Precision BioSciences AASLD Update Call Summary Company Overview - **Company**: Precision BioSciences (NasdaqCM:DTIL) - **Event**: AASLD Business Update Call - **Date**: November 11, 2025 Key Points Industry and Company Focus - Precision BioSciences is focused on gene editing technologies, particularly in the treatment of chronic hepatitis B (HBV) and Duchenne muscular dystrophy (DMD) [3][59] Core Programs and Developments - **Arcus Platform**: The backbone of Precision BioSciences, now delivering results in clinical settings [4][5] - **Chronic Hepatitis B Program**: The PBGene HBV in the ELIMINATE B trial is a key focus, with promising data presented at AASLD [6][7] - **Duchenne Muscular Dystrophy Program**: The PBGene DMD program is set to start clinical trials in 2026, targeting a broad patient population [58][59] Financial Updates - Precision BioSciences raised $75 million to secure cash and operational runway through 2028, supporting ongoing clinical trials [8][59] Clinical Trial Highlights - **ELIMINATE B Trial**: - Phase I study focusing on E antigen negative patients, with a goal to identify a dosing regimen that allows stopping nucleos(t)ide analogs (NUCs) and testing for a cure [21][22] - Cohort 3 has shown promising results with a dose of 0.8 mg/kg, indicating a near-term path towards stopping NUCs [38][44] - Safety profile has been manageable, with no dose-limiting toxicities observed [24][29] Efficacy Data - **S Antigen Reduction**: - Cohort 1 (0.2 mg/kg) showed a 50% reduction in S antigen after nine months [31] - Cohort 2 (0.4 mg/kg) demonstrated sustained S antigen decline in all patients [33] - Cohort 3 (0.8 mg/kg) showed significant declines in S antigen, indicating effective viral DNA removal [34][35] Safety Profile - Adverse events were predictable and consistent with LNP-related infusion reactions, resolving within 12 hours [24][29] - No evidence of liver dysfunction was observed, with transaminase elevations being transient [28][29] Future Directions - Completion of dosing in Cohort 3 is expected by 2026, with plans to test for a cure by stopping NUCs [44][45] - The company aims to file an IND for PBGene DMD by the end of 2025 and begin clinical trials in early 2026 [58][59] Regulatory Considerations - The FDA has outlined approvable efficacy endpoints, including sustained suppression of HBV DNA and S antigen off treatment for at least six months [46][48] Off-Target Editing and Genomic Integrity - Precision BioSciences conducts extensive preclinical evaluations to ensure the specificity of their gene editing technology, with no off-target effects detected at therapeutically relevant doses [89][95] Market Context - The company is addressing a significant unmet need in the chronic hepatitis B and DMD markets, with a focus on innovative gene editing solutions [12][49] Conclusion Precision BioSciences is making significant strides in gene editing therapies for chronic hepatitis B and Duchenne muscular dystrophy, with promising clinical data and a strong financial position to support ongoing and future trials. The focus on safety and efficacy, along with regulatory alignment, positions the company well for potential breakthroughs in these challenging therapeutic areas.

AASLD 2025 Business Update November 2025 Forward-Looking Statements This presentation contains forward-looking statements, as may any related presentations, within the meaning of the Private Securities Litigation Reform Act of 1995. The Company intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements c ...

Core Viewpoint - Precision BioSciences, Inc. has announced an underwritten offering to sell 10,815,000 shares of its common stock along with warrants to purchase up to 5,407,500 additional shares at a combined price of $6.14 [1] Company Summary - The company is a clinical stage gene editing firm utilizing its proprietary ARCUS® platform to develop in vivo gene editing therapies targeting diseases with high unmet needs [1] - The offering includes both common stock and accompanying warrants, indicating a strategy to raise capital for further development [1]

Core Insights - Precision BioSciences, Inc. is a clinical stage gene editing company focused on developing in vivo gene editing therapies for diseases with high unmet needs [1] Group 1 - The company announced a late-breaking oral presentation at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting® 2025 [1] - The presentation will include data from the ongoing ELIMINATE-B Phase 1 study [1] - The study evaluates PBGENE-HBV, which is described as a first-in-class in vivo gene editing therapy [1]

Core Insights - Precision BioSciences, Inc. reported financial results for Q3 2025, highlighting significant advancements in its gene editing pipeline and promising Phase 1 safety and efficacy data for its product PBG [1] Financial Performance - The company announced its financial results for the third quarter ended September 30, 2025, indicating progress in its operations [1] Business Update - Precision BioSciences emphasized strong progress in its gene editing pipeline throughout the third quarter, showcasing the effectiveness of its proprietary ARCUS® platform [1]

Financial Performance - Revenue for the three months ended September 30, 2025, was $13,000, a significant decrease of 97.7% compared to $576,000 for the same period in 2024[24]. - Operating loss for the nine months ended September 30, 2025, was $65,857,000, compared to a net income of $24,912,000 for the same period in 2024[24]. - The company reported a net loss of $21,772,000 for the three months ended September 30, 2025, compared to a net loss of $16,425,000 for the same period in 2024[24]. - For the nine months ended September 30, 2025, the company reported a net loss of $65.857 million compared to a net income of $24.912 million for the same period in 2024[27]. - Revenue for the nine months ended September 30, 2025, was $60,000, a significant decrease from $68.058 million in the same period of 2024[87]. - The net loss for Q3 2025 was $21.772 million compared to a net loss of $16.425 million in Q3 2024, representing a 32.5% increase in losses[81]. - The company reported a net loss of $65.9 million for the nine months ended September 30, 2025, with an accumulated deficit of $548.3 million[183]. Assets and Liabilities - Total current assets decreased from $94,989,000 as of December 31, 2024, to $51,705,000 as of September 30, 2025, a decline of approximately 45.4%[22]. - Cash and cash equivalents decreased from $85,899,000 as of December 31, 2024, to $44,870,000 as of September 30, 2025, a reduction of approximately 47.8%[22]. - Total liabilities decreased from $79,995,000 as of December 31, 2024, to $76,876,000 as of September 30, 2025, a decline of about 2.7%[22]. - Stockholders' equity decreased from $56,393,000 as of December 31, 2024, to $16,634,000 as of September 30, 2025, a drop of approximately 70.5%[22]. - As of September 30, 2025, the company had cash, cash equivalents, and restricted cash totaling $71.212 million, down from $121.328 million at the end of 2024[27]. - The company had a restricted cash balance of $26.342 million as of September 30, 2025, up from $22.576 million at the end of 2024[36]. Expenses - Research and development expenses for the three months ended September 30, 2025, were $13,352,000, slightly up from $13,084,000 in the same period of 2024[24]. - Total operating expenses for the nine months ended September 30, 2025, were $64.716 million, down from $69.374 million in 2024[87]. - General and administrative expenses decreased to $7.3 million for the three months ended September 30, 2025, down approximately $1.4 million from $8.8 million in 2024[129]. - General and administrative expenses for the nine months ended September 30, 2025, were $25.0 million, down $0.7 million from $25.7 million in 2024[141]. - Employee share-based compensation expense for Q3 2025 was $2.386 million, down 25.4% from $3.199 million in Q3 2024[69]. Financing Activities - The company reported a net cash provided by financing activities of $17.646 million for the nine months ended September 30, 2025, compared to $44.530 million in the prior year[27]. - The company entered into a public offering on March 1, 2024, resulting in approximately $37.0 million of net proceeds after expenses[56]. - The company issued 1,818,886 shares through an ATM facility, net of issuance costs, contributing $9,516,000 to additional paid-in capital[25]. - The company entered into a loan agreement in July 2024, securing a term loan of $22.5 million[191]. - The outstanding principal balance on the 2024 Term Loan was $22.5 million as of September 30, 2025, with a stated interest rate of 5.75%[163]. Research and Development - The company plans to expand the ELIMINATE-B clinical trial and file an investigational new drug application for the PBGENE-DMD program[30]. - PBGENE-HBV is in a global first-in-human clinical trial aimed at treating chronic Hepatitis B, with multiple ascending dose levels being investigated[95]. - The ELIMINATE-B trial for PBGENE-HBV is recruiting patients in the U.S., Moldova, Hong Kong, New Zealand, and the U.K.[95]. - PBGENE-DMD received Rare Pediatric Disease designation from the FDA in June 2025 and Orphan Drug Designation in July 2025[99]. - The Company anticipates filing for an investigational new drug (IND) for PBGENE-DMD by the end of 2025, with Phase 1 initiation expected in the first half of 2026[102]. - The ongoing OTC-HOPE study for neonatal onset ornithine transcarbamylase deficiency is expected to yield data in the first half of 2026[105]. Market and Competition - The company has not generated any revenue from product sales and does not expect to do so in the foreseeable future, relying instead on collaboration and license agreements[108]. - The company faces significant competition from larger pharmaceutical and biotechnology companies with greater resources and expertise[216]. - Adverse public perception of genome editing may negatively impact the developmental progress or commercial success of the company's products[212]. - The company may experience delays in achieving development milestones, which could harm its business and credibility[211]. Future Outlook - The company expects to incur significant expenses and operating losses for the foreseeable future as it advances product candidates through development and clinical trials[83]. - The company believes existing cash and cash equivalents will be sufficient to fund operations into the second half of 2027, pending supportive Phase 1 data readouts for ongoing studies[166]. - The company may need to seek additional funding sooner than planned due to various factors, including clinical trial outcomes and regulatory approvals[188]. - The company has incurred significant operating losses and does not expect to achieve profitability in the foreseeable future[183]. - The company expects its cash runway to be sufficient to fund operating expenses and capital expenditures into the second half of 2027[188]. Risks and Challenges - Investment in biopharmaceutical product development is highly speculative, with significant risks that product candidates may fail to demonstrate efficacy or safety, obtain regulatory approvals, or become commercially viable[200]. - The company faces challenges in forecasting accuracy, resource allocation, and gaining market acceptance in the rapidly evolving gene editing field[201]. - Limited prior experience in commercialization may hinder market penetration and revenue potential[219]. - Compliance with complex and changing regulatory requirements poses significant challenges[220]. - Economic weaknesses, such as inflation and political instability, could impact market performance[220]. - Production shortages may arise from disruptions in raw material supply or manufacturing capabilities[220]. - Longer accounts receivable collection times could affect cash flow[220]. - Foreign currency exchange rate fluctuations may impact financial results[220].

Financial Performance - Total revenues for Q3 2025 were less than $0.1 million, a decrease from $0.6 million in Q3 2024, primarily due to reduced billable efforts under the Novartis Agreement[17] - Revenue for Q3 2025 was $13,000, a significant decrease from $576,000 in Q3 2024, representing a decline of approximately 97.7%[29] - Net loss for Q3 2025 was $21.8 million, or ($1.84) per share, compared to a net loss of $16.4 million, or ($2.25) per share, in Q3 2024[20] - The net loss for Q3 2025 was $21,772,000, compared to a net loss of $16,425,000 in Q3 2024, indicating an increase in losses of approximately 32.5%[29] - Total operating expenses for Q3 2025 were $20,680,000, slightly down from $21,851,000 in Q3 2024, a decrease of about 5.4%[29] Cash and Assets - As of September 30, 2025, the company had approximately $71.2 million in cash, cash equivalents, and restricted cash, with a cash runway expected to extend into the second half of 2027[16] - Cash, cash equivalents, and restricted cash as of September 30, 2025, were $71,212,000, down from $108,468,000 at the end of 2024, a decrease of about 34.4%[31] - Total assets decreased to $93,510,000 as of September 30, 2025, from $136,388,000 at the end of 2024, a decline of approximately 31.4%[31] - Total stockholders' equity fell to $16,634,000 as of September 30, 2025, down from $56,393,000 at the end of 2024, a decrease of about 70.5%[31] Research and Development - Research and development expenses increased to $13.4 million in Q3 2025 from $13.1 million in Q3 2024, driven by the PBGENE-DMD program[18] - The company commenced dosing in Cohort 3 of the ELIMINATE-B trial during Q3 2025, with additional data readouts planned for early 2026[7] - The investigational new drug (IND) filing for PBGENE-DMD is anticipated by the end of 2025, with Phase 1 initiation expected in the first half of 2026[7] - The company anticipates an IND filing for PBGENE-DMD by the end of 2025 and expects to initiate a Phase 1 clinical trial in 2026[26] - The design of PBGENE-DMD targets over 60% of patients with DMD, aiming for durable functional improvement with a one-time lower dose of AAV[26] Clinical Trials and Milestones - PBGENE-HBV has shown a durable HBsAg reduction of approximately 50% from baseline in one patient, ongoing seven months post-treatment[6] - The company received an $8 million milestone payment from Imugene on October 31, 2025, related to the azer-cel program[13] - The company has been granted Breakthrough Therapy designation by the FDA for PBGENE-HBV, which is designed to eliminate the root cause of chronic Hepatitis B[21] - The company aims to complete enrollment for the OTC-HOPE clinical trial by late 2025, with data expected in the first half of 2026[26]

Core Insights - Precision BioSciences, Inc. is a clinical stage gene editing company focused on developing in vivo gene editing therapies for diseases with high unmet needs [1] - The company will publish its financial results for the third quarter of 2025 and provide a business update on November 3, 2025 [1] Company Overview - Precision BioSciences utilizes its proprietary ARCUS® platform for gene editing [1] - The company is dedicated to improving life through innovative gene editing solutions [1]

Summary of Precision BioSciences FY Conference Call Company Overview - **Company**: Precision BioSciences (NasdaqCM:DTIL) - **Technology**: Proprietary gene editing technology called Arcus, which is a nuclease-based system distinct from CRISPR Cas technologies [4][5] Key Points Discussed Industry Context - **Gene Editing Technologies**: The session focused on advancements in genome editing technologies, particularly the applications of gene insertion and targeting viral genomes [1][2] - **Participants**: Included executives from Precision BioSciences, eCure, Harbor Site, and Seamless Therapeutics, discussing their respective technologies and programs [2] Precision BioSciences' Arcus Technology - **Unique Features**: Arcus nucleases create staggered overhang cuts, allowing for high-efficiency gene insertion in both dividing and non-dividing cells [30] - **Clinical Applications**: The technology is being utilized in partnership with eCure for clinical gene insertion, specifically targeting conditions like ornithine transcarbamylase (OTC) deficiency [5][28] Clinical Data and Outcomes - **OTC Deficiency Treatment**: A patient treated at six and a half months of age showed significant improvement, with a 50% reduction in ammonium levels and no hyperammonemic events post-treatment [36][37] - **Regulatory Approval**: The program has received clearance to operate in multiple regions, including the US, UK, Spain, and Australia [32] Comparison with Traditional Gene Therapy - **Gene Insertion vs. Gene Addition**: Traditional gene addition approaches can lead to dilution of therapeutic effects in actively dividing tissues, whereas gene insertion ensures that the therapeutic gene is integrated into the genome, providing sustained expression [18][19] - **Targeting Multiple Mutations**: The ability to insert large DNA fragments addresses diseases with multiple mutations, which traditional gene editing systems cannot effectively target [12][15] Market Potential and Competitive Landscape - **Hepatitis B Program**: Precision BioSciences is pursuing a hepatitis B program aimed at eliminating cccDNA, which is crucial for curing chronic infections [54][56] - **Commercial Viability**: The potential market for hepatitis B treatments is significant, with a large patient population and a clear unmet need for effective therapies [62] Regulatory Outlook - **Positive Regulatory Interactions**: The company has had favorable interactions with regulatory agencies, indicating a supportive environment for gene editing technologies [81][83] - **Flexibility in Development**: Regulators are showing a willingness to adapt traditional trial designs to accommodate the unique challenges of rare diseases [86] Future Directions - **Expansion Beyond Rare Diseases**: There is a focus on applying gene editing technologies to larger patient populations and various indications beyond rare genetic disorders [94][95] - **Technological Advancements**: Continuous improvements in gene editing technologies and cost efficiencies are expected to enhance the commercial viability of these therapies [95] Additional Insights - **Investor Sentiment**: Investors are increasingly interested in technologies that address unmet medical needs and have clear commercial potential [76][78] - **Long-term Impact**: The ability to treat young patients with severe genetic disorders could have significant societal and economic benefits [90] This summary encapsulates the key discussions and insights from the Precision BioSciences FY Conference Call, highlighting the company's innovative approach to gene editing and its implications for future therapies.

Summary of Precision BioSciences Conference Call Company Overview - **Company**: Precision BioSciences - **Industry**: Biotechnology, specifically focusing on gene editing therapies for liver diseases, including hepatitis B virus (HBV) and hepatocellular carcinoma [2][4] Key Points and Arguments Arcus Gene Editing Platform - **Technology**: Precision BioSciences utilizes a proprietary gene editing platform called Arcus, which is distinct from CRISPR Cas9 technology [4][5] - **Unique Features**: - **Cut**: Arcus creates a four base pair three-prime overhang, enhancing gene insertion efficiency [8] - **Size**: Arcus is the smallest gene editor at approximately 1,000 bases, facilitating better delivery via lipid nanoparticle (LNP) and adeno-associated virus (AAV) technologies [10][11] - **Simplicity**: Arcus consists of a single protein that recognizes and cuts DNA, contrasting with CRISPR systems that require multiple components [12][13] Focus on Hepatitis B Virus (HBV) - **Unmet Need**: Current functional cure rates for HBV are only 1-2%, highlighting a significant unmet medical need [15][46] - **Differentiation**: The unique features of the Arcus platform make it particularly suitable for targeting covalently closed circular DNA (cccDNA) in HBV, which is crucial for achieving a complete cure rather than just viral suppression [17][22] Clinical Trials and Efficacy - **ILLUMINATE B Study**: A Phase 1 study testing the safety and antiviral response of PBG and HBV in patients on nucleoside analogs [24] - **Design**: Patients receive three administrations of PBG and HBV while continuing their nucleoside analog therapy to prevent reactivation [26] - **Cohort Data**: Initial data from Cohort 1 showed good tolerability and substantial S antigen reductions of 45-70% [31] - **Safety**: No greater than grade two adverse events (AEs) were observed, with infusion-related reactions being the most common [30] Future Directions - **Biopsy Data**: Liver biopsies will be conducted to assess the effects of PBG and HBV on cccDNA and integrated DNA levels [42][43] - **Dosing Interval**: The current dosing interval is eight weeks, but there is potential to shorten this based on safety data [36][38] - **Upcoming Milestones**: Key milestones include presentations at the AASLD liver meeting, completion of Cohorts 2 and 3, and potential adjustments to dosing intervals [49][51] Broader Implications - **Treatment Landscape**: The current treatment landscape for HBV has been underwhelming, with no effective means to eliminate cccDNA, which is essential for achieving a complete cure [46][47] - **Field Excitement**: The innovative approach of targeting the root cause of HBV infection has generated excitement within the field, as evidenced by the opportunity for a late breaker oral presentation at an upcoming conference [47][48] Additional Important Insights - **Long-term Goals**: The ultimate aim is to achieve complete viral clearance and eliminate the risk of reactivation, moving beyond the current focus on functional cures [22][27] - **Patient-Centric Approach**: The design of the trial considers patient safety and aims to optimize treatment duration and efficacy [38][51]