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-On track to begin CTX001 clinical study in hemoglobinopathies in 2018-
-On track to file IND in 2018 for CTX101, allogeneic CRISPR-based CAR-T targeted toward CD19+ malignancies-
“We continue to make rapid progress with our lead program, CTX001 for β-thalassemia and sickle cell disease, as well as our portfolio of allogeneic CRISPR-based CAR-T cell therapies,” said
Recent Highlights and Outlook
- Remains on track for CTX001 in β-thalassemia and sickle cell disease (SCD). After announcing the acceptance of the first Clinical Trial Application (CTA), CRISPR, together with its partner, Vertex, remains on track to initiate a Phase 1/2 trial to assess the safety and efficacy of CTX001 in patients with transfusion dependent β-thalassemia in the second half of 2018. In addition, the Company remains on track to begin clinical studies for CTX001 in SCD in 2018. CTX001 is an autologous gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies.
- Continuing to advance wholly-owned allogeneic CRISPR-based CAR-T cell therapies. CRISPR remains on track to file an IND for CTX101, its lead allogeneic CAR-T cell therapy targeted toward CD19+ malignancies, by year-end 2018. At the
American Association for Cancer Research(AACR) Annual Meeting held in April 2018, CRISPR presented data demonstrating the generation of CAR-T cells targeted toward BCMA and CD70 through CRISPR/Cas9 gene editing that have high editing rates, consistent expression, and selective and potent cell killing.
- Continued recruitment of top senior leadership. As CRISPR advances toward the clinic, the Company continues to recruit top talent across all functions. Earlier in 2018, CRISPR announced the hiring of Steve Caffé, M.D., as the Head of Regulatory of Affairs and
Shelby Walkeras the Head of Intellectual Property. The company has continued to expand its capabilities in all departments including Research, Clinical, Manufacturing, and other functions.
- Completed successful follow-on offering. In
January 2018, CRISPR announced the completion of a follow-on offering, including full exercise of the overallotment, totaling 5,750,000 shares of its common stock at a price of $22.75for net proceeds of $122.6 million.
First Quarter 2018 Financial Results
- Cash Position: Cash as of March 31, 2018 was
$341.8 million, compared to $239.8 million as of December 31, 2017, an increase of $102.0 million. The increase in cash was primarily driven by the January 2018follow-on offering.
- Revenues: Total collaboration revenues were $1.4 million for the first quarter of 2018 compared to $2.7 million for the first quarter of 2017. CRISPR’s collaboration revenue is primarily attributable to revenue recognized under the collaboration agreement with Vertex for work outside hemoglobinopathies. Cost sharing on the Vertex co-development and co-promotion agreement related to hemoglobinopathies is not included in revenue, but instead as an offset to expense in R&D.
- R&D Expenses: R&D expenses were $19.5 million for the first quarter of 2018 compared to $14.8 million for the first quarter of 2017. The increase in expense was driven by the advancement of our hemoglobinopathies program, the broadening of our wholly owned immuno-oncology portfolio, as well as increased investment in our CRISPR/Cas9 platform research.
- G&A Expenses: General and administrative expenses were $8.8 million for the first quarter of 2018 compared to $8.6 million for the first quarter of 2017.
- Net Loss: Net loss was $28.3 million for the first quarter of 2018 compared to a loss of
$21.5 millionfor the first quarter of 2017, driven predominantly by increased R&D expense in the quarter.
CRISPR Forward-Looking Statement
Certain statements set forth in this press release constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, statements concerning: the timing of filing of clinical trial applications and INDs, any approvals thereof and timing of commencement of clinical trials, the intellectual property coverage and positions of the Company, its licensors and third parties, the sufficiency of the Company’s cash resources and the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. You are cautioned that forward-looking statements are inherently uncertain. Although the Company believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, the 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: uncertainties regarding the intellectual property protection for our technology and intellectual property belonging to third parties; uncertainties inherent in the initiation and completion of preclinical studies for the Company’s 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; expectations for regulatory approvals to conduct trials or to market products; and those risks and uncertainties described under the heading “Risk Factors” in the Company’s most recent annual report on Form 10-K, and in any other subsequent filings made by the Company with the U.S. Securities and Exchange Commission (
Condensed Consolidated Statements of Operations
(Unaudited, In thousands except share data and per share data)
|Three Months Ended March 31,|
|Research and development||19,519||14,805|
|General and administrative||8,836||8,642|
|Total operating expenses||28,355||23,447|
|Loss from operations||(26,997||)||(20,744||)|
|Total other (expense) income, net||(1,217||)||(452||)|
|Net loss before income taxes||(28,214||)||(21,196||)|
|Provision for income taxes||(86||)||(279||)|
|Foreign currency translation adjustment||12||24|
|Reconciliation of net loss to net loss attributable to common shareholders:|
|Loss attributable to noncontrolling interest||-||-|
|Net loss attributable to common shareholders||$||(28,300||)||$||(21,475||)|
|Net loss per share attributable to common
shareholders - basic and diluted
|Weighted-average common shares outstanding used in
calculating net loss per share attributable to common
shareholders - basic and diluted
Condensed Consolidated Balance Sheets Data
(Unaudited, in thousands)
|March 31, 2018||December 31, 2017|
|Total shareholders' equity||290,335||187,832|
WCG on behalf of CRISPR
Westwicke Partners on behalf of CRISPR
Source: CRISPR Therapeutics AG