Research and development pipeline
As part of its research and development, Vertex Pharmaceuticals (VRTX) has been actively exploring new cystic fibrosis (or CF) drugs as well as other indications for its existing drug Kalydeco.
The above diagram shows the various research and development (or R&D) programs Vertex Pharmaceuticals (VRTX) is currently undertaking.
Lumacaftor and ivacaftor
The combination of lumacaftor and ivacaftor has already been approved in the United States for CF patients who are 12 years or older in age and suffer from the F508del mutation in their cystic fibrosis transmembrane conductance regulator (or CFTR) gene.
Known as Orkambi, this orphan drug enjoys substantial pricing flexibility, like other orphan drugs such as Alexion Pharmaceuticals’ (ALXN) Soliris (eculizumab), Amgen’s (AMGN) Blincyto (blinatumomab), and Gilead Sciences’ (GILD) Zydelig ((idelalisib). To further increase its patient base, Vertex Pharmaceuticals is conducting clinical trials to test the drug in CF patients six to 11 years of age.
VX-661 with ivacaftor
In the first quarter of 2015, Vertex Pharmaceuticals initiated four Phase 3 clinical trials to test the use of VX-661 with ivacaftor in groups of CF patients. Since CF is a rare disease affecting a very small number of patients, enrollment of patients in these clinical trials is expected to negatively impact Kalydeco’s revenues. Orkambi will, however, be protected from this switching effect, as the drug is currently approved only in the United States. The company is conducting clinical trials in Europe.
Vertex Pharmaceuticals has entered into a collaboration with Parion Sciences to develop a treatment for CF patients as well as other pulmonary disorders. Called epithelial sodium channel (or ENaC) inhibitors, this program is currently in Phase 2a clinical trials. Results from this program are expected in the middle of 2016.
Next-generation CFTR corrector compounds
In addition to the above programs, Vertex Pharmaceuticals is also involved in developing next-generation CFTR corrector compounds.
Instead of directly investing in Vertex Pharmaceuticals stock, investors can get exposure to the company’s strong R&D pipeline by investing in the iShares Nasdaq Biotechnology ETF (IBB). Vertex Pharmaceuticals accounts for 4.24% of IBB’s total holdings.