Business Context and Reporting Period
Precision BioSciences, Inc. (DTIL) filed this Form 8-K on December 11, 2021, to report program updates for its allogeneic chimeric antigen receptor T cell (CAR T) therapy pipeline. The updates were based on data presented at the 63rd American Society of Hematology (ASH) Annual Meeting.
Key Financial Metrics
Liquidity: As of November 30, 2021, the Company held approximately $152 million in cash and cash equivalents.
Capital Resources: Management expects existing cash, expected operational receipts, and available credit to be sufficient to fund operating expenses and capital expenditures into 2023.
Revenue and Profit: The filing text does not provide specific values for revenue, profit, cash flow, margins, or debt for this reporting period.
Material Changes and Clinical Updates
The filing details significant clinical progress across three primary candidates:
- PBCAR0191 (CD19-targeted for R/R NHL and B-ALL):
- In a cohort of 22 heavily pre-treated subjects, the therapy yielded a 73% overall response rate and a 59% complete response rate at a dose of 3 x 10^6 cells/kg.
- Safety profile included no Grade 3 or higher cytokine release syndrome (CRS) and one Grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS) that resolved within 72 hours.
- Relapsed Autologous CAR T Population: Among six subjects who previously failed autologous CAR T therapy, 100% responded, and 66% achieved a complete response. More than half experienced a longer duration of response with PBCAR0191 than with their prior treatment.
- PBCAR19B (Stealth Cell for R/R NHL):
- Phase 1 study is actively enrolling. The first three subjects have been dosed at Dose Level 1 (2.7 x 10^8 CAR T cells).
- Initial results are expected in mid-2022.
- PBCAR269A (BCMA-targeted for R/R Multiple Myeloma):
- Monotherapy data from 14 patients showed no Grade 3 or higher CRS or ICANS but demonstrated response rates not comparable to autologous CAR T profiles.
- The Company is now enrolling subjects in a combination cohort with nirogacestat (a gamma secretase inhibitor) to improve the therapeutic index.
- Initial combination data is expected in mid-2022.
Guidance, Outlook, and Risks
Outlook: The Company is actively enrolling additional patients in the relapse setting for PBCAR0191 to further evaluate its activity as a potential salvage treatment. Initial data for PBCAR19B and the PBCAR269A combination therapy are anticipated in mid-2022.
Risks and Contingencies: The filing includes standard forward-looking statement disclaimers, noting that future expectations involve known and unknown risks. Specific risks are referenced in the Company's Form 10-Q for the period ended September 30, 2021. One infectious death at Day 54 in the PBCAR0191 study was deemed possibly related to treatment.
Investor Verification Checklist
- Verify the durability of the 100% response rate in the six patients who previously failed autologous CAR T therapy.
- Monitor the mid-2022 data release for PBCAR19B to confirm safety and efficacy at higher dose levels.
- Assess the efficacy of the PBCAR269A combination with nirogacestat to determine if it achieves a therapeutic index comparable to autologous CAR T.
- Review the Company's cash burn rate to confirm the runway extends into 2023 as projected.
- Check for any updates on the one infectious death reported in the PBCAR0191 study.