CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Beta Thalassemia
April 16 2019 - 7:00AM
CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals
Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and
Drug Administration (FDA) has granted Fast Track Designation for
CTX001 for the treatment of transfusion-dependent beta thalassemia
(TDT). CTX001 is an investigational, autologous, gene-edited
hematopoietic stem cell therapy for patients suffering from severe
hemoglobinopathies.
The FDA’s Fast Track program is designed to
facilitate the development and expedite the review of drugs that
treat serious conditions and fill unmet medical needs. A drug
granted Fast Track Designation may be eligible for several
benefits, including more frequent meetings and communications with
the FDA and, if relevant criteria are met, the potential for
Accelerated Approval, Priority Review or Rolling Review of a
Biologics License Application (BLA).
In February 2019, CRISPR Therapeutics and Vertex
announced that the first patient had been treated with CTX001 in a
Phase 1/2 clinical study of patients with TDT, marking the first
company-sponsored use of a CRISPR/Cas9 therapy in a clinical trial.
The Phase 1/2 open-label trial is designed to assess the safety and
efficacy of a single dose of CTX001 in patients ages 18 to 35 with
TDT, non-beta zero/beta zero subtypes. The companies are also
evaluating CTX001 for the treatment of sickle cell disease (SCD)
and received Fast Track Designation for CTX001 from the FDA in
January 2019 for SCD. The companies announced in February 2019 that
the first patient had been enrolled in a Phase 1/2 clinical study
of CTX001 in severe SCD in the U.S. and is expected to be infused
with CTX001 in mid-2019. Enrollment in both studies is ongoing.
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 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) 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; (ii) 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; (iii) the scope and timing of ongoing and potential future
clinical trials; (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 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 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 and
life-threatening diseases. In addition to clinical development
programs in cystic fibrosis, Vertex has more than a dozen ongoing
research programs focused on the underlying mechanisms of other
serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's
headquarters is now located in Boston's Innovation District. Today,
the company has research and development sites and commercial
offices in the United States, Europe, Canada, Australia and Latin
America. Vertex is consistently recognized as one of the industry's
top places to work, including being named to Science magazine's Top
Employers in the life sciences ranking for nine years in a row.
For additional information and the latest
updates from the Company, please visit www.vrtx.com.
(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,
statements regarding the potential benefits of Fast Track
Designation and the ongoing clinical trials for CTX001. While
Vertex believes the forward-looking statements contained in this
press release are accurate, 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, among other things, risks related to
developing drug candidates and the other risks listed under Risk
Factors in Vertex's annual report and quarterly reports filed with
the Securities and Exchange Commission. Vertex disclaims any
obligation to update the information contained in this press
release as new information becomes available.
CRISPR Investor Contact:Susan Kim+1
617-307-7503susan.kim@crisprtx.com
CRISPR Media Contact:Jennifer PaganelliWCG on
behalf of CRISPR+1 347-658-8290jpaganelli@wcgworld.com
Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
+1 617-341-6108orEric Rojas, +1 617-961-7205orZach Barber, +1
617-341-6470
Media:
mediainfo@vrtx.com orNorth
America:Heather Nichols, +1
617-341-6992Heather_Nichols@vrtx.com
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