CRISPR Therapeutics Provides Business Update and Reports First Quarter 2020 Financial Results
April 28 2020 - 4:45PM
CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical
company focused on creating transformative gene-based medicines for
serious diseases, today reported financial results for the first
quarter ended March 31, 2020.
“We made substantial progress in the last quarter despite the
challenges posed by COVID-19. We are now progressing five
cell therapy clinical trials in parallel targeting
hemoglobinopathies and various cancers,” said Samarth Kulkarni,
Ph.D., Chief Executive Officer of CRISPR Therapeutics. “Our CTX001
and CTX110 programs continue to enroll patients, and we expect to
report data for these programs this year. We are pleased to
have begun treating patients in our CTX120 trial for the treatment
of relapsed or refractory multiple myeloma and we expect to begin
treating patients in our CTX130 trial in renal cell carcinoma and
T-cell and B-cell hematologic malignancies in the second half of
this year. Despite these unprecedented times, we continue to
execute on our programs and we remain focused on our commitment to
patients and their families.”
Recent Highlights and Outlook
- Beta Thalassemia and Sickle Cell Disease
- CRISPR Therapeutics and its partner Vertex remain on track to
provide additional data from the two ongoing Phase 1/2 studies of
the investigational CRISPR/Cas9 gene-editing therapy CTX001 in
patients with transfusion-dependent beta thalassemia and in
patients with severe sickle cell disease in 2020. New data expected
in 2020 include initial data from additional patients dosed in each
of the Phase 1/2 studies and longer duration follow-up data for the
first patients dosed in each study. Screening, enrollment and
mobilization in these studies is ongoing, however no additional
patients are scheduled to initiate conditioning or dosing at this
time due to COVID-19.
- Immuno-Oncology
- Patient enrollment continues in a clinical trial to assess the
safety and efficacy of CTX110, CRISPR Therapeutics’ wholly-owned
allogeneic CAR-T cell therapy targeting refractory CD19+ B-cell
malignancies. The multi-center, open label clinical trial is
designed to enroll up to 95 patients and investigate several dose
levels of CTX110. If successful, CTX110 could enable off-the-shelf
use of cell therapies and greatly expand their applicability and
accessibility in treating patients with these hematologic
malignancies. The Company expects to report top-line data for
CTX110 at the end of 2020.
- CRISPR Therapeutics has begun treating patients in a clinical
trial to assess the safety and efficacy of CTX120, its wholly-owned
allogeneic CAR-T cell therapy targeting BCMA for the treatment of
relapsed or refractory multiple myeloma. The multi-center, open
label trial is designed to enroll up to 80 patients and investigate
several dose levels of CTX120.
- Earlier this year, the U.S. Food and Drug Administration (FDA)
accepted CRISPR Therapeutics’ Investigational New Drug (IND)
application for CTX130, its wholly-owned allogeneic CAR-T cell
therapy targeting CD70 for the treatment of both solid tumors, such
as renal cell carcinoma, and T-cell and B-cell hematologic
malignancies. Additionally, CRISPR Therapeutics has obtained
approval from Health Canada for its Clinical Trial Application
(CTA). The Company expects to begin treating patients with CTX130
in the second half of this year.
- Other Corporate Matters
- Under the June 2019 collaboration agreement with Vertex to
discover and develop gene editing therapies for the treatment of
Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1
(DM1), CRISPR Therapeutics received a payment of $25 million from
Vertex related to the achievement of a research milestone in the
DM1 program. CRISPR Therapeutics is eligible to receive additional
milestone payments from Vertex of up to $800 million for these two
programs.
- CRISPR Therapeutics is scheduled to present two posters during
the virtual American Society of Gene & Cell Therapy 2020 Annual
Meeting, to be held from May 12 to 15, 2020, via
https://annualmeeting.asgct.org/am20/, as
follows:Title: Dual Guide CRISPR/Cas9 Editing of
the CCR5 Gene Provides Complete Protection Against HIV in Humanized
Mouse Models (abstract #1046)Session Title: Gene
Targeting and Gene CorrectionDate and Time:
Thursday, May 14, 2020; 5:30 PM - 6:30
PM Title: Multiplexing of Up to 10 Gene Edits
Using CRISPR/Cas9 to Generate CAR-T Cells with Improved Function
(abstract #1151)Session Title: Cancer -
Immunotherapy, Cancer VaccinesDate and Time:
Thursday, May 14, 2020; 5:30 PM - 6:30 PM
- In February, CRISPR Therapeutics announced its proposal to
elect Doug Treco, Ph.D. to its Board of Directors at the Company’s
upcoming annual general meeting to be held later this year.
First Quarter 2020 Financial Results
- Cash Position: Cash and cash equivalents as of
March 31, 2020, were $889.7 million, compared to $943.8 million as
of December 31, 2019, a decrease of $54.1 million. The decrease in
cash was primarily driven by cash used in operating activities of
$52.2 million to support spending on the Company’s clinical and
pre-clinical programs, as well as payroll and payroll-related
expenses to support growth. In April 2020, the Company received a
milestone payment of $25 million from Vertex under the
collaboration agreement for DMD and DM1, resulting in pro forma
cash exceeding $900 million.
- Revenue: Total collaboration revenue was $0.2
million for the first quarter of 2020 compared to $0.3 million for
first quarter of 2019. Collaboration revenue primarily consisted of
charges to partners for research activities.
- R&D Expenses: R&D expenses were $54.2
million for the first quarter of 2020 compared to $33.8 million for
the first quarter of 2019. The increase in expenses was driven by
increased headcount and development activities supporting the
advancement of the hemoglobinopathies program and wholly-owned
immuno-oncology programs.
- G&A Expenses: General and administrative
expenses were $19.6 million for the first quarter of 2020 compared
to $14.9 million for the first quarter of 2019. The increase in
general and administrative expenses for the year was driven by
headcount-related expense and higher facilities cost.
- Net Loss: Net loss was $69.7 million for the
first quarter of 2020 compared to net loss of $48.4 million for the
first quarter of 2019.
About CTX001™CTX001 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 CTX110™CTX110 is a
healthy donor-derived gene-edited allogeneic CAR-T therapy
targeting cluster of differentiation 19, or CD19, for the treatment
of CD19+ malignancies. A wholly-owned asset of CRISPR Therapeutics,
CTX110 is being investigated in a clinical trial designed to assess
the safety and efficacy of CTX110 for the treatment of relapsed or
refractory B-cell malignancies. The multi-center, open-label
clinical trial is designed to enroll up to 95 patients and
investigate several dose levels of CTX110.
About CTX120™CTX120 is a
healthy donor-derived gene-edited allogeneic CAR-T therapy
targeting B-cell maturation antigen, or BCMA. A wholly-owned asset
of CRISPR Therapeutics, CTX120 is being investigated in a clinical
trial designed to assess the safety and efficacy of CTX120 for the
treatment of relapsed or refractory multiple myeloma. The
multi-center, open-label clinical trial is designed to enroll up to
80 patients and investigate several dose levels of CTX120.
About CTX130™CTX130 is a
healthy donor-derived gene-edited allogeneic CAR-T therapy
targeting cluster of differentiation 70, or CD70, an antigen
expressed on various solid tumors and hematologic malignancies. A
wholly-owned asset of CRISPR Therapeutics, CTX130 is being
developed for the treatment of both solid tumors, such as renal
cell carcinoma, and T-cell and B-cell hematologic malignancies.
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
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 Cambridge, Massachusetts, and business offices in San Francisco,
California and 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) potential impacts due to the coronavirus
pandemic; (ii) the safety, efficacy and clinical progress of CRISPR
Therapeutics’ various clinical programs including CTX001, CTX110,
CTX120 and CTX130; (iii) the status of clinical trials (including,
without limitation, the timing of filing of clinical trial
applications and INDs, any approvals thereof, the timing of
commencement of clinical trials and the expected timing of data
releases), development timelines and discussions with regulatory
authorities related to product candidates under development by
CRISPR Therapeutics and its collaborators; (iv) 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; (v)
the intellectual property coverage and positions of CRISPR
Therapeutics, its licensors and third parties as well as the status
and potential outcome of proceedings involving any such
intellectual property; (vi) the sufficiency of CRISPR Therapeutics’
cash resources; (vii) the expected benefits of CRISPR Therapeutics’
collaborations; and (viii) 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 potential
impacts due to the coronavirus pandemic such as (x) delays in
regulatory review, manufacturing and supply chain interruptions,
adverse effects on healthcare systems and disruption of the global
economy; (y) the timing and progress of clinical trials,
preclinical studies and other research and development activities;
and (z) the overall impact of the coronavirus pandemic on its
business, financial condition and results of operations; the
potential for initial and preliminary data from any clinical trial
(including CTX001, CTX110, CTX120 and CTX130) not to be indicative
of final trial results; the risk that the initial data from a
limited number of patients (as is the case with CTX001 at this
time) may not be indicative of results from the full planned study
population; 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 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, 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.
CRISPR Therapeutics AG |
Condensed Consolidated Statements of
Operations |
(Unaudited, In thousands except share data and per share data) |
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
2020 |
|
|
|
2019 |
|
Collaboration revenue |
|
$ |
157 |
|
|
$ |
328 |
|
Operating expenses: |
|
|
|
|
Research and development |
|
|
54,193 |
|
|
|
33,822 |
|
General and administrative |
|
|
19,550 |
|
|
|
14,929 |
|
Total operating expenses |
|
|
73,743 |
|
|
|
48,751 |
|
Loss from operations |
|
|
(73,586 |
) |
|
|
(48,423 |
) |
Total other income, net |
|
|
4,232 |
|
|
|
100 |
|
Net loss before income
taxes |
|
|
(69,354 |
) |
|
|
(48,323 |
) |
Provision for income taxes |
|
|
(377 |
) |
|
|
(85 |
) |
Net loss |
|
|
(69,731 |
) |
|
|
(48,408 |
) |
Foreign currency translation adjustment |
|
|
(25 |
) |
|
|
8 |
|
Comprehensive loss |
|
$ |
(69,756 |
) |
|
$ |
(48,400 |
) |
Reconciliation of net loss to
net loss attributable to common shareholders: |
|
|
|
|
Net loss |
|
$ |
(69,731 |
) |
|
$ |
(48,408 |
) |
Net loss per share
attributable to common shareholders - basic |
|
$ |
(1.15 |
) |
|
$ |
(0.93 |
) |
Weighted-average common shares
outstanding used in calculating net loss per share attributable to
common shareholders - basic |
|
|
60,847,683 |
|
|
|
52,093,208 |
|
|
|
|
|
|
CRISPR Therapeutics AG |
Condensed Consolidated Balance Sheets Data |
(Unaudited, in thousands) |
|
|
|
As of |
|
|
March 31, 2020 |
|
December 31, 2019 |
Cash |
|
$ |
889,712 |
|
$ |
943,771 |
Working capital |
|
|
874,196 |
|
|
930,441 |
Total assets |
|
|
1,007,300 |
|
|
1,066,752 |
Total shareholders'
equity |
|
|
886,097 |
|
|
939,425 |
|
|
|
|
|
Investor Contact:Susan
Kimsusan.kim@crisprtx.com
Media Contact:Rachel EidesWCG on behalf of
CRISPR617-337-4167 reides@wcgworld.com
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