CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals
Incorporated (NASDAQ: VRTX) today announced positive, interim data
from the first two patients with severe hemoglobinopathies treated
with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in
ongoing Phase 1/2 clinical trials. One patient with
transfusion-dependent beta thalassemia (TDT) received CTX001 in the
first quarter of 2019 and data for this patient reflect nine months
of safety and efficacy follow-up. One patient with severe sickle
cell disease (SCD) received CTX001 in mid-2019 and data for this
patient reflect four months of safety and efficacy follow-up. These
studies are ongoing and patients will be followed for approximately
two years following infusion. Several additional patients have been
enrolled and have had drug product manufactured across the two
studies.
Transfusion-Dependent Beta
Thalassemia The patient with TDT has the β0/IVS-I-110
genotype and required 16.5 transfusions per year (annualized rate
during the two years prior to consenting for the study) before
enrolling in the clinical study. The patient achieved neutrophil
engraftment 33 days after CTX001 infusion and platelet engraftment
37 days after infusion. Two serious adverse events (SAEs) occurred,
neither of which the principal investigator (PI) considered related
to CTX001: pneumonia in the presence of neutropenia and
veno-occlusive liver disease attributed to busulfan conditioning;
both subsequently resolved. At nine months after CTX001 infusion,
the patient was transfusion independent and had total hemoglobin
levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells
(erythrocytes expressing fetal hemoglobin).
Sickle Cell Disease The patient
with SCD experienced seven vaso-occlusive crises (VOCs) per year
(annualized rate during the two years prior to consenting for the
study) before enrolling in the clinical study. The patient achieved
neutrophil and platelet engraftment 30 days after CTX001 infusion.
Three SAEs occurred, none of which the PI considered related to
CTX001: sepsis in the presence of neutropenia, cholelithiasis, and
abdominal pain, all of which resolved. At four months after CTX001
infusion, the patient was free of VOCs and had total hemoglobin
levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells
(erythrocytes expressing fetal hemoglobin).
“We are very encouraged by these preliminary
data, the first such data to be reported for patients with beta
thalassemia and sickle cell disease treated with our CRISPR/Cas9
edited autologous hematopoietic stem cell candidate, CTX001,” said
Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR
Therapeutics. “These data support our belief in the potential of
our therapies to have meaningful benefit for patients following a
one-time intervention. We continue to enroll these studies as we
drive forward to develop CRISPR/Cas9 therapies as a new class of
transformative medicines to treat serious diseases.”
“The data we announced today are remarkable and
demonstrate that CTX001 has the potential to be a curative
CRISPR/Cas9-based gene-editing therapy for people with sickle cell
disease and beta thalassemia,” said Jeffrey Leiden, M.D., Ph.D.,
Chairman, President and Chief Executive Officer of Vertex. “While
the data are exciting, we are still in the early phase of this
clinical program. We look forward to continuing to work with
physicians, patients, caregivers and families over the coming
months and years to bring forward the best possible therapy for
these two serious diseases and to continue to accelerate our
gene-editing programs for other serious diseases such as Duchenne
muscular dystrophy and myotonic dystrophy type 1.”
About the Phase 1/2 Study in
Transfusion-Dependent Beta
ThalassemiaThe 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 18 to 35 with TDT. The study
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 study. Enrollment is ongoing at six
clinical trial sites in the United States, Canada and Europe.
About the Phase 1/2 Study in Sickle Cell
DiseaseThe 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 18 to 35 with severe SCD.
The study 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 study. Enrollment is
ongoing at 12 clinical trial sites in the United States, Canada and
Europe.
About the
Gene-Editing Process in These
TrialsPatients who enroll in these studies will have
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.
CRISPR Therapeutics Conference Call and
WebcastCRISPR Therapeutics will host a conference call and
webcast today at 8:00 a.m. ET. The webcast and presentation
will be made available on the CRISPR Therapeutics website at
https://crisprtx.gcs-web.com/events in the Investors section under
Events and Presentations. Following the live audio webcast, a
replay will be available on the Company's website for approximately
30 days.
Dial-In InformationLive (U.S. /
Canada): (800) 895-3361Live (International): (785)
424-1062Conference ID: 87198237
About CTX001CTX001 is an
investigational ex vivo CRISPR gene-edited therapy that is being
evaluated for patients suffering from TDT or severe SCD in which a
patient’s hematopoietic stem cells are engineered 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 and is then replaced by the adult form of
hemoglobin. The elevation of HbF by CTX001 has the potential to
alleviate transfusion requirements for TDT patients and painful and
debilitating sickle crises for SCD patients.
CTX001 is being developed under a co-development
and co-commercialization agreement between CRISPR Therapeutics and
Vertex.
About the CRISPR-Vertex
Collaboration CRISPR Therapeutics and Vertex 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 treatment to emerge from the joint research
program. CRISPR Therapeutics and Vertex will jointly develop and
commercialize CTX001 and equally share all research and development
costs and profits worldwide.
About CRISPR TherapeuticsCRISPR
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 AG, 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 London, United
Kingdom. For more information, please visit www.crisprtx.com.
CRISPR Therapeutics Forward-Looking
StatementThis 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 regarding CRISPR Therapeutics’ expectations about any or
all of the following: (i) the safety, efficacy and clinical
progress of CRISPR Therapeutics’ CTX001 clinical program; (ii) the
status and scope of ongoing and potential future clinical trials
(including, without limitation, the timing of filing of clinical
trial applications and INDs, any approvals thereof and the timing
of commencement of clinical trials), development timelines and
discussions with regulatory authorities related to product
candidates under development by CRISPR Therapeutics and
its collaborators; (iii) the number of patients that will be
evaluated, the anticipated date by which enrollment will be
completed and the data that will be generated by ongoing and
planned clinical trials, and the ability to use that data for the
design and initiation of further clinical trials; v(iv) the
intellectual property coverage and positions of CRISPR
Therapeutics, its licensors and third parties; (v) the sufficiency
of CRISPR Therapeutics’ cash resources; and (vi) 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, forward-looking statements are neither
promises nor guarantees and they are necessarily subject to a high
degree of uncertainty and risk. 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
(including CTX001) not to be indicative of final trial results; the
risk that the initial data from a limited number of patients (as is
the case with CTX001 at this time) may not be indicative of results
from the full planned study population; the outcomes for each
CRISPR Therapeutics’ planned clinical trials and studies may not be
favorable; that one or more of CRISPR Therapeutics’ internal or
external product candidate programs will not proceed as planned for
technical, scientific or commercial reasons; that future
competitive or other market factors may adversely affect the
commercial potential for CRISPR Therapeutics’ product candidates;
uncertainties inherent in the initiation and completion of
preclinical studies for CRISPR Therapeutics’ product candidates;
availability and timing of results from preclinical studies;
whether results from a preclinical trial will be predictive of
future results of the future trials; uncertainties about regulatory
approvals to conduct trials or to market products; uncertainties
regarding the intellectual property protection for CRISPR
Therapeutics’ technology and intellectual property belonging to
third parties, and the outcome of proceedings (such as an
interference, an opposition or a similar proceeding) involving all
or any portion of such intellectual property; and those risks and
uncertainties described under the heading "Risk Factors" in CRISPR
Therapeutics’ most recent annual report on Form 10-K, 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.
Existing and prospective investors are cautioned not to place undue
reliance on these forward-looking statements, which speak only as
of the date they are made. 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.
About VertexVertex is a global
biotechnology company that invests in scientific innovation to
create transformative medicines for people with serious diseases.
The company has four 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 disease. In
addition, Vertex has a rapidly expanding pipeline of genetic and
cell 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, UK. 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 10 consecutive years on Science
magazine's Top Employers list and top five on the 2019 Best
Employers for Diversity list by Forbes. 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.
(VRTX-GEN)
Vertex Special Note Regarding
Forward-Looking StatementsThis press release contains
forward-looking statements as defined in the Private Securities
Litigation Reform Act of 1995, including, without limitation, the
information provided regarding the status of, and expectations with
respect to, the CTX001 clinical development 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 factors that could cause actual events or
results to differ materially from those indicated by such
forward-looking statements. Those risks and uncertainties include
that the development of CTX001 may not proceed due to safety,
efficacy or other reasons, and other risks listed under Risk
Factors in Vertex's annual report and quarterly reports filed with
the Securities and Exchange Commission and available
through the company's website at www.vrtx.com. Vertex
disclaims any obligation to update the information contained in
this press release as new information becomes available.
CRISPR Therapeutics
Investor Contact:Susan Kim, +1
617-307-7503susan.kim@crisprtx.com
CRISPR Therapeutics Media Contact:Jennifer
PaganelliWCG on behalf of CRISPR+1
347-658-8290jpaganelli@wcgworld.com
Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
+1 617-341-6108orZach Barber, +1 617-341-6470orLeah Gibson, +1
617-961-1507
Media: mediainfo@vrtx.com orNorth
America:Heather Nichols, +1 617-341-6992
Heather_Nichols@vrtx.com
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