Cheryl Barton is director of PharmaVision, 

Cheryl Barton

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

On 11 December 2025, the European Parliament and the Council of the European Union (EU) reached a political agreement on reforming the EU’s pharmaceutical legislation.

 This is the most significant overhaul of the regulatory framework in more than 2 decades and will help Europe modernize the development and authorization of medicines across the EU.

What Is the New Pharmaceutical Legislation?

The new pharmaceutical legislation replaces the existing legislation for human medicines (Regulation [EC] No 726/2004 and Directive 2001/83/EC), including those for children and rare diseases (Regulation [EC] No 1901/2006; Regulation [EC] No 141/2000).

 In addition, the new legislation introduces a series of targeted amendments to the advanced therapy medicinal products (ATMP) regulation (Regulation [EC] No 1394/2007), the clinical trials regulation (Regulation [EU] No 536/2014), and the European Medicines Agency’s (EMA’s) extended mandate regulation (Regulation [EU] 2022/123).

The new pharmaceutical legislation is designed to, as follows:

•address major public health challenges, including antimicrobial resistance

•adapt the EU regulatory framework to support digitalization and foster innovation

EMA will provide regular updates throughout 2026 on the changes introduced. The new legislation will focus on several areas to support innovation and reduce procedural complexities and administrative burdens, particularly in paediatric and rare disease medicines. It will review medicine shortages and the environmental sustainability of medicines and amend the structure of the EMA scientific committees to improve efficiency and the evaluation of medicines.

The new pharmaceutical legislation will enter into force in 2026, with the following 2 years serving as a transition period to allow EU Member States to align their national laws with the new rules. The European Commission (EC), EMA, and EU Member States will provide implementation guidance to stakeholders in 2026 (

Figure 1. Implementation timeline for new pharmaceutical legislation. (Figure courtesy of the author)

On 12 January 2025, the EU Health Technology Assessment (HTA) regulation came into effect for oncology and ATMPs, with all new medicinal products to be incorporated from 2030 onward.9 The regulation ensures a scientifically based, evidence-driven, multidisciplinary approach to evaluating the relative effectiveness and additional benefit of health technologies compared with existing alternatives. The main objectives are to:

•harmonize clinical assessment methodologies across the EU

•reduce duplication of national assessments

•expedite patient access to innovative therapies

•support sustainable health care systems by providing robust evidence for decision-making.

This marks a major shift in the assessment of health technologies, with clinical evaluations now conducted at the EU level through the joint clinical assessment, which will run in parallel with the EMA process. The EU HTA regulation aims to expedite patient access, reduce duplication of effort across Member States, and harmonize the methodologies used for clinical evaluation.

In October 2025, Regulation (EU) 2025/2086 came into force, significantly affecting the healthcare sector in the EU. It further streamlines the HTA process for medical and in vitro diagnostic devices and is expected to enhance the efficiency and consistency of HTAs. It will benefit patients and health care providers by providing clearer, more reliable information on the effectiveness and safety of new health technologies. 

How Is the UK Aligning Marketing Authorization and HTA Recommendations?

As of April 2026, the pharmaceutical industry will be able to use the Integrated Scientific Advice service from the Medicines and Healthcare products Regulatory Agency and the National Institute for Health and Care Excellence to obtain UK marketing authorization and HTA recommendations in parallel.

 The joint scientific advice service will provide companies with greater clarity on evidence requirements and more predictable timelines, thereby avoiding unnecessary delays. It should help reduce the time to patient access by 3 to 6 months.

The aligned pathway will be optional for companies with eligible drugs. If preferred, companies can request a later submission date and use the traditional sequential approach. This flexibility may be appealing where global development programmes or regulatory timelines do not align with the earlier submission requirements.

What Are the Implications for European Pharma?

The EU and the UK continue to update legislation to ensure competitiveness and expedite patient access to innovative medicines. The new legislation and alignment of regulatory pathways should help harmonize regulation, reduce complexity and administrative burden, enhance operational efficiency, and drive the delivery of a sustainable health care system.

Reform of the EU pharmaceutical legislation. European Medicines Agency; 2025. Accessed 19 March 2026. ema.europa.eu

Regulation (EC) No 726/2004. European Medicines Agency; 2022. Accessed 19 March 2026.ema.europa.eu

Directive 2001/83/EC. European Medicines Agency; 2025. Accessed 19 March 2026.ema.europa.eu

Regulation (EC) No 1901/2006. European Medicines Agency; 2019. Accessed 19 March 2026.ema.europa.eu

Regulation (EC) No 141/2000. European Medicines Agency; 2025. Accessed 19 March 2026.ema.europa.eu

Regulation (EC) No 1394/2007. European Medicines Agency; 2019. Accessed 19 March 2026.ema.europa.eu

Regulation (EU) No 536/2014. European Medicines Agency; 2022. Accessed 19 March 2026.ema.europa.eu

Regulation (EU) 2022/123. European Medicines Agency; 2022. Accessed 19 March 2026.ema.europa.eu

Regulation (EU) 2021/2282. European Medicines Agency; 2021. Accessed19 March 2026.ema.europa.eu

Johns A, Andrade A, Johal S, Ryan J. EU joint clinical assessment: a framework for optimising use with cost-effectiveness decision-making. 

Chirico G. How are countries implementing the EU HTA regulation? An updated overview. Remap Consulting; 2025. Accessed 19 March 2026 https://remapconsulting.com/hta/how-are-countries-implementing-the-eu-hta-regulation-an-updated-overview/12.

Regulation (EU) 2025/2086. European Medicines Agency; 2025. Accessed19 March 2026.ema.europa.eu

Medicines: get integrated scientific advice from the MHRA and NICE. MHRA. GOV.UK; 2026. Accessed 19 March 2026.https://www.gov.uk/guidance/medicines-get-scientific-advice-from-mhra

Grubert N. Simultaneous UK marketing authorisation and HTA recommendation. LinkedIn. November 20, 2025. AccessedMarch 19, 2026. https://www.linkedin.com/posts/neil-grubert_uk-mhra-nice-activity-7382473273948807168-XNB_/.

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

When referring to this article, please cite it as Barton C. The impact of new pharmaceutical and HTA legislation in Europe. 

Articles

Europe is implementing sweeping reforms to medicine regulation and health technology assessment, with major implications for oncology, ATMPs, paediatric and orphan products. A new EU pharmaceutical legislative package will replace core human medicines rules, amend ATMP and clinical trials frameworks, and update EMA committee structures, while also addressing antimicrobial resistance, shortages, sustainability, and digitalization. In parallel, EU-level joint clinical assessment for HTA is now live for oncology and ATMPs and will expand to all new medicines by 2030. The UK is introducing optional parallel MHRA–NICE advice to compress access timelines.

New legislation will improve patient access to innovative medicines in Europe and the United Kingdom.

The Impact of New Pharmaceutical and HTA Legislation in Europe

Cancer cells being destroyed by immunotherapy | Image Credit: ©Jennifer -stock.adobe.com

Cancer remains the leading cause of death worldwide. The approval of immune checkpoint inhibitors (ICIs), which target cytotoxic T-lymphocyte-associated protein 4 (CTLA-4), lymphocyte activation gene-3 (LAG-3), and programmed cell death protein 1 (PD-1) receptors on T and B immune cells, has transformed the landscape of cancer treatment.

However, they are not effective against all cancers, have variable efficacy, and some patients may develop resistance to treatment.

Researchers have identified several new targets that could overcome resistance and restore T-cell immune responses (

 These include B and T lymphocyte attenuator (BTLA), B7 homologue 3 protein (B7-H3), CD112R, also known as poliovirus receptor (PVR)-related Ig domain (PVRIG),glucocorticoids-induced TNF receptor family-related protein (GITR), inducible co-stimulatory receptor (ICOS), indoleamine 2,3-dioxygenase 1 (IDO-1), natural killer (NK) group 2A (NKG2A), T-cell immunoglobulin and ITIM domain (TIGIT), T-cell immunoglobulin and mucin-containing protein-3 (TIM-3), and V-domain Ig suppressor of T cell activation (VISTA).

These findings have paved the way for potential new treatments to be used as monotherapies or, more likely, in combination with existing ICIs.

 Current and novel targets for immune checkpoint inhibitors in cancer. Modified from reference 5. Figure courtesy of the author.

There has been significant progress in understanding how cells evade the immune system.

 Researchers at Lund University, Sweden, have discovered a new immune evasion mechanism regulated by signaling lymphocytic activation molecule 6 (SLAMF6, also known as Ly108).

 SLAMF6 receptors are primarily expressed on progenitor or stem-like exhausted T cells and show both activating and inhibitory functions, which could be effectively utilized in immunotherapy.

Preliminary data with SLAMF6-targeting monoclonal antibodies (mAbs) have demonstrated improvements in the functional integrity of T cells 

 and in mouse models; this is a vital finding, because ICI blockade can cause T cell exhaustion.

What ICI Therapies Are the Pharma Industry Investing In?

Many large pharmaceutical companies have invested in this area. Leading commercial products include Merck & Co’s Keytruda (pembrolizumab), Bristol-Myers Squibb's (BMS) Opdivo (nivolumab), Roche/Genentech’s Tecentriq (atezolizumab), and AstraZeneca’s Imfinzi (durvalumab), which together represent the majority share of global ICI revenues.

Leading pharma firms continue to expand their oncology pipelines and have formed strategic collaborations and acquisitions to access new ICIs. For example, BioNTech acquired Biotheus to gain full global rights to BNT327/PM8002 (PD-L1 × VEGF-A bispecific) and has since established a global strategic partnership with BMS to co-develop and co-commercialize BNT327.

In October 2024, AbbVie and EvolveImmune Therapeutics signed a collaboration and option-to-license agreement to develop multispecific T cell-engaging antibodies in oncology.

What Are Some European Players to Watch Out for in the ICI Space?

Several European biotechs are evaluating next-generation ICIs that target a variety of pathways 

 European biotech companies developing immune checkpoint inhibitors in clinical development. Source: Author

is a global biotechnology company that has developed a comprehensive oncology portfolio comprising three therapeutic modalities: mRNA cancer immunotherapies; next-generation immunomodulators, such as antibody-drug conjugates (ADCs) and chimeric antigen receptor (CAR) T cell therapies; and targeted therapies. The company is developing a range of immunomodulators, including gotistobart (optimised Fc anti-CTLA4), pumitamig (anti PD-L1/anti VEGF A), BNT314 (anti 4-1BB/anti-EpCAM), BNT317 (anti-PD-1/CD39), and BNT3213 (anti-TIGHT/OVRIG) bi-specific antibodies.

 It is also evaluating HER2, TROP2, B7H3, HER3, EGFRxHER3, and ADCs as combination partners for cancer therapies. BioNTech has established a broad set of relationships with multiple global and specialized pharmaceutical collaborators, including BMS, Duality Biologics, Fosun Pharma, Genentech, Genmab, MediLink, OncoC4, Pfizer, and Regeneron.

is an international biotechnology company established in 1999. It has developed a broad range of antibody-based therapeutic formats, including bispecific antibodies, ADCs, immune-modulating antibodies, and other next-generation modalities. It has a strategic focus on novel drug combinations, including ICIs with immunomodulators, such as TIGIT and LAG-3. In 2025, it terminated development of acasunlimab, a PD-L1x4-1BB bispecific antibody; BioNTech withdrew from its development in August 2024

Meanwhile, in September 2025, Genmab acquired Merus for approximately US$8 billion to expand its late-stage pipeline, gaining access to petosemtamab, an EGFRxLGR5 bispecific antibody in phase 3 for head and neck cancer and MCLA‑145, a bispecific antibody targeting CD137 and PD-1/PD-L1 for solid tumours.

 is a global clinical-stage oncology-focused biotech company. It is pioneering NK cell checkpoint modulators and monalizumab, an anti-NKG2A antibody, in collaboration with AstraZeneca, in non-small cell lung cancer.

Innate Pharma has also established a collaboration with Sanofi, as well as renowned academic and research institutions, to advance innovation in immuno-oncology. 

was founded in 2022 by Thoas Fioretos, MD, PhD, Niklas Landberg, MD, PhD, Carl Sandén, PhD, and Kjell Sjöström, MSc, a life science entrepreneur and founder of Innovagen AB. The company has raised US$770,000 from Seed Venture Capitalists.

 It is currently evaluating several mAbs that target different ICI pathways, including LB01, which targets SLAMF6, T-cell mediated elimination of cancer cells for the treatment of leukaemia and solid tumours; LB02, which targets C3AR, which is located on leukemic stem cells; and LB03, which targets SIRPα-CD47, which is important in the regulation of macrophage function.

In 2025, the company published a study showing that LB01 inhibits a previously unknown immune escape mechanism and eliminates acute myeloid leukaemia (AML) cells in advanced experimental models.

 Based on the promising results, clinical trials are expected to start within 2 to 3 years.

 The company has forged strategic partnerships with Lunds University, Medicon Village, RISE, SciLife Lab, Smile Venture Hub, and Vinnova to drive the development of its pipeline products.

Over the past decade, significant progress has been made in cancer immunotherapy, including ICIs and CAR T-cell therapy, leading to improved management of various cancers.

Treatment continues to advance as innovative biotech companies explore new ICI pathways that can enhance and combine the effects of existing therapies.

 The global ICI market was valued at US$42.3 billion in 2024 and is expected to reach US$49.7 billion in 2025, expanding further to US$156.5 billion by 2033, with a compound annual growth rate of 15.4% from 2025 to 2033. The increasing global cancer burden will drive this robust growth, with more ICI approvals across different tumour types and earlier treatment lines, higher adoption of combination therapies, and broader access to treatments in emerging markets.

Many large pharmaceutical companies, including AstraZeneca, BMS, Genentech, Merck & Co., Roche, and Sanofi, have already made significant investments in this area. Innovative European biotech firms such as BioNTech, GenMab, Innate Pharma SA, and Lead Biologics have been quick to capitalise on investor interest, enabling them to explore new targets that could be used as monotherapies or in combination with approved therapies to improve clinical outcomes in a broader range of cancers.

Bandara S and Raveendran S. Current landscape and future directions in cancer immunotherapy: therapies, trials, and challenges. 

Tang Z. and Veillette A. Inhibitory immune checkpoints in cancer immunotherapy. 

Borgeaud M.; Sandoval J.; Obeid M.; et al. Novel targets for immune-checkpoint inhibition in cancer. 

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Immunogenicity of cell death and cancer immunotherapy with immune checkpoint inhibitors

Top 13 Immune Checkpoint Inhibitors Companies in 2025

Sandén C; Landberg N; Peña-Martínez P.; et al. Aberrant expression of SLAMF6 constitutes a targetable immune escape mechanism in acute myeloid leukemia. 

SLAMF6 as a drug-targetable suppressor of T cell immunity against cancer. 

SLAMF6 discovery offers hope for non-responding cancer patients

SLAMF6: Drug Target to Boost T Cell Immunity

BioNTech to acquire Biotheus to boost oncology strategy

BioNTech and Bristol Myers Squibb announce global strategic partnership to co-develop and co-commercialize next-generation bispecific antibody candidate BNT327 broadly for multiple solid tumour types

AbbVie and EvolveImmune Therapeutics announce collaboration and option-to-license agreement to develop next-generation cancer biotherapeutics

. Company Presentation. November 11, 2025.

Genmab Takes Full Control of Acasunlimab Development Program.

Genmab to Acquire Merus, Expanding Late-Stage Pipeline and Accelerating into a Wholly Owned Model.

Phase I study of MCLA-145, a bispecific antibody targeting CD137 and PD-L1, in solid tumors, as monotherapy or in combination with pembrolizumab. 

Innate Pharma announces €15m investment by Sanofi. 

The landscape of combining immune checkpoint inhibitors with novel Therapies: Secret alliances against breast cancer. 

Moving the needle on immune checkpoint inhibitors with novel targets: are we being TIMid or LAGging behind?, 

Immune checkpoint inhibitors targeting CTLA-4, PD-1/PD-L1, and LAG-3 have reshaped oncology but show heterogeneous responses and acquired resistance. A broadened checkpoint landscape (eg, TIGIT, TIM-3, VISTA, NKG2A, BTLA, CD112R/PVRIG, ICOS, GITR, IDO1, B7-H3) is being pursued largely in combinations. SLAMF6/Ly108 has been identified as a targetable immune-evasion mechanism on progenitor exhausted T cells; early anti-SLAMF6 antibodies restore T-cell functionality in models. Key revenues remain anchored by pembrolizumab, nivolumab, atezolizumab, and durvalumab, as major pharma and European biotechs expand bispecific and multispecific portfolios amid strong market growth forecasts.

European biotech companies are exploring new therapeutic targets on T-cells to overcome drug resistance and improve clinical outcomes in cancer.

European Biotechs Pushing the Frontiers in Immunotherapy 

Amyloid plaques in Alzheimer's disease | Image Credit: ©Artur -stock.adobe.com


Dementia affects more than 55 million people worldwide, with Alzheimer’s disease (AD) accounting for approximately 70% of cases.

 By 2050, it is estimated that around 130 million people could suffer from AD, driven by an ageing global population. Approximately 7% of people over age 65 have dementia, and prevalence rises sharply with age, often doubling every five to six years after age 65.

A large proportion of dementia cases remain undiagnosed; new diagnostic tests are under development that could help speed up diagnosis and patient access to approved and emerging AD treatments

 Eli Lilly has developed a telehealth platform, LillyDirect, to expand access and potentially improve early diagnosis and treatment uptake.

 AD and dementia impose enormous societal and healthcare costs worldwide, with urgent calls for better detection, treatment, and care infrastructure.

Traditional AD treatments, such as Forest Laboratories' orally active Namenda (memantine), a cholinesterase inhibitor, address the symptoms of the disease rather than slow disease progression.

 More recently, new disease-modifying therapies (DMTs), such as intravenous (IV) administered anti-amyloid β monoclonal antibodies from Biogen/Eisai’s Leqembi (lecanemab) and Eli Lilly's Kisunla (donanemab), have been approved for early stages of AD with confirmed amyloid pathology.

 However, there remains a high unmet need for safer, easy-to-use drugs and therapies targeting non-amyloid/tau pathways. In 2025, the US Food and Drug Administration (FDA) accepted the supplemental biologics licensure application (BLA) for Leqembi Iqlik (lecanemab-irmb), a subcutaneous autoinjector formulation for once-weekly maintenance dosing in the United States.

There has been significant investment in the AD space in recent years, and more than 138 drugs were investigated in 182 clinical trials in 2025. These drugs target a range of novel targets beyond amyloid and tau, including pathways affecting bioenergetics and metabolism, the endocrine system, oxidative stress, synaptic plasticity, and the vasculature (

 New Alzheimer’s disease treatments target a wide range of biological pathways. Figure courtesy of the author. 

What AD therapies are the pharma industry investing in?

At present, Biogen, Eli Lilly, and Eisai monopolise the AD market; however, many large pharma firms also run AD programs. For example, Johnson & Johnson (J&J) and Roche/Genentech are developing anti-amyloid antibodies, posdienmab and trontinemab, respectively.

 Large pharma has been active in licensing assets and co-developing innovative platforms, ranging from anti-tau antibodies to natural killer (NK) cell therapies. Meanwhile, the regulators have published guidance and established frameworks, including FDA Fast Track and European Medicines Agency (EMA) adaptive pathways, to help align drug development and investment in innovative medicines for AD and other areas of unmet medical need.

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

Several European biotechs are evaluating next-generation anti-amyloid agents, tau and sigma inhibitors. European biotech leading the way include the following.

 is a clinical-stage biopharmaceutical company focusing on novel treatment for neurodegenerative diseases leveraging its clinically validated technology platforms, SupraAntigen and Morphomer. The company is developing a broad range of AD therapies, including ACI-35.030 (JNJ-2056), an anti-pTau active immunotherapy in phase 1b/2a targeting extracellular phosphorylated Tau (pTau), partnered with Janssen Pharmaceuticals; the company published trial data in eBioMedicine, and further data are expected in 1H26.

 ACI-24.060 targets misfolded Abeta to prevent accumulation and enhance clearance of amyloid plaques in AD and AD in Down syndrome and is currently being evaluated in the ABATE phase 1b/2 with Takeda. Results of this study are due to be released soon.

Anavex Life Science Corporation (Germany) 

focuses on novel small-molecule treatment for central nervous system diseases. Its lead candidate, blarcamesine, is a once-daily oral Sigma-1 receptor modulator that enhances autophagy. The company has filed a new drug application with EMA and FDA based on pivotal ATTENTION-AD and AD-004 trials in early AD, in which it demonstrated significant clinical benefit and slowed brain volume loss compared with the intent-to-treat population.

 In December 2025, the company requested a re-examination of the EMA's refusal of its marketing authorization because “the main study failed to demonstrate effectiveness and safety of blarcamesine in patients with early AD who do not have a mutation in the SIGMAR1 gene”.

 is a clinical-stage biotech company developing small molecules targeting tau protein aggregation. In July 2024, it completed a $100 million Series C financing round led by Novo Holdings, with new investment from EQT Life Sciences–LSP Dementia Fund, OrbiMed, and SR One, alongside existing investors M Ventures, Sofinnova Partners, GSK Equities Investments Limited and Johnson & Johnson Innovation (JJDC, Inc.).The funds were used to advance its lead asset, ASN51, an oral small-molecule drug designed to inhibit OGA, an enzyme implicated in protein aggregation in AD and Parkinson’s disease (PD).

 developed Leqembi in conjunction with Eisai, one of the first DMTs for early AD. The drug is now approved in more than 50 countries and continues to show strong sales growth. In September 2025, BioArtic received regulatory approval for monthly IV maintenance treatment in China. Eisai received US approval for Leqembi Iqlik weekly maintenance dosing, using a subcutaneous autoinjector. BioArtic signed a global out-licensing agreement with Bristol Myers Squibb (BMS) for two preclinical AD programmes: BAN1503, an antibody targeting pyroGlu Abeta, and BAN2803, an antibody targeting PyroGlu Abeta combined with BioArtic’s Brain Transporter technology.

 a UK biotechnology company, focuses on the treatment of AD by modulating retinoic acid receptor alpha (RARα), as identified in Professor Jonathan Corcoran's research at King's College London.

 has taken a different approach, targeting septin filaments, which regulate calcium entry into cells. This function is impaired in AD. In September 2025, the company published data from the phase 2a trial, which was halted prematurely due to dose-dependent elevations in liver enzymes that normalized after treatment was stopped.

 The company is developing REN392, a second-generation septin modulator that can help restore neuronal calcium balance, with an optimised safety profile and pharmacokinetic profile.

) are developing small-molecule tau aggregation inhibitors, and their lead programme is hydromethylthionine mesylate (HMTM), which has demonstrated promising results in the phase 3 LUCIDITY in the treatment of mild cognitive impairment in AD.

 is a spin-off company from the University of Cambridge (UK) and Lund University (Sweden). In January 2021, it closed a £12.4 million ($17 million) financing round; the total capital raised to date is approximately £33 million ($45 million).

 The funds have been used to advance its two misfolding small-molecule programs towards the clinic for the potential treatment of AD and PD.

Competition in the AD field is increasing, and several US biotechs—Alzheon, Annovis Bio, Alpha Cognition, Cognition Therapeutics, Longeveron, Neurodon—have R&D programs in this space and include NKGen Biotech, which is developing troculeucel, the first autologous enhanced NK cell therapy for AD.

 Asia-Pacific activity is growing, and several biotech companies—ADEL, AirBio Co. Ltd, GemVax, Shanghai Hengrui, and Therabest Korea—are developing AD agents and attracting pharma interest. In December 2025, Sanofi signed a US$1.04 billion licensing agreement with Korean biotech ADEL for ADEL-Y01, a tau-targeting antibody for AD.

Future opportunities in targeting the proteasome

Treatment for AD continues to advance, and innovative biotech companies are now looking beyond anti-amyloid and anti-tau programs to target the underlying causes of neuroinflammation and immune modulation in AD rather than focusing on symptomatic therapies.The global Alzheimer’s drugs market size was valued at US$3.52 billion in 2022 and is projected to grow from US$3.69 billion in 2023 to US$5.21 billion by 2030, exhibiting a compound average growth rate of 5.1% due to improvements in diagnosis and the emergence of new DMTs.

 Many large pharma companies, including Biogen, Bristol-Myers Squibb, Eisai, Genentech, Janssen, Roche, Sanofi, and Takeda, have already invested in the field, and several pivotal studies are due to report during the next 18 months that could stimulate further investment in this dynamic but high-risk market.

Alzheimer’s Disease Diagnosis: An Update and Review of Biomarkers and Clinical Practice

FDA OKs First Blood Test That Can Help Diagnose Alzheimer’s Disease.

Eli Lilly Announces Expanded Services to Early Diagnosis and Access Alzheimer's Disease Care.

Therapeutic Advances in Targeting the Amyloid-β Pathway for Alzheimer’s Disease.

Eisai Initiated Rolling Supplemental Biologics License Application to the US FDA for LEQEMBI IQLIK (lecanemab-irmb) as a Subcutaneous Starting Dose for the Treatment of Early Alzheimer’s Disease Under Fast Track Status.

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Genentech and Roche Present New Insights in Alzheimer’s Disease Research Across Its Diagnostics and Pharmaceutical Portfolios at AAIC

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NKGen Biotech to Present on the Use of Troculeucel for Neurodegenerative Diseases at the XXVII World Congress of Neurology. 

ADEL Signs $1.04 Billion Global License Agreement with Sanofi for ADEL-Y01, a Novel Investigational Alzheimer's Disease Therapy

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Cheryl Barton, PhD, founder and director, PharmaVision

Dementia prevalence is rising rapidly, with Alzheimer’s disease comprising most cases and substantial underdiagnosis persisting despite emerging biomarker-based diagnostics and telehealth-enabled access models. Symptomatic therapies remain in use, but disease-modifying anti-amyloid monoclonal antibodies now dominate early AD treatment, including movement toward subcutaneous, autoinjector maintenance dosing. The development pipeline is broadening beyond amyloid/tau into metabolism, oxidative stress, synaptic biology, vascular targets, and immune modulation. European biotechs and Asia-Pacific innovators are increasingly influential via novel mechanisms, financings, and major licensing deals.

New disease-modifying agents are being evaluated in the clinic that could change the course of treatment for Alzheimer’s disease patients.

European Biotechs Impacting Treatment of Alzheimer’s Disease


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.

 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.

 In recent years, significant progress has been made in the selective delivery of PROTACs, potentially extending their use to a broader range of conditions.

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   carfilzomib (Kyprolis), Bristol Myers Squibb’s ixazomib (Ninlaro), and Johnson & Johnson/Takeda’s bortezomib (Velcade). 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.

 In September 2025, Novartis initiated a collaboration with US-based Monte Rose to develop novel degraders or molecular glues to treat immune-mediated diseases.

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 3 VERITAC-2 clinical trial with vepdegestrant in metastatic breast cancer.

 Additionally, C4 Therapeutics and Nurix Therapeutics have protein degrader programs: cemisoldomide in phase 1/2 for multiple myeloma/non-Hodgkin lymphoma (NHL), and bexobrutideg in phase 2/3 for relapsed/refractory chronic lymphocytic leukaemia (R/R CLL), respectively.

More recently, US-based Kymera Therapeutics received fast track designation from the US FDA for KT-621, an oral signal transducer and activator of transcription 6 (STAT6) degrader for the treatment of atopic dermatitis.

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 2 trials for atopic dermatitis and hidradenitis suppurativa.

 Other US biotech companies working in this field include Accutar Biotech, Cleave Therapeutics, Dialectic Therapeutics, and Karyopharm Therapeutics.

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

focuses on developing small-molecule proteasome activators for neurodegenerative disease using its DGRADX platform. In October 2025, the company completed a US$15 million financing round led by life sciences investors Apollo Health Ventures and Novo Holding.

 Funds will be used to advance its multidisease pipeline.

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

QLi5 Therapeutics GmbH (German-Korean joint venture) 

is working on a new class of proteasome inhibitors that demonstrate strong anti-tumour and anti-inflammatory activity, offering promise in cancer, autoimmune, and inflammatory disorders.

 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.

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.

 Preliminary research indicated that proteasomes may have far-reaching effects and could be used to target new immune defence mechanisms.

 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 to 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. 

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Garber K. How protein-slayer drugs could beat some of the cruellest cancers. 

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Lee CH, Nguyen TM, Lee Y, et al. Overcoming therapeutic resistance in triple-negative breast cancer: targeting the undrugged kinome. 

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Yang X, Wang D, Li C, et al. Nano-PROTACs for precision medicine: engineering strategies for enhanced targeting and potency. 

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 Johnson & Johnson completes acquisition of Halda Therapeutics and its novel platform to revolutionize cancer treatment and enable next-generation oral therapies. Press release. Halda Therapeutics. Published December 29, 2025. Accessed February 19, 2026. 

https://haldatx.com/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. Press release. Monte Rosa Therapeutics. Published September 15, 2025. Accessed February 19, 2026. 

https://ir.monterosatx.com/news-releases/news-release-details/monte-rosa-therapeutics-announces-collaboration-novartis

 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. Press release. Arvinas. Published December 6, 2025. Accessed February 19, 2026. 

https://ir.arvinas.com/news-releases/news-release-details/arvinas-presents-preclinical-data-supporting-mechanistic

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.