CRISPR Therapeutics (Nasdaq: CRSP) and
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)
today announced data in seven patients from two ongoing Phase 1/2
clinical trials of the investigational CRISPR/Cas9 gene-editing
therapy CTX001 in severe hemoglobinopathies has been accepted for
an oral presentation during the Plenary Scientific Session at the
annual ASH Meeting and Exposition, which will take place virtually
from December 5-8, 2020. Haydar Frangoul, M.D., Medical Director of
Pediatric Hematology and Oncology at Sarah Cannon Research
Institute, HCA Healthcare’s TriStar Centennial Medical Center, will
deliver the presentation on behalf of all the authors on December
6, 2020.
An abstract posted online today includes data
from five patients with three months to 15 months of follow-up
after CTX001 infusion in the ongoing Phase 1/2 CLIMB-111 trial in
transfusion-dependent beta thalassemia (TDT) and data from two
patients with three months and 12 months of follow-up in the
ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease
(SCD). Additional data will be presented at ASH, including
longer-duration follow-up data for the patients included in the
abstract and data for additional patients with greater than three
months of follow-up.
CTX001 is being investigated in these two
ongoing clinical trials as a potential one-time curative therapy
for patients suffering from TDT and severe SCD.
The accepted abstract is now available on the
ASH conference website.
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
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,
which then switches to the adult form of hemoglobin. The elevation
of HbF by CTX001 has the potential to alleviate transfusion
requirements for TDT patients and reduce painful and debilitating
sickle crises for SCD patients.
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).
CTX001 has also been granted Orphan Drug Designation from the
European Commission for both TDT and SCD, as well as Priority
Medicines (PRIME) designation from the European Medicines Agency
(EMA) for SCD.
CTX001 is being developed under a co-development
and co-commercialization agreement between CRISPR Therapeutics and
Vertex. 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 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 CRISPR-Vertex
CollaborationCRISPR 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 potential 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, 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 status of clinical trials (including,
without limitation, the expected timing of data releases) related
to product candidates under development by CRISPR Therapeutics and
its collaborators, including expectations regarding the data and
plans to present data at the annual ASH meeting and exposition;
(ii) the expected benefits of CRISPR Therapeutics’ collaborations;
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, 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 and
initial data from a limited number of patients (as is the case with
CTX001 at this time) not to be indicative of final trial results;
the potential that CTX001 clinical trial results may not be
favorable; the potential impacts due to the coronavirus pandemic,
such as the timing and progress of clinical trials; 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 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, 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. 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.
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.
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
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. 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 regarding the expectations and plans to present data at
the annual ASH meeting and exposition, the development, including
expected timeline for development, and potential benefits of
CTX001, our plans and expectations for our clinical trials and
clinical trial sites, and 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 potential
outcomes. 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 the company's development programs,
including its programs with its collaborators, may not support
registration or further development of its compounds due to safety,
efficacy or other reasons, and other risks listed under Risk
Factors in Vertex's most recent annual report and subsequent
quarterly reports filed with the Securities and Exchange Commission
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)
CRISPR Therapeutics Investor
Contact:Susan Kim, +1
617-307-7503susan.kim@crisprtx.com
CRISPR Therapeutics Media
Contact:Rachel EidesWCG on behalf of CRISPR+1
617-337-4167reides@wcgworld.com
Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
+1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1
617-341-6187
Media:mediainfo@vrtx.com
orU.S.: +1 617-341-6992orHeather Nichols: +1
617-839-3607orInternational: +44 20 3204 5275
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