2025 was a year characterized by both challenges and opportunities for the biotechnology sector. Despite volatility, the segment remained on a high-growth trajectory, driven by several clinical and operational breakthroughs across categories. However, going forward, the segment is expected to face significant regulatory, political, and financial uncertainties.
On January 8, Boston Consulting Group published a detailed report titled “Biopharma Trends 2026,” highlighting what is in store for the sector for the coming year. This includes a significant shift in R&D spend, with greater focus on large populations and real-world applicability. Additionally, the industry will continue to see an uptick in M&A activity, as many biopharma companies pursue inorganic growth strategies. Another key priority for companies will be cost-optimization measures to help them achieve target margins. The report indicates a challenging landscape for biopharma players, which will require them to rethink their business models, incorporate AI in their operations, and maintain their focus on innovative therapies. That is the only way these businesses can sustain themselves.
Across public markets, the highly volatile nature of biotechnology stocks presents both risks and attractive opportunities. With careful selection, these stocks can help portfolios outperform the broader market by a wide margin. However, navigating this highly competitive, rapidly evolving segment remains a challenge for investors.
With that background, let’s explore our 17 Biotechnology Stocks With More Than 50% Upside.
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Our Methodology
To identify relevant stocks for this article, we screened U.S.-listed biotechnology companies with market capitalizations above $2 billion and with share prices above $5. Next, we identified stocks with at least 50% upside potential based on TipRanks consensus as of the February 12 closing. Finally, we selected 17 stocks with the highest upside and ranked them in ascending order.
Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter’s strategy selects 14 small-cap and large-cap stocks every quarter and has returned 427.7% since May 2014, beating its benchmark by 264 percentage points (see more details here).
17. BioMarin Pharmaceutical (NASDAQ:BMRN)
Number of Hedge Fund Holders: 54
Upside Potential: 47.3%
BioMarin Pharmaceutical (NASDAQ:BMRN) is one of the 17 biotechnology stocks with more than 50% upside.
As of February 12 closing, BioMarin Pharmaceutical (NASDAQ:BMRN) carried a moderately bullish analyst sentiment. Of the 15 analysts covering the stock, 10 assigned Buy ratings and 5 issued Hold ratings. With no Sell rating, the stock has a projected median 1-year price target of $88.29, implying more than 47% upside.
On February 6, Allison Bratzel from Piper Sandler reiterated her Overweight rating on BioMarin Pharmaceutical (NASDAQ:BMRN). The analyst reduced her price target on the stock from $122 to $84. Despite this downward revision, her estimates still imply upside potential of more than 40% from the prevailing level. Bratzel’s rating incorporates recent fourth-quarter pre-releases, which also prompted Piper Sandler to adjust its estimates and price targets for some commercial names.
BioMarin Pharmaceutical (NASDAQ:BMRN) develops and commercializes targeted therapies for life-threatening medical conditions and rare genetic diseases. Some of its major products include VIMIZIM, VOXZOGO, NAGLAZYME, and ALDURAZYME. The company operates in more than 70 countries and currently has several drugs in the development stage.
16. Apogee Therapeutics (NASDAQ:APGE)
Number of Hedge Fund Holders: 30
Upside Potential: 52.5%
Apogee Therapeutics (NASDAQ:APGE) is one of the 17 biotechnology stocks with more than 50% upside.
On January 25, Citi analyst Geoff Meacham maintained a Buy rating on Apogee Therapeutics (NASDAQ:APGE), with a $95 price target. His forecast indicates potential upside of more than 37%.
Selloff in Apogee Therapeutics (APGE) stock, following the release of Sanofi’s Phase 3 amlitelimab data, is viewed as a market overreaction by Meacham. He maintains that data readout does not negatively affect prospects for the company’s APG279. This candidate is a fixed-dose combination that targets both IL-13 and OX40L.
Back on January 22, Brian Abrahams from RBC Capital lowered his rating on Apogee Therapeutics (NASDAQ:APGE) from Outperform to Sector Perform. The analyst raised the price target from $70 to $83, implying nearly 20% upside.
Abrahams noted that the segment in which Apogee Therapeutics (NASDAQ:APGE) operates is “becoming increasingly crowded with well-entrenched existing players” despite the company’s primary drug, zumilokibart, showing potential as a long-acting IL-13 for atopic dermatitis. He reflected on the 108% surge in the share price over the past year, which limits the potential for additional gains.
Apogee Therapeutics (NASDAQ:APGE) is a clinical-stage biotechnology company developing novel biologics to treat inflammatory and immune diseases. These include atopic dermatitis, asthma, chronic obstructive pulmonary disease, eosinophilic esophagitis, and others. Its existing pipeline includes APG777, APG279, APG990, APG333, and APG808, all in different clinical trial stages.
15. Arcellx Incorporated (NASDAQ:ACLX)
Number of Hedge Fund Holders: 36
Upside Potential: 61.6%
Arcellx Incorporated (NASDAQ:ACLX) is one of 17 biotechnology stocks with more than 50% upside.
On February 12, Qize Ding from Rothschild & Co Redburn downgraded the rating on Arcellx Incorporated (NASDAQ:ACLX) from Buy to Neutral. In the process, he also reduced the price target from $113 to $82, which still yields a revised upside potential of almost 20%. Ding’s views are based on his conservative stance on the company’s CAR-T cell therapy class amid rising competition across larger markets in other modalities.
On February 9, Stifel analyst Stephen Willey reaffirmed his Buy rating on Arcellx Incorporated (NASDAQ:ACLX), with a $127 price target. His forecast leads to an upside potential of 85% from the current level.
Willey’s rating came after the company presented new laboratory study data at the TANDEM meeting. He highlighted that the findings support anito-cel’s potentially leading safety profile, showing lower unintended immune activity and fewer off-target effects than rival CAR-T treatments. The analyst also pointed to differences versus cilta-cel (Carvykti) and ide-cel (Abecma), suggesting its off-target interactions could be linked to severe delayed side effects seen in clinical use. Moreover, Willey believes the data strengthens anito-cel’s positioning as a potentially best-in-class therapy and continues to view the stock as a “core SMid-cap holding.”
Arcellx Incorporated (NASDAQ:ACLX) develops advanced immunotherapies for cancer patients and individuals with other incurable conditions. The company leverages its proprietary D-Domain technology to develop more effective treatments that target AML, multiple myeloma, and other malignancies. The current pipeline includes the development of ACLX-001 and ACLX-002, which target BCMA in rrMM and CD123 in AML/MDS.
