UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
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Item 2.02 Results of Operations and Financial Condition.
On February 15, 2022, CRISPR Therapeutics AG announced its financial results for the quarter ended and year ended December 31, 2021 and other business highlights. The full text of the press release issued in connection with the announcement is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information in this Form 8-K (including Exhibit 99.1) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits:
The following exhibits shall be deemed to be furnished, and not filed:
Exhibit |
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Description |
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99.1 |
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Press Release by CRISPR Therapeutics AG, dated February 15, 2022 |
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104 |
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Cover Page Interactive Data File (embedded within the Inline XBRL document) |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
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CRISPR Therapeutics AG |
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Date: |
February 15, 2022 |
By: |
/s/ Samarth Kulkarni |
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Samarth Kulkarni, Ph.D. |
Exhibit 99.1
CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2021 Financial Results
- More than 70 patients have been dosed with CTX001™ across CLIMB-Thal-111 and CLIMB-SCD-121 to date; enrollment complete and regulatory submissions planned for late 2022-
-Initiated and began dosing patients in the pivotal trial of CTX110™, targeting CD19+ B-cell malignancies; additional data expected to report in 2022-
-Top-line data expected to report in 1H2022 for ongoing CTX120™and CTX130™ clinical trials-
-First patient dosed in Phase 1 clinical trial of VCTX210 for the treatment of type 1 diabetes (T1D)-
ZUG, Switzerland and CAMBRIDGE, Mass., February 15, 2022 – 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, 2021.
“2021 was a productive year for CRISPR Therapeutics on all fronts. We and our partner Vertex completed enrollment in the CTX001 clinical trials and are on track for planned regulatory submissions in late 2022,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “Concurrently, we advanced our wholly-owned immuno-oncology pipeline, and have initiated dosing of patients in our CTX110 pivotal trial. Further, we and our partner ViaCyte, recently announced the dosing of the first patient in the Phase 1 clinical trial of VCTX210 for the treatment of type 1 diabetes. This investigational therapy has the potential to be a transformative treatment for patients with all insulin-requiring forms of diabetes. Looking ahead, we expect a productive 2022 with continued progress across our portfolio as we enter a new phase of growth for our Company. Given the breadth of our portfolio and our strong capital position, we remain well positioned to advance our pipeline and platform to develop transformative medicines for patients suffering from serious diseases.”
Recent Highlights and Outlook
Fourth Quarter and Full Year 2021 Financial Results
About CTX001
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are edited 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, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate or eliminate transfusion requirements for patients with TDT and reduce or eliminate painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published as a Brief Report in The New England Journal of Medicine in January of 2021.
Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.
Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.
About the CRISPR-Vertex Collaboration
Vertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics.
About CLIMB-111
The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.
About CLIMB-121
The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.
About CLIMB-131
This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.
About CTX110
CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON trial.
About CARBON
The ongoing Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies.
About CTX120
CTX120, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen, or BCMA. CTX120 is being investigated in an ongoing Phase 1 single-arm, multi-center, open-label clinical trial designed to assess the safety and efficacy of several dose levels of CTX120 for the treatment of relapsed or refractory multiple myeloma. CTX120 has been granted Orphan Drug designation from the FDA.
About CTX130
CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively.
About VCTX210
VCTX210 is an investigational, allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of T1D. VCTX210 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and ViaCyte, Inc.
About CRISPR Therapeutics
CRISPR 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, 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 Therapeutics Forward-Looking Statement
This 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 made by Dr. Kulkarni in this press release, as well as 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, CTX130, and VCTX210; (ii) the status of clinical trials (including, without limitation, expectations regarding the data that is being presented, the expected timing of data releases and development, as well as completion of clinical trials) and development timelines for CRISPR Therapeutics’ product candidates; (iii) 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, including expectations regarding the CTX001 and CTX110 data that was previously presented; (iv) its in vivo programs; (v) the actual or potential benefits of regulatory designations; (vi) 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; (vii) the sufficiency of CRISPR Therapeutics’ cash resources; (viii) the expected benefits of CRISPR Therapeutics’ collaborations; and (ix) 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 and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results 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 (including, without limitation, availability and timing of results and whether such results will be predictive of future results of the future trials); uncertainties about regulatory approvals to conduct trials or to market products; 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; 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, quarterly report on Form 10-Q 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® word mark and design logo, CTX001™, CTX110™, CTX120™, and CTX130™ are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.
Investor Contact:
Susan Kim
+1-617-307-7503
susan.kim@crisprtx.com
Media Contact:
Rachel Eides
+1-617-315-4493
rachel.eides@crisprtx.com
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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2021 |
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2020 |
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2021 |
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2020 |
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Revenue: |
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Collaboration revenue |
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$ |
12,348 |
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$ |
194 |
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$ |
913,081 |
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$ |
543 |
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Grant revenue |
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551 |
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176 |
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1,882 |
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176 |
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Total revenue |
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$ |
12,899 |
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$ |
370 |
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$ |
914,963 |
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$ |
719 |
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Operating expenses: |
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Research and development |
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134,470 |
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82,365 |
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438,633 |
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266,946 |
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General and administrative |
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24,127 |
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25,766 |
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102,802 |
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88,208 |
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Total operating expenses |
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158,597 |
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108,131 |
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541,435 |
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355,154 |
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(Loss) income from operations |
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(145,698 |
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(107,761 |
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373,528 |
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(354,435 |
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Total other income, net |
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2,197 |
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575 |
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6,003 |
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6,379 |
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Net (loss) income before income taxes |
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(143,501 |
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(107,186 |
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379,531 |
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(348,056 |
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Benefit (provision) for income taxes |
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2,253 |
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147 |
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(1,870 |
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(809 |
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Net (loss) income |
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(141,248 |
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(107,039 |
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377,661 |
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(348,865 |
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Foreign currency translation adjustment |
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3 |
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37 |
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(11 |
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40 |
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Unrealized loss on marketable securities |
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(4,300 |
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14 |
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(4,973 |
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(130 |
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Comprehensive (loss) income |
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$ |
(145,545 |
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$ |
(106,988 |
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$ |
372,677 |
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$ |
(348,955 |
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Net (loss) income per common share — basic |
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$ |
(1.84 |
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$ |
1.50 |
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$ |
4.97 |
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$ |
(5.29 |
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Basic weighted-average common shares outstanding |
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76,649,727 |
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71,282,096 |
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75,948,686 |
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65,949,672 |
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Net (loss) income per common share — diluted |
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$ |
(1.84 |
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$ |
1.50 |
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$ |
4.70 |
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$ |
(5.29 |
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Diluted weighted-average common shares outstanding |
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76,649,727 |
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71,282,096 |
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80,393,496 |
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65,949,672 |
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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, 2021 |
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December 31, 2020 |
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Cash |
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$ |
923,031 |
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$ |
1,168,620 |
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Marketable securities |
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1,456,098 |
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521,713 |
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Working capital |
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2,297,630 |
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1,622,361 |
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Total assets |
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2,751,877 |
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1,827,966 |
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Total shareholders' equity |
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2,399,460 |
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1,664,234 |
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