Financial Data and Key Metrics Changes - As of June 30, 2025, the company's cash, cash equivalents, and marketable securities totaled approximately $122.8 million [23] - Revenues for Q2 2025 were zero, compared to approximately $4 million for Q2 2024, and total revenues for 2025 were zero compared to approximately $8.9 million for 2024 [23][24] - Research and development expenses for Q2 2025 were approximately $20.1 million, up from approximately $11.7 million in Q2 2024, and total R&D expenses for 2025 were approximately $36.3 million compared to approximately $21.8 million for 2024 [25][26] - General and administrative expenses for Q2 2025 were approximately $4 million, compared to approximately $1.8 million in Q2 2024, and total G&A expenses for 2025 were approximately $7 million compared to approximately $3.6 million for 2024 [26] - The net loss for Q2 2025 was approximately $25.9 million, compared to a net loss of approximately $11 million for Q2 2024, and the total net loss for 2025 was approximately $50.3 million compared to approximately $20.8 million for 2024 [26] Business Line Data and Key Metrics Changes - The company is focused on advancing daramycin for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy, which is a significant unmet need in the DMD community [5][6] - The HOPE-three trial is fully enrolled with 104 patients, and the primary efficacy endpoint has been amended to Left Ventricular Ejection Fraction (LVEF) [9][10] Market Data and Key Metrics Changes - The company is actively working with the FDA to clarify the path forward for daramycin following a complete response letter received in July 2025 [6][12] - The company is also involved in Project NextGen, focusing on advancing next-generation vaccines for COVID-19 and other infectious diseases through its Stealth X vaccine [17][18] Company Strategy and Development Direction - The company aims to deliver transformative therapies for rare diseases, with a strong commitment to the DMD community [5] - The strategy includes working constructively with the FDA to define a clear path for daramycin's approval and preparing for potential commercial launch activities [8][16] - The company is also exploring opportunities in vaccine development through its exosome platform, which could lead to partnerships and expansion into other therapeutic areas [20][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strength of their data and the potential for daramycin to stabilize cardiac function in DMD patients [14][29] - The company remains focused on regulatory clarity and is optimistic about the upcoming Type A meeting with the FDA [35][64] - Management emphasized the importance of LVEF as a primary efficacy endpoint and the need for innovative thinking from the FDA regarding rare disease approvals [82][87] Other Important Information - The company has received formal acceptance of all items from its pre-license inspection, validating its quality systems and manufacturing capabilities [14][15] - The company is well-positioned financially, with over $120 million in cash, allowing it to support operations until late 2026 [28] Q&A Session Summary Question: What is the plan for unblinding the HOPE-three trial? - The company is waiting for FDA adjudication on requirements for HOPE-three before proceeding with unblinding [33] Question: What incremental data could be submitted to the FDA? - The company is considering submitting supplemental open-label extension data to support safety and efficacy [41] Question: How has the FDA review team changed? - The company is uncertain about changes in the review team but is looking forward to working with the FDA [111] Question: What are the key takeaways expected from the upcoming Type A meeting? - The company aims to clarify what is required for approval and the primary endpoint for HOPE-three [64] Question: What accounts for the increase in R&D expenses? - The increase is due to ongoing clinical studies and preparations for CMC endeavors [105]

Financial Data and Key Metrics Changes - Total revenue for the second quarter of 2025 was $8,500,000, an 18% decline from $10,400,000 in 2024 [28] - Core revenue increased to $8,200,000, representing an 8% year-over-year growth compared to $7,600,000 in the prior year [28] - Gross margin was 82% in 2025, down from 86% in the previous year [30] Business Line Data and Key Metrics Changes - Instrument revenue grew 22% year-over-year to $2,100,000, up from $1,800,000 in 2024 [28][12] - License revenue remained flat at $2,600,000 compared to the same quarter in 2024 [28] - Processing Assembly (PA) revenue increased to $3,100,000, up from $3,000,000 in 2024 [28] Market Data and Key Metrics Changes - 42% of core revenue was derived from SPL customers in 2025, down from 51% in the prior year [30] - SPL program-related revenue was $300,000 in 2025, significantly lower than $2,900,000 in 2024 [30] Company Strategy and Development Direction - The company is focusing on disciplined investments while aiming for profitability with existing capital [6] - MaxSight is integrating SecurDx, which is seen as a substantial long-term opportunity [6] - The company plans to return to growth in 2026, driven by new platform launches and increasing clinical programs [11] Management's Comments on Operating Environment and Future Outlook - Management acknowledged short-term external headwinds impacting growth expectations for the second half of 2025 [7] - The company is lowering its core revenue guidance for 2025 to flat to down 10% compared to 2024 [8] - Despite current challenges, management remains confident in the long-term value proposition of MaxSight within the cell and gene therapy industry [25] Other Important Information - The company officially delisted from the AIM markets on June 26, 2025, and is now solely listed on NASDAQ [26] - MaxSight expects to end 2025 with approximately $155,000,000 in cash equivalents and investments [34] Q&A Session Summary Question: Impact of manufacturing operations reorganization by the largest customer - Management confirmed that the reorganization is a short-term consolidation and will not impact future licensing revenue [36][37] Question: Opportunities for additional efficiencies within the organization - Management is focused on achieving profitability and is continuously looking for efficiencies across the organization [42][43] Question: Quarterly cadence expectations from Q3 to Q4 - Slight weight towards Q4 is expected, but not materially significant [46][47] Question: Performance expectations for instrumentation sales - Instrument sales are expected to continue performing well, with a focus on lower-priced systems [49][50] Question: SPL partnerships and program prioritization - There is a shift towards allogeneic therapies among partners, which is favorable for the company's systems [56][57] Question: PA revenue benefits and future expectations - PA revenue benefited from a single order, but this is not expected to have a material impact on future guidance [62][63] Question: Impact of macro headwinds on SPL pipeline - No significant impact on the SPL pipeline is observed, with continued confidence in signing new SPLs [65][66]

Financial Data and Key Metrics Changes - As of June 30, 2025, the company's cash, cash equivalents, and fixed-term deposits amounted to $230 million, a decrease of $33.2 million from $264 million as of December 31, 2024, primarily due to cash payments to suppliers and operational expenses [14][14][14] - The company reported a consolidated net loss attributable to shareholders for the six months ended June 30, 2025, with specific figures available in the press release [14] Business Line Data and Key Metrics Changes - The recruitment for the dose escalation component of the Phase I BALL E-one study evaluating UCART22 in relapsed/refractory B cell acute lymphoblastic leukemia has been completed, with a pivotal Phase II study expected to commence in the second half of 2025 [9][10] - The NATALI-one study assessing ETICELL product YOCAR T20 in relapsed/refractory non-Hodgkin's lymphoma is ongoing, with data presentation anticipated in late 2025 [5][11] Market Data and Key Metrics Changes - The company is expanding clinical trial sites in the United States and Europe to accelerate recruitment for the Phase II study, with plans to have sites open by the end of the year [10][11] Company Strategy and Development Direction - Cellectis is focused on advancing research and developing solutions for patients with unmet medical needs, with a strategic collaboration with AstraZeneca on three cell and gene therapy programs [4][5] - The company is preparing for an R&D Day on October 16, 2025, to present the Phase I dataset and late-stage development strategy for UCART22 [3][4] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress of ongoing clinical trials and the potential for their product candidates to lead to significant advancements in treating hard-to-treat cancers and genetic disorders [15] - The company remains optimistic about its financial position, indicating sufficient cash to fund operations into the second half of 2027 [14] Other Important Information - An arbitration decision regarding the licensing agreement with Servier is expected by December 15, 2025, with the company seeking compensation for losses incurred due to the lack of development of licensed products [5][6][14] - The company welcomed a new board member, Andre Muller, and acknowledged the contributions of departing directors [6][7] Q&A Session Summary Question: Contextualization of Servier arbitration scenarios and actions - Management indicated that it is complicated to predict outcomes but expressed hope for a favorable decision that would restore their CD19 products and provide compensation for losses [18] Question: High-level thoughts on pivotal Phase II trial design - Management confirmed productive interactions with both FDA and EMA, with clear agreement on endpoints and no concerns raised about the statistical plan [20][21] Question: Data points expected at R&D Day and FDA dynamics - The R&D Day will present the full Phase I dataset for LASMA cell, including safety, efficacy, and durability data, along with the late-stage development strategy [26][28] Question: Bar for success in relapsed refractory ALL - Management stated that details on endpoints and timing will be shared during the R&D Day, emphasizing the importance of durability of response [35][36] Question: Feedback from regulators on anti-CD52 inclusion - Management does not foresee changes to the pivotal study design based on Allogene's recent decisions, citing established safety profiles and risk-benefit assessments [58] Question: Cash runway and pivotal study completion - The cash runway into H2 2027 includes costs for pivotal studies for both LASMA cell and ETICELL, with prudent assumptions made regarding cash inflows [60] Question: Size of data set for ETICELL - The data set for ETICELL will be smaller than that for LASMA cell, reflecting the different stages of development [62] Question: Differentiation of ETICELL in dual targeting space - Management highlighted that ETICELL targets different antigens compared to competitors, positioning it uniquely in the market [70][71]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company had cash, cash equivalents, short-term investments, and restricted cash of $84.5 million, down from $98.1 million as of December 31, 2024 [19] - Research and development expenses increased to $9.9 million for Q1 2025 from $7.2 million in Q1 2024, primarily due to increased headcount for manufacturing scale-up [20] - General and administrative expenses rose to $9.8 million for Q1 2025 from $7.1 million in Q1 2024, also due to increased headcount related to the ZivaSkin launch [20] - The net loss for Q1 2025 was $12 million, or $0.24 loss per share, compared to a net loss of $31.6 million, or $1.16 loss per share, in Q1 2024 [21] Business Line Data and Key Metrics Changes - The company launched ZivaSkin, the first autologous cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), following FDA approval [5][6] - The first Qualified Treatment Center (QTC) was activated ahead of schedule, with Lurie Children's Hospital of Chicago being the first to offer ZivaSkin [8] - The company anticipates treating the first patient at Lurie in Q3 2025, with a goal of treating 10 to 14 patients by the end of 2025 [10][16] Market Data and Key Metrics Changes - The company has executed outcomes-based agreements with two payer contracting organizations to ensure access to ZivaSkin for patients [18] - The inbound interest from patients and families has been significant, with approximately 30 inquiries received in the first two weeks post-approval [12][13] Company Strategy and Development Direction - The company aims to achieve profitability driven by ZivaSkin revenues by early 2026, supported by the sale of a Priority Review Voucher (PRV) for $155 million [11][19] - The company plans to activate additional QTCs by the end of 2025, focusing on well-recognized institutions with experience in cell and gene therapy [14][15] - The company is also preparing to advance its ophthalmology pipeline, with plans to bring an asset into clinical trials in the second half of 2026 [45][46] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial launch of ZivaSkin and the positive response from patients and physicians [12][16] - The management highlighted the importance of payer agreements to ensure timely access to ZivaSkin for patients [18] - The company is confident in its financial position, stating that the proceeds from the PRV sale will fund operations for over two years [19] Other Important Information - The company presented two late-breaking abstracts at the Society for Investigative Dermatology Annual Meeting, reinforcing ZivaSkin's efficacy and safety profile [10] - The company is actively engaging with commercial payers and state Medicaid programs to expand access to ZivaSkin [19] Q&A Session Summary Question: What does site activation mean? - Site activation includes training staff and surgeons, allowing them to identify patients for ZivaSkin and begin the payer authorization process [25][27] Question: How many RDEB patients are eligible at Lurie Children's Hospital? - There are at least a couple of dozen RDEB patients treated at Lurie, with potential for more referrals from the local community [28][29] Question: What factors affect patient access to treatment? - Commercially insured patients are expected to have faster access compared to Medicaid patients, which may take longer [31][32] Question: What is the expected patient throughput at QTCs? - Initial throughput is expected to be around two patients per month per site, with potential to increase as centers gain experience [41][42] Question: What are the anticipated net proceeds from the PRV sale? - The net proceeds from the PRV sale are expected to be approximately $152 million [58] Question: How will billing and reimbursement work? - Revenue recognition will occur after patient treatment, with discussions already in place with insurance companies to ensure reimbursement [78][80]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company had cash, cash equivalents, short-term investments, and restricted cash of $84.5 million, down from $98.1 million as of December 31, 2024, before accounting for the proceeds from the Priority Review Voucher (PRV) sale [17] - Research and development expenses increased to $9.9 million for Q1 2025 from $7.2 million in Q1 2024, primarily due to increased headcount for manufacturing scale-up [18] - General and administrative expenses rose to $9.8 million for Q1 2025 from $7.1 million in Q1 2024, also due to increased headcount related to the ZivaSkin launch [18] - The net loss for Q1 2025 was $12 million, or $0.24 loss per share, compared to a net loss of $31.6 million, or $1.16 loss per share, in Q1 2024 [19] Business Line Data and Key Metrics Changes - The company launched ZivaSkin, the first autologous cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), following FDA approval [4][5] - The first Qualified Treatment Center (QTC) activated was Lurie Children's Hospital of Chicago, which is expected to treat the first patient in Q3 2025 [6][8] Market Data and Key Metrics Changes - The company has received inquiries from approximately 30 patients and families across the country since ZivaSkin's approval, indicating strong demand [10] - The company is actively engaging with commercial payers and state Medicaid programs to expand access to ZivaSkin [16] Company Strategy and Development Direction - The company aims to treat 10 to 14 patients with ZivaSkin in 2025 and anticipates a robust start to 2026 [13] - The company has entered into an agreement to sell the PRV for $155 million, which will strengthen its balance sheet and fund operations for over two years [9][17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial launch of ZivaSkin and the positive response from patients and physicians [10][13] - The company is focused on ensuring that the value of ZivaSkin is recognized by payers and that patients have timely access to the therapy [16] Other Important Information - The company expects to achieve profitability driven by ZivaSkin revenues in early 2026 [17] - The company is on track to activate additional QTCs by the end of 2025, with ongoing discussions with recognized institutions [12] Q&A Session Summary Question: What does site activation mean? - Site activation means that staff and surgeons are trained and ready to identify patients for ZivaSkin treatment [22][25] Question: How many RDEB patients are eligible at Lurie Children's Hospital? - There are at least a couple of dozen patients treated at Lurie, with potential for more referrals from the local community [26] Question: What factors affect patient access to treatment? - Commercially insured patients are expected to have faster access compared to Medicaid patients, which may take longer [29] Question: What is the expected patient throughput at QTCs? - Initial throughput is expected to be two patients per month per site, with potential to ramp up to four patients per month at some centers [39] Question: What are the anticipated net proceeds from the PRV sale? - The net proceeds from the PRV sale are expected to be about $152 million [54] Question: How will billing and reimbursement work? - Revenue recognition will occur after patient treatment, with discussions already in place with insurance companies to ensure reimbursement [72][75]

Financial Data and Key Metrics Changes - Total revenue for Q1 2025 was $8.5 million, an 11% increase from $7.6 million in Q1 2024 [7] - Gross margin improved to 60%, up from 59% in Q1 2024 [10] - Cash and cash equivalents decreased to $12.4 million from $20.1 million at the end of 2024 [11] Business Line Data and Key Metrics Changes - Biologics and Drug Delivery revenue increased by 9% to $4.7 million, driven by a $1.2 million increase in product revenue [8] - Neurosurgery Navigation revenue surged 70% to $3.3 million, attributed to higher sales of new product offerings [9] - Capital Equipment and Software revenue fell 63% to $500,000 due to fewer new placements of navigation and laser units [10] Market Data and Key Metrics Changes - The company activated two new sites in Q1 2025, which were both capital placements [51] - The introduction of the three point zero navigation software has allowed expansion into operating rooms, enhancing market reach [14] Company Strategy and Development Direction - The company aims to extend its lead in cell and gene therapy through a comprehensive drug delivery ecosystem [4] - Plans to evolve the product portfolio to focus on fast, simple, and predictable workflows to increase hospital efficiency [5] - The company is pursuing global scale by expanding its installed base and generating more access to its ecosystem [5] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential for additional cell and gene therapy platforms to gain approvals in the next two years [16] - The recent partnership with Overland Capital provides the company with the necessary funding to execute its strategy [6] - Management anticipates that operational cash burn will continue to increase but expects to generate meaningful cash flow as new drugs come online [31] Other Important Information - The company has implemented a Pathfinder subscription program to smooth capital revenue recognition over time [22] - The three point zero navigation software has been well received, with positive feedback from surgeons regarding efficiency and accuracy [18] Q&A Session Questions and Answers Question: Are there initiatives that can be prioritized now with access to incremental capital? - Management confirmed that there are opportunities to accelerate hiring and expand preclinical resources in response to partner requests [29] Question: How does the new capital runway relate to achieving sustained breakeven? - Management believes the new debt facility can support the company towards cash breakeven, especially with the flexibility of an interest-only period [31] Question: How is the neuro franchise tracking relative to expectations? - Management stated that the neuro franchise is on plan and expects significant growth throughout the year, with potential upside from new product launches [34] Question: What is the expected mix of MRI versus OR procedures in the future? - Management indicated that while MRI procedures currently dominate, there is potential for OR procedures to grow faster as new therapies are introduced [40] Question: What is the status of GLP certification and customer interest? - Management confirmed that they are on track for GLP certification by the end of the year and noted strong interest from pharma partners for studies once certified [42][46]