10-Q: Precision BioSciences Reports Q1 2025 Financial Results, Highlights Progress in In Vivo Gene Editing Programs

Sentiment:

Quarterly Report 15 May 2025 7:34 AM

📋All filings for Precision Biosciences INC

Precision BioSciences announces its Q1 2025 financial results, emphasizing advancements in its in vivo gene editing therapies, particularly PBGENE-HBV and PBGENE-DMD.

Delay expectedTo accelerate development of PBGENE-DMD and maintain operational capability to deliver PBGENE-HBV and PBGENE-DMD data through Phase 1 clinical results, we plan to pause development of PBGENE-3243 and will stage future development of PBGENE-3243 alone or with partners following completion of the Phase 1 ELIMINATE-B HBV trial and after the PBGENE-DMD program reaches the clinic.

Capital raiseThe company states that it will need substantial additional funding, and if it is unable to raise a sufficient amount of capital when needed on acceptable terms, or at all, it may be forced to delay, reduce or eliminate some or all of its research programs, product development activities and commercialization efforts.The company is subject to the Baby Shelf Rule, as defined and described in Risk Factors We will need substantial additional funding, and if we are unable to raise a sufficient amount of capital when needed on acceptable terms, or at all, we may be forced to delay, reduce or eliminate some or all of our research programs, product development activities and commercialization efforts. in Part II, Item 1A. of this Quarterly Report on Form 10-Q.As a result of the Baby Shelf Rule, we will be limited in the amount of proceeds we are able to raise by selling shares of our common stock using our Form S-3, including under our ATM facility, until such time as our public float exceeds $75 million.

Worse than expectedThe company's revenue decreased significantly compared to the same period last year, primarily due to decreased revenue from collaboration agreements.

Summary

Precision BioSciences reported a net loss of $20.6 million for the three months ended March 31, 2025.
Revenue for the quarter was less than $0.1 million, a significant decrease from $17.6 million in the same period of 2024.
Research and development expenses totaled $13.6 million, slightly up from $13.3 million in Q1 2024.
The company's cash, cash equivalents, and restricted cash amounted to $99.8 million as of March 31, 2025.
Precision BioSciences believes its current resources will fund operations into the second half of 2026.
The company is prioritizing PBGENE-DMD and plans to pause development of PBGENE-3243.
PBGENE-HBV clinical trials have been cleared to commence in the United States and the United Kingdom.
An IND and/or CTA for PBGENE-DMD is targeted for filing in 2025, with clinical data expected in 2026.

Sentiment

Score: 5

Explanation: The document presents a mixed picture. While there's progress in clinical trials and promising preclinical data, the significant revenue decline and continued losses raise concerns. The company's ability to extend its cash runway and prioritize key programs is a positive sign, but the need for additional funding remains a risk.

Positives

PBGENE-HBV was safe and well tolerated in all three participants in cohort 1 after the first administration of a 0.2 mg/kg dose.
PBGENE-HBV demonstrated a substantial reduction in Hepatitis B surface antigen (HBsAg) in two of the three participants following the first administration at the lowest dose level.
The U.S. FDA granted Fast Track designation to PBGENE-HBV for chronic Hepatitis B.
PBGENE-DMD demonstrated significant, durable functional improvement in a humanized DMD mouse model.
The company targets filing an IND and/or CTA for PBGENE-DMD in 2025, with clinical data expected in 2026.
The company believes its current resources will fund operations into the second half of 2026.

Negatives

Precision BioSciences reported a net loss of $20.6 million for Q1 2025.
Q1 2025 revenue was less than $0.1 million, down from $17.6 million in Q1 2024.
The company is pausing development of PBGENE-3243 to accelerate PBGENE-DMD and deliver PBGENE-HBV and PBGENE-DMD data through Phase 1 clinical results.

Risks

The company has incurred significant operating losses since its inception and expects to continue to incur losses for the foreseeable future.
The company will need substantial additional funding, and if it is unable to raise a sufficient amount of capital when needed on acceptable terms, or at all, it may be forced to delay, reduce or eliminate some or all of its research programs, product development activities and commercialization efforts.
The company has a limited operating history, which makes it difficult to evaluate its current business and future prospects and may increase the risk of investment.
ARCUS is a novel technology, making it difficult to predict the time, cost and potential success of product candidate development.
The company is heavily dependent on the successful development and translation of ARCUS, and due to the early stages of product development operations, it cannot give any assurance that any product candidates will be successfully developed and commercialized.
Adverse public perception of genome editing may negatively impact the developmental progress or commercial success of products that the company develops alone or with collaborators.
The company faces significant competition in industries experiencing rapid technological change, and there is a possibility that its competitors may achieve regulatory approval before it or develop product candidates or treatments that are safer or more effective than theirs, which may harm its financial condition and its ability to successfully market or commercialize any of its product candidates.

Future Outlook

Precision BioSciences expects its current cash resources, operational receipts, and ATM facility availability to fund operating expenses and capital expenditure requirements into the second half of 2026, sufficient to achieve first-in-human Phase 1 clinical data for two wholly-owned in vivo gene editing programs.

Management Comments

Throughout 2025, we plan to share updates on the full cohorts including antiviral efficacy at different dose levels as each cohort is completed following three dose administrations and appropriate follow up.
Based on these compelling data, the significant unmet need in DMD, and the clear regulatory guidance established for new therapeutics for DMD, we believe that PBGENE-DMD holds significant potential for the majority of patients with DMD.
To accelerate development of PBGENE-DMD and maintain operational capability to deliver PBGENE-HBV and PBGENE-DMD data through Phase 1 clinical results, we plan to pause development of PBGENE-3243 and will stage future development of PBGENE-3243 alone or with partners following completion of the Phase 1 ELIMINATE-B HBV trial and after the PBGENE-DMD program reaches the clinic.

Industry Context

The company is operating in the competitive gene editing field, facing competition from companies using technologies like CRISPR/Cas9, zinc finger nucleases, and TALENs. The company's focus on in vivo gene editing places it in competition with other gene therapy companies.

Comparison to Industry Standards

The report does not provide enough information to make a detailed comparison to industry standards.
However, the company's focus on in vivo gene editing and its progress in clinical trials for PBGENE-HBV and PBGENE-DMD can be compared to other companies in the gene therapy and gene editing space, such as CRISPR Therapeutics, Editas Medicine, and Intellia Therapeutics.
These companies are also developing gene editing therapies for various diseases and are at different stages of clinical development.
A thorough comparison would require analyzing the clinical trial data, financial performance, and market capitalization of these companies.

Stakeholder Impact

Shareholders face potential dilution from future capital raises.
Employees may experience changes in job security due to program prioritization.
Patients with HBV and DMD may benefit from the company's progress in developing new therapies.
Suppliers and manufacturers may be affected by changes in the company's development plans.
Creditors are exposed to risks associated with the company's ability to secure additional funding.

Next Steps

The company plans to share updates on the full cohorts including antiviral efficacy at different dose levels as each cohort is completed following three dose administrations and appropriate follow up.
The company targets filing an IND and/or CTA for PBGENE-DMD in 2025, with clinical data expected in 2026.
The company will stage future development of PBGENE-3243 alone or with partners following completion of the Phase 1 ELIMINATE-B HBV trial and after the PBGENE-DMD program reaches the clinic.

Key Dates

Date	Description
January 26, 2006	Precision BioSciences, Inc. was incorporated.
November 19, 2020	Precision BioSciences entered into a development and license agreement with Eli Lilly and Company.
April 9, 2021	Precision BioSciences entered into a program purchase agreement with Les Laboratoires Servier and Institut de Recherches Internationales Servier.
August 2021	Precision BioSciences entered into a development and license agreement with iECURE.
June 14, 2022	Precision BioSciences entered into a collaboration and license agreement with Novartis Pharma AG.
August 15, 2023	Precision BioSciences entered into an asset purchase agreement with Imugene Limited.
January 7, 2024	Precision BioSciences entered into a license agreement with TG Cell Therapy, Inc.
March 1, 2024	Precision BioSciences entered into an underwriting agreement relating to a public offering.
July 31, 2024	Precision BioSciences entered into an amended and restated loan and security agreement with Banc of California.
January 6, 2025	Precision BioSciences received a deferred cash payment of $2.5 million from TG Therapeutics.
February 2025	Precision BioSciences announced initial results from the first administration of PBGENE-HBV in cohort 1.
March 2025	The U.S. FDA cleared PBGENE-HBV to commence Phase 1 clinical trials in the United States.
April 2025	The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) cleared PGENE-HBV for a Phase 1 trial.
April 2025	The FDA granted Fast Track designation to PBGENE-HBV for chronic Hepatitis B.
May 8, 2025	Precision BioSciences presented initial safety for PBGENE-HBV at the European Association for the Study of the Liver Congress (EASL).
May 14, 2025	Precision BioSciences presented additional new data at the American Society of Gene and Cell Therapy (ASGCT).

Keywords

Precision BioSciences, gene editing, ARCUS, PBGENE-HBV, PBGENE-DMD, PBGENE-3243, clinical trials, financial results, biotechnology, pharmaceuticals

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