Vertex Pharma (VRTX) Updates on CF Progress; TRAFFIC, TRANSPORT Phase 3s Full Enrolled

October 17, 2013 9:25 AM EDT Send to a Friend
Vertex Pharmaceuticals, Inc. (Nasdaq: VRTX) provided a comprehensive update on recent progress in its research and development activities in cystic fibrosis (CF) aimed at helping more people with CF and enhancing the clinical benefit for these patients with our approved and investigational medicines. Vertex today announced that the TRAFFIC and TRANSPORT Phase 3 studies of lumacaftor (VX-809) in combination with ivacaftor in people with two copies of the F508del mutation are fully enrolled. Data from these studies are expected in mid-2014, and Vertex plans to submit a New Drug Application (NDA) in the U.S. and a Marketing Authorization Application (MAA) in Europe in the second half of 2014 for the combination of lumacaftor and ivacaftor. Vertex also today provided updates on multiple ongoing label-expansion studies for ivacaftor, ongoing and planned Phase 2 combination studies of lumacaftor and ivacaftor and VX-661 and ivacaftor, and research efforts aimed at beginning clinical development of a next-generation corrector.

These updates were made in conjunction with the 27th Annual North American Cystic Fibrosis Conference (NACFC) in Salt Lake City where additional data from a study of ivacaftor in people with non-G551D gating mutations and data from the PERSIST open-label rollover study will be presented. The company will webcast remarks from its investor presentation at the conference at 2:45 p.m. ET on Friday, October 18. The webcast can be accessed at www.vrtx.com/nacfc2013.

"KALYDECO was the first medicine to treat the underlying cause of CF, and we believe that KALYDECO is just the first step of our work in CF," said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer at Vertex. “Our goal in CF is to help many more people with this disease and to evaluate multiple combinations of CFTR modulators aimed at providing further benefit for people with CF.”

KALYDECOTM (ivacaftor) is currently approved for people with CF ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. More than 2,000 people with CF ages 6 and older have at least one copy of the G551D mutation in North America, Europe and Australia. Vertex has multiple studies planned and underway to evaluate whether ivacaftor may help additional people with CF who have other mutations that may respond to ivacaftor treatment. More than 7,000 people with CF, including those with the G551D mutation, have CFTR mutations that may respond to ivacaftor treatment. The company is also conducting studies to evaluate a combination of ivacaftor and a corrector in people with two copies of the most common CFTR mutation, F508del. Vertex today provided the following updates:

KALYDECO (ivacaftor)

* Global Availability of KALYDECO: KALYDECO is currently available to eligible patients in England, Scotland, Northern Ireland, Wales, the Republic of Ireland, France, Germany, the Netherlands, Austria, Denmark, Sweden, Norway, Greece and the U.S. Vertex is in active discussions with relevant agencies in Australia and Canada regarding public reimbursement of KALYDECO in these countries.
* PERSIST Data Presented at NACFC and Submitted to U.S. FDA: Data from the open-label PERSIST study will be presented as a poster at NACFC. PERSIST is an ongoing rollover study of people with CF ages 6 and older with a G551D mutation who took part in the Phase 3 STRIVE and ENVISON studies of KALYDECO. The data from PERSIST showed that 144 weeks of continuous treatment with KALYDECO provided durable treatment effects in lung function (as measured by FEV1), weight and other measures. The safety profile was consistent with what was observed in the 48-week Phase 3 studies that supported the approval of KALYDECO. The PERSIST data have been submitted to the U.S. Food and Drug Administration (FDA) for review for potential inclusion in the KALYDECO label.

Studies to Expand Number of People Eligible for Ivacaftor

Multiple ongoing studies of ivacaftor monotherapy are designed to evaluate whether additional people with CF may benefit from treatment with ivacaftor alone. These studies include three Phase 3 label-expansion studies and a Phase 2 proof-of-concept study:

* Gating Mutations Study: In September, Vertex announced submission of a supplemental NDA (sNDA) for people with CF ages 6 and older who have at least one non-G551D gating mutation. Vertex also recently submitted an MAA variation in Europe for the same group of people with CF. The sNDA and MAA variation submissions were based on data from a Phase 3 study that showed statistically significant improvements in lung function (FEV1), and other measures of disease. The safety and tolerability results observed in this study were consistent with those observed in prior Phase 3 studies of ivacaftor monotherapy in people with CF who have the G551D mutation. Additional data from this study will be presented at NACFC as part of Symposium III, "CFTR: Matching CFTR Mutations and Drugs," on October 19, 10:30 a.m. - 12:20 p.m. MT. In North America, Europe and Australia, approximately 400 people age 6 and older have at least one non-G551D gating mutation.
* R117H Study: A Phase 3 study of people ages 6 and older with one copy of the R117H mutation is ongoing and fully enrolled. Vertex expects data to be available from this study at the end of 2013 to support a potential sNDA submission in early 2014. A potential MAA variation is planned for the second quarter of 2014. In North America, Europe and Australia, approximately 1,100 people ages 6 and older have at least one R117H mutation. People who have the R117H mutation exhibit a range of severity and signs and symptoms of the disease.
* Study in Children Ages 2 to 5 with Gating Mutations: A Phase 3 study of ivacaftor in children with CF ages 2 to 5 who have a gating mutation is ongoing and fully enrolled. Data from this study are expected in the second quarter of 2014 to support a potential sNDA submission in the second half of 2014. In North America, Europe and Australia, approximately 300 children ages 2 to 5 have a gating mutation.
* Residual Function Study: Enrollment is complete in a Phase 2 proof-of-concept study evaluating ivacaftor in people with CF who have clinical evidence of residual CFTR function. Data from this study are expected in the second quarter of 2014. In North America, Europe and Australia, more than 3,000 people ages 6 and older have non-R117H CFTR mutations that result in residual function.

Phase 3 Program in People with Two Copies (homozygous) of the F508del Mutation

* Phase 3 TRAFFIC and TRANSPORT Studies: Vertex recently completed enrollment of the Phase 3 TRAFFIC and TRANSPORT studies evaluating lumacaftor (VX-809) in combination with ivacaftor in people with CF ages 12 and older who have two copies (homozygous) of the F508del mutation. Vertex expects data from these studies to be available in mid-2014 to support the potential submission of an NDA and MAA for the combination in people homozygous for the F508del mutation in the second half of 2014.
* Vertex also recently began enrollment in two additional studies of lumacaftor in combination with ivacaftor as part of the Phase 3 program. These additional studies are:
-- Study in Children Ages 6 to 11: Vertex recently completed dosing in the pharmacokinetics portion of a Phase 2 study of VX-809 in combination with ivacaftor in children with CF ages 6 to 11 who have two copies of the F508del mutation. Enrollment in the second part of this study is expected to begin in the first quarter of 2014. Data from this study is expected be used for potential subsequent registration of the combination in children ages 6 to 11 in the U.S.
-- Study in People Ages 18 and Older with One Copy (heterozygous) of the F508del mutation: Vertex recently began enrollment in an 8-week exploratory Phase 2 study of lumacaftor in combination with ivacaftor in people 18 and older with one copy (heterozygous) of the F508del mutation on one allele and a second mutation that is not expected to respond to either ivacaftor or lumacaftor alone. This study will evaluate the twice daily (q12h) administration of VX-809 (400mg) and ivacaftor (250mg) to provide additional safety and lung function data on the combination in heterozygous patients.

More than 28,000 people ages 6 and older have two copies of the F508del mutation in North America, Europe and Australia, and more than 17,000 people ages 6 and older have one copy of the F508del mutation and a mutation not expected to respond to ivacaftor treatment.

VX-661 in Combination with Ivacaftor

* 12-Week Phase 2b Study of VX-661 and Ivacaftor in People with Two Copies of the F508del Mutation: Following recent discussions with regulatory authorities, Vertex is preparing to conduct a 12-week study of VX-661 in combination with ivacaftor in people with CF who have two copies of the F508del mutation. The study is designed to evaluate safety, efficacy and dose-response information to characterize VX-661 for further clinical development. Enrollment in this study is expected to begin in the first quarter of 2014, pending protocol approval from regulatory agencies.
* 4-Week Study of VX-661 in Combination with KALYDECO in People with One Copy of Both the G551D and F508del mutation: Vertex recently began enrollment in a Phase 2 study evaluating a 4-week regimen of VX-661 in combination with KALYDECO in people with one copy of the G551D mutation and one copy of the F508del mutation. This is the first proof-of-concept study of a combination of a corrector and KALYDECO in people with these mutations. This study is intended to explore whether the addition of a corrector to treatment with KALYDECO can further enhance the clinical benefit received from KALYDECO alone in people with the G551D and F508del mutations. This combination treatment regimen is supported by in vitro data showing enhanced CFTR function with the combination of VX-661 and ivacaftor. Data from this study are expected in the first quarter of 2014.
* Prioritization of VX-661: Based on the emerging profiles for VX-661 and VX-983, Vertex has prioritized VX-661 over VX-983. The company does not intend to further develop VX-983.

Next-Generation Correctors

* Vertex’s goal is to advance a next-generation corrector into clinical development by the end of 2014. Next-generation correctors could be evaluated as part of potential dual-corrector regimens. The proposed use of a dual-corrector combination regimen is supported by in vitro data that showed a combination of two correctors with ivacaftor increased chloride transport in human bronchial epithelial cells with one or two copies of the F508del mutation, as compared to the use of a single corrector in combination with ivacaftor.

Additional NACFC Presentations

At NACFC, Fred Van Goor, Ph.D., Head of Biology for Vertex's CF program, and David Rodman, M.D., Vice President of Clinical Development for Vertex's CF program, will participate in invited talks regarding their work to discover and develop medicines that target the underlying cause of CF. The talks will take place during Symposium Sessions II and III:

* "Matching Novel Therapies to CF-Causing Mutations in Cell-based Systems." Fred Van Goor, Ph.D, will deliver an invited talk during Symposium Session III, "CFTR: Matching CFTR Mutations & Drugs," on October 19 at 11:30 a.m. MT.
* "Lessons Learned From the Development of First Generation CFTR Modulators." David Rodman, M.D., will deliver an invited talk during Symposium II, "NT/CFTR: Advances in the Therapeutic Pipeline for CFTR Repair (Combination)," on October 18 at 11:55 a.m. MT.


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