On August 7, 2025 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for diseases with high unmet need, reported financial results for the second quarter ended June 30, 2025, and provided a business update (Press release, Precision Biosciences, AUG 7, 2025, View Source [SID1234654994]).
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"Our team continues to be very disciplined about executing our plans and is making strong progress advancing our clinical stage PBGENE-HBV program while rapidly advancing PBGENE-DMD toward the clinic," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "The early Phase 1 safety and efficacy data for PBGENE-HBV from the first cohort of the Phase 1 ELIMINATE-B trial establishes proof of activity for our novel gene editing approach for chronic Hepatitis B. Our data shows that we have a novel, safe and active drug in all patients treated with a durable effect in one third of patients reinforcing the mechanism of PBGENE-HBV to eliminate cccDNA. We are very pleased with the safety profile demonstrated in Cohorts 1 and 2 which has enabled the Data Monitoring Committee to endorse enrolling Cohort 3 this month to test the next higher dose. Concurrently, we are accelerating the development of our second program, PBGENE-DMD, and were proud to receive both Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (FDA), underscoring the significant unmet need for new therapeutic options for patients living with DMD."
"Given the unmet need, opportunity and enthusiasm for PBGENE-HBV and PBGENE-DMD, we are taking proactive steps to invest fully in these two programs while extending our expected cash runway to the second half of 2027 through a significant reduction in our non-program related annual operating expenses. These actions are expected to enable commencement of a Phase 2 study for PBGENE-HBV and a potential pivotal trial for PBGENE-DMD. Our team remains committed to delivering transformative therapies in areas with significant unmet need and, with a longer cash runway, we believe we are now even better positioned to deliver the meaningful clinical data that is expected by patients and shareholders for both of our wholly-owned programs," added Mr. Amoroso.
Wholly Owned Portfolio
PBGENE-HBV (Viral Elimination Program): PBGENE-HBV is Precision’s wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to be a potentially curative treatment for chronic Hepatitis B infection. PBGENE-HBV is the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate the root cause of chronic Hepatitis B, cccDNA, while inactivating integrated HBV DNA. The ELIMINATE-B trial is investigating PBGENE-HBV at multiple ascending dose levels with three dose administrations per dose level in patients with chronic Hepatitis B.
On August 6, 2025, Precision announced Phase 1 safety and efficacy data for Cohort 1, the lowest dose level in the ELIMINATE-B trial. Cohort 1 consisted of three patients each of whom received three planned administrations of 0.2 mg/kg of PBGENE-HBV dosed approximately eight weeks apart. The primary objective is to characterize the safety of PBGENE-HBV. PBGENE-HBV was well-tolerated in all three patients in Cohort 1. Across Cohort 1, no patient experienced above a Grade 2 treatment-related adverse event, a serious adverse event, or dose-limiting toxicity. No clinically significant lab abnormalities were observed, including liver enzymes and platelets.
PBGENE-HBV demonstrated a substantial HBsAg reduction in all three patients in Cohort 1 with best response reductions of 56%, 69% and 47% compared to baseline HBsAg levels (ranging between 562-11,813 IU/mL) in patients one, two and three, respectively. One of three patients (33%) in Cohort 1 achieved a durable HBsAg reduction of approximately 50% from baseline that was maintained as of the data cutoff-date (July 28, 2025), which was seven months after initial dosing. This data provides evidence of the ability of PBGENE-HBV to drive a durable antiviral response by editing the viral DNA at the source of chronic Hepatitis B infection. There was no association between baseline HBsAg and efficacy in Cohort 1.
Precision also announced initial safety data from Cohort 2 (0.4 mg/kg) in the ELIMINATE-B study. As of the data cutoff, one patient received three dose administrations with two weeks of follow-up, and two patients received one dose administration with four weeks of follow-up. In these patients, no adverse events above Grade 2 were observed. No serious adverse events or dose limiting toxicities were observed, and no cumulative adverse effects were observed. There were no clinically significant elevations of liver transaminases. One additional patient did not complete their dose due to a transient infusion-related serious adverse event that resolved within minutes. The Data Monitoring Committee determined that this transient reaction was not dose-related or dose-limiting. Given the favorable safety profile of Cohorts 1 and 2, the Data Monitoring Committee recently recommended initiation of Cohort 3.
The Company is on track to complete dosing of all three patients across all dose administrations in Cohort 2 and commence dosing Cohort 3. The Company expects to provide a data update later in 2025.
PBGENE-DMD (Muscle Targeted Excision Program): PBGENE-DMD is Precision’s development program for the treatment of DMD. DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the U.S. alone. There are currently no approved therapies that can drive durable and significant functional improvements over time. PBGENE-DMD is designed to improve function for more than 60% of patients afflicted with DMD by employing two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene. The aim of this approach is to restore a near-full length functional dystrophin protein within the body that more closely resembles normal dystrophin as opposed to synthetic, truncated dystrophin approaches with minimal functional benefit.
The FDA granted PBGENE-DMD Rare Pediatric Disease designation in June 2025 for the treatment of DMD, highlighting the significant unmet need for new therapeutic options. This was followed in July 2025 by the FDA granting PBGENE-DMD Orphan Drug Designation, re-enforcing the need for new and better treatments. With the Rare Pediatric Disease designation, Precision may be eligible to receive a Priority Review Voucher upon FDA approval of PBGENE-DMD.
In preclinical data presented at the ASGCT (Free ASGCT Whitepaper) annual meeting in May 2025, PBGENE-DMD demonstrated significant and durable functional improvement in a humanized DMD mouse model. Following AAV delivery, PBGENE-DMD restored the body’s ability to produce a functional dystrophin protein broadly across multiple muscle types, including cardiac and skeletal muscles. Over the course of nine months, mice treated with PBGENE-DMD showed increased dystrophin protein expression resulting in substantial and sustained functional muscle improvement. In addition, PBGENE-DMD-edited dystrophin mRNA transcript in muscle satellite stem cells, which are progenitor cells for new muscle cells, supports the potential for long-term durability.
In July 2025, Precision announced new preclinical data building upon previous data shared at the ASGCT (Free ASGCT Whitepaper) annual meeting. These data demonstrated that PBGENE-DMD produced a three-fold increase in dystrophin-positive muscle cells between three and nine months in the quadricep, gastrocnemius (calf), heart, and diaphragm. In the gastrocnemius specifically, up to 85% of cells were dystrophin-positive, indicating a high degree of productive gene editing. This broad increase in dystrophin-positive cells combined with the increased dystrophin protein detected in tissues further validates the improved muscle function that was observed over time and may be attributable to edited satellite cells.
Precision is advancing the final U.S. investigational new drug (IND)-enabling toxicology studies with an anticipated IND and/or clinical trial application (CTA) filing targeted by the end of 2025 with initial clinical data expected in 2026.
PBGENE-3243 (Mutant Mitochondrial DNA Elimination Program): PBGENE-3243 is a first-of-its-kind potential treatment for m.3243-associated mitochondrial disease that is designed to specifically target and eliminate mutant m.3243G mitochondrial DNA, thereby eliminating the root cause of the disease. Precision has paused development of PBGENE-3243 to prioritize its two lead programs, PBGENE-HBV and PBGENE-DMD.
Partnered In Vivo Gene Editing Programs
iECURE-OTC (Gene Insertion Program): Led by iECURE, ECUR-506 is an ARCUS-mediated in vivo gene editing program currently in a first-in-human Phase 1/2 trial (OTC-HOPE) evaluating ECUR-506 as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. Preliminary data from the study presented at ASGCT (Free ASGCT Whitepaper) in May demonstrated a complete clinical response from three months post-exposure to the end of study at six months, as defined by the study protocol. The patient is now more than one year of age and is eating appropriate levels of protein for a child of his age. iECURE has reported that a second infant with severe OTC deficiency was dosed in the first half of 2025.
The OTC-HOPE study is ongoing in the U.K., the U.S., Australia, and Spain, and iECURE expects to complete enrollment in 2025 and anticipates complete data from the trial in the first half of 2026.
PBGENE-NVS (Gene Insertion Program): Precision continues to advance its gene editing program with Novartis to develop a custom ARCUS nuclease for patients with hemoglobinopathies, such as sickle cell disease and beta thalassemia. The collaborative intent is to insert, in vivo, a therapeutic transgene as a potential one-time transformative treatment administered directly to the patient to overcome disparities in patient access to treatment with other therapeutic technologies, including those that are targeting an ex vivo gene editing approach.
Non-Core Ex Vivo Programs
Azer-Cel (azercabtagene zapreleucel allogeneic CAR T treatment for cancer): Imugene Limited, Precision’s clinical stage partner developing azer-cel for oncology indications, announced updated clinical data in July 2025. In the updated data in patients diagnosed with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), two additional patients achieved a Complete Response and three additional patients achieved a Partial Response. Imugene reported that, as a result of the new data, the best overall response rate for azer-cel in DLBCL reached 75% and the Complete Response rate reached 55%. Based on the updated response rate and maturing durability data, as well as having been awarded FDA Fast Track Designation for DLBCL, Imugene expects to request an end of Phase 1 meeting with the FDA in the fourth quarter of 2025 to present the data and discuss designs for a pivotal/registrational trial for azer-cel.
Other Announcements
Mark Sulkowski, M.D., Professor of Medicine at the Johns Hopkins University School of Medicine and renowned expert in hepatic and infectious diseases has expanded his advisory role with Precision BioSciences. In the newly created role, Head Clinical Development Advisor, Dr. Sulkowski will work closely with Precision’s leadership and cross-functional teams to support clinical strategy across the development lifecycle for the Company’s on-going PBGENE-HBV Phase 1 clinical trial as well as initiation of later stage trials. His advisory role will focus on optimizing clinical trials, including translational integration, and aligning scientific rationale with regulatory objectives.
Quarter Ended June 30, 2025 Financial Results
"As Precision advances the ELIMINATE-B clinical trial and prepares to file an IND and/or CTA for the PBGENE-DMD program we have been closely managing our operating costs. Cost management is evident in our decision to pause development on PBGENE-3243 in the second quarter and is reflected in a $3.9 million reduction in our second quarter total operating expenses as compared to the same period last year," said Alex Kelly, Chief Financial Officer of Precision BioSciences. "We have also extended our expected cash runway to the second half of 2027 to enable meaningful clinical data readouts for PBGENE-HBV and PBGENE-DMD. In July 2025, we initiated an operating efficiency program, including reductions in early research, manufacturing and general & administrative operating expenses which are aimed at reducing our annual cash operating expenses in each of 2026 and 2027 by approximately $25 million compared to the 2025 annual cash expense level."
"In addition to significantly reducing our operating expenses, Precision will continue to pursue less dilutive sources of cash to even further extend the cash runway, including business development collaborations for future or deprioritized ARCUS programs as well as opportunities to monetize non-core program royalties and milestones," added Mr. Kelly.
Cash, Cash Equivalents, and Restricted Cash: As of June 30, 2025, Precision had approximately $84.8 million in cash, cash equivalents, and restricted cash. Based on its expected cash runway, Precision believes it is sufficiently capitalized to reach important milestones for both programs, including commencement of a Phase 2 study for PBGENE-HBV and a potential pivotal trial for PBGENE-DMD. The Company expects existing cash and cash equivalents, upfront and potential near-term cash from CAR T transactions, along with expected operating efficiencies, operational receipts, and availability of Precision’s at-the-market (ATM) facility to extend Precision’s cash runway into the second half of 2027.
Revenues: Total revenues for the quarter ended June 30, 2025, were less than $0.1 million, as compared to $49.9 million for the quarter ended June 30, 2024. The decrease was expected and primarily the result of $48.2 million of non-cash revenue recognized in the prior period which represented all remaining deferred revenue related to the Prevail Therapeutics Agreement at its conclusion in April 2024 under generally accepted accounting principles. The upfront cash from this collaboration was received and recorded on the balance sheet in January 2021.
Research and Development Expenses: Research and development expenses were $12.8 million for the quarter ended June 30, 2025, as compared to $17.2 million for the quarter ended June 30, 2024. The decrease of $4.4 million was primarily due to decreases in PBGENE-HBV direct expenses due to lower manufacturing and toxicology expenses as the program transitioned to the clinic in the fall of 2024. PBGENE-3243, which has been paused, and other research related expenses also decreased in the comparative period, offset by an increase in PBGENE-DMD expenses as the program was accelerated in the second quarter of 2025.
General and Administrative Expenses: General and administrative expenses were $9.1 million for the quarter ended June 30, 2025, as compared to $8.5 million for the quarter ended June 30, 2024 as an increase in employee-related costs, including non-cash employee-related costs, offset a decrease in other general and administrative expenses.
Net Loss: Net loss was $23.5 million, or ($2.13) per share (basic and diluted), for the quarter ended June 30, 2025. Net income was $32.7 million, or $4.70 per share (basic) and $4.67 per share (diluted), for the quarter ended June 30, 2024.