- Data from long-term follow-up of patients in
clinical trials further demonstrate durability of the
transformative benefits of CASGEVY™ -
- Safety profile consistent with busulfan
conditioning and autologous hematopoietic stem cell transplant
-
- Vertex provides update on progress in
bringing CASGEVY to patients -
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced longer-term data for CASGEVY™ (exagamglogene autotemcel)
from global clinical trials in people with severe sickle cell
disease (SCD) or transfusion-dependent beta thalassemia (TDT).
CASGEVY is the first and only approved CRISPR/Cas9 gene-edited
therapy.
The results, presented at the American Society of Hematology
(ASH) Annual Meeting and Exposition, continue to demonstrate the
transformative, durable clinical benefits of CASGEVY. The longest
follow up for both SCD and TDT patients now extends more than 5
years, with a median of 33.2 months and 38.1 months,
respectively.
“These comprehensive data provide additional evidence of the
benefits of eradicating transfusion requirements for people with
transfusion-dependent beta thalassemia and vaso-occlusive crises
for those with sickle cell disease,” said Franco Locatelli, M.D.,
Ph.D., Professor of Pediatrics at the Catholic University of the
Sacred Heart of Rome, Director of the Department of Pediatric
Hematology and Oncology at Bambino Gesù Children’s Hospital, Chair
of Vertex’s TDT Program Steering Committee, and Presenting Author
of the CASGEVY clinical data at ASH. “With median follow-up around
three years there is strong evidence for the durability of these
beneficial effects following treatment with CASGEVY.”
“CASGEVY is changing the outlook for people living with sickle
cell disease and beta thalassemia, with these data reinforcing the
immense clinical value a durable one-time therapy can provide to
patients,” said Carmen Bozic, M.D., Executive Vice President,
Global Medicines Development and Medical Affairs, and Chief Medical
Officer at Vertex. “We have a strong commitment to build on our
progress in bringing CASGEVY to patients around the world.”
New long-term follow-up data presented from the CASGEVY
trials
- In SCD, 39/42 (93%) evaluable patients (those with at least 16
months of follow-up) were free from vaso-occlusive crises (VOCs)
for at least 12 consecutive months (VF12) in CLIMB-121 and
CLIMB-131 combined. The mean duration of VOC-free was 30.9 months,
with a maximum of 59.6 months.
- The three evaluable patients who have not achieved VF12 have
derived meaningful clinical benefit including by reducing their
rate of hospitalization for VOCs by 91%, 71% and 100%.
- In TDT, 53/54 (98%) evaluable patients (those with at least 16
months of follow-up) achieved transfusion-independence for at least
12 consecutive months with a weighted average hemoglobin of at
least 9 g/dL (TI12) in CLIMB-111 and CLIMB-131 combined. The mean
duration of transfusion independence was 34.5 months, with a
maximum of 64.1 months.
- The one evaluable patient who has not yet achieved TI12 has
been transfusion free for 8.2 months.
- Both SCD and TDT patients reported sustained and clinically
meaningful improvements in their quality of life, including
physical, emotional, social/family and functional well-being, and
overall health status.
- The safety profile of CASGEVY continues to be generally
consistent with myeloablative conditioning with busulfan and
autologous hematopoietic stem cell transplant.
- Patients continue to demonstrate stable levels of fetal
hemoglobin (HbF) and allelic editing across all ages and genotypes
in the trials.
Vertex had seven abstracts accepted at the ASH annual meeting
as outlined below:
- Oral presentation, Abstract #512, entitled “Durable Clinical
Benefits with Exagamglogene Autotemcel for Transfusion-Dependent
β-Thalassemia”
- Poster presentation, Abstract #4954, entitled “Durable Clinical
Benefits with Exagamglogene Autotemcel for Severe Sickle Cell
Disease”
- Poster presentation, Abstract #1098, entitled “Estimated
Prevalence of β-Thalassemia in the United States in 2023”
- Publication only, Abstract #7454, entitled “Health-Related
Quality-of-Life Improvements after Exagamglogene Autotemcel in
Patients with Transfusion-Dependent Beta Thalassemia”
- Publication only, Abstract #7453, entitled “Health-Related
Quality-of-Life Improvements after Exagamglogene Autotemcel in
Patients with Severe Sickle Cell Disease”
- Publication only, Abstract #7660, entitled “Adherence, Clinical
and Economic Outcomes in Patients with Sickle Cell Disease with
Recurrent Vaso-Occlusive Crises Treated with L-Glutamine,
Voxelotor, or Crizanlizumab Covered By Medicaid and Commercial
Insurance in the United States”
- Publication only, Abstract #7661, entitled “Clinical
Complications and Healthcare Resource Utilization in Medicaid and
Commercially Insured Patients with Sickle Cell Disease Receiving
Frequent Red Blood Cell Transfusions”
Progress in bringing CASGEVY to patients around the
world
CASGEVY is approved for both SCD and TDT in the U.S., the
European Union, Great Britain, Canada, Switzerland, Bahrain and the
Kingdom of Saudi Arabia, and Vertex plans to make submissions in
the United Arab Emirates and Kuwait. More than 45 authorized
treatment centers have been activated globally to support the
delivery of CASGEVY, and more than 40 patients have had a first
cell collection.
Vertex is continuing to work with reimbursement authorities to
secure sustainable access for patients. Through this work, Vertex
has agreements to provide CASGEVY in multiple countries, including
the U.S., England (TDT), Austria, Bahrain and the Kingdom of Saudi
Arabia, and continues to make strong progress in others, including
positive Health Technology Assessments (HTAs) in Canada for both
diseases and advancing access negotiations for SCD patients in
England. In the U.S., Vertex recently secured an industry-first,
voluntary agreement with the Centers for Medicare & Medicaid
Services (CMS) on a single outcomes-based arrangement available to
all state Medicaid programs to ensure broad and equitable access to
CASGEVY. To support this progress on patient access and growing
patient demand, Vertex has received approval for a third
manufacturing facility for CASGEVY with our partner Lonza.
About Sickle Cell Disease (SCD)
SCD is a debilitating, progressive and life-shortening disease.
SCD patients report health-related quality of life scores well
below the general population, and the lifetime health care costs in
the U.S. of managing SCD for patients with recurrent VOCs is
estimated between $4 and $6 million. SCD is an inherited blood
disorder that affects the red blood cells, which are essential for
carrying oxygen to all organs and tissues of the body. SCD causes
severe pain, organ damage and shortened life span due to misshapen
or “sickled” red blood cells. The clinical hallmark of SCD is VOCs,
which are caused by blockages of blood vessels by sickled red blood
cells and result in severe and debilitating pain that can happen
anywhere in the body at any time. SCD requires a lifetime of
treatment and results in a reduced life expectancy. In the U.S.,
the median age of death for patients living with SCD is
approximately 45 years. A cure for SCD today is a stem cell
transplant from a matched donor, but this option is only available
to a small fraction of patients living with SCD because of the lack
of available donors.
About Transfusion-Dependent Beta Thalassemia (TDT)
TDT is a serious, life-threatening genetic disease. TDT patients
report health-related quality of life scores below the general
population and the lifetime health care costs in the U.S. of
managing TDT are estimated between $5 and $5.7 million. TDT
requires frequent blood transfusions and iron chelation therapy
throughout a person’s life. Due to anemia, patients living with TDT
may experience fatigue and shortness of breath, and infants may
develop failure to thrive, jaundice and feeding problems.
Complications of TDT can also include an enlarged spleen, liver
and/or heart, misshapen bones and delayed puberty. TDT requires
lifelong treatment and significant use of health care resources,
and ultimately results in reduced life expectancy, decreased
quality of life and reduced lifetime earnings and productivity. In
the U.S., the median age of death for patients living with TDT is
37 years. Stem cell transplant from a matched donor is a curative
option but is only available to a small fraction of people living
with TDT because of the lack of available donors.
About CASGEVY™ (exagamglogene autotemcel [exa-cel])
CASGEVY™ is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell
therapy for eligible patients with SCD or TDT, in which a patient’s
own hematopoietic stem and progenitor cells are edited at the
erythroid specific enhancer region of the BCL11A gene through a
precise double-strand break. This edit results in the production of
high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood
cells. HbF is the form of the oxygen-carrying hemoglobin that is
naturally present during fetal development, which then switches to
the adult form of hemoglobin after birth. CASGEVY has been shown to
reduce or eliminate VOCs for patients with SCD and transfusion
requirements for patients with TDT.
CASGEVY is approved for eligible SCD and TDT patients 12 years
and older by multiple regulatory bodies around the world.
About the CLIMB Trials
The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and
CLIMB-121, are designed to assess the safety and efficacy of a
single dose of CASGEVY in patients ages 12 to 35 years with TDT or
with SCD and recurrent VOCs. The trials are closed for enrollment.
Patients will be followed for approximately two years after CASGEVY
infusion in these trials. Each patient will be asked to participate
in the ongoing long-term, open-label trial, CLIMB-131. CLIMB-131 is
designed to evaluate the long-term safety and efficacy of CASGEVY
in patients who received CASGEVY, including those in other CLIMB
trials. The trial is designed to follow patients for up to 15 years
after CASGEVY infusion.
U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY
(exagamglogene autotemcel)
WHAT IS CASGEVY?
CASGEVY is a one-time therapy used to treat people aged 12 years
and older with:
• sickle cell disease (SCD) who have frequent vaso-occlusive
crises or VOCs
• beta thalassemia (β-thalassemia) who need regular blood
transfusions
CASGEVY is made specifically for each patient, using the
patient’s own edited blood stem cells, and increases the production
of a special type of hemoglobin called hemoglobin F (fetal
hemoglobin or HbF). Having more HbF increases overall hemoglobin
levels and has been shown to improve the production and function of
red blood cells. This can eliminate VOCs in people with sickle cell
disease and eliminate the need for regular blood transfusions in
people with beta thalassemia.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about
CASGEVY?
After treatment with CASGEVY, you will have fewer blood cells
for a while until CASGEVY takes hold (engrafts) into your bone
marrow. This includes low levels of platelets (cells that usually
help the blood to clot) and white blood cells (cells that usually
fight infections). Your doctor will monitor this and give you
treatment as required. The doctor will tell you when blood cell
levels return to safe levels.
- Tell your healthcare provider right away if you
experience any of the following, which could be signs of low levels
of platelet cells:
- severe headache
- abnormal bruising
- prolonged bleeding
- bleeding without injury such as nosebleeds; bleeding from gums;
blood in your urine, stool, or vomit; or coughing up blood
- Tell your healthcare provider right away if you
experience any of the following, which could be signs of low levels
of white blood cells:
You may experience side effects associated with other medicines
administered as part of the treatment regimen for CASGEVY. Talk to
your physician regarding those possible side effects. Your
healthcare provider may give you other medicines to treat your side
effects.
How will I receive CASGEVY?
Your healthcare provider will give you other medicines,
including a conditioning medicine, as part of your treatment with
CASGEVY. It’s important to talk to your healthcare provider about
the risks and benefits of all medicines involved in your
treatment.
After receiving the conditioning medicine, it may not be
possible for you to become pregnant or father a child. You should
discuss options for fertility preservation with your healthcare
provider before treatment.
STEP 1: Before CASGEVY treatment, a doctor will give you
mobilization medicine(s). This medicine moves blood stem cells from
your bone marrow into the blood stream. The blood stem cells are
then collected in a machine that separates the different blood
cells (this is called apheresis). This entire process may happen
more than once. Each time, it can take up to one week.
During this step rescue cells are also collected and stored at
the hospital. These are your existing blood stem cells and are kept
untreated just in case there is a problem in the treatment process.
If CASGEVY cannot be given after the conditioning medicine, or if
the modified blood stem cells do not take hold (engraft) in the
body, these rescue cells will be given back to you. If you are
given rescue cells, you will not have any treatment benefit from
CASGEVY.
STEP 2: After they are collected, your blood stem cells
will be sent to the manufacturing site where they are used to make
CASGEVY. It may take up to 6 months from the time your cells are
collected to manufacture and test CASGEVY before it is sent back to
your healthcare provider.
STEP 3: Shortly before your stem cell transplant, your
healthcare provider will give you a conditioning medicine for a few
days in hospital. This will prepare you for treatment by clearing
cells from the bone marrow, so they can be replaced with the
modified cells in CASGEVY. After you are given this medicine, your
blood cell levels will fall to very low levels. You will stay in
the hospital for this step and remain in the hospital until after
the infusion with CASGEVY.
STEP 4: One or more vials of CASGEVY will be given into a
vein (intravenous infusion) over a short period of time.
After the CASGEVY infusion, you will stay in hospital so that
your healthcare provider can closely monitor your recovery. This
can take 4-6 weeks, but times can vary. Your healthcare provider
will decide when you can go home.
What should I avoid after receiving CASGEVY?
- Do not donate blood, organs, tissues, or cells at any time in
the future
What are the possible or reasonably likely side effects of
CASGEVY?
The most common side effects of CASGEVY include:
- Low levels of platelet cells, which may reduce the ability of
blood to clot and may cause bleeding
- Low levels of white blood cells, which may make you more
susceptible to infection
Your healthcare provider will test your blood to check for low
levels of blood cells (including platelets and white blood cells).
Tell your healthcare provider right away if you get any of the
following symptoms:
- fever
- chills
- infections
- severe headache
- abnormal bruising
- prolonged bleeding
- bleeding without injury such as nosebleeds; bleeding from gums;
blood in your urine, stool, or vomit; or coughing up blood
These are not all the possible side effects of CASGEVY. Call
your doctor for medical advice about side effects. You may report
side effects to FDA at 1-800-FDA-1088.
General information about the safe and effective use of
CASGEVY
Talk to your healthcare provider about any health concerns.
Please see full Prescribing Information including Patient
Information for CASGEVY.
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, primary
membranous nephropathy, autosomal dominant polycystic kidney
disease, type 1 diabetes and myotonic dystrophy type 1.
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 15 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 X.
(VRTX-GEN)
Vertex 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, the statements by Franco
Locatelli, M.D., Ph.D. and Carmen Bozic, M.D., in this press
release, and statements regarding expectations for the anticipated
transformative, durable clinical benefits of CASGEVY, plans to
continue working with reimbursement authorities to secure
sustainable access for patients, including our expectations for
progress in Canada and England, and our plans for and design of the
CLIMB studies. While we believe 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 eligible patient access to CASGEVY may not be achieved
on the anticipated timeline, or at all, that data from the
company's development programs may not support registration or
further development of its compounds due to safety, efficacy, and
other reasons, 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.
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Vertex Pharmaceuticals Incorporated Investors:
InvestorInfo@vrtx.com
Media: mediainfo@vrtx.com or International: +44 20 3204
5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607
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