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 November 5, 2024, CRISPR Therapeutics AG announced its financial results for the quarter ended September 30, 2024 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 No. |
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Description |
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99.1 |
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Press Release by CRISPR Therapeutics AG, dated November 5, 2024 |
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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: |
November 5, 2024 |
By: |
/s/ Samarth Kulkarni |
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Samarth Kulkarni, Ph.D. Chief Executive Officer |
Exhibit 99.1
CRISPR Therapeutics Provides Business Update and Reports Third Quarter 2024 Financial Results
-CASGEVY™ approved for the treatment of patients 12 years of age and older with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in Switzerland and Canada-
-45 authorized treatment centers (ATCs) activated globally for CASGEVY and approximately 40 patients have had cells collected across all regions as of mid-October-
-Two clinical trials are ongoing for next generation CAR T product candidate, CTX112™ targeting CD19, in B-cell malignancies and systemic lupus erythematosus-
-Two clinical trials are ongoing for next generation CAR T product candidate, CTX131™, targeting CD70, in solid tumors and hematological malignancies-
-Company plans to provide an update on the Phase 1 dose escalation study of CTX112 in B cell malignancies at the American Society of Hematology (ASH) 2024 Annual Meeting-
-Clinical trials ongoing for in vivo gene editing product candidates, CTX310™ and CTX320™ targeting ANGPTL3 and LPA, respectively-
-Clinical trial ongoing for CTX211™, an allogeneic, hypoimmune, gene-edited, stem cell derived product candidate for the treatment of Type 1 Diabetes (T1D)-
-Strong balance sheet with approximately $1.9 billion in cash, cash equivalents, and marketable securities as of September 30, 2024-
ZUG, Switzerland and BOSTON, November 5, 2024 -- 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, 2024.
“We continue to make significant progress across our pipeline of in vivo and ex vivo CRISPR-based therapies,” said Samarth Kulkarni, Ph.D., Chief Executive Officer and Chairman of CRISPR Therapeutics. “In addition to the continued momentum of CASGEVY’s launch, we are pleased to share that CASGEVY has received regulatory approvals for the treatment of patients 12 years of age and older with SCD or TDT in Switzerland and Canada. In parallel, we remain focused on advancing our portfolio of clinical trials across oncology, autoimmune, diabetes and cardiovascular indications in a capital efficient manner. We look forward to a number of important data catalysts over the next 9-12 months as we advance our portfolio.”
Recent Highlights and Outlook
About CASGEVY (exagamglogene autotemcel [exa-cel])
CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for certain indications in multiple jurisdictions for eligible patients.
About the CRISPR Therapeutics-Vertex Collaboration
CRISPR Therapeutics and Vertex 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. CASGEVY represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing, and commercialization of CASGEVY and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics. Vertex is the manufacturer and exclusive license holder of CASGEVY.
About CTX112
CTX112 is being developed for both oncology and autoimmune indications. CTX112 is a next-generation, wholly-owned, allogeneic CAR T product candidate targeting Cluster of Differentiation 19, or CD19, which incorporates edits designed to evade the immune system, enhance CAR T potency and reduce CAR T exhaustion. CTX112 is being investigated in an ongoing clinical trial designed to assess safety and efficacy of the product candidate in adult patients with relapsed or refractory CD19-positive B-cell malignancies who have received at least two prior lines of therapy. In addition, CTX112 is being investigated in an ongoing clinical trial designed to assess safety and efficacy of the product candidate in adult patients with system lupus erythematosus.
About CTX131
CTX131 is being developed for both solid tumors and hematologic malignancies, including T cell lymphomas (TCL). CTX131 is a next-generation, wholly-owned, allogeneic CAR T product candidate targeting Cluster of Differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX131 incorporates edits designed to evade the immune system, prevent fratricide, enhance CAR T potency and reduce CAR T exhaustion. CTX131 is being investigated in ongoing clinical trials designed to assess the safety and efficacy of the product candidate in adult patients with relapsed or refractory solid tumors and hematologic malignancies, including TCL.
About In Vivo Programs
CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) platform for the delivery of CRISPR/Cas9 to the liver. The Company’s in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards LPA, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are targeting validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company’s research and preclinical development candidates include CTX340 and CTX450, targeting angiotensinogen (AGT) for refractory hypertension and 5’-aminolevulinate synthase 1 (ALAS1) for acute hepatic porphyria (AHP), respectively.
About CTX211
CTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of type 1 diabetes (T1D), which incorporates gene edits that aim to make cells hypoimmune and enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose. A Phase 1 clinical trial for CTX211 for the treatment of T1D is ongoing.
About CRISPR Therapeutics
Since its inception over a decade ago, CRISPR Therapeutics has transformed from a research-stage company advancing programs in the field of gene editing, to a company that recently celebrated the historic approval of the first-ever CRISPR-based therapy and has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic in 2018 to investigate the treatment of sickle cell disease or transfusion-dependent beta thalassemia, and beginning in late 2023, CASGEVY (exagamglogene autotemcel [exa-cel]) was approved in some countries to treat eligible patients with either of those conditions. The Nobel Prize-winning CRISPR science has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer and Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. To learn more, visit www.crisprtx.com.
CRISPR THERAPEUTICS® standard character mark and design logo, CTX110®, CTX112™, CTX131™, CTX211™, CTX310™, CTX320™, CTX340™ and CTX450™ are trademarks and registered trademarks of CRISPR Therapeutics AG. The CASGEVY™ word mark and design are trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners.
CRISPR Therapeutics Forward-Looking Statement
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials, potential expansion into new indications and expectations regarding data, safety and efficacy generally; as well as its plans to and the clinical data being presented at the 2024 ASH Annual Meeting; (ii) its strategy, goals, anticipated financial performance and the sufficiency of its cash resources; (iii) regulatory submissions and authorizations, including timelines for and expectations regarding additional regulatory agency decisions; (iv) the expected benefits of its collaborations; and (v) the therapeutic value, development, and commercial potential of gene editing technologies and therapies, including CRISPR/Cas9. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading “Risk Factors” in its 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. 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. We disclaim 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.
This press release discusses CRISPR/Cas9 gene editing investigational therapies and is not intended to convey conclusions about efficacy or safety as to those investigational therapies or uses of such investigational therapies. There is no guarantee that any investigational therapy will successfully complete clinical development or gain approval from applicable regulatory authorities.
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 September 30, |
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Nine Months Ended September 30, |
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2024 |
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2023 |
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2024 |
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2023 |
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Revenue: |
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Collaboration revenue |
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$ |
— |
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$ |
— |
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$ |
— |
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$ |
170,000 |
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Grant revenue |
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602 |
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— |
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1,623 |
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— |
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Total revenue |
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$ |
602 |
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$ |
— |
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$ |
1,623 |
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$ |
170,000 |
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Operating expenses: |
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Research and development |
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82,160 |
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90,698 |
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238,498 |
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292,188 |
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General and administrative |
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17,419 |
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18,291 |
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54,853 |
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59,683 |
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Collaboration expense, net |
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11,153 |
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23,422 |
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110,250 |
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110,250 |
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Total operating expenses |
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110,732 |
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132,411 |
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403,601 |
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462,121 |
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Loss from operations |
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(110,130 |
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(132,411 |
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(401,978 |
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(292,121 |
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Total other income, net |
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25,064 |
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20,671 |
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75,924 |
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51,819 |
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Net loss before income taxes |
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(85,066 |
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(111,740 |
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(326,054 |
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(240,302 |
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Provision for income taxes |
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(876 |
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(412 |
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(2,887 |
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(2,655 |
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Net loss |
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(85,942 |
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(112,152 |
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(328,941 |
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(242,957 |
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Foreign currency translation adjustment |
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76 |
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(49 |
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66 |
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12 |
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Unrealized gain on marketable securities |
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13,368 |
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2,160 |
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8,586 |
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8,838 |
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Comprehensive loss |
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$ |
(72,498 |
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$ |
(110,041 |
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$ |
(320,289 |
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$ |
(234,107 |
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Net loss per common share — basic |
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$ |
(1.01 |
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$ |
(1.41 |
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$ |
(3.92 |
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$ |
(3.07 |
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Basic weighted-average common shares outstanding |
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85,234,926 |
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79,414,098 |
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83,988,063 |
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79,063,415 |
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Net loss per common share — diluted |
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$ |
(1.01 |
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$ |
(1.41 |
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$ |
(3.92 |
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$ |
(3.07 |
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Diluted weighted-average common shares outstanding |
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85,234,926 |
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79,414,098 |
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83,988,063 |
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79,063,415 |
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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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September 30, 2024 |
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December 31, 2023 |
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Cash and cash equivalents |
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$ |
225,670 |
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$ |
389,477 |
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Marketable securities |
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1,709,975 |
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1,304,215 |
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Marketable securities, non-current |
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— |
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1,973 |
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Working capital |
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1,854,081 |
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1,799,287 |
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Total assets |
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2,256,130 |
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2,229,571 |
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Total shareholders' equity |
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1,939,658 |
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1,882,803 |
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