Investing in Biotech Stocks in 2025

Axsome's revenue is soaring thanks to strong momentum for two drugs -- Auvelity and Sunosi. The company launched Auvelity in October 2022 as a treatment for major depressive disorder. It acquired sleep disorder drug Sunosi from Jazz Pharmaceuticals (NASDAQ:JAZZ) in May 2022.

In January 2025, Axsome added a third product to its lineup with the U.S. Food and Drug Administration (FDA) approval of Sybravo in treating migraines. The drug could have significant commercial potential, with current migraine therapies not as effective as desired for many patients.

Other regulatory approvals could be on the way. Axsome plans to file for FDA approvals of AXS-05 (Auvelity) in treating Alzheimer's disease and AXS-12 in treating narcolepsy in 2025.

BioNTech hit the jackpot with its 2020 collaboration with Pfizer (NYSE:PFE) to develop a COVID-19 vaccine. Although sales for the vaccine the two partners market together, Comirnaty, have fallen since the end of the COVID-19 pandemic, BioNTech now has a huge cash stockpile as a result of the vaccine's success.

The German biotech innovator is still working on developing new vaccines for fighting viruses. It's evaluating a seasonal flu vaccine and a combination flu-COVID vaccine in late-stage testing. Both programs are collaborations with Pfizer.

However, BioNTech's big opportunity is in treating cancer. The company's pipeline includes two late-stage cancer therapies and 12 programs in phase 2 testing.

3. Exelixis

Exelixis has developed four drugs that are already on the market. Its biggest winner, by far, is Cabometyx, which is approved to treat renal cell carcinoma (RCC) and hepatocellular carcinoma (HC) -- the most common types of kidney cancer and liver cancer, respectively -- as well as thyroid cancer.

Exelixis and big drugmaker Bristol Myers Squibb (NYSE:BMY) won U.S. regulatory approval in early 2021 for the use of Cabometyx in combination with Bristol Myers' immunotherapy drug Opdivo. The company is also evaluating Cabometyx in combination with other drugs in late-stage studies targeting several types of cancer.

The next major growth driver for Exelixis could be zanzalintinib. The company expects to report results in the second half of 2025 from late-stage studies of the drug targeting colorectal cancer and non-clear cell renal cell carcinoma.

4. Regeneron

The biggest moneymaker for Regeneron is Eylea, an eye disease drug that the company makes in collaboration with Bayer (OTC:BAYR.Y). All net sales from Eylea in the U.S. are awarded to Regeneron, and the company splits the revenue earned from markets outside the U.S. with Bayer.

Regeneron also has a lucrative partnership with Sanofi (NASDAQ:SNY), another life sciences and pharmaceutical company. Together, the two companies are marketing and selling the autoimmune disease drugs Dupixent and Kevzara, the cancer drugs Libtayo and Zaltrap, and the cholesterol drug Praluent.

The company won initial approval from the FDA in February 2021 for Evkeeza in treating homozygous familial hypercholesterolemia (HoFH), a rare form of high cholesterol, in patients ages 12 and older. It also picked up an additional approval for the drug in March 2023 for treating HoFH patients between the ages of 5 and 11.

Regeneron could soon add another drug to its lineup. The company anticipates FDA approval of linvoseltamab in treating relapsed/refractory multiple myeloma by mid-2025. Its pipeline also includes 13 late-stage programs.

Summit Therapeutics doesn't have any products on the market yet. However, that status could change in the not-too-distant future.

The company plans to announce results from a late-stage study evaluating ivonescimab in combination with chemotherapy as a second-line treatment for non-small cell lung cancer (NSCLC) in mid-2025. If these results are successful, it should pave the way for Summit to file for FDA approval of its experimental immunotherapy.

Ivonescimab is already approved in China as a second-line treatment for NSCLC in combination with chemotherapy. It's marketed in the country by Summit's partner, Akeso (OTC:AKES.F). The drug also proved to be effective as a first-line treatment for NSCLC in a late-stage study conducted by Akeso. In this study, ivonescimab achieved significantly higher progression-free survival in first-line NSCLC patients than Merck's (NYSE:MRK) Keytruda.

Vertex, which makes multiple cystic fibrosis (CF) drugs, enjoys a monopoly over sales of treatments for the underlying cause of CF. Its dominance in CF was bolstered by the December 2024 approval of Alyftrek.

CF isn't Vertex's only market now, though. The big biotech company is ramping up commercialization efforts for Casgevy, a gene-editing therapy approved to treat two rare blood disorders -- sickle cell disease and transfusion-dependent beta-thalassemia.

Vertex also won FDA approval in January 2025 for Journavx (suzetrigine). This non-opioid drug is the first new type of pain medication approved in more than 20 years.

The company's pipeline looks especially promising. Vertex has four late-stage programs. It's evaluating suzetrigine in treating painful diabetic peripheral neuropathy. Inaxaplin is a potential treatment for APOL1-mediated kidney disease. Povetacicept targets another chronic kidney disease, IgA nephropathy. Vertex is also developing a potential cure for severe type 1 diabetes.

Some scientists believe that we’re in the golden age of biotechnology. Scientific advances are creating new and previously only imaginable ways to treat and prevent diseases.

The biotech sector is also presenting exciting opportunities for investors. Many of the best biotech companies have both strong drug candidate pipelines and winning drugs already on the market.

1. Axsome Therapeutics

2. BioNTech

5. Summit Therapeutics

6. Vertex Pharmaceuticals

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