• The first targeted C3 therapy approved in Canada
  
• Approval based on results from head-to-head PEGASUS phase 3 study where Empaveli™ demonstrated superiority to eculizumab in improving hemoglobin levels¹
• On trial, transfusion avoidance was achieved by 85.4% patients in the Empaveli group versus 15.4% in the eculizumab group¹
  

WALTHAM, Mass., Dec. 09, 2022 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi^®) (STO:SOBI), announced today that Health Canada has approved Empaveli™ (pegcetacoplan), a complement inhibitor, for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to, or are intolerant of, a C5 inhibitor. Empaveli™ is a targeted C3 therapy designed to regulate the activity of the complement cascade, which can lead to the onset and progression of many serious diseases.

PNH is a rare, life-threatening hematologic disorder, acquired genetically, with numerous manifestations. PNH is characterized by the destruction of red blood cells (hemolysis) and release of free hemoglobin that can cause thrombosis and require frequent transfusions. Hemolysis can reduce quality of life by causing debilitating symptoms such as dyspnea, pain, erectile dysfunction and severe fatigue caused by anemia.^2-4 The global incidence of PNH is estimated to be 1-1.5 cases per million individuals and is estimated to impact fewer than 1,000 people across Canada.⁴ Despite reductions in hemolysis with C5 inhibitor treatment, approximately 70 percent of people with PNH treated with C5 inhibitors remain anemic, according to a retrospective study and a cross-sectional study.^5,6

“Today’s approval by Health Canada is a significant milestone for those living with PNH across Canada,” said Bob McLay, Vice President, General Manager of Sobi Canada. “With the addition of Empaveli, patients now have the added flexibility of a treatment option that has demonstrated superiority to the previous standard of care and can be self-administered, reducing the need to schedule infusions in a clinic and giving them back the gift of time.”

The approval is based on the results from the head-to-head PEGASUS phase 3 study, which evaluated the efficacy and safety of Empaveli compared to eculizumab at 16 weeks in adults with PNH who had persistent anemia despite treatment with eculizumab. The full safety and efficacy results were published in The New England Journal of Medicine in March 2021.¹

“Many of our PNH patients treated with C5 inhibitors remain anemic despite full terminal complement control. Some of them have persistent fatigue, reduced quality of life, and may require ongoing transfusions” said Christopher Patriquin, hematologist at Toronto General Hospital and an investigator on the PEGASUS trial. “Today’s approval represents an important new treatment option for Canadian PNH patients who may have an inadequate response to currently available therapies.”

About Empaveli™
Empaveli (pegcetacoplan) is a targeted C3 therapy designed to regulate activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Empaveli is approved in the United States and the United Arab Emirates for the treatment of adults with PNH. In Australia, Empaveli is approved for the treatment of adult patients with PNH who have an inadequate response to, or are intolerant of, a C5 inhibitor. Aspaveli, the brand name in the EU and the UK, is approved as an orphan drug for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who are anemic after treatment with a C5 inhibitor for at least three months. The therapy is also being investigated in several other rare diseases across hematology, nephrology, and neurology.

About the Sobi and Apellis Collaboration
Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-US commercialization rights for systemic pegcetacoplan, and Apellis has exclusive US commercialization rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.

References
1. Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. DOI: 10.1056/NEJMoa2029073.
2. Borowitz MJ, et al. Cytometry B Clin Cytom. 2010;78(4):211-230.
3. National Cancer Institute. Hemolysis. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/hemolysis. Accessed November 4, 2022.
4. Hill A, et al. Nat Rev Dis Primers. 2017;3:17028.
5. McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130:3471.
6. Dingli D, Matos JE, Lehrhaupt K, Krishnan S, Yeh M, Fishman J, Sarda SP, Baver SB (2022) The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol 101(2):251–263

About Sobi North America
As the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobi’s vision of providing access to innovative treatments that make a significant difference in the lives of individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and rare disease. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at www.sobi-northamerica.com or at www.sobi.com.

About Sobi^®
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,600 employees across Europe, North America, the Middle East and Asia. In 2021, revenue amounted to SEK 15.5 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, sobi-northamerica.com, LinkedIn and YouTube.

Contacts
To contact the Sobi Investor Relations Team, please email thomas.kudsklarsen@sobi.com.
For Sobi media contacts, please email NACommunications@sobi.com or call +1 289-291-3852.