Vertex Pharmaceuticals
Jul 25, 2013
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Vertex Provides Update on Ongoing All-Oral Studies of VX-135 in Hepatitis C

-U.S. Study: FDA places partial clinical hold on ongoing Phase 2 U.S. study of VX-135, preventing evaluation of 200 mg dose following observation of elevated liver enzymes in patients receiving 400 mg of VX-135 in combination with ribavirin in Phase 2 study in Europe; evaluation of 100 mg dose continues in U.S.-

-European Study: 12-week dosing complete in 100 mg and 200 mg VX-135 dose groups in combination with ribavirin in Phase 2 study; 70% and 80%, respectively, of patients achieved undetectable HCV RNA by week 4 and treatment was well tolerated with no discontinuations or serious adverse events reported through 12 weeks-

-New Zealand Study: dosing ongoing in Phase 2 study of 100 mg and 200 mg of VX-135 in combination with daclatasvir, an NS5A replication complex inhibitor-

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the company has received notice from the U.S. Food and Drug Administration (FDA) that a partial clinical hold has been placed on Vertex's ongoing Phase 2 U.S. study of the nucleotide analogue hepatitis C virus (HCV) polymerase inhibitor VX-135. The partial clinical hold prevents evaluation of a 200 mg dose of VX-135 in the U.S. study following observation of reversible elevated liver enzymes in patients receiving 400 mg of VX-135 in combination with ribavirin in a Phase 2 study in Europe. Evaluation of a 100 mg dose of VX-135 in combination with ribavirin as part of the 12-week Phase 2 study in the U.S. is continuing as planned.

Vertex recently completed dosing of 100 mg and 200 mg of VX-135 in combination with ribavirin as part of the 12-week Phase 2 study in Europe, and both doses were well tolerated with no discontinuations. No serious adverse events have been reported and no liver or cardiac safety issues have been identified. Vertex also recently initiated dosing of 100 and 200 mg of VX-135 in combination with daclatasvir as part of a Phase 2 study in New Zealand.

"Developing safe and effective medicines for patients is our goal," said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer at Vertex. "We are committed to continuing to work closely with the FDA to provide the data needed to support evaluation of a 200 mg dose of VX-135 in the U.S."

Ongoing Studies of VX-135

Multiple studies of VX-135 as part of all-oral treatment regimens are ongoing, including:

About VX-135

VX-135 is a uridine nucleotide analogue pro-drug designed to inhibit the replication of the hepatitis C virus by acting on the NS5B polymerase. Vertex gained worldwide rights to ALS-2200, known as VX-135 in Phase 2 studies, through an exclusive licensing agreement signed with Alios BioPharma, Inc. in June 2011.

About Vertex

Vertex creates new possibilities in medicine. Our team discovers, develops and commercializes innovative therapies so people with serious diseases can lead better lives.

Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, rheumatoid arthritis and other life-threatening diseases.

Founded more than 20 years ago in Cambridge, Mass., we now have ongoing worldwide research programs and sites in the U.S., U.K. and Canada. Today, Vertex has more than 2,000 employees around the world, and for three years in a row, Science magazine has named Vertex one of its Top Employers in the life sciences.

Vertex Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Kauffman's statements in the third paragraph of the press release and statements regarding (i) Vertex's expectation that it will complete submission of additional clinical, preclinical and pharmacokinetic data in the fourth quarter of 2013; (ii) the plan to complete the evaluation of the 100 mg dose of VX-135 in the United States; (iii) the timing of availability of data from the U.S. and European studies of VX-135 in combination with ribavirin and from the first part of the study of VX-135 in combination with daclatasvir; (iv) the plan to expand the study of VX-135 and daclatasvir to enroll additional patients with both genotypes 1 and 3 HCV infection; and (v) the planned combination study of VX-135 and simeprevir. While Vertex believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the partial clinical hold prevents evaluation of the 200 mg dose of VX-135 in the United States, that the clinical development program for VX-135 may be delayed by the partial clinical hold, that the FDA may not lift the partial clinical hold on VX-135 or allow the company to pursue further development of VX-135 in the United States, that the outcomes of Vertex's planned and ongoing clinical studies of VX-135 may not be favorable, that VX-135 may not be safe or efficacious and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

VRTX — GEN

Vertex Contacts:
Media:
Zach Barber, 617-341-6470
mediainfo@vrtx.com
or
Investors:
Michael Partridge, 617-341-6108
or
Kelly Lewis, 617-961-7530

Source: Vertex Pharmaceuticals Incorporated

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