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Precision BioSciences(DTIL) - 2025 Q1 - Quarterly Results

First Quarter 2025 Financial Results and Business Update Precision BioSciences reported strong Q1 2025 momentum with significant clinical data, regulatory approvals, and accelerated program development CEO Commentary CEO Michael Amoroso highlighted strong Q1 2025 momentum, significant clinical data generation, and accelerated program development - ARCUS in vivo gene insertion validated clinically with a complete response in an infant with OTC-deficiency3 - Phase 1 ELIMINATE-B trial for chronic Hepatitis B (PBGENE-HBV) progressing, with encouraging safety data after repeat dosing presented at EASL Congress3 - U.S. IND approval and Fast Track Designation received for PBGENE-HBV, underscoring its potential for a curative treatment3 - PBGENE-DMD program accelerated, targeting IND/CTA in 2025 with clinical data in 2026, based on preclinical proof of protein expression and improved muscle function34 First Quarter 2025 Business Highlights Q1 2025 achievements include new clinical data, initial safety results, regulatory clearances, program acceleration, and extended cash runway - New clinical data announced for two programs, validating safety and efficacy of ARCUS in vivo gene editing4 - Initial safety data from Phase 1 ELIMINATE-B trial (PBGENE-HBV) presented at EASL, with ongoing clinical updates expected throughout 20254 - Regulatory clearance of IND from U.S. FDA to expand ELIMINATE-B trial and CTA approved by MHRA for UK expansion; PBGENE-HBV granted U.S. FDA Fast Track Designation4 - PBGENE-DMD program accelerated to be a first-in-class in vivo gene editing approach for Duchenne's Muscular Dystrophy, targeting IND/CTA in 2025 and clinical data in 20264 - Expected cash runway into second half of 2026, enabling data readouts from lead wholly-owned programs (PBGENE-HBV and PBGENE-DMD)4 Pipeline Update Precision BioSciences is advancing its wholly-owned and partnered programs, achieving key clinical and preclinical milestones Wholly Owned Portfolio The company is prioritizing PBGENE-HBV and PBGENE-DMD, showing promising clinical and preclinical data, while pausing PBGENE-3243 PBGENE-HBV (Viral Elimination Program) PBGENE-HBV demonstrated initial safety and HBsAg reduction, receiving U.S. IND and Fast Track, and U.K. CTA clearances - PBGENE-HBV is the first and only gene editing program in clinic designed to eliminate cccDNA and inactivate integrated HBV DNA for chronic Hepatitis B5 - Initial results from cohort 1 showed PBGENE-HBV was safe and well tolerated, with substantial reduction in Hepatitis B surface antigen (HBsAg) in two of three participants at the lowest dose6 - Received U.S. FDA clearance for Phase 1 trials and Fast Track designation in March/April 2025; U.K. MHRA also cleared for Phase 1 trial, making it the fifth country7 - Initial safety data presented at EASL Congress on May 8, 2025, supported pre-planned repeat dosing and dose escalation8 PBGENE-DMD (Muscle Targeted Excision Program) PBGENE-DMD showed significant functional improvement in preclinical models and is prioritized for 2025 IND/CTA filing - PBGENE-DMD addresses over 60% of DMD patients by excising exons 45-55 to restore a near-full length functional dystrophin protein9 - Preclinical data presented at MDA and ASGCT demonstrated significant, durable functional improvement and restoration of dystrophin protein across multiple muscles, including cardiac tissue, in a humanized DMD mouse model1011 - The program is prioritized as the second wholly-owned clinical program, targeting IND/CTA filing in 2025 and clinical data in 202612 PBGENE-3243 (Mutant Mitochondrial DNA Elimination Program) PBGENE-3243 showed preclinical efficacy in mutant mtDNA elimination but is paused to focus on lead programs - PBGENE-3243 is designed to specifically target and eliminate mutant m.3243G mitochondrial DNA, addressing the root cause of m.3243-associated mitochondrial disease13 - New preclinical data at ASGCT showed an ARCUS nuclease can eliminate mutant mitochondrial DNA and achieve therapeutically meaningful heteroplasmy shifts in vivo14 - Development of PBGENE-3243 is paused to accelerate PBGENE-DMD, with future development to be staged after PBGENE-HBV Phase 1 completion and PBGENE-DMD reaching the clinic15 Partnered Programs Partnered programs iECURE-OTC and PBGENE-NVS are progressing, with iECURE-OTC showing a complete clinical response iECURE-OTC (Gene Insertion Program) iECURE-OTC reported a complete clinical response in an OTC-deficient infant, with full enrollment expected in 2025 - iECURE-OTC (ECUR-506) is an ARCUS-mediated in vivo gene editing program for neonatal onset OTC deficiency, currently in a Phase 1/2 trial (OTC-HOPE)16 - Clinical efficacy and safety data from the first patient showed a complete clinical response from three months post-exposure to six months, with the patient now over one year old and eating age-appropriate protein levels16 - The OTC-HOPE study is ongoing in multiple countries, with enrollment expected to complete in 2025 and complete data anticipated in the first half of 202617 PBGENE-NVS (Gene Insertion Program) PBGENE-NVS, in collaboration with Novartis, aims for an in vivo gene insertion therapy for hemoglobinopathies - Precision continues to advance its gene editing program with Novartis (PBGENE-NVS) for hemoglobinopathies such as sickle cell disease and beta thalassemia18 - The program aims to insert a therapeutic transgene in vivo as a potential one-time transformative treatment, overcoming access disparities of ex vivo approaches18 First Quarter 2025 Financial Performance Precision BioSciences reported a net loss in Q1 2025 due to decreased revenue, with R&D and G&A expenses remaining stable Cash, Cash Equivalents, and Restricted Cash Cash, cash equivalents, and restricted cash totaled approximately $100 million, extending the cash runway into H2 2026 Cash Position | Metric | March 31, 2025 | December 31, 2024 | | :-------------------------------- | :------------- | :---------------- | | Cash, cash equivalents, and restricted cash | $99,789 thousand | $108,468 thousand | - Company expects cash runway into the second half of 2026, enabling data readouts from PBGENE-HBV and PBGENE-DMD through Phase 119 Revenues Total revenues significantly decreased to less than $0.1 million in Q1 2025 due to concluded collaboration agreements Revenue Comparison (Q1 2025 vs Q1 2024) | Metric | Q1 2025 | Q1 2024 | Change | | :------- | :------ | :------ | :----- | | Revenue | < $0.1M | $17.6M | -$17.6M | - Decrease due to revenue recognized from TG Therapeutics and Caribou Biosciences agreements in Q1 2024, decrease in Prevail agreement revenue (concluded April 2024), and decrease in Novartis Agreement revenue as preclinical work nears completion20 Research and Development Expenses R&D expenses slightly increased to $13.6 million in Q1 2025, driven by the advancement of the PBGENE-DMD program R&D Expenses Comparison (Q1 2025 vs Q1 2024) | Metric | Q1 2025 | Q1 2024 | Change | | :------------------------ | :-------- | :-------- | :----- | | Research and development | $13.6M | $13.3M | +$0.3M | - Increase primarily due to increased direct expense for PBGENE-DMD as the program advances towards the clinic21 General and Administrative Expenses G&A expenses rose slightly to $8.6 million in Q1 2025, primarily due to higher employee-related costs G&A Expenses Comparison (Q1 2025 vs Q1 2024) | Metric | Q1 2025 | Q1 2024 | Change | | :-------------------------- | :-------- | :-------- | :----- | | General and administrative | $8.6M | $8.4M | +$0.2M | - Increase primarily due to employee-related costs, partially offset by decreases in depreciation, amortization, taxes, and insurance22 Net Loss The company reported a net loss of $(20.6) million, or $(2.21) per share, in Q1 2025 Net (Loss) Income and EPS Comparison (Q1 2025 vs Q1 2024) | Metric | Q1 2025 | Q1 2024 | | :---------------------- | :-------- | :-------- | | Net (loss) income | $(20.6)M | $8.6M | | Net (loss) income per share (Basic) | $(2.21) | $1.70 | | Net (loss) income per share (Diluted) | $(2.21) | $1.70 | Company Information Precision BioSciences is a clinical-stage gene editing company leveraging its ARCUS® platform for in vivo therapies Company Profile Precision BioSciences is a clinical-stage gene editing company developing in vivo therapies for high unmet medical needs - Precision BioSciences is a clinical stage gene editing company utilizing its proprietary ARCUS® genome editing platform24 - Lead programs are PBGENE-HBV for chronic Hepatitis B and PBGENE-DMD for Duchenne muscular dystrophy, targeting diseases with high unmet need24 ARCUS® Genome Editing Platform The ARCUS® platform is a unique genome editing technology enabling gene insertion, elimination, and excision capabilities - ARCUS® platform differs from other technologies in its cutting mechanism, smaller size, and simpler structure24 - Capabilities include gene insertion (adding function), elimination (removing viral DNA), and excision (removing defective gene portions)25 Financial Statements Financial statements detail Q1 2025 performance, including a significant revenue decrease, operating loss, and balance sheet changes Statements of Operations Statements of Operations show a Q1 2025 net loss of $(20.6) million due to decreased revenue, with stable expenses Statements of Operations (In thousands, except share and per share amounts) | Metric | For the Three Months Ended March 31, 2025 | For the Three Months Ended March 31, 2024 | | :-------------------------------------- | :---------------------------------------- | :---------------------------------------- | | Revenue | $29 | $17,584 | | Research and development | $13,588 | $13,343 | | General and administrative | $8,553 | $8,428 | | Total operating expenses | $22,141 | $21,771 | | Operating loss | $(22,112) | $(4,187) | | Total other income | $1,547 | $12,775 | | Net (loss) income | $(20,565) | $8,588 | | Net (loss) income per share (Basic) | $(2.21) | $1.70 | | Net (loss) income per share (Diluted) | $(2.21) | $1.70 | | Weighted-average shares outstanding (Basic) | 9,292,066 | 5,060,978 | | Weighted-average shares outstanding (Diluted) | 9,292,066 | 5,063,406 | Balance Sheets Data Balance sheets data indicates a decrease in cash, total assets, and liabilities as of March 31, 2025 Balance Sheets Data (In thousands, except share amounts) | Metric | March 31, 2025 | December 31, 2024 | | :-------------------------------- | :------------- | :---------------- | | Cash, cash equivalents, and restricted cash | $99,789 | $108,468 | | Working capital | $69,710 | $80,009 | | Total assets | $124,411 | $136,388 | | Total liabilities | $75,074 | $79,995 | | Total stockholders' equity | $49,337 | $56,393 | | Common stock outstanding | 10,548,852 | 8,202,715 | Forward-Looking Statements This section outlines future expectations, beliefs, and assumptions, along with inherent risks and uncertainties that may impact actual results