Core Business Highlights - Sangamo Therapeutics announced positive topline results from the registrational Phase 1/2 STAAR study for isaralgagene civaparvovec (ST-920) in Fabry disease, showing a mean annualized estimated glomerular filtration rate (eGFR) slope of 1.965 mL/min/1.73m²/year at 52 weeks across all 32 patients dosed [6][4] - The FDA has agreed that the eGFR slope will serve as the primary basis for approval under the Accelerated Approval pathway [6] - The company raised approximately $21 million in net proceeds from an underwritten registered equity offering [5] Financial Performance - For Q2 2025, Sangamo reported revenues of $18.3 million, a significant increase from $0.3 million in Q2 2024, primarily due to an upfront license payment from Eli Lilly [8] - The consolidated net loss for Q2 2025 was $20.0 million, or $0.08 per share, compared to a net loss of $36.1 million, or $0.18 per share, for the same period in 2024 [7] - Total operating expenses for Q2 2025 were $36.2 million, a decrease from $37.4 million in Q2 2024, driven by cost reductions from strategic realignment [11][10] Pipeline Developments - The first clinical site for the Phase 1/2 STAND study in chronic neuropathic pain has been initiated, with the first patient expected to be dosed in fall 2025 and preliminary efficacy data anticipated in late 2026 [2][4] - Sangamo plans to present additional clinical data at the 15th International Congress of Inborn Errors of Metabolism in September 2025 [9] - The company is advancing preparations for a Clinical Trial Application (CTA) submission for ST-506, an investigational treatment for prion disease, expected as early as mid-2026 [9] Cash Position and Financial Guidance - As of June 30, 2025, cash and cash equivalents were $38.3 million, down from $41.9 million at the end of 2024, but expected to fund operations into Q4 2025 [12] - The company reiterated its financial guidance for 2025, expecting total operating expenses in the range of approximately $135 million to $155 million on a GAAP basis [13][14]

Core Points - Sangamo Therapeutics, Inc. is set to release its second quarter 2025 financial results on August 7, 2025, after market close [1] - A conference call will be held at 4:30 p.m. Eastern on the same day to discuss financial results and provide business updates [1] - Participants can register for the call via a provided link and are encouraged to join 10 minutes early [2] Company Overview - Sangamo Therapeutics focuses on genomic medicine, aiming to develop treatments for serious neurological diseases lacking adequate options [4] - The company utilizes zinc finger epigenetic regulators and a capsid discovery platform to potentially address neurological disorders and expand delivery methods [4] - Sangamo's pipeline includes multiple partnered programs and opportunities for further partnerships and investments [4]

Financial Data and Key Metrics Changes - The company reported a significant reduction in non-GAAP operating expenses by 50% year-on-year in 2024, focusing on key priorities to enhance efficiency [17] - An equity offering was announced to extend the company's cash runway, with proceeds expected to last until late Q3 2025 [16][12] Business Line Data and Key Metrics Changes - The capsid engineering platform saw a new licensing agreement with Eli Lilly, which includes an $18 million upfront fee and potential total payments of up to $1.4 billion across five targets [6][7] - The neurology pipeline is advancing, with preparations for a Phase 1/2 study of ST503 for chronic neuropathic pain expected to begin in mid-2025 [8][10] Market Data and Key Metrics Changes - The company is actively engaging in discussions with potential partners for its Fabry disease program, with a focus on securing a commercial partnership that aligns with its neurology mission [11][19] Company Strategy and Development Direction - The long-term vision is to position Sangamo as a neurology-focused genomic medicine company, aiming to deliver transformational therapies and significant shareholder value [15] - The company is committed to operating efficiently while advancing its neurology programs, with a disciplined approach to capital allocation [17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the FDA's engagement and the clarity provided during the Type B meeting, which is crucial for the BLA submission process [32] - The company remains focused on securing funding and partnerships to support its neurology pipeline and Fabry disease program [19] Other Important Information - The company plans to present data at the ASGCT Annual Meeting, showcasing advancements in its neurology pipeline and the effectiveness of its technologies [19][9] Q&A Session Summary Question: Can you provide more color on the top line eGFR data? - Management confirmed that the updated mean eGFR slope will be shared, along with additional information at a later date [24] Question: How many potential partners are you currently in conversations with for STABBI? - Management indicated that multiple potential partners are being engaged, and the Type B meeting was beneficial for discussions [27] Question: Has the pace of conversations with potential partners changed due to the macro landscape? - Management noted that interactions with the FDA have been positive and have not impacted the program [33] Question: Will you file based on 52-week eGFR data? - Management confirmed that they are pursuing the agreement with the FDA to use 52-week eGFR data for the entire patient population [38] Question: Are all patients who were on ERT still off ERT? - Management confirmed that all 18 patients who started on ERT are still off ERT [47]

Financial Data and Key Metrics Changes - The company reported a significant reduction in non-GAAP operating expenses by 50% year-on-year in 2024, focusing on key priorities to enhance efficiency [16][11] - An equity offering was announced to extend the company's cash runway, which is expected to last until late in the third quarter of 2025 [15][11] Business Line Data and Key Metrics Changes - The company signed a third capsid license agreement with Eli Lilly, receiving an upfront fee of $18 million and potential additional payments of up to $1.4 billion across five disease targets [5][6] - The Fabry program achieved key de-risking milestones, with all patients in the Phase 1/2 STAR study completing at least 52 weeks of follow-up, indicating a positive mean eGFR slope [9][10] Market Data and Key Metrics Changes - The company is actively engaging in discussions with multiple potential partners for its Fabry disease program, indicating strong interest in its technology [12][32] - The FDA's recent interactions have been positive, with no significant changes in their approach to the company's programs, including Fabry disease [32][33] Company Strategy and Development Direction - The long-term vision is to focus on neurology as a core area, aiming to deliver transformational therapies while maximizing shareholder value [13][15] - The company is committed to securing a commercial partnership for the Fabry program to support its neurology mission and ensure sustainable funding [11][18] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the FDA's engagement and the clarity provided regarding the chemistry, manufacturing, and controls pathway for the BLA submission [10][32] - The company remains focused on advancing its neurology pipeline and securing necessary funding to support ongoing clinical trials [11][18] Other Important Information - The company plans to present data at the ASGCT Annual Meeting, showcasing advancements in its neurology pipeline and the effectiveness of its capsid delivery technology [8][18] - The company is exploring additional funding opportunities through collaborations related to its technology platforms [12] Q&A Session Summary Question: Can you provide more color on what exactly you plan to show in the top line eGFR data? - Management confirmed that they will share the updated mean eGFR slope and additional information at a later date [21][23] Question: How many potential partners are you currently in conversations with regarding STABBI? - Management indicated that there are multiple potential partners but did not disclose specific details [26] Question: Has the pace of conversations with potential partners changed due to the macro landscape? - Management reported that interactions with the FDA have been positive and have not impacted their program [33] Question: Do you still plan to file based on 52-week eGFR data? - Management confirmed that they are pursuing the agreement with the FDA to use 52-week eGFR data for the entire patient population [37] Question: Are all patients who were on ERT still off ERT? - Management confirmed that all 18 patients who started on ERT are still off ERT [46] Question: Is there any threshold for the eGFR slope that should be looked at? - Management stated they are not commenting on specific statistical significance at this time but confirmed the mean eGFR slope remains positive [48][49]

Core Viewpoint - Sangamo Therapeutics reported a quarterly loss of $0.14 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.11, marking an earnings surprise of -27.27% [1] - The company generated revenues of $6.44 million for the quarter, missing the consensus estimate by 29.77%, compared to $0.48 million in the same quarter last year [2] Group 1: Financial Performance - The company has surpassed consensus EPS estimates only once in the last four quarters [2] - Sangamo shares have declined approximately 31.4% year-to-date, contrasting with the S&P 500's decline of -3.8% [3] Group 2: Future Outlook - The current consensus EPS estimate for the upcoming quarter is -$0.09 on revenues of $14.8 million, and for the current fiscal year, it is -$0.39 on revenues of $46.97 million [7] - The estimate revisions trend for Sangamo is currently unfavorable, resulting in a Zacks Rank 4 (Sell), indicating expected underperformance in the near future [6] Group 3: Industry Context - The Medical - Biomedical and Genetics industry is ranked in the top 34% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors [5]

Neurology Pipeline and Technology - Sangamo's STAC-BBB capsid demonstrates industry-leading performance with 700-fold higher transgene expression than benchmark capsid AAV9[25] - ST-503 for chronic neuropathic pain is advancing with patient enrollment and dosing expected in mid-2025 and preliminary proof of efficacy data anticipated in Q4 2026[16, 35, 39] - Prion disease program is progressing with a CTA submission anticipated in Q1 2026 and preliminary clinical data expected in Q4 2026[12, 20, 35, 39] - ST-503 resulted in approximately 70% repression of the SCN9A gene in mice, reducing pain hypersensitivity[18] - Intrathecal delivery of ZFR in NHPs by AAV9 demonstrated up to 60% repression of SCN9A in dorsal root ganglia (DRG) tissue[18] Partnerships and Financials - Sangamo received an $18 million upfront license fee from Lilly for a capsid license agreement to deliver genomic medicines for up to five diseases of the CNS, with potential for up to $1.4 billion in additional licensed target fees and milestone payments, plus tiered royalties on potential net sales[30, 36] - STAC-BBB partnerships have the potential for up to $4.6 billion in future milestones and exercise fees, assuming exercise of all options and targets, plus additional potential product royalties[26] - As of March 31, 2025, Sangamo had approximately $25.2 million in cash and cash equivalents[36] Fabry Disease Program - A potential BLA submission for Isaralgagene civaparvovec is anticipated as early as the first quarter of 2026[13, 28, 35, 39] - All dosed patients (32) in Phase 1/2 STAAR study data have now completed at least 52-weeks of follow-up, with preliminary analysis indicating a positive mean eGFR slope[35, 39, 130] - In the Phase 1/2 STAAR study, a positive mean eGFR slope of 3.061 mL/min/1.73m2/year (95% confidence interval: 0.863, 5.258) was observed in the 23 patients that have reached 1-year follow-up[136]

PART I [Financial Statements](index=7&type=section&id=Item%201.%20Financial%20Statements) Q1 2025 financial statements show reduced net loss and expenses, but declining cash and equity raise substantial doubt about the company's going concern Condensed Consolidated Balance Sheet Highlights (Unaudited) | Account | March 31, 2025 ($ in thousands) | December 31, 2024 ($ in thousands) | Change | | :--- | :--- | :--- | :--- | | Cash and cash equivalents | 25,180 | 41,918 | ▼ $16,738 | | Total assets | 86,166 | 101,635 | ▼ $15,469 | | Total liabilities | 81,260 | 78,865 | ▲ $2,395 | | Total stockholders' equity | 4,906 | 22,770 | ▼ $17,864 | Condensed Consolidated Statements of Operations (Unaudited) | Account | Three Months Ended Mar 31, 2025 ($ in thousands) | Three Months Ended Mar 31, 2024 ($ in thousands) | Change | | :--- | :--- | :--- | :--- | | Revenues | 6,437 | 481 | ▲ $5,956 | | Research and development | 26,006 | 35,891 | ▼ $9,885 | | General and administrative | 10,059 | 11,767 | ▼ $1,708 | | Loss from operations | (29,628) | (51,526) | ▲ $21,898 | | Net loss | (30,597) | (49,089) | ▲ $18,492 | | Net loss per share | (0.14) | (0.27) | ▲ $0.13 | Condensed Consolidated Statements of Cash Flows (Unaudited) | Activity | Three Months Ended Mar 31, 2025 ($ in thousands) | Three Months Ended Mar 31, 2024 ($ in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | (26,149) | (48,663) | | Net cash provided by investing activities | — | 35,840 | | Net cash provided by financing activities | 8,134 | 22,122 | | Net (decrease) increase in cash | (16,738) | 8,629 | - The company has substantial doubt about its ability to continue as a going concern due to a history of significant losses, negative cash flows, and limited liquidity, with existing capital resources as of March 31, 2025, at only **$25.2 million**[36](index=36&type=chunk)[39](index=39&type=chunk) - Subsequent to the quarter end, on April 3, 2025, Sangamo entered into a global capsid delivery license agreement with Eli Lilly, receiving an **$18.0 million** upfront payment and eligible for up to **$1.4 billion** in future milestones and royalties[136](index=136&type=chunk)[137](index=137&type=chunk) - The collaboration with Pfizer for giroctocogene fitelparvovec was terminated for convenience by Pfizer, effective April 21, 2025, with Sangamo recognizing a final **$5.0 million** in revenue from this agreement in Q1 2025[100](index=100&type=chunk)[107](index=107&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=27&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management reiterates going concern risk, with current capital funding operations only into early Q3 2025, emphasizing the critical need for a Fabry disease program partner and additional funding - There is substantial doubt about the company's ability to continue as a going concern, as cash on hand as of March 31, 2025, plus subsequent proceeds from the Lilly agreement (**$18.0M**) and ATM sales (**$5.1M**), is only sufficient to fund operations into **early Q3 2025**[144](index=144&type=chunk)[184](index=184&type=chunk) - Securing a commercialization partner for the Fabry disease program is **critical**, as failure to do so in the near term will substantially impair the company's ability to raise the additional capital needed to continue operations[145](index=145&type=chunk)[186](index=186&type=chunk) - Key clinical milestones include a pivotal read-out for the Fabry disease program (isaralgagene civaparvovec) expected by the **end of Q2 2025**, with a potential BLA submission as early as **Q1 2026**, and the company is also seeking a partner for its hemophilia A program following Pfizer's termination[151](index=151&type=chunk)[156](index=156&type=chunk) Revenue and Operating Expense Changes (Q1 2025 vs Q1 2024) | Item | Q1 2025 ($ in thousands) | Q1 2024 ($ in thousands) | Change ($ in thousands) | Reason for Change | | :--- | :--- | :--- | :--- | :--- | | **Revenues** | **6,437** | **481** | **+5,956** | **$5.0M from Pfizer termination, $1.0M from Sigma license.** | | Research & Development | 26,006 | 35,891 | (9,885) | Lower headcount, program reprioritization, lower overhead. | | General & Administrative | 10,059 | 11,767 | (1,708) | Lower headcount, lower professional services fees. | | Impairment of long-lived assets | — | 4,349 | (4,349) | No impairment in 2025; charge in 2024 related to restructuring. | - Net cash used in operating activities decreased to **$26.1 million** in Q1 2025 from **$48.7 million** in Q1 2024, primarily due to a lower net loss and changes in working capital[188](index=188&type=chunk)[189](index=189&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=36&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) As a smaller reporting company, Sangamo is not required to provide quantitative and qualitative disclosures about market risk - As a smaller reporting company, Sangamo is not required to provide quantitative and qualitative disclosures about market risk[195](index=195&type=chunk) [Controls and Procedures](index=38&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded the company's disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal control over financial reporting during the quarter - The principal executive officer and principal financial officer concluded that the company's disclosure controls and procedures were effective as of March 31, 2025[198](index=198&type=chunk) - There were no changes in internal control over financial reporting during the quarter that materially affected, or are reasonably likely to materially affect, these controls[200](index=200&type=chunk) PART II [Legal Proceedings](index=39&type=section&id=Item%201.%20Legal%20Proceedings) The company reports it is not a party to any material pending legal proceedings - Sangamo is not currently involved in any material pending legal proceedings[202](index=202&type=chunk) [Risk Factors](index=39&type=section&id=Item%201A.%20Risk%20Factors) New risks include a Nasdaq deficiency notice for low stock price, potential delisting, and the impact of FDA policy changes or international trade policies on product approvals and supply chain - On **April 30, 2025**, Sangamo received a deficiency notice from Nasdaq because its common stock bid price had closed below **$1.00** per share for **30 consecutive business days**, with the company having until **October 27, 2025**, to regain compliance or face potential delisting[204](index=204&type=chunk) - A potential delisting from Nasdaq could adversely affect the stock's liquidity and market price, and would substantially impair the company's ability to raise additional capital and continue as a going concern[204](index=204&type=chunk)[207](index=207&type=chunk) - The company faces risks from potential disruptions at the FDA, including workforce reductions or policy changes, which could delay product approvals, with a specific risk being the potential discontinuation of the **Accelerated Approval program**, the planned regulatory pathway for its Fabry disease candidate, isaralgagene civaparvovec[213](index=213&type=chunk)[215](index=215&type=chunk) - International trade policies, including tariffs and sanctions, may adversely affect the business by increasing costs for materials, disrupting the supply chain, and creating a complex and unpredictable trade landscape[208](index=208&type=chunk)[209](index=209&type=chunk)[210](index=210&type=chunk) [Other Information (Items 2-6)](index=41&type=section&id=Item%202-6) This section covers standard reporting items, including no unregistered equity sales, no defaults on senior securities, no mine safety disclosures, and no other material information - Item 2: No unregistered sales of equity securities were reported for the period[216](index=216&type=chunk) - Items 3, 4, and 5 were marked as 'Not applicable' or 'None', indicating no defaults on senior securities, no mine safety disclosures, and no other information to report[217](index=217&type=chunk)[218](index=218&type=chunk)[219](index=219&type=chunk)

Group 1 - Sangamo Therapeutics, Inc. (NASDAQ: SGMO) has established a deal with Eli Lilly and Company (LLY) to advance up to 5 neurological programs [2] - The article is authored by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace, providing in-depth analysis of pharmaceutical companies [1][2] - The Biotech Analysis Central service includes a library of over 600 biotech investing articles and a model portfolio of more than 10 small and mid-cap stocks [2]

Financial Data and Key Metrics Changes - Sangamo Therapeutics reduced non-GAAP operating expenses by nearly half year-over-year since 2023 [9] - The company raised over $100 million in funding through non-dilutive license fees, milestone payments, and equity financing in 2024 [9] Business Line Data and Key Metrics Changes - The company advanced its neurology therapies, securing its first-ever neurology IND for idiopathic small fiber neuropathy [7] - The Fabry gene therapy study continues to generate best-in-class data, with pivotal data readout expected in mid-2025 [9][22] Market Data and Key Metrics Changes - Interest in the Fabry program has been strong, with ongoing business development negotiations for a commercial partner [11] - The company is actively engaged in advanced contract negotiations for a third STAC-BBB license agreement [13] Company Strategy and Development Direction - Sangamo's number one priority is addressing financing needs to ensure the company is well-capitalized for future success [11] - The company aims to secure a partnership for the Fabry program by the second quarter of 2025 [12] - The regulatory pathway for accelerated approval in Fabry disease could reduce the time to potential approval by approximately three years [8][29] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress made in transitioning to a clinical-stage neurology company [28] - The company is focused on raising additional capital to support its programs and is in late-stage negotiations for a third STAC-BBB license agreement [30] Other Important Information - The company plans to begin patient enrollment and dosing for ST-503 in mid-2025, with preliminary proof of efficacy data expected in the fourth quarter of 2026 [19] - The FDA has provided a clear regulatory pathway to accelerate approval for ST-920, with full 52-week data expected in the first half of 2025 [25] Q&A Session Summary Question: Is the company still waiting on any data for the Fabry program? - Management confirmed they are in late-phase discussions with several partners and are looking forward to positive data from the ongoing studies [34] Question: Have potential partners seen any data beyond the WORLDSymposium data? - Management indicated that while partners have seen broader data, they have not seen later efficacy data beyond what was presented [45] Question: What is the status of the STAC-BBB deal? - Management hopes to finalize the deal by the end of the quarter and indicated that the partner is a logical blue-chip choice [55] Question: How is the company managing operating expenses going forward? - The company has reduced operating expenses significantly and plans to maintain the same level of expenses as last year while advancing its neurology pipeline [58] Question: What are the patient enrollment criteria for the Nav1.7% study? - Management stated that the criteria will be published on clinical trial registries, emphasizing the importance of a clear result for the one-time treatment [73]

Financial Data and Key Metrics Changes - Sangamo Therapeutics reduced non-GAAP operating expenses by nearly half year-over-year since 2023 [9] - The company raised over $100 million in funding through non-dilutive license fees, milestone payments, and equity financing in 2024 [9] Business Line Data and Key Metrics Changes - The company advanced its neurology therapies, securing its first-ever neurology IND for idiopathic small fiber neuropathy [7] - The Fabry gene therapy study continues to generate best-in-class data, with pivotal data readout expected in mid-2025 [9][22] Market Data and Key Metrics Changes - Interest in the Fabry program has been strong, with ongoing business development negotiations for a commercial partner [11] - The company is actively engaged in advanced contract negotiations for a third STAC-BBB license agreement [12] Company Strategy and Development Direction - Sangamo's number one priority is addressing its financing needs to fulfill its potential [11] - The company aims to secure a partnership for Fabry that provides capital for executing other programs [12] - The regulatory pathway for accelerated approval in Fabry disease could reduce the time to potential approval by approximately three years [8][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress made towards becoming a clinical-stage neurology company [28] - The company is focused on raising additional capital to support its operations and development [30] Other Important Information - The company plans to begin patient enrollment and dosing for ST-503 in mid-2025, with preliminary proof of efficacy data expected in the fourth quarter of 2026 [19] - The FDA has provided a clear regulatory pathway to accelerate approval for ST-920, with a potential BLA submission in the second half of 2025 [25][26] Q&A Session Summary Question: Is the company still waiting on any data for the Fabry program? - Management confirmed they are in late-phase discussions with several partners and look forward to seeing the one-year data for the last patient soon [34][36] Question: Have potential partners seen any data beyond the WORLDSymposium data? - Management indicated that partners have not seen efficacy data beyond what was presented at the WORLDSymposium [45] Question: What is the status of the STAC-BBB deal? - Management hopes to finalize the deal by the end of the quarter and indicated that the partner is a logical blue-chip choice [55] Question: How is the company managing operating expenses going forward? - Management has reduced operating expenses by nearly half year-over-year and plans to maintain the same level of expenses as last year while advancing the neurology pipeline [58] Question: What are the patient enrollment criteria for the Nav1.7% study? - Management stated that the criteria will be published on clinical trial registries and emphasized the importance of a clear result for the one-time treatment [73][76]