Precision BioSciences Receives Pre-IND Feedback from US FDA for PBGENE-HBV as it Advances Towards Clinical Readiness
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Insights
The update on Precision BioSciences' PBGENE-HBV program is a significant stride in the gene editing field, particularly for the treatment of Chronic Hepatitis B (HBV). The feedback from regulatory bodies such as the FDA is a critical step in drug development, as it often signals a move towards human clinical trials, which can have substantial implications for both the company's valuation and the broader biotech sector.
From a medical research perspective, the potential of PBGENE-HBV to eliminate cccDNA and inactivate integrated HBV DNA is noteworthy. cccDNA, or covalently closed circular DNA, is a unique form of the virus's genetic material that is notoriously difficult to eradicate and is responsible for the persistence of the infection. The ability to target this aspect of the virus could indeed represent a leap towards a functional cure, a milestone that current treatments have not achieved.
Furthermore, the specificity of the ARCUS® platform in editing genes could reduce off-target effects, which are a common concern in gene therapy. Precision's progress towards an IND (Investigational New Drug application) and/or CTA (Clinical Trial Application) is therefore not only pivotal for its own pipeline but could also set a precedent for similar gene editing therapies in the future.
Investors closely monitor developments such as these because they can lead to significant changes in a company's market value. The positive regulatory feedback positions Precision BioSciences favorably in the eyes of the investment community, as it de-risks the clinical development path to a certain extent. The anticipation of an IND/CTA filing in 2024 provides a clear timeline for investors and could influence investment decisions in the interim.
However, it is essential to consider the competitive landscape. Precision BioSciences' claim that PBGENE-HBV will be the first and potentially only curative gene editing program for HBV in clinical trials is a bold one. The uniqueness of this claim, if substantiated by clinical outcomes, may provide Precision with a significant competitive advantage. Nonetheless, the biotech industry is known for rapid innovation and competitors may emerge with alternative or complementary solutions.
Lastly, the expansion of site selection for global clinical Phase 1 studies highlights the company's commitment to a broad and diverse patient population. This could enhance the potential market reach of PBGENE-HBV upon successful trial outcomes and eventual commercialization.
From a financial standpoint, the transition of PBGENE-HBV towards clinical trials is a capital-intensive phase and Precision BioSciences will likely experience increased cash burn as it ramps up manufacturing and clinical site preparations. Investors should monitor the company's cash reserves and fundraising activities, as these will be crucial for sustaining operations through this period.
Furthermore, the long-term potential revenue from a successful HBV therapy could be substantial, given the chronic nature of the disease and the global prevalence. The market for HBV treatments is substantial and a functional cure could command a premium price. However, the actual financial impact will depend on the clinical trial results, pricing, insurance coverage and competitive dynamics at the time of potential market entry.
It is also important to note that while regulatory feedback is positive, it is not a guarantee of future approval. Investors should weigh the risks of clinical trial outcomes against the potential rewards of a successful market entry. Precision BioSciences' stock performance will likely reflect the evolving risk-reward profile as the PBGENE-HBV program progresses.
- Regulatory feedback and alignment from both
- IND/CTA enabling studies with final clinical candidate underway for PBGENE-HBV program; expect to file IND and/or CTA in 2024
- Expected to be the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA
Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion, excision, and elimination, today announced that the company has received pre-IND regulatory feedback from the
“We believe the regulatory feedback we have received to date informs and strengthens our clinical strategy and provides a clear pathway towards an IND and/or CTA filing for our lead gene editing program PBGENE-HBV,” said Michael Amoroso, President and Chief Executive Officer at Precision BioSciences. “Based on this feedback, we have initiated the final preclinical studies as well as site selection efforts as we move towards clinical readiness. We look forward to filing regulatory applications in 2024 and continue to believe that PBGENE-HBV has the potential to achieve a functional cure for patients suffering with Chronic Hepatitis B."
The pre-IND meeting with FDA provided feedback on overall design for the proposed first-in-human clinical study as well as feedback on the toxicology and specificity assessments. This interaction builds on the engagement and alignment achieved through early scientific advice during the FDA INTERACT meeting in July 2023. Similar, productive consultations have been held with regulators in ex-
Collectively, the feedback and overall alignment obtained from multiple regulatory authorities validates Precision’s path towards a planned IND and/or CTA filing for PBGENE-HBV in 2024. Precision is committed to rapidly advancing PBGENE-HBV into a global clinical Phase 1 study and is currently expanding site selection efforts with specialized centers focused on the treatment of Chronic Hepatitis B in several countries. Simultaneously, manufacturing is proceeding to supply drug for all planned nonclinical and clinical activities.
PBGENE-HBV is expected to be the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA.
“Current treatment options for patients with HBV are unable to both eliminate cccDNA and inactivate HBsAg expression from integrated HBV DNA genomes in the hepatocyte, which is essential to drive a durable loss of viral markers and potentially achieve a functional cure for patients with HBV,” said Dr. Geoffrey Dusheiko, MD, FCP(SA), FRCS. Emeritus Professor of Medicine, King’s College Hospital & University College London. “Given the constructive feedback from the FDA and other regulatory agencies worldwide, I look forward to seeing this novel modality progressing to further assessment in the clinic.”
About Hepatitis B and PBGENE-HBV:
Hepatitis B is a leading cause of morbidity in the US and death globally, with no curative options currently available for patients. In 2019, despite the availability of approved antiviral therapies, an estimated 300 million people globally and more than 1 million people in the US were estimated to have chronic hepatitis B infection. An estimated
Chronic hepatitis B infection is primarily driven by persistence of HBV cccDNA and integration of HBV DNA into the human genome in liver cells, the primary source of HBsAg in late-stage disease. Current treatments for patients with HBV infection include agents that result in long-term viral suppression as indicated by reduction of circulating HBV DNA, but these therapies do not eradicate HBV cccDNA, rarely lead to functional cure, and require lifelong administration. PBGENE-HBV is a highly specific, novel therapeutic approach to treating patients with chronic HBV infection. It’s designed to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, resulting in degraded cccDNA and a reduction in HBsAg.
About ARCUS
ARCUS is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert, excise, or eliminate DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI, that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA and stimulate gene insertion at the cut site by homologous recombination. Precision's platform and products are protected by a comprehensive portfolio including more than 130 granted patents to date.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is an advanced gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The Company intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our product candidates and gene editing approaches including ARCUS’s potential editing efficiency and differentiating aspects and the ability for the ARCUS genome editing platform to develop differentiated programs; the suitability of ARCUS nucleases for gene or viral elimination, large excision, gene insertion, and other complex gene editing approaches to drive defined outcomes; the potential of PBGENE-HBV to durably achieve a near-complete reduction across viral markers including HBsAg, HBeAg, HBV DNA and HBV RNA and as a potentially curative treatment for HBV with no off-target editing at maximal on-target editing dose; and the expected timing of manufacturing activities and regulatory processes, including timing of expected CTA and/or IND filings. The words “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “designed”, “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “promise,” “pursue,” “should,” “target,” “will,” “would,” and other similar words or expressions, or the negative of these words or similar words or expressions, are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions.
Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. These statements are neither promises nor guarantees, but involve number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with raising additional capital and requirements under our current debt instruments and effects of restrictions thereunder; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical studies and clinical trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ ability to identify, develop and commercialize product candidates; potential product liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the
All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
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Investor and Media Contact:
Naresh Tanna
Vice President, Investor Relations
naresh.tanna@precisionbiosciences.com
Source: Precision BioSciences
FAQ
What is the regulatory feedback received by Precision BioSciences for the PBGENE-HBV program?
What is the expected timeline for filing IND and/or CTA for the PBGENE-HBV program?
What makes PBGENE-HBV unique in its approach?
What feedback was provided by the FDA during the pre-IND meeting?
Who is leading the clinical development efforts for PBGENE-HBV at Precision BioSciences?
