Vertex Pharmaceuticals Outlines 2015 Business Priorities

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) outlined 2015 business priorities to support the development, approval and launch of new medicines that treat the underlying cause of cystic fibrosis or CF.

Vertex said it entered 2015 with approximately $1.4 billion in cash, cash equivalents and marketable securities. In July 2014, Vertex entered into a credit agreement that provides for a secured loan of up to $500 million, $300 million of which Vertex received in July 2014 and is currently outstanding.

Vertex expects total 2015 Kalydeco net revenues of $560 million to $580 million. Vertex expects to report total 2014 Kalydeco net revenues of approximately $460 million and fourth quarter kalydeco net revenues of approximately $120 million.

The company expects that its combined non-GAAP R&D and SG&A expenses in 2015 will be in the range of $1.05 billion to $1.1 billion. The increase as compared to 2014 is primarily a result of launch preparation activities for lumacaftor in combination with ivacaftor and the planned pivotal Phase 3 development program for VX-661 in combination with ivacaftor. Vertex's expected non-GAAP R&D and SG&A expenses exclude stock-based compensation expense and certain other expenses recorded in 2015.

Following the expansion of the KALYDECO label throughout 2014, more than 3,100 people ages 6 and older are currently eligible for treatment with KALYDECO, including people with one of eight gating mutations and, in the United States, people with the R117H mutation. Vertex currently expects that by the end of 2015, more than 3,700 people may be eligible for treatment with KALYDECO, including children ages 2 to 5 and, in Europe, adults with the R117H mutation.

Vertex announced that its New Drug Application (NDA) for the use of ivacaftor in children ages 2 to 5 who have the G551D or one of the eight additional gating mutations was accepted for filing by the Food and Drug Administration (FDA), and a target review date of March 17, 2015 was set under the Prescription Drug User Fee Act (PDUFA) for the FDA's approval decision.

Vertex announced that the FDA has accepted its NDA for the combination of lumacaftor and ivacaftor in people with CF ages 12 and older who have two copies of the F508del mutation. The FDA granted Vertex's request for Priority Review, with a target review (PDUFA) date of July 5, 2015 for the FDA's approval decision.

In Europe, the MAA for this combination regimen has been validated by the EMA and is under review by the CHMP. The CHMP granted Vertex's request for Accelerated Assessment of the MAA, which is given to new medicines of major public health interest and shortens the review time from approximately 210 to 150 days for the CHMP to give an opinion following the start of the review.

Vertex also plans to initiate a study of lumacaftor in combination with ivacaftor in children ages 6 to 11 who have two copies of the F508del mutation in the first half of 2015. The study is expected to evaluate the combination regimen as part of a single-arm, open-label design in approximately 50 children. The primary endpoint of the study will be safety and pharmacokinetics.

Vertex is currently conducting a 12-week Phase 2 study of VX-661 in combination with ivacaftor in people with CF who have two copies of the F508del mutation. Vertex completed enrollment in the study in October 2014. The study enrolled approximately 20 patients who received placebo and approximately 20 patients who received one of two doses of VX-661 (50 mg q12h or 100 mg QD) in combination with ivacaftor (150 mg q12h). The primary endpoint of the study is safety, and additional secondary endpoints will evaluate the pharmacokinetics and efficacy of VX-661. 12-week data from this study are expected in the first quarter of 2015.

Vertex stated that it plans to initiate a Phase 3 pivotal program of VX-661 in combination with ivacaftor in February. The initiation of the Phase 3 program is based on safety and efficacy data from Phase 2 studies of VX-661, including interim data from the ongoing 12-week Phase 2 study and previously completed studies of VX-661 in combination with ivacaftor in people with two copies of the F508del mutation and in people with one copy of the F508del mutation and one copy of the G551D mutation, and recent regulatory discussions regarding the design of the Phase 3 program.