– Patient 1 showed blood glucose time-in-range
change from 40.1% on 34.0 units per day of exogenous insulin at
baseline to 99.9% and insulin independence at Day 270 –
– Patient 2 showed blood glucose time-in-range
change from 35.9% on 25.9 units per day of exogenous insulin at
baseline to 51.9% with a 30% reduction in exogenous insulin use at
Day 150 –
– As previously reported, proof-of-concept
achieved with first two patients treated at half the target dose of
VX-880 demonstrating glucose responsive insulin secretion,
improvements in HbA1c and reductions in exogenous insulin –
– VX-880 generally well tolerated in all
patients dosed to date; majority of adverse events were mild or
moderate –
– Two additional abstracts also accepted for
presentation –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
provided additional data on patients dosed in Part A of its Phase
1/2 clinical trial of VX‑880, an investigational stem cell-derived,
fully differentiated pancreatic islet cell replacement therapy for
people with T1D with impaired hypoglycemic awareness and severe
hypoglycemia. As reported last month, both patients treated with
half the target dose of VX-880 achieved glucose-responsive insulin
production, improvements in glycemic control and reductions in
exogenous insulin requirements. The data presented today show
significant increases in the blood glucose time-in-range compared
to baseline, following treatment with VX-880. Time spent in target
range is a clinically important metric in diabetes management that
reflects the amount of time a patient’s blood glucose level is
measured in the desired, target blood sugar range. The accepted
American Diabetes Association (ADA)/European Association for the
Study of Diabetes (EASD) target for time-in-range (blood glucose
between 70 and 180 mg/dL) is 70%.
These data were presented during the American Diabetes
Association 82nd Scientific Sessions Conference on June 6, 2022 in
New Orleans, Louisiana from 9:00 to 9:15 a.m. CDT as an oral
abstract presentation, “Stem Cell-Derived, Fully Differentiated
Islet Cells for Type 1 Diabetes” (abstract/publication #259-OR). A
link to the presentation is included here.
The presentation included data from both patients dosed in Part
A of the study, designed to assess the safety profile of VX-880 at
half the target dose. Patient 1 showed a blood glucose
time-in-range increase from 40.1% at baseline to 99.9% at Day 270
and was insulin independent. Patient 2 showed a time-in-range
increase from 35.9% at baseline to 51.9% at Day 150 with a 30%
reduction in exogenous insulin use.
VX-880 has been generally well tolerated in all patients dosed
to date, with the majority of adverse events being mild or
moderate.
“The glucose time-in-range data presented today at ADA
demonstrate the remarkable glycemic control that can be achieved
after treatment with VX‑880,” said Bastiano Sanna, Ph.D., Executive
Vice President and Chief of Cell and Genetic Therapies at Vertex.
“Elevations in blood sugar are important to control, as are
fluctuations over time, as both increase the risk of complications
in patients with T1D. The first two patients treated with VX-880
have not only achieved improved HbA1c and decreased insulin
requirements, but also higher time-in-range. Taken altogether,
these data provide further evidence of the potential for VX-880 as
a functional cure for people living with T1D.”
“As a treating physician, I have seen the profound burden of
this disease on patients, especially those who experience severe
hypoglycemia. The ability to restore a patient’s islet function and
improve glycemic control, and subsequently reduce exogenous insulin
dependence, has significant potential to improve patients’ lives,”
said Camillo Ricordi, M.D., Professor of Surgery, Director of the
Diabetes Research Institute and the Cell Transplant Center at the
University of Miami Miller School of Medicine, and Steering
Committee Chair for the VX-880 clinical trial. “These results from
the first two patients treated with half of the target dose are
remarkable and encouraging as we continue investigating treating
patients with type 1 diabetes with this stem cell-derived
therapy.”
“The potential impact of this treatment on patients cannot be
overstated,” said James Markmann, M.D., Ph.D., Professor of Surgery
and Chief of the Division of Transplant Surgery at Massachusetts
General Hospital, who treated Patient 1. “This study shows a
significant leap forward in the potential treatment of patients
with type 1 diabetes.”
Additional Vertex presentations at ADA this year included an
oral presentation, “Persistence of Impaired Awareness of
Hypoglycemia, Severe Hypoglycemic Events and Suboptimal Glycemic
Control Despite Advanced Diabetes Technologies”
(abstract/publication #92-OR), and poster, “Gaps Remain in
Achieving Target T1D Glycemic Goals Despite Advanced Technologies”
(poster #652-P).
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.
Encapsulated Islet Cell Program & Development of
Hypoimmune Cells
Vertex is also pursuing additional programs in T1D, including
one in which these same stem cell-derived islets are encapsulated
in an immunoprotective device to be surgically implanted without
the use of concomitant immunosuppression, and another program where
cells are modified to produce hypoimmune stem cell-derived islets.
IND-enabling studies for the encapsulated cells program are
underway, and the company remains on track to submit an IND in
2022.
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 full 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. The Phase 1/2 study is on
clinical hold in the U.S. and is ongoing in Canada.
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
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, as
amended, including, without limitation, (i) statements by Bastiano
Sanna, Ph.D., Dr. Camillo Ricordi and Dr. Jim Markmann 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 IND-enabling studies for the
encapsulated islet cell program, including anticipated regulatory
filings in 2022, 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
617-429-6891
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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