Vertex Announces FDA Clearance of Investigational New Drug (IND) Application for VX-880, a Novel Cell Therapy for the Treatme...
January 28 2021 - 4:11PM
Business Wire
- Vertex will initiate a Phase 1/2 clinical
trial in first half of 2021 -
- VX-880 is the first stem cell-derived therapy
evaluating fully differentiated pancreatic islet cells for the
treatment of T1D -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced that the U.S. Food and Drug Administration (FDA) has
cleared the IND, enabling the company to proceed with initiating a
clinical trial for VX-880, an investigational stem cell-derived,
fully differentiated pancreatic islet cell therapy to treat T1D.
Vertex plans to initiate a Phase 1/2 clinical trial in the first
half of 2021 in patients who have T1D with impaired hypoglycemic
awareness and severe hypoglycemia.
“As we celebrate the 100th anniversary of the discovery of
insulin this year, we are excited to bring a first-in-class cell
therapy to the clinic with the potential to meaningfully impact
people living with T1D,” said Bastiano Sanna, Ph.D., Executive Vice
President and Chief of Cell and Genetic Therapies at Vertex. “We
look forward to getting our clinical program underway and testing
our unique approach of replacing pancreatic islet cells, which are
destroyed in people with type 1 diabetes, with our stem
cell-derived fully differentiated insulin-producing pancreatic
islet cells.”
About VX-880 VX-880, formerly known as STx-02, is an
investigational allogeneic human stem cell-derived islet cell
therapy that 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
insulin production.
The VX-880 clinical trial will involve an infusion of fully
differentiated, functional islet cells, as well as the chronic
administration of concomitant 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, single-arm, open-label study in subjects who have T1D
with impaired hypoglycemic awareness and severe hypoglycemia. This
will be a sequential, multi-part clinical trial to evaluate the
safety and efficacy of different doses of VX-880. Approximately 17
patients will be enrolled in the clinical trial.
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 patients with T1D.
Hypoglycemia remains a critical limiting factor in glycemic
management, and severe hypoglycemia can cause loss of
consciousness, coma, seizures, injury, and can be fatal.
There are currently 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 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 cell and
genetic 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. 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 11 consecutive years on Science magazine's Top
Employers list and a best place to work for LGBTQ equality by the
Human Rights Campaign. 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, statements made by Bastiano Sanna, Ph.D., in
this press release, statements regarding the development, plans and
expectations for our T1D pipeline program, including our plans to
initiate a Phase 1/2 clinical trial in people with T1D and expected
timeline of our clinical trials, statements regarding patient
enrollment and dosing, statements regarding potential clinical
trial results and anticipated benefits of VX-880, and our plans to
provide further updates on our T1D pipeline 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 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 the FDA may not
approve our IND, that data from a limited number of patients may
not be indicative of final clinical trial results, that data from
the company's development programs may not support registration or
further development due to safety, efficacy or other reasons, that
the COVID-19 pandemic may impact the status or progress of our
clinical trials, 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. 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 Brenda Eustace, +1
617-341-6187 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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