10 biotech companies to watch in 2025

Last year, we documented nine biotechs we thought you should watch in 2024. Now, as the year has flown by and the end of 2024 is approaching, biotech companies will be setting out their plans for next year as they continue to work towards bringing their innovative therapies through the clinic and to the market in an attempt to bring new and groundbreaking medicines to patients suffering from debilitating diseases. In this article, we take a look at 10 biotech companies we think are worth keeping an eye out for in 2025. 

Anavex Life Sciences

• Lead candidate: blarcamesine, for neurodegenerative diseases
• Anavex submitted marketing authorization application for blarcamesine for treatment of Alzheimer’s
• Blarcamesine received FDA orphan drug designation for Rett syndrome, fragile X syndrome, and infantile spasms

A neuroscience company, Anavex Life Sciences, is developing small-molecule treatments for CNS disorders like Alzheimer’s disease, Parkinson’s disease, and Rett syndrome. The company’s proprietary SIGMACEPTOR platform produces orally available therapeutic candidates that modulate the Sigma-1 receptor (SIGMAR1). The company says that targeting SIGMAR1 in this way has the capability of unlocking the body’s own defenses to potentially treat CNS conditions, ultimately restoring neural cell homeostasis and neuroplasticity.

Anavex’s lead candidate, ANAVEX 2-73 (blarcamesine), has shown promise in clinical trials, potentially offering scalable therapeutic solutions for several neurodegenerative diseases. On November 26, 2024, the company announced that it had submitted a marketing authorization application (MAA) for blarcamesine for the treatment of Alzheimer’s to the European Medicines Agency (EMA). This came after a phase 2b/3 trial showed that treatment with the drug resulted in significant slowed clinical decline on several notable endpoints, and also showed that it was safe, with no associated neuroimaging adverse events.

Blarcamesine has also successfully completed a phase 2 proof-of-concept study in Parkinson’s disease dementia, and a phase 2 and a phase 3 study in adult patients, and one phase 2/3 study in pediatric patients, with Rett syndrome, a rare genetic disorder that affects brain development, resulting in severe mental and physical disability. The drug has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for Rett syndrome, as well as for fragile X syndrome and infantile spasms. 

With Anavex awaiting the outcome of its MAA application, as well as managing multiple late-stage trials for its lead candidate, we can expect 2025 to potentially be a busy year for the company.

Candid Therapeutics

• Candid launched in September 2024 with $370 million
• Candid acquired Vignette Bio and TRC 2004 along with lead T cell engager assets 
• Candid hopes for clinical data in 2025 for assets in autoimmune diseases

Candid Therapeutics launched in September 2024 with one of the biggest private investments of the month after it managed to raise $370 million in capital. The company is developing T cell engager antibodies that can deplete specific B lymphocyte cell populations for the treatment of autoimmune diseases. It is aiming to become the first company to bring these novel therapies to the market.

In an unusual move, Candid’s launch also involved the acquisition of two biotech companies, Vignette Bio and TRC 2004, along with their lead assets, CND106, which targets B-cell maturation antigen, and CND261, which binds to CD20, respectively. Both candidates then also bind to CD3 on the surface of T cells. 

CND106 and CND261 have both completed phase 1 dose escalation studies with combined data from over 130 oncology patients. Candid is now focused on filing IND applications for them so that the company can study them in autoimmune diseases. Ken Song, chairman, president, and chief executive officer (CEO) of Candid, told Fierce Biotech that he hopes they will have clinical data on at least the drugs’ safety by 2025.

Enterprise Therapeutics

• Lead candidate: ETD001, for cystic fibrosis
• ETD001 in phase 2a trial; results expected in 2025
• ETD001 granted rare pediatric designation by FDA

Enterprise Therapeutics is a drug discovery company dedicated to the research and development of novel therapies for the treatment of respiratory diseases, including cystic fibrosis. It has a pipeline of novel low molecular weight compounds with first-in-class and best-in-class potential.

The company’s lead asset for the treatment of cystic fibrosis, ETD001, works by targeting the sodium channel (ENaC) in the airway epithelium to increase the hydration and clearance of mucus. The company commenced a phase 2a trial for the candidate in July. Shortly afterward, it was granted rare pediatric designation by the FDA, as cystic fibrosis is regarded as a serious or life-threatening disease. Enterprise is expected to share the results from this study in 2025. 

The company has also recently made several key leadership appointments to support the ongoing trial for ETD001, including appointing Renu Gupta as chief medical officer (CMO), Janet Hammond as a non-executive director, and Annabella Amatulli as head of Regulatory Affairs. Furthermore, in January 2024, the cystic fibrosis company closed a £26 million ($33.1 million) series B follow-on financing round, which it said would fund the phase 2a clinical trial for ETD001. 

Jazz Pharmaceuticals

• Received FDA approval for Ziihera in November; first treatment of its kind for biliary tract cancer
• Positive phase 3 topline results for Zepzelca in combination with PD-L1 inhibitor atezolizumab
• In 2025, Jazz will focus on launch and commercialization of Ziihera

With several drugs already approved in different countries for oncology and neuroscience indications, Jazz Pharmaceuticals is continuing to evolve its broad pipeline and commercial operations, focusing on bringing new, first-of-their-kind treatments to small, often rare, patient populations.

The company has recently grown its oncology portfolio even further after receiving FDA approval in late November for zanidatamab, sold under the brand name Ziihera, for the treatment of adults with previously treated, unresectable, or metastatic HER2-positive biliary tract cancer. This approval makes Jazz’s drug the first dual HER2-targeted bispecific antibody specifically indicated for patients with HER2+ metastatic biliary tract cancer, which is a rare and devastating cancer with a poor prognosis for patients.

In another recent update, Jazz announced positive top-line results from a phase 3 clinical trial evaluating Zepzelca (lurbinectedin) in combination with the PD-L1 inhibitor atezolizumab (Tecentriq) compared to atezolizumab alone when administered as a maintenance treatment for adults with extensive-stage small cell lung cancer (ES-SCLC). The combination demonstrated a statistically significant improvement in the primary endpoints of overall survival and progression-free survival compared to treatment with atezolizumab alone.

In the coming year, Jazz will be focused on the launch and commercialization of Ziihera. 

Life Biosciences

• Two core platforms: partial epigenetic reprogramming platform and chaperone-mediated autophagy (CMA) platform
• Lead program: ER-100; from partial epigenetic reprogramming platform
• 2025 plans: Life Bio aims to initiate first clinical studies evaluating ER-100 for optic neuropathies

Life Biosciences is advancing an innovative cellular rejuvenation platform to reverse diseases of aging and injury and ultimately restore health for patients. The company has two core platforms: the partial epigenetic reprogramming platform and the chaperone-mediated autophagy (CMA) platform. 

The epigenetic reprogramming platform reprograms the epigenome of adult cells to resemble the epigenome of younger cells via the expression of three Yamanaka factors, which are a group of protein transcription factors that play a vital role in the creation of induced pluripotent stem cells, and control how DNA is copied for translation into other proteins. This “partial programming” approach retains the original cell identity to safely restore youthful cellular function without tumor formation. 

Meanwhile, the company’s other platform is based on the process of CMA, by which unwanted proteins are degraded in cells, with CMA activity declining during aging due to a reduced expression of a protein called LAMP2A, leading to the accumulation of insoluble protein aggregates that disrupt cellular function. With its CMA platform, the company has identified novel small-molecule oral compounds that increase the expression of multiple proteins in the CMA pathway. According to the company, these CMA activator compounds have been shown to have efficacy in preclinical models of frontotemporal dementia, Alzheimer’s disease, and retinal degeneration.

The company’s lead program is a gene therapy called ER-100, which uses Life Bio’s epigenetic reprogramming platform. In the second half of 2025, the company aims to initiate the first human clinical studies evaluating ER-100 for optic neuropathies, including NAION and glaucoma, making it the first company to bring cellular rejuvenation with partial epigenetic reprogramming to the clinic to treat optic neuropathies. 

Ovid Therapeutics

• Lead candidate: OV888; a ROCK2 inhibitor for cerebral cavernous malformation
• Second clinical candidate: OV329; a GABA-AT inhibitor for chronic epilepsy, acute seizures, and status epilepticus
• KCC2 portfolio: OV350 expected to begin phase 1 study in 2025

Focused on neurological diseases, Ovid Therapeutics is developing the next generation of targeted small-molecule therapies that seek to reduce seizures and deliver improved safety and tolerability. 

Ovid currently has a pipeline of small-molecule therapies with novel mechanisms of action. Its lead asset, OV888, is a ROCK2 inhibitor (the ROCK2 pathway is thought to be hyperactivated in multiple neurological diseases) for the treatment of a rare seizure-related disorder called cerebral cavernous malformation (CCM), while its other clinical candidate, OV329, is a GABA-AT inhibitor (low levels of GABA in the brain have been linked to neuronal hyperexcitability, which can lead to seizures) with potential as an oral treatment for chronic epilepsy and as an intravenous formulation for acute seizures and status epilepticus. 

The company is also doing work on a novel drug target, called K+Cl– cotransporter 2 (KCC2), which it believes could revolutionize therapeutics for a broad range of CNS conditions. The company exclusively in-licensed its KCC2 portfolio from AstraZeneca in 2022. Its lead candidate in this area is called OV350 and works by directly activating KCC2, which has the potential to restore neuronal inhibitory and excitatory balance to bring a hyperexcited neuron back into homeostasis – a state of balance among all the body systems needed for the body to survive and function correctly.

In 2022, Ovid began optimizing OV350 for multiple possible formulations. Its desire is to achieve both intravenous and oral administration formulations for the drug, as dual formulations are optimal for patients who are treated acutely in the hospital and need to maintain seizure reduction in an out-patient setting. The company intends to initiate the phase 1 study in 2025. 

Skye Bioscience

• Lead candidate: nimacimab; a peripherally restricted CB1 inhibitor for obesity
• Nimacimab currently being tested in phase 2 trial 
• Final data from phase 2 trial expected in Q4 2025

Skye Bioscience is developing next-generation molecules to treat obesity and metabolic diseases. Going against the grain, the company believes that, despite their success, currently approved GLP-1 agonist therapies still have a number of flaws that suggest they do not modify the underlying metabolic disorders associated with obesity and, therefore, may not be conducive to achieving long-term health improvements. This is why the company is working on a different mechanism of action for its weight loss drug that it believes will offer patients the opportunity to achieve healthier weight loss.

Its lead candidate, nimacimab, is a peripherally restricted CB1 inhibitor. CB1 is expressed throughout the body, especially in tissues important to metabolism and metabolic disorders such as adipose (fat), muscle, stomach, liver, and kidneys. According to Skye, preclinical studies have demonstrated that blocking CB1 receptors in these tissues can result in significant weight loss through multiple mechanisms. For example, CB1 inhibition in fat tissue promotes the expenditure of energy stored in fat, and therefore, the breakdown of fat, and inhibition of CB1 in the gut also appears to modulate appetite-regulating hormones that act on the brain and support reduced caloric intake. These mechanisms may result in weight loss while preserving muscle and may allow patients to better maintain weight loss for longer periods of time. 

In August 2024, Skye launched a phase 2 trial of nimacimab in patients with obesity. The study is assessing parameters increasingly viewed as important to the long-term quality and/or sustainability of weight loss, including gastrointestinal tolerability (GI) and lean mass retention. 

In Q2 2025, the company expects to announce interim data from this phase 2 trial, before reporting final data in Q4 2025. 

TG Therapeutics

• FDA-approved drug: Briumvi; an anti-CD20 monoclonal antibody for relapsing multiple sclerosis
• At time of launch, Briumvi was the only drug of its kind for relapsing multiple sclerosis with one-hour infusion time following starting dose
• In November 2025, TG Therapeutics was ranked number one fastest-growing company

Multiple sclerosis company TG Therapeutics is focused on the acquisition, development, and commercialization of novel treatments for B-cell diseases. In December 2022, the company announced that the FDA had approved its drug BRIUMVI (ublituximab) for the treatment of patients with relapsing forms of multiple sclerosis, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease. 

Briumvi works by targeting an epitope on CD20-expressing B-cells. When the drug binds to the B-cell, it triggers a series of immunological reactions, including antibody-dependent cellular cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC), leading to the destruction of the cell. The drug is also designed to lack certain sugar molecules normally expressed on the antibody. This is because the removal of these sugar molecules, a process called glycoengineering, has been shown to enhance the potency of the drug, particularly when it comes to ADCC activity.

As the company launched Briumvi with advantages in administration time and pricing that resonate with doctors and patients in this market, it has demonstrated strong early sales and continues to gain market share through practical advantages over existing treatments. When it was launched, Briumvi became the first and only anti-CD20 monoclonal antibody approved for patients with relapsing multiple sclerosis that can be administered in a one-hour infusion following the starting dose. 

Furthermore, in September 2024, TG Therapeutics reported that after five years of treatment with Briumvi, 92% of patients with relapsing multiple sclerosis were free from disability progression. 

To further accentuate why TG Therapeutics is a company to watch out for next year, in November 2024, it was announced that the biotech ranked number one on the Deloitte Technology Fast 500, a ranking of the 500 fastest-growing technology, media, telecommunications, life sciences, fintech, and energy tech companies in North America. This is due to the company’s growth between fiscal years 2020 and 2023, fueled by BRIUMVI revenues.

Wave Life Sciences

• Lead candidate: WVE-006; a synthetic RNA molecule for alpha-1 antitrypsin deficiency 
• WVE-006 achieved positive results in phase 1b/2a study, marking first-ever clinical demonstration of RNA editing in humans
• Wave Life Sciences formed partnership with GSK

RNA editing company Wave Life Sciences is committed to developing best-in-class therapies across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization, and production of stereopure oligonucleotides.

In September 2023, Wave Life Sciences submitted a clinical trial application for its lead RNA editing candidate, WVE-006, with dosing for that trial beginning in the U.K. last year. This was a milestone achievement, as it marked the first time an RNA editing candidate had managed to reach the clinic. Then, crossing yet another milestone, in October 2024, Wave revealed positive proof-of-mechanism data from this phase 1b/2a study in patients with alpha-1 antitrypsin deficiency (AATD), marking the first-ever clinical demonstration of RNA editing in humans.

WVE-006 is a synthetic RNA molecule that aims to correct the single base mutation in messenger RNA (mRNA) coded by the SERPINA1 Z allele, enabling the restoration and circulation of functional M-AAT protein in order to prevent liver damage and protect the lungs. 

The initiation of the trial for WVE-006 saw Wave achieve its first milestone in its collaboration with GSK, which resulted in a $20 million payment to the RNA editing company. As part of the collaboration, Wave is eligible to receive up to $505 million in additional development, launch, and sales-related milestone payments, as well as tiered royalties on net sales.

Quotient Therapeutics

• Launched in 2023 with $50 million
• Somatic genomics platform is a world-first; designed to find links between genes and diseases for drug discovery
• Partnered with Pfizer earlier this year to identify new drug targets to address heart and kidney diseases

Born out of the venture capital company Flagship Pioneering, Quotient Therapeutics made its $50 million launch in late 2023. The newly formed genomics sequencing company, which has headquarters in both the U.S. and the U.K., has come up with a world-first genetic library platform that will aid in drug discovery.

Quotient’s somatic genomics platform is designed to find the links between genes and diseases in order to develop medicines that can imitate or inhibit drug targets – the proteins that code for the disease-related genes. The company says that the platform can identify a broad scope of genes undetected by traditional population genetics approaches using fewer patients, enabling unprecedented insight and the ability to create truly transformative therapies.

Earlier this year, the biotech formed an alliance with pharma giant Pfizer to identify new drug targets to address heart and kidney diseases. The partnership will see the two collaborators analyze somatic mutations present in the tissues of people with diseases to treat them.