Top 5 Small-cap Pharma Stocks in 2026
Today’s pharmaceutical stocks are facing the challenges of government-imposed drug price caps, waning demand for COVID-19 vaccines and global stock market upheaval. However, the industry’s major underlying drivers — higher rates of cancer and chronic disease — are still at play and not expected to dissipate.
The US reigns supreme in the pharma market, both in terms of drug demand and development. In 2025, 46 novel medicines were approved by the US Food and Drug Administration (FDA), compared to 50 such approvals in 2024.
Big pharma largely steals the show when people discuss pharmaceutical companies, but some small- and mid-cap NASDAQ pharma stocks have also made gains.
Below the Investing News Network profiles the top five small-cap pharma stocks on the NASDAQ by year-to-date share price performance. Data was compiled on November 7, 2026, using TradingView’s stock screener, and pharma companies with market caps between US$50 million and US$500 million at that time were considered.
Read on to learn more about their activities this year.
1. AN2 Therapeutics (NASDAQ:ANTX)
Year-to-date gain: 279.46 percent
Market cap: US$160.38 million
Share price: US$4.25
AN2 Therapeutics develops pill-based drugs built around boron, an element that can make covalent bonds that are still reversible, according to the company.
Its lead candidate, epetraborole, is an inhibitor of bacterial leucyl-tRNA synthetase being studied for multiple indications, including treatment of mycobacterium abscessus, a hard-to-treat lung infection. The company is now also testing it in polycythemia vera, a rare blood cancer in which the body makes too many red blood cells.
On November 2, AN2 received US Food and Drug Administration clearance to test epetraborole in patients with polycythemia vera. It now has approval to run the Phase 2 trial in the US, Australia and India, and it expects to start the trial during Q4. Two days later, the company announced it selected its AN2-504661 as a development candidate for hypophosphatasia, a rare inherited disease that weakens bones and teeth. It expects the candidate to be ready for human testing in the second half of 2027. In its Q2 2026 results released in August, AN2 said its cash should fund operations into 2029.
2. Turn Therapeutics (NASDAQ:TTRX)
Year-to-date gain: 133.89 percent
Market cap: US$299.22 million
Share price: US$9.80
Turn Therapeutics develops precision treatments for inflammatory skin diseases that work on the skin’s surface rather than through the whole body. Its lead candidate, GX-03, is a long-acting skin treatment built on polyhexanide, a common antiseptic. It is being tested in moderate-to-severe eczema.
In June, Turn reported interim results from the first 50 patients in its Phase 2 trial. Most patients treated with GX-03 saw the severity of their eczema at least halved within four weeks. The company then expanded the Phase 2 trial to about 120 to 135 patients…
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