- Vertex announces three additional
abstracts on the burden of beta thalassemia and sickle cell disease
accepted for poster presentation –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR
Therapeutics (NASDAQ: CRSP) today announced new late-breaking
clinical data accepted for oral presentation at the 2022 European
Hematology Association (EHA) Congress. Vertex also announced three
abstracts accepted for poster presentation at EHA.
Late-breaking abstract #LB2367 entitled “Efficacy and Safety of
A Single Dose of CTX001 For Transfusion-Dependent Βeta-Thalassemia
and Severe Sickle Cell Disease,” will be an oral presentation on
Sunday, June 12 at 09:45‑11:15 CEST. The abstract from Vertex and
CRISPR Therapeutics includes data on patients treated in CLIMB‑111
and CLIMB‑121 and followed in CLIMB‑131 with CTX001, now known as
exagamglogene autotemcel (exa-cel). This abstract has been selected
for the media briefing program and is therefore embargoed until
Saturday, June 11 at 09:00 am CEST.
In addition, three real-world evidence and health economics
abstracts from Vertex have been accepted for poster
presentation.
- Abstract #P1704 entitled “Projected Lifetime Economic Burden of
Severe Sickle Cell Disease in the United States,” will be a poster
presentation on Friday, June 10 at 16:30‑17:45 CEST. The abstract
posted online projects the per-patient lifetime direct health care
cost of severe sickle cell disease (SCD) from a U.S. health care
payer perspective using an economic model developed based on
published model frameworks.
- Abstract #P1703 entitled “Economic Burden of
Transfusion‑Dependent Beta‑Thalassemia in the United States,” will
be a poster presentation on Friday, June 10 at 16:30‑17:45 CEST.
The abstract posted online estimates the economic burden of
transfusion-dependent beta thalassemia (TDT) using administrative
claims data to estimate the costs and health care utilization
associated with disease management in the U.S.
- Abstract #P1482 entitled “Patients With Severe Sickle Cell
Disease on Standard-of-Care Treatment Are Very Unlikely to Become
VOC‑Free for One Year: A Cohort Study of Medicaid Enrollees,” will
be a poster presentation on Friday, June 10 at 16:30‑17:45 CEST.
The abstract posted online contextualizes the efficacy of exa‑cel
in eliminating vaso‑occlusive crises (VOCs) in patients with SCD
using nationwide U.S. Medicaid claims data from 2000 to 2014 to
assess the proportion of patients with recurrent VOCs who became
VOC‑free during a 1‑year follow up on standard of care.
The accepted abstracts are now available online on the EHA
website.
Exa‑cel is being investigated in multiple ongoing clinical
trials as a potential one-time therapy for patients with either TDT
or SCD.
About exagamglogene autotemcel (exa-cel)
Exa‑cel, formerly known as CTX001, is an investigational,
autologous, ex vivo CRISPR/Cas9 gene‑edited therapy that is being
evaluated for patients with TDT or SCD characterized by recurrent
VOCs, in which a patient’s own hematopoietic stem cells are edited
to produce 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. The elevation
of HbF by exa‑cel 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 in The New England Journal
of Medicine in January of 2021.
Based on progress in this program to date, exa‑cel 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. Exa-cel
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 evaluated for TDT and SCD,
exa‑cel is the furthest advanced in clinical development.
About CLIMB‑111 and CLIMB‑121
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 exa‑cel 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 exa‑cel infusion. Each patient will
be asked to participate in CLIMB‑131, a long‑term follow‑up
trial.
About CLIMB-131
This is a long‑term, open‑label trial to evaluate the safety and
efficacy of exa‑cel in patients who received exa‑cel in CLIMB‑111,
CLIMB‑121, CLIMB‑141 or CLIMB‑151. The trial is designed to follow
participants for up to 15 years after exa‑cel infusion.
About CLIMB‑141 and CLIMB‑151
The ongoing Phase 3 open-label trials, CLIMB‑141 and CLIMB‑151,
are designed to assess the safety and efficacy of a single dose of
exa‑cel in patients ages 2 to 11 years with TDT or with SCD,
characterized by recurrent VOCs, respectively. The trials are now
open for enrollment and currently enrolling patients ages 5 to 11
years of age and will plan to extend to ages 2 to less than 5 years
of age at a later date. Each trial will enroll up to 12 patients.
Patients will be followed for approximately two years after
infusion. Each patient will be asked to participate in CLIMB-131, a
long‑term follow‑up trial.
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, exa‑cel, will then be infused back
into the patient as part of an autologous hematopoietic stem cell
transplant (HSCT), a process which involves a patient being treated
with myeloablative busulfan conditioning. Patients undergoing HSCT
may also encounter side effects (ranging from mild to severe) that
are unrelated to the administration of exa‑cel. 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 exa‑cel 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. Exa‑cel represents the first
potential treatment to emerge from the joint research program.
Under an amended collaboration agreement, Vertex now leads global
development, manufacturing and commercialization of exa‑cel and
splits 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, cell and genetic
therapies in other serious diseases where it has deep insight into
causal human biology, including sickle cell disease, beta
thalassemia, APOL1‑mediated kidney disease, pain, type 1 diabetes,
alpha‑1 antitrypsin deficiency and Duchenne muscular dystrophy.
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 12 consecutive years on Science magazine's Top
Employers list and one of the 2021 Seramount (formerly Working
Mother Media) 100 Best Companies. 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
Statements
This press release contains forward-looking statements as
defined in the Private Securities Litigation Reform Act of 1995, as
amended, including, without limitation, our plans and expectations
to present clinical data from the ongoing exa-cel clinical trials
during the EHA Congress, expectations regarding the abstracts that
will be made available on the virtual platform and the clinical
data that will be presented during the EHA Congress, including
anticipated projections and estimates related to the various
economic impacts of SCD and TDT, the potential benefits, efficacy,
and safety of exa-cel, including the potentially transformative
nature of the therapy and the potential of the treatment for
patients, 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, patient enrollment and
expectations regarding clinical trial follow-up. 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 internal or external
factors that could delay, divert, or change our plans and
objectives with respect to our research and development programs,
that future competitive or other market factors may adversely
affect the commercial potential for exa-cel, 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 (SEC) and available through the company's
website at www.vrtx.com and on the SEC’s website at www.sec.gov.
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.
(CRSP-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 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, as well as statements regarding
CRISPR Therapeutics’ expectations about any or all of the
following: i) the safety, efficacy and clinical progress of the
ongoing exa-cel clinical trials, including expectations regarding
the abstract that will be made available on the virtual platform
and our plans to present and the clinical data that are being
presented during the EHA Congress; and (ii) 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
exa-cel at this time) not to be indicative of final or future trial
results; the potential that the exa-cel 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 exa-cel; CRISPR
Therapeutics may not realize the potential benefits of its
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.
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version on businesswire.com: https://www.businesswire.com/news/home/20220601006221/en/
Vertex Pharmaceuticals Incorporated Investors:
Michael Partridge, +1 617-341-6108 Or Manisha Pai, +1 617-961-1899
Or Miroslava Minkova, +1 617-341-6135
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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