Promising News from Vertex for the Cystic Fibrosis Community

enniob

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Vertex trial results recently presented at the NACFC, spurred several stock analysts to increase expectations for the company’s shares. 22 out of 34 analysts covering Vertex now have a positive rating on the stock.

Vertex recently provided data showing its experimental drug, lumacaftor, and its approved drug, Kalydeco, continue to improve lung function in cystic fibrosis patients after 48 weeks of treatment. That confirms the effects reported in June from patients who were on the drug combination for 24 weeks, and helped dispel lingering concerns stemming from early-stage tests that the two drugs might counteract each other.

Vertex also outlined the progress in identifying new drugs for the disease in the last several months, and plans to start human tests of one or two of those drugs next year. Those drugs would also be designed to be used in combination with Kalydeco or similar drugs, which work by helping the maturation and function of the CFTR protein, which when defective, cause cystic fibrosis.

Since Kalydeco only works in about 4 percent of cystic fibrosis patients, Vertex is developing other drugs — including lumacaftor and another drug in mid-stage trials — that will allow more CFTR to actually reach the cell surface. That would effectively expand the treatable population to well over half of all cystic fibrosis patients. This larger market share should hopefully decrease the price of drugs like Kalydeco and improve availability and affordability of the drug in a larger treatable population.

Vertex also announced its intention to make Kalydeco available to children ages 2-5 and has applied to the relevant authorities for usage of Kalydeco in this age group. Currently, Kalydeco is only approved for usages in CF patients 6 years and older.

Street forecasted sales of Vertex cystic fibrosis drugs are estimated to generate $1 billion in 2015 and $3.1 billion in 2016, assuming approval of lumacaftor. Vertex posted losses for at least the last six quarters, but analysts expect Vertex to return to profitability by the end of 2015.

Below is a diagram of how potentiator drugs like Kalydeco can work synergistically with corrector drugs like lumacaftor to achieve greater expression and function of CFTR protein on the surface of cells in people with CF.
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Source (and special thanks for the well written coverage of Vertex): http://t.co/QtzSgLNYE0

- Chris Baldwin, PhD
CEO @ CysticFibrosis.com
 
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