N91115 CFTR Stabilizer

Nivalis Therapeutics (NVLS) is a clinical stage company that specializes in treatments for Cystic Fibrosis, with it's lead candidate stabilizer N91115.  N91115 is the only clinical stage candidate designed to stabilize CFTR (Cystic Fibrosis Transmembrane Conductance) inside the cell and at the cell surface.  The company expects N91115 to be complimentary to existing and future CFTR modulators, and is currently in a phase 2 clinical trial with approved drug Orkambi for people with the F508del mutation.  The trial is designed to see what effect N91115 may have as addition to what Orkambi achieves.  Below is the clinical trial and efficacy goals the company expects from this trial.

- N91115 in Patients With CF Homozygous for the F508del-CFTR Mutation.
- The company is targeting a 5% improvement, up and over Orkambi's 3% range in ppFEV1.
- Readout of data will be 2nd half 2016.
- First readout for a triple with three distinct therapies, for F508del homozygous patients.
- Have received Orphan and Fast Track designations from FDA in 2016.
- The patent for N91115 runs until 2031 at the very earliest.

The company plans to couple N91115 with leading potentiator's to potentially increase efficacy for combo therapy.  Either Kalydeco, CTP-656 or GLPG1837 are the current group of potentiator's that hold the most promise to date.  Thank you for reading.