In this article, we will look at the top 14 Biotech Stocks with High Potential.
On October 1, Evan Seigerman, head of healthcare research at BMO Capital Markets, appeared on CNBC’s “The Exchange” to talk about what the future may be like for healthcare stocks.
Healthcare was the second worst performer this year, with pharma and biotech companies under pressure. However, the market is speculating whether the sector is poised to bring value or is a value trap under increased clarity regarding the Trump administration’s drug pricing efforts.
READ ALSO: 13 Most Undervalued Retail Stocks to Invest In and 13 Most Oversold Large Cap Stocks So Far in 2025.
Discussing this scenario, Seigerman opined that deals could be struck in the domain, which could help the sector. He added that valuations had been suppressed for a long time due to fears regarding drug pricing and tariff implementation. However, some of these fears have been uplifted with the Trump/Pfizer press conference offering a clearer path forward.
Seigerman further stated that while the dwindling uncertainty is a significant positive, it is too early to claim whether the cloud on the sector has fully lifted. It has, however, provided meaningful clarity.
With these trends in view, let’s look at the best biotech stocks with high potential.
Our Methodology
We used stock screeners to make a list of the best biotech stocks with high analyst upside potential and selected the top 13 with the highest upside. We also added the number of hedge fund holders for each stock as of Q2 2025, sourcing the hedge fund sentiment data from Insider Monkey’s database. The list is sorted in ascending order of analyst upside.
Note: All data was sourced on October 6.
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 373.4% since May 2014, beating its benchmark by 218 percentage points (see more details here).
14 Biotech Stocks with High Potential
14. Summit Therapeutics Inc. (NASDAQ:SMMT)
Analyst Upside: 60.99%
Number of Hedge Fund Holders: 30
Summit Therapeutics Inc. (NASDAQ:SMMT) is one of the best biotech stocks with high potential. In a report released on September 29, Leerink Partners maintained a Sell rating on Summit Therapeutics Inc. (NASDAQ:SMMT) without assigning a price target.
In addition, Barclays also maintained a Sell rating on Summit Therapeutics Inc. (NASDAQ:SMMT) on September 25 with a $13 price target.
However, Asthika Goonewardene from Truist Financial maintained a Buy rating on Summit Therapeutics Inc. (NASDAQ:SMMT) the same day, without assigning a price target.
In addition to Asthika Goonewardene, Citi’s Yigal Nochomovitz also expressed bullish sentiments for Summit Therapeutics Inc. (NASDAQ:SMMT) in a report released on September 24, assigning the stock a Buy rating with a $35 price target.
Summit Therapeutics Inc. (NASDAQ:SMMT) is a biopharmaceutical company that specializes in creating novel medications for infectious and cancerous diseases.
13. IDEAYA Biosciences, Inc. (NASDAQ:IDYA)
Analyst Upside: 62.81%
Number of Hedge Fund Holders: 32
IDEAYA Biosciences, Inc. (NASDAQ:IDYA) is one of the best biotech stocks with high potential. On September 23, J.P. Morgan analyst Anupam Rama maintained a bullish stance on IDEAYA Biosciences, Inc. (NASDAQ:IDYA), giving the stock a Buy rating with a $74 price target.
The analyst based the rating on several promising developments associated with the company’s lead asset, darovasertib, stating that IDEAYA Biosciences, Inc. (NASDAQ:IDYA) is anticipated to release notable updates on darovasertib in several treatment settings. This is expected to support significant value creation.
In another report released on September 22, UBS analyst David Dai reiterated a Buy rating on IDEAYA Biosciences, Inc. (NASDAQ:IDYA) and set a price target of $50.00.
IDEAYA Biosciences, Inc. (NASDAQ:IDYA) develops oncology-focused precision medicine, focusing on targeted therapeutics for patients selected using molecular diagnostics. The company’s product candidate is IDE196, a protein kinase C inhibitor that targets genetically defined cancers with GNAQ or GNA11 gene mutations.
12. Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX)
Analyst Upside: 63.15%
Number of Hedge Fund Holders: 32
Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) is one of the best biotech stocks with high potential. HC Wainwright reiterated a Buy rating on Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) on September 17 and set a $35 price target.
Similarly, in a report released on September 11, Jason Gerberry from Bank of America Securities also reiterated a Buy rating on Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) and set a price target of $34.00.
The analyst consensus rating on Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) is a Strong Buy, with the stock’s median price target of $20.71 implying an upside of 64.17% from current levels.
Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) is a commercial-stage, biopharmaceutical company involved in the development, in-licensing, and commercialization of novel medicines for patients with difficult-to-treat and rare diseases.
11. Beam Therapeutics Inc. (NASDAQ:BEAM)
Analyst Upside: 66.99%
Number of Hedge Fund Holders: 31
Beam Therapeutics Inc. (NASDAQ:BEAM) is one of the best biotech stocks with high potential. H.C. Wainwright analyst Patrick Trucchio reiterated a Buy rating on Beam Therapeutics Inc. (NASDAQ:BEAM) on September 15, retaining the price target of $80.
The analyst supported the optimistic rating with the company’s strong momentum in its in vivo and ex vivo base editing programs.
Similarly, in a report released on September 10, Kostas Biliouris from BMO Capital also maintained a Buy rating on Beam Therapeutics Inc. (NASDAQ:BEAM) and set a price target of $40.00.
Biliouris supported the optimistic rating with the company’s technological advancements and strong position in the AATD market, adding that management’s confidence in the competitive positioning of BEAM-302 is a crucial reason for the bullish stance.
Beam Therapeutics Inc. (NASDAQ:BEAM) is a biotechnology company that develops precision genetic medicines through base editing, a technology that allows efficient and predictable single-base changes at targeted genomic sequences. The company’s lead programs focus on sickle cell disease and alpha-1 antitrypsin deficiency.
Beam Therapeutics Inc. (NASDAQ:BEAM) is advancing programs in other genetic diseases as well, along with oncology and immunology.
10. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)
Analyst Upside: 74.09%
Number of Hedge Fund Holders: 58
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best biotech stocks with high potential. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported total revenues of $825 million in fiscal Q2 2025, up 16% year-over-year and up 17% year-over-year at constant currency. GAAP diluted earnings per share (EPS) for the quarter reached $1.23.
In a report released on September 17, Christopher Raymond from Raymond James maintained a Buy rating on BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) and set a price target of $85.00.
In addition to Raymond James, Wedbush’s Yun Zhong also expressed bullish sentiments for BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) in a report released on September 8, assigning the stock a Buy rating. However, the same day, H.C. Wainwright initiated coverage of the stock with a Hold rating and a $60 price target.
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) develops and commercializes therapies for serious and life-threatening medical conditions and rare diseases. The company’s product pipeline includes Valoctocogene roxaparvovec, Vosoritide, and BMN 307.
9. Ocular Therapeutix Inc. (NASDAQ:OCUL)
Analyst Upside: 75.28%
Number of Hedge Fund Holders: 27
Ocular Therapeutix Inc. (NASDAQ:OCUL) is one of the best biotech stocks with high potential. On October 3, Piper Sandler raised the firm’s price target on Ocular Therapeutix Inc. (NASDAQ:OCUL) to $31 from $21 while keeping an Overweight rating on the shares.
The rating update came after the firm hosted Ocular Therapeutix Inc.’s (NASDAQ:OCUL) management at an investor dinner after its investor day, discussing aspects such as the newly disclosed diabetic retinopathy pivotal trial designs and the anticipated SOL-1 readout in Q1 2026.
The firm further stated that it raised its peak risk-adjusted sales estimates for AXPAXLI from $1.9 billion to $3.2 billion, based on the increased confidence in the wet AMD and NPDR Phase 3 programs.
Ocular Therapeutix Inc. (NASDAQ:OCUL) is a biopharmaceutical company that develops and commercializes therapies for conditions and diseases related to the eye. The company’s product pipeline includes Dextenza, OTX-TP, and OTX-TIC.
8. Amicus Therapeutics, Inc. (NASDAQ:FOLD)
Analyst Upside: 82.93%
Number of Hedge Fund Holders: 40
Amicus Therapeutics, Inc. (NASDAQ:FOLD) is one of the best biotech stocks with high potential. On September 18, Needham analyst Gil Blum upgraded the rating on Amicus Therapeutics, Inc. (NASDAQ:FOLD) to a Buy, setting a price target of $14.00.
Similarly, Morgan Stanley analyst Maxwell Skor also maintained a Buy rating on Amicus Therapeutics, Inc. (NASDAQ:FOLD) on September 17, setting a $12.00 price target.
The analyst consensus rating on Amicus Therapeutics, Inc. (NASDAQ:FOLD) is a Strong Buy, and the stock’s median price target of $8.41 implies an upside of 78.25% from current levels.
Amicus Therapeutics, Inc. (NASDAQ:FOLD) is a biotech company that discovers, develops, and delivers medicines to treat metabolic diseases.
Its product portfolio includes the first and only approved oral precision medicine to treat Fabry disease, a clinical-stage treatment paradigm for Pompe disease, and a rare disease gene therapy portfolio.
7. Crinetics Pharmaceuticals, Inc. (NASDAQ:CRNX)
Analyst Upside: 98.24%
Number of Hedge Fund Holders: 28
Crinetics Pharmaceuticals, Inc. (NASDAQ:CRNX) is one of the best biotech stocks with high potential. On October 1, Cantor Fitzgerald analyst Joshua Schimmer maintained a Buy rating on Crinetics Pharmaceuticals, Inc. (NASDAQ:CRNX) and set a price target of $90.00.
In other news, Crinetics Pharmaceuticals, Inc. (NASDAQ:CRNX) announced the FDA approval of PALSONIFYTM (paltusotine) as a “first-line treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option” on September 25.
PALSONIFY is a selectively-targeted somatostatin receptor type 2 nonpeptide (SST2) agonist, and management stated that the FDA approval makes it the first once-daily, oral treatment approved for acromegaly in adults.
Crinetics Pharmaceuticals, Inc. (NASDAQ:CRNX) is a clinical-stage pharmaceutical company that develops and commercializes novel therapeutics for rare endocrine diseases and endocrine-related tumors.
The company’s product candidate, CRN00808, is an oral treatment for acromegaly. It is also developing other oral treatments for neuroendocrine tumors, hyperinsulinism, and Cushing’s disease.
6. Soleno Therapeutics, Inc. (NASDAQ:SLNO)
Analyst Upside: 102.67%
Number of Hedge Fund Holders: 52
Soleno Therapeutics, Inc. (NASDAQ:SLNO) is one of the best biotech stocks with high potential. In a report released on October 3, Yasmeen Rahimi from Piper Sandler maintained a Buy rating on Soleno Therapeutics, Inc. (NASDAQ:SLNO) and set a price target of $145.00.
Soleno Therapeutics, Inc. (NASDAQ:SLNO) reported in its fiscal Q2 2025 results that it used $12.6 million of cash in its operating activities during the three months ended June 30, 2025. The company had $293.8 million of cash, cash equivalents, and marketable securities at quarter end
In addition, cash at the end of fiscal Q2 2025 excluded $230 million of gross proceeds that Soleno Therapeutics, Inc. (NASDAQ:SLNO) raised in July through an underwritten offering of common stock.
Soleno Therapeutics, Inc. (NASDAQ:SLNO) also reported that product revenue, net, for the quarter reached $32.7 million.
Soleno Therapeutics, Inc. (NASDAQ:SLNO) is a clinical-stage biopharmaceutical company that develops and commercializes novel therapeutics to treat rare diseases.
The company focuses on the treatment of neurobehavioral and metabolic disorders. Its lead candidate, Diazoxide Choline Controlled-Release (DCCR), is an oral tablet that treats Prader-Willi Syndrome (PWS).
5. Legend Biotech Corporation (NASDAQ:LEGN)
Analyst Upside: 137.06%
Number of Hedge Fund Holders: 39
Legend Biotech Corporation (NASDAQ:LEGN) is one of the best biotech stocks with high potential. On October 3, TD Cowen analyst Yaron Werber reiterated a Buy rating on Legend Biotech Corporation (NASDAQ:LEGN), keeping the price target at $62.00.
The analyst supported the optimistic rating with the company’s future growth potential and strategic positioning.
He stated that while Legend Biotech Corporation (NASDAQ:LEGN) is experiencing supply constraints with its Carvykti product at present, management is confident about the company’s potential to overcome these limitations by the end of 2025, supported by planned capacity expansions at key sites.
According to him, this confidence can be seen in Legend Biotech Corporation’s (NASDAQ:LEGN) expectations to meet the consensus sales target of $1.9 billion for the fiscal year 2025.
Legend Biotech Corporation (NASDAQ:LEGN) is a clinical-stage company that develops, discovers, manufactures, and commercializes novel therapies for oncology and other indications.
It develops advanced cell therapies across an elaborate range of technology platforms. The company operates in the US, China, and other geographical segments.
4. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)
Analyst Upside: 157.93%
Number of Hedge Fund Holders: 60
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the best biotech stocks with high potential. On October 3, Bank of America Securities analyst Tazeen Ahmad maintained a Buy rating on Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and set a price target of $79.00.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) reported $166 million in total revenue for fiscal Q2 2025, with Crysvita® revenue of $120 million and Dojolvi® revenue of $23 million.
The company also reaffirmed its 2025 revenue guidance and now expects total revenue in the $640 million to $670 million range
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops, acquires, and commercializes novel products to treat genetic diseases. Its product portfolio includes Crysvita, Mepsevii, Dojolvi, and Evkeeza.
3. Immunovant, Inc. (NASDAQ:IMVT)
Analyst Upside: 163.31%
Number of Hedge Fund Holders: 31
Immunovant, Inc. (NASDAQ:IMVT) is one of the best biotech stocks with high potential. On September 30, JPMorgan analyst Brian Cheng lowered the firm’s price target on Immunovant, Inc. (NASDAQ:IMVT) to $33 from $37 while keeping an Overweight rating on the shares.
Immunovant, Inc. (NASDAQ:IMVT) reported that its cash and cash equivalents totaled approximately $598.9 million in the quarter ending June 30, 2025, providing runway for announced indications through GD readout expected in 2027.
Immunovant, Inc. (NASDAQ:IMVT) develops treatments for autoimmune diseases. Its product pipeline includes batoclimab and IMVT-1402. Both are novel antibodies targeting the neonatal fragment crystallizable receptor (FcRn).
2. Vaxcyte, Inc. (NASDAQ:PCVX)
Analyst Upside: 171.92%
Number of Hedge Fund Holders: 44
Vaxcyte, Inc. (NASDAQ:PCVX) is one of the best biotech stocks with high potential. In a report released on October 3, Jason Gerberry from Bank of America Securities maintained a Buy rating on Vaxcyte, Inc. (NASDAQ:PCVX) and set a price target of $134.00.
In other news, Vaxcyte, Inc. (NASDAQ:PCVX) reported on September 30 that it entered into a new agreement with Thermo Fisher Scientific Inc. to “bring additional fill-finish commercial manufacturing for Vaxcyte’s broad-spectrum pneumococcal conjugate vaccines (PCVs) to the United States”.
Management reported that Thermo Fisher would “provide custom commercial fill-finish capacity for the Company’s broad-spectrum PCVs at its Greenville, North Carolina facility”.
The agreement, including both manufacturing and related services, marks a long-term US commercial manufacturing commitment for Vaxcyte, Inc. (NASDAQ:PCVX), representing up to $1 billion.
Vaxcyte, Inc. (NASDAQ:PCVX) is a clinical-stage vaccine innovation company that develops high-fidelity vaccines. It develops broad-spectrum conjugate and novel protein vaccines to prevent or treat bacterial infectious diseases.
The company re-engineers the creation of highly complex vaccines through advanced chemistry and modern synthetic techniques.
1. Viking Therapeutics, Inc. (NASDAQ:VKTX)
Analyst Upside: 236.47%
Number of Hedge Fund Holders: 43
Viking Therapeutics, Inc. (NASDAQ:VKTX) is one of the best biotech stocks with high potential. H.C. Wainwright analyst Joseph Pantginis reiterated a bullish stance on Viking Therapeutics, Inc. (NASDAQ:VKTX) on September 22, giving the stock a Buy rating with a $102 price target.
The analyst supported the rating with Viking Therapeutics, Inc.’s (NASDAQ:VKTX) promising market position, stating that the company’s competitive efficacy and safety data from its oral and subcutaneous GLP-1 programs take the lead as its notable strengths.
The same day, BTIG analyst Justin Zelin also reiterated a Buy rating on Viking Therapeutics, Inc. (NASDAQ:VKTX) and set a $125.00 price target, supporting the rating with the company’s solid position in the metabolic disease and obesity market.
Viking Therapeutics, Inc. (NASDAQ:VKTX) is a clinical-stage company that develops therapies for metabolic and endocrine disorders.
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