Vertex Pharma (VRTX) Misses Q3 EPS by 2c; Plans to Commence Two CF-Focused Phase 2s

October 25, 2016 4:04 PM EDT

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Vertex Pharma (NASDAQ: VRTX) reported Q3 EPS of $0.16, $0.02 worse than the analyst estimate of $0.18. Revenue for the quarter came in at $410 million versus the consensus estimate of $418.37 million.

Vertex also announced the planned initiation of two Phase 2 studies to evaluate the next-generation correctors VX-440 and VX-152 in triple combination regimens with tezacaftor (VX-661) and ivacaftor in people with cystic fibrosis (CF). The Phase 2 study of VX-440 is designed to evaluate the safety and efficacy of 4-week dosing of VX-440 in combination with tezacaftor and ivacaftor in approximately 40 people with CF who have one F508del mutation and one minimal function mutation and approximately 25 people with two copies of the F508del mutation. The first data from this study are expected in the second half of 2017 and are intended to support the initiation of Phase 3 development for VX-440. The Phase 2 study of VX-152 will evaluate 2 weeks of triple combination dosing in approximately 35 people with CF who have one F508del mutation and one minimal function mutation and approximately 25 people with two copies of the F508del mutation. Data from the study of VX-152 are also expected in the second half of 2017 and are intended to support the initiation of a longer-duration Phase 2b or registrational program for VX-152.

Vertex has submitted Investigational New Drug applications to the U.S. Food and Drug Administration (FDA) for both VX-440 and VX-152 and expects to start both studies by the end of 2016.

Vertex also announced today that it plans to begin Phase 1 development of an additional next-generation corrector, VX-659, by the end of 2016. The Phase 1 study is expected to enroll healthy volunteers and will also include an arm to evaluate triple combination dosing in CF patients who have one F508del mutation and one minimal function mutation. Based on data from this study, Vertex plans to start a Phase 2 study of VX-659 in the second half of 2017. The company also expects to advance a fourth next-generation corrector into Phase 1 development in 2017.

“We are committed to advancing multiple next-generation correctors in parallel to bring the best potential treatments to all people with CF who have at least one F508del mutation,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We believe that the combination of a next-generation corrector with tezacaftor and ivacaftor has the potential to benefit a broad range of people with this disease, including those with minimal function mutations who do not yet have a medicine to treat the underlying cause of their CF.”

“KALYDECO and ORKAMBI are significant medical advances for many people with CF, however a very large number of our patients still do not have medicine to treat the cause of their disease,” said Patrick Flume, M.D., Director of the Medical University of South Carolina Cystic Fibrosis Center and Principal Investigator for the Phase 2 VX-152 study. “The studies announced today are a promising step forward for patients and for the treatment of this devastating disease. I look forward to working with Vertex to move these potential medicines through development and toward patients as rapidly as possible.”

The initiation of the Phase 2 studies of VX-440 and VX-152 is based on data from preclinical studies as well as recently completed Phase 1 studies for VX-440 and VX-152 that each enrolled approximately 100 healthy volunteers. Additional details for each Phase 2 study are provided below.

About the VX-440 Phase 2 Study

The Phase 2 study of VX-440 includes three parts. Part A of the study is designed to evaluate triple combination dosing for 4 weeks in approximately 40 patients ages 18 and older with one F508del mutation and one minimal function mutation. Part B of the study is designed to evaluate triple combination dosing for 4 weeks in approximately 25 patients ages 18 and older with two copies of the F508del mutation. A detailed study design is provided in Table 1.

Part A Cohort 1a will evaluate low-dose VX-440 in combination with tezacaftor and ivacaftor. Cohort 1a is designed to confirm that the safety and pharmacokinetic properties observed in healthy volunteers are similar in people with CF. The doses for Part A Cohort 1b and Part B will be confirmed based on review of data from Part A Cohort 1a.

The primary endpoints of the study are safety and tolerability and the absolute change in percent predicted forced expiratory volume in one second (ppFEV1). Secondary endpoints will evaluate relative improvement in ppFEV1, change in sweat chloride and change in the CF questionnaire-revised (CFQ-R) respiratory domain score, among others. Women of childbearing potential who enroll in the Phase 2 study of VX-440 will be required to use pre-specified, non-hormonal methods of contraception.

Pending completion of enrollment, Vertex expects to report data from both cohorts of Part A and from Part B in the second half of 2017. The initiation of Phase 3 development is pending data from Parts A and B and discussions with regulatory authorities.

A third part of the study (Part C) is designed to evaluate triple combination dosing for 12 weeks in approximately 130 patients ages 12 and older with one F508del mutation and one minimal function mutation. Part C will be initiated based on data from Part A and Part B and would run in parallel with potential Phase 3 development. Part C is intended to generate 12-week safety and efficacy data and to demonstrate the contribution of the components to the overall effect of the triple combination regimen.

About the VX-152 Phase 2 Study

The Phase 2 study of VX-152 includes two parts. Part A of the study includes up to three dose escalation cohorts and is designed to evaluate triple combination dosing for 2 weeks in approximately 35 patients ages 18 and older with one F508del mutation and one minimal function mutation. Part B of the study includes up to two cohorts and is designed to evaluate triple combination dosing for 2 weeks in approximately 25 patients ages 18 and older with two copies of the F508del mutation. A detailed study design is provided in Table 2.

The primary endpoint of the study is safety and tolerability. Secondary endpoints will evaluate absolute and relative improvement in ppFEV1, change in sweat chloride, and change in the CFQ-R respiratory domain score, among others. Part A Cohort 1a, will evaluate low-dose VX-152 in combination with tezacaftor and ivacaftor. The doses for Part A Cohort 1b, Cohort 1c and Part B will be based on review of data from prior cohorts.

Pending completion of enrollment, Vertex expects to report data from Parts A and B of this study in the second half of 2017. These data are intended to support the initiation of a longer-duration Phase 2b or registrational program.

Advancing Additional Next-Generation Correctors into Phase 1

As part of Vertex’s commitment to find new medicines to treat the cause of CF for all people with the disease, the company is evaluating multiple next-generation correctors in parallel, both in research and development, to maximize the probability of rapidly advancing the best regimens to help the greatest number of people with CF. Consistent with that strategy, Vertex today announced that it plans to begin clinical development of a third next-generation corrector, VX-659, in 2016.

In human bronchial epithelial (HBE) cells with multiple different CFTR genotypes, including cells with two copies of the F508del mutation and cells with one copy of the F508del mutation and one copy of a mutation known to result in minimal CFTR function, the triple combination of VX-659, tezacaftor and ivacaftor exhibited higher maximal efficacy (as measured by percent of normal chloride transport) and enhanced potency compared to other next-generation correctors in triple combination.

A Phase 1 study of VX-659 is expected to begin by the end of 2016 and will evaluate single ascending doses, multiple ascending doses and triple combination dosing with tezacaftor and ivacaftor in healthy volunteers. The Phase 1 study will also include an arm to evaluate triple combination, or placebo, dosing in people with CF who have one copy of the F508del mutation and one copy of a minimal function mutation. Pending results of this study, Vertex plans to initiate a Phase 2 study to evaluate VX-659 in people with CF in the second half of 2017.

In addition to VX-659, Vertex expects to move a fourth next-generation corrector from research into clinical development in 2017.

For earnings history and earnings-related data on Vertex Pharma (VRTX) click here.



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