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[{"orgOrder":0,"company":"Prothena","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Prothena to Present Data on Survival Benefit Observed in Completed Phase 3 Study of Drug Candidate Birtamimab in Patients with Mayo Stage IV AL Amyloidosis at the ASH 2022 Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Prothena"},{"orgOrder":0,"company":"Prothena","sponsor":"Jefferies","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Prothena Announces Proposed Public Offering of Ordinary Shares","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Prothena"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Announces FDA Acceptance of New Drug Application for Oleogel-S10 for the Treatment of Epidermolysis Bullosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Announces Positive Interim Analysis Data From Open Label Phase of EASE Phase 3 Trial in EB","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"}]

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            Birtamimab, formerly known as NEOD001, is an investigational monoclonal antibody designed to specifically and selectively target and clear the amyloid that accumulates and causes organ dysfunction and failure in patients with AL amyloidosis.

            Lead Product(s): Birtamimab,Cyclophosphamide,Bortezomib

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Jefferies

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Public Offering December 13, 2022

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            Birtamimab is an investigational monoclonal antibody designed to specifically and selectively target and clear the amyloid that accumulates and causes organ dysfunction and failure in patients with AL amyloidosis.

            Lead Product(s): Birtamimab,Cyclophosphamide,Bortezomib

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 03, 2022

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            The trial met its primary endpoint in the double-blind phase (DBP; 90 days); target wounds treated with Oleogel-S10 reached first complete wound closure by Day 45 more frequently than wounds treated with control gel, with statistical significance (p=0.013).

            Lead Product(s): Episalvan

            Therapeutic Area: Genetic Disease Product Name: Oleogel-S10

            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 March 26, 2022

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            Oleogel-S10 is a potential treatment for the cutaneous manifestations of Junctional and Dystrophic EB, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment.

            Lead Product(s): Oleogel-S10

            Therapeutic Area: Genetic Disease Product Name: Filsuvez

            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 02, 2021

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