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[{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme reports positive arimoclomol data from open-label phase 2\/3 extension in Niemann-Pick disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"CytRx Corporation","pharmaFlowCategory":"D","amount":"$120.0 million","upfrontCash":"Undisclosed","newsHeadline":"CytRx Corporation Highlights Updated Arimoclomol Milestone Guidance","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Neurology","graph2":"Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme\u2019s Arimoclomol Receives US Fast Track Designation in Amyotrophic Lateral Sclerosis","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Neurology","graph2":"Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Initiates Rolling Submission Of New Drug Application For Arimoclomol With FDA in Niemann-Pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Phase 2 Study of Arimoclomol In Gaucher Disease Demonstrates Marked Improvements in Key Clinical Markers","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CytRx Corporation Highlights Orphazyme's Announcement of its Phase 2 Study Results with Arimoclomol in Gaucher Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Michael J. Fox Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orphazyme to Collaborate with The Michael J. Fox Foundation on Parkinson\u2019s Disease Research","therapeuticArea":"Neurology","highestDevelopmentStatus":"Preclinical","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Neurology","graph2":"Preclinical"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Completes Rolling Submission of New Drug Application to U.S. FDA for Arimoclomol for Niemann-Pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Announces U.S. FDA Acceptance and Priority Review of New Drug Application for Arimoclomol for Niemann-Pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"CytRx Corporation","pharmaFlowCategory":"D","amount":"$83.7 million","upfrontCash":"Undisclosed","newsHeadline":"CytRx Issues Statement Regarding Orphazyme\u2019s Global Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Accelerates Arimoclomol Pre-Launch Activities and Updates Financial Outlook for 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Provides Regulatory Update on Arimoclomol for NPC","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme\u2019s Arimoclomol Expected to Be Used with Zavesca After Likely FDA Approval in Niemann-Pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme to Showcase Data on Arimoclomol in Niemann-Pick Disease Type C During the 2021 Annual WORLDSymposium\u2122","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Announces Topline Results from Pivotal Trial of Arimoclomol for Inclusion Body Myositis (IBM)","therapeuticArea":"Immunology","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Immunology","graph2":"Phase II"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Announces Topline Data from Pivotal Trial of Arimoclomol in Amyotrophic Lateral Sclerosis (ALS)","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Neurology","graph2":"Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Presents 36-Month Data Supporting Durable Response to Arimoclomol During Parseghian Scientific Conference for Npc Research","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Provides Regulatory Update Following Type A Meeting with FDA on Arimoclomol in Niemann-Pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Provides Update for Planned NDA Resubmission for Arimoclomol for the Treatment of Niemann-Pick disease type C in the United States","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme to Present Additional Data on Arimoclomol\u2019s Mechanism of Action During 2022 Annual WORLDSymposium\u2122","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Announces Withdrawal of European Marketing Authorisation Application for Arimoclomol for the Treatment of Niemann-pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"KemPharm","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$12.8 million","newsHeadline":"KemPharm Announces Strategic Acquisition of Arimoclomol from Orphazyme, Expanding its Rare CNS Diseases Pipeline","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III"}]

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            Details:

            The acquisition of arimoclomol aligns perfectly with the strategy to build KemPharm’s value via the advancement and commercialization of novel treatments that address rare CNS conditions, including the lead clinical candidate, KP1077 in idiopathic hypersomnia.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: KemPharm

            Deal Size: Undisclosed Upfront Cash: $12.8 million

            Deal Type: Acquisition May 15, 2022

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            Decision to withdraw the European Marketing Authorisation Application for BRX-345 (arimoclomol) for the treatment of Niemann-Pick disease Type C (NPC) ahead of a final vote and opinion by the CHMP on the MAA scheduled for later this month.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease Product Name: BRX-345

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 22, 2022

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            Orphazyme has made progress towards resubmission of the NDA for BRX-345 (arimoclomol citrate), amplifies the production of heat shock proteins for the treatment of NPC to the FDA and plans to request a Type C Meeting in Q2 2022.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease Product Name: BRX-345

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 11, 2022

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            Arimoclomol, is an investigational drug candidate that amplifies the production of heat shock proteins (HSPs). Arimoclomol is administered orally, and has now been studied in Phase 1, Phase 2, and pivotal Phase 2/3 trials.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 07, 2022

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            arimoclomol, a heat shock protein co-inducer that promotes nascent protein folding, in patients with rapidly progressive SOD1 amyotrophic lateral sclerosis (ALS) and Niemann-Pick disease type C.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 31, 2021

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            NPC patients receiving routine clinical care was estimated to be a mean increase of 5.2 points after three years, based on a statistical model combining placebo data from the NPC-002 double-blind study and prospective data from the observational NPC-001 study.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 28, 2021

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            Orphazyme announced that ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS. No important safety signals were reported in the trial.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Neurology Product Name: Miplyffa

            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 07, 2021

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            Orphazyme's phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis (IBM), a progressively debilitating muscle-wasting disease, did not meet its primary and secondary endpoints.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Immunology Product Name: Undisclosed

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 29, 2021

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            Orphazyme plans to present a series of data on its investigational drug arimoclomol during the 17th Annual WORLDSymposium Scientific Meeting, to be held virtually on February 8-12, 2021.

            Lead Product(s): Arimoclomol,Miglustat

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 04, 2021

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            The long-term data demonstrate a continued positive impact on disease progression over two years. Furthermore, separate data from a post-hoc genetic sub-group analysis of patients provides more evidence of the efficacy of arimoclomol.

            Lead Product(s): Arimoclomol,Miglustat

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 22, 2021

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