Specialty Care Business area Growth in the newly formed business area Specialty Care reached 5 per cent in 2017. Ambitions for the unit are higher than this, as Sobi explores several opportunities for growth that build on proven capabilities in bringing products to patients in complex markets. The business area Specialty Care was formed during 2017 by merging Sobi operations in Partner Products, Genetics & Metabolism and Inflammation. The focus is on rare diseases and niche indications, and the products aim to make a significant difference for patients. The unit has a distribution network across EMENAR and North America used to sell both proprietary and partner products. Sales in 2017 amounted to SEK 2,829 M. The unit s largest products are Kineret and Orfadin, representing 40 and 30 per cent of sales respectively, followed by Xiapex representing 6 per cent. The port folio currently consists of 37 products. We are actively seeking new products for our Specialty Care portfolio, to enhance efficiency and improve our ability to meet the evolving needs of patients. To make better use of our capabilities, we are also seeking to attain more extensive product rights through external opportunities. Based on more than 25 years of experience, we offer partners strong collaborative skills and well-developed processes that efficiently manage partnerships covering several markets with efficient distribution and extensive market knowledge. Our organisation has a competitive advantage thanks to our ability to launch products 22
Direct sales Other sales areas rapidly in both larger and smaller markets, offering our partners an efficient path to market. We have a thorough understanding of local regulatory environments and the drivers among payers and healthcare providers in various markets. Partnerships span over many years: apart from strategies for regulatory approval, pricing and reimbursement, they may include launch preparations, marketing activities, tender management and efficient logistics. We assume a comprehensive responsibility for products to ensure that medical needs are met in the best possible manner. Our current organisation has offices in 19 countries, managing products in over 70 countries. Markets are evolving in many countries as their standards of living rise. We are looking to match increased demand with a greater market presence, particularly in more populous countries. Better meeting patient needs In recent years, we have employed active strategies to address patient needs even Long experience of commercialising products Sobi has extensive experience of launching and commercialising products in Europe. Kineret, Orfadin, Xiapex and 34 other products can be found in 70 markets in the EMENAR region. Kineret, Orfadin and Kepivance are also made available by Sobi in North America. better. This can entail product development and innovation driven by medical needs. New medical needs may arise when people with certain rare diseases reach adolescence and adulthood for the first time thanks to the treatments we provide. Sobi also supports rare-disease communities in various ways, for example through ambassador programmes, financial support to bridge reimbursement, or educational tools for healthcare providers, children and adults. Financial performance of the business area 2,829 SEK M Total revenue for the Specialty Care business unit was SEK 2,829 M, up 5 per cent from SEK 2,695 M in 2016. Understanding the rare disease space also gives us capabilities to leverage in specialty-care and niche indications Norbert Oppitz, Head of Specialty Care THIS IS SOBI BUSINESS AREAS INNOVATION SUSTAINABILITY HISTORY & FINANCIAL OVERVIEW REPORTING CORPORATE GOVERNANCE SUSTAINABILITY MANAGEMENT 23
The Orfadin Ambassador programme in the US allows patients and caregivers to connect with other people who have experience of living with HT-1. For a patient population of 1000 people worldwide, this is a rare opportunity. Orfadin Before Orfadin was first launched in 2002, few patients living with hereditary tyrosinaemia type 1 (HT-1) would celebrate their second birthday. Today, thanks to improved newborn screening, effective treatment and dietary management, we are seeing patients with HT-1 reach an age where they are starting their own families. Sobi has worked closely with the HT-1 community to help people to manage their disease not just at diagnosis but throughout their lives. We made a commitment to make treatment as simple as possible, and in recent years we have developed new formulations to achieve this goal on several fronts. Firstly, as new-born screening for HT-1 has improved and patients are being identified at birth, we developed a liquid formulation of Orfadin that makes it easy to administer a precise dose to infants and young children. Secondly, because patients are growing up and becoming adults, we have developed a 20 mg capsule, allowing patients to reduce the number of capsules they take each day. Lastly, we have conducted studies allowing Orfadin to be taken as a once-daily dosing option for patients five years of age and older. These new dosing options are unique to Sobi and a result of our insights concerning the medical needs of these patients. In 2017, competition entered the nitisinone market in the US, Canada and Europe in the form of generic treatments. Our commitment to the community and our understanding of ORFADIN patient needs have proven valuable not only to HT-1 patients but also to Sobi. Despite the new generics, Orfadin sales continued to increase during 2017, driven by the launch of the new formulations and comprehensive patient support services. People with HT-1 are unable to break down an amino acid called tyrosine. Toxic byproducts are formed and accumulate in the body, which can cause liver, renal and neurological complications. In the most common form of the disease, symptoms arise within the first six months of the child s life. Orfadin (nitisinone) blocks the breakdown of tyrosine, thereby reducing the amount of toxic tyrosine by-products in the body. Patients must maintain a special diet in combination with Orfadin treatment as tyrosine is not adequately broken down. Orfadin is a proprietary product, developed and marketed globally by Sobi. Orfadin is available in five dosage strengths: 2 mg, 5 mg, 10 mg, 20 mg capsules and 4 mg/ml oral suspension. For full European prescribing information, please visit the EMA website. For full US prescribing information please see orfadin.com 24
Kineret Kineret is a biologic that can reduce the activity of interleukin-1 (IL-1), a key mediator of inflammation in autoinflammatory and autoimmune diseases. Kineret blocks the biological activity of IL-1a and IL-1b by binding to the interleukin-1 type 1 receptor (IL-1RI), expressed in a variety of tissues and organs, and thereby blocking interleukin-1 (IL-1) signalling. This signal blockade helps manage excess levels of IL-1 in the body, and consequently, inflammation and other symptoms. Kineret has a well-characterised safety profile, a quick onset of action and a short half-life. In 2017, Sobi started clinical trials for acute gout (phase 2) and Still s disease, including SJIA and adult onset Still s disease (AOSD) (phase 3). The Still s study (anastills), is required for approval in the US market. Developing Kineret s market potential in the US In 2014, the US team decided to take a closer look at how we could improve the Kineret business from a relatively flat business to a growing and thriving one. Sobi in North America was established in 2012 and the full rights for Kineret were bought in 2013. At that time there was little understanding of patient needs: half of the people prescribed the drug did not even start treatment due to reimbursement issues. Making matters worse, half of those who actually started dropped out during the first two months. We set out to better understand the patients and the market, and then we leveraged that understanding to bring about change, says Rami Levin, Head of Sobi North America. Our goals were twofold to remove the barriers that were preventing patients from starting treatment, and to understand and address treatment challenges so that patients could stay on treatment. To allow more patients to access the treatment, Sobi applied a rare-disease approach, addressing doctors who were familiar with the treatment and working together with them to identify appropriate patients. Sobi also invested in supporting patients through the very complex In 2013, at the time we received the CAPS (cryo pyrin-associated periodic syndrome) indication in Europe, we made a fundamental revision of our strategy for Kineret. A focused and tailored patient and customer-centric organisation was formed to meet market needs in the best way possible. Listening to healthcare providers and patients has helped us better meet their needs and take an important role in the market, one which has also resulted in steadily increasing product sales. Increasing interest in and understanding of the IL-1 field is supporting the growth of Kineret. Our work with Kineret in the US is described below. r eimbursement process and launched the Kineret On Track patient support programme, which includes nurse support, training on how to inject the drug, assistance in applying for reimbursement, and financing until reimbursement is received. The changes Sobi enacted increased both retention and compliance for Kineret by about 15 percentage points each. I cherish the fact that we have strengthened our relationships with the rheumatoid arthritis and NOMID communities. And I am also proud that we so strongly have contributed to the growth of Kineret, says Rami. Five-year global Kineret sales 1 MSEK 1 200 1 000 800 600 400 200 0 2013 2014 2015 2016 2017 1. Sales in the US and EMENAR follow the same trend. KINERET Kineret (anakinra) is approved for the treatment of rheumatoid arthritis (RA) in adults, neonatal-onset multisystem inflammatory disease (NOMID) in children and adults (in the US and Canada), and cryo pyrin-associated periodic syndrome (CAPS) in adult patients, and in children from eight months and older (in the EU). It is also approved in Australia for the treatment of RA, CAPS and also for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in patients aged two years and above who have failed to respond adequately to non-biological disease modifying anti-rheumatic drugs, and in Israel for RA and CAPS. For full European prescribing information, please visit the EMA website. For full US prescribing information please see kineretrx.com. We set out to better understand the patients and the market, and then use that understanding to bring about change Rami Levin, Head of North America THIS IS SOBI BUSINESS AREAS INNOVATION SUSTAINABILITY HISTORY & FINANCIAL OVERVIEW REPORTING CORPORATE GOVERNANCE SUSTAINABILITY MANAGEMENT 25
Xiapex Xiapex was first granted marketing authorisation by the European Commission for the treatment of the disabling hand condition Dupuytren s contracture in 2011. In 2015, the treatment was approved in Europe for Peyronie s disease, a condition resulting in an abnormally curved penis. Since 2013, when we partnered with Auxilium, now Endo International, for Xiapex, sales of the product have grown by an average of 36 per cent per year (CAGR). In 2017, sales increased by 7 per cent to SEK 164 M with growth stemming from both indications During 2017, people in England with Dupuytren s contracture were given access to Xiapex on the National Health Service for the first time after a recommendation from the National Institute for Health and Care Excellence (NICE), offering an alternative to surgery. Dupuytren s contracture is believed to be more common than Peyronie s disease, and has less stigma attached. In 2017, we continued to raise awareness about Peyronie s disease, a hidden condition that is not spoken about publicly. More men were subsequently given access to treatment. XIAPEX Sobi and the specialty pharmaceutical company Endo International are partners for the commercialisation of Xiapex (collagenase clostridium histolyticum). Sobi has the exclusive rights to commercialise Xiapex for the treatment of Dupuytren s contracture and Peyronie s disease in 71 Eurasian and African countries. Sobi is the Marketing Authorisation Holder (MAH) for Xiapex in the 28 EU Member States, as well as Norway and Iceland. Xiaflex is the trade name for Xiapex used in the United States. Xiapex is used to treat Dupuytren s contracture and Peyronie s disease in adults. Dupuytren s contracture is a condition where one or more fingers are bent forwards toward the palm and cannot be fully straightened. Peyronie s disease is a condition in which men develop plaques of fibrous, scar-like tissue in their penis, causing it to become abnormally curved. For full prescribing information, please visit the EMA s website. Long experience from working with partners in a complex environment. 26