No matter the market environment, you’ll always find entertainment in the biotech space. There will always be biotech stocks making huge swings in both directions and these stocks can make or break your portfolio in a very small amount of time due to the nature of how important clinical trial results are for these companies.
The United Nations forecasts that the over-65 age group will grow by 150% from 800 million in 2024 to 2 billion by 2067. This “older, richer & sicker” population is expected to push global healthcare spending on medicines up 12% to $2.3 trillion by 2028. If you combine that with AI making breakthroughs in healthcare more frequent, you’ll see many more biotech stocks starting to pop to the upside over the coming years.
So far into 2025, here are 15 that have done just that. More gains could come if the positive news driving these stocks sticks around.
Scientist in a lab working on a research project, focusing on biotechnology and healthcare advancements.
Methodology
For this article, I screened the top-performing biotech stocks year-to-date.
I will also mention the number of hedge fund investors in these stocks. 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).
15. Vigil Neuroscience Inc (NASDAQ:VIGL)
Number of Hedge Fund Holders In Q4 2024: 18
Vigil Neuroscience Inc (NASDAQ:VIGL) is a biotech company that makes treatments for rare and common neurodegenerative diseases by restoring the vigilance of microglia, the sentinel immune cells of the brain.
The stock is up significantly so far in 2025 in anticipation of upcoming clinical trial data readouts in the first half of 2025.
It already reported positive Phase 1 clinical trial data for VG-3927 (small molecule TREM2 agonist) for Alzheimer’s disease. Plus, a final analysis from the IGNITE Phase 2 clinical trial evaluating iluzanebart in adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is planned for Q2 2025.
In addition, it surged following announcements about promising progress in clinical trials for Alzheimer’s and ALSP in January.
The consensus price target of $19.75 implies 699.6% upside.
VIGL is up 45% year-to-date.
14. SpringWorks Therapeutics Inc (NASDAQ:SWTX)
Number of Hedge Fund Holders In Q4 2024: 40
SpringWorks Therapeutics Inc (NASDAQ:SWTX) is a biopharma company making medicines for people with severe rare diseases and cancer.
The stock is up significantly so far in 2025 due to advanced acquisition talks with Merck KGaA.
Furthermore, on February 11, 2025, the FDA approved GOMEKLI™ (mirdametinib), SpringWorks’ MEK inhibitor, for the treatment of adult and pediatric patients 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.
With the above approval, SpringWorks was granted a rare pediatric disease priority review voucher (PRV) by the FDA.
The consensus price target of $73.2 implies 37.05% upside.
SWTX is up 47.80% year-to-date.
13. Axsome Therapeutics Inc (NASDAQ:AXSM)
Number of Hedge Fund Holders In Q4 2024: 44
Axsome Therapeutics Inc (NASDAQ:AXSM) is a biopharmaceutical company that makes treatments for CNS disorders that don’t have reliable alternative treatments.
The stock is up significantly so far in 2025 due to the settlement of patent litigation with Teva Pharmaceuticals regarding AUVELITY, their treatment for major depressive disorder. This agreement prevents Teva from launching a generic version until at least September 2038 (or March 2039 if pediatric exclusivity is granted).
Another major catalyst was the FDA approval of SYMBRAVO for the acute treatment of migraine with or without aura in adults. This was announced in early 2025.
The company also has positive Phase 3 clinical trials for two key pipeline candidates for Alzheimer’s and narcolepsy.
The consensus price target of $165.36 implies 30.81% upside.
AXSM is up 49.39% year-to-date.
12. BeiGene Ltd. (NASDAQ:ONC)
Number of Hedge Fund Holders In Q4 2024: 30
BeiGene Ltd. (NASDAQ:ONC) is a company that makes cancer treatments.
The stock is up significantly so far in 2025 after solid Q4 and full-year results.
It reported global revenue growth of 78% year-over-year to $1.1 billion in Q4 2024. Full-year revenue grew 55% to $3.8 billion.
The company also guided for $4.9-5.3 billion in 2025 revenue and exceeded Wall Street’s expectations of $4.74 billion. It also anticipates positive GAAP operating income and cash flow.
ONC is up 50.71% year-to-date.
11. GH Research PLC (NASDAQ:GHRS)
Number of Hedge Fund Holders In Q4 2024: 10
GH Research PLC (NASDAQ:GHRS) is a biopharma company working on psychiatric and neurological disorders by developing novel and proprietary mebufotenin (5-MeO-DMT) therapies for conditions like treatment-resistant depression.
The stock is up significantly so far in 2025 after the announcement of positive Phase 2b clinical trial results for their lead product candidate GH001 in treatment-resistant depression (TRD).
It showed a significant placebo-adjusted MADRS reduction of -15.5 on Day 8 and an impressive 57.5% remission rate compared to 0% in the placebo group. 78% of participants sustained remission.
The consensus price target of $30.6 implies 187.32% upside.
GHRS is up 52.14% year-to-date.
10. Absci Corp (NASDAQ:ABSI)
Number of Hedge Fund Holders In Q4 2024: 18
Absci Corp (NASDAQ:ABSI) sells itself as an AI-integrated drug company.
The stock is up significantly so far in 2025 as it announced a collaboration with AMD. This included a $20 million strategic investment from AMD through private investment in public equity (PIPE) and will involve deploying AMD Instinct™ accelerators and ROCm™ software to power Absci’s AI drug discovery workloads.
It also partnered up with Invetx in January and advanced its ABS-101 drug and has plans to submit an Investigational New Drug application in Q1 2025.
The consensus price target of $8.57 implies 112.16% upside.
ABSI is up 54.58% year-to-date.
9. Precigen Inc (NASDAQ:PGEN)
Number of Hedge Fund Holders In Q4 2024: 10
Precigen Inc (NASDAQ:PGEN) is a company that makes gene and cell therapies with precision technology. It targets diseases in therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases.
The stock is up significantly so far in 2025 as the FDA granted Priority Review to the company’s Biologics License Application (BLA) for PRGN-2012.
This is an investigational AdenoVerse gene therapy that could treat adults with recurrent respiratory papillomatosis (RRP). This announcement came on February 25, 2025, with the FDA setting a Prescription Drug User Fee Act (PDUFA) target action date of August 27, 2025.
The consensus price target of $7 implies 300% upside.
PGEN is up 55.36% year-to-date.
8. Palvella Therapeutics Inc (NASDAQ:PVLA)
Number of Hedge Fund Holders In Q4 2024: 9
Palvella Therapeutics Inc (NASDAQ:PVLA) is a clinical-stage biopharmaceutical company that makes therapies for rare genetic skin diseases with no FDA-approved therapies.
The stock is up significantly so far in 2025 after Palvella’s announcement on February 10, 2025, that it would expand its Phase 3 SELVA clinical trial of QTORIN™ rapamycin for microcystic lymphatic malformations to include younger pediatric patients aged 3 to 5 years old.
Before that, Palvella announced the publication of results from its Phase 2 clinical trial of QTORIN™ rapamycin for microcystic lymphatic malformations in the Journal of Vascular Anomalies, reporting that 100% of participants were either “Much Improved” or “Very Much Improved” as rated by the Clinician Global Impression of Change.
It also announced that the first patients had been dosed in its Phase 2 TOIVA clinical trial of QTORIN™ rapamycin for the treatment of cutaneous venous malformations.
The consensus price target of $40.33 implies 113.97% upside.
PVLA is up 56.17% year-to-date.
7. Sana Biotechnology Inc (NASDAQ:SANA)
Number of Hedge Fund Holders In Q4 2024: 24
Sana Biotechnology Inc (NASDAQ:SANA) is a biotech company that makes engineered cells as medicines. It is making therapies for conditions including type 1 diabetes, autoimmune diseases, and cancer.
The stock is up significantly so far in 2025 as it announced positive clinical results from its type 1 diabetes study. The company reported the successful transplantation of UP421, which is an allogeneic primary islet cell treatment using their hypoimmune technology in a type 1 diabetes patient without requiring immunosuppressive drugs.
This caused the stock to skyrocket.
The consensus price target of $14.25 implies 450.19% upside.
SANA is up 58.90% year-to-date.
6. Tiziana Life Sciences Ltd (NASDAQ:TLSA)
Number of Hedge Fund Holders In Q4 2024: N/A
Tiziana Life Sciences Ltd (NASDAQ:TLSA) is a biotech company that makes immunomodulation therapies. Its lead candidate is a fully human anti-CD3 monoclonal antibody for treating neurodegenerative and inflammatory diseases. It is called intranasal foralumab.
The stock is up significantly so far in 2025 due to the publication of a study in Nature Neuroscience showing positive results for nasal anti-CD3 therapy in traumatic brain injury.
Moreover, it announced that its Long COVID study using foralumab is nearing completion in Q2 2025.
It also announced a product development services agreement with Renaissance Lakewood. This is a contract development and manufacturing organization specializing in nasal drug delivery.
TLSA is up 62.31% year-to-date.
5. INmune Bio Inc (NASDAQ:INMB)
Number of Hedge Fund Holders In Q4 2024: 10
INmune Bio Inc (NASDAQ:INMB) is a biotech company that makes treatments that target the innate immune system to fight disease, with two main product platforms: the Dominant-Negative Tumor Necrosis Factor (DN-TNF) platform and the Natural Killer Cell Priming Platform.
The stock is up significantly so far in 2025 as it announced plans to submit a Biologics License Application (BLA) to the FDA seeking approval of CORDStrom for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) in pediatric patients.
This announcement followed positive results from the MissionEB study. INmune Bio also plans to submit Marketing Authorization Applications (MAAs) to the European Union and UK in 2026.
The consensus price target of $22.8 implies 190.45% upside.
INMB is up 68.09% year-to-date.
4. Akero Therapeutics Inc (NASDAQ:AKRO)
Number of Hedge Fund Holders In Q4 2024: 41
Akero Therapeutics Inc (NASDAQ:AKRO) is a biotech company making treatments for metabolic diseases, particularly focusing on metabolic dysfunction-associated steatohepatitis (MASH).
The stock is up significantly so far in 2025 after it announced Phase 2b SYMMETRY study results showing a statistically significant reversal of compensated cirrhosis (F4) due to MASH after 96 weeks of treatment with efruxifermin (EFX).
Among patients with baseline and week 96 biopsies, 39% of the 50mg EFX group showed ≥1 stage improvement in fibrosis with no worsening of MASH. The company also posted its Q4 earnings results very recently.
The consensus price target of $75.86 implies 58.86% upside.
AKRO is up 71.75% year-to-date.
3. Genelux Corp (NASDAQ:GNLX)
Number of Hedge Fund Holders In Q4 2024: 8
Genelux Corp (NASDAQ:GNLX) is a biopharma company making oncolytic viral immunotherapies for patients suffering from aggressive and/or difficult-to-treat solid tumor types.
The stock is up significantly so far in 2025 as investors are optimistic about the company’s OnPrime Phase 3 registrational trial in platinum-resistant/refractory ovarian cancer. Results are expected in the second half of 2025.
Also, interim data from the NSCLC (non-small cell lung cancer) trial is expected in mid-2025. Topline results from the Phase 3 OnPrime/GOG-3076 registration trial in platinum-resistant/refractory ovarian cancer (PRROC) are expected in the second half of 2025.
The consensus price target of $18.25 implies 323.43% upside.
GNLX is up 82.63% year-to-date.
2. Compass Therapeutics Inc (NASDAQ:CMPX)
Number of Hedge Fund Holders In Q4 2024: 14
Compass Therapeutics Inc (NASDAQ:CMPX) is a biopharmaceutical company that makes antibody-based therapies for several diseases.
The stock is up significantly so far in 2025 due to it reporting 2024 results and its anticipated top-line Phase 2/3 data readout for COMPANION-002, which is evaluating tovecimig (CTX-009, a DLL4 x VEGF-A bispecific antibody) in patients with biliary tract cancer (BTC). It is expected by the end of the first quarter of 2025.
In addition, Compass Therapeutics announced in January 2025 that it is advancing a new drug candidate, CTX-10726, a PD-1 x VEGF-A bispecific antibody, with IND submission expected by the end of 2025.
The consensus price target of $11.38 implies 289.55% upside.
CMPX is up 101.38% year-to-date.
1. Invivyd Inc (NASDAQ:IVVD)
Number of Hedge Fund Holders In Q4 2024: 14
Invivyd Inc (NASDAQ:IVVD) is a biopharmaceutical company that makes antibody-based therapies for infectious diseases.
The stock is up significantly so far in 2025 after the announcement of positive Phase 1/2 clinical data for VYD2311. This monoclonal antibody candidate is designed to be a better alternative to COVID-19 vaccination for the broad population.
The clinical trial showed more favorable safety and tolerability compared to vaccines, with no activation of the subject immune systems, and demonstrated potential as a novel, potent, long-acting COVID-19 treatment option.
On the same day, Invivyd announced preliminary fourth quarter 2024 financial results showing strong revenue growth. Their PEMGARDA™ (pemivibart) product grew revenue by 48% to $13.8 million.
The consensus price target of $7.89 implies 536.09% upside.
IVVD is up 179.85% year-to-date.
While we acknowledge the potential of IVVD as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than IVVD but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.
READ NEXT: Why These 15 E-Commerce Stocks Are Skyrocketing So Far In 2025 and Why These 15 Insurance Stocks Are Skyrocketing So Far In 2025
Disclosure: None. Insider Monkey focuses on uncovering the best investment ideas of hedge funds and insiders. Please subscribe to our free daily e-newsletter to get the latest investment ideas from hedge funds’ investor letters by entering your email address below.
