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.