Precision BioSciences Q1 2024 Financial Results and Business Update

Precision BioSciences, Inc., a leading gene editing company using its proprietary ARCUS® platform, announced its financial results for the first quarter ending March 31, 2024, and provided significant business updates. The company's primary focus for the year is the advancement of its wholly owned gene editing programs for the Hepatitis B virus (HBV) and primary mitochondrial myopathy (PMM), targeting clinical trial applications in 2024 and 2025 respectively.

CEO Michael Amoroso highlighted the company's progress in gene editing, emphasizing the advancement of the first in vivo ARCUS gene editing program for OTC deficiency into clinical trials through a partnership with iECURE. This milestone underscores the potential of ARCUS in gene insertion and sets regulatory precedents for global markets.

Precision BioSciences has regained control of three preclinical programs from Prevail Therapeutics, showcasing significant promise across various therapeutic domains. These programs leverage ARCUS' unique attributes and are under consideration for internal development or re-partnership. The company plans to provide updates upon finalizing its strategy.

Financially, Precision BioSciences bolstered its cash reserves by monetizing CAR T assets through licensing agreements with TG Therapeutics and Caribou Biosciences. A $40 million common stock offering has extended the financial runway into the second half of 2026, supporting continued program development.

Among the lead programs, PBGENE-HBV is being developed as a potential curative treatment for chronic Hepatitis B. The program aims to eliminate cccDNA and inactivate integrated HBV DNA. Precision has received pre-IND regulatory feedback from the U.S. FDA and other agencies, aligning its clinical strategy for an anticipated IND/CTA submission in 2024.

Similarly, PBGENE-PMM targets m.3243-associated primary mitochondrial myopathy by editing and eliminating mutant mitochondrial DNA. This program has shown high specificity in preclinical studies, with plans for an IND/CTA submission in 2025.

In addition to the wholly owned programs, Precision is evaluating three newly returned programs for internal development or new partnerships. These include PBGENE-DMD, an approach for Duchenne Muscular Dystrophy; PBGENE-LIVER, focusing on liver gene insertion; and PBGENE-CNS, targeting central nervous system diseases.

Partnered programs also show progress, with iECURE leading the advancement of ECUR-506 for neonatal onset ornithine transcarbamylase (OTC) deficiency. This first-in-human Phase 1/2 trial is underway, with initial data expected by late 2024 or 2025. Precision continues its collaboration with Novartis to develop a gene editing treatment for hemoglobinopathies, aiming for a transformative, one-time treatment.

Precision BioSciences also completed significant business transactions, including a licensing deal with TG Therapeutics for the development of Azercabtagene Zapreleucel (azer-cel) in autoimmune diseases and a non-exclusive patent license agreement with Caribou Biosciences for CRISPR-based therapies.

Financially, the company reported $17.6 million in revenues for Q1 2024, a substantial increase from the previous year, driven by cell therapy transactions. Research and development expenses rose to $13.3 million due to increased investment in gene editing programs, while general and administrative expenses decreased to $8.4 million. The net income for the quarter was $8.6 million, contrasting with a net loss in the same period last year.

Precision BioSciences had approximately $137.8 million in cash and cash equivalents as of March 31, 2024. This financial stability is expected to support the development of its gene editing programs through Phase 1 readouts and the initiation of a new program.

Overall, Precision BioSciences remains focused on leveraging its ARCUS platform to develop groundbreaking gene editing therapies, with ongoing efforts to advance its pipeline and secure further financial and strategic partnerships.