Vertex Quietly Terminates Small Phase II CF Trial


September 12, 2017
By Alex Keown, Breaking News Staff


BOSTON – Shares of Vertex Pharmaceuticals (VRTX) went on something of an overnight ride. Prices dipped Monday afternoon, presumably due to an article published by Stat News, reporting the termination of a long-acting trial combining a recently acquired therapy with its cystic fibrosis drug, Kalydeco

Stocks rallied somewhat as the day went on, then jumped in overnight trading. Shares are trading at $157.68 this morning. However, this morning in pre-market trading, share prices have slipped again. In the article, Stat’s Adam Feuerstein reported the company had terminated a Phase II trial evaluating the longer-acting Kalydeco combination as a treatment for CF patients with CFTR mutations.

The experimental drug in question, CTP-656, was acquired from Concert Pharmaceuticals (CNCE) earlier this year. CTP-656 is an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator designed for use as part of a once-daily drug combination for the CTFR mutation. Vertex handed over $160 million to Concert for the experimental drug. Concert stood to gain an additional $90 million from Vertex if CTP-656 was approved as part of a combination treatment, according to terms of the deal. Now though that is not likely to happen. According to the Phase II program has been terminated.

When Feuerstein initially spoke with the company, they allegedly indicated the development of the drug combination was continuing, despite the trial having been terminated, according to a brief review on Seeking Alpha. Vertex has not indicated why the trial was terminated, according to the report.

Despite this trial termination, Vertex has been making headway in its CF program. In July, the company announced positive data from Phase I and Phase II studies of three different triple combination regimens for cystic fibrosis patients with strains that have been hard to treat using current medications on the market. Data from those trials demonstrated the potential to treat the underlying cause of cystic fibrosis in patients who have one F508del mutation and one minimal function mutation (F508del/Min) – one of the most difficult types of CF to treat. The company has accelerated these trials in hopes of gaining regulatory approval and is also looking at other triple combination options, Vertex indicated in July.

On Monday during the Morgan Stanley Healthcare conference, Chief Commercial Officer Stuart Arbuckle told reporters and investors the company has many irons in the fire. While he did not address the trial termination of CTP-656 in any media reports, he pointed to a looming U.S. Food and Drug Administration decision for the company’s two-drug combination of tezacaftor and Kalydeco. Writing in the Motley Fool, analyst Keith Speights said the drug combination posted stellar Phase II results and the FDA could make a decision by Feb. 28, 2018. Arbuckle, Speights said, believes the market life for this drug combination could be short if it’s approved due to a number of pending combination drug therapies in development. He said the drug though could be a great option for patients who have not yet taken Vertex’s other big CF drug, Orkambi, or have discontinued the medication due to side effects.

“Vertex’s triple-drug combos present the company’s greatest long-term opportunity. Arbuckle said positive data from studies so far gives the biotech confidence that it will be able to deliver on its mission of developing an effective daily treatment for 90 percent of cystic fibrosis patients,” Speights wrote.



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