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    [{"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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                            01

                            Ascendis Pharma

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                            Ascendis Pharma

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                            Lead Product(s) : Navepegritide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            TransCon CNP Shows Growth & Bone Gains in Children with Achondroplasia

                            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

                            Ascendis Pharma

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                            Ascendis Pharma

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                            Lead Product(s) : Navepegritide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Ascendis Submits NDA for TransCon CNP for Achondroplasia in Kids

                            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

                            NMD PHARMA

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                            Lead Product(s) : NMD670

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            NMD Pharma’s NMD670 Gains FDA Orphan Status for Charcot-Marie-Tooth

                            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

                            NMD PHARMA

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                            NMD PHARMA

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                            Lead Product(s) : NMD670

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            NMD Pharma Starts Phase 2 NMD670 Study for Charcot-Marie-Tooth Disease

                            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

                            Ascendis Pharma

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                            Ascendis Pharma

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                            Lead Product(s) : Navepegritide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Ascendis' Dwarfism Drug Accelerates Growth in Phase 3, Teeing up Challenge to BioMarin

                            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

                            NMD PHARMA

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                            NMD PHARMA

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                            Lead Product(s) : NMD670

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Organovo Holdings

                            Deal Size : $80.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            NMD Pharma Raises €75 Million (~$80 million) in a Series B Financing

                            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

                            NMD PHARMA

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                            NMD PHARMA

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                            Discovery Europe
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                            Lead Product(s) : NMD670

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            NMD Pharma initiates Phase II trial of NMD670 in Spinal Muscular Atrophy

                            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

                            Ascendis Pharma

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                            Ascendis Pharma

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                            Lead Product(s) : Navepegritide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Once-Weekly TransCon™ CNP Achieved Primary Efficacy Objective, with Superiority at 100 µg/kg/week, in Ascen...

                            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

                            Saniona

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                            Saniona

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                            Lead Product(s) : Tesofensine,Metoprolol Tartrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Saniona Provides Update on Ongoing Review of Tesofensine in Mexico

                            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

                            Saniona

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                            Saniona

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                            Lead Product(s) : Tesofensine,Metoprolol Tartrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Saniona Initiates Phase 2b Clinical Trial of Tesomet for Prader-Willi Syndrome

                            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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