2014 Full Year Results Investor Factbook 2014 Full Year Results 1 Our Company ....................................................................................................................................................................................... 3 Vision 3 Strategy 3 Board Members 4 Executive Committee 4 Employees 5 Locations 5 UCB: A Solid Heritage 6 Financials ............................................................................................................................................................................................. 7 Key Financials 7 Net sales 7 Net sales by region and product 8 R & D Expenses (€ m) 8 Shareholder structure and free-float by region 9 Target Therapy Areas ........................................................................................................................................................................ 10 Disease Areas .................................................................................................................................................................................... 11 Epilepsy 11 Parkinson’s disease 11 Restless legs syndrome 11 Rheumatoid arthritis 11 Crohn’s disease 12 Osteoporosis 13 Systemic lupus erythematosus 13 Core Medicines: Cimzia®, Vimpat®, Neupro® (CVN)........................................................................................................................ 14 Cimzia® 14 Vimpat® 15 Neupro® 15 Development Pipeline Information ..................................................................................................................................................... 16 Partners ............................................................................................................................................................................................. 17 Drug Development Process ............................................................................................................................................................... 18 Research & Development .................................................................................................................................................................. 19 2015 Financial Calendar .................................................................................................................................................................... 20 Disclaimer and Safe Harbor ............................................................................................................................................................... 20 Contacts ............................................................................................................................................................................................. 20 Investor FactBook FY2014 2 Our Company Vision UCB aspires to be the patient-centric global biopharmaceutical leader transforming the lives of people living with severe diseases. Strategy Since 2004, our strategy has been focused on delivering superior and sustainable solutions to people living with severe diseases, targeting two areas: neurological diseases and diseases of the immune system. In each of these areas, we constantly strive to obtain better patient and healthcare consumer insights while moving science forward to create unique solutions and efficient ways to deliver them. In 2015, we continue to focus on our strategic growth priorities: ® ® ® Grow Cimzia , Vimpat , and Neupro Advance and prepare launch of the next wave of promising new solutions: brivaracetam, epratuzumab, and romosozumab; Deliver breakthrough medicines Reach competitive profitability; Constantly ensure quality and compliance with laws and regulations and; Develop passionately engaged colleagues and business partners. These priorities are underpinned by constantly ensuring quality and compliance with laws and regulations, and developing passionately engaged colleagues and business partners. In our early stage pipeline, we focus on potential breakthroughs (i.e. new medicines that can transform patients’ lives and UCB) that offer true differentiation and systematically discontinue projects that do not. The productivity, wealth and quality of our pipeline – internal and external – allow us to make these choices. Our established research strategy for the breakthrough focuses on first or second-in class innovative approaches, prioritizing projects that have a clear proof of concept and clear end points. We focus our manufacturing network on R&D scale-up until launch. Where appropriate, we augment our internal capabilities using strategic partnerships, both for large and small molecules, in the commercial phase of the product, while securing cost-effective supply. As for our commercial strategy, we have our own presence covering specialist physicians, payers and patient groups, in North America, Europe and major emerging markets. In all areas, we continue to learn from best-in-class companies outside the biopharma industry in areas such as innovation, customer insights, cost management and activity-based management. As a result of our strategy, UCB aims to exceed the biopharma industry’s compounded growth. We also expect to invest more than our peers in R&D proportionally to our sales, while accelerating towards peer profitability through reallocation of resources to best impact. With no major patent expiry for many years and with three new core medicines fueling our growth, plus a rich pipeline and cutting edge science, UCB is now poised to build a strong global presence in neurology and immunology – and to deliver significant returns to shareholders. Thanks to the current performance and growth of our core medicines, we ® ® ® confirm our ambition to reach more than 1.5 million patients with Cimzia , Vimpat and Neupro , representing peak sales of at least € 3.1 billion in the second half of the decade. Investor FactBook FY2014 3 Board Members The Board of Directors is made up of 12 members: First appointed as End of term Director of office Independent Audit Scientific Director Committee Committee Gerhard Mayr, Chairman 2005 2015 x x Evelyn du Monceau, Vice Chair 1984 2015 Jean-Christophe Tellier, CEO & Executive Director 2014 2018 Albrecht De Graeve 2010 2017 Arnoud de Pret 2005 2015 Harriet Edelman 2012 2016 x Kay Davies 2014 2018 x x Charles-Antoine Janssen 2012 2016 Jean-Pierre Kinet 2008 2015 x x Tom McKillop 2009 2016 x Norman J. Ornstein 2008 2015 x Cédric van Rijckevorsel 2014 2018 Governance, Nomination & Compensation Committee x x x x x x For additional information about UCB Board members and the Board Committees, please refer to: http://www.ucb.com/investors/Governance/Corporate-governance Executive Committee The Executive Committee is made up of 10 members Jean-Christophe Tellier, CEO and Chair of the Executive Committee Joined UCB Appointed June 2011 July. 2011 Emmanuel Caeymaex, Immunology Patient Value Unit Head Mar 1994 Feb. 2015 Fabrice Enderlin, Chief Talent Officer Jan. 2008 March 2008 Ismail Kola, New Medicines Patient Value Unit Head and Chief Scientific Officer Nov. 2009 Nov. 2009 Iris Löw-Friedrich, Chief Medical Officer Sept. 2006 March 2008 Mark McDade, Chief Operating Officer April 2008 Sept. 2008 Anna Richo, General Counsel Nov. 2012 Nov. 2012 Mar 2015 Mar 2015 Sept. 2006 Jan. 2007 Oct. 2010 Feb. 2015 Bharat Tewarie, Chief Marketing Officer Detlef Thielgen, Chief Financial Officer Jeff Wren, Neurology Patient Value Unit Head For additional information about UCB Executive Committee, please refer to: http://www.ucb.com/investors/Governance/Corporate-governance Investor FactBook FY2014 4 Employees (headcount) Employees by function (2014) Total: 8 684 Employees by region (2014) Total: 8 684 Total Employee Headcount (FY) 9 048 8 927 8 732 8 684 2013 2014 8 506 2010 2011 2012 Headquartered in Brussels (Belgium), UCB is present in 36 countries. Investor FactBook FY2014 5 UCB: A Solid Heritage 1990s: approval of Keppra®, a novel anti-epileptic 1928: Emmanuel Janssen establishes UCB in Brussels 2006: UCB acquires German pharma company Schwarz Pharma Today 2005: UCB divests non-pharma business 1980s: UCB registers its novel antihistamine Zyrtec® 2004: UCB acquires British biotech company Celltech 1936: UCB enters the United States 1928 1936 Chemical Group Investor FactBook FY2014 1980s 1990s Primary Care Pharma 2004 Launch of new medicines 2005 2006 2008-2012 Today Specialty Bio-Pharma Focus: CNS + immunology 6 Financials Key Financials 2010 € million 2011 2012 2013 (restated) (restated) (restated) 2014 Revenue 3 218 3 246 3 462 3 133 3 344 Net sales 2 786 2 876 3 070 2 795 2 938 705 778 861 886 928 22% 24% 25% 28% 28% Recurring EBIT (REBIT) 467 439 444 297 379 Recurring EBITDA 731 687 684 536 609 23% 21% 20% 17% 18% Net profit including non-controlling interests 103 238 245 145 199 Core EPS (€ per non-diluted share) 1.99 1.91 2.10 1.24 1.69 1 525 1 548 1 766 1 998 1 611 Ratio net debt / REBITDA 2.09 2.25 2.58 3.73 2.65 Equity ratio 51% 51% 49% 44% 48% 506 292 355 288 512 78 137 221 344 161 R&D expenses Ratio R&D expenses / revenue Ratio REBITDA / revenue Net debt Cash flow from operating activities Capital expenditure (including intangible assets) Mature Products Net Sales FY2014 665 Keppra® 712 163 Zyrtec® 204 Total mature products sales € 1 470 million (-9%)* 96 Xyzal® 114 55 Nootropil® 58 433 Other 482 0 100 (€ million) 200 300 400 2014 500 600 700 800 2013 *Adjusted for product divestitures (-2%) Investor FactBook FY2014 7 Net sales by region and product 2013 net sales - geographical area 2014 net sales - geographical area France 6% France 5% North America 37% North America 39% Germany 8% Germany 8% Italy 5% Italy 5% Spain 5% Spain 5% RoW other 4% Emerging markets (BRICMT) 11% Europe other Japan 12% 8% RoW other 4% U.K. & Ireland 4% Emerging markets (BRICMT) 11% 2013 net sales - by product therapeutic area (€ million) Neupro®, 182 Keppra®, 665 Immunolo gy Allergy, 318 Vimpat®, 411 U.K. & Ireland 4% 2014 net sales - by product / therapeutic area (€ million) Cimzia®, 594 Keppra®, 712 Europe other Japan 12% 7% Cimzia® , 797 Neupro®, 200 Other, 546 Immunolo gy Allergy, 259 Vimpat®, 471 Other, 530 CNS other, 16 CNS other, 984 Ratio gross profit / revenue 69% 69% R & D Expenses (€ m) 69% 28% 28% 24% 22% 25% 69% 67% 3 218 3 246 3 462 3 133 3 344 705 778 861 886 926 2010 2011 restated 2012 restated 2013 2014 2010 2011 restated 2012 restated 2013 2014 Revenue Investor FactBook FY2014 Margin R & D expenses R&D expenses as % of revenue 8 Share price history (Jan 1, 2014 – Dec 31, 2014) (rebased to 100) 1000 stock performance 750 100 500 50 250 - daily volume in mn of shares 150 0 Jan-14 Feb-14 Mar-14 Apr-14 Volume May-14 UCB Jun-14 Jul-14 Aug-14 MSCI EU Pharma Sep-14 Oct-14 Nov-14 Dec-14 MSCI US Pharma & Biotech Shareholder structure and free-float by region (January 2015) th Since January 5 2015, UCB’s capital amounts to € 583 516 974, divided in 194 505 658 ordinary shares with no nominal value. UCB’s main shareholder is Financière de Tubize S.A. (34.12%), a company listed on Euronext Brussels. “Free float” (64%) investors by region Investor FactBook FY2014 9 Target Therapy Areas UCB focuses on therapy innovation for people living with severe diseases of the central nervous system and in immunology. CNS1 The central nervous system (CNS) controls most functions of the body and mind. It consists of two parts: the brain and the spinal cord. The brain is the center of our thoughts, the interpreter of our external environment, and the origin of control over body movement. Like a central computer, it interprets information from our eyes (sight), ears (sound), nose (smell), tongue (taste), and skin (touch), as well as from internal organs such as the stomach. The spinal cord is the highway for communication between the body and the brain. When the spinal cord is injured, the exchange of information between the brain and other parts of the body is disrupted. Immunology2 The immune system is a complex network designed to defend the body against attacks by foreign bodies. The word immunity comes from the Latin immunis, or “exempt.” Usually these attackers are bacteria, fungi or parasites. If the immune system loses the ability to distinguish between self and non-self (or foreign targets), then autoimmunity can occur, leading to diseases such as rheumatoid arthritis, inflammatory bowel disease or lupus. The immune system is made up of cells, tissues and organs, including T and B cells, lymph nodes, mucosal (lining) surfaces of the intestines, the spleen and tonsils, as well as chemical mediators such as cytokines (eg tumor necrosis factor or TNF) and receptors. The immune system has complex mechanisms of communication enabling it to recognize and defend the body against thousands of different foreign attackers. Immunology is a branch of biomedical science that covers the study of the immune system in all organisms. Immunology examines the physiological functioning of the immune system in states of both health and disease. 1. 2. Source: http://www.christopherreeve.org/site/c.ddJFKRNoFiG/b.4452157/k.3E9D/What_is_the_Central_Nervous_System.htm Adapted from http://www.niaid.nih.gov/topics/immunesystem/pages/whatisimmunesystem.aspx Investor FactBook FY2014 10 Disease Areas Epilepsy Epilepsy is excessive electrical activity in the nerve cells in the brain that leads to epileptic seizures. Seizures are brief episodes with a variety of symptoms ranging from strange sensations, emotions and behavior, jerking or stiffening of the limbs and body with muscle spasms and loss of consciousness. In partial seizures the abnormal electrical activity is confined to a part of the brain, while in generalized seizures the disturbance involves large parts on both sides of the brain. • Prevalence : • Incidence : • 1 2 5.2 million people 50.4 cases / 100 000 population (U.S. & EU) 1 Market size : USD 2.9 billion Parkinson’s disease Parkinson’s disease (PD) is a progressive disorder of the nervous system caused by a gradual loss of nerve cells in the brain that produce a chemical called dopamine. Symptoms of the disorder include motor symptoms such as tremors, stiffness, and slowing of movement. Additionally, non-motor symptoms are common including thinking difficulties, emotional changes, sleep problems, bladder problems, constipation, and sexual dysfunction. As the disease progresses, the increasing motor disability affects activities of daily living including washing, getting dressed, speaking, and writing. Treatments aim to restore dopamine levels and can help symptoms, but there is no cure for the disorder. Treatments include dopamine agonists (usually given early in the disease) and leva dopa (usually given later in the course of the disease). Surgery on certain regions of the brain are sometimes employed when drugs are not effective. • 3 Prevalence : • Incidence : • Market size : 3 4.1 – 4.6 million people 10 - 14 cases / 100 000 population (worldwide) 4 USD 2.3 billion Restless legs syndrome Restless legs syndrome (RLS) usually occurs deep within the lower part of the leg. It is characterized by a strong urge to move the legs and is accompanied by an extremely bothersome sensation in the legs. Symptoms become worse in the evening and during the night, preventing patients from falling asleep and making it impossible to have a restful sleep. Sleep can also be interrupted by involuntary leg movements. Although the exact cause of RLS still is unclear, dys-regulations in the endogenous dopaminergic system might play a role. For the majority of patients, for whom a genetic basis of their disease is obvious, dopamine-agonists are regarded as a first line treatment. For a smaller proportion of patients there might be secondary reasons involved, like iron deficit in the peripheral blood. RLS affects more women than men (roughly 2:1) and although it can affect all ages, usually the diagnosis is made in middle-aged people and the elderly. • Prevalence : • Incidence : 1 5 54.4 million people ~1 000 – 2 000 cases / 100 000 population (U.S. & EU) • Market size : 1. 2. 3. 4. 5. Decision Resources 2009 – 2013; 7 major markets include: France, Germany, Japan, Italy, Spain, U.K. and U.S. http://www.neurology.org/content/77/10/1005.full.html WHO Neurological Disorders Report , 2006 Decision Resources, July 2013 Szentkiraly 2011 1 Investor FactBook FY2014 USD 193 million 11 Rheumatoid arthritis Rheumatoid arthritis (RA) is a chronic, systemic disorder that causes the immune system, which usually fights infection, to rapidly attack the joints. The result is painful inflammation, known as arthritis, and joint damage that can be disabling. This slows patients down, and ultimately leads to a substantial loss of functioning and mobility that limits a patient’s ability to live their life. This impacts both patient’s physical abilities as well as their mental health and social functioning. Over 5 million people suffer from RA in the major seven markets. Women are three times more likely to be affected than men. Although RA can affect people of all ages, the onset of the disease usually occurs between 30-50 years of 1 age . • 2 Prevalence : • Incidence : • Market size : 3 5 million people ~20-50 cases / 100 000 population (U.S. & EU) 2 USD 10.3 billion Crohn’s disease Crohn’s disease is an idiopathic, chronic inflammatory process of the gastrointestinal (GI) tract that can involve any part of the gastrointestinal tract from the mouth to the anus. The effect appears to be due to the body’s immune system attacking healthy cells in the gastrointestinal tract, causing inflammation. Because it is caused by the immune system, CD is classified medically as an autoimmune disorder. This means that the body is producing antibodies against itself. Together with ulcerative colitis, CD belongs to the group of illnesses called inflammatory bowel disease (IBD). The lives of people with CD are frequently disrupted by flare-ups of the condition. Most common symptoms are: fever, abdominal pain, weight loss and diarrhea; which can lead to an urgent need to use the bathroom. Coupled with the fact that the condition is often diagnosed in young adults, a time in life when one is typically faced with major lifedecisions such as college, new jobs and relationships, CD limits a sufferer’s ability to lead a normal life. There is still some uncertainty about how CD is caused. A number of genetic and environmental factors are associated with it but their role is not clear. However, the disease is thought to be an abnormal response of the body’s immune system to bacteria found in the intestines. • Prevalence : • Incidence : • Market size : 1. 2. 3. 4. 5. Rindfleisch JA, Muller D. Am Fam Physician. 2005; 72: 1037-1047. Decision Resources (2011). The 7 major markets include: France, Germany, Japan, Italy, Spain, the UK and the US. Tobón GJ, Youinou P, Saraux A. J Autoimmun. 2010; 35: 10-14 Decision Resources 2012 World Journal of Gastroenterology 2012 4 5 918 000 people ~3-10 cases / 100 000 population (U.S. & EU) 4 Investor FactBook FY2014 USD 3.8 billion 12 Osteoporosis1,2,3 Osteoporosis affects many women after menopause and is a disease that weakens bones over time, making them thinner and more likely to break. Post-menopausal women with osteoporosis have a greater risk for breaking a bone. Such a break, or fracture, may be a life-changing event. About one in three women over age 50 will have an osteoporosis-related fracture, and once that happens, the chances of another are much higher. The International Osteoporosis Foundation urges governments worldwide to make osteoporosis a healthcare priority. • Prevalence : • Incidence : • Market size : 4 200+ million people 5 1.66 million people 6 USD 451 million Systemic lupus erythematosus Systemic lupus erythematosus (SLE), or lupus, is a chronic, autoimmune disease that can damage any part of the body, including skin, joints, and organs inside the body, resulting in inflammation and tissue damage. Immunological aberrations in SLE patients produce excessive amounts of antibodies directed against self, “auto-antibodies”. Chronic means that the signs and symptoms tend to last longer than several weeks and often for many years. It is a disease of flares (the symptoms worsen and you feel ill) and remissions (the symptoms improve and you feel better). Some patients experience a relatively benign disease with little medical intervention, while others can have a serious and aggressive progression that can lead to significant and potentially life-threatening damage to organs such as the kidneys, brain, heart and lungs. No cure for SLE yet exists. There is a 85% five-year survival rate. The morbidity and mortality of SLE results from tissue damage due to disease progression. However, people with non-organ threatening aspects of SLE can lead a full and normal life. The cause of SLE is unknown, but is thought to be multi-factorial with genetic, hormonal and environmental factors playing a role. • Diagnosed prevalence : • Incidence : • 1. 2. 3. 4. 5. 6. 7. 7 7 622 000 million people ~3.4 cases / 100 000 population (US & EU) 7 Market size : USD 910 million Osteoporosis Foundation. “What is Osteoporosis.” Accessed September 2013 from http://www.nof.org/articles/7. International National Osteoporosis Foundation.” Facts and Statistics.” Accessed September 2013 from http://www.iofbonehealth.org/factsstatistics#category-16. International Osteoporosis Foundation. "Osteoporosis in the European Union in 2008: Ten years of progress and ongoing challenges." Accessed September 2013 from http://www.iofbonehealth.org/osteoporosis-european-union-ten-years-progress-and-ongoing-challenges http://www.iofbonehealth.org/epidemiology http://www.who.int/nutrition/topics/5_population_nutrient/en/index25.html; denotes hip fractures only Decision Resources (2011). The 7 major markets include: France, Germany, Japan, Italy, Spain, the UK and the US. Pharmacor, Decision Resources, Nov 2013; 7 major markets (include: France, Germany, Japan, Italy, Spain, U.K. and U.S.) Investor FactBook FY2014 13 Core Medicines: Cimzia®, Vimpat®, Neupro® (CVN) ® ® ® Our “core medicines” are: Cimzia , Vimpat and Neupro , which we continue to grow through new indications and global launch roll-outs. 797 Cimzia® 594 471 Vimpat® Total core medicines sales € 1 468 million (+24%) 411 200 182 Neupro® 0 100 200 (€ million) 300 400 2014 500 600 700 800 900 2013 Cimzia® quarterly net sales Patient access: • • • • • • Rheumatoid arthritis: available in 48 countries, including U.S., EU, Japan Crohn’s disease: available in 9 countries, including U.S. Psoriatic arthritis: available in 38 countries, including U.S. & EU Axial spondyloarthritis / ankylosing spondylitis: available in 38 countries, including U.S. (AS only) & EU Juvenile idiopathic arthritis: Phase 3 clinical trial started March 2013 with results expected in H1 2016 ® Psoriasis: strategic partnership with Dermira for the exclusive rights to develop Cimzia in psoriasis in the U.S., Canada and the EU Investor FactBook FY2014 14 Vimpat® quarterly net sales Patient access: • Partial-onset seizures, adjunctive therapy: approved in 43 countries, including U.S. & EU • Partial-onset seizures - monotherapy (US): approved September 2014 • Partial-onset seizures - monotherapy (EU): Phase 3 clinical trial program on-going; results expected in Q4 2015 • Partial-onset seizures, adjunctive therapy in Asia: Phase 3 results (October 2014); submission to Japanese and Chinese authorities planned in 2015 • Partial-onset seizures - adjunctive therapy in children: Phase 3 clinical trial started in September 2013; results expected in 2017 • Phase 3 clinical trial in PGTCS adjunctive therapy to start in 2015 Neupro® quarterly net sales Patient access: • Parkinson’s disease: approved in 44 countries, including U.S., EU & Japan • Restless Legs Syndrome : approved in 14 countries, including U.S., EU & Japan • Parkinson’s disease in China: Phase 3 results (February 2015); submission planned in 2015 Investor FactBook FY2014 15 UCB’s Development Pipeline brivaracetam UCB is evaluating the potential of brivaracetam in the treatment of epilepsy as adjunctive therapy in partial onset seizures (POS). Brivaracetam is a novel high-affinity synaptic vesicle protein 2A (SV2A) ligand. The positive data from the most recent Phase 3 study demonstrated robust and clinically relevant seizure reduction in predominantly treatment resistant patients, and tolerability was consistent with previous brivaracetam trials. In January 2015, the New Drug Application (NDA) for the Food & Drug Administration (FDA) in the United States and the Marketing Authorization Application (MSS) for the European Medicines Agency (EMA) were both filed to review brivaracetam as adjunctive therapy for the treatment of partial-onset seizures in patients aged 16+ with epilepsy. epratuzumab Epratuzumab is a CD22-targeted monoclonal antibody under investigation for the treatment of SLE. CD22 is a B cell specific surface protein that influences B cells migration and activation. The product was licensed from Immunomedics. Under the agreement, UCB owns the rights and is responsible for the clinical development, and commercialization of epratuzumab in all autoimmune disorders including SLE. romosozumab1,2 Romosozumab is a bone-forming agent that inhibits sclerostin. It is currently being studied for its potential to reduce fracture risk in an extensive global Phase 3 program. This program includes two pivotal studies evaluating romosozumab against both placebo and active in more than 10,000 women with post-menopausal osteoporosis. An additional Phase 3 study began in June 2014 to compare the efficacy and safety of romosozumab in men with osteoporosis, with first results expected in H1 2016. Both Phase 3 programs are in collaboration with Amgen. 1. Partial onset seizures 2. ClinicalTrials.gov Accessed September 2013 from http://clinicaltrials.gov/ct2/show/NCT01631214?term=Romosozumab&rank 3. ClinicalTrials.gov Accessed September 2013 from http://clinicaltrials.gov/ct2/show/NCT01575834?term=AMG+785&rank=12 Investor FactBook FY2014 16 Partners UCB is committed to accessing assets and capabilities through strong, mutually-advantageous agreements with external organizations. We seek to find these agreements across our value chain. In drug discovery, we have research agreements with leading universities and research institutions in Asia, Europe and North America; in drug development, we have agreements with, amongst others, Amgen, Millenium and Wilex; in drug manufacturing, we partner with Boehringer Ingelheim, Lonza and Sandoz, amongst others; and in drug commercialization, we are partnering with AstraZeneca, GSK, Novartis, Otsuka, Pfizer and Sanofi Aventis, amongst others. • Amgen Inc.: A partnership aimed at the research, development and commercialization of romosozumab, an antibody which works against sclerostin, a protein discovered by UCB, for the treatment of bone diseases and disorders such as osteoporosis. • Astellas Pharma Inc. (“Astellas”): UCB entered into an agreement with Astellas in January 2012 to jointly ® develop and commercialize Cimzia for rheumatoid arthritis (RA) in Japan. Under this agreement, UCB manufactures and supplies the product for commercialization. Astellas manages the distribution exclusively, ® and both Astellas and UCB jointly develop and commercialize Cimzia in Japan. • Daiichi Sankyo: UCB and Daiichi Sankyo entered into a strategic partnership in November 2014 to jointly ® commercialize Vimpat for epilepsy patients in Japan. • Dermira: UCB and Dermira entered into a strategic collaboration in dermatology to broaden patient access to ® Cimzia . • Immunomedics Inc.: Immunomedics Inc. has granted to UCB the exclusive worldwide rights to develop, market and sell epratuzumab for all non-cancerous human diseases including autoimmune disease indications. • Otsuka Pharmaceuticals: UCB and Otsuka Pharmaceuticals have entered into collaboration agreements ® pertaining to the development, license and supply of Neupro in Japan, a development and commercialization ® contract relating to Keppra in Japan. • PRA: In 2011, UCB announced that it entered into a strategic partnership with PRA to drive UCB’s operational clinical development activities. The agreements are effective for all of UCB’s new clinical study programs on a global basis. • Parexel: UCB has entered into a strategic regulatory partnership with Parexel providing comprehensive lifecycle maintenance and other regulatory support for UCB’s legacy products. • Sanofi: UCB and Sanofi entered into a scientific and strategic collaboration for the discovery and development of innovative anti-inflammatory small molecules which have the potential to treat a wide range of immune-mediated diseases in areas such as gastroenterology and arthritis. Investor FactBook FY2014 17 Drug Development Process Year 1 2 3 4 5 6 7 8 9 10 11 12 13 Product registration & approval Marketing & launch Development A pre-marketing strategy may have been instigated as early as Phase I trials to ensure that the market's needs are incorporated into the new drug's overall development, but more usually during the later phases when clinical results are promoted at international symposia in order to develop an awareness amongst the medical community who will ultimately be prescribing the new product. A Sales force will be trained and will begin an intense sales and marketing campaign prior to launch. New drug application Phase IV trials When a product is considered safe and effective from Phase III trials, it must be authorized in each individual country before it can be marketed. All data generated about the molecule is collected and submitted to the regulatory authorities in the US (FDA), European Union (EMA) and Japan (PMDA) and other countries which may require their own national approvals. Phase IV trials are conducted after a new drug has been granted a license, approved and launched. In these studies, the new drug is prescribed in an everyday healthcare environment using a much larger group of participants (two to five thousand patients). This enables new treatment uses of the new drug to be developed, comparisons with other treatments for the same condition to be made, and determination of the clinical effectiveness of the new drug in a a wider variety of patient types, and more rare side effects, if any, may be detected. Clinical development Phase I Phase II Phase III Phase I trials are conducted primarily to determine how the new drug works in humans, its safety profile and to predict its dosage range. It typically involves between fifty and one hundred healthy volunteers. Phase II trials test for efficacy as well as safety and side effects in a group of between one hundred to three hundred patients with the condition for which the new drug is being developed. Phase III trials involved a much larger group of patients, between several hundred and several thousand, which will help determine if the new drug can be considered both safe and effective. It will usually involve a control group using standard treatment or a placebo as a comparison. Pre-clinical phase Manufacturing Pre-clinical dev. IND application M anufacturing Pre-clinical development Application for Investigational new Drug The manufacturing process for the new drug is initiated and developed to produce it in sufficient quantities for pre-clinical testing and clinical trial purposes. Pre-clinical development begins before clinical trials or testing in humans may begin and during which important safety and pharmacology data are collected. An application for an IND is made to the FDA, EMA and/or other regulatory agencies for permission to administer a new drug to humans in clinical trials. Research phase Lead discovery and lead optimization Research The initial molecules are further tested in a wider range of biochemical and other models in order to establish that the lead compounds have the potential to become a drug. Exploratory Research Hit identification, UCB SLAM The hypothesis is validated using biochemical methods and in vivo testing to ensure that the scientific approach is relevant to the disease of interest. The relevant biology is investigated and drug starting points identified. Investor FactBook FY2014 18 Research & Development Patient Value Units - New Medicines The goal of New Medicines is to build a prolific pipeline of best-for-patient (first and/or best-in-class molecules of maximum value to patients, physicians and payers) molecules that enables UCB to launch one new product every other year, by channeling exquisite science for industry-beating performance across six key priorities: 1. Deliver differentiated molecules 2. Increase success rates 3. Implement extreme networking and open innovation model 4. Enhance scientific excellence 5. Increase efficiency 6. Strategic asset partnering Patient Value Practices - Development / Medical Practices The mission of Development is to support the development of products with proven value to patients suffering from severe diseases. As the Patient Solution Company UCB focuses on patients and empowers Patient Value Practices teams of experts to drive their patient solutions in comprehensive missions along the value chain. Within the Patient Value Practices we strive for operational excellence in clinical development, medical affairs, regulatory affairs, project management, and leadership supporting PVPs within and beyond Development. In doing so, we aim to be a “best-in-class” development organization that brings innovative solutions to patients – minimizing the time to our global market and maximizing value and quality of our products to ensure successful patient access all over the globe. Two main research centers • Biologics pilot plant – Braine-l’Alleud, Belgium • Biologics R&D centre - Slough, U.K. World-class expertise in selected therapeutic areas and disease mechanisms. Connecting science in new ways - chemistry (small, chemically-derived molecules) and biology (large antibody-based molecules), to leverage the potential of these two disciplines. “Open innovation” model based on strong partnerships with other biotech companies and prominent universities. Investor FactBook FY2014 19 2015 Financial Calendar 9M Interim Report FY 2014 Results February April July October 27 30 30 29 3M Interim Report and AGM 2015 HY Results Disclaimer and Safe Harbor This FactBook contains forward-looking statements based on current plans, estimates and beliefs of management. All statements, other than statements of historical fact, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, political, regulatory or clinical results and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions which could cause actual results to differ materially from those that may be implied by such forward-looking statements contained in this FactBook. Important factors that could result in such differences include: changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, product liability claims, challenges to patent protection for products or product candidates, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws and hiring and retention of its employees. Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction. UCB is providing this information as of the date of this FactBook and expressly disclaims any duty to update any information contained in this FactBook, either to confirm the actual results or to report a change in its expectations. There is no guarantee that new product candidates in the pipeline will progress to product approval or that new indications for existing products will be developed and approved. Products or potential products which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences between the partners. Also, UCB or others could discover safety, side effects or manufacturing problems with its products after they are marketed. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement. Contacts Antje Witte VP Investor Relations Tel: +32 2 559 9414 E-mail: [email protected] www.ucb.com Investor FactBook FY2014 20
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