2 Immunology solutions: where deep insight and innovative science change lives

2 Immunology solutions: where deep insight and innovative science change lives

Within the Immunology Solutions group, we are laser focused on creating value for people living with psoriasis, psoriatic arthritis, axial spondyloarthritis (axSpA), rheumatoid arthritis and in the future, lupus. We are committed to understanding how best to address these chronic inflammatory diseases which profoundly impact patients’ lives.

With the development of an exciting, differentiated portfolio of rheumatology and immuno-dermatology therapies, we continue to deliver on UCB’s Patient Value Strategy by connecting innovative science with the unmet needs of patients.

Emmanuel Caeymaex, Executive Vice President, Patient Value Immunology & US solutions, UCB (portrait)

At UCB we aim to create unique value for specific patient populations. We are proud of the difference we are making for patients that didn’t have a solution before, like those suffering from a debilitating inflammatory back condition called non-radiographic axial spondyloarthritis.

Emmanuel Caeymaex, Executive Vice President, Immunology Solutions & Head of U.S.

Watch video in the online version of the report

Cimzia®

A prime example is the continued growth of our biologic, Cimzia® (certolizumab pegol), the only Fc-free, pegylated anti-TNF therapy. In March 2019, Cimzia® became the first and only treatment to gain approval from the U.S. Food and Drug Administration (FDA) for the treatment of active non-radiographic axial spondyloarthritis (nr-axSpA) with objective signs of inflammation. The approval was based on the unique 52-week placebo-controlled C-AXSPAND study, which demonstrated that nr-axSpA patients experienced a rapid and substantial improvement in their disease when treated with Cimzia®, compared to placebo, when added to standard therapy. The study showed a meaningful improvement in patients’ disease activity, including pain, physical function, mobility, and objective signs of inflammation.1

Importantly, our in-depth understanding of the debilitating symptoms that negatively impact the lives of patients with nr-axSpA life reinforced the need for a therapy that aims to treat the underlying inflammation. The FDA approval and the publication of our C-AXSPAND study marked an important advance for people with nr-axSpA. More treatment options, earlier diagnosis, and awareness building will lead to better outcomes for people living with the disease. Our efforts have increased the understanding and recognition of this disease worldwide.

We are also committed to understanding and addressing the unique needs of women with chronic inflammatory diseases. For these women, family planning can present complex challenges. Adequate disease control before and during pregnancy is crucial to ensure the best fetal and maternal health and a large proportion of women with chronic inflammatory diseases depend on medication to keep their symptoms under control during pregnancy.2,3,4,5 Additionally, after pregnancy, disease flares occur post-partum in 40-90% of new mothers (depending on the disease) – sometimes as soon as four weeks after giving birth – often leading to a trade-off between treatment and breastfeeding.6,7 Our clinical research and subsequent label updates made Cimzia® the first anti-TNF treatment option that could be considered for women with chronic inflammatory diseases, during both pregnancy (when clinically needed) and breastfeeding. We continue to support these women with innovative patient education and disease awareness initiatives conducted via social media and international advocacy efforts.

Ultimately, we want to ensure that patients have an optimal experience of their treatment, while maintaining a sustainable model for society. In China, with this objective in mind, we are partnering with CinKate to leverage the potential of digital strategies to identify patients that are most likely to benefit from Cimzia® and facilitate their connection with an appropriate healthcare provider. In the U.S., we operate a best-in-class support solution to ensure patients can obtain seamless access to Cimzia®. The program provides patients with tools and support to empower them to manage their condition such as our nurse program CIMplicity® – this initiative aims to answer patient questions, provide injection training, discuss nutrition and wellness information and support the patient during treatment.

Evenity®

Recently approved in the European Union, the U.S, Japan, Canada, Australia and South Korea, Evenity® is a bone forming anti-sclerostin antibody with a novel dual effect that increases bone formation whilst to a lesser extent decreases bone resorption. The origins of its discovery were based on the rare, inherited condition of sclerosteosis (sclerostin deficiency), characterized by bone overgrowth in sufferers. Research identified that sclerosteosis is caused by a mutation in the sclerostin gene.8,9 At first glance, sclerosteosis and osteoporosis appear very different since sclerosteosis patients do not produce sclerostin and their bones are thicker and stronger than normal, while osteoporosis patients have bones that become weak and brittle.

However, with these findings on the cause of sclerosteosis, our scientists correctly hypothesized that they could create a new medicine that could bind to – and inhibit – sclerostin and thus promote new bone formation to address low bone mass disorders such as osteoporosis.10

Today, UCB, together with our partner Amgen*, is the first company that developed an anti-sclerostin therapy, and the first company in the last decade to successfully bring an osteoporosis treatment to patients in all major markets, including Europe.10 The gene-to-drug development of Evenity® demonstrates how we translated a genetic discovery into a new medicine, turning conceptual science into a reality; a real journey from patient to science to solution.11

To learn more about our investigational new drugs bimekizumab, dapirolizumab pegol visit Research and Development and Key events sections.

Improving care for people living with chronic inflammatory disease improves their outcomes and their lives. Helping patients live their best life is our ultimate ambition. Knowing that we are making a difference is what drives us forward every day.

* UCB and Amgen are co-developing and co-commercializing Evenity®.

From patient to science to solution – Evenity®’s discovery and development story

We aim to truly understand the needs of patients. The inspiration for new medicines is sometimes found in extraordinary places or people. Nowhere is this more evident than in our Evenity® story. It’s a tale of inspiration from an extraordinary place, and how, together with our partner Amgen*, we turned a genetic discovery into a new medicine.

The story begins in South Africa with a rare inherited genetic condition called sclerosteosis. Sclerosteosis (first characterized in the 1960’s) affects less than 100 people worldwide and is a condition that causes excess bone formation due to the lack of or low levels of a protein called sclerostin.12 At first glance osteoporosis and sclerosteosis seem very different but scientists made several exciting discoveries:

  • X-rays showed that people with sclerosteosis have high bone mass leading to large and strong bones that have shown fracture-resistance even in traumatic situations (effectively the opposite of osteoporosis).
  • The strong bones of sclerosteosis patients were found to be caused by a mutation in a previously undiscovered SOST gene encoding a protein named sclerostin.13
  • Sclerostin is predominantly expressed in bone and inhibits bone formation. Because sclerosteosis patients don’t produce sclerostin, their bones are thicker and stronger than normal.

Based on these discoveries our scientists correctly hypothesized that they could create a new medicine that could bind to and inhibit sclerostin and thus promote bone growth to address low bone mass disorders such as osteoporosis. With this hypothesis in mind, our scientists worked together screening thousands of antibodies in the search for the best candidate to inhibit sclerostin and move into the next phase of development.

From there, our drug development teams identified a clinical candidate antibody that bound to and inhibited the activity of sclerostin. It worked by having a dual effect on bone, both building new bone and slowing existing bone loss. This antibody was named romosozumab and moved into clinical testing.14,15,16

Hanneke, living with osteoporosis (portrait)
Hanneke, living with osteoporosis

Then came a unique opportunity. In 2011, UCB and Amgen received a request from NASA to test a version of Evenity® in space – where the lack of gravity can cause astronauts to lose bone mass. This study of mice in orbit showed promising results: the bone strength of mice given the medicine increased compared to the mice that were not treated.

Back on Earth, after successful Phase 1 and 2 clinical trials, an extensive Phase 3 program was started. This program included two large fracture trials comparing Evenity® to either placebo or active comparator in more than 10 000 postmenopausal women with osteoporosis. The results of the Phase 3 program showed that Evenity® was effective in increasing patients’ bone strength and significantly reducing their risk of fracture with 12 monthly doses.

We are proud that this new bone-forming treatment providing a way to improve bone mass and reducing the risk of life changing fractures in those with osteoporosis at high risk of fracture is approved in the European Union, the U.S, Japan, Canada, Australia and South Korea.17

* UCB and Amgen are co-developing and co-commercializing Evenity®.

1 Deodhar A, et al. A Fifty-Two-Week, Randomized, Placebo-Controlled Trial of Certolizumab Pegol in Nonradiographic Axial Spondyloarthritis. Arthritis Rheumatol. 2019;71(7):1101-1111.

2 Jetwa H, Lam S, Smith C, et al. Does Rheumatoid Arthritis Really Improve During Pregnancy? A Systematic Review And Metaanlysis. J Rheumatol. 2019; 46(3):245-50.

3 Zbinden A, van den Brandt S, Østensen M. Risk for adverse pregnancy outcome in axial spondyloarthritis and rheumatoid arthritis: disease activity matters. Rheum 2018;57(7):1235-1242.

4 Polachek A., Li S., Polachek I.S., Chandran V., Gladman D. Psoriatic arthritis disease activity during pregnancy and the first-year postpartum Semin Arthritis Rheum 2017; 46(6):740-5.

5 Murase JE, Chan KK, Garite TJ, Cooper DM, Weinstein GD. Hormonal effect on psoriasis in pregnancy and post partum. Arch Dermatol 2005;141:601-6.

6 Tincani A., Taylor P., Fischer-Betz R., Ecoffet C., Chakravarty E. Fears and misconceptions of women with chronic rheumatic diseases on their journey to motherhood. Annals of the rheumatic diseases 2018; 77(Suppl 2):866, abs FRI0693 EULAR 2018 Annual European Congress of Rheumatology; 13-16 June 2018; Amsterdam, Netherlands.

7 Murase J., Simone C. de, Fischer-Betz R., Ecoffet C., Tincani A. Fears and misconceptions of women with chronic inflammatory diseases on their journey of motherhood J Am Acad Dermatol 2019; 81(Suppl 1):AB65 Annual Meeting of the American Academy of Dermatology (AAD), 1-5 March 2019; Washington, D.C., USA.

8 Robinson MK et al. Sclerostin: how human mutations have helped reveal a new target for the treatment of osteoporosis. Drug Discov Today. 2013 18(13-14): 637-43.

9 Brunkow ME, et al. Bone dysplasia sclerosteosis results from loss of the SOST gene product, a novel cysteine knot-containing protein Am J Hum Genet 2001;68: 577-589.

10 Padhi D, et al. Multiple doses of sclerostin antibody romosozumab in healthy men and postmenopausal women with low bone mass: a randomized, double-blind, placebo-controlled study J Clin Pharmacol 2014;54(2):168-178.

11 EU SmPC, Evenity®, accessed on 14 February 2020.

12 Robinson MK et al. Sclerostin: how human mutations have helped reveal a new target for the treatment of osteoporosis. Drug Discov Today. 2013 18(13-14): 637-43.

13 Brunkow ME, et al. Bone dysplasia sclerosteosis results from loss of the SOST gene product, a novel cysteine knot-containing protein Am J Hum Genet 2001;68: 577-589.

14 Padhi D, et al. Multiple doses of sclerostin antibody romosozumab in healthy men and postmenopausal women with low bone mass: a randomized, double-blind, placebo-controlled study J Clin Pharmacol 2014;54(2):168-178.

15 Saag KG, et al. Romosozumab or alendronate for fracture prevention in women with osteoporosis. N Engl J Med 2017;377:1417-1427.

16 Cosman F, et al. Romosozumab treatment in postmenopausal women with osteoporosis. N Engl J Med 2016;375:1532-1543.

17 EU SmPC, Evenity®, accessed on 14 February 2020.
USPI, Evenity®, accessed on 14 February 2020.