Precision BioSciences (NasdaqCM:DTIL) Conference Transcript

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]