- Beta thalassemia: All 15 patients were
transfusion independent after CTX001 infusion - - Sickle cell
disease: All seven patients were free of vaso-occlusive crises
after CTX001 infusion -
Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) and CRISPR
Therapeutics (Nasdaq:CRSP) today announced new data on 22 patients,
with follow-up of at least 3 months, and ranging from 4 months to
26 months, treated with the investigational CRISPR/Cas9-based
gene-editing therapy, CTX001, that show a consistent and sustained
response to treatment. CTX001 is being investigated in two ongoing
Phase 1/2 clinical trials as a potential one-time therapy for
patients suffering from transfusion-dependent beta thalassemia
(TDT) and severe sickle cell disease (SCD). In total, more than 40
patients have been dosed across both studies to date.
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All 15 patients with TDT,
including six who have the beta zero/beta zero or other severe genotypes, were
transfusion-free at last follow-up, and all seven patients with
severe SCD were free of vaso-occlusive crises (VOCs) from CTX001
infusion through last follow-up. Five patients with TDT and two
patients with SCD now have follow-up of greater than one year,
demonstrating a stable and durable response to treatment. These
data are available as e-posters beginning on June 11, 2021, at
09:00 CEST, and a partial presentation of these data were presented
during the Joint EHA-ASH Symposium on June 10, 2021 from
17:30-18:30 CEST. A summary of the results from the CLIMB-111 and
CLIMB-121 Phase 1/2 clinical trials is provided below.
“The data presented today in 22 patients are impressive in both
the consistency and durability of effect. These results add to the
growing body of evidence that CTX001 may hold the promise for a
one-time functional cure for sickle cell disease and beta
thalassemia. We are working with urgency to complete enrollment and
look forward to finalizing regulatory discussions and moving
towards filing,” said Reshma Kewalramani, M.D., Chief Executive
Officer and President at Vertex.
“The continued progress and momentum of CTX001 validate the role
that CRISPR gene-editing technology could have in the future of
therapeutics,” added Samarth Kulkarni, Ph.D., Chief Executive
Officer at CRISPR Therapeutics. “We are excited about these results
and look forward to additional longer-term data and to moving this
investigational medicine forward for a larger population of
patients with these two devastating diseases.”
“As a physician caring for patients suffering from beta
thalassemia, I have a high sense of urgency for novel and
efficacious treatments,” said Dr. Franco Locatelli, Professor of
Pediatrics at the Sapienza University of Rome, Director of the
Department of Pediatric Hematology and Oncology at Bambino Gesù
Children’s Hospital. “These results suggest the potential for a
durable benefit for patients with transfusion-dependent beta
thalassemia.”
“It is thrilling to work on a groundbreaking program like
CTX001,” said Dr. Stephan Grupp, Section Chief, Cellular Therapy
and Transplant, Division of Oncology, Children's Hospital of
Philadelphia. “This approach uses CRISPR/Cas9 gene editing to
enable the patient’s own cells to produce fetal hemoglobin, and to
see results that demonstrate the potential for a treatment that may
transform the lives of many patients is an exciting time for me and
the team.”
CLIMB-111 Trial in TDT: Updated Results The 15 patients
with TDT reported at EHA are patients who had reached at least
three months of follow-up after CTX001 dosing and therefore could
be assessed for initial safety and efficacy. All 15 patients showed
a similar pattern of response, with rapid and sustained increases
in total hemoglobin, fetal hemoglobin and transfusion
independence.
All 15 patients were transfusion independent with follow-up
ranging from 4 to 26 months after CTX001 infusion and had
clinically meaningful improvements in total hemoglobin from 8.9 to
16.9 g/dL and fetal hemoglobin from 67.3% to 99.6% at last
visit.
Bone marrow allelic editing data collected from 10 patients with
at least 6 months of follow-up, of which five patients had at least
12 months of follow-up and one patient had at least 24 months of
follow-up, demonstrated a durable effect.
The safety data from all 15 patients were generally consistent
with an autologous stem cell transplant and myeloablative
conditioning. As previously reported, there were four serious
adverse events (SAEs) considered related or possibly related to
CTX001 reported in one patient: headache, hemophagocytic
lymphohistiocytosis (HLH), acute respiratory distress syndrome and
idiopathic pneumonia syndrome. All four SAEs occurred in the
context of HLH and have resolved. The majority of non-serious
adverse events were considered mild to moderate.
The presentations at EHA and the data summarized in this press
release cover all TDT patients dosed with CTX001 with three or more
months of follow-up as of the data cut on March 30, 2021. In
addition to the data presented above, a TDT patient, with less than
three months of follow-up and therefore not included in the data
cut, experienced an SAE; this SAE of cerebellar hemorrhage, which
was considered related to busulfan conditioning, has resolved.
Enrollment and dosing are ongoing.
CLIMB-121 Trial in Severe SCD: Updated Results The seven
patients reported at EHA are patients who had reached at least
three months of follow-up after CTX001 dosing and therefore could
be assessed for initial safety and efficacy. All seven patients
showed a similar pattern of response, with rapid and sustained
increases in total hemoglobin and fetal hemoglobin, as well as
elimination of VOCs.
All seven patients remained VOC-free with follow-up ranging from
five to 22 months after CTX001 infusion and had clinically
meaningful improvements in total hemoglobin from 11 to 15.9 g/dL
and fetal hemoglobin levels from 39.6% to 49.6% at last visit.
Bone marrow allelic editing data collected from four patients
who have at least six months of follow-up, of which two had 12
months of follow-up, demonstrated a durable effect.
The safety data from all seven patients were generally
consistent with an autologous stem cell transplant and
myeloablative conditioning. There were no SAEs considered related
to CTX001, and the majority of non-serious adverse events were
considered mild to moderate.
Enrollment and dosing are ongoing.
About CTX001 CTX001 is an investigational, autologous, ex
vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for
patients suffering from TDT or severe SCD, in which a patient’s
hematopoietic stem cells are edited to produce high levels of fetal
hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of
the oxygen-carrying hemoglobin that is naturally present at birth,
which then switches to the adult form of hemoglobin. The elevation
of HbF by CTX001 has the potential to alleviate or eliminate
transfusion requirements for patients with TDT and reduce or
eliminate painful and debilitating sickle crises for patients with
SCD. Earlier results from these ongoing trials were published as a
Brief Report in The New England Journal of Medicine in January of
2021.
Based on progress in this program to date, CTX001 has been
granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track,
Orphan Drug, and Rare Pediatric Disease designations from the U.S.
Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has
also been granted Orphan Drug Designation from the European
Commission, as well as Priority Medicines (PRIME) designation from
the European Medicines Agency (EMA), for both TDT and SCD.
Among gene-editing approaches being investigated/evaluated for
TDT and SCD, CTX001 is the furthest advanced in clinical
development.
About CLIMB-111 The ongoing Phase 1/2 open-label trial,
CLIMB-Thal-111, is designed to assess the safety and efficacy of a
single dose of CTX001 in patients ages 12 to 35 with TDT. The trial
will enroll up to 45 patients and follow patients for approximately
two years after infusion. Each patient will be asked to participate
in a long-term follow-up trial.
About CLIMB-121 The ongoing Phase 1/2 open-label trial,
CLIMB-SCD-121, is designed to assess the safety and efficacy of a
single dose of CTX001 in patients ages 12 to 35 with severe SCD.
The trial will enroll up to 45 patients and follow patients for
approximately two years after infusion. Each patient will be asked
to participate in a long-term follow-up trial.
About CLIMB-131 This is a long-term, open-label trial to
evaluate the safety and efficacy of CTX001 in patients who received
CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow
participants for up to 15 years after CTX001 infusion.
About the Gene-Editing Process in These Trials Patients
who enroll in these trials will have their own hematopoietic stem
and progenitor cells collected from peripheral blood. The patient’s
cells will be edited using the CRISPR/Cas9 technology. The edited
cells, CTX001, will then be infused back into the patient as part
of a stem cell transplant, a process which involves, among other
things, a patient being treated with myeloablative busulfan
conditioning. Patients undergoing stem cell transplants may also
encounter side effects (ranging from mild to severe) that are
unrelated to the administration of CTX001. Patients will initially
be monitored to determine when the edited cells begin to produce
mature blood cells, a process known as engraftment. After
engraftment, patients will continue to be monitored to track the
impact of CTX001 on multiple measures of disease and for
safety.
About the Vertex-CRISPR Collaboration Vertex and CRISPR
Therapeutics entered into a strategic research collaboration in
2015 focused on the use of CRISPR/Cas9 to discover and develop
potential new treatments aimed at the underlying genetic causes of
human disease. CTX001 represents the first potential treatment to
emerge from the joint research program. Under a recently amended
collaboration agreement, Vertex will lead global development,
manufacturing and commercialization of CTX001 and split program
costs and profits worldwide 60/40 with CRISPR Therapeutics.
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.
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, including,
without limitation, statements made by Dr. Reshma Kewalramani, Dr.
Samarth Kulkarni, Dr. Franco Locatelli, and Dr. Stephan Grupp, and
statements regarding expectations that data will be presented and
available as e-posters beginning on June 11, 2021, the potential
benefits of CTX001, the potential benefits of our collaboration
with CRISPR and CRISPR gene-editing technology, our plans and
expectations for our clinical trials, including our expectations
for the gene-editing process in these clinical trials, the status
of our clinical trials of our product candidates under development
by us and our collaborators, including activities at the clinical
trial sites and patient enrollment, our expectations and plans
regarding our regulatory discussions and future regulatory filings,
and our expectations regarding the future activities of the parties
pursuant to the amended collaboration agreement with CRISPR. 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
development programs, including its programs with its
collaborators, may not support registration or further development
of its compounds due to safety and/or efficacy, or other reasons,
that the COVID-19 pandemic may impact the status or progress of our
clinical trials and clinical trial sites and the clinical trials
and clinical trial sites of our collaborators, including patient
enrollment, or 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.
(VRTX-GEN)
About CRISPR Therapeutics CRISPR Therapeutics is a
leading gene editing company focused on developing transformative
gene-based medicines for serious diseases using its proprietary
CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing
technology that allows for precise, directed changes to genomic
DNA. CRISPR Therapeutics has established a portfolio of therapeutic
programs across a broad range of disease areas including
hemoglobinopathies, oncology, regenerative medicine and rare
diseases. To accelerate and expand its efforts, CRISPR Therapeutics
has established strategic collaborations with leading companies
including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR
Therapeutics AG is headquartered in Zug, Switzerland, with its
wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and
R&D operations based in Cambridge, Massachusetts, and business
offices in San Francisco, California and London, United Kingdom.
For more information, please visit www.crisprtx.com.
CRISPR THERAPEUTICS® word mark and design logo and CTX001™ are
trademarks and registered trademarks of CRISPR Therapeutics AG. All
other trademarks and registered trademarks are the property of
their respective owners.
CRISPR Therapeutics Forward-Looking Statement This press
release may contain a number of “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, including statements made by Dr. Reshma
Kewalramani, Dr. Samarth Kulkarni, Dr. Franco Locatelli, and Dr.
Stephan Grupp in this press release, as well as statements
regarding CRISPR Therapeutics’ expectations about any or all of the
following: (i) the safety, efficacy and clinical progress of CRISPR
Therapeutics’ various clinical programs, including CTX001,
including expectations regarding the data presented and available
as e-posters beginning on June 11, 2021 and a partial presentation
of such data during the Joint EHA-ASH Symposium on June 10, 2021;
(ii) the potential and expected benefits of CRISPR Therapeutics’
collaboration with Vertex; and (iii) the therapeutic value,
development, and commercial potential of CRISPR/Cas9 gene editing
technologies and therapies. Without limiting the foregoing, the
words “believes,” “anticipates,” “plans,” “expects” and similar
expressions are intended to identify forward-looking statements.
You are cautioned that forward-looking statements are inherently
uncertain. Although CRISPR Therapeutics believes that such
statements are based on reasonable assumptions within the bounds of
its knowledge of its business and operations, existing and
prospective investors are cautioned that forward-looking statements
are inherently uncertain, are neither promises nor guarantees and
not to place undue reliance on such statements, which speak only as
of the date they are made. Actual performance and results may
differ materially from those projected or suggested in the
forward-looking statements due to various risks and uncertainties.
These risks and uncertainties include, among others: the potential
for initial and preliminary data from any clinical trial and
initial data from a limited number of patients (as is the case with
CTX001 at this time) not to be indicative of final or future trial
results; the potential that CTX001 clinical trial results may not
be favorable or may not support registration or further
development; the potential that future competitive or other market
factors may adversely affect the commercial potential for CTX001;
CRISPR Therapeutics may not realize the potential benefits of the
collaboration with Vertex; potential impacts due to the coronavirus
pandemic, such as to the timing and progress of clinical trials;
uncertainties regarding the intellectual property protection for
CRISPR Therapeutics’ technology and intellectual property belonging
to third parties; and those risks and uncertainties described under
the heading “Risk Factors” in CRISPR Therapeutics’ most recent
annual report on Form 10-K, quarterly report on Form 10-Q, and in
any other subsequent filings made by CRISPR Therapeutics with the
U.S. Securities and Exchange Commission, which are available on the
SEC's website at www.sec.gov. CRISPR Therapeutics disclaims any
obligation or undertaking to update or revise any forward-looking
statements contained in this press release, other than to the
extent required by law.
(CRSP-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
CRISPR Therapeutics Investors: Susan Kim, +1
617-307-7503 susan.kim@crisprtx.com
Media: Rachel Eides, +1-617-315-4493
Rachel.Eides@crisprtx.com
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