CRISPR Therapeutics Provides Business Update and Reports Third Quarter 2019 Financial Results
October 28 2019 - 7:00AM
CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical
company focused on creating transformative gene-based medicines for
serious diseases, today reported financial results for the third
quarter ended September 30, 2019.
“In 2019, we’ve made significant progress across several
development programs, including ongoing enrollment of our CTX001
trials in beta thalassemia and severe sickle cell disease, with
preliminary data expected from these programs later this year. We
also began treating patients in our clinical trial for CTX110, our
allogeneic CAR-T therapy, and are advancing additional CAR-T
candidates toward clinical development,” said Samarth Kulkarni,
Ph.D., Chief Executive Officer of CRISPR Therapeutics. “Looking
forward, we expect a robust 2020, with continued focus on execution
as we anticipate conducting five clinical trials in parallel.”
Recent Highlights and
Outlook
- Beta Thalassemia and Sickle Cell Disease
- Enrollment is ongoing at six clinical trial sites in the U.S.,
Canada and Europe for the Phase 1/2 study of CTX001 in patients
with transfusion-dependent beta thalassemia (TDT) and at ten
clinical trial sites in the U.S., Canada and Europe for the study
in patients with severe sickle cell disease (SCD).
- The European Medicines Agency (EMA) Committee for Orphan
Medicinal Products (COMP) issued a positive opinion for orphan drug
designation (ODD) of CTX001 for the treatment of TDT. In addition,
we are expanding the TDT patient population for CTX001 to include
beta zero/beta zero subtypes.
- The Company expects to release preliminary safety and efficacy
data from the ongoing Phase 1/2 clinical trials in late 2019.
- Immuno-Oncology
- The Company has begun treating patients in a Phase 1/2 trial to
assess the safety and efficacy of CTX110, its wholly-owned
allogeneic CAR-T cell therapy targeting CD19+ malignancies. The
multi-center, open label trial is designed to enroll up to 95
patients and investigate several dose levels of CTX110. The study
is currently enrolling at five clinical trial sites in the U.S. and
Australia. In addition, the Company obtained approval from Health
Canada for its Clinical Trial Application (CTA). The Company
believes its CRISPR-based allogeneic CAR-Ts may have a superior
product profile compared to current autologous therapies and allow
accessibility to broader patient populations.
- The Company expects to initiate a Phase 1/2 clinical trial of
CTX120™, targeting B-cell maturation antigen (BCMA) for the
treatment of multiple myeloma, in the first half of 2020. CRISPR
Therapeutics continues to advance additional allogeneic CAR-T
candidates toward clinical development including CTX130™, targeting
CD70 for the treatment of solid tumors and hematologic
malignancies. The Company continues to scale its capabilities to
enable rapid advancement of these programs into and through the
clinic.
- The Company recently announced it entered into a license
agreement with KSQ Therapeutics whereby CRISPR Therapeutics gained
access to KSQ intellectual property (IP) for editing certain novel
gene targets in its allogeneic oncology cell therapy programs, and
KSQ gained access to CRISPR Therapeutics’ IP for editing novel gene
targets identified by KSQ as part of its current and future eTILTM
(engineered tumor infiltrating lymphocyte) cell programs. The
agreement further strengthens the Company’s proprietary allogeneic
CAR-T platform.
- The Company will present a poster at the Society for
Immunotherapy of Cancer (SITC) Conference on November 9, 2019
related to single-cell RNA sequencing and functional assessment of
healthy donor and cancer patient-derived T and CAR-T cells
(#P187).
- Regenerative Medicine
- On September 17, 2019, CRISPR Therapeutics, in collaboration
with ViaCyte, presented positive in vitro data for a potential
immune-evasive cell replacement therapy for diabetes at the 55th
Annual Meeting of the European Association for the Study of
Diabetes (EASD) in Barcelona, Spain. The oral presentation included
new data that demonstrate the successful differentiation of
CRISPR-edited human pluripotent stem cells to pancreatic precursor
cells.
- Other Corporate Matters
- Vertex has exercised the options granted under the
collaboration it established with CRISPR Therapeutics in 2015 to
in-license three additional targets for the development of
treatments using CRISPR-based gene editing. The targets include the
cystic fibrosis transmembrane conductance regulator (CFTR) gene and
two undisclosed targets. Under the terms of the agreement, CRISPR
Therapeutics will receive an upfront payment of $30 million in
connection with the option exercise and has the potential to
receive up to $410 million in development, regulatory and
commercial milestones and royalties on net product sales for each
of the three targets, and Vertex will receive exclusive rights to
develop and commercialize products related to these targets
globally. The research term of the Company’s 2015 collaboration
with Vertex has now expired, and Vertex no longer holds rights to
in-license additional targets under that agreement.
- The Company recently announced proposed plans that Casebia
Therapeutics, previously a joint venture between CRISPR
Therapeutics and Bayer, would operate under the direct management
of CRISPR Therapeutics. Upon closing of the transaction, Casebia
Therapeutics would focus on the development of its lead programs in
hemophilia, ophthalmology and autoimmune diseases, with Bayer
having opt-in rights for two products at IND submission. The
transaction is subject to negotiation and execution of definitive
agreements as well as certain customary conditions. The
Company and Bayer are negotiating the definitive agreements and,
subject to the finalization of the definitive agreements and
satisfaction of closing conditions, anticipate to close the
transaction in the fourth quarter of 2019.
Third Quarter 2019 Financial
Results
- Cash Position: Cash as of September 30, 2019,
was $629.7 million, compared to $427.9 million as of June 30, 2019,
an increase of $201.8 million as increased cash operating expenses
were offset by $68.6 million net proceeds from financing activities
and $175 million upfront payments received from Vertex related to
the 2019 collaboration agreement announced in June.
- Revenues: Total collaboration revenues were
$211.9 million for the third quarter of 2019 compared to $0.6
million for third quarter of 2018 with the increase primarily
driven by the collaboration agreement with Vertex.
- R&D Expenses: R&D expenses were $57.2
million for the third quarter of 2019 compared to $39.8 million for
the third quarter of 2018. The increase was driven by increased
headcount and services expense supporting the advancement of the
hemoglobinopathies program, the broadening of the Company’s
wholly-owned immuno-oncology portfolio, as well as increased
investment in the Company’s CRISPR/Cas9 platform research and some
non-cash expense related to the Company’s collaboration with
Vertex.
- G&A Expenses: General and administrative
expenses were $15.5 million for the third quarter of 2019 compared
to $10.2 million for the third quarter of 2018. The increase
was driven by increased headcount-related expense and external
professional and consulting service expense.
- Net Income (Loss): Net Income was $138.4
million for the third quarter of 2019 compared to a loss of $50.7
million for the third quarter of 2018, driven predominantly by
increased revenue recognized in connection with the Company’s
collaboration with Vertex.
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) the safety, efficacy and clinical
progress of CRISPR Therapeutics’ various clinical programs
including CTX001, CTX110, CTX 120 and CTX 130; (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
proposed transaction involving 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 KSQ 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 (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’ and/or Casebia
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; uncertainties inherent in the proposed
transaction involving Casebia Therapeutics, including the expected
timing for completion of such transaction and the possibility such
transaction is not consummated; 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.
eTILTM is a trademark of KSQ Therapeutics, Inc. All other
trademarks referenced herein are the property of CRISPR
Therapeutics.
CRISPR Therapeutics
AGCondensed Consolidated Statements of
Operations(Unaudited, In thousands except share data and
per share data)
|
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
|
|
2019 |
|
|
2018 |
|
|
2019 |
|
|
2018 |
|
Collaboration revenue |
|
$ |
211,928 |
|
|
$ |
563 |
|
|
$ |
212,574 |
|
|
$ |
3,009 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
57,246 |
|
|
|
39,820 |
|
|
|
130,601 |
|
|
|
84,972 |
|
General and administrative |
|
|
15,519 |
|
|
|
10,175 |
|
|
|
46,216 |
|
|
|
31,752 |
|
Total operating expenses |
|
|
72,765 |
|
|
|
49,995 |
|
|
|
176,817 |
|
|
|
116,724 |
|
Income (loss) from operations |
|
|
139,163 |
|
|
|
(49,432 |
) |
|
|
35,757 |
|
|
|
(113,715 |
) |
Total other income (expense), net |
|
|
(466 |
) |
|
|
(1,142 |
) |
|
|
1,003 |
|
|
|
(3,357 |
) |
Net income (loss) before income taxes |
|
|
138,697 |
|
|
|
(50,574 |
) |
|
|
36,760 |
|
|
|
(117,072 |
) |
Provision for income taxes |
|
|
(274 |
) |
|
|
(137 |
) |
|
|
(444 |
) |
|
|
(319 |
) |
Net income (loss) |
|
|
138,423 |
|
|
|
(50,711 |
) |
|
|
36,316 |
|
|
|
(117,391 |
) |
Foreign currency translation adjustment |
|
|
(12 |
) |
|
|
(6 |
) |
|
|
(14 |
) |
|
|
(15 |
) |
Comprehensive income (loss) |
|
$ |
138,411 |
|
|
$ |
(50,717 |
) |
|
$ |
36,302 |
|
|
$ |
(117,406 |
) |
Reconciliation of net income (loss) to net income (loss)
attributable to common shareholders: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net income (loss) |
|
$ |
138,423 |
|
|
$ |
(50,711 |
) |
|
$ |
36,316 |
|
|
$ |
(117,391 |
) |
Net income (loss) per share attributable to common shareholders -
basic |
|
$ |
2.52 |
|
|
$ |
(1.07 |
) |
|
$ |
0.68 |
|
|
$ |
(2.51 |
) |
Weighted-average common shares outstanding used in calculating net
loss per share attributable to common shareholders - basic |
|
|
54,829,057 |
|
|
|
47,391,988 |
|
|
|
53,380,123 |
|
|
|
46,709,388 |
|
Net income (loss) per share attributable to common shareholders -
diluted |
|
$ |
2.40 |
|
|
$ |
(1.07 |
) |
|
$ |
0.65 |
|
|
$ |
(2.51 |
) |
Weighted-average common shares outstanding used in calculating net
loss per share attributable to common shareholders - diluted |
|
|
57,598,901 |
|
|
|
47,391,988 |
|
|
|
55,821,420 |
|
|
|
46,709,388 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
CRISPR Therapeutics
AGCondensed Consolidated Balance Sheets
Data(Unaudited, in thousands)
|
|
As of |
|
|
|
30-Sep-19 |
|
|
31-Dec-18 |
|
Cash |
|
$ |
629,717 |
|
|
$ |
456,649 |
|
Working capital |
|
|
584,286 |
|
|
|
438,649 |
|
Total assets |
|
|
720,590 |
|
|
|
489,016 |
|
Total shareholders' equity |
|
|
591,878 |
|
|
|
392,195 |
|
Investor Contact:Susan
Kimsusan.kim@crisprtx.com
Media Contact:Jennifer PaganelliWCG on behalf
of CRISPR347-658-8290jpaganelli@wcgworld.com
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