CRISPR Therapeutics(CRSP) - 2025 Q2 - Quarterly Results

CRISPR Therapeutics Business Update and Q2 2025 Financial Results Business Highlights and Outlook The company reports strong commercial and clinical program momentum, with key data updates expected and a solid cash position of approximately $1.7 billion - The company is experiencing momentum across its commercial and clinical programs, with several key milestones anticipated in the second half of 2025[2] - Maintains a strong balance sheet with approximately $1.7 billion in cash, cash equivalents, and marketable securities as of June 30, 2025[1] Hemoglobinopathies and CASGEVY® The commercial launch of CASGEVY® is progressing with 75 treatment centers activated globally and reimbursement agreements secured in 10 countries - Achieved the target of activating 75 authorized treatment centers (ATCs) globally for CASGEVY®[3] - As of June 30, approximately 115 patients have completed cell collection, and 29 patients have received CASGEVY® infusions, with 16 of those occurring in Q2[3] - Vertex has secured reimbursement agreements for eligible patients in 10 countries, including recent additions of Northern Ireland, Scotland, and Denmark[6] - Advancing next-generation approaches, including an anti-CD117 ADC and an in vivo editing platform, to expand the addressable patient population[6] Immuno-Oncology and Autoimmune Disease Programs The company is advancing its next-generation CAR T candidates, with CTX112™ receiving RMAT designation and broad data updates expected in H2 2025 - CTX112, targeting CD19, received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for relapsed or refractory follicular lymphoma and marginal zone lymphoma[6] - A Phase 1 trial of CTX112 is ongoing for autoimmune indications, including systemic lupus erythematosus (SLE)[6] - Broad updates for CTX112 in oncology and autoimmune disease are expected in H2 2025, with updates for the CTX131 program also expected in 2025[1][6] In Vivo Liver Editing Programs The in vivo pipeline shows progress with CTX310™ demonstrating significant reductions in triglycerides and LDL, with a full data presentation expected in H2 2025 CTX310™ Preliminary Phase 1 Data Highlights | Metric | Peak Reduction | | :--- | :--- | | Triglycerides (TG) | up to 82% | | Low-density lipoprotein (LDL) | up to 86% | - The company anticipates presenting the complete Phase 1 data for CTX310 at a medical meeting in the second half of 2025[1][10] - A clinical trial for CTX320™, targeting the LPA gene, is ongoing, with an update expected in the first half of 2026[1][10] SRSD107 and Sirius Therapeutics Collaboration A strategic collaboration with Sirius Therapeutics was initiated to co-develop siRNA therapies, with SRSD107 receiving EMA authorization for a Phase 2 trial - Entered a strategic collaboration with Sirius Therapeutics to jointly develop and commercialize siRNA therapies, beginning with SRSD107[10] - Received European Medicines Agency (EMA) authorization to initiate a Phase 2 clinical trial of SRSD107 for thromboembolic disorders[1][10] Regenerative Medicine Programs The company is advancing its regenerative medicine programs for Type 1 Diabetes, with an update on next-generation therapies expected in 2025 - Developing next-generation programs for Type 1 Diabetes (T1D) using iPSC-derived, allogeneic, gene-edited beta islet cell precursors to enable insulin independence without immunosuppression[9] - An update on the regenerative medicine programs is expected in 2025[9] Second Quarter 2025 Financial Results The company reported a net loss of $208.5 million for Q2 2025, driven by a one-time R&D expense, while maintaining a strong cash position of $1.72 billion Financial Performance Summary The company's cash position decreased to $1.72 billion, while net loss increased to $208.5 million due to a $96.3 million in-process R&D expense Q2 2025 Financial Highlights (vs. Q2 2024) | Metric | Q2 2025 | Q2 2024 | Change Driver | | :--- | :--- | :--- | :--- | | Cash Position (June 30) | $1,721.2M | N/A | Decrease from Dec 31, 2024 ($1,903.8M) due to operating expenses and Sirius payment | | R&D Expenses | $69.9M | $80.2M | Decrease in employee-related expenses | | Acquired In-Process R&D | $96.3M | $0 | Costs incurred from Sirius Agreement | | G&A Expenses | $18.9M | $19.5M | Remained relatively stable | | Collaboration Expense, net | $45.2M | $52.1M | Decrease due to higher CASGEVY revenue and lower program expenses | | Net Loss | ($208.5M) | ($126.4M) | Increased primarily due to acquired in-process R&D expense | Condensed Consolidated Statements of Operations For Q2 2025, the company reported total revenues of $0.89 million and a net loss of $208.5 million, or ($2.40) per share Condensed Consolidated Statements of Operations (Unaudited, In thousands) | | Three Months Ended June 30, | Six Months Ended June 30, | | :--- | :--- | :--- | | | 2025 | 2024 | 2025 | 2024 | | Total revenue | $892 | $517 | $1,757 | $1,021 | | Research and development | $69,894 | $80,165 | $142,378 | $156,338 | | Acquired in-process R&D | $96,253 | $— | $96,253 | $— | | General and administrative | $18,916 | $19,481 | $38,212 | $37,434 | | Collaboration expense, net | $45,153 | $52,131 | $102,662 | $99,097 | | Total operating expenses | $230,216 | $151,777 | $379,505 | $292,869 | | Loss from operations | ($229,324) | ($151,260) | ($377,748) | ($291,848) | | Net loss | ($208,549) | ($126,408) | ($344,545) | ($242,999) | | Net loss per common share — basic | ($2.40) | ($1.49) | ($3.98) | ($2.92) | Condensed Consolidated Balance Sheets Data As of June 30, 2025, the company held $1.72 billion in cash and marketable securities, with total assets of $2.03 billion Condensed Consolidated Balance Sheets Data (Unaudited, in thousands) | | As of June 30, 2025 | As of December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $193,618 | $298,257 | | Marketable securities | $1,527,619 | $1,605,569 | | Working capital | $1,629,304 | $1,849,350 | | Total assets | $2,029,711 | $2,242,034 | | Total shareholders' equity | $1,711,125 | $1,932,080 |