Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
FDA Grants Fast Track Designation to PTC518 Huntington's Disease Program
Details : PTC518, is an oral small molecule, which reduces mHTT production of the mutated Huntingtin protein that leads to injury and death of the neuron, resulting disease progression.
Product Name : PTC518
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
September 26, 2024
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Interim PIVOT-HD Results Show Favorable Effects in Huntington's Disease Patients
Details : PTC518, is an oral small molecule, which reduces mHTT production of the mutated Huntingtin protein that leads to injury and death of the neuron, resulting disease progression.
Product Name : PTC518
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
June 20, 2024
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Novartis Pharmaceuticals Corporation
Deal Size : $2,900.0 million
Deal Type : Licensing Agreement
PTC, Novartis Collaborate on Huntington's Disease Drug PTC518 Through Licensing Deal
Details : Under the licensing agreement, Novartis will assume responsibility for PTC518 (Votoplam) development, manufacturing and commercialization for the treatment of Huntington's disease.
Product Name : PTC518
Product Type : Other Small Molecule
Upfront Cash : $1,000.0 million
February 12, 2024
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Novartis Pharmaceuticals Corporation
Deal Size : $2,900.0 million
Deal Type : Licensing Agreement
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : PTC518, is a small molecule that can be taken orally, reduces the production of the mutated Huntingtin protein that leads to injury and death of the neuron, which results in disease progression.
Product Name : PTC518
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
June 21, 2023
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
PTC Provides Update on Ongoing Global PIVOT-HD Trial For PTC518
Details : The Phase 2 PIVOT-HD study consists of two parts; an initial 12-week placebo-controlled part focused on PTC518 pharmacology and pharmacodynamic effect, followed by a 9-month placebo-controlled part, during which blood, CSF, and radiographic biomarker dat...
Product Name : PTC518
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
October 18, 2022
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : PTC518 is a small molecule splicing modifier that acts via a unique mechanism to promote the inclusion of a novel pseudoexon containing a premature termination codon, thus triggering HTT mRNA degradation and subsequent reduction in HTT protein levels.
Product Name : PTC518
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
March 30, 2022
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : PTC518 is a small molecule splicing modifier that acts via a unique mechanism to promote the inclusion of a novel pseudoexon containing a premature termination codon against Huntington's Disease.
Product Name : PTC518
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
September 20, 2021
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : PTC518 is an orally bioavailable molecule with broad central nervous system and systemic distribution and has been designed to target Huntingtin protein expression with high selectivity and specificity.
Product Name : PTC518
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
November 17, 2020
Lead Product(s) : Votoplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable