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                          Therapeutic Area by Lead Product

                          Study Phase by Lead Product

                          Company by Lead Product

                          Top Deals by Deal Size (USD bn)

                          01

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Under the licensing agreement, Sobi will hold the exclusive rights in countries outside the U.S., Canada to commercialize Tryngolza (olezarsen sodium) as a potential treatment for FCS.

                          Product Name : Tryngolza

                          Product Type : Oligonucleotide

                          Upfront Cash : Undisclosed

                          March 26, 2025

                          Lead Product(s) : Olezarsen Sodium

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Recipient : Ionis Pharmaceuticals

                          Deal Size : Undisclosed

                          Deal Type : Licensing Agreement

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                          02

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Gamifant (emapalumab) is an anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. Emapalumab is currently in Phase lll development for MAS.

                          Product Name : Gamifant

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          February 27, 2025

                          Lead Product(s) : Emapalumab

                          Therapeutic Area : Immunology

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Aspaveli (pegcetacoplan) is the first and only approved therapy for geographic atrophy. Now it is being evaluated for the treatment of C3 glomerulopathy & IC-MPGN.

                          Product Name : Aspaveli

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          February 20, 2025

                          Lead Product(s) : Pegcetacoplan

                          Therapeutic Area : Nephrology

                          Highest Development Status : Phase III

                          Sponsor : Apellis Pharmaceuticals

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Doptelet (avatrombopag maleate), an oral thrombopoietin receptor agonist, which is indicated for the treatment of chronic immune thrombocytopenia in pediatric patients one year and older.

                          Product Name : Doptelet

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 12, 2024

                          Lead Product(s) : Avatrombopag Maleate

                          Therapeutic Area : Immunology

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : The agreement aims for the international development and distribution for the enFuse Injector, for the subcutaneous delivery of Aspaveli (pegcetacoplan), a targeted C3 therapy, for PNH.

                          Product Name : Aspaveli

                          Product Type : Peptide

                          Upfront Cash : Undisclosed

                          September 12, 2024

                          Lead Product(s) : Pegcetacoplan

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Recipient : Enable Injections

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          06

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Syfovre (pegcetacoplan injection) targets C3, being investigated for C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis.

                          Product Name : Syfovre

                          Product Type : Peptide

                          Upfront Cash : Not Applicable

                          August 08, 2024

                          Lead Product(s) : Pegcetacoplan

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Recipient : Apellis Pharmaceuticals

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Altuvoct (efanesoctocog alfa) is a once-weekly and high-sustained factor VIII replacement therapy, approved for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A.

                          Product Name : Altuvoct

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          June 19, 2024

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Gamifant (emapalumab) is an anti-IFNγ monoclonal antibody that binds free and receptor-bound IFNγ, neutralizing its biological activity. Emapalumab is currently in Phase lll development for MAS.

                          Product Name : Gamifant

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          May 24, 2024

                          Lead Product(s) : Emapalumab

                          Therapeutic Area : Immunology

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : Aspaveli (pegcetacoplan) received an indication extension for PNH. It binds to complement protein C3 and is indicated for adults with paroxysmal nocturnal hemoglobinuria who have hemolytic anemia.

                          Product Name : Aspaveli

                          Product Type : Peptide

                          Upfront Cash : Not Applicable

                          May 08, 2024

                          Lead Product(s) : Pegcetacoplan

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          ELCC
                          Not Confirmed
                          ELCC
                          Not Confirmed

                          Details : BIVV001 (efanesoctocog alfa), a once-weekly Factor VIII therapy, is being evaluated for bleed prevention and perioperative prophylaxis in hemophilia A.

                          Product Name : BIVV001

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          April 26, 2024

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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