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            Lead Product(s): Plasminogen

            Therapeutic Area: Genetic Disease Product Name: Ryplazim

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 07, 2020

            Details:

            Two posters were presented that highlighted individual case histories of patients with C-PLGD treated with the investigational intravenous Ryplazim® (plasminogen) in compassionate use and expanded access cases.

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            Lead Product(s): Plasminogen

            Therapeutic Area: Genetic Disease Product Name: Ryplazim

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 08, 2020

            Details:

            The BLA resubmission for Ryplazim® was supported by data from a Phase 2/3 clinical trial, which evaluated 15 patients, both pediatric and adults, with C-PLGD over 48 weeks of therapy with Ryplazim®.

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            Lead Product(s): Plasminogen

            Therapeutic Area: Genetic Disease Product Name: Ryplazim

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 05, 2020

            Details:

            Two abstracts related to the Company’s clinical development activities of Ryplazim® for the treatment of Congenital Plasminogen Deficiency will be presented at the 62nd American Society of Hematology Annual Meeting and Exposition scheduled to take place in a virtual format.

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            Lead Product(s): Plasminogen

            Therapeutic Area: Genetic Disease Product Name: Ryplazim

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 21, 2020

            Details:

            The BLA resubmission for Ryplazim® is supported by data from a Phase 2/3 clinical study which evaluated 15 patients, both pediatric and adults, with congenital plasminogen deficiency over 48 weeks of therapy with Ryplazim®.

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            Lead Product(s): Plasminogen

            Therapeutic Area: Genetic Disease Product Name: Ryplazim

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 08, 2020

            Details:

            In a pivotal phase 2/3 clinical trial for the treatment of C-PLGD, all of the patients treated with Ryplazim® achieved at least the targeted increase from baseline in their individual trough plasminogen activity levels through 12 weeks of therapy.