Vertex Announces Sustained Viral Response Rate (SVR4) Data from All-Oral Study of VX-135 in Combination with Daclatasvir in

  Vertex Announces Sustained Viral Response Rate (SVR4) Data from All-Oral
  Study of VX-135 in Combination with Daclatasvir in Hepatitis C

Business Wire

BOSTON -- January 9, 2014

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the first
data from the initial cohorts of an open-label Phase 2a study of VX-135,
Vertex’s nucleotide analogue hepatitis C virus (HCV) polymerase inhibitor, in
combination with daclatasvir, Bristol-Myers Squibb's NS5A replication complex
inhibitor. In an intent-to-treat analysis, the sustained viral response rate
four weeks after the completion of treatment (SVR4) was 83 percent (10 of 12)
in treatment-naïve genotype 1 patients who received 200 mg of VX-135 in
combination with daclatasvir. In this arm, one patient discontinued treatment
after the first dose due to a serious adverse event of vomiting/nausea. The 11
other patients in this arm completed 12 weeks of treatment, and 91 percent (10
of 11) achieved SVR4. In the study, the majority of adverse events were mild.

“We are encouraged by these initial Phase 2a data for VX-135 in combination
with another direct acting antiviral medicine,” said Robert Kauffman, M.D.,
Ph.D., Senior Vice President and Chief Medical Officer at Vertex. “We believe
that VX-135 has the potential to play an important future role in the
treatment of hepatitis C, and we are currently evaluating these data with BMS
to determine the next steps for this combination in people with hepatitis C,
including people with genotypes 1 and 3.”

About the Phase 2a Study

The data announced today are from the first two cohorts of an open-label Phase
2a study of VX-135 in combination with daclatasvir. The initial two cohorts of
the study evaluated 100 mg and 200 mg once-daily doses of VX-135 in
combination with daclatasvir once daily (60 mg) for 12 weeks of total
treatment. Twenty-three people with chronic genotype 1 hepatitis C who were
new to treatment (treatment-naïve) and did not have liver cirrhosis were
enrolled in these cohorts. More than 75 percent of all patients enrolled had
genotype 1a HCV. The majority of adverse events observed in the study were
mild. The most common adverse events observed in greater than 10 percent of
patients across the study were fatigue, headache and nausea. Safety and
efficacy data for the two arms of the study are provided below:

  *200 mg of VX-135 in Combination with Daclatasvir (60 mg): In an
    intent-to-treat analysis, 58 percent (7 of 12) of patients had
    undetectable HCV RNA after 4 weeks of treatment and 83 percent (10 of 12)
    of patients had undetectable HCV RNA four weeks after the completion of
    treatment (SVR4). One patient in this arm experienced a serious adverse
    event of vomiting/nausea, discontinued treatment after the first dose and
    did not acheive SVR4. The 11 other patients in this arm completed 12 weeks
    of treatment, and 91 percent (10 of 11) achieved SVR4. One patient
    relapsed during the follow-up period and did not achieve SVR4.
  *100 mg of VX-135 in Combination with Daclatasvir (60 mg): In an
    intent-to-treat analysis, 73 percent (8 of 11) of patients achieved
    undetectable HCV RNA after 4 weeks of treatment and 73 percent (8 of 11)
    of patients had undetectable HCV RNA four weeks after the completion of
    treatment (SVR4). Two patients in this arm experienced viral breakthrough
    while receiving the combination regimen, and one patient relapsed during
    the follow-up period.

Vertex expects to submit these data for presentation at a medical meeting in
2014.

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 is a global biotechnology company that aims to discover, develop and
commercialize innovative medicines 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 cystic fibrosis,
hepatitis C, rheumatoid arthritis and other life-threatening diseases. In
addition to our clinical development programs, Vertex has more than a dozen
ongoing preclinical programs aimed at other serious and life-threatening
diseases.

Founded in 1989 in Cambridge, Mass., Vertex today has research and development
sites and commercial offices in the United States, Europe, Canada and
Australia. For four years in a row, Science magazine has named Vertex one of
its Top Employers in the life sciences. For additional information and the
latest updates from the company, please visit www.vrtx.com.

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 second paragraph of the press
release. 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 there is a partial clinical hold on VX-135 in the United
States, that the outcomes of Vertex's clinical studies of VX-135 may not be
favorable or support further development of VX-135 due to safety, efficacy, or
other reasons, and the 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 atwww.vrtx.com. Vertex
disclaims any obligation to update the information contained in this press
release as new information becomes available.

(VRTX – GEN)

Contact:

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