Vertex Announces FDA Has Lifted the Clinical Hold on VX-880 Phase 1/2 Clinical Trial for the Treatment of Type 1 Diabetes
05 July 2022 - 10:00PM
Business Wire
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration (FDA) has
lifted the clinical hold placed on the Phase 1/2 clinical trial of
VX-880, an investigational stem cell-derived, fully differentiated
pancreatic islet cell replacement therapy for people with type 1
diabetes (T1D) with impaired hypoglycemic awareness and severe
hypoglycemia. As a result, the Phase 1/2 trial will be reopened for
screening, enrollment and dosing at multiple sites in the U.S.
To date, three patients have been dosed in the Phase 1/2 study
with VX-880. Two patients received half the target dose of cells in
Part A of the study. A third patient has received the full target
dose in Part B of the study. Part B will evaluate safety and
efficacy in five patients at the target dose before expanding to
additional patients in Part C.
About VX-880
VX-880 is an investigational allogeneic stem cell-derived, fully
differentiated, insulin-producing islet cell therapy manufactured
using proprietary technology. VX-880 is being evaluated for
patients who have T1D with impaired hypoglycemic awareness and
severe hypoglycemia. VX-880 has the potential to restore the body’s
ability to regulate glucose levels by restoring pancreatic islet
cell function, including glucose responsive insulin production.
VX-880 is delivered by an infusion into the hepatic portal vein and
requires maintenance immunosuppressive therapy to protect the islet
cells from immune rejection.
About the Phase 1/2 Clinical Trial
The clinical trial is a Phase 1/2, multi-center, single-arm,
open-label study in patients who have T1D with impaired
hypoglycemic awareness and severe hypoglycemia. This study is
designed as a sequential, multi-part clinical trial to evaluate the
safety and efficacy of VX-880. In Part A, the first two patients
received half the target dose. In Part B, five patients will
receive the target dose, after which concurrent dosing at the full
target dose will occur in Part C. Approximately 17 patients will be
enrolled in the clinical trial. Enrollment is ongoing in this
study.
About Type 1 Diabetes
T1D results from the autoimmune destruction of insulin-producing
islet cells in the pancreas, leading to loss of insulin production
and impairment of blood glucose control. The absence of insulin
leads to abnormalities in how the body processes nutrients, leading
to high blood glucose levels. High blood glucose can lead to
diabetic ketoacidosis and over time, to complications such as
kidney disease/failure, eye disease (including vision loss), heart
disease, stroke, nerve damage and even death.
Due to the limitations and complexities of insulin delivery
systems, it can be difficult to achieve and maintain balance in
glucose control in people with T1D. Hypoglycemia often results
because of the difficulty in balancing the different factors that
impact glucose levels, including insulin, diet and exercise.
Hypoglycemia remains a critical limiting factor in glycemic
management, and severe hypoglycemia can cause loss of
consciousness, coma, seizures, injury and can be fatal. Over time,
patients with T1D can develop impaired awareness of hypoglycemia,
meaning they are no longer able to perceive the early signs of a
hypoglycemic event, which can be dangerous and result in
life-threatening events.
Current standards of care do not address the underlying causes
of the disease, and there are limited treatment options beyond
insulin for the management of T1D.
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, cell and genetic
therapies in other serious diseases where it has deep insight into
causal human biology, including sickle cell disease, beta
thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes,
alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.
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. 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 12 consecutive years on Science magazine's Top
Employers list and one of the 2021 Seramount (formerly Working
Mother Media) 100 Best Companies. For company updates and to learn
more about Vertex's history of innovation, visit www.vrtx.com or
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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, as
amended, including, without limitation, (i) statements in this
press release, (ii) our plans, expectations for, and the potential
benefits of VX-880, (iii) our plans to continue to progress the
Phase 1/2 program for VX-880 and (iv) our plans for dosing and
enrollment of patients. 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 risks
and uncertainties that could cause actual events or results to
differ materially from those expressed or implied by such
forward-looking statements. Those risks and uncertainties include,
among other things, that data from a limited number of patients may
not be indicative of final clinical trial results, that data from
the company's research and development programs may not support
registration or further development of its compounds due to safety,
efficacy, and other risks listed under the heading “Risk Factors”
in Vertex's most recent annual report and subsequent quarterly
reports filed with the Securities and Exchange Commission at
www.sec.gov and available through the company's website at
www.vrtx.com. You should not place undue reliance on these
statements, or the scientific data presented. Vertex disclaims any
obligation to update the information contained in this press
release as new information becomes available.
(VRTX-GEN)
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Vertex Pharmaceuticals Incorporated Investors:
Michael Partridge, +1 617-341-6108 or Manisha Pai, +1 617-961-1899
or Miroslava Minkova, +1 617-341-6135
Media: mediainfo@vrtx.com or U.S.: +1 617-341-6992 or
Heather Nichols: +1 617-839-3607 or International: +44 20 3204
5275
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