10 Best Performing Small-Cap Stocks in 2024

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1. Avidity Biosciences Inc. (NASDAQ:RNA)

Year-to-Date Performance as of September 23: 388.73%

Market Cap as of September 23: $5.17 billion

Number of Hedge Fund Holders: 31

Avidity Biosciences Inc. (NASDAQ:RNA) a biopharmaceutical company engaged in the delivery of RNA therapeutics. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat diseases previously untreatable with RNA therapeutics, creating RNA-based drugs that can target and correct underlying genetic defects.

In June, Avidity initiated the global Phase 3 HARBORTM trial evaluating del-desiran for myotonic dystrophy type 1 (DM1). The FDA granted del-desiran Breakthrough Therapy designation for DM1 earlier in May.

In August, it reported positive initial data from the Phase 1/2 EXPLORE44™ trial evaluating del-zota (delpacibart zotadirsen), a potential treatment for Duchenne muscular dystrophy (DMD44). Del-zota demonstrated favorable safety and tolerability in 25 participants across two dose levels (5 mg/kg and 10 mg/kg). Enrollment for the EXPLORE44 trial is now complete, and the company plans to enroll additional patients in the EXPLORE44 Open-label Extension study (OLE).

The company also reported positive initial data from the Phase 1/2 FORTITUDE trial evaluating del-brax for facioscapulohumeral muscular dystrophy (FSHD) in this quarter. The trial demonstrated unprecedented and consistent reductions of over 50% in DUX4 regulated genes, trends of functional improvement, and favorable safety and tolerability.

The company faced some challenges and experienced an 11.70% year-over-year decline, due to a rather heavy focus on R&D investment. Management announced plans to unveil its lead target from its precision cardiology program by Q4 2024, indicating an expansion beyond rare neuromuscular diseases into broader therapeutic areas. Such expansions position the company to become a leader in its industry.

While we acknowledge the growth potential of Avidity Biosciences Inc. (NASDAQ:RNA), our conviction lies in the belief that AI stocks hold great promise for delivering high returns and doing so within a shorter timeframe. If you are looking for an AI stock that is more promising than RNA but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.

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