Investors Elated as Vertex Picks Two Top CF Triple Combos for Phase III Trials

Vertex is keeping its promise of advancing triple combination treatments for patients with cystic fibrosis and investors are happy. Shares of Vertex Pharmaceuticals were up more than 4 percent in premarket trading after the company announced it was pushing two next-generation correctors into Phase III development in combination with another therapy to treat cystic fibrosis – a core focus of the company.

Vertex said it was advancing VX-659 and VX-445 into Phase III development as part of two different triple combination regimens for people with a difficult form of cystic fibrosis. The combination treatments will target 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 believes these treatments, if successful, could provide treatment for up to 90 percent of people with cystic fibrosis. Boston-based Vertex estimates there are about 75,000 people in North America, Europe and Australia who have cystic fibrosis. Patients diagnosed with cystic fibrosis typically experience difficulty breathing due to a thickening of the mucus lining the lungs. Vertex already markets CF drugs Kalydeco and Orkambi, which allows the company to already treat about 30,000 CF patients.

Vertex Chief Medical Officer Jeffrey Chodakewitz said the company hopes to soon conclude regulatory discussions and advance the treatments into Phase III. He said the company has the goal of bringing a triple action combination regimen to patients as quickly as possible.

VX-659 and VX-445 were selected based on data collected in Phase II, as well as new data from an ongoing Phase II trial, that showed mean absolute improvements in percent predicted forced expiratory volume in one second of up to 13.3 and 13.8 percentage points from baseline through four weeks of treatment.

Vertex has been high on these two compounds for some time. In July, the company announced it had accelerated the development programs for VX-445 and VX-659. Now the company is ready to move the programs into late-stage development in the first half of this year. Following the start of a study of F508del/Min patients, Vertex said it intends to launch a second study to evaluate the VX-659 triple combination in people with two copies of the F508del mutation (F508del/F508del).

Vertex plans to initiate a Phase III program in mid-2018 to evaluate VX-445 with tezacaftor and VX-561 as a once-daily regimen in F508del/Min and F508del/F508delpatients.

Jennifer Taylor-Cousar, co-chair of Vertex’s Triple Combination Steering Committee, said the data the company has gathered so far demonstrated the "rapid advances" being made in treating the underlying causes of cystic fibrosis.

"Together, all the Phase II data to date provide further evidence that the addition of a next-generation corrector to tezacaftor and ivacaftor has the potential to provide substantial clinical benefits to patients with one F508del and one minimal function mutation who don’t currently have a medicine to treat the underlying cause of their CF, as well as to provide additional benefits to patients with at least one F508delmutation who are already eligible for CFTR modulator therapies," Taylor-Cousar said in a statement.

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