CRISPR Therapeutics AG

-Regulatory submissions complete for exagamglogene autotemcel (exa-cel), formerly known as CTX001, in the U.S. for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD)-

-EU and U.K. submissions validated by European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA); exa-cel has been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA-

-Enrollment and dosing ongoing for CTX110, targeting CD19+ B-cell malignancies, and CTX130, targeting CD70 for the treatment of T cell lymphomas-

-Initiated clinical trials for next generation CAR T candidates, CTX112 targeting CD19+ B-cell malignancies and CTX131, targeting CD70+ solid tumors-

-Enrollment and dosing ongoing in a Phase 1/2 clinical trial of VCTX211 for the treatment of Type 1 Diabetes (T1D)-

-Continues to advance its lead in vivo program, CTX310, targeting angiopoietin-related protein 3 (ANGPTL3) into clinical trials this year-

ZUG, Switzerland and BOSTON, May 08, 2023 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the first quarter ended March 31, 2023.

In the first quarter of 2023, we continued strong momentum across our portfolio. We and our partner Vertex have now completed regulatory submissions for exa-cel in the United States, European Union and United Kingdom, positioning exa-cel to potentially become the first approved CRISPR-based therapy in the world, a remarkable pace of progress considering the discovery of the CRISPR platform took place a little more than a decade ago, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. In parallel, we continue to drive our portfolio programs, including the initiation of clinical trials for our next-generation CAR T candidates, CTX112 and CTX131. In addition, we expect to advance CTX310, our lead in vivo program targeting ANGPTL3, into clinical trials later this year. We are well-positioned to drive towards our mission of bringing transformative and potentially curative therapies to patients in need.

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Recent Highlights and Outlook

FirstQuarter 2023 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $1,889.5 million as of March 31, 2023, compared to $1,868.4 million as of December 31, 2022. The increase in cash of $21.1 million was primarily driven by the upfront payment received from Vertex in connection with a non-exclusive license agreement and a benefit from changes in net-working capital, offset by operating expenses.

Revenue: Total collaboration revenue was $100.0 million for the quarter ended March 31, 2023. Collaboration revenue for the first quarter of 2022 was not material. Collaboration revenue recognized in the first quarter of 2023 was primarily attributable to revenue recognized in connection with the upfront payment from Vertex.

R&D Expenses: R&D expenses were $99.9 million for the first quarter of 2023, compared to $118.2 million for the first quarter of 2022. The decrease in R&D expense was primarily driven by reduced variable external research and manufacturing costs.

G&A Expenses: General and administrative expenses were $22.4 million for the first quarter of 2023, compared to $28.0 million for the first quarter of 2022. The decrease in G&A expense was primarily driven by a decrease in external professional costs.

Collaboration Expense: Collaboration expense, net, was $42.2 million for the first quarter of 2023, compared to $30.6 million for the first quarter of 2022. The increase in collaboration expense, net, was primarily driven by an increase in manufacturing and pre-commercial costs associated with the exa-cel program.

Net Loss: Net loss was $53.1 million for the first quarter of 2023, compared to a net loss of $179.2 million for the first quarter of 2022.

About exagamglogene autotemcel (exa-cel)Exa-cel, formerly known as CTX001, is an investigational, autologous,ex vivoCRISPR/Cas9 gene-edited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent vaso-occlusive crises (VOCs), in which a patients own hematopoietic stem cells are edited to produce 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. The elevation of HbF by exa-cel has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published inThe New England Journal of Medicinein January of 2021.

Based on progress in this program to date, exa-cel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from theFDA for both TDT and SCD. Exa-cel has also been granted Orphan Drug Designation from theEuropean Commission, as well as Priority Medicines (PRIME) designation from theEuropean Medicines Agency(EMA), for both TDT and SCD. In the U.K., exa-cel has been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.

About CLIMB-111 and CLIMB-121The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after exa-cel infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up trial.

About CLIMB-131This is a long-term, open-label trial to evaluate the safety and efficacy of exa-cel in patients who received exa-cel in CLIMB-111, CLIMB-121, CLIMB-141 or CLIMB-151. The trial is designed to follow participants for up to 15 years after exa-cel infusion.

About CLIMB-141 and CLIMB-151The ongoing Phase 3 open-label trials, CLIMB-141 and CLIMB-151, are designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 2 to 11 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now open for enrollment and currently enrolling patients ages 5 to 11 years of age and will plan to extend to patients 2 to less than 5 years of age at a later date. Each trial will enroll approximately 12 patients. Patients will be followed for approximately two years after infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up- trial.

About the CRISPR-Vertex CollaborationCRISPR Therapeuticsand Vertex Pharmaceuticals 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. Exa-cel 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 exa-cel and splits program costs and profits worldwide 60/40 withCRISPR Therapeutics.

About CTX110 and CTX112CTX110, a wholly owned program ofCRISPR 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 clinical trial, which is designed to assess the safety and efficacy of CTX110 in adult patients with relapsed or refractory CD19-positive B-cell malignancies who have received at least two prior lines of therapy. CTX110 has been granted RMAT designation by the FDA. In addition, CTX112, a next-generation allogeneic CAR T cell therapy targeting CD19, is being investigated in a clinical trial. CTX112 includes two additional edits beyond CTX110 that are designed to enhance the potency of the CAR T cells.

About CTX130 and CTX131CTX130, a wholly owned program ofCRISPR 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 investigated for the treatment of relapsed or refractory T-cell hematologic malignancies in the COBALT-LYM trial and for renal cell carcinoma in the COBALT-RCC trial. CTX130 has been granted Orphan Drug designation for the treatment of T cell lymphoma by the FDA and RMAT designation for the treatment of relapsed or refractory Mycosis Fungoides and Szary Syndrome (MF/SS), types of cutaneous T cell lymphoma (CTCL). In addition, CTX131, a next-generation allogeneic CAR T cell therapy targeting CD70, is being assessed for safety and efficacy in a clinical trial investigating a basket of select solid tumors. CTX131 includes two additional edits beyond CTX130 that are designed to enhance the potency of the CAR T cells.

About VCTX210 and VCTX211VCTX210 is an investigational, allogeneic, gene-edited, immune-evasive, stem cell-derived investigational therapy for the treatment of T1D. VCTX210 is being developed under a co-development and co-commercialization agreement betweenCRISPR TherapeuticsandViaCyte, Inc. VCTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of T1D, which incorporates additional gene edits that aim to further enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose.

AboutCRISPR TherapeuticsCRISPR Therapeuticsis 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 Therapeuticshas 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 Therapeuticshas established strategic partnerships with leading companies includingBayer, Vertex PharmaceuticalsandViaCyte, Inc.CRISPR Therapeutics AGis headquartered inZug, Switzerland, with its wholly-ownedU.S.subsidiary,CRISPR Therapeutics, Inc., and R&D operations based inBoston, MassachusettsandSan Francisco, California, and business offices inLondon, United Kingdom. For more information, please visitwww.crisprtx.com.

CRISPR THERAPEUTICS standard character mark and design logo,COBALT, CTX001, CTX110, CTX112, CTX130, CTX131, CTX310, VCTX210, VCTX211 are trademarks and registered trademarks ofCRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

CRISPR Therapeutics 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 made byDr. Kulkarniin this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) its preclinical studies, clinical trials and pipeline products and programs, including, without limitation, the status of such studies and trials, regulatory filings for exa-cel and timing of data releases and regulatory submissions; (ii) potential benefits of exa-cel and the FDAs review of the BLAs and impact of Priority Review on such timing; (iii) benefits of Dr. Prasads employment; (iv) the sufficiency of its cash resources; (v) benefits of its collaborations, including potential milestone payments and royalties on future products under the non-exclusive license agreement; and (vi) 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. AlthoughCRISPR Therapeuticsbelieves 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 preliminary data from any clinical trial not to be indicative of final trial results; the potential that clinical trial results may not be favorable; that one or more of its 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 its product candidates; uncertainties inherent in the initiation and completion of preclinical studies for its product candidates and whether results from such studies will be predictive of future results of future studies or clinical trials; uncertainties about regulatory approvals to conduct trials or to market products; it may not realize the potential benefits of its collaborations; uncertainties regarding the intellectual property protection for its 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 byCRISPR Therapeuticswith theU.S. Securities and Exchange Commission, which are available on theSEC'swebsite atwww.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 Therapeuticsdisclaims 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.

Investor Contact:Susan Kim+1-617-307-7503susan.kim@crisprtx.com

Media Contact:Rachel Eides+1-617-315-4493rachel.eides@crisprtx.com

CRISPR Therapeutics AGCondensed Consolidated Statements of Operations(Unaudited, In thousands except share data and per share data)

Three Months Ended March 31,

2023

2022

Revenue:

Collaboration revenue

$

100,000

$

178

Grant revenue

762

Total revenue

$

100,000

$

940

Operating expenses:

Research and development

99,935

118,245

General and administrative

22,360

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CRISPR Therapeutics Provides Business Update and Reports First Quarter 2023 Financial Results - Yahoo Finance