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[{"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":"Small molecule","graph2":"Orphazyme"},{"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":"Small molecule","graph2":"Orphazyme"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Receives Positive Feedback from FDA on Regulatory Path for Tesomet in Prader-Willi Syndrome (PWS)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona receives Pre-IND Feedback from FDA on Regulatory Path for Tesomet in Prader-Willi Syndrome (PWS) and Hypothalamic Obesity (HO)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Updates Tesomet Clinical Development Timelines Based on Manufacturing Feedback from U.S. FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Initiates Phase 2b Clinical Trial of Tesomet for Prader-Willi Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Provides Update on Ongoing Review of Tesofensine in Mexico","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NMD Pharma initiates Phase II trial of NMD670 in Spinal Muscular Atrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"NMD PHARMA"},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Novo Holdings","pharmaFlowCategory":"D","amount":"$80.0 million","upfrontCash":"Undisclosed","newsHeadline":"NMD Pharma Raises \u20ac75 Million (~$80 million) in a Series B Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"NMD PHARMA"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ascendis Pharma A\/S Receives Orphan Designation for TransCon\u2122 CNP for the Treatment of Achondroplasia in Europe","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Ascendis Pharma"},{"orgOrder":0,"company":"Ascendis Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Once-Weekly TransCon\u2122 CNP Achieved Primary Efficacy Objective, with Superiority at 100 \u00b5g\/kg\/week, in Ascendis Pharma\u2019s Phase 2 ACcomplisH Trial of Children with Achondroplasia Aged 2 to 10 Years","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Peptide","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Ascendis Pharma"}]

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

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            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.

            Lead Product(s): NMD670

            Therapeutic Area: Genetic Disease Product Name: NMD670

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Novo Holdings

            Deal Size: $80.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing November 15, 2023

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            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.

            Lead Product(s): NMD670

            Therapeutic Area: Genetic Disease Product Name: NMD670

            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 September 26, 2023

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            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.

            Lead Product(s): Natriuretic Peptide, C-Type

            Therapeutic Area: Genetic Disease Product Name: TransCon CNP

            Highest Development Status: Phase II Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 13, 2022

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

            Lead Product(s): Tesofensine,Metoprolol Tartrate

            Therapeutic Area: Genetic Disease Product Name: Tesomet

            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 May 20, 2022

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            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 the study.

            Lead Product(s): Tesofensine,Metoprolol Tartrate

            Therapeutic Area: Genetic Disease Product Name: Tesomet

            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 December 28, 2021

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            Details:

            The company requested FDA feedback, and the FDA agreed with the company’s proposal but requested additional information regarding Tesomet manufacturing.

            Lead Product(s): Tesofensine,Metoprolol Tartrate

            Therapeutic Area: Genetic Disease Product Name: Tesomet

            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 April 21, 2021

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            Details:

            FDA recommended that Saniona conduct a supportive Phase 2b study prior to initiating a Phase 3 study to confirm the safety and efficacy of the doses intended for commercialization.

            Lead Product(s): Tesofensine,Metoprolol Tartrate

            Therapeutic Area: Genetic Disease Product Name: Tesomet

            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 October 09, 2020

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            Details:

            TransCon CNP is a investigational long-acting prodrug of CNP, designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose.

            Lead Product(s): Natriuretic Peptide, C-Type

            Therapeutic Area: Genetic Disease Product Name: TransCon CNP

            Highest Development Status: Phase II Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 12, 2020

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            Details:

            Arimoclomol demonstrated a marked and clinically meaningful dose-dependent reduction in liver and spleen size. Data supports Orphazyme's intention to proceed with pivotal stage clinical development in Gaucher disease .

            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease 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 June 29, 2020

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            Details:

            The data show a dose-dependent effect of arimoclomol on certain disease-relevant clinical secondary endpoints, such as liver and spleen size.

            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease 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 June 24, 2020

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