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[{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera Completes Enrollment of Phase 3 SIDEROS Study with Puldysa\u00ae (Idebenone) in Duchenne Muscular Dystrophy (DMD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Highbridge Capital Management","pharmaFlowCategory":"D","amount":"$21.1 million","upfrontCash":"Undisclosed","newsHeadline":"Santhera Receives Financing Commitment of up to CHF 20 Million from Fund Managed by Highbridge Capital Management","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"UK\u2019s MHRA renews EAMS Scientific Opinion for Santhera\u2019s Idebenone in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera to Discontinue Phase 3 SIDEROS Study and Development of Puldysa\u00ae in Duchenne Muscular Dystrophy (DMD) and Focus on Vamorolone","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera to Discontinue Phase 3 SIDEROS Study and Development of\n Puldysa\u00ae in Duchenne Muscular Dystrophy (DMD) and Focus on Vamorolone","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Chiesi Group","pharmaFlowCategory":"D","amount":"$104.0 million","upfrontCash":"$49.2 million","newsHeadline":"Santhera Completes Divestment of Raxone\u00ae\/Idebenone Business to Chiesi Group","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"July 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"}]

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            Details:

            Under the terms of the agreement, Chiesi Group acquired all assets and certain liabilities related to Raxone (idebenone) in all indications worldwide, including Raxone in leber hereditary optic neuropathy (LHON).

            Lead Product(s): Idebenone

            Therapeutic Area: Genetic Disease Product Name: Raxone

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Chiesi Group

            Deal Size: $104.0 million Upfront Cash: $49.2 million

            Deal Type: Divestment July 31, 2023

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            Details:

            Data from an interim analysis conducted by the independent Data and Safety Monitoring Board concluded that the Phase 3 SIDEROS study with Puldysa® (idebenone) was unlikely to meet its primary endpoint.

            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 October 06, 2020

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            Data from an interim analysis conducted by the (DSMB) concluded that the study was unlikely to meet its primary endpoint. Santhera will discontinue the study, withdraw the European marketing authorization application and end the global development program for Puldysa.

            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 October 06, 2020

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            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.

            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

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            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.

            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

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            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.

            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

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