Precision BioSciences — Q3 2026 Earnings Summary

Financial Performance:

• Reported no revenue for the quarter ended September 30, 2026, compared to less than $0.1 million in the same period in 2025, which was derived from the Novartis Agreement.
• Research and development expenses were $12.7 million for Q3 2026, a decrease from $13.4 million in Q3 2025, driven by the completion of pre-IND research for PBGENE-HBV and PBGENE-DMD, partially offset by increased clinical program costs for ELIMINATE-B.
• General and administrative expenses increased to $7.7 million in Q3 2026 from $7.3 million in Q3 2025.
• Total other income was $12.8 million for Q3 2026, a significant increase from a total other expense of $1.1 million in Q3 2025, primarily due to a non-cash gain of $12.7 million on the change in fair value of the warrant liability.
• Net loss was $7.6 million, or $0.28 per share (basic and diluted), for Q3 2026, compared to a net loss of $21.8 million, or $1.84 per share, in Q3 2025; the reduction is attributed to the non-cash warrant liability gain.
• Cash, cash equivalents, and restricted cash totaled $108.0 million as of September 30, 2026, down from $137.2 million as of December 31, 2025.
• Working capital was $78.3 million as of September 30, 2026, compared to $109.8 million as of December 31, 2025.
• Total assets were $120.4 million as of September 30, 2026, compared to $154.4 million as of December 31, 2025.
• Total liabilities increased to $68.6 million as of September 30, 2026, from $62.2 million as of December 31, 2025.
• Total stockholders' equity was $51.8 million as of September 30, 2026, compared to $92.2 million as of December 31, 2025.
• Common stock outstanding increased to 27,893,065 shares as of September 30, 2026, from 24,088,425 shares as of December 31, 2025.

Guidance and Future Outlook:

• The company expects its current cash position and operating discipline to provide a runway through 2028, enabling key clinical data readouts for PBGENE-HBV and PBGENE-DMD.
• Initial safety data for the FUNCTION-DMD study is targeted for year-end 2026, with efficacy data expected at distinct timepoints in H1 and H2 2027.
• The company plans to present late-breaking oral data for the ELIMINATE-B trial at AASLD's The Liver Meeting on November 8, 2026.
• Management expects to continue advancing the ELIMINATE-B trial by enrolling additional patients and expanding cohorts to support Phase 2 expansion dosing schedule selection.

Business Segments and Product Lines:

• PBGENE-HBV: Advanced the ELIMINATE-B global clinical trial by enrolling additional patients and expanding 0.4 mg/kg and 0.65 mg/kg cohorts; collecting biopsy and blood biomarker data to validate cccDNA elimination findings.
• PBGENE-DMD: Dosed the first patient in the Phase 1/2 FUNCTION-DMD study at Arkansas Children's Hospital in August 2026; multiple sites are active, including Washington University School of Medicine; the trial targets ambulatory DMD patients aged 2-7 with mutations between exons 45-55.
• ECUR-506 (iECURE): Phase 1/2 dosing completed across all cohorts in the OTC-HOPE trial; preliminary data showed a 57% reduction in annualized hyperammonemic events (HAEs) and a 74% reduction in annualized hyperammonemic crises (HACs); generally well tolerated with no unexpected safety events.
• Azer-cel (MS): Evaluated by TG Therapeutics in a Phase 1 trial for progressive multiple sclerosis; 89% of evaluable participants showed stable or improved disability scores at Day 90; no high-grade CAR-T associated toxicities observed.
• Azer-cel (Cancer): Evaluated by Imugene Limited for diffuse large B-cell lymphoma; received FDA written guidance on the registrational pathway; overall response rates reached 81% in cohort 2 and 86% in cohort 3.

Market and Competitive Landscape:

• PBGENE-HBV is the only clinical stage program targeting and eliminating cccDNA, the sole source of HBV replication, and the first in vivo gene elimination approach to prospectively employ repeat lipid nanoparticle (LNP) administration.
• PBGENE-DMD is the first clinical stage gene editing approach aimed at restoring near full-length dystrophin protein from the native gene in DMD patients.
• PBGENE-DMD received Orphan Drug Designation in July 2025, Fast Track designation in February 2026, and is eligible for a Priority Review Voucher via the Rare Pediatric Disease program.
• PBGENE-HBV has been granted Fast Track designation by the FDA.

Risks and Challenges:

• Forward-looking statements highlight risks including the ability to become profitable, procure sufficient funding, and manage anticipated cash runway.
• Risks include potential delays in clinical trial initiation, cost, timing, and results, as well as the possibility that product candidates may not work as intended or cause undesirable side effects.
• The company faces risks related to regulatory approval, market acceptance, competition from other genome-editing technologies, and dependence on its ARCUS technology.
• Potential risks include intellectual property litigation, manufacturing problems, and the inability to obtain or maintain regulatory designations or approvals.

Management Commentary and Tone:

• CEO Michael Amoroso stated the company entered 2026 with a clear plan to deliver milestones and made significant progress executing against that plan.
• Management expressed excitement about the upcoming ELIMINATE-B data presentation and the milestone of dosing the first patient in the FUNCTION-DMD trial.
• Amoroso noted broad support and excitement from patient families, advocacy groups, and physicians regarding the PBGENE-DMD approach.
• Management emphasized remaining on track to deliver data updates from both programs by year-end, supported by a financial position providing runway through 2028.

Other Key Points:

• The company has an at-the-market facility available to fund its cash runway through 2028.
• The PBGENE-DMD program targets up to 60% of boys living with DMD by focusing on mutations between exons 45 and 55.
• The OTC-HOPE trial for ECUR-506 is ongoing in the U.K., the U.S., Australia, and Spain.
• The azer-cel cancer program received clear FDA alignment on dosing, patient population, endpoints, and manufacturing readiness for a pivotal study.
• The company's ARCUS platform utilizes smaller, simpler nucleases for gene elimination, excision, and insertion.