3 Neurology solutions: delivering effective and meaningful solutions that patients truly value

3 Neurology solutions: delivering effective and meaningful solutions that patients truly value

Across our Neurology Solutions group, we are driven by our passion for helping people with severe diseases live their best life and addressing the impact of serious neurological and autoantibody-mediated conditions including epilepsy, Parkinson’s disease and rare diseases such as myasthenia gravis.

We aspire to develop a real understanding of patients’ needs. By amplifying patient voices, listening to their expert opinions, and learning about how they experience and live with neurological conditions, we are well positioned to deliver long-term solutions which will make a difference to their lives.

Our heritage, experience, and long-standing leadership in epilepsy, combined with a commitment to expand into specific populations suffering from neurodegenerative and neuro-immunological diseases, truly sets us apart. This unique combination provides robust foundations for patient value creation today, tomorrow, and in the long-term.

Vimpat®

Vimpat® demonstrates our continued commitment to addressing the needs of epilepsy patients. In the first half of 2019, UCB obtained approval for Vimpat® in Japan for partial onset seizures in children aged four and above. We have furthermore obtained approval for Vimpat® in China through an innovative and pioneering approach using real-world evidence and data extrapolation. Following this approach, Vimpat® was the first neurological drug to be approved in China for pediatric patients (four years and above) based on extrapolation, after having been approved following a similar methodology for the treatment of adult patients.

Nayzilam®

Our approach provides many unique opportunities to deliver sustainable patient value. It also comes with a responsibility to consistently strive to improve outcomes and experiences for the people who rely on our medicines every day. Whether through cutting edge science, progressive approaches to access, or pioneering health-tech solutions, our approach is patient centric throughout. This can be seen with Nayzilam® (midazolam) nasal spray CIV, a benzodiazepine indicated for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity that are distinct from a patient’s usual seizure pattern in patients with epilepsy 12 years of age and older. Nayzilam® now provides patients and caregivers with the first FDA-approved nasal option for treating seizure clusters.

To learn about our investigational new drug padsevonil and our innovative approaches in research and development visit the Research and Development section.

Charl van Zyl, Executive Vice President, Patient Value Neurology EU/IM (portrait)

We are committed to developing solutions that address areas of acute need while delivering sustainable value for people living with myasthenia gravis and other serious, rare, immune-related neurological diseases.

Charl van Zyl, Executive Vice President, Neurology Solutions & Head of EU/International

Watch video in the online version of the report

Another step towards providing differentiated solutions to patients with myasthenia gravis

In the IgG autoantibody-mediated autoimmune diseases space, UCB has made significant strides to provide solutions for patients with myasthenia gravis (MG), at different stages of this debilitating disease.

Internally, UCB is developing an investigational compound, rozanolixizumab, as an advanced subcutaneous anti-FcRn therapy. In 2019 UCB also announced an agreement to acquire Ra Pharmaceuticals, Inc. (Ra Pharma). Closing of this transaction (expected to occur by the end of Q1 2020) will add Ra Pharma’s zilucoplan to UCB’s product pipeline. zilucoplan is a peptide inhibitor of complement component 5 (C5), currently in Phase 3 trials. Because of its different mechanism of action, alongside UCB’s anti-FcRn, rozanolixizumab, zilucoplan, would create an opportunity to provide more people living with MG with additional treatment options.

Beyond MG, the planned acquisition of Ra Pharmaceuticals has the potential to enable UCB to offer new treatment opportunities for several rare diseases in neurology and immunology as well as different delivery forms, including extended release and orally available products. The combined portfolio may also offer synergies in the outreach to people with rare diseases and the healthcare market. UCB will also further strengthen its presence in the U.S., through expansion of the innovation hub in Boston, Massachusetts (U.S.).

In everything we do, we are realizing solutions by focusing on what patients care about and always keeping their needs in mind. We continue to forge paths where our patient value strategy, sustainable access, and medical advances align, helping people living with epilepsy today, and in the future helping patients living with MG and other neurological conditions.

To learn more about myasthenia gravis visit the Research and Development section.

Myasthenia gravis

At UCB, patients’ needs fuel our approach to innovation. We are partnering with the global patient community to refine our focus on delivering solutions for unreached patient populations with immunoglobulin G (IgG) autoantibody-mediated autoimmune diseases. These diseases – including myasthenia gravis (MG), immune thrombocytopenia (ITP) and chronic inflammatory demyelinating polyneuropathy (CIDP) – are characterized by life-limiting, and sometimes life-threatening, symptoms.

MG is a chronic neuromuscular condition where the body’s immune system mistakenly targets the connection between the nerves and the muscles, leading to weakness and fatigue of the skeletal muscle. We have made significant progress in 2019 to better understand the mechanisms underlying these debilitating diseases and the clinical potential of new treatment approaches. Patient feedback has reinforced our scientific efforts. Indeed, it has helped accelerate the development of rozanolixizumab for these patients.

Who does myasthenia gravis affect?

  • ~20 cases per 100 000 people worldwide1
  • 2x as many women as men2

What are the signs and symptoms of MG?

  • In approximately 65% of people, the first signs of MG are problems with the eyes, such as double vision or drooping eyelids.3,4
  • 15-20% of people with MG will experience a myasthenic crisis, that can lead to problems swallowing & respiratory failure.5
  • About 75% of people will develop more generalized weakness of muscles across the body.6

Employment challenges are common for MG patients

  • 27-59% patients with MG experienced employment challenges such as unemployment or the need to stop working7,8
  • 27-47% experienced long-term sickness absence9
  • 36-48% experienced decreased income10

It’s very hard to understand a disease where so many different people go through so many different symptoms and then also undergo so many different treatments. With these variable experiences in mind, I can’t tell you how pleasing it is to have a company out there listening to and learning from patients and working towards a treatment to help improve lives.

Tommy, living with myasthenia gravis

We have undertaken multiple collaborative initiatives to build on these insights, including:

  • Engaging with a global network of patients and advocacy organizations to provide insights on treatment, patient care, and unmet needs in MG, drug delivery devices and clinical trial design.
  • Generation of robust real-world evidence drawing on deep knowledge and understanding with the aim of securing broad patient access.
  • Inviting patients to our U.S. and global investigator meetings to highlight their unique experiences with MG and the importance of clinical trials to the MG community.

Through our research and advocacy work to date, we have built our knowledge about the extent to which MG symptoms can affect patients physically, socially and emotionally. This is compounded by the high treatment burden associated with the current standard of care, suggesting an urgent need for safe, effective, less invasive, less time-consuming treatment options.

With these unmet needs in mind, we are excited about the potential of rozanolixizumab to improve the experience and quality of life of people living with MG. In addition, closing of the planned merger of Ra Pharmaceuticals, Inc. would create an opportunity for UCB to provide more treatment options to a broader range of MG patients.

We will continue to use patient insights to inform our scientific advances and to build solutions which will deliver sustainable value. This will help to ensure that people living with MG can achieve their full potential.

We believe this approach is critical to our future long-term success, helping to generate a leadership position from the outset. In this way we will continue to support patients around the world to live their lives to their fullest.

1 National Institutes of Health – U.S. National Library of Medicine. Genetics Home Reference – Your guide to understanding genetic conditions: Myasthenia Gravis. Retrieved from: https://ghr.nlm.nih.gov/condition/myasthenia-gravis#statistics. Accessed November 2019.

2 Li Y, Arora Y, Levin K. Myasthenia gravis: newer therapies offer sustained improvement. Cleve Clin J Med. 2013 Nov. 80(11):711-21.

3 Robertson NP et al. Myasthenia gravis: a population based epidemiological study in Cambridgeshire, England. J Neurol Neurosurg Psychiatry 1999;65:492-496.

4 Kupersmith MJ et al. Development of generalized disease at 2 years in patients with ocular myasthenic gravis. Arch Neurol 2003;60(2):243-248.

5 Bershad EM et al. Myasthenia gravis crisis. Southern Medical Journal 2008;101(1):63-69.

6 Robertson NP et al. Myasthenia gravis: a population based epidemiological study in Cambridgeshire, England. J Neurol Neurosurg Psychiatry 1999;65:492-496.

7 CIE. The cost to patients and the community of Myasthenia Gravis: Understanding the patient experience and community wide impact: The Centre for International Economics; 2013.

8 Kulkantrakorn K, Jarungkiatkul W. Quality of life of myasthenia gravis patients. J Med Assoc Thai 2010;93:1167-71.

9 Frost A., Svendsen M.L., Rahbek J., Stapelfeldt C.M., Nielsen C.V. et al. Labour market participation and sick leave among patients diagnosed with myasthenia gravis in Denmark 1997-2011 BMC neurology 2016; 16(1):224.

10 Nagane Y, Murai H, Imai T, et al. Social disadvantages associated with myasthenia gravis and its treatment: a multicentre cross-sectional study. BMJ open 2017;7:e013278.