Top 19 most-performing biotech stocks of 2024

2024 has been described as a year of transition between a couple of rougher years and a potentially brighter period for biotech. While we can only speculate on what 2025 will be like in the industry, we can still take a look at the companies that have shown more than resilience in 2024. Indeed some biotech stocks recorded spectacular performance this year. 

Nicolas Schmitz, senior manager and strategic market analyst at KBI Biopharma, recently listed the top-performing biotech stocks of 2024. Building on his insight, we delve into the standout performers to uncover emerging trends and understand what they might reveal about the industry’s trajectory.

General overview of biotech stocks in 2024

In 2024, the biotechnology sector exhibited a notable resurgence, overcoming prior challenges and setting a foundation for future growth. Venture capital funding in biotech rose to $28.1 billion from $21.2 billion in 2023, indicating renewed investor confidence. 2024 was also a better year for initial public offerings (IPOs), and mergers and acquisitions (M&A) of private, venture-backed biotechs saw a significant uptick. However, the year was overall marked by resilience, in a trajectory to go back to more optimistic levels.

Amidst this cautious optimism in 2024, a select few biotechs managed to truly stand out in terms of stock performance.

A key driver of biotech stock performance in 2024 was regulatory approval, which serves as a key moment for companies in this space. For instance, the approval of Verona Pharma’s Ohtuvayre by the U.S. Food and Drug Administration (FDA) provided a much-needed non-steroid inhaled treatment option for patients with chronic obstructive pulmonary disease (COPD), resulting in a 134% surge in the company’s stock. Regulatory wins like this attract the attention of investors eager to capitalize on these milestones.

Breakthrough therapies also played an important role in shaping the biotech landscape this year. Companies developing solutions for unmet medical needs saw significant gains. A notable example is Guardant Health, whose stock rose following Medicare’s decision to reimburse its colon cancer blood test, Guardant Reveal. 

Clinical trial successes continued to drive investor enthusiasm, as positive data often translates into higher market valuations. Companies like Viking Therapeutics, which reported promising progress in its phase 2a trials for an obesity drug candidate, demonstrated how advancements in clinical pipelines can strengthen market confidence and spark stock appreciation.

While the overall sector showed resilience, certain companies stood out as outliers due to their remarkable stock performance. Bright Minds Biosciences led the pack with a staggering 1,857% year-over-year increase, driven by its progress in developing treatments for neuropsychiatric disorders. Monopar Therapeutics followed, with its stock surging by 1,089%, fueled by advancements in its cancer drug pipeline. Summit Therapeutics also impressed with a 576% increase, tied to the success of its therapeutic programs. 

Spotlight on the top 3 performing biotech stocks of 2024

In 2024, several biotech companies achieved remarkable stock performance – notably, Bright Minds Biosciences, Monopar Therapeutics, and Summit Therapeutics.

​​Bright Minds Biosciences: A pioneer in psychedelic therapies

Bright Minds Biosciences is focused on developing next-generation therapies inspired by psychedelics. The company aims to create treatments that retain the therapeutic benefits of psychedelics while minimizing their psychoactive side effects. Bright Minds specializes in precision-designed serotonergic drugs, targeting specific receptor subtypes in the central nervous system (CNS) to treat a range of neurological and psychiatric disorders.

At the core of Bright Minds’ technology is its work on the serotonin 5-HT receptor family, particularly the 5-HT2C receptor. The company’s lead candidate, BMB-101, is a 5-HT2C receptor superagonist designed to address disorders like refractory epilepsy and certain neuropsychiatric conditions. The selective targeting of this receptor subtype offers a promising therapeutic approach, as it avoids activating 5-HT2B receptors, which are linked to potential cardiac risks, and 5-HT2A receptors, responsible for hallucinogenic effects.

The biotech company’s stock performance skyrocketed by an astounding 1,857% in 2024, primarily driven by external market dynamics and its promising pipeline. A major turning point was Lundbeck’s $2.6 billion acquisition of Longboard Pharmaceuticals, which fueled investor enthusiasm. Longboard’s lead candidate, bexicaserin, is a 5-HT2C receptor superagonist, a class of drugs similar to Bright Minds’ BMB-101. This acquisition triggered a growing market interest in 5-HT2C-targeted therapies, elevating Bright Minds’ profile as a leader in this space.

Investors likely drew parallels between the two companies, as both focus on developing therapies targeting the 5-HT2C receptor. 

Additionally, after having reported positive phase 1 results for BMB-101 for epilepsy in mid-2023, Bright Minds initiated its phase 2 trials in 2024. The company also secured a $35 million private placement towards the end of 2024.

The growing acceptance of psychedelic-inspired therapies in mainstream medicine also contributed to the stock’s rise. Psychedelics, once stigmatized, are now gaining traction as a legitimate therapeutic modality, particularly in mental health and neurological disorders.

Monopar Therapeutics: Marching on several fronts

The company’s pipeline includes treatments designed to improve outcomes for patients undergoing cancer therapy and those affected by rare diseases. 

Monopar was best known for its lead candidate, Validive (clonidine mucobuccal tablet), a late-stage drug being developed to reduce severe oral mucositis in patients undergoing chemotherapy for head and neck cancers. However, the company announced the drug did not meet its efficacy threshold in phase 2b/3 in March 2023.

Monopar Therapeutics’ stock surged approximately 1,080% in 2024, driven by two major developments that reinforced investor confidence in the company’s strategy and pipeline.

A key milestone was Monopar’s exclusive worldwide license agreement with Alexion Pharmaceuticals to develop ALXN-1840, a late-stage candidate for Wilson disease, a rare genetic disorder that causes copper accumulation in the body, leading to severe organ damage. Wilson disease currently has limited treatment options, and ALXN-1840 represents a significant option to address it.

Alongside the ALXN-1840 licensing agreement, Monopar announced the dosing of the first patient in the phase 1 trial of MNPR-101-Lu, a novel radiopharmaceutical treating aggressive solid tumors. This is the candidate Monopar decided to prioritize after Validive’s mitigated results. 

This compound combines MNPR-101, a humanized monoclonal antibody that selectively binds to the urokinase plasminogen activator receptor (uPAR), with the radioactive isotope lutetium-177 (Lu-177). uPAR is a receptor overexpressed in various aggressive cancers, including pancreatic, ovarian, triple-negative breast, and colorectal cancers.

This is consistent with the radiopharmaceutical boom that is currently happening in the industry.

Summit Therapeutics: Advancing bispecific antibody therapies

Summit Therapeutics’ flagship program centers on ivonescimab, a bispecific antibody designed to enhance cancer treatment outcomes by combining two key mechanisms of action: immune checkpoint inhibition and anti-angiogenesis.

Ivonescimab is a bispecific antibody that simultaneously targets PD-1 (programmed death-1) and VEGF (vascular endothelial growth factor). PD-1 inhibitors, such as Merck’s Keytruda, have become a cornerstone of immunotherapy by blocking the PD-1 protein, which allows cancer cells to evade immune detection. VEGF, on the other hand, promotes tumor angiogenesis, or the formation of new blood vessels, which allows cancer to grow and spread. By targeting both PD-1 and VEGF, ivonescimab boosts immune system activity against tumors while simultaneously cutting off their blood supply.

This dual mechanism gives ivonescimab a significant advantage over traditional single-target therapies, as it addresses two critical processes involved in tumor survival and progression. This approach reflects a broader trend in oncology, where bispecific antibodies are gaining traction for their ability to enhance efficacy by tackling multiple pathways simultaneously.

The biotech’s stock saw a remarkable increase of 576% in 2024, primarily driven by the success of ivonescimab in clinical trials. The company presented data at the 2024 World Conference on Lung Cancer, showcasing ivonescimab’s superior efficacy in patients with advanced non-small cell lung cancer (NSCLC). Specifically, the trial demonstrated a statistically significant improvement in progression-free survival (PFS) compared to Merck’s Keytruda. Additionally, Summit successfully raised $235 million in a private placement in September 2024. 

Cancer, gene therapy, psychedelics, radiopharmaceuticals… Which will be the top performers of 2025?

Taking a look at the different technologies and disease areas the companies with the top-performing biotech stocks of 2024 are focused on, there isn’t a clear trend. Of course, as always cancer is well represented with, for instance, Summit Therapeutics or Cardiff Oncology.

Gene-related therapies have also driven significant growth among the top-performing stocks in biotech notably with RNA technologies such as Benitect’s RNA interference (RNAi) platform.

Psychedelics-inspired therapies rise as the unexpected leader with Bright Minds Biosciences leading the way by far in stock performance. Once an enthusiasts-only area of research, the progress biotechs have accomplished to isolate the psychedelics benefits from its hallucinogenic properties is finally tangible. The years to come might be determining for non-hallucinogenic psychedelic drugs. 

Another technology that we would see more rarely in the past, radiopharmaceuticals are gaining traction as their safety profiles improve. 

Similarly, the success of bispecific antibodies, exemplified by Summit Therapeutics’ ivonescimab, signals a shift in oncology toward therapies that combine multiple mechanisms of action. This innovation addresses the complexity of cancer biology, improving outcomes for patients and broadening the scope of immunotherapy applications. Overall, the key drivers for stock performance are of course the individual successes of companies – clinical trial successes,  regulatory approvals, or significant deals. This might be an indicator of which companies will be the top performers in 2025.