Cheryl Barton is director of PharmaVision, 

Cheryl Barton


Proteasomes are molecular machines for breaking down proteins called proteolysis, only target proteins that have been marked for destruction called ubiquitin to the target protein, 3d rendering | Image Credit: ©

Momentum is building for proteolysis-targeting chimeras (PROTAC) treatments that hijack the ubiquitin-proteasome system (UPS) to catalytically degrade pathogenic proteins and eliminate disease-causing proteins (1–2). This approach enables scientists to target previously "undruggable" proteins and exert sustained pharmacological effects, including those responsible for difficult-to-treat childhood cancers and drug-resistant triple-negative breast cancer (1, 3). In recent years, significant progress has been made in the selective delivery of PROTACs, potentially extending their use to a broader range of conditions (4).

How is the pharma industry investing in proteasome-targeting therapies?

A number of proteasome-targeting therapies have been approved in Europe and the United States, such as Amgen’s Kyprolis (carfilzomib), Bristol Myers Squibb’s Ninlaro (ixazomib), and Johnson & Johnson/Takeda’s Velcade (bortezomib). Several Big Pharma companies—AbbVie, AstraZeneca, Eli Lilly, Merck & Co, Novartis, Pfizer, Roche, and Sanofi—have products in clinical development. Interest in this field has catalyzed some interesting acquisitions and partnerships. For instance, in 2025, Johnson & Johnson completed its acquisition of Halda Therapeutics to gain access to its novel Regulated Induced Proximity Targeting Chimaeras (RIPTACs), a protein degradation approach that connects with proteasome pathways (5). In September 2025, Novartis initiated a collaboration with US-based Monte Rose to develop novel degraders or molecular glues to treat immune-mediated diseases (6).

Several biotech companies in the US are developing second-generation PROTACs, including Arvinas' vepdegestrant (ARV-471). In March 2025, Pfizer and Arvinas announced positive topline results from the Phase III VERITAC-2 clinical trial with vepdegestrant in metastatic breast cancer (7). Additionally, C4 Therapeutics and Nurix Therapeutics have protein degrader programs: cemisoldomide in Phase I/II for multiple myeloma/non-Hodgkin lymphoma (NHL), and bexobrutideg in Phase II/III for relapsed/refractory chronic lymphocytic leukaemia (r/r CLL), respectively (8–10).

More recently, US-based Kymera Therapeutics received fast-track designation from FDA for KT-621, an oral signal transducer and activator of transcription 6 (STAT6) degrader for the treatment of atopic dermatitis (11). In 2025, Sanofi modified its collaboration with Kymera, discontinuing development of KT-474, an Interleukin-1 receptor-associated kinase 4 (IRAK4) degrader, while continuing development of KT-485, which is in Phase II trials for atopic dermatitis and hidradenitis suppurativa (12). Other US biotech companies working in this field include Accutar Biotech, Cleave Therapeutics, Dialectic Therapeutics, and Karyopharm Therapeutics.

What are some European players to watch out for in the proteasome space?

Several European biotechs are evaluating a new class of inhibitors that harness the UPS, hijacking the proteasome to provide disease-specific protein degradation with a wide range of therapeutic applications from autoimmune to oncology.

has developed its proprietary next-generation targeted protein degradation (TPD) technology to develop Targeted Glues. Its lead program, AMX-883, can induce BRD9 degradation to block patient-derived tumor growth 

 as a monotherapy, with superiority to venetoclax in acute myeloid leukaemia (AML) (13).

focuses on developing small-molecule proteasome activators for neurodegenerative disease using its DGRADX platform. In October 2025, the company completed a $15 million financing round led by life sciences investors Apollo Health Ventures and Novo Holding (14). Funds will be used to advance its multi-disease pipeline.

focuses on the development of precision medicine using site-restricted targeted therapeutics. OncoOne’s research encompasses the development of targeted Immunotherapeutics that modulate tumor degradation using its Pre-Targ-it platform. The company is exploring therapies that target the proteasome-Bcl-2 axis as novel anticancer agents (15).

QLi5 Therapeutics GmbH (German-Korean joint venture) 

is working on a new class of proteasome inhibitors that demonstrate strong anti-tumor and anti-inflammatory activity that offer promise in cancer, autoimmune, and inflammatory disorders (16). In September 2025, Qli5 closed a €10 million (US$12 million) Series A financing round with an international consortium of investors, including SV Investment (Korea), KHAN Technology Transfer Fund I (Germany), Atinum Investment (Korea), and DAOL Investment (formerly KTB, Korea). The proceeds will advance its pipeline of proteasome inhibitors in clinical development (17).

Future opportunities in targeting the proteasome

According to market research, the global proteasome inhibitor market was valued at approximately US$2.7 billion in 2024 and is projected to reach US$3.03 billion in 2024, growing to approximately US$6.1 billion by 2034, with a compound annual growth rate of 8.70% from 2025 to 2034 (18). Preliminary research indicated that proteasomes may have far-reaching effects and could be used to target new immune defence mechanisms (19). Currently, companies are focusing on developing proteasome inhibitors for oncology; however, these agents may also present new opportunities to treat patients with inflammatory and autoimmune diseases. Many large pharmaceutical companies have already forged strategic partnerships with research institutions and biotech companies to advance PROATCs and UPS, either as standalone therapies or in combination with existing therapies. Several late-stage clinical trials will read out over the next 12–24 months, and promising results could stimulate further investment in this area.

Fu, Z.; Pan, M.; Yang, C.; et al. Rational Modification of PROTACs for Tumor-Selective Protein Degradation. 

. Jan. 9, 2026. DOI:10.1016/j.addr.2026.115775

How Protein-Slayer Drugs Could Beat Some of the Cruellest Cancers

Lee, C.H.; Minh Nguyen, T.; Lee, Y.; et al. Overcoming Therapeutic Resistance in Triple-Negative Breast Cancer: Targeting the Undrugged Kinome. 

Yang, X.; Wang, D.; Li, C.; et al. Nano-PROTACs for Precision Medicine: Engineering Strategies for Enhanced Targeting and Potency. 

Johnson & Johnson Completes acquisition of Halda Therapeutics and Its Novel Platform to Revolutionize Cancer Treatment and Enable Next-Generation Oral Therapies. 

Monte Rosa Therapeutics Announces Collaboration with Novartis for Degraders to Treat Immune-mediated Diseases

Arvinas Presents Preclinical Data Supporting Mechanistic Synergies and Enhanced Antitumor Activity with the Combination of ARV-393 and Glofitamab at the 2025 American Society of Hematology Annual Meeting and Exposition.

. https://c4therapeutics.com/our-pipeline/cemsidomide/ (accessed Jan 13, 2026).

Nurix Therapeutics Outlines 2026 Goals and Objectives for Advancing Bexobrutideg and Its Pipeline of Novel Degrader-Based Medicines in Cancer and Autoimmune Diseases.

Arvinas and Pfizer Announce Positive Topline Results from Phase 3 VERITAC-2 Clinical Trial

Kymera Therapeutics Announces U.S. FDA Fast Track Designation for KT-621, a First-in-Class, Oral STAT6 Degrader for the Treatment of Atopic Dermatitis

Kymera Therapeutics Announces Sanofi IRAK4 Collaboration

Amphista Therapeutics Presents New Preclinical Data Showing the Potential of its BRD9 Targeted Glue™, AMX-883, to Transform the Treatment Paradigm for Acute Myeloid Leukaemia - Amphista Therapeutics. 

Booster Therapeutics Launches to Pioneer New Class of Proteasome Activator Medicines for the Treatment of a Range of Complex Diseases.

. https://www.oncoone.com/ (accessed Jan. 13, 2026).

Next Generation Proteasome Inhibitors for Unmet Medical Needs Deployed by an International Research & Development Network

QLi5 Therapeutics Attracts EUR 10 Million in Series A Financing.

Proteasome Inhibitors Market Size & Share Report, 2025 – 2034

This Scientist Found a New Trick of the Immune System by Digging through Cellular Rubbish

Cheryl Barton, PhD, is founder and director of PharmaVision, Pharmavision.co.uk.

Articles

Proteolysis-targeting chimeras (PROTACs) are gaining momentum as they leverage the ubiquitin-proteasome system to degrade pathogenic proteins, offering potential treatments for previously "undruggable" targets, including challenging cancers. The pharmaceutical industry is heavily investing in proteasome-targeting therapies, with several companies developing and testing new drugs. European biotech firms are also advancing in this field, exploring novel inhibitors and therapeutic applications. The global proteasome inhibitor market is projected to grow significantly, with potential applications extending beyond oncology to autoimmune and inflammatory diseases.

Proteasome-targeting therapies may eliminate disease-causing proteins in a broad range of diseases.

Proteasomes: A Novel Approach to Target the Immune System

Fluorescent Imaging immunofluorescence of cancer cells growing in 2D with nuclei in blue, cytoplasm in red and DNA damage foci in green | Image Credit: ©drimafilm - stock.adobe.com

Live-cell imaging techniques offer dynamic, spatio-temporal insights into cellular processes and provide a comprehensive, 

cost-effective way to objectively examine drug effects across different cellular models 

(1). When used with fluorescence reporters or label-free methods alongside artificial intelligence (AI)/machine learning (ML) techniques, it can deliver detailed phenotypic screening, enabling thorough compound profiling, understanding mechanisms of action (MoA), enhancing translational predictivity, and speeding up lead optimization (

Figure 1: Key advantages of utilising live cell imaging in driving drug discovery. Source: modified from references 2,3. Figure courtesy of the author.

Which European frontrunners are leading the field in live-cell imaging technologies?

Several European biotechs, including Cellbox Labs, Evotec SE, Nanolive SA, and Salipro Biotech AB, have developed innovative high-throughput screening methods to improve 

 translation (IVIVE) and accelerate the discovery of new chemical entities (NCEs) for clinical evaluation.

was founded in 2020 by Artūrs Ābols, Gatis Mozoļevskis, and Roberts Rimša. The company focuses on developing modular “organ-on-chip” (OOC) systems to improve drug discovery, in which endothelial and epithelial layers are stacked in vertical channels separated by permeable membranes, enabling organ modelling in a controlled environment. The company has developed various OOCs, including the blood-brain barrier, kidney, gut, lung, and pancreas, to accurately predict individual responses to drugs, food, and probiotics (4). The OOCs can be mass-produced to enhance reproducibility and cost-efficiency, and can be scaled up from 8 to 24 chips, if needed.

In 2024, Cellbox raised a pre-seed round of €935,000 led by Latvian Buildit VC, with participation from LatBAN angels, ASP Asset Management AIFP, and private investor Ansis Spridzans (5). In August 2025, Cellbox attracted a further €3.3 million in funding from Tech4Cure, a state-funded Important Project of Common European Interest (IPCEI). These funds will be used to expand Cellbox’s OOC high-throughput technology and to integrate biological sensors for improved IVIVE (6). Earlier this year, Cellbox Labs began a collaboration with the Latvian Biomedical Research and Study Centre, KTH Royal Institute of Technology, Teadusmosaiik OÜ, and Fewer Moving Parts to develop a first-in-class vascularised Gut-Brain-Axis (GBA) on-chip platform, a multi-OOC model that, if successful, could have significant applications in real-world drug evaluation (7).

In August 2025, Cellbox forged a collaboration with Altis Biosystems to scale an automated, gut-on-chip model using iPSCs and patient-derived microbiota, and it plans to integrate oxygen and pH sensors directly into the OOC to provide more detailed analysis for AI-driven drug discovery (7). Cellbox is also leveraging its pancreatic islet OOC to assess and benchmark biosimilars and glucagon-like protein 1 (GLP-1) generics under dynamic flow conditions, and compare the data generated with traditional 

 animal models to help improve biosimilar testing. In addition, Cellbox Labs is working with ESQlabs and MPSlabs to develop digital twin models to enhance the predictive power of the OOC platforms for human outcomes. Overall, these next-generation OCCs could improve the translational prediction of compound effects compared to classic monolayer cultures and help de-risk and accelerate preclinical drug development (8).

using induced pluripotent stem cell (iPSC) screening

 to model human diseases and enable patient-specific drug testing. Initially, the company focused on 2D cultures to improve translational prediction and is now investigating more physiologically relevant models, including multilineage cultures, organoids, and co-cultures, to improve fidelity. The proprietary platforms integrate live-cell imaging with iPSC-derived cell types for specific conditions, including neurodegenerative and metabolic diseases (9). Evotec has leveraged its iPSC phenotypic screening and imaging platform to establish strategic collaborations with biopharma. In January 2022, Boehringer Ingelheim (BI) entered a partnership with Evotec to combine its PanOmics Platform with iPSC-based disease modelling and phenotypic screening to identify novel candidates for vision-related diseases (10). In the second quarter of 2025, Evotec reported key advancements in its neuroscience collaboration with Bristol Myers Squibb, resulting in a US$20 million research payment to the company. To date, the company has received US$75 million in programme-based payments, and two partnered assets have entered Phase II clinical trials (11).

Evotec has also established collaborations with Esperon and identified a preclinical candidate for the treatment of Primary Sclerosing Cholangitis. Another partner, Dewpoint Therapeutics, submitted an investigational new drug (IND) application for a first-in-class condensate modulator, DPTX3186, for Wnt-driven cancers, earlier this year (12). In addition, Evotec Ventures funds translational projects with academia through its Biomedical Research, Innovation & Development Generation Efficiency (BRIDGE) model. Since its establishment, it has created eight BRIDGE partnerships and made three investments in start-ups (e.g., Autobalm Labs, ArgioBio, and Extend) (13). In November 2025, the company reported strong nine-month financial results. It announced a US$650 million deal for Sandoz to acquire its Just-Evotec Biologics (JEB) EU in Toulouse and license its continuous manufacturing platform technology. The company is cash-rich and is on target to deliver its strategic objectives, enhance its live cell offerings, and build strategic partnerships with biopharma to secure future growth (11).

provides label-free live-cell holotomographic imaging platforms that measure the quantitative refractive index of cells, enabling continuous imaging of living cells over long periods without fluorescent reporters. Its proprietary LIVE Cytotoxicity Assay and LIVE T Cell Assay can be combined with AI-powered analysis to determine interaction dynamics, viability, and organelle changes (14). This technology has the potential to accelerate the discovery of chimeric antigen receptor T (CAR-T), T-cell receptor T (TCR-T), bispecific antibodies, and other immunomodulators. For example, Pagliuca 

 deployed Nanolive’s technology to monitor real-time interactions between Vγ9Vδ2 T cells and tumour cells following treatment with BTN2A1-agonist antibody, which has potential in haematological and solid tumours (15). Nanolive continues to expand its offerings and, earlier in 2025, launched its Smart Mitochondrial AssayLIVE for real-time mitochondrial analysis, enabling researchers to evaluate metabolic function, cellular stress, and drug-induced toxicity. This assay could be a helpful addition for researchers evaluating NCEs targeting mitochondrial diseases and oncology (16).

develops nanomembrane-protein stabilisation technology, Salipro DirectMX, to improve the development of therapeutic antibodies and small molecules targeting membrane proteins, such as G-protein-coupled receptors (GPCRs), ion channels, and transporters (17). This technology can be used in downstream applications such as B-cell sorting, immunisation, mass spectrometry, phage display, radioligand binding, and other processes to facilitate drug discovery. In May 2023, 

screened and identified several small-molecule binders using the Salipro and DEL (DNA encoded library) platform, successfully discovering binders against a challenging membrane protein target. This partnership provided proof-of-concept data demonstrating that Salipro’s platform enhances the discovery of new drugs targeting previously undruggable proteins (18). Salipro published a paper with Bio-Rad Laboratories, showing that Salipro platform technology, combined with Bio-Rad’s Pioneer Platform, can generate a synthetic human antibody phage display library to identify several high-affinity antibodies targeting CXC chemokine receptor type 4 (CXCR4), which play a central role in immune-oncology (19). Salipro has formed strategic partnerships with AstraZeneca, Bayer, Boehringer Ingelheim, GlaxoSmithKline, and Merck, Sharp and Dohme (MSD) to unlock drug targets for various drug development programmes. BI signed a deal to utilize Salipro’s platform technology to accelerate target discovery in therapeutic areas such as mental health and cardio-renal-metabolic diseases (20). Salipro technology complements imaging and phenotypic tools and can support drug discovery, particularly for membrane-bound protein targets that have proven challenging to study using traditional methods.

Future opportunities in live-cell imaging in drug discovery

According to market research, the global live cell imaging market is projected to reach US$3.03 billion in 2024 and grow to approximately US$6.73 billion by 2034, with a compound annual growth rate (CAGR) of 8.30% from 2024 to 2034 (21). Live-cell imaging increasingly enables phenotypically and mechanistically informed, translationally relevant screening, aiding in drug discovery and personalised medicine. Additionally, regulators aim to phase out animal testing and promote the use of new approach methodologies, including OOCs and live-cell imaging, to evaluate the safety and efficacy of new medicines during non-clinical development (22). The integration of advanced cell models, sophisticated imaging, and AI/ML analytics is creating a new generation of discovery platforms that facilitate quicker identification, validation, and progression of NCEs into clinical trials. Cellbox Labs, Evotec, Nanolive, and Salipro Biotech are leading in Europe, offering diverse approaches to accelerate drug discovery and reduce biopharma’s investment in 

 studies. Over the coming years, platforms capable of combining scale, physiological relevance, mechanistic insight, and actionable decision-making will gain momentum in the market, leading to improved target/compound selection and ultimately a higher hit-to-clinical success ratio, thereby reducing late-stage drug attrition and supporting faster go/no-go decisions.

Mimori-Kiyosue, Y.; Koizumi, T.; Washio, T. Potential of High-Spatiotemporal Resolution Live Cell Imaging for Drug Discovery and Development. In: Satoh, H., Funatsu, K., Yamamoto, H. (eds) 

Drug Development Supported by Informatics

Carreras-Puigvert, J.; Spjuth, O. Artificial Intelligence for High Content Imaging in Drug Discovery. 

Chen, B.; Yin, Z.; Ng, B.W.; et al. Label-Free Live Cell Recognition and Tracking for Biological Discoveries and Translational Applications. 

Riga-based Developer of Industrial Organ-on-Chip Technology Cellbox Labs Raised €935K

Latvian BioTech Startup Cellbox Labs Lands €3.3 million for Organ-on-Chip Innovation without Animal Testing

Cellbox Labs Kicks Off Project Developing a First-in-Class Vascularized Gut-Brain-Axis-on-Chip Platform for Accelerating Research and Drug Discovery (GBA-Connect)

Zhou, L.; Huang, J.; Li, C.; et al. Organoids and Organs-on-Chips: Recent Advances, Applications in Drug Development, and Regulatory Challenges. 

Evotec Enters Ipsc-Based Drug Discovery Partnership With Boehringer Ingelheim In Ophthalmology

Evotec SE Reports 9M 2025 Results: Continued Strong Execution On Strategic Priorities

Evotec-Partner Dewpoint Therapeutics Announces An Open IND For First-In-Class Condensate Modulator DPTX3186 For Wnt-Driven Cancers

New Gen LIVE T Cell Assay » Nanolive - A Complete Solution For Your Label-Free Live Cell Imaging

Le Floch, A.C.; Imbert, C.; Boucherit, N.; et al. Targeting BTN2A1 Enhances Vγ9Vδ2 T-Cell Effector Functions and Triggers Tumor Cell Pyroptosis. 

Automatically Quantify the Dynamics of Live Mitochondrial Assays

Salipro Biotech and DyNAbind Announce Milestone Achievement in Collaboration to Enable DEL for Membrane Proteins.

Pioneer: a synthetic human antibody phage display library for rapid therapeutic lead generation

Salipro Biotech and Boehringer Ingelheim Enter Research and License Agreement to Accelerate Multiple Boehringer Ingelheim Pipeline Programs

Live Cell Imaging Market Size, Share, and Trends 2024 to 2034

Regulatory acceptance of new approach methodologies: how to interact with EMA

Live-cell imaging techniques, combined with AI/ML, are revolutionizing drug discovery by providing detailed phenotypic screening and enhancing translational predictivity. European companies like Cellbox Labs, Evotec SE, Nanolive SA, and Salipro Biotech AB are at the forefront, developing innovative technologies to improve in vitro–in vivo translation and accelerate new chemical entity discovery. These advancements are crucial as the global live-cell imaging market is projected to grow significantly, driven by the need for more efficient drug discovery methods and the phasing out of animal testing in favor of new approach methodologies.

Live cell phenotypic screening can enhance translational predictability and speed up lead optimization.

Live Cell High-Content Imaging Is Driving Drug Discovery

Landkarte *** Europa | Image credit: ©beugdesign - Stock.adobe.com

In September 2025, the European Commission (EC), together with the Heads of Medicines Agencies (HMA) and the European Medicines Agency (EMA), revealed two new goals for clinical trials to ensure Europe maintains its leadership in clinical research (1). By 2030, the European Union aims to:

Increase the number of multinational clinical trials by 100 annually; currently, an average of 900 are authorized each year.

Increase the speed of patient recruitment in clinical trials; currently, about 50% of trials recruit patients within 200 calendar days or less from the application submission date. The goal is to raise this to two-thirds (66%).

In early 2025, the United Kingdom (UK) government unveiled its plans to speed up clinical trials, unlock National Health System (NHS) data for research, and improve patient access to new treatments earlier (2). The global clinical trial arena is becoming increasingly competitive, serving as a significant revenue generator. According to market research, the global clinical trials market accounted for US$59 billion (€44 billion) in 2024 and is anticipated to grow from US$62.4 billion (€54.35 billion) in 2025 to US$98.9 billion (€86.15 billion) in 2034, growing at a compound annual growth rate of 5.2% (3).

An analysis of clinical trials between 2005 and March 2025 highlights that the United States hosts the majority of clinical trials, followed by China, France, Canada, Turkey, and the UK (

). The majority of clinical trials were conducted in a limited geographical distribution. Specifically, 26% of clinical trials were conducted in multiple locations, and only 8.2% of the total clinical trials were multinational clinical trials (4). However, market research indicates that the main barriers in clinical trials are due to poor trial design, lack of inclusivity, and inadequate clinical trial infrastructure (5). Therefore, if the EU wants to be a serious contender, it needs to strengthen its clinical trial ecosystem, promote ethical research, encourage stakeholder collaboration, and implement strategic investments.

Figure 1: Distribution of clinical trials by continents and disease, 2005-2025. (Figure courtesy of the author and modified from reference 4).

How is the EU driving innovative clinical research?

These new goals are part of the EU's ongoing effort to create a more supportive environment for clinical research following the launch of the Accelerating Clinical Trials in the EU (ACT EU) initiative in January 2022, a collaboration between EC, HMA, and EMA (6). It also builds on the EU Life Science to enhance public health, create jobs, and foster innovation by 2030, positioning the EU as a global leader (7).

The EU pharmaceutical legislation continues to evolve, and the EC has started a comprehensive review to create an agile, competitive, and equitable regulatory framework. The ACT EU is one of the first steps to strengthen clinical research in Europe (8). The ACT EU initiative has launched a trial map to help patients find nearby clinical trials. Additionally, it provides guidance to clinical trial sponsors on designing effective trials and submitting successful applications for authorisation, including marketing authorization. In addition, they can provide support for the implementation of the revised International Council for Harmonisation (ICH) E6 R3, 

 guideline and assistance for non-commercial sponsors to conduct more multinational trials.

In addition, the EU has several complementary initiatives underway to help the EU meet these targets, including:

The Clinical Trials Regulation (CTR) Collaborate initiative aims to foster interaction between national authorities and ethics committees to promote harmonized procedures and reduce administrative burden (9).

The COMBINE initiative aims to understand challenges and obstacles to align the interface between the three frameworks: CTR, In Vitro Diagnostics Regulation (IVDR), and Medical Device Regulation (MDR) (10). In June 2025, the first pilot was launched to coordinate clinical and performance study assessments into a single streamlined process (11).

The MedEthicsEU is a forum of national representatives from medical research ethics committees, established by the EC, to support discussion and mutual learning between EU/EEA Member States Ethics Committees (12).

Meanwhile, the European Federation of Pharmaceutical Industries and Associations (EFPIA) has been actively promoting innovation in this area. It has launched its Clinical Trials Strategy 2030+ with the goal of re-establishing Europe as a leading hub for faster, smarter, and more patient-centric clinical trials (

) (13). EFPIA is collaborating closely with other industry associations, patient organizations, academia, and national trade bodies to engage with regulators and policymakers, to help address the challenges in the current legislative framework (e.g., CTR, IVDR, MDR) and proactively proposing solutions (14). They are involved in multiple Innovative Health Initiatives (IHIs) that focus on patient-centred clinical research, aiming to improve trial design and conduct, enhance patient engagement and experience, increase the efficiency of clinical trials, and address health disparities.

Figure 2: Clinical Trials Strategy 2030+. (Figure courtesy of the author and modified from reference 13).

What are the implications of new clinical trial targets for pharma?

These new EC trial targets present new opportunities for the pharmaceutical industry to expand the number of multinational trials, provided they can meet patient recruitment timelines and comply with the CTR. It could catalyse changes in how clinical trials are conducted and managed in the EU, and allow sponsors to include more flexible trial design and market access strategies. This will help to increase speed to market and enable European patients to receive new therapies more quickly. The EU continues to streamline the trial process and reduce administrative burdens. Pharma companies must proactively engage with regulators and stakeholders to capitalize on the EU's strategy and strengthen their position in this multi-billion-dollar arena.

EMA. New Targets for Clinical Trials in Europe. Press Release. 23 Sept 2025. 

https://www.ema.europa.eu/en/news/new-targets-clinical-trials-europe

Wise, J. UK Launches Plan to Speed Up Clinical Trials and Improve Access to New Treatments 

Clinical Trials Market Size & Share Report, 2025 – 2034

Joshi, G.; et al. The Current Landscape of Clinical Trials. 

Apr 7;14(7):2519. DOI: 10.3390/jcm14072519.

The Lancet. Clinical Trials in Global Health 2025. 25 Mar 2025. 

https://www.thelancet.com/series-do/clinical-trials

Accelerating Clinical Trials in the EU (ACT EU)

https://accelerating-clinical-trials.europa.eu/about/initiative_en

EU Regulatory Activity is Driving Comprehensive Reforms for Pharma in 2025

https://www.osborneclarke.com/insights/eu-regulatory-activity-driving-comprehensive-reforms-pharma-2025

. 16 Apr. 2014. https://eur-lex.europa.eu/eli/reg/2014/536/oj

EC. ‘COMBINE’ Initiative–The Regulatory Landscape for Combined Studies on the IVDR/MDR/CTR Interface. 

. December 2024. https://health.ec.europa.eu/document/download/44bbffa5-e61e-4056-bfaf-50a46eeecb7b_en?filename=md_combine_presentation_en.pdf

EC. COMBINE Programme Launched a Pilot Coordinated Assessment for Clinical Trials and Performance Studies. 

https://ec.europa.eu/newsroom/sante/newsletter-archives/64447

EC.Clinical Trials–MedEthicsEU–Group of National Representatives of Medical Research Ethics Committees. 

https://health.ec.europa.eu/medicinal-products/clinical-trials/medethicseu_en

clinical-trial-strategy-in-a-snapshot.pdf

EFPIA. Improving EU Clinical Trials: Proposals to Overcome Current Challenges and Strengthen the Ecosystem

efpias-list-of-proposals-clinical-trials-15-apr-2025.pdf

The European Union (EU) is setting ambitious goals to enhance its clinical trial landscape by 2030, aiming to increase multinational trials and expedite patient recruitment. This initiative is part of the Accelerating Clinical Trials in the EU (ACT EU) program, which seeks to create a supportive environment for clinical research. The EU is also implementing complementary initiatives to streamline processes and reduce administrative burdens. These efforts present opportunities for the pharmaceutical industry to expand multinational trials, improve trial design, and accelerate market access, ultimately benefiting European patients with quicker access to new therapies.

The European Union aims to become the most attractive destination for clinical research.

New Targets for Clinical Trials in the European Union

On 2 July 2025, the European Commission (EC) announced a “bold and ambitious” new life science strategy to position the European Union (EU) as the world’s most attractive place for life sciences by 2030 (1). This strategy aims to positively impact public health, create jobs, and provide access to innovative, environmentally friendly technologies, thereby enhancing the EU's strategic resilience. Previously, President Ursula von der Leyen emphasized that the strategy for European life sciences was a priority in the political guidelines for 2024–2029, stating that, “we need to put research and innovation at the heart of our economy” (2). In January 2025, the EC also published the Competitiveness Compass, focusing on how to improve research and innovation (R&I) to boost competitiveness and promote innovation in biotechnology (3).

The EC has identified several strengths, including the presence of biotech clusters and leading academic institutions that operate with freedom and encourage diversity and inclusion. Additionally, the EU ranks second behind the United States (US) in terms of global high-value patents, holding 18% share of patents compared to 39% in the US (1). The life science industry continues to invest heavily in R&I, with 15% of the world’s top firms residing in the EU (1). However, the EC also recognizes that to improve R&I it must overcome numerous barriers, such as turning scientific breakthroughs into real-world applications, improving scalability in the EU, creating a more cohesive R&D ecosystem, utilizing healthcare data and artificial intelligence (AI) more effectively, and streamlining the regulatory process to reduce complexity and duplication (1).

How will this strategy unlock Europe’s life science potential?

To overcome these issues and achieve the life science strategy goals, the EC has outlined three interconnected phases that will impact the entire life science value chain:

optimising the R&I ecosystem to achieve a globally competitive life science sector

ensuring smooth and rapid market access for life science innovations

boosting the uptake and use of life science innovation.

The EC recognizes the need to optimize the R&I ecosystem and develop a globally competitive life science sector. This can be achieved through enhanced cooperation, more efficient resource use, and a comprehensive approach that leverages data and AI, develops adequate skills, and promotes a sustainable industry. The EC will provide new incentives for long-term multi-country clinical trials and rare disease research, strengthen EU bioclusters, and establish a network of European Centres of Excellence in advanced therapy medicinal products (ATMPs) (4). The EC also aims to establish a European Life Sciences R&I Data Assembly to improve cross-regulatory collaboration and enhance the interpretation of data regulations (5).

To ensure life science innovations reach the market smoothly and quickly, the European Medicines Agency (EMA) will need to establish more innovation-friendly regulations, and the EU will need to increase investments from both private and public sectors. A number of legislative changes and regulatory reforms are underway to simplify and modernize the approval processes, such as the Biotech Act and the EU Pharma Package, and these should help make the EU’s regulatory environment more conducive to biotech innovation (5–6). For instance, the Biotech Act, announced in July 2024, seeks to tackle barriers through legislative and policy changes across five key areas: regulation, financing, scale, skills, and data (7).

The creation of new knowledge is an essential basis for a vibrant life sciences ecosystem and for the development of technologies and innovations. The EC will invest more than €10 billion (US$11.9 billion) in various programs, such as Digital Europe, EU4Health, and Horizon Europe, to help build this knowledge base (8–10). Meanwhile, Member States and stakeholders must strengthen their connection with citizens to help reduce misinformation and build trust. They also need to collaborate more closely with end-users to ensure solutions meet their specific needs. Overall, this should help accelerate the adoption and use of life science innovations (1).

What is the future outlook for EU life sciences?

Although the EU is currently in a strong R&I position, the biotechnology sector remains highly competitive. In recent years, China has increased biotech investments following regulatory reforms, improvements in domestic manufacturing, and the implementation of the Healthy China 2030 initiative (11). Additionally, the implementation of US–EU framework on an agreement for reciprocal, fair, and balanced trade has resulted in a 15% tariff on pharmaceutical raw ingredients, APIs, and completed drug products imported into the US. This has raised expenses for branded pharmaceuticals developed by multinational corporations and created market uncertainty. Private investors are concerned that potential investments may slow due to pricing pressures, and biotech companies may hesitate to invest in new projects to expand operations, given the risks associated with tariffs (12–13).

Now more than ever, the EU needs to boost its investment in the life sciences sector and tackle longstanding issues that have hindered the region’s competitiveness and limited European patients' access to the latest treatments. Plans to introduce the Biotech Act in 2025, along with enhanced support for small and medium-sized enterprises and improvements to the regulatory framework, are positive developments. The European Federation of Pharmaceutical Industries and Association (EFPIA) welcomed the news; although, Nathalie Moll, Director General of EFPIA, warned that “for Europe to truly lead, it needs to invest in medicines, attract investment, accelerate turning that investment into new treatments and economic growth, and ensure that patients across the EU have access to them at the same pace as other regions” (14).

EC. A Competitiveness Compass for the EU 

Five Facts to Know: EU Life Sciences Strategy

. https://www.edelmanglobaladvisory.com. 2 July 2025. https://www.edelmanglobaladvisory.com/insights/five-facts-know-eu-life-sciences-strategy

 May 2025. https://www.europarl.europa.eu/RegData/etudes/BRIE/2025/772866/EPRS_BRI(2025)772866_EN.pdf

The New EU “Pharma Package”: Preparing for the Trilogue

. Crowell.com. 30 June 2025. https://www.crowell.com/en/insights/client-alerts/the-new-eu-pharma-package-preparing-for-the-trilogues

European Commission Sets Out New EU Life Sciences Strategy

. 1 Aug. 2025. https://www.osborneclarke.com/insights/european-commission-sets-out-new-eu-life-sciences-strategy

EU4Health Programme 2021-2027–A Vision for a Healthier European Union

. EC.europa.eu. (accessed 30 Sept. 2025). https://health.ec.europa.eu/funding/eu4health-programme-2021-2027-vision-healthier-european-union_en

(accessed 30 Sept. 2025). https://research-and-innovation.ec.europa.eu/funding/funding-opportunities/funding-programmes-and-open-calls/horizon-europe_en

Healthy China 2030: A Vision for Health Care

. https://www.ispor.org/docs/default-source/publications/newsletter/commentary_health-care_china_2030.pdf

Assessing the Impact of Tariffs on the Pharmaceutical Industry

. https://www.pharmafocuseurope.com/articles/impact-of-tariffs-on-pharmaceutical-industry.

The Impact of Tariffs on the Life Sciences Industry - Healthcare & Life Sciences Blog

31 Mar. 2025. https://healthcarelifesciences.bakermckenzie.com/2025/03/13/the-impact-of-tariffs-on-the-life-sciences-industry/

EU Life Science Strategy: A Springboard for European Life Sciences

. Efpia.eu. 2 July 2025. https://www.efpia.eu/news-events/the-efpia-view/statements-press-releases/eu-life-science-strategy-a-springboard-for-european-life-sciences/

Cheryl Barton is the founder and director of PharmaVision, Pharmavision.co.uk.

The European Commission has unveiled a comprehensive life science strategy to establish the EU as a global leader in life sciences by 2030. This initiative aims to enhance public health, create jobs, and foster innovation through environmentally friendly technologies. The strategy focuses on optimizing the research and innovation ecosystem, ensuring rapid market access for innovations, and increasing the adoption of life science advancements. Key measures include regulatory reforms, increased investments, and the establishment of European Centres of Excellence. The strategy also addresses challenges such as scalability, data utilization, and regulatory complexity.

The European Union aims to become the most attractive place for life science by 2030.

European Union Life Science Strategy

Metabolic dysfunction-associated steatohepatitis (MASH) is a type of non-alcoholic fatty liver disease (NAFLD) affecting more than 250 million people worldwide, and the number of cases in advanced stages is expected to double by 2030. Underlying conditions such as obesity, type 3 diabetes, and metabolic syndrome can increase the risk of MASH. For many years, MASH has been difficult to treat, and several products have failed in clinical development, including Genfit’s peroxisome proliferator-activated receptor (PPAR) agonist elafibranor, Gilead's ASK1 inhibitor selonsertib, Intercept Pharmaceuticals' farnesoid X receptor (FXR) agonist obeticholic acid, and Novartis/Conatus' pan-caspase inhibitor emricasan.

In the past few years, significant progress has been made, and new products have entered the MASH market. In March 2024, the US Food and Drug Administration (FDA) approved Madrigal Pharmaceuticals’ orally active, selective thyroid hormone receptor (THR)-β agonist, Rezdiffra (resmetirom), for adults with MASH with moderate to advanced liver scarring (fibrosis), alongside diet and exercise (1). In June 2025, the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) recommended granting conditional marketing authorization for Rezdiffra in MASH (2). On 19 Aug. 2026, the European Commission approved Rezidiffra, and the company is planning its first European launch in Germany in the fourth quarter of 2025 (3, 4).

In August 2025, Novo Nordisk received FDA approval for its injectable weight-loss drug Wegovy (semaglutide) for patients with MASH and moderate to advanced liver fibrosis, without cirrhosis, in combination with a reduced-calorie diet and exercise (5). The conditional clearance was based on Phase III results, where the glucagon-like peptide-1 receptor agonist (GLP-1RA) demonstrated improved liver scarring without worsening the condition and resolved MASH without making scarring worse (6,7). Eli Lilly is also seeking approval for its injectable dual-action gastric inhibitory polypeptide (GIP)/GLP-1RA, Zepbound (tirzepatide), based on its Phase II SYNERGY-MASH trial in patients with and without type 2 diabetes, and with stage 2 or 3 fibrosis (scarring) (8).

What investments are Big Pharma making in MASH?

MASH is seen as the next frontier in metabolic diseases, and numerous clinical trials are underway evaluating hormone analogs, receptor agonists, and enzyme inhibitors 

MASH drug development has attracted significant interest from pharma and private investors. In the GLP-1 space, Boehringer Ingelheim has teamed up with UK-based Zealand Pharma to develop survodutide. This dual glucagon/GLP-1 agonist has demonstrated a placebo-adjusted 64.8% improvement in fatty liver disease (9).

Table I. Clinical development of therapies targeting Metabolic dysfunction-associated steatohepatitis (MASH).

Table I. Clinical development of therapies targeting Metabolic dysfunction-associated steatohepatitis (MASH) (Continued).

In May 2025, GSK acquired Boston Pharmaceuticals' lead asset efimosfermin for US$1.2 billion plus US$800 million in milestone payments. Efimosfermin is a novel, once-monthly fibroblast growth factor 21 (FGF21) analog therapeutic in Phase II clinical development for the treatment of MASH (10). US-based Akero Therapeutics and 89bio are also developing FGF21 analogs, efruxifermin and pegozafermin, respectively (11, 12).

Which European frontrunners are targeting MASH?

Several European biotechs are developing innovative approaches to target the underlying cause of MASH and liver fibrosis. These include:

focuses on developing novel small-molecule therapies targeting fibrosis, lysosomal storage disorders, and oncology. Its lead program, lanifibranor (IVA337), is an orally active pan-PPAR agonist. It demonstrated promising results in a Phase IIb placebo-controlled trial in patients with active MASH, improving the resolution of MASH without worsening fibrosis (13). Lanifibranor is currently being evaluated in NATiV3, a pivotal Phase III clinical trial in patients with MASH and fibrosis stages F2 and F3; the trial is due to complete in September 2026 (14).

 is a clinical-stage biopharmaceutical company specializing in fibrolytic and anti-inflammatory diseases. Its lead compound, Vonafexor, is an orally active, non-steroidal agonist of NR1H4 (FXR, or bile acid receptor). In the Phase II LIVIFY study, Vonafexor significantly reduced liver fibrosis and inflammation markers, decreased liver fat and weight, and improved liver and kidney functions (15). In January 2024, the company raised €39 million (US$45.39 million) in Series C funding to develop Vonafexor further (16).

, is a biotechnology company focused on discovering and developing innovative peptide-based medicines. It has created several candidates targeting the metabolic brain axis, including Survodutide, a glucagon/GLP-1 dual agonist, which is being developed with Boehringer Ingelheim for MASH (Phase II) and obesity (Phase II), and petrelintide, an amylin analogue that it is co-developing with Roche. This compound could represent a new class of treatment for obesity (17).

In the United States, several biotech companies are working on MASH candidates, including MetaVia, which is developing DA-1726, a novel oxyntomodulin (OXM) analogue that functions as a glucagon-like peptide-1 receptor (GLP1R) and glucagon receptor (GCGR) dual agonist (18).

Additionally, significant progress has been made in developing biomarkers and diagnostics to identify patients at risk of MASH. For example, France-based Genfit has created a non-invasive diagnostic program that includes an 

test to detect NIS4+ and NIS2+. At the same time, Nordic Bioscience has identified biomarkers targeting neo-epitopes of collagen fragments that can be used to risk-stratify patients. (19, 20).

The global MASH market was valued at US$7.9 billion in 2024 and is forecast to grow to US$31.8 billion by 2033, with a compound annual growth rate of 17.7% from 2025 to 2033 (21). After many failed attempts, the industry has finally identified new ways to reduce liver fat buildup. While Madrigal's orally active Rezdiffra was the first to enter the US and European Union markets, it now faces competition from Novo’s injectable Wegovy, and Eli Lilly is not far behind. It will be interesting to see how the MASH landscape evolves and whether new approaches, such as oxyntomodulin analogues and gene silencing agents, can capture a share of this lucrative market in the future.

FDA Approves First Treatment for Patients with Liver Scarring Due to Fatty Liver Disease

Madrigal Receives European Commission Approval for Rezdiffra™ (resmetirom) for the Treatment of MASH with Moderate to Advanced Liver Fibrosis.

Madrigal Pharmaceuticals Reports Second-Quarter 2025 Financial Results and Provides Corporate Updates.

FDA Approves Treatment for Serious Liver Disease Known as ‘MASH’

Sanyal, A. J.; Newsome, P. N.; Kliers, I.; et al.

 Phase 3 Trial of Semaglutide in Metabolic Dysfunction-Associated Steatohepatitis

Semaglutide 2.4 mg Demonstrates Superior Improvement in Both Liver Fibrosis and MASH Resolution in the ESSENCE Trial.

Loomba, R.; Hartman, M. L.; Lawitz, E. J.; et al. (2024). T

irzepatide for Metabolic Dysfunction-Associated Steatohepatitis with Liver Fibrosis

Survodutide US FDA Breakthrough Therapy Phase 3 Trials MASH.

GSK. GSK to Acquire Efimosfermin, a Phase III-Ready Potential Best-in-Class Specialty Medicine to Treat and Prevent Progression of Steatotic Liver Disease (SLD). Press Release. 14 May 2025.

 akerotx.com/clinical-trials (accessed 20 Aug. 2025).

89bio Receives EMA PRIME Status for Pegozafermin in the Treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH) with Fibrosis and Compensated Cirrhosis.

Metabolic dysfunction-associated steatohepatitis (MASH), a subtype of non-alcoholic fatty liver disease, is projected to see a significant increase in advanced cases by 2030. Recent advancements include FDA and EMA approvals of Madrigal Pharmaceuticals' Rezdiffra and Novo Nordisk's Wegovy for MASH treatment. Big Pharma is heavily investing in MASH, with companies like GSK acquiring assets and others developing innovative therapies. European biotechs are also making strides with novel treatments. The MASH market is expected to grow substantially, driven by new therapeutic approaches and diagnostic advancements.

Novo Nordisk’s Wegovy will be competing with Madrigal Pharmaceuticals’ Rezdiffra in the metabolic dysfunction-associated steatohepatitis arena. 