CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical
company focused on creating transformative gene-based medicines for
serious diseases, today provided an update on its immuno-oncology
pipeline of CRISPR/Cas9 gene-edited allogeneic chimeric antigen
receptor (CAR) T cell product candidates.
The Company’s first-generation allogeneic CAR T
candidates, CTX110 and CTX130, provided important proof of concept
that allogeneic CAR T cells can produce durable remissions
following a standard lymphodepletion regimen. Preliminary data from
ongoing clinical trials of its next-generation candidates, CTX112
targeting CD19 and CTX131 targeting CD70, suggest that these
candidates may improve upon that clinical profile. Emerging
pharmacology data, including pharmacokinetics, indicate that the
novel potency gene edits in CTX112 and CTX131 lead to significantly
higher CAR T cell expansion and functional persistence in patients
compared to the first-generation candidates. In addition, the
next-generation candidates exhibit increased manufacturing
robustness, with a higher and more consistent number of CAR T cells
produced per batch. Based on these considerations, the Company is
focusing on the development of CTX112 and CTX131 and will be
transitioning patients treated with CTX110 and CTX130 to long-term
follow-up where applicable.
“Our next-generation allogeneic CAR T candidates
reflect our mission of innovating continuously to bring potentially
transformative medicines to patients as quickly as possible,” said
Samarth Kulkarni, Ph.D., Chief Executive Officer and Chairman of
the Board of CRISPR Therapeutics. “We are excited about our
next-generation CAR T platform, and focusing our efforts on these
candidates will allow us to advance these potentially best-in-class
CAR T therapies more efficiently and rapidly.”
“We are very encouraged by the progress and early clinical data
from our next-generation candidates. While we saw benefits from
consolidation dosing with CTX110, we believe CTX112 could result in
even better outcomes for patients,” said PK Morrow, M.D., Chief
Medical Officer of CRISPR Therapeutics. “We thank the patients,
families, and investigators who have participated in our clinical
trials of CTX110 and CTX130 and look forward to applying learnings
from these programs to expedite the development of CTX112 and
CTX131.”
In December 2022, the Company presented data
from Part A of the Phase 1/2 clinical trial of CTX110 that showed
the potential for CTX110 to produce durable complete remissions in
heavily pre-treated patients following a standard lymphodepletion
regimen. In new data updated today, Part B of the trial
demonstrated an increased 6-month complete response (CR) rate
following the inclusion of consolidation dosing, as shown in the
table below. The safety profile of CTX110 in Part B remained
consistent with the positively differentiated safety profile
observed in Part A.
|
Part ASingle dose with optionalre-dosing at ≥DL3
(N=27) |
Part BConsolidation dosing atDL4 (N=31) |
ORR |
67% |
65% |
CR rate |
41% |
39% |
6-month CR rate |
19% |
23% |
|
|
|
CTX112 and CTX131 have the potential to improve
upon the efficacy observed with CTX110 and CTX130. These
next-generation candidates each incorporate two novel gene
edits—knock-out of Regnase-1 and transforming growth factor-beta
receptor type 2 (TGFBR2)—that have the potential to enhance CAR T
potency and reduce CAR T exhaustion. Editing Regnase-1 removes an
intrinsic “brake” on T cell function while editing TGFBR2 removes a
key extrinsic “brake” on T cell anti-tumor activity. CRISPR
Therapeutics identified this combination of edits through
systematic screening of dozens of new and previously described
genes. In preclinical studies, these edits synergistically improved
potency approximately 10-fold compared to the first-generation
candidates. Clinical trials are ongoing for CTX112 in B-cell
malignancies and for CTX131 in solid tumors. The Company is
producing CTX112 and CTX131 for clinical trials at its internal GMP
manufacturing facility.
Furthermore, CRISPR Therapeutics announced plans
to initiate new clinical trials of CTX112 and CTX131 in additional
indications. The Company plans to expand the evaluation of CTX112
beyond oncology into autoimmune diseases. Early clinical studies
have shown that CD19-directed autologous CAR T therapy can produce
long-lasting remissions in multiple autoimmune indications. CTX112
has the potential to provide similar results with several
advantages, including greater scalability, lower cost of goods, and
no patient apheresis. The Company plans to initiate a clinical
trial in systemic lupus erythematosus (SLE) in the first half of
2024, with the potential to expand into additional autoimmune
indications in the future.
About CD19
Candidates CTX110 is a wholly-owned, healthy
donor-derived gene-edited allogeneic CAR T investigational therapy
targeting cluster of differentiation 19, or CD19, and CTX112 is a
next-generation, wholly-owned, allogeneic CAR T product candidate
targeting CD19, which incorporates additional edits designed to
enhance CAR T potency and reduce CAR T exhaustion. CTX112 is being
investigated in an ongoing clinical trial designed to assess safety
and efficacy of the product candidate in adult patients with
relapsed or refractory CD19-positive B-cell malignancies who have
received at least two prior lines of therapy.
About CD70
Candidates CTX130 is a wholly-owned, healthy
donor-derived gene-edited allogeneic CAR T investigational therapy
targeting cluster of differentiation 70, or CD70, an antigen
expressed on various solid tumors and hematologic malignancies, and
CTX131 is a next-generation, wholly-owned, allogeneic CAR T product
candidate targeting CD70, which incorporates additional edits
designed to enhance CAR T potency and reduce CAR T exhaustion.
CTX131 is being investigated in a clinical trial designed to assess
the safety and efficacy of the product candidate in adult patients
with relapsed or refractory solid tumors.
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
cardiometabolic diseases. To accelerate and expand its
efforts, CRISPR Therapeutics has established strategic
partnerships 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 Boston,
Massachusetts and San Francisco, California, and business
offices in London, United Kingdom. For more information,
please visit www.crisprtx.com.
CRISPR THERAPEUTICS® standard character mark and
design logo, CTX110®, CTX112™, CTX130™, and CTX131™ 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 Drs. Kulkarni and Morrow in this press
release, as well as statements regarding CRISPR Therapeutics’
expectations about any or all of the following: (i) its plans for
and its preclinical studies, clinical trials and pipeline products
and programs, including, without limitation, manufacturing, status
of such studies and trials, potential expansion into new
indications and expectations regarding data generally, as well as
the data presented in this press release; (ii) the safety, efficacy
and clinical progress of our various clinical and preclinical
programs, including our immuno-oncology programs; (iii) 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; 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,
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 efficacy and safety results from ongoing
clinical trials will not continue or be repeated in ongoing or
planned clinical trials or may not support regulatory submissions;
clinical trial results may not be favorable or support further
development; one or more of its product candidate programs will not
proceed as planned for technical, scientific or commercial reasons;
future competitive or other market factors may adversely affect the
commercial potential for its product candidates; uncertainties
inherent in the initiation and completion of preclinical studies
for its product candidates and whether results from such studies
will be predictive of future results of future studies or clinical
trials; uncertainties about regulatory approvals to conduct trials
or to market products; uncertainties inherent in the operation of a
manufacturing facility; uncertainties regarding the intellectual
property protection for its 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.
Investor Contact:Susan
Kim+1-617-307-7503susan.kim@crisprtx.com
Media Contact:Rachel
Eides+1-617-315-4493rachel.eides@crisprtx.com
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