Top 5 Small-cap Pharma Stocks (Updated January 2026)

1. Galectin Therapeutics (NASDAQ:GALT)

Year-to-date gain: 211.45 percent
Market cap: US$263.08 million
Share price: US$4.08

Galectin Therapeutics is developing therapies for patients with chronic liver disease and cancer.

The clinical-stage biopharma company's lead drug candidate, carbohydrate-based belapectin, targets multiple inflammatory, fibrotic and malignant diseases by inhibiting the galectin-3 protein. Belapectin has been granted fast-track designation by the FDA.

In 2025, Galectin Therapeutics reported positive topline data from its Phase 2b/3 trial evaluating the efficacy and safety of using belapectin intravenously in patients with metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis and portal hyper tension. The results demonstrated that belapectin significantly reduced the development of new esophageal varices and stabilized liver stiffness, demonstrating potential to halt the progression of MASH cirrhosis.

Galectin is currently designing its pivotal Phase 3 study intended to support a formal new drug application. Based on a December 2025 response from the FDA, the company said it believes it has achieved alignment with the agency on the patient population for its upcoming registration-level trials.

2. CytomX Therapeutics (NASDAQ:CTMX)

Year-to-date gain: 136.63 percent
Market cap: US$375.74 million
Share price: US$2.38

CytomX Therapeutics is a clinical-stage biopharma firm with a focus on developing safer, more effective oncology treatments. It collaborates with a number of leading oncology firms, including Amgen (NASDAQ:AMGN), Bristol-Myers Squibb (NYSE:BMY), Regeneron Pharmaceuticals (NASDAQ:REGN) and Moderna (NASDAQ:MRNA).

The company's pipeline is based on its PROBODY therapeutic platform, which it uses to produce localized biologics that target tumors. This includes multiple treatment modalities such as antibody-drug conjugates, T-cell engagers and immune modulators such as cytokines. Its clinical-stage pipeline includes CX-2051 and CX-801.

In mid-May 2025, CytomX's share price shot up significantly after the company provided its Q1 business update and closed on a US$100 million underwritten offering of common stock.

In the update, CytomX included positive interim clinical results for an ongoing Phase 1 dose escalation study of its lead candidate, CX-2051, in advanced colorectal cancer. The company has initiated further Phase 1 dose expansions, with data expected out by Q1 2026. In its Q3 update, CytomX reported it plans to initiate a Phase 1b study of CX-2051 in combination with bevacizumab to treat colorectal cancer, also expected in the first quarter of the new year.

On May 19, the first patient was dosed in CytomX's ongoing Phase 1 dose escalation study with CX-801 in combination with Merck & Company's (NYSE:MRK) Keytruda in patients with metastatic melanoma. The company released initial translational data in November.

3. Eton Pharmaceuticals (NASDAQ:ETON)

Year-to-date gain: 25.37 percent
Market cap: US$450.53 million
Share price: US$16.80

Eton Pharmaceuticals is a high-growth pharmaceutical company developing treatments for rare diseases. Headquartered in Deer Park, Illinois, the company has successfully transitioned from a development-stage firm into a commercially focused entity with a diversified portfolio of orphan drugs.

2025 included the successful launch of KHINDIVI, the first FDA-approved oral solution formulation of hydrocortisone, in June. KHINDIVI was approved in May for pediatric patients five and older with adrenocortical insufficiency. The company is looking to expand the indication to younger patients with a revised formulation, and a bioequivalence study is expected to begin in early 2026.

The year also included high-performing relaunches of acquired assets Increlex, which treats a rare condition in which a child’s body does not produce enough growth factor-1, and the zinc therapy Galzin, a maintenance treatment for Wilson disease.

As of December, its portfolio included eight commercial products and five in its pipeline. The FDA is reviewing its new drug application for ET-600, with a decision scheduled for late February.

4. Fennec Pharmaceuticals (NASDAQ:FENC)

Year-to-date gain: 20.91 percent
Market cap: US$262.54 million
Share price: US$7.69

Fennec Pharmaceuticals is a commercial-stage specialty pharmaceutical company focused on preventing ototoxicity, meaning permanent hearing loss, in pediatric cancer patients undergoing cisplatin-based chemotherapy.

The company’s sole commercial product, Pedmark, is the first and only FDA-approved therapy specifically indicated to reduce the risk of hearing loss associated with cisplatin in patients one month of age and older with non-metastatic solid tumors.

Fennec experienced a pivotal year in 2025, marked by record revenue growth, entry into international markets and the elimination of corporate debt.

Additionally, data from a Phase 2/3 clinical study in Japan showed a significant reduction in the percentage of patients who experienced hearing loss, setting the stage for a 2026 global registration.

The company also began exploring its first major expansion into the adult cancer market through a new trial in metastatic testicular cancer.

5. Zevra Therapeutics (NASDAQ:ZVRA)

Year-to-date gain: 5.25 percent
Market cap: US$496.54 million
Share price: US$8.82

Zevra Therapeutics is a commercial-stage rare disease company that utilizes data-driven strategies to develop and commercialize transformational therapies for ultra-rare conditions.

Formerly known as KemPharm, the company rebranded in 2023 to reflect its evolution into a fully integrated pharmaceutical entity with a focus on high-unmet-need pediatric and metabolic disorders.

At the end of December, Zevra executed a strategic distribution agreement with Uniphar to provide its flagship product, Miplyffa, to patients outside of the US and Europe, broadening the drug's global footprint. Miplyffa was approved by the FDA in 2024, and is indicated as a treatment for Niemann-Pick disease type C administered in combination with miglustat.

This announcement followed a strong Q3, in which the company reported a 605 percent year-over-year revenue increase, largely driven by the early success of Miplyffa.