Precision BioSciences Q3 2024 Financial Results and Business Update

Precision BioSciences, Inc., a clinical-stage gene editing company based in Durham, N.C., has released its financial results for the third quarter of 2024 and provided updates on its business activities. The company is leveraging its proprietary ARCUS® platform to develop gene editing therapies, specifically targeting chronic hepatitis B (HBV) and mitochondrial diseases.

Precision BioSciences announced that it has received clearance for its first clinical trial application (CTA) for PBGENE-HBV, its lead program aimed at treating chronic hepatitis B. This milestone marks a significant step for the company as it transitions to a clinical-stage gene editing organization. The team is preparing to dose patients and is also seeking additional regulatory approvals globally to expedite the enrollment process for the PBGENE-HBV phase 1 trial. PBGENE-HBV is unique in that it targets the elimination of cccDNA and the inactivation of integrated HBV genomes, which are the root causes of viral persistence in chronic hepatitis B.

Looking forward, Precision expects to report phase 1 data for PBGENE-HBV throughout 2025. Additionally, the company is working on another wholly owned gene editing program, PBGENE-3243, which targets m.3243-associated mitochondrial disease. This program aims to treat mitochondrial diseases by targeting mutant mitochondrial DNA, allowing healthy mitochondrial DNA to repopulate and improve cellular function. Precision plans to submit an investigational new drug (IND) application or a CTA for this program in 2025.

In October 2024, Precision received CTA approval for PBGENE-HBV in Moldova, where investigators have significant experience in early- and mid-stage hepatitis B clinical trials. The preclinical safety data for PBGENE-HBV indicate that it can specifically target and eliminate hepatitis B cccDNA without affecting other genes in the human genome, and non-human primate data showed that the treatment was well-tolerated across multiple doses.

The PBGENE-3243 program has shown promising results in preclinical studies, including its ability to localize exclusively to mitochondria and avoid off-target editing in the nuclear genome. Precision presented additional data from this program at the UMDF Conference earlier this year, highlighting improvements in mitochondrial function.

Precision also reclaimed rights to three programs from its collaboration with Prevail Therapeutics Inc. These programs include PBGENE-DMD for Duchenne Muscular Dystrophy, PBGENE-LIVER for gene insertion in liver cells, and PBGENE-CNS for targeting central nervous system diseases. The company is currently assessing these programs for internal development or potential partnerships.

In its partnered programs, iECURE is leading the development of ECUR-506, an ARCUS-mediated gene editing program for treating neonatal onset ornithine transcarbamylase (OTC) deficiency. Precision is also collaborating with Novartis to advance a gene editing program for hemoglobinopathies like sickle cell disease and beta thalassemia.

Precision BioSciences reported its financial results for the quarter ended September 30, 2024. The company had $121.3 million in cash, cash equivalents, and restricted cash. Total revenue for the quarter was $0.6 million, a significant decrease from $13.1 million in the same period in 2023, primarily due to reduced revenue from agreements with Novartis and Prevail. Research and development expenses were $13.1 million, down from $15.9 million in 2023, while general and administrative expenses decreased to $8.8 million from $9.6 million. The net loss for the quarter was $16.4 million, compared to $8.1 million in 2023.

Precision has strengthened its leadership team with the appointment of Dr. Murray Abramson as Senior Vice President, Head of Clinical Development, and John Fry as Strategic Clinical Advisor, Hepatitis. The company also announced the retirement of Alan List, MD, who will now serve on the Scientific Advisory Board as a Clinical Consultant.

Lastly, Precision amended its loan and security agreement with Banc of California, securing a $22.5 million term loan with a maturity date of June 30, 2027. The company believes that its current cash runway is sufficient to support its programs through their next phases of development into 2026.