Svensk version All offices All contacts Home About Sobi Patients Products Pipeline Partnering Healthcare Professionals Investors & Media You are here: Start>About Sobi>Report by the CEO Who we are Quick facts Report by the CEO Rare Diseases Specialty Pharmaceuticals Market Operations Our Leadership Team Board of Directors Corporate Governance Corporate Responsibility Careers Contacts and addresses Sobi's history Related Information Financial Reports Presentations Products Development Pipeline Report by the CEO CEO Geoffrey McDonough Annual Report 2013 Pioneer in Rare Diseases 2013 was eventful and positive for Sobi. We made significant progress in many areas towards our goal of being a leading international speciality healthcare company dedicated to rare diseases. The operating business showed strong momentum and our pipeline programmes achieved several key milestones during the year. Progress in our operating business Revenues in 2013 increased by 13 per cent compared to the previous year, our gross margin improved by 5 percentage points to 59 per cent, and we strengthened our company financially through stable positive cash flow and improved operating profit. Our key Therapeutic Areas of Inflammation and Genetics & Metabolism continued their trajectory from 2012, expanding the business in both existing and new markets and growing revenues by 10 per cent. Kineret® was approved in Europe for a new adult and paediatric indication, Cryopyrin-Associated Periodic Syndromes (CAPS), shortly before year end, and we reached an agreement with our distributor for Orfadin® in the US to assume direct responsibility for Orfadin in the US, Canada and Latin America. These developments will continue to support the growth of these businesses. In addition, we have strengthened our Partner Products portfolio with four significant new partnership agreements: Auxilium Pharmaceuticals for the development and commercialisation of Xiapex®, a new generation biologic for the treatment of Dupuytren’s contracture, a connective tissue disorder. PharmaSwiss for marketing of Megace®, Monopril®, Cefzil® and Duricef® which are approved in the areas of oncology, cardiovascular and anti-inflammatory therapies. Exelixis to support the distribution and commercialisation of Cometriq® for the treatment of metastasised thyroid cancer within the EU and potentially also in other countries. Hyperion Therapeutics for the sole rights in seven countries in the Middle East for the distribution of a liquid form of Ravicti®, a drug to treat patients with Urea Cycle Disorders (UCD). Finally, our ReFacto® manufacturing business was stable in 2013 with growth of 9 per cent. Our commercial progress forms the platform for the launch of our pipeline. Progress in our Development Programmes In addition to the launch of Kineret for the treatment of Neonatal Onset Multisystem Inflammatory Disease (NOMID) in the US and the CAPS approval in the EU for children and adults, we also signed an agreement with Amgen giving us the full rights to develop and commercialise Kineret for all therapeutic indications. This supports our long-term goal of exploring the full potential of Kineret and we are now working on firming up our plans in this area. We are developing a liquid formulation of Orfadin to facilitate the ease and accuracy of administration for Orfadin in paediatric patients. In 2013 we filed registration applications in the EU and plan to do so in the US in 2014. We expect a decision in the EU in 2014 and in 2015 in the US. Sobi is a founding member of the European Commission funded DevelopAKUre project to develop nitisinone as a potential treatment of the chronic debilitating disease Alkaptonuria (AKU). The programme has progressed during the year with the completion of SONIA 1, an international, randomised dose-finding open-label study. The DevelopAKUre collaboration was chosen from over 200 nominations, to win the RARE Champions of Hope Award for Collaborations in Science. We also acquired additional rights for Kepivance® which allows us to explore a potential new indication based on the results from two phase 3 studies conducted by Amgen. These studies show that Kepivance has the potential to reduce the incidence and duration of severe oral mucositis in patients undergoing treatment for advanced head and neck cancer. This Kepivance programme was presented for the first time at our Capital Market Day events in Stockholm and New York in November 2013. Before year end, SOBI002 began phase 1 clinical trials. SOBI002 is a novel small biologic molecule that works as a potent and selective inhibitor of complement protein C5, a key protein in human immunological and inflammatory processes and central to a number of important diseases. In the course of 2013 we completed enrolment of the LAIF trial (Lipase Added to Infant Feeding), our phase 3 study investigating the effect of rhBSSL (recombinant human Bile Salt Stimulated Lipase) on the growth and development of preterm infants born before 32 weeks gestation. In Q1 2014 we learned that rhBSSL did not meet its primary endpoint in this trial – growth velocity measured after four weeks of treatment showed no statistically significant improvement compared to placebo. We expect to evaluate further data from LAIF in the course of 2014, and to assess the financial impact of the topline results in Q2 2014. Solid foundation for Haemophilia During 2013 we laid a solid foundation from which to begin commercialisation of our two long-lasting haemophilia drug candidates (rFVIIIFc and rFIXFc), developed together with our partner Biogen Idec. We reinforced our Haemophilia senior medical and commercial teams by recruiting several highly experienced professionals. Biogen Idec expects to launch both products in the US in 2014, and as of Q1 2014 rFIXFc (AlprolixTM) had been approved in both Canada and in the United States. At the end of 2013 we had two ongoing paediatric phase 3 studies with our haemophilia drug candidates in children under the age of 12. Interim results from the studies, which were presented in December at the Annual Meeting of the American Society of Hematology were consistent with pharmacokinetic results in adults and adolescents; prolonged half-life compared to the trial patients’ previous treatment regimens. Pending the outcome of the paediatric studies, we plan to file for marketing authorisation of the products in our territories i.e. Europe, Russia, North Africa and the Middle East. Priorities in 2014 Looking forward, we will continue to focus on the growth and geographical expansion of our base business, to continuously improve the efficiency of our business model. In addition, we aim to continue our work as a pioneer in rare diseases by advancing our programmes to address the needs of children. Thanks to our employees, our partners and shareholders for your support and contribution to our successes during the year. Geoffrey McDonough President and CEO To full Annual Report 2013 Updated 2012-07-14 Tip A Friend Print Contact Swedish Orphan Biovitrum AB (publ) SE-112 76 Stockholm, Sweden Phone: +46 8 697 20 00 Reg.No. 556038-9321 © Swedish Orphan Biovitrum AB, 2013 Quick Links Terms of use & cookies All contacts Follow us Press releases News Financial reports