[{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Novartis Pharmaceuticals Corporation","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Votoplam","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : PTC518, is an oral small molecule, which reduces mHTT production of the mutated Huntingtin protein that leads to injury and death of the neuron, resulting disease progression.

                          Product Name : PTC518

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 26, 2024

                          Lead Product(s) : Votoplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : PTC518, is an oral small molecule, which reduces mHTT production of the mutated Huntingtin protein that leads to injury and death of the neuron, resulting disease progression.

                          Product Name : PTC518

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          June 20, 2024

                          Lead Product(s) : Votoplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : Under the licensing agreement, Novartis will assume responsibility for PTC518 (Votoplam) development, manufacturing and commercialization for the treatment of Huntington's disease.

                          Product Name : PTC518

                          Product Type : Other Small Molecule

                          Upfront Cash : $1,000.0 million

                          February 12, 2024

                          Lead Product(s) : Votoplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Novartis Pharmaceuticals Corporation

                          Deal Size : $2,900.0 million

                          Deal Type : Licensing Agreement

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                          04

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : PTC518, is a small molecule that can be taken orally, reduces the production of the mutated Huntingtin protein that leads to injury and death of the neuron, which results in disease progression.

                          Product Name : PTC518

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          June 21, 2023

                          Lead Product(s) : Votoplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : The Phase 2 PIVOT-HD study consists of two parts; an initial 12-week placebo-controlled part focused on PTC518 pharmacology and pharmacodynamic effect, followed by a 9-month placebo-controlled part, during which blood, CSF, and radiographic biomarker dat...

                          Product Name : PTC518

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          October 18, 2022

                          Lead Product(s) : Votoplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : PTC518 is a small molecule splicing modifier that acts via a unique mechanism to promote the inclusion of a novel pseudoexon containing a premature termination codon, thus triggering HTT mRNA degradation and subsequent reduction in HTT protein levels.

                          Product Name : PTC518

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          March 30, 2022

                          Lead Product(s) : Votoplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : PTC518 is a small molecule splicing modifier that acts via a unique mechanism to promote the inclusion of a novel pseudoexon containing a premature termination codon against Huntington's Disease.

                          Product Name : PTC518

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 20, 2021

                          Lead Product(s) : Votoplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          AAN
                          Not Confirmed
                          AAN
                          Not Confirmed

                          Details : PTC518 is an orally bioavailable molecule with broad central nervous system and systemic distribution and has been designed to target Huntingtin protein expression with high selectivity and specificity.

                          Product Name : PTC518

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 17, 2020

                          Lead Product(s) : Votoplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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