CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2019 Financial Results
February 12 2020 - 4:05PM
CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical
company focused on creating transformative gene-based medicines for
serious diseases, today reported financial results for the fourth
quarter and full year ended December 31, 2019.
“In 2019, CRISPR Therapeutics achieved important milestones and
momentum across key programs. We announced positive interim safety
and efficacy data from the first two patients in our ongoing CTX001
clinical trials, one patient with beta thalassemia and one patient
with sickle cell disease. These preliminary data support our belief
in the potential of CTX001 to have meaningful benefit for patients
following a one-time intervention,” said Samarth Kulkarni, Ph.D.,
Chief Executive Officer of CRISPR Therapeutics. “In addition, we
advanced our first allogeneic CAR-T cell therapy, CTX110, targeting
CD19+ malignancies and, building on this progress, today announced
that we have begun enrolling patients in a clinical trial for our
second allogeneic CAR-T therapy, CTX120, targeting BCMA for the
treatment of relapsed or refractory multiple myeloma.”
Dr. Kulkarni added: “2020 has the potential to be a pivotal year
in our company’s growth. We expect to conduct clinical trials in
five indications, and we anticipate new data from our
immuno-oncology and hemoglobinopathies programs. Our continued
progress brings us closer to potentially providing transformative
therapies to patients with serious diseases.”
2019 Highlights and Outlook |
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Beta Thalassemia and Sickle Cell Disease |
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In November 2019, CRISPR Therapeutics and its partner Vertex
announced positive, interim data from the first two patients with
severe hemoglobinopathies - one patient with transfusion-dependent
beta thalassemia (TDT) and one patient with severe sickle cell
disease (SCD) - treated with the investigational CRISPR/Cas9
gene-editing therapy CTX001 in the ongoing Phase 1/2 CLIMB clinical
trials. Enrollment is ongoing in both trials and the companies
expect to provide additional data for these programs in 2020. |
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CTX001 has been granted orphan drug designation (ODD) by the
European Commission for the treatment of SCD. The European
Commission previously granted CTX001 ODD for the treatment of
TDT. |
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Immuno-Oncology |
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Patient enrollment continues in a clinical trial to assess the
safety and efficacy of CTX110, its 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
immuno-oncology. |
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CRISPR Therapeutics has begun enrolling 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. |
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CRISPR Therapeutics continues to advance additional allogeneic
CAR-T candidates toward clinical development, including 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. The Company
continues to scale its capabilities to enable rapid advancement of
these programs into and through the clinic. |
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Other Corporate Matters |
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In November 2019, CRISPR Therapeutics announced the pricing of an
underwritten public offering of 4,250,000 common shares at a public
offering price of $64.50 per share, plus the exercise in full of
the underwriters’ option to purchase 637,500 additional common
shares. Gross proceeds from the offering (including the exercise of
the underwriters’ option), before deducting underwriting discounts
and commissions and other offering expenses, were $315.2 million.
The initial offering closed in November 2019 and the closing of the
option to purchase additional shares occurred in December
2019. |
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In 2019, CRISPR Therapeutics broadened its business development
efforts through additional transactions, including an agreement
with Bayer to place Casebia Therapeutics, previously a joint
venture between Bayer and CRISPR Therapeutics, under CRISPR
Therapeutics’ direct management; a license agreement with KSQ
Therapeutics whereby CRISPR Therapeutics gains access to KSQ
intellectual property (IP) for editing certain novel gene targets
in its allogeneic oncology cell therapy programs, and KSQ gains
access to CRISPR Therapeutics’ IP for editing novel gene targets
for certain applications; a collaboration and license agreement
with ProBioGen focused on the development of novel in vivo delivery
modalities for CRISPR/Cas9 leveraging ProBioGen’s existing
technology and expertise; and a collaboration with StrideBio, which
expands upon an existing agreement to generate engineered
adeno-associated viruses (AAV) capsids with improved properties for
in vivo gene editing programs and includes additional undisclosed
applications. |
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In June 2019, CRISPR Therapeutics and Vertex expanded their
collaboration and entered into an exclusive licensing agreement to
discover and develop gene editing therapies for the treatment of
Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1
(DM1). In connection with this agreement, CRISPR Therapeutics
received a $175.0 million up-front payment from Vertex, and is
eligible to receive milestone payments from Vertex of up to $825.0
million in the aggregate. CRISPR Therapeutics continues to make
advancements with programs utilizing an in vivo approach, which
remains a key area of focus. |
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In June 2019, CRISPR Therapeutics received notification that the
United States Patent and Trademark Office (USPTO) has initiated an
interference proceeding at the Patent Trial and Appeal Board
between certain pending U.S. patent applications co-owned by the
University of California, the University of Vienna and Dr.
Emmanuelle Charpentier (collectively, the “CVC Group”) and certain
patents and a patent application currently owned by the Broad
Institute, Harvard University and the Massachusetts Institute of
Technology, all of which are related to the single guide format of
CRISPR/Cas9 genome editing technology in eukaryotic cells. As of
December 2019, the USPTO has granted twenty-one patents to the CVC
group. None of these issued patents are involved in the
interference. |
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Fourth Quarter 2019 Financial Results |
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Cash Position: Cash and cash equivalents as of
December 31, 2019, were $943.8 million, compared to $456.6 million
as of December 31, 2018, an increase of $487.2 million. The
increase in cash was primarily driven by cash from financing
activities of $430.9 million from our at-the market offering,
completed in July 2019; from our November public offering; and from
stock options exercised during 2019. In addition, the Company
generated cash from operations in the current year of $56.7
million, driven by cash received from Vertex for milestone and
option payments of $208.0 million as well as cash obtained from the
Bayer transaction of $31.8 million, offset by operating
expenses. |
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Revenue: Total collaboration revenue was $77.0
million for the fourth quarter of 2019 compared to $0.1 million for
fourth quarter of 2018, and $289.6 million for the year ended
December 31, 2019, compared to $3.1 million for the year ended
December 31, 2018. The increase in revenue was primarily
attributable to revenue recognized in connection with the Company’s
collaboration agreements with Vertex. |
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R&D Expenses: R&D expenses were $48.8
million for the fourth quarter of 2019 compared to $28.8 million
for the fourth quarter of 2018, and $179.4 million for the year
ended December 31, 2019 compared to $113.8 million for the year
ended December 31, 2018. The increase in expense for the year was
driven by increased headcount and development activities supporting
the advancement of the hemoglobinopathies program, the broadening
of the Company’s wholly-owned immuno-oncology portfolio and $10.0
million of non-cash expense related to the Company’s collaboration
with Vertex. |
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G&A Expenses: General and administrative
expenses were $17.3 million for the fourth quarter of 2019 compared
to $16.5 million for the fourth quarter of 2018, and $63.5 million
for the year ended December 31, 2019, compared to $48.3 million for
the year ended December 31, 2018. The increase in general and
administrative expenses for the year was driven by
headcount-related expense and external professional and consulting
service expense. |
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Net Income/Loss: Net income was $30.5 million for
the fourth quarter of 2019 compared to net loss of $47.6 million
for the fourth quarter of 2018, and net income was $66.9 million
for the year ended December 31, 2019, compared to a loss of $165.0
million for the year ended December 31, 2018. |
About CTX001TMCTX001 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 CTX110TMCTX110 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 in a clinical trial designed to assess the safety and
efficacy of CTX110 in 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 CTX120TMCTX120 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 in a clinical trial designed to
assess the safety and efficacy of CTX120 in 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 CTX130TMCTX130 is a
healthy donor-derived gene-edited allogeneic CAR-T therapy
targeting CD70, an antigen expressed on hematologic cancers. A
wholly-owned asset of CRISPR Therapeutics, CTX130 is in development
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) the safety, efficacy and clinical
progress of CRISPR Therapeutics’ various clinical programs
including CTX001, CTX110, CTX120 and CTX130; (ii) the status of
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; (iii) 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; (iv) the
integration of Casebia Therapeutics; (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, including
those with Bayer, KSQ, ProBioGen, StrideBio and Vertex; 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 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 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’; 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; the risk that the CRISPR Therapeutics’
business and Casebia Therapeutics’ business will not be integrated
successfully; 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.
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CRISPR Therapeutics AG |
Condensed Consolidated Statements of
Operations |
(Unaudited, In thousands except share data and per share data) |
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Three Months Ended December 31, |
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Year Ended December 31, |
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2019 |
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2018 |
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2019 |
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2018 |
|
Collaboration revenue |
$ |
77,016 |
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$ |
115 |
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$ |
289,590 |
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$ |
3,124 |
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Operating expenses: |
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|
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|
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|
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Research and development |
|
48,762 |
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|
28,801 |
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|
179,362 |
|
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|
113,773 |
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General and administrative |
|
17,271 |
|
|
|
16,542 |
|
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|
63,488 |
|
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|
48,294 |
|
Total operating expenses |
|
66,033 |
|
|
|
45,343 |
|
|
|
242,850 |
|
|
|
162,067 |
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Income (loss) from operations |
|
10,983 |
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(45,228 |
) |
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46,740 |
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(158,943 |
) |
Total other income (expense), net |
|
19,563 |
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(2,128 |
) |
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20,566 |
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|
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(5,485 |
) |
Net income (loss) before income taxes |
|
30,546 |
|
|
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(47,356 |
) |
|
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67,306 |
|
|
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(164,428 |
) |
Provision for income taxes |
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(4 |
) |
|
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(234 |
) |
|
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(448 |
) |
|
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(553 |
) |
Net income (loss) |
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30,542 |
|
|
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(47,590 |
) |
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|
66,858 |
|
|
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(164,981 |
) |
Foreign currency translation adjustment |
|
29 |
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(7 |
) |
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|
15 |
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(22 |
) |
Comprehensive income (loss) |
$ |
30,571 |
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$ |
(47,597 |
) |
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$ |
66,873 |
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$ |
(165,003 |
) |
Reconciliation of net income (loss) to net income (loss)
attributable to common shareholders: |
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Net income (loss) |
$ |
30,542 |
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$ |
(47,590 |
) |
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$ |
66,858 |
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$ |
(164,981 |
) |
Net income (loss) per share attributable to common shareholders -
basic |
$ |
0.53 |
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$ |
(0.92 |
) |
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$ |
1.23 |
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$ |
(3.44 |
) |
Weighted-average common shares outstanding used in calculating net
loss per share attributable to common shareholders - basic |
|
57,395,839 |
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51,688,383 |
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54,392,304 |
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47,964,368 |
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Net income (loss) per share attributable to common shareholders -
diluted |
$ |
0.51 |
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$ |
(0.92 |
) |
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$ |
1.17 |
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$ |
(3.44 |
) |
Weighted-average common shares
outstanding used in calculating net loss per share attributable to
common shareholders - diluted |
|
60,233,927 |
|
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51,688,383 |
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56,932,798 |
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|
47,964,368 |
|
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CRISPR Therapeutics AG |
Condensed Consolidated Balance Sheets Data |
(Unaudited, in thousands) |
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As of |
|
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December 31, 2019 |
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December 31, 2018 |
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Cash |
$ |
943,771 |
|
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$ |
456,649 |
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Working capital |
|
930,441 |
|
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|
438,649 |
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Total assets |
|
1,066,752 |
|
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|
489,016 |
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Total shareholders' equity |
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939,425 |
|
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|
392,195 |
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Investor Contact:Susan
Kimsusan.kim@crisprtx.com
Media Contact:Rachel EidesWCG on behalf of
CRISPR617-337-4167 reides@wcgworld.com
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