In this article, we will list the 5 Best Biotech Stocks with Highest Upside Potential. Please visit 10 Best Biotech Stocks with Highest Upside Potential if you’d like to see an extended list and how we came up with the list of best biotechnology stocks.
5. Legend Biotech Corp. (NASDAQ:LEGN)
Legend Biotech Corp. (NASDAQ:LEGN) is one of the 10 best biotech stocks with highest upside potential.
On April 20, H.C. Wainwright maintained its Buy rating for Legend Biotech Corp. (NASDAQ:LEGN). The firm has projected a target price of $50, which yields more than 109% potential upside for investors. It highlighted the company’s CAR-T therapies and attributed its bullish view of the stock to the ongoing progress related to those programs. It noted that the trial data for vivo CD19/CD20 CAR-T will be revealed soon.
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The firm also reflected on the management’s strong indication of critical medical meeting readouts, which appear to be imminent in the foreseeable future. These relate to the preliminary dosages given to patients during last summer, leading to 6 months of data for patients who have undergone the longest treatments.
H.C. Wainwright also acknowledged the continued strength shown by the CARVYKTI electronic order book during the entirety of the first quarter. With favorable operational momentum witnessed from January through April, the firm labelled the IQVIA prescription data oscillation as mere external noise. Such momentum is in line with the company’s 64% topline growth, exceeding $1 billion during the prior 12 months.
Legend Biotech Corp. (NASDAQ:LEGN) is a commercial-stage biopharmaceutical company that develops innovative therapies. Being an end-to-end cell therapy company, they focus on medication for serious diseases such as hematologic malignancies and solid tumors.
4. Crinetics Pharmaceuticals Inc. (NASDAQ:CRNX)
Crinetics Pharmaceuticals Inc. (NASDAQ:CRNX) is one of the 10 best biotech stocks with highest upside potential.
As of the April 24 closing, the stock carries a strongly bullish consensus sentiment. With a price target of $86.71, it yields more than 126% upside potential. It received Buy ratings from all 8 analysts who provided coverage.
On March 26, Crinetics Pharmaceuticals Inc. (NASDAQ:CRNX) filed a Marketing Authorization Application with the National Health Surveillance Agency in Brazil for Palsonify, a novel once-daily orally administered somatostatin receptor subtype 2 selective non-peptide agonist, for the treatment of acromegaly in adult patients.
Founder and CEO of Crinetics, Scott Struthers, has observed that this application marks yet another landmark on a global level. The once-a-day oral Palsonify medication has revolutionized the treatment protocol after its approval by the FDA in the United States. The submission is based on data collected from eighteen clinical trials, two of which were Phase 3 trials with positive results for both primary and secondary endpoints.
The drug was highly tolerable, producing biochemical and symptomatic control, compared to placebo. In Europe, the CHMP recently issued a positive opinion in relation to the MAA application of Crinetics.
Crinetics Pharmaceuticals Inc. (NASDAQ:CRNX) is engaged in developing and marketing of innovative treatments for rare endocrine diseases and endocrine-related tumors. The company offers a range of therapies that are currently in different stages of clinical trials. These include Paltusotine, Atumelnant, CRN09682, oral GLP-1 and GIP nonpeptide.
3. Structure Therapeutics Inc. (NASDAQ:GPCR)
Structure Therapeutics Inc. (NASDAQ:GPCR) is one of the 10 best biotech stocks with highest upside potential.
As of April 24 closing, the stock carries a strongly bullish consensus sentiment, receiving Buy ratings from all 14 analysts. Based on a 1-year median target price of $107.77, it currently offers an upside potential of almost 138%.
Back on March 16, Structure Therapeutics Inc. (NASDAQ:GPCR) released encouraging results of the Phase 2 ACCESS II study conducted on its investigational product called aleniglipron. It is an oral GLP-1 receptor agonist for use against obesity. It achieved 16.3% weight reduction when taken at 180 mg and 16% weight loss when taken at a higher dose of 240 mg at 44 weeks.
The open-label extension trial reported further weight reduction up to 16.2% for the 120 mg dose after 56 weeks without any sign of weight loss plateauing. It should be mentioned that the lower 2.5 mg starting dose significantly enhanced the tolerability of the drug by lowering the rate of adverse events associated with drug discontinuations to only 2 to 3.4%.
The end-of-phase 2 meeting with the FDA is scheduled to take place during the second quarter of 2026, while phase 3 is still set to start in the second half of 2026. According to Chief Executive Raymond Stevens, the data shows that the company’s drug is significantly different from others because it achieves the highest amount of weight loss in an oral GLP-1 drug.
Structure Therapeutics Inc. (NASDAQ:GPCR) is involved in the development and marketing of novel oral small molecule therapies for the treatment of several chronic diseases with unmet medical needs. The company is currently working on several drug candidates, which include GSBR-1290, ACCG-2671, ACCG-3535, LTSE-2578, and ANPA-0073. Additionally, it also develops programs like the GCG and GIPR programs.
2. Ocular Therapeutix Inc. (NASDAQ:OCUL)
Ocular Therapeutix Inc. (NASDAQ:OCUL) is one of the 10 best biotech stocks with highest upside potential.
On April 13, William Blair maintained an Outperform rating on Ocular Therapeutix (NASDAQ:OCUL) right after the presentation of additional SOL-1 clinical trial data. According to the firm, this post-hoc investigation unequivocally supports Axpaxli’s overall effectiveness and durability. In particular, starting at week 8, the treatment showed prolonged disease control over aflibercept, resulting in improvements in the median time to more than or equal to 30-micrometer and 75-micrometer increases in central subfield thickness at 23 and 22 weeks.
Additionally, patients with poorer initial baseline measurements showed higher treatment improvements, according to quartile analyses contextualizing visual acuity increases. The underlying safety characteristics are essentially unchanged. In addition to publishing detailed patient-level information about past intraocular inflammation instances, the company has said that drug particles that were first classified as vitreous floaters were totally resorbed after a 20-week mean duration.
William Blair reiterated that Sustained anatomic control is a crucial leading signal of vision loss and significantly influences therapy choices within this particular indication. Based only on these stand-alone SOL-1 trial findings, the company is now moving closer to submitting a formal New Drug Application.
Ocular Therapeutix Inc. (NASDAQ:OCUL) focuses on developing and commercializing therapeutics for different eye conditions, including retinal diseases, using its bioresorbable hydrogel-based formulation technology. Its portfolio includes DEXTENZA, AXPAXLI in phase 3 of clinical trial, and OTX-TIC in phase 2 of clinical trial.
1. Viking Therapeutics Inc. (NASDAQ:VKTX)
Viking Therapeutics Inc. (NASDAQ:VKTX) is one of the 10 best biotech stocks with highest upside potential.
Back on March 26, Viking Therapeutics Inc. (NASDAQ:VKTX) reported the final recruitment of patients into the Phase 3 VANQUISH-2 study of its investigational subcutaneous drug VK2735, a dual agonist of the GLP-1 and GIP receptors. The drug is being studied for oral and subcutaneous administration in the treatment of various metabolic diseases.
Furthermore, Viking is performing the Phase 3 VANQUISH-1 trial of VK2735 administered subcutaneously to patients for the treatment of obesity or being overweight. The enrollment for the VANQUISH-1 trial was completed in November 2025. Additionally, Viking is performing a Phase 1 trial assessing multiple maintenance dosing strategies for VK2735. The primary goals of the trial involve assessing the safety, tolerability, and pharmacokinetics of VK2735 administered under various dosing schemes.
Secondary goals measure the body weight changes from the baseline and after the switch to maintenance dosing. The results of this trial are expected to be announced in the third quarter of 2026.
Viking Therapeutics Inc. (NASDAQ:VKTX) specializes in metabolic and endocrine disorder therapies. As a clinical-stage biopharmaceutical company, they direct their research efforts towards small-molecule drugs to cure obesity, fatty liver, and diabetes.
While we acknowledge the potential of VKTX to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than VKTX and that has 100x upside potential, check out our report about the cheapest AI stock.
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