[Sangamo Therapeutics Q2 2025 Earnings Release](index=1&type=section&id=Sangamo%20Therapeutics%20Q2%202025%20Earnings%20Release) Sangamo Therapeutics reports significant Q2 2025 progress in its Fabry disease and neurology pipelines, alongside improved financial results driven by increased revenues and reduced net loss [Business Highlights](index=1&type=section&id=Recent%20Business%20Highlights) Sangamo Therapeutics announced significant progress across its pipeline in Q2 2025, including positive Fabry disease study results, new clinical site initiation for neuropathic pain, and productive discussions for its prion disease program, alongside a $21 million equity offering [Corporate Updates](index=1&type=section&id=Corporate%20Updates) The company successfully raised approximately $21 million in net proceeds from a recent equity offering - Raised approximately **$21 million** in net proceeds from an underwritten registered equity offering[5](index=5&type=chunk) [Fabry Disease Program (isaralgagene civaparvovec / ST-920)](index=1&type=section&id=Fabry%20Disease) Positive topline results from the STAAR study for Fabry disease gene therapy support an accelerated approval pathway with the FDA - Announced positive topline results from the registrational Phase 1/2 STAAR study for isaralgagene civaparvovec (ST-920) in adults with Fabry disease[1](index=1&type=chunk)[6](index=6&type=chunk) Key Efficacy Endpoints from STAAR Study | Endpoint | Result | Follow-up Period | Patient Count | | :--- | :--- | :--- | :--- | | **Mean Annualized eGFR Slope** | +1.965 mL/min/1.73m²/year | 52 weeks | 32 | | **Mean Annualized eGFR Slope** | +1.747 mL/min/1.73m²/year | 104 weeks | 19 | | **α-Gal A Activity** | Maintained elevated expression | Up to 4.5 years | Longest treated patient | | **Plasma lyso-Gb3 Levels** | Remained generally stable | Post-ERT withdrawal | N/A | - The FDA has agreed that the positive mean annualized eGFR slope at 52 weeks will serve as the primary basis for a Biologics License Application (BLA) submission under the Accelerated Approval pathway, anticipated as early as Q1 2026[1](index=1&type=chunk)[6](index=6&type=chunk)[11](index=11&type=chunk) - The treatment demonstrated a favorable safety and tolerability profile without the need for preconditioning, with most adverse events being grade 1-2[11](index=11&type=chunk) [Core Neurology Pipeline](index=2&type=section&id=Core%20Neurology%20Pipeline) Sangamo initiated its first clinical site for chronic neuropathic pain and aligned on study design for its prion disease program - **Chronic Neuropathic Pain (ST-503):** The first clinical site has been initiated for the Phase 1/2 STAND study, with the first patient expected to be dosed in fall 2025 and preliminary efficacy data anticipated in Q4 2026[2](index=2&type=chunk)[11](index=11&type=chunk) - **Prion Disease (ST-506):** Held a productive meeting with the UK's MHRA, aligning on study design, with a Clinical Trial Application (CTA) submission expected as early as mid-2026[2](index=2&type=chunk)[11](index=11&type=chunk) [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) For Q2 2025, Sangamo reported significantly increased revenues to $18.3 million and a narrowed net loss, with cash and equivalents of $38.3 million expected to fund operations into Q4 2025 [Key Financial Metrics](index=2&type=section&id=Key%20Financial%20Metrics) Sangamo's Q2 2025 revenues significantly increased to $18.3 million, leading to a reduced net loss of $20.0 million Q2 2025 vs. Q2 2024 Performance | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | **Revenues** | $18.3 million | $0.3 million | | **Net Loss** | ($20.0 million) | ($36.1 million) | | **Net Loss per Share** | ($0.08) | ($0.18) | - The **$18.0 million** increase in revenues was primarily attributable to an upfront license payment received under a capsid license agreement with Eli Lilly and Company[10](index=10&type=chunk) [Operating Expenses](index=3&type=section&id=GAAP%20and%20Non-GAAP%20Operating%20Expenses) GAAP operating expenses decreased in Q2 2025 due to strategic realignment, while non-GAAP expenses slightly increased Operating Expenses for Q2 (in millions) | Expense Type | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | **GAAP Operating Expenses** | $36.2 | $37.4 | | **Non-GAAP Operating Expenses** | $33.0 | $31.9 | - The decrease in GAAP operating expenses was mainly due to cost reductions from strategic realignment, including lower headcount and facilities costs, partially offset by increased expenses for BLA readiness activities for the Fabry disease program[13](index=13&type=chunk) [Cash Position and Financial Guidance](index=3&type=section&id=Cash%20and%20Financial%20Guidance) Sangamo's cash and equivalents of $38.3 million are projected to fund operations into Q4 2025, with full-year 2025 operating expense guidance reiterated - Cash and cash equivalents were **$38.3 million** as of June 30, 2025, sufficient to fund planned operations into the fourth quarter of 2025 with recent at-the-market offering proceeds[14](index=14&type=chunk) Reiterated Full-Year 2025 Financial Guidance | Metric | Expected Range (in millions) | | :--- | :--- | | **GAAP Total Operating Expenses** | $135 - $155 | | **Non-GAAP Total Operating Expenses** | $125 - $145 | [Corporate Information and Events](index=3&type=section&id=Corporate%20Information%20and%20Events) Sangamo Therapeutics will participate in two investor conferences in September 2025 and held a conference call on August 7, 2025, to provide updates - Sangamo plans to participate in the Cantor Global Healthcare Conference and the Wells Fargo Healthcare Conference from September 3-5, 2025[17](index=17&type=chunk)[19](index=19&type=chunk) - The management team held a conference call on August 7, 2025, to discuss program and financial updates[18](index=18&type=chunk) [Financial Statements](index=6&type=section&id=SELECTED%20CONSOLIDATED%20FINANCIAL%20DATA) The consolidated financial statements detail Sangamo's Q2 2025 performance, showing increased revenues, reduced net loss, and a cash position of $38.3 million [Statement of Operations](index=6&type=section&id=Statement%20of%20Operations%20Data) The Statement of Operations shows a significant increase in revenues and a reduction in net loss for both the three and six months ended June 30, 2025 Consolidated Statement of Operations Data (Unaudited, in thousands) | | Three Months Ended June 30, | Six Months Ended June 30, | | :--- | :--- | :--- | :--- | :--- | | | **2025** | **2024** | **2025** | **2024** | | **Revenues** | $18,306 | $356 | $24,743 | $837 | | **Total operating expenses** | $36,161 | $37,440 | $72,226 | $89,447 | | **Loss from operations** | ($17,855) | ($37,084) | ($47,483) | ($88,610) | | **Net loss** | ($19,986) | ($36,128) | ($50,583) | ($85,217) | | **Net loss per share** | ($0.08) | ($0.18) | ($0.21) | ($0.44) | [Balance Sheet](index=6&type=section&id=Selected%20Balance%20Sheet%20Data) The Balance Sheet indicates total assets of $97.6 million and cash and cash equivalents of $38.3 million as of June 30, 2025 Selected Balance Sheet Data (Unaudited, in thousands) | | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Cash and cash equivalents** | $38,344 | $41,918 | | **Total assets** | $97,558 | $101,635 | | **Total stockholders' equity** | $19,602 | $22,770 | [Disclosures](index=4&type=section&id=Disclosures) This section outlines forward-looking statements regarding financial risks and clinical uncertainties, and explains the rationale for using non-GAAP financial measures - **Forward-Looking Statements:** Key risks highlighted include the company's lack of capital resources, the need for substantial additional funding to continue as a going concern, the uncertain R&D process, and the unpredictable regulatory approval process[23](index=23&type=chunk) - **Non-GAAP Financial Measures:** The company presents non-GAAP operating expenses, which exclude items like depreciation, stock-based compensation, and impairment charges, to provide what it believes is a more meaningful comparison of its operational performance over time[25](index=25&type=chunk)

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]

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)

Financial Performance - Novavax reported quarterly earnings of $2.93 per share, significantly exceeding the Zacks Consensus Estimate of $0.71 per share, and compared to a loss of $1.05 per share a year ago, representing an earnings surprise of 312.68% [1] - The company posted revenues of $666.66 million for the quarter ended March 2025, surpassing the Zacks Consensus Estimate by 215.04%, and compared to year-ago revenues of $93.86 million [2] Market Performance - Novavax shares have declined approximately 26% since the beginning of the year, while the S&P 500 has decreased by 4.3% [3] - The current consensus EPS estimate for the upcoming quarter is $0.07 on revenues of $149.29 million, and for the current fiscal year, it is $0.84 on revenues of $633.26 million [7] Industry Outlook - The Medical - Biomedical and Genetics industry, to which Novavax belongs, is currently ranked in the top 33% of over 250 Zacks industries, indicating a favorable outlook [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact Novavax's stock performance [5]

Core Viewpoint - Regenxbio (RGNX) is anticipated to report a significant year-over-year increase in earnings and revenues for the quarter ended March 2025, which could influence its stock price depending on the actual results compared to estimates [1][2]. Earnings Expectations - The upcoming earnings report is expected to be released on May 12, 2025, with a consensus estimate of $0.41 per share, reflecting a year-over-year increase of +129.7% [3]. - Revenues are projected to reach $109.98 million, representing a substantial increase of 604.1% from the same quarter last year [3]. Estimate Revisions - Over the last 30 days, the consensus EPS estimate has been revised down by 6.26%, indicating a reassessment by analysts [4]. - The Most Accurate Estimate for Regenxbio is higher than the Zacks Consensus Estimate, resulting in a positive Earnings ESP of +57.77% [10][11]. Earnings Surprise Prediction - The Zacks Earnings ESP model suggests that a positive Earnings ESP reading is a strong predictor of an earnings beat, especially when combined with a Zacks Rank of 1 (Strong Buy), 2 (Buy), or 3 (Hold) [8]. - Regenxbio currently holds a Zacks Rank of 3, indicating a likelihood of beating the consensus EPS estimate [11]. Historical Performance - In the last reported quarter, Regenxbio was expected to post a loss of $1.27 per share but actually reported a loss of $1.01, resulting in a surprise of +20.47% [12]. - Over the past four quarters, the company has surpassed consensus EPS estimates two times [13]. Industry Context - In the Zacks Medical - Biomedical and Genetics industry, Sangamo Therapeutics (SGMO) is expected to report a loss of $0.11 per share for the same quarter, with a year-over-year change of +59.3% [17]. - Sangamo's revenue is projected to be $9.17 million, up 1810.4% from the previous year [17]. - Despite a recent downward revision of 23.8% in consensus EPS estimates, Sangamo has an Earnings ESP of 27.27%, but it holds a Zacks Rank of 4 (Sell), making predictions of an earnings beat uncertain [18].

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]