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- On track to begin first company-sponsored clinical trials of a CRISPR-based therapy -
- Wholly-owned CRISPR-based allogeneic CAR-T programs advancing rapidly -
ZUG,
“2017 was a momentous year for
“In 2018 we expect to build on our success by beginning a Phase 1/2 study of CTX001 in β-thalassemia, the first company-sponsored clinical trial for a CRISPR-based therapeutic. We also plan to begin clinical trials of CTX001 for sickle cell disease in
Recent Highlights and Outlook:
- Initiating Phase 1/2 trial of CTX001 in β-thalassemia in 2018. CRISPR, together with
Vertex Pharmaceuticals , has filed clinical trial applications (CTAs) in various European countries to conduct a Phase 1/2 trial of CTX001, an autologous gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. CRISPR has received approval for the first of these CTAs and expects to initiate clinical trials inEurope in 2018. The Phase 1/2 trial of CTX001 is designed to assess its safety and efficacy in adult transfusion-dependent β-thalassemia patients. Additionally, the Company plans to file an Investigational New Drug (IND) Application for CTX001 in SCD in the US in the first-half of 2018. - Updated preclinical data for CTX001. CRISPR presented new data at the
American Society of Hematology (ASH) Annual Meeting inDecember 2017 demonstrating that its CRISPR gene-editing approach results in high editing efficiency, with >90% of the hematopoietic stem cells edited at the target site. The expression levels of fetal hemoglobin in these cells were above the level believed to be sufficient to ameliorate symptoms in patients with β-thalassemia and sickle cell disease. - Initiated manufacturing and other IND-enabling activities for CTX101, an allogeneic CAR-T cell therapy targeting CD19+ malignancies. The Company presented in vitro and in vivo data demonstrating potent anti-tumor activity during a poster session at the
Society for Immunotherapy in Cancer (SITC ) 32nd Annual Meeting. The Company believes its allogeneic CAR-T cell therapies may have distinct advantages over the current generation of autologous cell therapies including greater access due to their “off-the-shelf” nature, and greater efficacy due to the homogeneity and consistency of the product. CRISPR plans to file an IND for CTX101 in the US by year-end 2018. - Added two new candidates to the CRISPR-based allogeneic CAR-T portfolio. During a company-sponsored reception at the
SITC 32nd Annual Meeting, CRISPR announced it will be advancing the next two candidates targeting B-cell maturation antigen (BCMA) and CD70. The Company believes it has the necessary capabilities in place to rapidly advance these product candidates to the clinic. - Continued advancement of the pipeline through collaborations with CureVac and StrideBio for in vivo programs. In
November 2017 , CRISPR entered into a collaboration with CureVac to develop novel Cas9 mRNA constructs with improved properties for gene editing applications that could include increased potency, decreased duration of expression and reduced potential for immunogenicity. The Company obtained an exclusive license to the improved constructs for use in three of their in vivo gene-editing programs in liver diseases. Additionally,CRISPR Therapeutics entered into a collaboration with StrideBio to develop AAV vectors with greater tissue specificity, and reduced immunogenicity. - Continued organizational growth and senior leadership changes. CRISPR continues to recruit top talent across all functions as it evolves into a clinical-stage company. Notably, in 2017, the Company appointed Dr.
Samarth Kulkarni as Chief Executive Officer and added key executives in positions including Head of Research and Development, Chief Financial Officer and General Counsel, each of whom bring an extensive track-record of success and leadership in biopharma. - Completed an offering of 5,750,000 common shares. The offering, including full exercise of the overallotment at
$22.75 per share, resulted in net proceeds to the Company of greater than$120 million .
Fourth Quarter and Full Year 2017 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities as of
December 31, 2017 were$239.8 million , compared to$315.5 million as ofDecember 31, 2016 , a decrease of$75.7 million , which was primarily driven by expenditures on research and development. Additionally, onJanuary 5, 2018 , CRISPR announced a follow-on offering that raised$122.6 million of net proceeds that are not reflected in year-end cash. Combined with the 2017 year-end cash balance,CRISPR Therapeutics enters 2018 with more than$360 million available to advance its portfolio. - Revenue: Total collaboration revenue was
$32.3 million for the fourth quarter of 2017 compared to$2.3 million for fourth quarter of 2016, and$41.0 million for the year endedDecember 31, 2017 , compared to$5.2 million for the year endedDecember 31, 2016 . The increase in annual revenue is primarily attributable to deferred revenue recognized in conjunction with the execution of the Company’s collaboration agreement with Vertex. - R&D Expenses: R&D expenses were
$20.0 million for the fourth quarter of 2017 compared to$15.6 million for the fourth quarter of 2016, and$69.8 million for the year endedDecember 31, 2017 compared to$42.2 million for the year endedDecember 31, 2016 . The increase in expense was driven by greater investment in CRISPR’s lead hemoglobinopathies program partnered with Vertex, in addition to accelerating the Company’s wholly owned immuno-oncology and in vivo programs. - G&A Expenses: General and administrative expenses were
$11.3 million for the fourth quarter of 2017 compared to$12.1 million for the fourth quarter of 2016, and$35.8 million for the year endedDecember 31, 2017 compared to$31.1 million for the year endedDecember 31, 2016 . The increase in general and administrative expenses for the year was driven by increases in employee-related costs associated with our growing organization. - Net Income/Loss: Net income was
$0.1 million for the fourth quarter of 2017 compared to$17.1 million for the fourth quarter of 2016, and net loss was$68.3 million for the year endedDecember 31, 2017 compared to$23.2 million for the year endedDecember 31, 2016 .
About
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 (
CRISPR Therapeutics AG | |||||||||||||||
Condensed Consolidated Statements of Operations | |||||||||||||||
(Unaudited, In thousands except share data and per share data) | |||||||||||||||
Three Months Ended December 31, |
Year Ended December 31, | ||||||||||||||
2017 | 2016 | 2017 | 2016 | ||||||||||||
Collaboration revenue | $ | 32,325 | $ | 2,344 | $ | 40,997 | $ | 5,164 | |||||||
Operating expenses: | |||||||||||||||
Research and development | 20,030 | 15,572 | 69,800 | 42,238 | |||||||||||
General and administrative | 11,323 | 12,082 | 35,845 | 31,056 | |||||||||||
Total operating expenses | 31,353 | 27,654 | 105,645 | 73,294 | |||||||||||
Income (Loss) from operations | 972 | (25,310 | ) | (64,648 | ) | (68,130 | ) | ||||||||
Total other (expense) income, net | (413 | ) | 42,808 | (1,960 | ) | 45,412 | |||||||||
Net income(loss) before income taxes | 559 | 17,498 | (66,608 | ) | (22,718 | ) | |||||||||
Provision for income taxes | (419 | ) | (400 | ) | (1,749 | ) | (484 | ) | |||||||
Net income (Loss) | 140 | 17,098 | (68,357 | ) | (23,202 | ) | |||||||||
Foreign currency translation adjustment | 40 | (18 | ) | ||||||||||||
Comprehensive Income (Loss) | $ | 140 | $ | 17,098 | $ | (68,317 | ) | $ | (23,220 | ) | |||||
Reconciliation of net loss to net loss attributable to common shareholders: | |||||||||||||||
Net income (Loss) | $ | 140 | $ | 17,098 | $ | (68,357 | ) | $ | (23,202 | ) | |||||
Loss attributable to noncontrolling interest | - | 1 | - | 25 | |||||||||||
Dividends attributable to Redeemable Convertible Preferred Stock | - | (763 | ) | - | - | ||||||||||
Net income allocated to participating securities | - | (2,299 | ) | - | - | ||||||||||
Net Income (loss) attributable to common shareholders | $ | 140 | $ | 14,037 | $ | (68,357 | ) | $ | (23,177 | ) | |||||
Net income (loss) per share attributable to common shareholders - basic |
$ | - | $ | 0.43 | $ | (1.71 | ) | $ | (1.89 | ) | |||||
Net income (loss) loss per share attributable to common shareholders - diluted |
$ | - | $ | 0.40 | $ | (1.71 | ) | $ | (1.89 | ) | |||||
Weighted-average common shares outstanding used in calculating net income (loss) per share attributable to common shareholders – basic |
40,509,897 | 32,987,335 | 40,057,365 | 12,257,483 | |||||||||||
Weighted-average common shares outstanding used in calculating net income (loss) per share attributable to common shareholders – diluted |
41,635,843 | 34,989,218 | 40,057,365 | 12,257,483 |
CRISPR Therapeutics AG | ||||||
Condensed Consolidated Balance Sheets Data | ||||||
(Unaudited, in thousands) | ||||||
As of | ||||||
December 31, 2017 | December 31, 2016 | |||||
Cash | $ | 239,758 | $ | 315,520 | ||
Total assets | 271,346 | 344,962 | ||||
Total liabilities | 83,514 | 112,116 | ||||
Total shareholders' equity | 187,832 | 232,846 |
Media Contacts:
WCG on behalf of CRISPR
1 347-658-8290
jpaganelli@wcgworld.com
Investor Contact:
617.307.7227
chris.erdman@crisprtx.com
Westwicke Partners on behalf of CRISPR
339-970-2843
chris.brinzey@westwicke.com