[{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2025","type":"Inapplicable","leadProduct":"Navepegritide","moa":"FGFR 3","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2020","type":"Inapplicable","leadProduct":"Navepegritide","moa":"C-type natriuretic peptide","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2022","type":"Inapplicable","leadProduct":"Navepegritide","moa":"C-type natriuretic peptide","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2024","type":"Inapplicable","leadProduct":"Navepegritide","moa":"C-type natriuretic peptide","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ascendis Pharma \/ Inapplicable"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"NMD PHARMA \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Inapplicable"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"NMD PHARMA \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Inapplicable"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"NMD PHARMA \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Inapplicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Arimoclomol Citrate","moa":"HSF-1 pathway","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Orphazyme \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Orphazyme \/ Inapplicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Arimoclomol Citrate","moa":"HSF-1 pathway","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Orphazyme \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Orphazyme \/ Inapplicable"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"NMD PHARMA \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Inapplicable"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Organovo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2023","type":"Series B Financing","leadProduct":"NMD670","moa":"ClC-1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NMD PHARMA","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"NMD PHARMA \/ Organovo Holdings","highestDevelopmentStatusID":"8","companyTruncated":"NMD PHARMA \/ Organovo Holdings"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Foundation for Prader-Willi Research","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2021","type":"Partnership","leadProduct":"Tesofensine","moa":"||Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Saniona \/ Foundation for Prader-Willi Research","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Foundation for Prader-Willi Research"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Saniona","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Tesofensine","moa":"||Triple monoamine reuptake","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Saniona","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Saniona \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Saniona \/ Inapplicable"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Peptide","year":"2025","type":"Inapplicable","leadProduct":"Navepegritide","moa":"NPRC","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Ascendis Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ascendis Pharma \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Ascendis Pharma \/ Inapplicable"}]

Find Drugs for Genetic Disease in Phase II Clinical Development in DENMARK

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                          Top Deals by Deal Size (USD bn)

                          01

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : TransCon CNP (navepegritide) is an investigational prodrug of CNP administered once weekly providing continuous exposure of active CNP to receptors on tissues throughout the body.

                          Product Name : TransCon CNP

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          May 13, 2025

                          Lead Product(s) : Navepegritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : TransCon CNP (c-type natriuretic peptide prodrug) is a FGFR 3 inhibitor. It is being evaluated for the treatment of children with achondroplasia (ACH) aged upto 3 years.

                          Product Name : TransCon CNP

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          March 31, 2025

                          Lead Product(s) : Navepegritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : NMD670 is a first-in-class small molecule inhibitor of ClC-1. It is being developed by NMD Pharma for the treatment of patients with charcot-marie-tooth disease.

                          Product Name : NMD670

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 06, 2025

                          Lead Product(s) : NMD670

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : NMD670 is a first-in-class small molecule inhibitor of ClC-1. It is being developed by NMD Pharma for the treatment of patients with charcot-marie-tooth disease.

                          Product Name : NMD670

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 18, 2024

                          Lead Product(s) : NMD670

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : TransCon CNP (Navepegritide) is a FGFR 3 inhibitor. It is being evaluated for the treatment of children with achondroplasia (ACH) aged 2 to 11 years.

                          Product Name : TransCon CNP

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          September 17, 2024

                          Lead Product(s) : Navepegritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : The proceeds will be used to complete three Phase 2 clinical studies of first-in-class ClC-1 molecule inhibitor NMD670 in myasthenia gravis, spinal muscular atrophy and Charcot-Marie-Tooth disease.

                          Product Name : NMD670

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          November 15, 2023

                          Lead Product(s) : NMD670

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Organovo Holdings

                          Deal Size : $80.0 million

                          Deal Type : Series B Financing

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                          07

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : NMD670 is a first-in-class small molecule inhibitor of the muscle-specific chloride ion channel, the ClC-1 ion channel, which is investigated for the treatmet of spinal muscular atrophy.

                          Product Name : NMD670

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 26, 2023

                          Lead Product(s) : NMD670

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : TransCon CNP is an investigational prodrug of CNP in development for the treatment of ACH in children. Designed to provide continuous CNP exposure, goal of TransCon CNP is to optimize efficacy with a safe and convenient once-weekly dose.

                          Product Name : TransCon CNP

                          Product Type : Peptide

                          Upfront Cash : Inapplicable

                          November 13, 2022

                          Lead Product(s) : Navepegritide

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : Tesomet (Tesofensine) is a serotonin–noradrenaline–dopamine reuptake inhibitor from the phenyltropane family of drugs, which is being developed for the treatment of obesity.

                          Product Name : Tesomet

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          May 20, 2022

                          Lead Product(s) : Tesofensine,Metoprolol Tartrate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : Adult patients receiving Tesomet achieved a statistically significant reduction in hyperphagia, as well as a reduction in body weight. Adolescent patients demonstrated dose-dependent reductions in hyperphagia and body weight in open-label extensions of t...

                          Product Name : Tesomet

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          December 28, 2021

                          Lead Product(s) : Tesofensine,Metoprolol Tartrate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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