Swedish Orphan Biovitrum AB (publ) Stock

Swedish Orphan Biovitrum AB (publ) operates as an international pharmaceutical company that focuses on rare diseases.

The company operates throughout the entire value chain, from early research, over to clinical development and into the commercial market. It operates through two business areas: Haemophilia and Specialty Care. The company’s research and product portfolio is primarily focused on haemophilia and specialty care, including inflammation and genetic and metabolic diseases. The company’s products Elocta and Alprolix are approved in Europe and the Middle East for the treatment of haemophilia A and haemophilia B respectively in children, adolescents and adults.

Elocta

Elocta is used to treat Alprolix to treat haemophilia B. Elocta (efmoroctocog alfa) is a recombinant clotting factor VIII therapy developed for haemophilia A with prolonged circulation in the body using Fc fusion technology.

Elocta is approved for the treatment of haemophilia A in adults and children of all ages and marketed by the company in the EU, Iceland, Liechtenstein, Norway, Switzerland, Kuwait and Saudi Arabia. Eloctate is approved for the treatment of haemophilia A in the United States, Japan, Canada, Australia and other countries, and marketed by Bioverativ.

Alprolix

In 2016, the company and Bioverativ announced that the European Commission had approved Alprolix for the treatment of haemophilia B. In connection with the approval, the deposit was transferred to Bioverativ and offset against the liability.

Alprolix (eftrenonacog alfa) is a recombinant clotting factor therapy developed for haemophilia B using Fc fusion technology to prolong circulation in the body. Alprolix is approved for the treatment of haemophilia B in adults and children of all ages in the EU, Iceland, Liechtenstein, Norway, Switzerland, Kuwait and Saudi Arabia, where it is marketed by the company. Bioverativ holds the marketing rights for the United States, Canada, Japan, Australia, New Zealand, Brazil and other countries.

BIVV001 (rFVIIIFc-VWF-XTEN)

In 2014, the company decided to include the preclinical development programme for the potentially long-acting haemophilia A treatment BIVV001 (rFVIIIFc- VWF-XTEN) in the agreement with Bioverativ. Under the agreement between the company and Bioverativ. Bioverativ initiated phase 1/2 trial with next generation EHL product in haemophilia A, rFVIIIFc-VWF-XTEN (BIVV001).

BIVV002 (rFIXFc-XTEN)

In 2017, the company decided to include the preclinical development programme for the potentially long-acting haemophilia B treatment BIVV002 (rFIXFc-XTEN) in the agreement with Bioverativ.

The company’s product Kineret is approved in Canada for the treatment of NOMID. Orfadin once-daily dosing approved in the EU and the U.S. New Orfadin formulations approved in Saudi Arabia, Canada, Algeria and Tunisia. Orfadin is used for the treatment of hereditary tyrosinaemia type 1 (HT-1) and Kineret is used for the treatment of autoinflammatory conditions. Xiapex is approved for the treatment of Dupuytren’s contracture and Peyronie’s disease in the EU and marketed by the company on behalf of Endo Pharmaceuticals. The company also markets approximately 30 products on behalf of partners in Europe, the Middle East, North Africa and Russia.

The company and the specialty pharmaceutical company Endo International are partners for the commercialisation of Xiapex (collagenase clostridium histolyticum). The co many has the exclusive rights to commercialise Xiapex for the treatment of Dupuytren’s contracture and Peyronie’s disease in 71 Eurasian and African countries. The company 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.

Orfadin is a proprietary product, developed and marketed worldwide by the company. Orfadin is available in five dosage strengths: 2 mg, 5 mg, 10 mg, 20 mg capsules and 4 mg/ml oral suspension. 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 U.S. and Canada), and cryopyrin- 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 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.

SOBI003 is the company’s major advanced early-stage project. It is being developed for mucopolysaccharidosis type IIIA or Sanfilippo A syndrome, a progressive, life-threatening and rare inherited metabolic disorder affecting children from early age.

During 2017, SOBI003 was granted orphan status by the US Food & Drug Administration, adding to the previously granted orphan designation in the E.U. SOBI003 is preparing to enter phase 1/2 clinical studies during 2018, being tested on patients for the first time. SOBI003 has been developed in-house by the company.

Strategy

The company’s strategy builds on its strengths and aims to capitalise on the potential in haemophilia and the rare disease areas. The company’s strategy for growth is aimed at balancing this geographic footprint by expanding Specialty Care in other markets, including North America.

The key elements of the company’s strategy include driving Haemophilia commercial effectiveness and internationalization; developing specialty care and become preferred partner; taking leading position in Haemophilia in EMENAR and expanding U.S. business; and strengthening pipeline and building foundation for self-sustained research and development.