CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company
focused on creating transformative gene-based medicines for serious
diseases, today reported financial results for the second quarter
ended June 30, 2024.
“In addition to the continued momentum of
CASGEVY’s launch, we are making significant progress across the
rest of our pipeline,” said Samarth Kulkarni, Ph.D., Chief
Executive Officer and Chairman of CRISPR Therapeutics. “We continue
to advance our next generation CD19-directed CAR T cell program,
CTX112, which has the potential to be best-in-class in both
oncology and autoimmune indications. We have opened the clinical
trial for CTX131 in hematologic malignancies, and continue to
dose-escalate with our in vivo directed programs, CTX310 and
CTX320. We are making outstanding progress across our early stage
discovery efforts and are well-positioned to realize our mission of
bringing multiple transformative medicines to patients in
need.”
Recent Highlights and
Outlook
- Hemoglobinopathies and CASGEVY™ (exagamglogene
autotemcel [exa-cel])
- CRISPR Therapeutics has two next
generation approaches with the potential to significantly expand
the addressable population with SCD and TDT. The Company continues
to advance its internally developed targeted conditioning program,
an anti-CD117 (c-Kit) antibody-drug conjugate (ADC), through
preclinical studies. Additionally, the Company has ongoing research
efforts to enable in vivo editing of hematopoietic stem cells. This
work could obviate the need for conditioning altogether, expand
geographic reach, and enable the treatment of multiple additional
other diseases beyond SCD and TDT.
- Enrollment has been completed in
two global Phase 3 studies of CASGEVY in people 5 to 11 years of
age with SCD or TDT and the trials are ongoing.
- In June, positive long-term data
from CLIMB-111, CLIMB-121 and the long-term follow-up study of
CASGEVY were presented at the 2024 Annual European Hematology
Association Congress. These long-term data from more than 100
patients dosed with CASGEVY, with the longest follow-up of more
than five years, confirm the transformative, consistent, and
durable clinical benefits of CASGEVY over time.
- The French National Authority for
Health (HAS) approved Vertex’s request for the implementation of an
early access program (EAP) for the use of CASGEVY in indicated
patients with SCD. HAS previously approved the implementation of an
EAP for CASGEVY in indicated patients with TDT in the first quarter
of 2024.
- As of mid-July, more than 35
authorized treatment centers (ATCs) have been activated globally,
including centers in all regions where CASGEVY is approved, and
approximately 20 patients have had cells collected across all
regions.
- CASGEVY is approved in the U.S.,
Great Britain, the European Union (EU), the Kingdom of Saudi Arabia
(KSA), and the Kingdom of Bahrain (Bahrain) for the treatment of
both sickle cell disease (SCD) and transfusion-dependent beta
thalassemia (TDT), and launches are ongoing. Regulatory submissions
for CASGEVY have been completed in both SCD and TDT in Switzerland
and Canada where it received Priority Review. CASGEVY is the
first therapy to emerge from a strategic partnership between CRISPR
Therapeutics and Vertex Pharmaceuticals established in 2015. As
part of an amendment to the collaboration agreement in 2021, Vertex
now leads global development, manufacturing, regulatory and
commercialization of CASGEVY with support from CRISPR
Therapeutics.
- Immuno-Oncology and Autoimmune Diseases
- CTX131 is currently in an ongoing
clinical trial in solid tumors. In addition, the Company has opened
a clinical trial for CTX131 in hematologic malignancies including T
cell lymphomas (TCL). Allogeneic CAR T approaches for TCL may have
greater potential to meet the unmet need in this patient population
given the patients’ own T cells are not suitable for autologous
manufacturing.
- CRISPR Therapeutics opened a
clinical trial for CTX112 in systemic lupus erythematosus (SLE),
with the potential to expand into additional autoimmune indications
in the future. Early clinical studies conducted by third parties
have shown that CD19-directed autologous CAR T therapy can produce
long-lasting remissions in multiple autoimmune indications by
deeply depleting B cells. The Company’s first generation allogeneic
CD19-directed CAR T program has demonstrated effective depletion of
B cells in oncology settings, which supports the potential for
CTX112 in autoimmune diseases.
- CTX112 is being developed for both
oncology and autoimmune indications. In oncology settings, CTX112
is in a Phase 1/2 trial for CD19 positive relapsed or refractory
B-cell malignancies, and the Company expects to report preliminary
clinical data this year.
- CRISPR Therapeutics’ next
generation allogeneic CAR T candidates reflect the Company’s
mission of innovating continuously to bring potentially
transformative medicines to patients as quickly as possible.
Clinical trials are ongoing for the Company’s next generation CAR T
product candidates, CTX112™ and CTX131™, targeting CD19 and CD70,
respectively, across multiple indications. CTX112 and CTX131 both
contain novel potency edits which can lead to significantly higher
CAR T cell expansion and cytotoxicity, potentially representing
best-in-class allogeneic CAR T products for these targets.
- In Vivo
- CRISPR Therapeutics has established
a proprietary lipid nanoparticle (LNP) platform for the delivery of
CRISPR/Cas9 to the liver. The first two in vivo programs utilizing
this proprietary platform, CTX310™ and CTX320™, are directed
towards validated therapeutic targets associated with
cardiovascular disease.
- CTX310 is currently in an ongoing
Phase 1 trial targeting ANGPTL3 in patients with homozygous
familial hypercholesterolemia (HoFH), severe hypertriglyceridemia
(SHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed
dyslipidemias. Natural loss-of-function mutations in ANGPTL3 are
associated with reduced low-density lipoprotein (LDL-C),
triglycerides (TG) and atherosclerotic cardiovascular disease
(ASCVD) risk without any negative impact on overall health.
- CTX320 is currently in an ongoing
Phase 1 trial targeting LPA in patients with elevated
lipoprotein(a) [Lp(a)], which has shown to have an independent
association with major adverse cardiovascular events (MACE). Up to
20% of the global population has elevated Lp(a) levels.
- The Company continues to advance
two additional preclinical programs, CTX340™ targeting
angiotensinogen (AGT) for the treatment of refractory hypertension
and CTX450™ targeting 5’ aminolevulinic acid synthase (ALAS1) for
the treatment of acute hepatic porphyrias (AHP). CRISPR
Therapeutics has initiated IND/CTA-enabling studies for CTX340,
targeting hepatic AGT for hypertension, and expects to initiate
both clinical trials in the second half of 2025.
- Regenerative Medicine
- CTX211™, an allogeneic,
gene-edited, stem cell-derived beta islet cell precursor, is
currently in an ongoing Phase 1 clinical trial for the treatment of
Type 1 Diabetes (T1D). CRISPR Therapeutics remains committed to its
goal of developing a beta-cell replacement product that does not
require chronic immunosuppression.
- Vertex has non-exclusive rights to
certain CRISPR Therapeutics’ CRISPR/Cas9 technology to accelerate
development of potentially curative cell therapies for T1D. CRISPR
Therapeutics remains eligible for development milestones and would
receive royalties on any future products resulting from this
agreement.
- Other Corporate Matters
- In May, CRISPR Therapeutics
announced the appointment of Naimish Patel, M.D., as Chief
Medical Officer. Dr. Patel brings in-depth experience in
successfully accelerating innovation and advancing drug candidates
across a breadth of modalities and disease areas. Dr. Patel is an
experienced drug developer who has worked across a wide range of
disease areas, including his most recent leadership role as the
Global Development Therapeutic Area Head of Immunology and
Inflammation at Sanofi. In addition, the Company announced the
promotions of (i) Julianne Bruno, M.B.A., to Chief Operating
Officer; Ms. Bruno previously served as the Company’s Senior Vice
President and Head of Programs & Portfolio Management; and (ii)
Susan Kim to Senior Vice President, Investor Relations and
Corporate Communications; Ms. Kim previously served as the
Company’s Vice President of Investor Relations and Corporate
Communications.
- Second Quarter 2024 Financial Results
- Cash Position:
Cash, cash equivalents, and marketable securities were $2,012.8
million as of June 30, 2024, compared to $1,695.7 million as of
December 31, 2023. The increase in cash was primarily driven by
proceeds from the $280.0 million February 2024 registered direct
offering, a $200.0 million milestone payment received from Vertex
in connection with the approval of CASGEVY, proceeds from employee
option exercises as well as interest income, offset by operating
expenses.
- Revenue: Total
collaboration revenue for the second quarter of 2024 was not
material. Collaboration revenue for the second quarter of 2023 was
$70.0 million. Collaboration revenue recognized in the second
quarter of 2023 was primarily attributable to a research milestone
achieved during the current quarter in connection with a
non-exclusive license agreement with Vertex.
- R&D Expenses:
R&D expenses were $80.2 million for the second quarter of 2024,
compared to $101.6 million for the second quarter of 2023. The
decrease in R&D expense was primarily driven by reduced
variable external research and manufacturing costs.
- G&A Expenses:
General and administrative expenses were $19.5 million for the
second quarter of 2024, compared to $19.0 million for the second
quarter of 2023.
- Collaboration
Expense: Collaboration expense, net, was $52.1 million for
the second quarter of 2024, compared to $44.6 million for the
second quarter of 2023. The increase in collaboration expense, net,
was primarily attributable to manufacturing and commercial costs
under the CASGEVY collaboration with Vertex.
- Net Loss: Net loss
was $126.4 million for the second quarter of 2024, compared to a
net loss of $77.7 million for the second quarter of 2023.
About CASGEVY™ (exagamglogene autotemcel
[exa-cel]) CASGEVY™ is a non-viral, ex
vivo CRISPR/Cas9 gene-edited cell therapy for eligible
patients with SCD or TDT, in which a patient’s own hematopoietic
stem and progenitor cells are edited at the erythroid specific
enhancer region of the BCL11A gene. This edit results in
the production of 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.
CASGEVY has been shown to reduce or eliminate VOCs for patients
with SCD and transfusion requirements for patients with TDT.
CASGEVY is approved for certain indications in multiple
jurisdictions for eligible patients.
About the CRISPR Therapeutics-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. CASGEVY
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 CASGEVY and splits program costs and profits worldwide 60/40
with CRISPR Therapeutics. Vertex is the manufacturer and
exclusive license holder of CASGEVY™.
About CTX112 CTX112 is being
developed for both oncology and autoimmune indications. CTX112 is a
next-generation, wholly-owned, allogeneic CAR T product candidate
targeting Cluster of Differentiation 19, or 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. In addition, the Company has opened a
clinical trial of CTX112 in systemic lupus erythematosus.
About CTX131 CTX131 is being
developed for both solid tumors and hematologic malignancies,
including T cell lymphomas (TCL). CTX131 is a next-generation,
wholly-owned, allogeneic CAR T product candidate targeting Cluster
of Differentiation 70, or CD70, an antigen expressed on various
solid tumors and hematologic malignancies. CTX131 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. In
addition, we have opened a clinical trial of CTX131 in hematologic
malignancies, including TCL.
About In Vivo Programs
CRISPR Therapeutics has established a proprietary LNP platform for
the delivery of CRISPR/Cas9 to the liver. The Company’s in vivo
portfolio includes its lead investigational programs, CTX310
(directed towards angiopoietin-related protein 3 (ANGPTL3)) and
CTX320 (directed towards LPA, the gene encoding apo(a), a critical
component of lipoprotein(a) [Lp(a)]), targeting two validated
therapeutic targets for cardiovascular disease. CTX310 and CTX320
are in ongoing clinical trials in patients with heterozygous
familial hypercholesterolemia, homozygous familial
hypercholesterolemia, mixed dyslipidemias, or severe
hypertriglyceridemia, and in patients with elevated lipoprotein(a),
respectively. In addition, the Company’s research and preclinical
development candidates include CTX340 and CTX450, targeting
angiotensinogen (AGT) for refractory hypertension and
5’-aminolevulinate synthase 1 (ALAS1) for acute hepatic porphyria
(AHP), respectively.
About CTX211 CTX211 is an
allogeneic, gene-edited, stem cell-derived investigational therapy
for the treatment of type 1 diabetes (T1D), which incorporates gene
edits that aim to make cells hypoimmune and enhance cell fitness.
This immune-evasive cell replacement therapy is designed to enable
patients to produce their own insulin in response to glucose. A
Phase 1 clinical trial for CTX211 for the treatment of T1D is
ongoing.
About CRISPR Therapeutics
Since its inception over a decade ago, CRISPR Therapeutics has
transformed from a research-stage company advancing programs in the
field of gene editing, to a company that recently celebrated the
historic approval of the first-ever CRISPR-based therapy and has a
diverse portfolio of product candidates across a broad range of
disease areas including hemoglobinopathies, oncology, regenerative
medicine, cardiovascular, autoimmune, and rare diseases. CRISPR
Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited
therapy into the clinic in 2018 to investigate the treatment of
sickle cell disease or transfusion-dependent beta thalassemia, and
beginning in late 2023, CASGEVY™ (exagamglogene autotemcel
[exa-cel]) was approved in some countries to treat eligible
patients with either of those conditions. The Nobel Prize-winning
CRISPR science has revolutionized biomedical research and
represents a powerful, clinically validated approach with the
potential to create a new class of potentially transformative
medicines. To accelerate and expand its efforts, CRISPR
Therapeutics has established strategic partnerships with leading
companies including Bayer and Vertex Pharmaceuticals. 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. To learn more, visit www.crisprtx.com.
CRISPR THERAPEUTICS® standard character mark and
design logo, CTX112™, CTX131™, CTX211™, CTX310™, CTX320™, CTX340™
and CTX450™ are trademarks and registered trademarks of CRISPR
Therapeutics AG. The CASGEVY™ word mark and design are trademarks
of Vertex Pharmaceuticals Incorporated. All other trademarks and
registered trademarks are the property of their respective
owners.
CRISPR Therapeutics Forward-Looking
Statement Statements contained in this press release
regarding matters that are not historical facts are
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995. Because such statements
are subject to risks and uncertainties, actual results may differ
materially from those expressed or implied by such forward-looking
statements. Such statements include, but are not limited to,
statements regarding any or all of the following: (i) CRISPR
Therapeutics preclinical studies, clinical trials and pipeline
products and programs, including, without limitation, manufacturing
capabilities, status of such studies and trials, potential
expansion into new indications and expectations regarding data,
safety and efficacy generally; (ii) its strategy, goals,
anticipated financial performance and the sufficiency of its cash
resources; (iii) regulatory submissions and authorizations,
including timelines for and expectations regarding additional
regulatory agency decisions; (iv) the expected benefits of its
collaborations; and (v) the therapeutic value, development, and
commercial potential of CRISPR/Cas9 gene editing technologies and
therapies, including as compared to other therapies. Risks that
contribute to the uncertain nature of the forward-looking
statements include, without limitation, the risks and uncertainties
discussed under the heading “Risk Factors” in its 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. 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. We disclaim 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.
This press release discusses CRISPR/Cas9 gene
editing investigational therapies and is not intended to convey
conclusions about efficacy or safety as to those investigational
therapies or uses of such investigational therapies. There is no
guarantee that any investigational therapy will successfully
complete clinical development or gain approval from applicable
regulatory authorities.
Investor Contact: Susie Kim
+1-617-307-7503 susan.kim@crisprtx.com
Media Contact: Rachel Eides
+1-617-315-4493 rachel.eides@crisprtx.com
CRISPR Therapeutics AG
Condensed Consolidated Statements of Operations
(Unaudited, In thousands except share data and per share data)
|
|
Three Months Ended June 30, |
|
|
Six Months Ended June 30, |
|
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
Revenue: |
|
|
|
|
|
|
|
|
|
|
|
|
Collaboration revenue |
|
$ |
— |
|
|
$ |
70,000 |
|
|
$ |
— |
|
|
$ |
170,000 |
|
Grant revenue |
|
|
517 |
|
|
|
— |
|
|
|
1,021 |
|
|
|
— |
|
Total revenue |
|
|
517 |
|
|
$ |
70,000 |
|
|
$ |
1,021 |
|
|
$ |
170,000 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
80,165 |
|
|
|
101,555 |
|
|
|
156,338 |
|
|
|
201,490 |
|
General and administrative |
|
|
19,481 |
|
|
|
19,032 |
|
|
|
37,434 |
|
|
|
41,392 |
|
Collaboration expense, net |
|
|
52,131 |
|
|
|
44,636 |
|
|
|
99,097 |
|
|
|
86,828 |
|
Total operating expenses |
|
|
151,777 |
|
|
|
165,223 |
|
|
|
292,869 |
|
|
|
329,710 |
|
Loss from
operations |
|
|
(151,260 |
) |
|
|
(95,223 |
) |
|
|
(291,848 |
) |
|
|
(159,710 |
) |
Total other
income, net |
|
|
26,139 |
|
|
|
18,406 |
|
|
|
50,860 |
|
|
|
31,148 |
|
Net loss
before income taxes |
|
|
(125,121 |
) |
|
|
(76,817 |
) |
|
|
(240,988 |
) |
|
|
(128,562 |
) |
Provision for income taxes |
|
|
(1,287 |
) |
|
|
(923 |
) |
|
|
(2,011 |
) |
|
|
(2,243 |
) |
Net
loss |
|
|
(126,408 |
) |
|
|
(77,740 |
) |
|
|
(242,999 |
) |
|
|
(130,805 |
) |
Foreign currency translation adjustment |
|
|
2 |
|
|
|
28 |
|
|
|
(9 |
) |
|
|
60 |
|
Unrealized (loss) gain on marketable securities |
|
|
(1,329 |
) |
|
|
452 |
|
|
|
(4,783 |
) |
|
|
6,679 |
|
Comprehensive loss |
|
$ |
(127,735 |
) |
|
$ |
(77,260 |
) |
|
$ |
(247,791 |
) |
|
$ |
(124,066 |
) |
Net loss per
common share — basic |
|
$ |
(1.49 |
) |
|
$ |
(0.98 |
) |
|
$ |
(2.92 |
) |
|
$ |
(1.66 |
) |
Basic
weighted-average common shares outstanding |
|
|
84,920,929 |
|
|
|
79,091,061 |
|
|
|
83,357,780 |
|
|
|
78,885,168 |
|
Net loss per
common share — diluted |
|
$ |
(1.49 |
) |
|
$ |
(0.98 |
) |
|
$ |
(2.92 |
) |
|
$ |
(1.66 |
) |
Diluted
weighted-average common shares outstanding |
|
|
84,920,929 |
|
|
|
79,091,061 |
|
|
|
83,357,780 |
|
|
|
78,885,168 |
|
CRISPR Therapeutics AG
Condensed Consolidated Balance Sheets Data
(Unaudited, in thousands)
|
|
As of |
|
|
June 30, 2024 |
|
December 31, 2023 |
Cash and cash equivalents |
|
$ |
484,472 |
|
$ |
389,477 |
Marketable
securities |
|
|
1,517,147 |
|
|
1,304,215 |
Marketable
securities, non-current |
|
|
11,216 |
|
|
1,973 |
Working
capital |
|
|
1,882,584 |
|
|
1,799,287 |
Total
assets |
|
|
2,339,853 |
|
|
2,229,571 |
Total
shareholders' equity |
|
|
1,980,949 |
|
|
1,882,803 |
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