In this article, we will discuss: 13 Biotech Stocks with Huge Upside Potential.

Biotech and biopharmaceutical firms are involved in the research, discovery, development, and production of novel drugs and drug-related technology.

The FDA Commissioner Marty Makary stated the FDA will reach its 2025 drug approval targets despite severe workforce reductions. “The FDA will have no problem approving new drugs on time,” Makary asserted during a Jefferies investor conference. That is the agency’s top priority. He underlined that the cuts maintained essential regulatory skills and had no effect on scientific reviewers or inspectors. Makary has made transparency a top priority since taking over in April, and he has started a “listening tour” to interact with leaders in the biotech and pharmaceutical industries. He pointed to a move away from closed decision-making and toward more transparent communication, saying, “We’ve tried to create a glass box instead of a black box pretty much every single day.

The FDA has adopted a more cautious approach to COVID-19 vaccinations under its direction, imposing more stringent clearance criteria and requiring more information before authorizing booster shots for healthy people. These policy changes are part of a larger effort to strengthen scientific integrity and public trust.

With that said, here are the 13 Biotech Stocks with Huge Upside Potential.

Our Methodology

For this list, we compiled an initial list of 20 Biotech stocks from different credible sources. Then we selected the 13 stocks that had the highest upside potential as of June 9, 2025. We have only included stocks in our list with an upside potential of 6% or higher. The stocks are ranked in ascending order of the upside potential.

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).

13. Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY)

Analysts’ Upside Potential as of June 9: 6.68%

Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) declared on June 9, 2025, that AMVUTTRA® (vutrisiran) has been approved by the European Commission to treat wild-type or hereditary ATTR amyloidosis with cardiomyopathy (ATTR-CM). It is one of the stocks with biggest upside.

As a result, AMVUTTRA is the first and only RNAi treatment approved in the EU for transthyretin amyloidosis symptoms that show as cardiomyopathy and polyneuropathy. The HELIOS-B Phase 3 research, which revealed a 36% mortality reduction over 42 months and a 28% decrease in all-cause mortality and recurrent cardiovascular events, served as the basis for the approval.

More than 6,000 patient-years of safety data support the quarterly administration of the medication. The drug is already authorized in Brazil and the United States.

Up to 100,000 individuals in Europe have ATTR amyloidosis, mostly as cardiomyopathy. An RNAi treatment called vutrisiran provides continuous knockdown by stopping TTR production at its source with quarterly injections.

Functional and quality-of-life gains were validated by the HELIOS-B trial, which involved patients on SGLT2 inhibitors and tafamidis. Elevations of liver enzymes and injection site responses are frequent adverse effects.

Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is an innovator in the investigation of RNA interference medicines.

12. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)

Analysts’ Upside Potential as of June 9: 14.83%

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) shared new data at the 48th European Cystic Fibrosis Conference, revealing that ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor) is linked with improved clinical outcomes and quality of life in cystic fibrosis (CF) patients.

A pooled research found that sweat chloride (SwCl) levels below 60 mmol/L were associated with improved lung function, fewer exacerbations, and better nutritional and quality of life outcomes. Even larger numerical improvements were observed for SwCl below 30 mmol/L. According to post hoc data from Phase 3 trials, ALYFTREK outperformed TRIKAFTA® in terms of health outcomes for adults, adolescents, and children aged 6 to 11.

Approximately 109,000 people worldwide, 94,000 of whom live in North America, Europe, and Australia, have cystic fibrosis. The majority of patients have at least one F508del mutation, which is the result of mutations in the CFTR gene.

TRIKAFTA® is being donated in 14 lower-income countries, and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) medications currently serve over 75,000 people in 60+ countries, or almost two-thirds of eligible patients. It is among the stocks with the biggest upside.

11. ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD)

Analysts’ Upside Potential as of June 9: 21.08%

ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD) declared that the U.S. Court of Appeals maintained the legality of its composition of matter patent for the medication Nuplazid, which treats psychosis in Parkinson’s disease.

The verdict supports ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD) in its legal struggle with India-based MSN Laboratories and upholds a December 2023 ruling by the U.S. District Court of Delaware. A later patent claim with the same priority date cannot be declared invalid by the court.

According to CEO Catherine Owen Adams, Nuplazid’s patent protection has been extended to 2030. The 34 mg capsule is also protected by a separate formulation patent that runs through 2038.

The U.S. FDA authorized Nuplazid in 2016 to treat delusions and hallucinations linked to psychosis in Parkinson’s disease. ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD)’s legal stance in a different action against Aurobindo Pharma was further strengthened by the latest decision.

When taken as a whole, these results solidify the company’s portfolio of intellectual property in the face of generic competition, guaranteeing prolonged market exclusivity for its flagship neurological medication.

ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD) is a biopharmaceutical firm that specializes in the creation and marketing of medications that treat uncommon diseases and disorders of the central nervous system.

10. Ascendis Pharma A/S (NASDAQ:ASND)

Analysts’ Upside Potential as of June 9: 24.42%

Ascendis Pharma A/S (NASDAQ:ASND) reported interim Week 26 data from its Phase 2 COACH Trial, confirming that combining TransCon hGH (lonapegsomatropin) with TransCon CNP (navepegritide) raised annualized growth velocity (AGV) and height Z-scores in children with achondroplasia.

A mean AGV of 9.14 cm/year and a +0.53 Z-score rise were attained by treatment-naïve youngsters (N=12). Children (N=9) treated with TransCon CNP in the past achieved an AGV of 8.25 cm/year with a +0.44 Z-score improvement. The majority of side effects were modest, and safety remained consistent with monotherapies.

The COACH Trial is the first to assess this combination in kids between the ages of two and eleven. While TransCon CNP is undergoing FDA priority review, TransCon hGH is approved as SKYTROFA® for juvenile growth hormone insufficiency. The treatment focuses on the FGFR3-CNP signaling imbalance that is at the heart of achondroplasia, a disorder that affects more than 250,000 people worldwide.

It is anticipated that the results of week 52 and a scheduled Phase 3 trial in Q4 2025 will further clarify efficacy and long-term benefits.

The biopharmaceutical business Ascendis Pharma A/S (NASDAQ:ASND) uses its TransCon technology platform to significantly improve patient outcomes. It is one of the stocks with the biggest upside.

9. Insmed Incorporated (NASDAQ:INSM)

Analysts’ Upside Potential as of June 9: 35.72%

Kelly Shi of Jefferies has given Insmed Incorporated (NASDAQ:INSM) a buy recommendation, stating that she is confident in the TPIP treatment’s impending Phase 2 data for pulmonary arterial hypertension.

Shi anticipates a substantial decrease in pulmonary vascular resistance, the main outcome of the trial, of 20%, which is regarded as a definite success. If the results are encouraging, TPIP may replace Tyvaso, the current standard treatment.

On June 4, Wells Fargo reaffirmed its Buy recommendation, with a price objective of $107.

Shi points out that TPIP’s once-daily dosage is a significant benefit over Tyvaso’s four-times-daily schedule, which could increase adherence and make it more appealing to patients as well as physicians. The efficacy potential of TPIP is supported by interim study results that show additional advantages in six-minute walk distance (6MWD).

Shi expects significant market penetration in light of these considerations, which could lead to an increase in Insmed Incorporated (NASDAQ:INSM)’s valuation. Both analysts highlight that if future trial results live up to predictions, TPIP has the potential to completely change the way that PAHs are treated.

Insmed Incorporated (NASDAQ:INSM) is a biopharmaceutical firm that improves the lives of individuals suffering from serious and rare diseases.

8. BridgeBio Pharma, Inc. (NASDAQ:BBIO)

Analysts’ Upside Potential as of June 9: 46.96%

In a recent report, Raghuram Selvaraju, an expert at H.C. Wainwright, raised the price target for BridgeBio Pharma, Inc. (NASDAQ:BBIO) from $53 to $56 and kept the stock’s buy rating.

Positive momentum for the company is proven by the fast uptake of the Attruby product, which is the driving force behind this bullish forecast.

For adult patients with ATTR-CM, Attruby is the only nearly full (≥90%) stabilizer of Transthyretin (TTR) that has been approved in the United States to lower cardiovascular mortality and hospitalizations linked to cardiovascular disease. In general, Attruby was highly received. Abdominal pain and diarrhea were the most frequent, moderate side effects that went away without stopping the medication. BridgeBio Pharma, Inc. (NASDAQ:BBIO) provides a wide range of initiatives to facilitate patients’ access to our medications.

BridgeBio Pharma, Inc. (NASDAQ:BBIO) is a novel biopharmaceutical business that was established to find, develop, test, and distribute revolutionary medications to treat people with hereditary disorders. The development programs in its pipeline span from early science to advanced clinical trials. BBIO is among the stocks with the biggest upside.

7. Metsera, Inc. (NASDAQ:MTSR)

Analysts’ Upside Potential as of June 9: 54.66%

Metsera, Inc. (NASDAQ:MTSR) reported encouraging Phase 1 results for MET-233i, its once-monthly amylin analog, which showed a mean weight loss of up to 8.4% at Day 36 after subtracting the placebo.

The candidate supported monthly dosing with the longest half-life of any known amylin analog, 19 days. MET-233i had no safety indications and was well tolerated. The trial included 80 overweight or obese participants, with individual cases resulting in weight loss of up to 10.2%.

MET-233i displayed high tolerability and dose-linear pharmacokinetics when tested in both single and multiple ascending dose formats. The majority of adverse gastrointestinal events occurred in the first week and were mild and dose-dependent.

According to preliminary findings, it might make it practical to use its fully-biased GLP-1 RA candidate, MET-097i, in the first monthly GLP-1 + amylin combination therapy. Topline results from combination trials and an ongoing monotherapy study with MET-097i are anticipated in late 2025. Metsera, Inc. (NASDAQ:MTSR)’s HALO™ peptide stabilization platform supports the program.

Metsera, Inc. (NASDAQ:MTSR) is a clinical-stage biopharmaceutical business focused on developing new treatments for obesity and metabolic diseases. It is one of the stocks with the biggest upside.

6. Axsome Therapeutics, Inc. (NASDAQ:AXSM)

Analysts’ Upside Potential as of June 9: 55.54%

The FDA’s Division of Anesthesiology, Addiction Medicine, and Pain Medicine has issued a Refusal to File letter for Axsome Therapeutics, Inc. (NASDAQ:AXSM) AXS-14 new drug application.

The submission was deemed incomplete by the agency due to the use of an 8-week primary endpoint in one of the two supporting trials and a flexible-dose schedule. Vikram Purohit, an analyst at Morgan Stanley, stated that Axsome Therapeutics, Inc. (NASDAQ:AXSM) intends to start a fresh 12-week primary endpoint trial with a fixed dose in Q4. The stock is still rated as overweight by Morgan Stanley, which has set a price target of $190.

AXS-14’s regulatory setback is viewed as a temporary setback. The regulatory development of AXS-05 for Alzheimer’s disease agitation and the commercial success of Auvelity in major depressive disorder are seen by Morgan Stanley as Axsome Therapeutics, Inc. (NASDAQ:AXSM)’s primary 2025 value drivers.

The company is nevertheless optimistic about Axsome Therapeutics, Inc. (NASDAQ:AXSM)’s overall pipeline momentum and prospects for near-term revenue growth despite the AXS-14 filing struggle.

Axsome Therapeutics, Inc. (NASDAQ:AXSM) is a biopharmaceutical business in the clinical stage. It is involved in the development of new treatments for conditions of the central nervous system or CNS, for which there are few available options. The business is among the stocks with the biggest upside.

5. Jazz Pharmaceuticals plc (NASDAQ:JAZZ)

Analysts’ Upside Potential as of June 9: 62.69%

Jazz Pharmaceuticals plc (NASDAQ:JAZZ) is one of the stocks with the biggest upside. It presented late-breaking Phase 4 data on Xywav® at SLEEP 2025, which included findings from the XYLO and DUET trials.

In the XYLO study (n=43), moving from high-sodium to low-sodium oxybate resulted in a substantial decrease in 24-hour ambulatory systolic blood pressure (−4.1 mmHg; P=0.0019). SBP reduction during the day (−5.1 mmHg; P=0.0003) and while seated (−9.2 mmHg; P<0.0001) were secondary objectives.

The TEAEs were moderate or mild. DUET data from 24 subjects taking 9-12g Xywav nightly displayed improvement in excessive daytime sleepiness and narcolepsy severity with an average dose of 11.2g during the stable period.

Xywav is the sole FDA-approved low-sodium oxybate for cataplexy or EDS in narcolepsy and idiopathic hypersomnia. Xywav reduces nightly sodium intake by up to 92% compared to Xyrem®, lowering cardiovascular risk, which is a serious issue in narcolepsy patients. Both indications were awarded Orphan Drug Exclusivity by the FDA.

Jazz Pharmaceuticals plc (NASDAQ:JAZZ)’s data underscores how crucial it is to lower sodium intake in long-term sleep disorder treatment plans.

4. Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN)

Analysts’ Upside Potential as of June 9: 65.04%

JPMorgan has maintained its Overweight rating on Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) after reducing its price objective from $950 to $800.

Following poor IL-33 data in late May, the company believes that the recent selloff in Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) shares is “very much overdone.”

JPMorgan pointed out that IL-33 was never a key component of its model, though. The company stressed that, even without accounting for any pipeline contribution, Dupixent, Eylea, and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN)’ solid cash position alone warrants a valuation far higher than current levels. JPMorgan anticipates several catalysts over the next six months that will help reset its value.

Recently, the firm’s valuation was under pressure due to its IL-33 program’s lackluster Phase results. JPMorgan is still optimistic in spite of this, concentrating on important assets like Dupixent and Eylea that support the company’s financial stability. The company’s updated $800 estimate, which reflects confidence in Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN)’s core business and future potential milestones, still suggests significant upside.

Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) discovers, develops, and commercializes drugs to treat eye disease, cardiovascular disease, cancer, and inflammation. It is one of the stocks with the huge upside potential.

3. Avidity Biosciences, Inc. (NASDAQ:RNA)

Analysts’ Upside Potential as of June 9: 106.43%

Raymond James has started coverage of Avidity Biosciences, Inc. (NASDAQ:RNA), with a Strong Buy rating and a price objective of $65.

The rating is part of a larger biotech sector note, in which the firm sees favorable risk/reward across the board. Raymond James points out that less de-risked platforms can produce the most outsized returns, even if companies with de-risked, high probability-of-success assets are frequently viewed with greater certainty.

The firm stresses its confidence in del-zota, which is anticipated to be the first medicine from Avidity Biosciences, Inc. (NASDAQ:RNA)’s antibody oligonucleotide conjugate (AOC) platform to be approved.

Avidity Biosciences, Inc. (NASDAQ:RNA) is becoming more well-known for their AOC platform, which targets muscle disorders by combining oligonucleotides and antibodies. According to Raymond James, one of the platform’s top candidates, Del-Zota, has a strong chance of being approved.

The $65 objective is in line with the firm’s opinion that early-stage, less-risked assets can yield higher returns and points out faith in the platform’s revolutionary potential.

Avidity Biosciences, Inc. (NASDAQ:RNA) is a biopharmaceutical firm dedicated to developing a novel type of RNA treatment known as Antibody Oligonucleotide Conjugates, or AOCs. It is ranked third on our list of the stocks with the biggest upside.

2. NewAmsterdam Pharma Company N.V. (NASDAQ:NAMS)

Analysts’ Upside Potential as of June 9: 112.45%

NewAmsterdam Pharma Company N.V. (NASDAQ:NAMS) is now under Stifel’s coverage, with a buy rating and a $44 price target.

According to the firm, NewAmsterdam Pharma Company N.V. (NASDAQ:NAMS) is a distinct late-stage cardiovascular company with a short-term trajectory toward regulatory filings. Its core asset, obicetrapib, has the potential to differentiate itself in the growing lipid-lowering market, according to Stifel.

The analyst points out that obicetrapib potentially differs from earlier attempts in the CETP inhibitor market due to the company’s cardiovascular outcomes trial, PREVAIL. Stifel anticipates significant upside even under cautious commercial assumptions.

The CETP inhibitor obicetrapib, which is being developed by NewAmsterdam Pharma Company N.V. (NASDAQ:NAMS), is intended to decrease cholesterol in the treatment of cardiovascular disease. Stifel cites the PREVAIL study and an extensive amount of supporting data to show that obicetrapib differs significantly from previous CETP failures. New Amsterdam has a legitimate route to market access due to this distinction and impending regulatory milestones.

Stifel’s $44 price estimate shows that they are confident in the drug’s clinical uniqueness and market potential.

1. Cytokinetics, Incorporated (NASDAQ:CYTK)

Analysts’ Upside Potential as of June 9: 114.07%

Tessa Romero, an analyst at JPMorgan, has maintained her Overweight rating on Cytokinetics, Incorporated (NASDAQ:CYTK) but reduced the firm’s price objective from $71 to $53.

Aficamten’s revised revenue projections, which anticipate a slower launch trajectory, are the basis for the modification. The management’s latest remarks, which reflect the Camzyos launch pace for obstructive hypertrophic cardiomyopathy (oHCM), are consistent with this reevaluation. According to JPMorgan, expectations for adoption will be significantly influenced by the final medicine label.

Cytokinetics, Incorporated (NASDAQ:CYTK) experimental treatment for oHCM, Aficamten, is still a valuable tool with promising clinical outcomes. Although JPMorgan admits that the launch trajectory was slower than anticipated, the company is still confident in the drug’s unique profile when compared to Camzyos, especially with regard to pharmacology, safety, and dosing flexibility.

These qualities and corroborating evidence support JPMorgan’s assessment of aficamten’s acceptability. The lower price objective is not a reflection of a shift in opinion about the drug’s strategic worth or efficacy, but rather of modified commercial ramp assumptions.

Cytokinetics, Incorporated (NASDAQ:CYTK) is one of the best biotech stocks on our list. It is a late-stage biopharmaceutical business that focuses on discovering, developing, and bringing to market first-in-class muscle activators and next-in-class muscle inhibitors as possible cures for fatal diseases where muscle function is impaired or deteriorating.

While we acknowledge the potential of CYTK 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 CYTK and that has 100x upside potential, check out our report about this cheapest AI stock.

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