## Promising Small-Cap Pharma Stocks: A Closer Look at Five Medicine Share Price Winners in 2025

The pharmaceutical industry is navigating significant headwinds — price regulation pressures, declining COVID-19 vaccine demand, and market volatility. Yet beneath these challenges lies a persistent reality: cancer rates and chronic disease prevalence continue climbing globally. While large pharmaceutical companies dominate headlines, several smaller players on the NASDAQ have quietly delivered exceptional returns to investors, with select medicine share price performances reaching triple-digit gains.

This analysis examines five small-cap pharma stocks that outperformed the broader market in 2025, based on year-to-date performance data compiled on December 29, 2025. The focus covers companies with market capitalizations between US$50 million and US$500 million.

### The Standout Performer: Galectin Therapeutics

Galectin Therapeutics (NASDAQ:GALT) emerged as the clear winner among the five, posting a remarkable 211.45 percent year-to-date gain with a share price of US$4.08 and market cap of US$263.08 million. The clinical-stage biopharma company has captured investor attention through its development of belapectin, a carbohydrate-based therapeutic targeting inflammatory, fibrotic, and malignant diseases.

The breakthrough came with positive Phase 2b/3 trial results released in 2025 for patients with metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis. Belapectin demonstrated the ability to significantly reduce esophageal varices development and stabilize liver stiffness — potentially halting disease progression. Following FDA fast-track designation, the company indicated alignment with regulators on patient populations for its pivotal Phase 3 registration trials. This regulatory clarity has fueled investor confidence in the medicine's pathway to approval.

### Mid-Tier Gains: CytomX Therapeutics and Its Platform Advantage

CytomX Therapeutics (NASDAQ:CTMX) delivered the second-highest return at 136.63 percent, trading at US$2.38 per share with a US$375.74 million valuation. The company's differentiated PROBODY platform generates localized biologics designed to minimize systemic toxicity — a critical advantage in oncology.

Two catalysts accelerated CytomX's share price appreciation in 2025. First, the company secured a US$100 million underwritten offering in May, signaling strong investor backing. More importantly, interim Phase 1 data for CX-2051 in advanced colorectal cancer proved compelling enough to warrant dose expansion studies. By mid-year, CytomX initiated Phase 1b combination trials pairing CX-2051 with bevacizumab. Additionally, first-patient dosing occurred for CX-801 combined with Keytruda in metastatic melanoma patients, with initial translational data released by November.

### Established Players With Slower But Steady Momentum

Eton Pharmaceuticals (NASDAQ:ETON) represents a different profile — a transition story from development-stage to commercial entity. While its 25.37 percent gain appears modest compared to peers, the US$450.53 million market cap and US$16.80 share price reflect a more mature business model. The 2025 launch of KHINDIVI, the first FDA-approved oral hydrocortisone solution, marked a key validation of Eton's commercialization capability. Additionally, successful relaunches of Increlex and Galzin expanded its revenue-generating portfolio to eight commercial products. With the FDA reviewing ET-600's new drug application for late February decision, 2026 could accelerate momentum.

Fennec Pharmaceuticals (NASDAQ:FENC) gained 20.91 percent, reaching US$7.69 with US$262.54 million in market cap. As the sole company with an FDA-approved therapy specifically preventing cisplatin-induced hearing loss in pediatric cancer patients, Fennec achieved record revenue growth, expanded internationally, and eliminated corporate debt. Recent Phase 2/3 data from Japan demonstrated significant hearing loss reduction, positioning the company for 2026 global registration. The company's entry into adult cancer indications signals potential for market expansion.

### The Ultra-Rare Disease Specialist

Zevra Therapeutics (NASDAQ:ZVRA) posted the most modest return at 5.25 percent, with a US$496.54 million market cap and US$8.82 share price. Yet this masks significant underlying momentum. A December distribution agreement with Uniphar expanded Miplyffa's global reach beyond the US and Europe. Q3 results showcased a stunning 605 percent year-over-year revenue increase, demonstrating strong market adoption of this Niemann-Pick disease type C therapy. The company's rebranding in 2023 reflects its evolution into a fully integrated pharmaceutical enterprise targeting high-unmet-need pediatric and metabolic disorders.

### Investment Takeaway

These five small-cap pharma stocks illustrate how medicine share price appreciation extends well beyond mega-cap manufacturers. Investors seeking exposure to healthcare innovation are finding opportunity in clinical-stage companies with clear FDA pathways and commercial-stage players generating early revenue traction. The convergence of unmet medical needs, regulatory clarity, and commercial execution continues driving investor interest in the small-cap pharma segment.