- First CRISPR gene-editing filings to be
accepted for review by FDA -
- FDA grants Priority Review for severe sickle
cell disease (SCD) and Standard Review for transfusion-dependent
beta thalassemia (TDT) –
- PDUFA target action date of December 8, 2023,
for SCD and March 30, 2024, for TDT –
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR
Therapeutics (Nasdaq: CRSP) today announced that the U.S. Food and
Drug Administration (FDA) has accepted the Biologics License
Applications (BLAs) for the investigational treatment exagamglogene
autotemcel (exa-cel) for severe sickle cell disease (SCD) and
transfusion-dependent beta thalassemia (TDT). The FDA has granted
Priority Review for SCD and Standard Review for TDT and assigned
Prescription Drug User Fee Act (PDUFA) target action dates of
December 8, 2023, and March 30, 2024, respectively. Updated data
from the pivotal trials supporting the regulatory submissions will
be presented at the Annual European Hematology Association Congress
on June 11, 2023.
“We are very pleased with the acceptance of the submissions and
the Priority Review designation for SCD by the FDA, as well as the
progress of the exa-cel filings in the EU and U.K.,” said Reshma
Kewalramani, M.D., Chief Executive Officer and President of Vertex.
“Exa-cel holds the promise to be the first CRISPR gene-editing
therapy to be approved, and we continue to work with urgency to
bring this treatment with transformative potential to patients who
are waiting.”
“We are glad to see that the unmet need and urgency for
innovative therapies in SCD was recognized by the FDA with Priority
Review,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of
CRISPR Therapeutics. “This is an exciting milestone for the CRISPR
platform, and we look forward to continuing the close collaboration
with our partners at Vertex to bring this medicine to patients in
need.”
In the U.S., exa-cel has been granted Regenerative Medicine
Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare
Pediatric Disease designations from the FDA for both TDT and
SCD.
In Europe, the Marketing Authorization Applications (MAAs) for
exa-cel were submitted in December 2022 and validated by the
European Medicines Agency (EMA) and the Medicines and Healthcare
products Regulatory Agency (MHRA) in January 2023. In the EU,
exa-cel has been granted Orphan Drug Designation from the European
Commission, as well as Priority Medicines (PRIME) designation from
the EMA, for both SCD and TDT. In the U.K., exa-cel has also been
granted an Innovation Passport under the Innovative Licensing and
Access Pathway (ILAP) from the MHRA.
About exagamglogene autotemcel (exa-cel)
Exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9
gene-edited therapy that is being evaluated for patients with SCD
or TDT, 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 reduce or
eliminate painful and debilitating VOCs for patients with SCD and
alleviate transfusion requirements for patients with TDT. Earlier
results from these ongoing trials were published in The New England
Journal of Medicine in January of 2021 and presented at the
American Society of Hematology Annual Congress in 2022.
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
The ongoing long-term, open-label trial, CLIMB-131, is designed
to evaluate the safety and efficacy of exa-cel in patients who
received exa-cel in CLIMB-111, CLIMB-121, CLIMB-141, CLIMB-151 or
CLIMB-161. 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 with the plan to extend to ages 2 to less than 5 years at a
later date. Each trial will enroll approximately 15 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 CLIMB-161
The ongoing Phase 3b trial, CLIMB-161, is to support expansion
of our manufacturing footprint after initial potential approval and
launch. This trial will enroll approximately 12 patients with
either TDT or with SCD, characterized by recurrent VOCs, ages 12 to
35 years. Patients will be followed for approximately one year
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 and ‑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
clinical pipeline of investigational small molecule, mRNA, cell and
genetic therapies (including gene editing) in other serious
diseases where it has deep insight into causal human biology,
including sickle cell disease, beta thalassemia, APOL1-mediated
kidney disease, acute and neuropathic pain, type 1 diabetes and
alpha-1 antitrypsin deficiency.
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 13 consecutive years on Science magazine's Top
Employers list and one of Fortune’s 100 Best Companies to Work For.
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, statements made by Reshma
Kewalramani, M.D., and Samarth Kulkarni, Ph.D., in this press
release, our plans and expectations to present updated clinical
data for exa-cel at the Annual European Hematology Association
Congress, the status of our clinical trials of our product
candidates under development by us and our collaborators, including
activities at the clinical trial sites, the gene-editing process,
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 regulatory
authorities may not approve, or approve on a timely basis, the
exa-cel BLAs, 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 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 in
Boston, Massachusetts and San Francisco, California, and business
offices in London, United Kingdom. For more information, please
visit www.crisprtx.com.
CRISPR THERAPEUTICS® word mark and design logo 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
statements made by Reshma Kewalramani, M.D., and Samarth Kulkarni,
Ph.D., 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 the
ongoing exa-cel clinical trials, including plans to present updated
clinical data at the European Hematology Association Congress; (ii)
timelines for and expectations regarding a regulatory agency
decision (iii) the benefits of its collaboration with Vertex; and
(iv) 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: data from a limited number of patients may not to be
indicative of final or future clinical 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;
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/20230608005819/en/
Vertex Pharmaceuticals Incorporated Investors:
Susie Lisa, +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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