Prime Medicine to Highlight New Preclinical Data, Including In Vivo Data in Wilson’s Disease, at Upcoming Scientific Meetings

Rhea-AI Summary

Prime Medicine (Nasdaq: PRME) announced it will present new preclinical data from its Prime Editing pipeline at two upcoming scientific meetings: the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress in Rome (October 22-25, 2024) and the American Association for the Study of Liver Diseases (AASLD) in San Diego (November 15-19, 2024).

The company will showcase in vivo data from its Wilson's Disease program and its universal liver-directed lipid nanoparticle (LNP) platform. Prime Medicine is on track to initiate IND-enabling activities for the Wilson's Disease program in Q4 2024, with an IND or CTA filing expected in H1 2026.

Presentations will cover various topics, including glycemic control restoration in Glycogen Storage Disease Type 1b, Prime Edited cell products for Chronic Granulomatous Disease, and advancements in Prime Editing for Wilson's Disease and Retinitis Pigmentosa.

Positive

• On track to initiate IND-enabling activities for Wilson's Disease program in Q4 2024
• IND or CTA filing for Wilson's Disease program expected in H1 2026
• New preclinical data, including in vivo data for Wilson's Disease, to be presented
• Advancements in universal liver-directed lipid nanoparticle (LNP) platform

Negative

• None.

Insights

Medical Research Analyst

The presentation of new preclinical data, particularly in vivo data for Wilson's Disease, represents a significant milestone for Prime Medicine. This progress in their Prime Editing pipeline could have substantial implications for treating genetic liver disorders.

Key points to consider:

• The company is on track to initiate IND-enabling activities for Wilson's Disease in Q4 2024, with an IND or CTA filing expected in H1 2026. This timeline provides a clear roadmap for investors to track progress.
• Prime Medicine's focus on Wilson's Disease is strategic, as it's a large, genetically defined condition with no approved disease-modifying treatments. This presents a significant market opportunity if their approach proves successful.
• The development of a universal liver-directed lipid nanoparticle (LNP) platform could have far-reaching implications, potentially allowing for broader application across multiple indications.
• Presentations at ESGCT and AASLD, prestigious scientific conferences, provide validation of the company's research and increase visibility in the scientific community.

While these advancements are promising, investors should note that the company is still in preclinical stages, with several years before potential commercialization. The success of Prime Editing technology in humans remains to be proven in clinical trials.

Financial Analyst

From a financial perspective, Prime Medicine's progress in preclinical development, particularly for Wilson's Disease, is a positive indicator for the company's potential. However, investors should consider several factors:

• With a market cap of $439,312,776, PRME is a relatively small biotech company, which often implies higher risk and volatility.
• The timeline to potential revenue is long, with IND filing not expected until H1 2026. This means continued cash burn and possible need for additional financing in the coming years.
• The broad applicability of their Prime Editing technology and LNP platform could provide multiple shots on goal, potentially reducing risk and increasing long-term value.
• Partnerships or licensing deals could become increasingly likely as the company progresses, potentially providing non-dilutive funding.

While the scientific progress is encouraging, financial performance will largely depend on successful translation to human trials and eventual commercialization. Investors should closely monitor cash runway, upcoming milestones and any potential partnerships or collaborations that could validate the technology and provide additional resources.

-- On-track to initiate IND-enabling activities for Wilson’s Disease program in 4Q 2024, with IND or CTA filing expected in 1H 2026 --

CAMBRIDGE, Mass., Oct. 15, 2024 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced the Company will highlight advances from across its Prime Editing pipeline and platform at the upcoming European Society of Gene and Cell Therapy (ESGCT) 31^st Annual Congress, being held October 22-25, 2024 in Rome, and the American Association for the Study of Liver Diseases (AASLD), being held November 15-19, 2024 in San Diego.

“At the ESGCT and AASLD meetings, we will present new preclinical data from across our pipeline, including from our efforts in liver disease,” said Jeremy Duffield, M.D., Ph.D., Chief Scientific Officer of Prime Medicine. “We are particularly excited to share in vivo data from our Wilson’s Disease program, as well as from our universal liver-directed lipid nanoparticle (LNP) platform. Together, these data reinforce our strategy of prioritizing Wilson’s Disease – a large, genetically defined condition for which there are no currently approved disease-modifying treatments, and which we believe is uniquely suited to a Prime Editing-based approach – and reinforce the potential for our modular universal LNP to be used broadly across current and future programs. We look forward to advancing our Wilson’s Disease program into IND-enabling studies later this year and, longer term, to potentially leveraging the modularity of our platform to expand into numerous follow-on indications.”

Details of the presentations are as follows:

European Society of Gene and Cell Therapy 31^st Annual Congress (ESGCT); October 22-25, 2024 in Rome

• Presentation Title: LNP delivered Prime Editors restore glycemic control in humanized rodent models of Glycogen Storage Disease Type 1b (GSD1b)
  Date & Time: October 24, 2024, 2:00 p.m. CEST

• Poster Title: Development of PM359, a Prime Edited CD34+ cell drug product for the treatment of p47phox Chronic Granulomatous Disease
  Date & Time: October 24, 2024, 2:00 – 2:30 p.m. CEST

• Poster Title: An all-Prime Editing one-step approach for non-viral generation of a multiplex-edited allogeneic CAR-T cell product
  Date & Time: October 24, 2024, 2:00 – 2:30 p.m. CEST

• Poster Title: Methods for genome-wide detection of single strand breaks induced by gene editors reveals the specificity of SpCas9 nuclease domains and provides comprehensive lists of potential off-targets for Prime Editors
  Date & Time: October 24, 2024, 2:00 – 2:30 p.m. CEST

• Poster Title: Exploring the roles of DNA repair processes across diverse Prime Editing strategies with pooled screens and Knock-Knock Prime
  Date & Time: October 24, 2024, 6:00 – 7:30 p.m. CEST

• Poster Title: Prime Editing advancements enable in vivo therapeutic correction of ATP7B p.H1069Q and p.R778L mutations in Wilson’s Disease
  Date & Time: October 24, 2024, 6:00 – 7:30 p.m. CEST

• Poster Title: Prime Editors precisely correct pathogenic mutations in RHO and USH2A associated Retinitis Pigmentosa and prevent retinal degeneration
  Date & Time: October 24, 2024, 6:00 – 7:30 p.m. CEST

American Association for the Study of Liver Diseases (AASLD); November 15-19, 2024 in San Diego

• Presentation Title: LNP delivered Prime Editors restore glycemic control in humanized rodent models of Glycogen Storage Disease Type 1b (GSD1b)
  Date & Time: Sunday, November 17, 2024, 5:00 – 6:30 p.m. PT

• Presentation Title: Advances in Prime Editing enable in vivo therapeutic correction of the ATP7B p.H1069Q and p.R778L mutations causing Wilson’s Disease
  Date & Time: Monday, November 18, 2024, 8:00 – 9:30 a.m. PT

About Prime Medicine

Prime Medicine is a leading biotechnology company dedicated to creating and delivering the next generation of gene editing therapies to patients. The Company is deploying its proprietary Prime Editing platform, a versatile, precise and efficient gene editing technology, to develop a new class of differentiated one-time curative genetic therapies. Designed to make only the right edit at the right position within a gene while minimizing unwanted DNA modifications, Prime Editors have the potential to repair almost all types of genetic mutations and work in many different tissues, organs and cell types. Taken together, Prime Editing’s versatile gene editing capabilities could unlock opportunities across thousands of potential indications.

Prime Medicine is currently progressing a diversified portfolio of investigational therapeutic programs organized around our core areas of focus: hematology, immunology and oncology, liver and lung. Across each core area, Prime Medicine is focused initially on a set of high value programs, each targeting a disease with well-understood biology and a clearly defined clinical development and regulatory path, and each expected to provide the foundation for expansion into additional opportunities. Over time, the Company intends to maximize Prime Editing’s broad and versatile therapeutic potential, as well as the modularity of the Prime Editing platform, to rapidly and efficiently expand beyond the diseases in its current pipeline, potentially including additional genetic diseases, immunological diseases, cancers, infectious diseases, and targeting genetic risk factors in common diseases, which collectively impact millions of people. For more information, please visit www.primemedicine.com.

© 2024 Prime Medicine, Inc. All rights reserved. PRIME MEDICINE, the Prime Medicine logos, and PASSIGE are trademarks of Prime Medicine, Inc. All other trademarks referred to herein are the property of their respective owners.

Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements about Prime Medicine’s beliefs and expectations regarding: the timing, progress, and results of its Wilson’s Disease program, including the timing of IND-enabling activities and opening an IND and/or CTA application; the initiation, timing, progress, and results of its research and development programs, preclinical studies and future clinical trials; the modularity of the Prime Editing platform and the benefits thereof; the potential for its modular universal LNP to be used broadly across current and future programs; the potential for Prime Editors to more precisely and effectively achieve genetic modification; the potential for Prime Editors to repair genetic mutations and offer curative genetic therapies for a wide spectrum of diseases; the potential of Prime Editors to reproducibly correct disease-causing genetic mutations across different tissues, organs and cell types; its expectations regarding the breadth of Prime Editing technology and the implementation of its strategic plans for its business, programs, and technology; and the potential of Prime Editing to unlock opportunities across thousands of potential indications. The words “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “expect,” “estimate,” “seek,” “predict,” “future,” “project,” “potential,” “continue,” “target” and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks associated with: the authorization, initiation, and conduct of preclinical and IND-enabling studies and other development requirements for potential product candidates, including uncertainties related to opening INDs and obtaining regulatory approvals; risks related to the development and optimization of new technologies, the results of preclinical studies, or clinical studies not being predictive of future results in connection with future studies; the scope of protection Prime Medicine is able to establish and maintain for intellectual property rights covering its Prime Editing technology; and the effect of unfavorable macroeconomic conditions or market volatility resulting from general economic, industry and market conditions, including rising interest rates, inflation, and adverse developments affecting the financial services industry. These and other risks and uncertainties are described in greater detail in the section entitled “Risk Factors” in Prime Medicine’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent Prime Medicine’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Prime Medicine explicitly disclaims any obligation to update any forward-looking statements subject to any obligations under applicable law. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.

Investor Contact
Hannah Deresiewicz
Precision AQ
212-362-1200
hannah.deresiewicz@precisionaq.com

Media Contact
Dan Budwick, 1AB
dan@1ABmedia.com

FAQ

What new data will Prime Medicine (PRME) present at the upcoming scientific meetings?

Prime Medicine will present new preclinical data, including in vivo data from its Wilson's Disease program and advancements in its universal liver-directed lipid nanoparticle (LNP) platform, at the ESGCT Annual Congress and AASLD meeting in October and November 2024, respectively.

When does Prime Medicine (PRME) expect to file an IND or CTA for its Wilson's Disease program?

Prime Medicine expects to file an IND or CTA for its Wilson's Disease program in the first half of 2026, following the initiation of IND-enabling activities in the fourth quarter of 2024.

What other diseases will Prime Medicine (PRME) present data on at the upcoming meetings?

Prime Medicine will present data on Glycogen Storage Disease Type 1b, Chronic Granulomatous Disease, and Retinitis Pigmentosa, among others, at the upcoming ESGCT and AASLD meetings in 2024.

How does Prime Medicine (PRME) plan to leverage its platform technology?

Prime Medicine plans to leverage the modularity of its platform to potentially expand into numerous follow-on indications beyond its current focus on Wilson's Disease and other liver-related conditions.