– All 12 patients who received the full dose of
VX-880 as a single infusion demonstrated islet cell engraftment and
glucose-responsive insulin production by Day 90 –
– All patients achieved ADA-recommended target
HbA1c levels <7.0% and >70% time-in-range (70-180 mg/dL), and
11 of 12 patients reduced or eliminated use of exogenous insulin
–
- 3 patients with at least 12 months of
follow-up, and therefore evaluable, met the primary endpoint of
elimination of severe hypoglycemic events (SHEs) with HbA1c
<7.0%, and the secondary endpoint of insulin independence –
- Trial expanded to enroll ~37 participants as
company progresses toward pivotal development –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
presented new data from its Phase 1/2 clinical trial of VX-880, an
investigational stem cell-derived, fully differentiated islet cell
therapy, in people with type 1 diabetes (T1D) with impaired
hypoglycemic awareness and severe hypoglycemic events (SHEs). These
updated data on 12 patients who received the full dose as a single
infusion in Parts B and C of the trial are consistent with
previously reported positive results in the VX-880 trial and
reinforce the transformative potential of this therapy.
At baseline, all patients in the study had undetectable fasting
C-peptide (a marker of endogenous insulin secretion), a history of
recurrent SHEs in the year prior to screening, and required an
average of 39.3 (min, max; 19.8, 52.0) units of insulin per day.
Following a single infusion of VX-880 at the full dose, all 12
patients demonstrated islet cell engraftment and glucose-responsive
insulin production by Day 90. At the latest visit, all patients had
improved glycemic control and achieved ADA-recommended targets for
both HbA1c below 7.0% and time-in-range above 70% on continuous
glucose monitoring. Nearly all participants (11 of 12) had a
reduction or elimination of exogenous insulin use at their last
visit. All patients had elimination of SHEs during the evaluation
period (from Day 90 onward). Finally, all three patients who had at
least one year of follow-up, and are therefore evaluable for the
primary endpoint, met the primary endpoint of elimination of SHEs
(from Day 90 after infusion) with HbA1c <7.0% and the secondary
endpoint of insulin independence.
VX-880 has been generally well tolerated. The majority of
adverse events (AEs) were mild or moderate, and there were no
serious AEs related to VX-880 treatment. As previously reported,
two patient deaths occurred, both unrelated to treatment with
VX-880. The safety profile is generally consistent with the
immunosuppressive regimen used in the study, the infusion
procedure, and complications from long-standing diabetes.
“These remarkable data add to the growing body of evidence for
VX-880 as a potentially curative therapy for T1D,” said Carmen
Bozic, M.D., Executive Vice President, Global Medicines Development
and Medical Affairs, and Chief Medical Officer at Vertex. “As we
plan toward pivotal development, we are pleased to have secured
regulatory approval to expand study enrollment and look forward to
advancing this program for patients who have long awaited a
transformative therapy.”
“The data we’ve seen to-date in this study are extremely
exciting. Stem cell-derived islets regulate blood glucose control
as well as natural human islets,” said Piotr Witkowski, M.D.,
Ph.D., Professor of Surgery and Director, Pancreatic, and Islet
Transplant Program, University of Chicago Medicine, one of the
investigators on the study, and a member of Vertex’s VX-880
Steering Committee. “The marked improvements seen across several
key glycemic measures, the elimination of severe hypoglycemic
episodes, and the reduction or total elimination of reliance on
exogenous insulin have the potential to fundamentally change the
treatment landscape for T1D and alleviate the significant burden
this disease carries for patients.”
These data were presented during the American Diabetes
Association 84th Scientific Sessions Conference on June 21, 2024,
in Orlando, Florida at an invited talk, “Update on Clinical Trials
Using Stem Cell Replacement,” as part of the joint ADA/IPITA
symposium from 3:45 – 5:15 p.m. ET.
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 chronic immunosuppressive therapy to protect the islet
cells from immune rejection. The VX-880 trial has expanded to
additional sites that are currently active and enrolling in the
U.S., Canada, and Europe.
VX-880 has been granted Regenerative Medicine Advanced Therapy
(RMAT) and Fast Track designations by the U.S. FDA, and PRIME
designation by the European Medicines Agency.
About the VX-880 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.
The original 17-patient study is fully enrolled. Fourteen
patients have been dosed in Parts A, B, and C of the study, and the
remaining patients will be dosed soon. Twelve patients received the
full dose as a single infusion in Parts B and C.
At baseline, all 14 patients dosed in the study had undetectable
fasting C-peptide, a history of recurrent SHEs in the year prior to
screening, and required an average of 39.3 (min, max; 19.8, 52.0)
units of insulin per day. Following an infusion of VX-880, all
patients demonstrated islet cell engraftment and glucose-responsive
insulin production by Day 90.
Based on the positive data shown to date, the study has been
expanded to approximately 37 patients.
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. 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;
there is currently no cure for diabetes.
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 approved medicines that
treat the underlying causes of multiple chronic, life-shortening
genetic diseases — cystic fibrosis, sickle cell disease, and
transfusion-dependent beta thalassemia — and continues to advance
clinical and research programs in these diseases. Vertex also has a
robust clinical pipeline of investigational therapies across a
range of modalities in other serious diseases where it has deep
insight into causal human biology, including acute and neuropathic
pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal
dominant polycystic kidney disease, type 1 diabetes, myotonic
dystrophy type 1, and alpha-1 antitrypsin deficiency.
Vertex was founded in 1989 and has its global headquarters in
Boston, with international headquarters in London. Additionally,
the company has research and development sites and commercial
offices in North America, Europe, Australia, Latin America, and the
Middle East. Vertex is consistently recognized as one of the
industry's top places to work, including 14 consecutive years on
Science magazine's Top Employers list and one of Fortune’s 100 Best
Companies to Work For. For company updates and to learn more about
Vertex's history of innovation, visit www.vrtx.com or follow us on
LinkedIn, Facebook, Instagram, YouTube, and Twitter/X.
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 by Carmen Bozic,
M.D., and Piotr Witkowski, M.D., Ph.D., in this press release, (ii)
our plans, expectations for, and the potential benefits of VX-880,
and (iii) our plans for expanding the 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:
Susie Lisa, +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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