Vertex Provides Update on its Clinical Programs Targeting Alpha-1 Antitrypsin Deficiency
October 14 2020 - 4:30PM
Business Wire
- Phase 2 study of VX-814 in patients with
alpha-1 antitrypsin deficiency discontinued based upon safety and
pharmacokinetic data -
- Phase 2 study of VX-864 continues to enroll
and dose patients; data expected in H1 2021 -
- AATD research program continues to progress
-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
provided an update on its clinical programs targeting the small
molecule correction of alpha-1 antitrypsin deficiency (AATD).
VX-814 Phase 2 Proof-of-Concept Study
Based on the safety and pharmacokinetic (PK) profile of VX-814
observed to date in a Phase 2 trial in AATD, Vertex has decided to
stop dosing in the trial and will discontinue development of
VX-814. The randomized, double-blind, placebo-controlled Phase 2
study of approximately 50 patients was designed to evaluate the
safety and PK of VX-814, and the ability of VX-814 to increase
functional levels of alpha-1 antitrypsin over 28 days of dosing.
Elevated liver enzymes (AST/ALT) were observed in several patients.
In four patients, across different doses studied, elevations
greater than 8 times the upper limit of normal were noted; the
elevated liver enzymes have either resolved or are resolving. No
patients had concomitant elevation of bilirubin levels and all
patients were asymptomatic. Analysis of the PK data from the study
indicated that exposures achieved were low. Based on these data,
Vertex concluded that it would not be feasible to safely reach
targeted exposure levels, and thus meaningful increases in AAT
levels, with VX-814, and the study was stopped.
“Based on the liver enzyme elevations observed, along with the
determination that we would not be able to safely achieve targeted
exposure levels with VX-814, we are discontinuing further
development of this molecule,” said Carmen Bozic, M.D., Executive
Vice President, Global Medicines Development and Medical Affairs,
and Chief Medical Officer at Vertex. “We are grateful to the AATD
patients and investigators who participated in the VX-814 studies
and we remain committed to transforming the treatment of this
disease. We look forward to continuing clinical study of VX-864 and
other molecules targeting the underlying cause of AATD.”
VX-864 Phase 2 Proof-of-Concept Study
A Phase 2 trial of VX-864, which is structurally distinct from
VX-814, was initiated in July 2020 and is ongoing. This randomized,
double-blind, placebo-controlled study of approximately 40 patients
is designed to evaluate the safety and pharmacokinetics of VX-864,
and the ability of VX-864 to increase functional levels of alpha-1
antitrypsin over 28 days of dosing. Patients continue to enroll and
be dosed in this study, and clinical data from this study are
anticipated in the first half of 2021.
AATD Research Program
Vertex also continues to advance multiple small molecule
correctors in late stage research with a goal of advancing at least
one additional molecule into development in 2021.
About Vertex
Vertex is a global biotechnology company that invests in
scientific innovation to create transformative medicines for people
with serious diseases. The company has multiple approved medicines
that treat the underlying cause of cystic fibrosis (CF) — a rare,
life-threatening genetic disease — and has several ongoing clinical
and research programs in CF. Beyond CF, Vertex has a robust
pipeline of investigational small molecule medicines in other
serious diseases where it has deep insight into causal human
biology, including pain, alpha-1 antitrypsin deficiency and
APOL1-mediated kidney diseases. In addition, Vertex has a rapidly
expanding pipeline of genetic and cell therapies for diseases such
as sickle cell disease, beta thalassemia, Duchenne muscular
dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global
headquarters is now located in Boston's Innovation District and its
international headquarters is in London, UK. Additionally, the
company has research and development sites and commercial offices
in North America, Europe, Australia and Latin America. Vertex is
consistently recognized as one of the industry's top places to
work, including 10 consecutive years on Science magazine's Top
Employers list and top five on the 2019 Best Employers for
Diversity list by Forbes. For company updates and to learn more
about Vertex's history of innovation, visit www.vrtx.com or follow
us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
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. Bozic’s quote and statements
regarding Vertex’s plans and expectations for the clinical trial
evaluating VX-864 and Vertex’s AATD research program. While Vertex
believes the forward-looking statements contained in this press
release are accurate, these forward-looking statements represent
the company's beliefs only as of the date of this press release and
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 data from the company's research and
development programs may not support development or registration of
its compounds due to safety, efficacy or other reasons, 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)
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Media: mediainfo@vrtx.com or U.S.: 617-341-6992 or
International: +44 20 3204 5275
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