Potential: 109.38%

Annexon Inc. (NASDAQ:ANNX)  is one of the small cap stocks with the highest upside potential. On March 30, the company reported its 2025 year-end financial results, highlighting a period of significant clinical and regulatory progress for its neuroinflammatory disease platform. The company ended the year with a strong balance sheet featuring $238.3 million in cash and short-term investments, a figure supported by an $86.3 million public offering in late 2025.

This capital is expected to provide a financial runway into H2 2027, supporting the advancement of two late-stage registrational programs and an emerging oral inhibitor. The company’s lead candidate, vonaprument, is currently being evaluated in the ARCHER II Phase 3 trial for geographic atrophy/GA, with topline data anticipated in Q4 2026. Unlike existing treatments that target downstream proteins, vonaprument utilizes an upstream C1q blockade designed to protect photoreceptor neurons and preserve vision.

The program has notably received PRIME designation from the European Medicines Agency, establishing a clear global regulatory path for what could be the first vision-preserving therapy for the millions affected by GA worldwide. Simultaneously, Annexon Inc. (NASDAQ:ANNX) is advancing tanruprubart for the treatment of Guillain-Barré Syndrome/GBS.

Annexon Inc. (NASDAQ:ANNX) is a clinical-stage biopharmaceutical company advancing a platform of targeted immunotherapies for neuroinflammatory and autoimmune diseases.

While we acknowledge the potential of ANNX to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than ANNX and that has 100x upside potential, check out our report about the cheapest AI stock.

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