After Hep C Flop, Has Vertex Learned From Its Past Mistakes?

After Hep C Flop, Has Vertex Learned From Its Past Mistakes? October 18, 2016
By Alex Keown, BioSpace.com Breaking News Staff

BOSTON – It’s no secret that despite its successes in developing treatments for cystic fibrosis, Vertex Pharmaceuticals has turned a profit one time. While the Boston-based company is entrenched in the CF market, it’s only a matter of time before other drugs supplant Orkambi and Kalydeco.

It would not be the first time something like that happened to Vertex. The company is no stranger to disappointment. It once had a promising hepatitis drug, telaprevir that had fewer side effects and better cure rates than previous drugs on the market. When it was launched, analysts predicted telaprevir would generate about $2 billion in annual sales. But, with the coming of Gilead Sciences ’s Sovaldi and Harvoni, Vertex’s drug has gone by the wayside. Last year Gilead’s two drugs generated more than $20 billion in combined revenues.

Kalydeco and Orkambi generated combined revenue of about $1 billion in 2015 and are likely to earn more this year. Unlike Harvoni and Sovaldi though, the two CF drugs are not cures, and as Xconomy’s Ben Fidler notes, “competition looms.” Fidler published a lengthy piece on the Vertex’s history, including the financial blows it took in the HCV market, including the company’s 2014 decision to remove telaprevir from the market and abandoning research into HCV.

Although the company is now one of the key leaders in the cystic fibrosis market, Fidler said Vertex cannot become complacent and should look to “diversify into new areas without losing its key franchise.” Diversification is necessary since Vertex lowers its sales projections for 2016, Fidler said. As a result, Vertex tapped a new chief of research and development, David Altshuler, to develop treatments for other illnesses, particularly those caused by easily identifiable genetic mutations. Jeffrey Leiden, Vertex’s chief executive officer, told Fidler the company is now aiming at making transformative medicines for serious specialty diseases.” Right now though, Fidler notes, the Vertex pipeline does not reflect that new shift, with projects aimed at cancer, spinal cord injury and pain treatments.

While Vertex may be looking to dive deeper into the rare disease market, the company has not given up on its cystic fibrosis drugs. It continues to focus on finding new treatment options—either developed in-house or by other companies. Leiden said the company scours the industry for CF drugs. Additionally, Vertex is looking at cutting edge methods of gene editing or messenger RNA treatments for cystic fibrosis in hopes of developing long-lasting or permanent cures for the lung disease, Fidler said.

Earlier this year, Vertex terminated clinical development of one late stage trial studying the combination of its investigational cystic fibrosis compound VX-661 approved cystic fibrosis drug Kalydeco following the recommendation of an independent data safety monitoring board.

Although Vertex has halted the combination trial of VX-661 and Kalydeco for the one copy mutation, the company is continuing with other studies for the two medication combination—a study of VX-661 and Kalydeco in people with two copies of the F508del mutation and another trial for patients with one copy of the F508del mutation and one copy of a residual function mutation. Vertex said it completed enrollment this week for the trial of CF patients with two copies and anticipated enrollment of the second trial in September.

Despite the setback in the combination trial, Vertex said it plans to submit a New Drug Application to the U.S. Food and Drug Administration in the second half of 2017 for the combination treatment pending data from the other Phase III programs. The NDA is expected to include data from the study in people with minimal function mutations, the company said.

Leiden said the company is being aggressive in the CF market because it does not want to repeat its past mistakes in the HCV market when it lost out to Gilead and discontinued telaprevir.

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