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            Lead Product(s): Idebenone

            Therapeutic Area: Genetic Disease Product Name: Puldysa

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 23, 2020

            Details:

            Uk's Medicines and Healthcare products Regulatory Agency has renewed for a further year the Early Access to Medicines Scheme scientific opinion for idebenone for patients with Duchenne muscular dystrophy in respiratory function decline who are not taking glucocorticoids.

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            Lead Product(s): Idebenone

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Highbridge Capital Management

            Deal Size: $21.1 million Upfront Cash: Undisclosed

            Deal Type: Financing June 04, 2020

            Details:

            In combination with Santhera's existing cash and cash equivalents, this financing will provide the Company with sufficient funding to complete regulatory work for Puldysa®, including the CHMP review, and with the achievement of Company milestones.

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            Lead Product(s): Idebenone

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 20, 2020

            Details:

            The large Phase 3 SIDEROS study was designed to confirm the efficacy of idebenone in patients with respiratory function decline who are concurrently taking glucocorticoids.