Business Context and Reporting Period
Precision BioSciences, Inc. (DTIL) filed a Form 8-K on September 9, 2021, to disclose a Regulation FD event and other material developments. The company is an emerging growth company focused on in vivo gene editing using its proprietary ARCUS platform. The filing details a strategic R&D event and a new collaboration agreement.
Key Financial Metrics
The filing provides limited financial data, noting that the information is unaudited and does not present a full understanding of the company's financial condition.
- Cash and Cash Equivalents: Approximately $167 million as of August 31, 2021.
- Liquidity Outlook: Management expects existing cash to fund planned operations into 2023.
- Revenue, Profit, and Debt: The filing text does not provide clear values for revenue, net income, operating margins, or debt levels.
Material Changes and Strategic Developments
The primary material change is the announcement of a license and collaboration agreement with iECURE, a mutation-agnostic in vivo gene editing company co-founded by James M. Wilson, M.D., Ph.D.
- iECURE Collaboration: iECURE will advance Precision's PBGENE-PCSK9 candidate for familial hypercholesterolemia (FH) into Phase 1 clinical studies, with a Clinical Trial Application (CTA) expected as early as 2022.
- Consideration: Precision will receive an equity stake in iECURE and is eligible for milestone and royalty payments on sales of iECURE products developed with ARCUS.
- Rights Retention: Precision retains rights to PBGENE-PCSK9 for FH and all genetic indications except those licensed to iECURE.
Guidance, Outlook, and Pipeline Updates
Management outlined a clinical development strategy expecting three preclinical programs to advance to IND/CTA filings within the next three years:
- PBGENE-PCSK9 (FH): CTA filing expected as early as 2022 via iECURE.
- PBGENE-PH1 (Primary Hyperoxaluria Type 1): IND application expected in 2023 using LNP delivery.
- PBGENE-HBV (Chronic Hepatitis B): IND/CTA expected in 2024 using LNP delivery.
Preclinical Data Highlights:
- Gene Insertion: ARCUS demonstrated higher efficiency than CRISPR for inserting a Factor IX transgene into the PCSK9 locus in non-human primates (NHPs).
- HBV: ARCUS reduced HBV S-antigen expression by up to 95% in primary human hepatocytes and mouse models.
- Mitochondrial Editing: mitoARCUS converted hybrid cells to >99% wild-type mitochondrial genomes and depleted mutant mtDNA in mouse models without detectable nuclear off-target editing.
- FH: NHP data showed up to 82% reduction in PCSK9 levels and 62% reduction in LDL levels over three years.
- PH1: NHP data showed ~98% reduction in HAO1 mRNA and protein after a single AAV administration.
- DMD: Collaboration with Lilly continues on PBGENE-DMD, targeting exons 45-55 of the dystrophin gene.
Investor Verification Checklist
- Verify the specific terms of the equity stake and royalty percentages in the iECURE agreement.
- Confirm the timeline for the iECURE-led Phase 1 study for PBGENE-PCSK9.
- Review the full risk factors in the most recent Form 10-Q regarding the ARCUS platform and clinical trial execution.
- Monitor the company's cash burn rate to validate the runway into 2023.
- Assess the regulatory pathway for mitochondrial genome editing, a novel therapeutic approach.