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[{"orgOrder":0,"company":"Khondrion","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Khondrion announces first patients dosed in Phase IIb study of Sonlicromanol for mitochondrial diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Khondrion"},{"orgOrder":0,"company":"Khondrion","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Khondrion Shares Progress of Sonlicromanol Development Programme at Mitochondrial Medicine 2020 Virtual Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Khondrion"},{"orgOrder":0,"company":"Khondrion","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Khondrion Receives EMA Agreement on its Paediatric Investigation Plan for Sonlicromanol","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Khondrion"},{"orgOrder":0,"company":"Khondrion","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Khondrion Completes Enrolment in KHENERGYZE Phase IIb Trial Evaluating Sonlicromanol in Adult Patients With MELAS Spectrum Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Khondrion"},{"orgOrder":0,"company":"Khondrion","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Khondrion Announces Sonlicromanol Phase IIb Progress Supporting Phase III Development in Melas Spectrum Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Khondrion"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Azafaros"}]

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

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            AZ-3102 is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NP-C).

            Lead Product(s): AZ-3102

            Therapeutic Area: Genetic Disease Product Name: AZ-3102

            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 08, 2023

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            Patients treated with KH176 (sonlicromanol) for 28 days did not achieve statistically significant improvement in attention domain score of cognitive functioning, as assessed by the computerised Cogstate visual identification test, compared to those who received placebo.

            Lead Product(s): Sonlicromanol

            Therapeutic Area: Genetic Disease Product Name: KH176

            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 November 22, 2022

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            Topline data from KHENERGYZE Phase IIb trial for KH176 (sonlicromanol) is expected in the third quarter of 2022, Phase IIa study showed a significant improvement in attention and mood-related outcomes in patients with MELAS spectrum disorder.

            Lead Product(s): Sonlicromanol

            Therapeutic Area: Genetic Disease Product Name: KH176

            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 January 10, 2022

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            With the acceptance of the PIP, Khondrion plans to initiate a paediatric trial to evaluate the safety and efficacy of sonlicromanol in children from birth to less than 18 years with mitochondrial disease affecting oxidative phosphorylation and symptoms of motor impairment.

            Lead Product(s): Sonlicromanol

            Therapeutic Area: Genetic Disease Product Name: KH176

            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 January 18, 2021

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            An ongoing Phase 2B clinical trial in patients with MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) spectrum disorders is investigating the effect of sonlicromanol on cognitive functioning.

            Lead Product(s): Sonlicromanol

            Therapeutic Area: Genetic Disease Product Name: KH176

            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 November 27, 2020

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            The study’s primary objective is to evaluate the dose-effect of Sonlicromanol on the attention domain score of cognitive functioning, Results are expected in the second half of 2020.

            Lead Product(s): Sonlicromanol

            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 January 27, 2020

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