Building value for patients, partners and shareholders
Our mission at Sobi is to develop and deliver innovative therapies that improve the lives of patients with rare diseases and, in doing so, to create value for our partners, society and shareholders.
Patient and Customer Centric commercialisation
At Sobi we believe that an integrated approach to delivering therapies is essential to ensuring that patients will benefit in practice from the innovative medicines we develop. The patient journey, from diagnosis and treatment, to on-going disease management and long-term outcomes is at the centre of how we prioritise our capabilities and investments. Our objective is to identify where we can add value for patients and their physicians, by reducing the time to diagnosis, improving diagnostic accuracy, developing and delivering monitoring tools, understanding the barriers to consistent health outcomes, and working with physicians and other healthcare providers to provide sustainable solutions for patients in the real world. By creating and maintaining dialogue with this community, and also with governments and payers, we seek to ensure that our treatments are delivered in a sustainable way. At Sobi we refer to this as a Patient and Customer Centric approach to commercialisation.
Our business focus
Our key Therapeutic Areas are Inflammation and Genetic diseases, with a growing focus on Haemophilia and Neonatology. Kineret®, a biological inhibitor of Interleukin-1 (IL-1) for Rheumatoid Arthritis (RA) and for Neonatal-Onset Multisystem Inflammatory Disorder (NOMID) is the focus in Inflammation. Orfadin®, Ammonaps® and Ammonul® are the focus in Genetic diseases. Our Development Programmes include two longlasting recombinant coagulation factors for the treatment of haemophilia A and B which are being developed in partnership with Biogen Idec, and Kiobrina®, an oral enzyme replacement therapy for preterm infants, which we hope will form the basis of our emerging neonatology Therapeutic Area. These activities are supported by a diversified and growing base business. Our Partner Products portfolio is composed of products which we commercialise on behalf of speciality and rare disease partner companies. We represent our partners in European markets with the same care, clinical acumen and dedication to patient service as we do for our proprietary products. Finally, we are the longstanding manufacturer of ReFacto AF, a recombinant factor VIII, sold by Pfizer for the treatment of haemophilia A.
Late-stage Development Programmes
2012 was an important and exciting year in the evolution of our late-stage development pipeline. In the second half of the year together with our partner Biogen Idec, we announced positive results from two separate phase III clinical studies which evaluated two new long-lasting recombinant coagulation factors in people with haemophilia A and haemophilia B, rare inherited disorders that impair blood coagulation. There is a significant demand amongst patients and healthcare professionals for long-lasting agents for the treatment of haemophilia A and haemophilia B. The findings from these studies may represent a major step forward for the haemophilia community and a significant commercial opportunity for the company.
We are also developing recombinant human bile salt stimulating stable lipase (BSSL) to improve growth in preterm infants. BSSL is essential for normal growth in the neonatal period, and is an enzyme naturally secreted in breast milk. Many babies who are born prematurely lack access to this enzyme. Kiobrina – an investigational orally delivered, enzyme replacement therapy may become an important therapy for premature infants, if they are not fed with fresh breast milk, by increasing growth and thereby reducing short- and long-term health complications caused by prematurity. Kiobrina will possibly reduce length of stay in the Neonatal Intensive Care Unit (NICU) in the first instance and, for the longer term, reduce complication rates for these babies and their families.
Foundations of future success
As anticipated at the outset of the year, 2012 was a crucial period in our development. We have focused on building a sustainable operating platform via the growth in key Therapeutic Areas, via a new set of partnerships in our Partner Product portfolio, and by improving key operating metrics such as our gross margin and cash flow from operations. The positive read-out from the haemophilia A and haemophilia B studies, combined with our on-going work with Kiobrina and the positive developments to potentially apply Kineret to specific paediatric inflammatory syndromes, provide significant momentum for building on this platform in the future.
Thank you for your support and interest in our work.
Geoffrey McDonough
President and CEO
Read the full comment in the Annual Report for 2012.