New treatment for Nethertons syndrome
| Reference number | |
| Coordinator | GU VENTURES AB - Holdingbolaget vid Göteborgs universitet AB |
| Funding from Vinnova | SEK 2 000 000 |
| Project duration | April 2014 - April 2016 |
| Status | Completed |
Purpose and goal
The funding has enabled Sixera to advance the development of novel drug for Netherton syndrome in relation to IPR, technology and business relations. This has resulted in new investments by original and new owners. The project has generated intense collaborations between Sixera and industrial partners such as SP Technical Research Int. of Sweden, and PSR Orphan Experts. In addition, close interactions with a network of key consultants and clinical opinion leaders have been established. New IPR has been filed and research grant application in Horizon 2020 program is being prepared.
Results and expected effects
Main project achievements: -IPR covering the lead compound SXR1096 submitted -CMC: Technical batches of high purity SXR1096 produced; clinically relevant dermal formulation developed -Safety: Indicative toxicology assessment of SXR1096 did not show any safety concerns -Efficacy: Proof-of-principle for Sixera´s dermal product demonstrated in a mouse model -Regulatory: Orphan designation for SXR1096 granted in EC and US -Clinical: Clinical trial synopses developed; clinical opinion leader panel established -BD: Intense contacts with industrial partners and VC investors
Approach and implementation
The project activities, time plan and budget have followed the original project plan. The planned studies have been successfully conducted, and the project is brought to the stage where GLP toxicology studies, followed by clinical development program, can be initiated.