1 Research and Development: innovating for patients
1 Research and Development: innovating for patients
We constantly innovate to deliver solutions for patient populations in disease areas within our current expertise of neurology, immunology, neuro-immunology and by expansion within adjacent areas.
In 2019, a strong multi-year investment in research and development has progressed two new treatments for patients in our core areas of immunology and neurology, while advancing potential solutions for new patient populations.
In the long-term our aspiration is to move from treatment to disease modification, and eventually, towards a cure for several severe chronic diseases. We already see progress in this direction with potentially disease modifying treatments in our pipeline such as our early development compounds targeting Tau and a-Syn proteins that play a role in neurological and degenerative diseases.
Innovation in research, aiming for a cure
The journey towards differentiated solutions for specific patients starts early in research. Our research process is grounded in strong science. We aim to understand the evolving knowledge underpinning disease biology and combine this with groundbreaking technologies and platforms to develop novel therapies.
Our small molecule platform, together with our insights into pathways in epilepsy delivered padsevonil, a rationally-designed medicine with a unique mode of action. padsevonil has now progressed to Phase 3 clinical studies for a sub-population of patients with drug-resistant epilepsy.
A differentiated solution means providing treatments to address real patient needs, where it matters most to them.Dhaval Patel, Executive Vice President & Chief Scientific Officer
Our research continues to explore new treatment options to support patients with Parkinson’s disease. Together with patients and experts in this space, we are working closely to validate and improve the way in which clinical studies are conducted, with the aim of incorporating real-world evidence into our trial designs.
We continue to evolve our world-class capabilities in antibody research. The progress of bimekizumab in late stage clinical development has demonstrated UCB’s ability to transform scientific innovation into a differentiated medicine for patients. Scientific understanding of pathways driving severe skin disease in psoriasis – and the importance of dual inhibition of IL-17A and IL-17F in a novel antibody – has enabled UCB to show a benefit for patients with psoriasis in Phase 3 clinical studies of bimekizumab.
In the IgG antibody-mediated autoimmune diseases space, which includes the rare diseases myasthenia gravis (MG), immune thrombocytopenia (ITP) and chronic inflammatory demyelinating polyneuropathy (CIDP), we are focused on the patient journey and areas of unmet need to incubate new solutions. Our progress to better understand the mechanism of these diseases, the scientific potential of new modalities, and the patient experience, has been significant. Our novel antibody rozanolixizumab specifically targets the human neonatal Fc receptor (FcRn) addressing immunoglobulin G (IgG) autoantibody-mediated diseases.1,2 The subcutaneous formulation of this molecule offers a potentially transformative option for patients, allowing them to move away from infusion centers to self-administration.
In 2019, UCB entered into an agreement to acquire Ra Pharmaceuticals, Inc. (Ra Pharma). When finalized, this acquisition will continue to broaden the scope of our scientific expertise by giving us access to a proprietary technology platform to produce synthetic macrocyclic peptides. The platform, known as ExtremeDiversity™, is based on messenger ribonucleic acid (mRNA) display and combines the diversity, specificity and high affinity of therapeutic antibodies with the attractive pharmacological properties of small molecules. It has the potential to augment UCB’s drug discovery capabilities and provide access to Ra Pharma’s proven expertise and talent in this area.
New modalities such as gene therapy offer the potential to drive a fundamental change in how diseases are treated. The ability to remove or add disease-related proteins with a single treatment, gene therapy could offer the potential of a cure in defined patient populations. UCB is already exploring new science and technologies to make balanced strategic investments in this extremely exciting field.
In epilepsy, for example, we anticipate a move from chronic symptomatic treatment towards addressing the burden of seizures through new disease-modifying medicines, targeted gene therapies and drug-technology combinations. With our scientific and technology partners, we are committed to being at the forefront of this evolution.
Innovating in Development, bringing differentiation to life
A promising pipeline
In 2019, we made notable strides towards achieving our ambition of delivering differentiated solutions. Compared to last year, we have gained approval for two new medicines and started five Phase 3 programs, involving more than 4 000 patients.
A unique outcome is an outcome that is clearly recognized as impacting the health and life of people with severe diseases in the eyes of all stakeholders, not only patients, but also payers and physicians.Iris Löw-Friedrich, Executive Vice President & Chief Medical Officer
We also built a technology transformation initiative across our clinical development activities to increase the efficiency of our clinical development activities. For example, UCB continues to explore decentralized trials in our innovative partnership with Science 37, where we will bring the trial into patients’ homes using the latest technologies. This is expected to accelerate development times, with patients enrolled faster in trials at a lower cost.
Beyond this, we are trying to improve every aspect of the patient experience in trials: we have developed ‘lay summaries’ of our clinical studies for our website, written in non-technical language and therefore accessible for any patient who is interested in our trials.
To learn more about our pipeline visit the business performance review.
Knowing that my experiences could really help further science, understanding and patient care is important to me. I think that participating in clinical trials and the drug development process is something we, as individuals, can give to people who come after us.Kelly, living with myasthenia gravis
Looking towards the future in neurology
In 2019, we continued the development of padsevonil, by launching a Phase 3 clinical program as planned. padsevonil could deliver significantly improved outcomes for patients with drug-resistant epilepsy who currently have few treatment options.
We accelerated the development of our novel subcutaneous anti-FcRn monoclonal antibody, rozanolixizumab, having achieved proof-of-concept in a Phase 2 study in patients with myasthenia gravis (MG) at the end of 2018. A confirmatory study in MG started in the second half of 2019. Building on the potential clinical utility of rozanolixizumab in other neurological conditions driven by pathogenic immunoglobulin G (IgG) autoantibodies, we initiated a Phase 2 study in patients with chronic inflammatory demyelinating polyneuropathy (CIDP). UCB is also advancing development in immune thrombocytopenia (ITP), with positive results from a Phase 2 study announced this year.3
To learn more about UCB’s commitment to people with myasthenia gravis visit the MG spotlight in our Neurology solutions section.
We strive to move beyond symptomatic treatment towards disease modification across different neuro-degenerative diseases. UCB has several investigational new drugs currently in development, including an anti-Tau monoclonal antibody being investigated as a potential new treatment option for people living with Progressive Supranuclear Palsy (PSP). In partnership with advocacy groups, we are learning more about the lived PSP experiences which is shaping our approach to clinical development.
Looking towards the future in immunology
At the end of 2019, our bimekizumab Phase 3 clinical program in psoriasis delivered impressive results, including demonstrating superiority to two widely used psoriasis biologic therapies. We now have strong evidence that bimekizumab, our investigational IL-17A and IL-17F dual inhibitor, has the potential to improve skin clearance rates, as well as improvements in itch, pain, and scaling, all of which are critically important in positively impacting the lives of psoriasis patients. UCB is now preparing for a bimekizumab submission to regulatory authorities in key markets by mid-2020. The efficacy and safety of bimekizumab is also currently being assessed in Phase 3 trials in psoriatic arthritis, ankylosing spondyloarthritis, nr-axSpA and hidradenitis suppurativa.
In 2019, with our partner Biogen, we decided to initiate a Phase 3 program with dapirolizumab pegol in patients with systemic lupus erythematosus (SLE) in 2020. dapirolizumab is a pegylated, Fc free antibody blocking CD40L, a critical molecule in the activation of autoimmune T and B lymphocytes in SLE. dapirolizumab pegol represents a leading and innovative mechanism of action. Additionally, UCB and Biogen have identified a better way to select patients in Phase 3 so that the recruited population truly reflects the target population with unmet medical needs. This hypothesis has been validated across multiple study databases.
1 Kiessling, P., R. Lledo-Garcia, S. Watanabe et al. The FcRn inhibitor rozanolixizumab reduces human serum IgG concentration: A randomized Phase 1 study. Sci Transl Med. 2017; 9(414).
2 Smith B, Kiessling A, Lledo-Garcia R, et al. Generation and characterization of a high affinity anti-human FcRn antibody, rozanolixizumab, and the effects of different molecular formats on the reduction of plasma IgG concentration. MAbs 2018;10: 1111-30.
3 Robak T., Kaźmierczak M., Jarque I., Musteata V., Treliński J. et al. Rozanolixizumab, an AntiFcRn Antibody: Final Results from a Phase II, Multiple-Dose Study in Patients with Primary Immune Thrombocytopenia Blood 2019; 134(suppl.1), abs 897 ASH (2019) American Society of Hematology – 61st Annual Meeting, 7-10 December 2019; Orlando, USA.