Lead Product(s) : Navepegritide
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : Navepegritide
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : NMD670
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : NMD670
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : Navepegritide
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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
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
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
Lead Product(s) : NMD670
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : Navepegritide
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : Tesofensine,Metoprolol Tartrate
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : Tesofensine,Metoprolol Tartrate
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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