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Vertex Announces Completion of Phase 2 Study of VX-765 in People with Epilepsy who did not Respond to Previous Treatment
-Results support continued development of VX-765 in people with treatment-resistant epilepsy-
"The use of anti-inflammatory medicines is a completely new way to
approach the treatment of epilepsy, and this study was an important
first step toward understanding whether VX-765 could help people with
this disease," said
About the Study
The double-blind, randomized, placebo-controlled Phase 2a study of VX-765 enrolled 60 people with treatment-resistant partial onset epilepsy, which is a type of epilepsy where the seizures start and occur in a specific part of the brain. The study enrolled and dosed people who did not benefit from the use of at least two currently available medicines for partial epilepsy. Patients received six weeks of treatment with VX-765 or placebo following a six-week baseline period to monitor seizure frequency. All patients in the study had at least six partial seizures during the baseline period. Patients were followed for six weeks after the treatment phase to collect additional safety and seizure information. Patients continued to receive standard medicines for epilepsy throughout the study, in addition to VX-765 or placebo. In the study, 48 people received 900 mg of VX-765 three-times-daily and 12 people received placebo three-times-daily.
Preliminary Analysis
The primary endpoint of the study was the safety and tolerability of VX-765. In the study, the safety profile for VX-765 was similar to that for placebo. The most common adverse events observed across both treatment arms were headache, dizziness, fatigue and gastrointestinal disorders, and the majority of these adverse events were mild to moderate. The only adverse event that was 10 percent or greater in frequency among those treated with VX-765 compared to placebo was dizziness. One person discontinued treatment due to adverse events during the study and was in the VX-765 treatment group.
Key secondary endpoints focused on the clinical activity of VX-765 during the study based on the following:
(1) Percent reduction in seizure rate
(2) Percent of patients with
a 50 percent or greater reduction in seizure frequency, known as the
responder-rate
(3) Percent of patients who were seizure-free in the
last two weeks of treatment
In addition to the endpoints specified in the study protocol, a number of additional analyses were conducted to evaluate the clinical activity of VX-765 during the last two weeks of the treatment phase and first two weeks of the follow-up period. These evaluations included:
(1) Percent of patients who were seizure-free during the first two weeks
of the follow-up period only
(2) Percent reduction in seizure rate
during the last two weeks of the treatment phase and first two weeks of
the follow-up period only
(3) Percent of patients with a 50 percent
or greater reduction in seizure frequency, known as the responder-rate,
during the last two weeks of the treatment phase and first two weeks of
the follow-up period only
Results for these endpoints and additional evaluations are as follows:
| Key Secondary Endpoints: | VX-765 | Placebo | Treatment Difference | |||||||||
|
-- Percent reduction in seizure rate -- Percent of patients with a 50 percent or greater reduction in seizure frequency, known as the responder-rate -- Percent of patients who were seizure-free in the last two weeks of treatment |
13% - 19% | 0% - 9% | 9% - 13% | |||||||||
| Additional Analyses: | ||||||||||||
|
-- Percent of patients who were seizure-free during the first two weeks of the follow-up period only -- Percent reduction in seizure rate during the last two weeks of the treatment phase and first two weeks of the follow-up period only -- Percent of patients with a 50 percent or greater reduction in seizure frequency, known as the responder-rate, during the last two weeks of the treatment phase and first two weeks of the follow-up period only |
19% - 31% | 0% - 9% | 19% - 23% | |||||||||
While none of the observed differences outlined above were statistically
significant, the improvements observed in the last two weeks of the
treatment phase and first two weeks of the follow-up period suggest that
the clinical activity observed for VX-765 was greater at the end of the
six-week treatment phase and continued during the start of the follow-up
phase. These results indicate that a longer-duration study may be needed
to characterize any additional or more robust clinical activity of
VX-765. Vertex plans to further evaluate VX-765 as part of a larger
Phase
About VX-765
VX-765 is an oral medicine in development that is designed to inhibit Caspase-1, an enzyme involved in the production of IL-1 beta and linked to a wide range of immune and inflammatory responses. VX-765 has been shown to inhibit acute partial seizures in preclinical models and has shown activity in preclinical models of chronic partial epilepsy that do not respond to currently available medicines for epilepsy. Vertex holds worldwide rights to VX-765.
About Epilepsy
Epilepsy is a chronic neurological disorder characterized by recurrent
seizures resulting from overactive neurons in the brain. Recent studies
suggest that inflammation and overproduction of IL-1 beta may be
associated with epileptic seizures.1
About Vertex
Vertex creates new possibilities in medicine. Our team aims to discover, develop and commercialize 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, epilepsy and other life-threatening diseases.
Founded more than 20 years ago in
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 statements
regarding (i) the results from the completed Phase 2 study supporting
continued development of VX-765 in people with treatment-resistant
epilepsy; (ii) the expectation that Vertex will begin a larger and
longer-duration Phase
1 Current Opinion in Investigational Drugs. 2010;
11(1): 34-50
2 Hirtz D, Thurman DJ, Gwinn-Hardy K,
Mohamed M, Chaudhuri AR, Zalutsky R: How common are the "common"
neurologic disorders? Neurology 2007, 68:326-337
3Current Opinion in Investigational Drugs. 2010; 11(1): 34-50
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