Cellectis S.A.

Cellectis S.A. is a clinical-stage biotechnology company that leverages its proprietary gene-editing technologies to develop allogeneic Chimeric Antigen Receptor T-cells (UCART) product candidates for immuno-oncology and gene therapy product candidates for other therapeutic indications [Item 4, Business Overview]. The company's core business model revolves around the development of "off-the-shelf" UCART products derived from healthy donors, aiming to provide cost-effective and globally distributable cancer treatments [Item 4, Business Overview]. Revenue generation is primarily through licensing agreements and potential future product sales, with a mix of upfront payments, milestone payments, and tiered royalties from partners [Item 4, Business Overview]. Primary customer segments, once products are approved, would be cancer patients, with a focus on hematologic cancers such as Acute Lymphoblastic Leukemia (ALL) and Non-Hodgkin Lymphoma (NHL) [Item 4, Business Overview].

The company's product and service lines are centered on its UCART pipeline and gene therapy programs. Key product candidates include lasme-cel (previously UCART22) for B-cell Acute Lymphoblastic Leukemia (B-ALL) and eti-cel (previously UCART20x22) for B-cell Non-Hodgkin's Lymphoma (B-NHL) [Item 4, UCART Pipeline]. Lasme-cel is designed to target CD22-expressing hematologic malignancies, with features like TCR inactivation to avoid GvHD and CD52 gene suppression for compatibility with alemtuzumab lymphodepletion [Item 4, Lasme-cel for B-ALL]. Eti-cel targets both CD20 and CD22 to potentially prevent tumor escape and also features TCR and CD52 gene suppression [Item 4, Eti-cel for NHL]. The company also develops self-owned UCART programs for solid tumors, currently in the preclinical phase, including UCARTFAP and UCARTMUC1 [Item 4, Self-owned UCART programs for solid tumors]. Additionally, Cellectis has licensing agreements with AstraZeneca, Allogene, and Servier for various product candidates, including cema-cel (formerly ALLO-501A) for NHL and CLL, and ALLO-316 for Renal Cell Carcinoma (RCC) [Item 4, Our Candidate Products; Item 4, Programs Under Licensing Agreements].

For the fiscal year ended December 31, 2025, Cellectis reported a net loss of $67.6 million from continuing operations [Item 3, Risks Related to Our Therapeutics Business]. Research and development expenses were $93.5 million [Item 3, Risks Related to Our Therapeutics Business]. As of December 31, 2025, the company had cash and cash equivalents of $61.5 million and fixed-term deposits (classified as current financial assets) of $144.8 million [Item 3, Risks Related to Our Therapeutics Business]. The accumulated deficit attributable to the shareholders of Cellectis was $334.1 million as of December 31, 2025 [Item 3, Risks Related to Our Therapeutics Business]. Total capital expenditures related to tangible and intangible assets for the year ended December 31, 2025, amounted to $3.5 million [Item 4, History and Development of the Company].

The company has seen significant operational developments, including the initiation of the pivotal Phase 2 for lasme-cel in the fourth quarter of 2025 [Item 4, Lasme-cel for B-ALL]. Preliminary results for eti-cel, presented in December 2025, showed an overall response rate (ORR) of 88% and a complete response (CR) rate of 63% (n=8) at the current dose level [Item 4, Eti-cel for NHL]. A joint research and collaboration agreement with AstraZeneca was entered into on November 1, 2023, which included an upfront payment of $25 million from AZ Ireland [Item 4, Joint Research and Collaboration Agreement]. AstraZeneca Holdings B.V. also made an initial equity investment of $80 million for 16,000,000 ordinary shares at $5.00 per share on November 1, 2023, and a subsequent investment of $140 million in May 2024 for 10,000,000 Class A Preferred Shares and 18,000,000 Class B Preferred Shares, also at $5.00 per share [Item 4, Initial Investment Agreement; Item 4, Subsequent Investment Agreement]. In December 2025, an arbitral tribunal ordered the partial termination of the Servier License Agreement with respect to UCART19 V1 [Item 4, UCART19 for ALL (discontinued)].

Cellectis anticipates that its cash and cash equivalents of $61.5 million, combined with fixed-term deposits of $144.8 million as of December 31, 2025, will be sufficient to fund its operations into the second half of 2027, covering at least twelve months following the consolidated financial statements' publication [Item 3, Risks Related to Our Therapeutics Business]. The company expects to continue incurring significant expenses and operating losses for the foreseeable future, with expenditures projected to increase as it conducts clinical studies, prepares regulatory filings for new product candidates, advances preclinical testing, and invests in deploying and scaling its manufacturing capabilities [Item 3, Risks Related to Our Therapeutics Business].

A major growth area for Cellectis is the advancement of its self-owned allogeneic UCART portfolio of product candidates up to Biologics License Application (BLA) and subsequent commercialization [Item 4, Our Strategy]. The pivotal Phase 2 of the BALLI-01 Study for lasme-cel was initiated in the fourth quarter of 2025, targeting transplant ineligible patients with relapsed/refractory B-ALL in the third line or beyond [Item 4, Lasme-cel for B-ALL]. Clinical data from Phase 1 of BALLI-01 showed an overall response rate of 68% with lasme-cel Process 2 (n=22), 83% at the Recommended Phase 2 Dose (n=12), and 100% in the target Phase 2 population (n=9), with 78% of patients in the target Phase 2 population proceeding to transplant [Item 4, Clinical Findings]. Eti-cel, targeting CD20 and CD22 for relapsed or refractory B-cell Non-Hodgkin's Lymphoma, demonstrated an encouraging overall response rate of 88% and a complete response rate of 63% (n=8) at the current dose level in preliminary results from the NATHALI-01 Study in December 2025 [Item 4, Eti-cel for NHL]. The company also plans to continue research and development of its gene therapy programs and self-owned UCART programs for solid tumors, which are currently in the preclinical phase [Item 4, Our Strategy; Item 4, Self-owned UCART programs for solid tumors].

Another significant growth vector is the collaboration with AstraZeneca under the Joint Research and Collaboration Agreement (AZ JRCA), which commenced on November 1, 2023 [Item 4, Joint Research and Collaboration Agreement]. This agreement involves the development of up to 10 novel cell and gene therapy candidate products, with AZ Ireland holding an exclusive option for a worldwide exclusive license on these candidates [Item 4, Joint Research and Collaboration Agreement]. Cellectis is eligible to receive an option exercise fee and development, regulatory, and sales-related milestone payments ranging from approximately $80 million up to $253 million per candidate product, plus tiered royalties from mid-single to low-double digits based on net sales of Licensed Products [Item 4, Joint Research and Collaboration Agreement]. The initial collaboration term is five years, during which Cellectis will be reimbursed for its budgeted research costs [Item 4, Joint Research and Collaboration Agreement].

Operationally, Cellectis aims to continue utilizing its self-owned manufacturing network to produce commercial-grade UCART products for clinical use, as well as critical raw and starting materials [Item 4, Our Strategy]. The Raleigh, North Carolina facility, approximately 80,000 sq. ft., is dedicated to clinical and commercial UCART product production, while the Paris, France facility, approximately 14,000 sq. ft., focuses on critical raw and starting material production [Item 4, Towards manufacturing autonomy with two state-of-the-art plants]. The company expects its capital expenditures to increase in absolute terms in the near term, primarily in France and the United States, to advance research and development programs and grow operations, with 2026 capital expenditure anticipated to be financed from existing cash and cash equivalents [Item 4, History and Development of the Company].

Planned capital allocation includes continued investment in research and development, with expenses for the year ended December 31, 2025, at $93.5 million [Item 3, Risks Related to Our Therapeutics Business]. Capital expenditures for tangible and intangible assets were $3.5 million in 2025 [Item 4, History and Development of the Company]. The company expects to finance its 2026 capital expenditure from its cash and cash equivalents on hand [Item 4, History and Development of the Company]. Cellectis has not declared or paid any cash dividends and does not intend to do so for the foreseeable future, planning to invest future earnings into growth [Item 3, French law may limit the amount of dividends we are able to distribute, and we do not currently intend to pay dividends].

Management has explicitly flagged structural headwinds and execution risks, including the inherent difficulties and high attrition rate in biopharmaceutical product development, the unpredictability of clinical trial outcomes, and the potential for unforeseen expenses, complications, or delays [Item 3, Risks Related to Our Therapeutics Business]. The novel nature of gene-editing technology and CAR T-cell immunotherapy also presents challenges, such as the risk of unintended DNA changes like oncogenesis, and the rapid development in the field could render current technologies obsolete [Item 3, Risks Related to the Discovery, Development and Commercialization of Our Therapeutic Product Candidates]. The company also faces substantial competition from other biotechnology and pharmaceutical companies, many with significantly greater resources [Item 3, Risks Related to Our Therapeutics Business]. The discontinuation of Servier's involvement in the development of CD19 Products may delay their development and commercialization [Item 3, Servier’s discontinuation of its involvement in the development of CD19 Products may have adverse consequences].

Geographic, regulatory, and macro factors identified as constraints include the rigorous, complex, and evolving regulatory framework for novel gene-editing and cell therapy products, which can lead to significant compliance costs, lengthy approval processes, and unpredictable outcomes [Item 3, Risks Related to Regulatory Approvals for Our Product Candidates]. Unstable market, economic, and geopolitical conditions, including inflation and interest-rate volatility, could adversely affect the business, making financing more difficult or costly, and potentially disrupting supply chains [Item 3, Unstable market, economic and geo-political conditions may have serious adverse consequences on our business, financial condition and stock price]. Operating in global markets subjects the company to additional risks such as differing regulatory, tax, accounting, and legal requirements, import/export licensing, and reduced intellectual property protection in some foreign countries [Item 3, Our future profitability, if any, depends, in part, on our ability to penetrate global markets, where we would be subject to additional regulatory burdens and other risks and uncertainties].

Cellectis faces material risks across several domains. Macroeconomic risks include unstable market, economic, and geopolitical conditions, which could lead to diminished liquidity, credit availability, elevated inflationary pressures, and interest-rate volatility, making debt or equity financing more difficult, costly, and dilutive [Item 3, Unstable market, economic and geo-political conditions may have serious adverse consequences on our business, financial condition and stock price]. Competitive risks are substantial, with many competitors possessing significantly greater financial, technical, and other resources, potentially developing more effective, safer, or less costly alternatives, or securing patent protection that could impede Cellectis' development [Item 3, We face substantial competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively]. Regulatory risks are high due to the rigorous, complex, and evolving regulatory framework for novel gene-editing and cell therapy products, which can result in significant compliance costs, unpredictable development and approval timelines, and potential delays or discontinuation of product development [Item 3, The regulatory landscape that governs our product candidates is uncertain; regulations relating to more established gene therapy and cell therapy products are still developing, and changes in regulatory requirements could result in delays or discontinuation of development of our product candidates or unexpected costs in obtaining regulatory approval]. Geopolitical risks, such as military and regional conflicts, could materially adversely affect global trade, currency exchange rates, inflation, and regional economies, increasing costs, disrupting supply chains, and impairing access to capital [Item 3, Unstable market, economic and geo-political conditions may have serious adverse consequences on our business, financial condition and stock price]. Operational risks include the inherent difficulties in managing development and expansion, such as recruiting and retaining qualified personnel, and the potential for product liability claims inherent in biopharmaceutical development, which could divert resources and result in substantial liabilities [Item 3, We will need to develop and expand our company, and we may encounter difficulties in managing this development and expansion, which could disrupt our operations; Item 3, Product liability lawsuits could divert our resources, result in substantial liabilities and reduce the commercial potential of our product candidates]. Furthermore, the company's internal computer systems or those of third-party contractors are vulnerable to security breaches, which could disrupt product development programs or lead to loss of personal data [Item 3, Our internal computer systems, or those of our third-party contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our product development programs or loss of personal data]. A specific quantified exposure is the lease guaranty for Cibus's Minnesota offices, representing a potential obligation of $19.9 million over the remaining 12-year lease period, with uncertainty regarding Cibus's ability to make payments or indemnify Cellectis [Item 3, Risks Related to Calyxt, Inc.].

Management's message to shareholders emphasizes leveraging the transformative potential of its unique gene-editing technologies and expertise through its cell therapy platform. The company's strategic priorities for the period ahead are to advance its self-owned allogeneic UCART portfolio of product candidates up to Biologics License Application (BLA) and commercialization, continue to utilize its self-owned manufacturing network for clinical and commercial UCART products and critical raw materials, structure a commercial launch plan for its self-owned product candidates, and continue the research and development of its gene therapy programs [Item 4, Our Strategy]. Management believes its cash and cash equivalents of $61.5 million and fixed-term deposits of $144.8 million as of December 31, 2025, will be sufficient to fund operations into the second half of 2027 [Item 3, Risks Related to Our Therapeutics Business]. They acknowledge the highly speculative nature of biopharmaceutical product development, the significant operating losses incurred since inception, and the expectation of continued substantial net losses for the foreseeable future, with research and development expenses for the year ended December 31, 2025, at $93.5 million [Item 3, Risks Related to Our Therapeutics Business]. Management also highlights the substantial competition and the need for additional funding, which may not always be available on acceptable terms [Item 3, We face substantial competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively; Item 3, We may need to raise additional funding, which may not be available on acceptable terms or at all].