- Results from CLIMB-111, -121 and -131
accepted for oral presentation -
- Data from these trials, with the longest
follow-up of more than five years, demonstrate transformative,
consistent and durable benefit of CASGEVY™ -
- Safety profile consistent with busulfan
conditioning and autologous hematopoietic stem cell transplant
-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today
announced longer-term data for CASGEVY™ (exagamglogene autotemcel
[exa-cel]) from global clinical trials in people with severe sickle
cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).
The results, presented at the annual European Hematology
Association (EHA) Congress, confirm the transformative, consistent
and durable clinical benefits of CASGEVY over time. CASGEVY is the
first and only approved CRISPR-based gene-editing therapy.
The data being presented are from more than 100 patients (46
SCD; 56 TDT) treated with exa-cel in clinical trials, with the
longest follow-up now extending more than 5 years. The efficacy
results are consistent with the previously reported primary and key
secondary endpoints analyses from these exa-cel studies and
continue to demonstrate transformative clinical benefit with
durable and stable levels of fetal hemoglobin (HbF) and allelic
editing.
“The transformative benefit seen in patients with sickle cell
disease in the trial is impressive given the significant and
cumulative burden of disease faced by people living with this blood
disorder,” said Haydar Frangoul, M.D., M.S., Medical Director of
Pediatric Hematology and Oncology at Sarah Cannon Research
Institute and HCA Healthcare’s TriStar Centennial Children’s
Hospital. “I am eager to offer this therapy and the opportunity of
a potential functional cure to my eligible patients.”
“The comprehensive data set presented today for adult and
adolescent TDT patients adds to the growing body of evidence for
CASGEVY, and it is important to now ensure the availability of this
innovative treatment to patients in the real world as soon as
possible,” 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. “With the longest follow up now
more than five years, alongside stable editing and sustained fetal
hemoglobin levels, I have conviction in the durable benefit to the
patients treated with CASGEVY.”
New data presented from CASGEVY pivotal trials
- In SCD 36/39 (92.3%) 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), consistent with the
previously reported primary endpoint data. Mean duration of
VOC-free was 27.9 months, with a maximum of 54.8 months.
- 38/39 (97.4%) patients with at least 16 months of follow-up
were free from hospitalizations related to VOCs for at least 12
consecutive months (HF12), consistent with the previously reported
key secondary endpoint data.
- In TDT 49/52 (94.2%) evaluable patients (those with at least 16
months of follow-up) were transfusion-independent for at least 12
consecutive months with a mean weighted hemoglobin of at least 9
g/dL (TI12), consistent with the previously reported primary
endpoint data. Mean duration of transfusion independence was 31.0
months, with a maximum of 59.4 months.
- All TDT patients dosed with at least 16 months of follow up are
transfusion free.
- Two of the three patients who did not achieve TI12 in CLIMB-111
achieved TI12 in the long-term follow-up study, CLIMB-131, and have
been transfusion independent for over one year. The third has been
transfusion free for 3.4 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.
In both SCD and TDT patients, edited levels of BCL11A alleles
were stable over time in bone marrow and peripheral blood
indicating successful editing in the long-term hematopoietic stem
cells. All patients engrafted neutrophils and platelets after
exa-cel infusion. The safety profile of exa-cel was generally
consistent with myeloablative conditioning with busulfan and
autologous hematopoietic stem cell transplant.
These longer-term data for CASGEVY from the CLIMB clinical
trials will be shared as outlined below:
- Abstracts S273 and S274 will be oral presentations entitled
“Exagamglogene Autotemcel For Severe Sickle Cell Disease,” and
“Exagamglogene Autotemcel For Transfusion-Dependent
Beta-Thalassemia,” on Sunday, June 16 at 12:15 CEST and 12:30 CEST,
respectively.
- Abstracts P1493 and P1525 will be poster presentations entitled
“Health-Related Quality Of Life Improvements After Exagamglogene
Autotemcel In Patients With Severe Sickle Cell Disease,” and
“Health-Related Quality Of Life Improvements After Exagamglogene
Autotemcel In Patients With Transfusion-Dependent
Βeta-Thalassemia,” on Friday, June 14 at 18:00 CEST.
- These presentations will include updated pivotal trial data
from patients treated with CASGEVY in CLIMB-111 and CLIMB-121 and
followed in CLIMB-131.
Vertex will also share five health economics abstracts at the
EHA Congress.
- Abstract P1483 is entitled “Adherence, Treatment Use, and
Clinical Outcomes in Patients With Sickle Cell Disease With
Recurrent Vaso-Occlusive Crises Treated With L-Glutamine,
Voxelotor, or Crizanlizumab in the United States.”
- Abstract P1506 is entitled “Mortality and Clinical
Complications Among Patients with Sickle Cell Disease With
Recurrent VOCs in Canada.”
- Abstract P1507 is entitled “Treatment Utilization and Clinical
Complications in Patients with Sickle Cell Disease Receiving
Frequent Red Blood Cell Transfusions in the United States.”
- Abstract P2191 is entitled “Clinical Complications and
Treatment Use Among Patients With Sickle Cell Disease With
Recurrent Vaso-Occlusive Crises in the Netherlands.”
- Abstract PB3248 is entitled “Clinical Complications Among
Patients With Transfusion-Dependent Beta-Thalassemia in the
Netherlands.”
About Sickle Cell Disease (SCD)
SCD is a debilitating, progressive, life shortening genetic
disease. SCD patients report health-related quality of life scores
well below the general population and significant health care
resource utilization. SCD 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 vaso-occlusive crises (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 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 Europe, the mean age of death for
patients living with SCD is around 40 years. Stem cell transplant
from a matched donor is a potentially curative option but is only
available to a small fraction of people 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 significant health care resource utilization. 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
Europe, the mean age of death for patients living with TDT is 50-55
years. Stem cell transplant from a matched donor is a potentially
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 certain indications in multiple
jurisdictions for eligible patients.
About the CLIMB Studies
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, characterized by recurrent VOCs, respectively. The trials
are now closed for enrollment. Patients will be followed for
approximately two years after CASGEVY infusion. Each patient will
be asked to participate in the ongoing long-term, open-label trial,
CLIMB-131. CLIMB-131 is designed to evaluate the safety and
efficacy of CASGEVY in patients who received CASGEVY in other CLIMB
studies. 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, 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, YouTube and Twitter/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 Haydar
Frangoul, M.D., M.S., and Franco Locatelli, M.D., Ph.D., in this
press release, and statements regarding our expectations for and
the anticipated benefits of CASGEVY, our plans to share longer-term
data for CASGEVY from the CLIMB clinical trials and to share health
economics abstracts at the EHA Congress, 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 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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