Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced three abstracts, including two oral presentations and one
poster, detailing data on type 1 diabetes (T1D) and its Phase 1/2
trial of VX-880, a stem cell‑derived, fully differentiated
pancreatic islet cell replacement therapy, have been accepted for
presentation during the American Diabetes Association 82nd Annual
Scientific Sessions Conference held June 3-7, 2022 in New Orleans,
Louisiana.
Abstract and publication #259-OR entitled “Stem Cell-Derived,
Fully Differentiated Islet Cells for Type 1 Diabetes,” will be
presented on Monday, June 6, 2022 at 9:00 a.m. CDT during the oral
presentation session “Other Therapeutic Agents,” which runs from
8:00 to 10:00 a.m. CDT. The abstract posted today details results
from the Phase 1/2 clinical trial, demonstrating VX‑880 can restore
insulin production and glucose control in T1D. Data will be updated
and additional information will be presented at the conference.
Abstract and publication #92‑OR entitled “Persistence of
Impaired Awareness of Hypoglycemia, Severe Hypoglycemic Events and
Suboptimal Glycemic Control Despite Advanced Diabetes
Technologies,” to be presented Saturday, June 4, 2022 at 1:45 p.m.
CDT during the oral presentation session “Glucose Monitoring and
Sensing,” which runs from 1:45 to 3:45 p.m. CDT. The abstract
posted today, describing an online survey of 2,044 adults with T1D
in the T1D Exchange registry and online communities, shows that in
patients with high rates of diabetes technology adoption, such as
continuous glucose monitoring, insulin pumps and hybrid closed loop
systems, impaired awareness of hypoglycemia (IAH) and severe
hypoglycemic events (SHEs) persist, with a substantial proportion
of patients not achieving target glycemic control, indicating the
need for novel T1D treatments.
Abstract and poster #652-P entitled “Gaps Remain in Achieving
Target T1D Glycemic Goals Despite Advanced Technologies,” will be
available as part of the general poster session to be held
Saturday, June 4, 2022 from 11:30 a.m. to 2:30 p.m. CDT. The
abstract posted today highlights that, despite improvements in
glycemic control with diabetes technologies, many patients with T1D
are still unable to achieve clinical targets and experience severe
hypoglycemia, emphasizing the need for novel T1D treatments.
The accepted abstracts are now available on the ADA website.
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, statements regarding (1)
the potential benefits, safety, and efficacy of VX‑880, (2) our
plans to present data about VX‑880 at the American Diabetes
Association Annual Conference, including data from our VX‑880
clinical trial and additional scientific presentations regarding
VX‑880, and (3) our plans to continue to progress our Phase 1/2
program for VX‑880. 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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