Traffick Therapeutics Inc. has characterized a series of small molecules acting as correctors and potentiators of the CF transmembrane conductance regulator (CFTR) protein which is mutated in patients with CF.
Our goal is to identify an oral therapy that will correct the defective trafficking of the F508del-CFTR mutant and transform Cystic Fibrosis (CF) treatment by moving from symptom treatment to disease modification.
- Optimize our current corrector lead series to deliver a clinical candidate achieving a better correction and properties than the current reference compound VX-809 in CF Human Bronchial Epithelium Cells (HBE) function, for Cystic Fibrosis treatment.
- To develop a novel lead series displaying an additive effect with our current lead series.
- To develop our potentiator series to a lead compound with much improved properties for the treatment of COPD and other respiratory diseases.
We have already found and characterized highly potent CFTR corrector/potentiator molecules: a lead series with a lead corrector (MG277) which combined encouraging activity in HBE from CF patient and promising developability properties:
- validated hit correctors that need to enter a hit-to-lead phase and SAR
- identified hit potentiators that need to enter a hit-to-lead phase and SAR
- preliminary data suggesting an additive effect between our current lead series and our hits
- novel assays and reagents for testing correctors and potentiator.
The next step:
- Lead optimization of our current corrector lead series to identify a candidate molecule suitable for oral therapy and its progression to be ready for a FTIH clinical study.
- To develop our new hit corrector and potentiators (from our screening campaign) to lead series
- To establish the optimal combination and concentration for each component.
- To identify the target of action of each compound to help begin to understand their mechanism of action