Company Overview
Precision BioSciences, Inc. (NASDAQ:DTIL) is a clinical-stage gene editing company at the forefront of developing transformative in vivo therapies using its proprietary ARCUS platform. With a focus on tackling some of the most challenging genetic diseases, Precision BioSciences is leveraging ARCUS’s unique capabilities to unlock new frontiers in gene editing and deliver on the promise of precision medicine.
Company History
Founded in 2006 and based in Durham, North Carolina, Precision BioSciences has steadily built an impressive portfolio of in vivo gene editing programs targeting a diverse range of indications, from chronic hepatitis B to mitochondrial diseases and hemoglobinopathies. The company’s unwavering commitment to innovation has positioned it as a leader in the rapidly evolving gene editing landscape. In its early years, Precision BioSciences devoted substantial resources to research and development activities, recruiting talent, and establishing its intellectual property portfolio.
Throughout its history, the company has faced numerous challenges common to early-stage biotechnology companies. Like many in the industry, Precision BioSciences has encountered obstacles in preclinical studies and early clinical trials as it sought to demonstrate the safety and efficacy of its product candidates. Additionally, the company has had to navigate a complex and evolving regulatory landscape for genome editing technologies. Despite these hurdles, Precision BioSciences has persevered, drawing on its internal expertise and collaborating with external partners to further the development of its pipeline.
A significant milestone in the company’s journey came in 2019 when Precision BioSciences completed its initial public offering, providing additional capital to fund its research and development efforts. This influx of resources has allowed the company to continue building out its team and infrastructure, acquiring new talent and facilities to support its growing operations. Precision BioSciences has also expanded its partnerships, working with pharmaceutical companies and other organizations to leverage its ARCUS platform across a range of therapeutic areas.
ARCUS Platform
At the heart of Precision BioSciences’ success is the ARCUS platform, a novel genome editing technology that sets it apart from traditional approaches. ARCUS utilizes a proprietary, sequence-specific DNA-cutting enzyme derived from a natural homing endonuclease, enabling precise and efficient gene edits. Unlike other gene editing tools, ARCUS boasts a relatively small size and simpler structure, potentially allowing for more versatile delivery and broader therapeutic applications.
Pipeline and Programs
Precision BioSciences’ pipeline showcases the versatility of the ARCUS platform, with programs targeting a variety of disease targets and therapeutic strategies. The company’s lead program, PBGENE-HBV, is designed to address the unmet need in chronic hepatitis B, a condition that affects an estimated 296 million people globally. Leveraging ARCUS’s ability to precisely eliminate covalently closed circular DNA (cccDNA) and inactivate integrated hepatitis B virus (HBV) DNA, PBGENE-HBV holds the promise of delivering a functional cure for this persistent viral infection.
In October 2024, Precision BioSciences announced that it had received the first approval of a clinical trial application (CTA) to initiate a phase 1 trial for PBGENE-HBV, marking a significant milestone in the program’s development. The company is currently recruiting patients for the ELIMINATE-B study, a phase 1 dose escalation and expansion trial designed to assess the safety and efficacy of PBGENE-HBV in reducing key viral biomarkers. Precision BioSciences expects to submit an investigational new drug (IND) application for PBGENE-HBV in the United States in 2025, further advancing its efforts to bring this potentially transformative therapy to patients.
Beyond PBGENE-HBV, Precision BioSciences’ pipeline showcases the breadth of its gene editing capabilities. The company’s PBGENE-3243 program, formerly known as PBGENE-PMM, is a first-of-its-kind potential treatment for m.3243 associated mitochondrial disease, a debilitating genetic disorder affecting an estimated 20,000 individuals in the United States alone. Leveraging ARCUS’s ability to selectively eliminate mutant mitochondrial DNA while preserving healthy mitochondria, PBGENE-3243 aims to shift heteroplasmy and restore cellular function. The company plans to submit an IND or CTA application for this program in 2025.
Strategic Collaborations
Precision BioSciences’ commitment to innovation extends beyond its internal programs, as evidenced by its strategic collaborations. In June 2022, the company entered into an exclusive in vivo gene editing research and development collaboration and license agreement with Novartis Pharma AG. This partnership is focused on developing a custom ARCUS nuclease to insert a therapeutic transgene at a safe harbor location in the genome, potentially paving the way for transformative one-time treatments for diseases such as certain hemoglobinopathies.
In January 2024, Precision BioSciences granted TG Therapeutics exclusive rights to develop and commercialize its allogeneic CAR T therapy, azer-cel, for autoimmune disease and other non-oncology indications. The company received upfront and potential near-term payments valued at up to $17.5 million, as well as the potential for additional milestone payments up to $288.6 million and royalties on net sales.
In August 2023, Precision BioSciences divested its CAR T infrastructure and licensed its azer-cel program for oncology applications to Imugene. The company is eligible to receive milestone payments of up to $206 million upon successful development and regulatory milestones, as well as royalties on net sales of licensed products.
Additionally, in February 2024, the company granted Caribou Biosciences, Inc. a non-exclusive license to use its foundational cell therapy patent families for CRISPR-based therapies.
Financials and Liquidity
Precision BioSciences’ financial position remains strong, with a cash balance of $98.8 million as of September 30, 2024. This, combined with expected operational receipts, including upfront and potential near-term consideration from licensees, and the availability of the company’s at-the-market (ATM) facility, is expected to fund Precision BioSciences’ operations into the second half of 2026. The company anticipates this runway will be sufficient to achieve first-in-human phase 1 clinical data for two of its wholly-owned in vivo gene editing programs.
For the fiscal year 2023, Precision BioSciences reported revenue of $48.73 million and a net loss of $42.53 million. The company’s operating cash flow for 2023 was -$84.11 million, with a free cash flow of -$86.39 million.
In the most recent quarter (Q3 2024), the company reported revenue of $576,000 and a net loss of $16.43 million. The operating cash flow for Q3 2024 was -$5.91 million, with a free cash flow of -$5.95 million. The decrease in revenue in Q3 2024 compared to the prior year was primarily due to a $7 million decrease in revenue recognized under the Novartis Agreement as the company nears completion of its pre-clinical workplan, as well as a $5.5 million decrease in revenue recognized under the terminated Prevail Agreement.
Precision BioSciences’ liquidity position remains solid, with a debt-to-equity ratio of 0.36. As of September 30, 2024, the company had $98.75 million in cash and cash equivalents, along with $22.58 million in restricted cash. In July 2024, the company terminated its previous revolving credit facility and entered into a $22.5 million term loan agreement with Banc of California. The company’s current ratio and quick ratio both stand at 9.22, indicating a strong ability to meet short-term obligations.
Outlook and Future Prospects
Despite the challenges faced by the broader biopharmaceutical industry, Precision BioSciences has demonstrated its resilience and ability to navigate uncertain waters. The company has continued to make steady progress, advancing its pipeline, strengthening its leadership team, and forging strategic partnerships to unlock the full potential of the ARCUS platform.
As Precision BioSciences continues to push the boundaries of gene editing, investors and patients alike will be closely monitoring the company’s journey. With a robust pipeline, a talented team, and a transformative technology at its core, Precision BioSciences is poised to make a significant impact in the field of precision medicine, offering hope to those affected by some of the most complex and debilitating genetic disorders.
Disclaimer: This article is for informational purposes only. It does not constitute financial, legal, or other types of advice. While every effort has been made to ensure the accuracy of the information presented here, the author and the publisher do not make any guarantees about the completeness, reliability, and accuracy of this information.