Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR
Therapeutics (Nasdaq: CRSP) today announced two abstracts detailing
updated data from the ongoing CTX001 clinical trials have been
accepted for presentation during the European Hematology
Association (EHA) 2021 Virtual Congress.
Abstract #EP736 entitled “CTX001 for Sickle Cell
Disease: Safety and Efficacy Results from the Ongoing CLIMB SCD-121
Study of Autologous Crispr-Cas9-Modified CD34+ Hematopoietic Stem
and Progenitor Cells,” will be made available on the virtual
platform as an e-poster Friday, June 11 at 9:00 CEST. The abstract
posted online today includes data on patients with severe sickle
cell disease with more than 3 months of follow-up, as of the
interim data cut on January 28, 2021. Data will be updated and
information on additional patients will be included for the
congress.
Abstract #EP733 entitled “CTX001 for
Transfusion-Dependent Β-Thalassemia: Safety and Efficacy Results
from the Ongoing CLIMB Thal-111 Study of Autologous
Crispr-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor
Cells,” will be made available on the virtual platform as an
e-poster Friday, June 11 at 9:00 CEST. The abstract posted online
today includes data on patients with transfusion-dependent beta
thalassemia (TDT) with more than 3 months of follow-up, including
patients with the most severe genotypes, as of the interim data cut
on January 21, 2021. Data will be updated and information on
additional patients will be included for the congress.
The accepted abstracts are now available online
on the EHA website
https://library.ehaweb.org/eha/#!*menu=6*browseby=8*sortby=2*media=3*ce_id=2035*label=21989*ot_id=25562*marker=1286.
CTX001 is being investigated in two ongoing
clinical trials as a potential one-time curative therapy for
patients suffering from TDT and severe SCD.
About CTX001CTX001 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 transfusion requirements for patients
with TDT and reduce 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-111The 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-121The 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-131This 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
TrialsPatients 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
CollaborationVertex 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. This amendment is subject
to customary closing conditions and clearances, including clearance
under the Hart-Scott Rodino Antitrust Improvements Act.
About VertexVertex 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 StatementsThis press release contains
forward-looking statements as defined in the Private Securities
Litigation Reform Act of 1995, including, without limitation, our
plans and expectations to present clinical data from the ongoing
CTX001 clinical trials during the EHA Virtual Congress,
expectations regarding the abstracts that will be made available on
the virtual platform, the expectation that data will be updated for
the conference, the potential benefits of CTX001, our plans and
expectations for our clinical trials and pipeline products, 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, and our
expectations regarding the transaction contemplated by the amended
collaboration agreement with CRISPR, including satisfaction of
closing conditions and antitrust clearances, and the future
activities of the parties pursuant to the amended collaboration
agreement. 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 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 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, 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 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, 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 abstracts
that will be made available on the virtual platform and the
clinical data that are being presented from the ongoing CTX001
clinical trials during the EHA Virtual Congress; (ii) the timing of
the potential closing of the transaction contemplated by the
amended collaboration agreement, future activities of the parties
pursuant to the collaboration and the potential 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; that future competitive or other market
factors may adversely affect the commercial potential for CTX001;
the transaction contemplated by the amended collaboration agreement
is subject to certain closing conditions, including the expiration
of the waiting period under the Hart-Scott-Rodino Antitrust
Improvements Act; 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; the potential that future competitive or other
market factors may adversely affect the commercial potential for
CTX001; 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.
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.
Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
+1 617-341-6108orBrenda Eustace, +1 617-341-6187OrManisha Pai, +1
617-429-6891
Media:mediainfo@vrtx.com
orU.S.: +1 617-341-6992orHeather Nichols: +1
617-839-3607orInternational: +44 20 3204 5275
CRISPR Therapeutics
Investors:Susan Kim, +1
617-307-7503susan.kim@crisprtx.com
Media:Rachel Eides,
+1-617-315-4493Rachel.Eides@crisprtx.com
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