ATP7B

IN013 restores functional protein levels to reduce systemic copper build-up in patients with Wilson’s disease. Image credit: Shutterstock / Kateryna Kon. The US Food and Drug Administration (FDA) has granted rare paediatric disease and orphan drug designations for IN013, Innorna’s mRNA therapeutic for Wilson’s disease. China-headquartered Innorna will now be in line for tax credits for US-based clinical trials. If IN013 is approved, the potential for seven years of market exclusivity in the designated indication, in accordance with orphan drug status benefits. The rare paediatric disease designation, meanwhile, given to biotechs developing treatments for diseases that affect fewer than 200,000 US paediatric patients, could mean Innorna receives a priority review voucher (PRV) if the therapy is approved. This voucher would allow the company to fast-track a future candidate of its choice through FDA review, meaning it will reach the market four months sooner if approved.  Innorna could also sell the voucher for a quicker cash injection – vouchers have typically been sold for around $100m over the past few years. Wilson’s disease is a rare inherited disease that causes copper levels to build up in the body’s organs, including the liver and brain. The disease is caused by mutations in the ATP7B gene, which normally encodes for the protein responsible for eliminating excess copper. Symptoms of the illness include hepatic and neurological damage, ocular abnormalities, and kidney injury, among others. It is estimated that one in every 30,000 individuals is affected by the disease, with isolated populations at higher risk due to consanguineous marriages. The current standard of care is the administration of copper chelating agents – compounds that bind to copper ions to reduce toxicity and facilitate the removal. In 2022, the FDA greenlit the first new treatment for Wilson’s disease in 30 years, Orphalan’s Cuvrior (trientine tetrahydrochloride). Comprising a copper chelating agent, Cuvrior is for patients who are de-coppered and able to take D-penicillamine, another drug approved for the disease. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Innorna states that current treatments offer limited efficacy and carry risks, such as hepatorenal toxicity or exacerbated neurological symptoms. Some Wilson’s disease patients taking penicillamine have experienced nervous system deterioration . Unlike current approaches that regulate copper levels downstream, Innorna’s IN013 enables the production of the ATP7B protein. The biotech states its asset, developed on an mRNA lipid nanoparticle platform, can restore functional protein levels to reduce systemic copper build-up. Other companies are also developing new treatments for Wilson’s disease. Vivet Therapeutics and Pfizer are developing a gene therapy called VTX-801, currently in a Phase I/II trial (NCT04537377). The candidate acts by inserting a fully functional version of ATP7B into the body. The FDA designations for Innorna come at a time of regulatory spotlight on rare disease pipelines, amid agency job cuts by US President Donald Trump and programme expirations. The PRV programme for paediatric rare diseases, for example, entered its ‘sunsetting’ phase in December 2024 after US Congress failed to reauthorise it. Experts have said the programme’s expiration, along with reduced FDA capacity, could slow down rare disease development. mRNA vaccine coverage on Pharmaceutical Technology is supported by Trilink . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Enhancing Potency, Stability, and Efficiency in mRNA Read this technical note to explore the frontiers of mRNA capping in advanced therapeutic development. As mRNA-based treatments evolve, optimizing capping strategies becomes critical for enhancing stability, translation efficiency, and potency. This comparative analysis of TriLink’s CleanCap® technologies versus traditional enzymatic capping reveals the key innovations setting new benchmarks. Discover how co-transcriptional methods, structural modifications, and dose-sparing potential are accelerating the path from synthesis to impactful protein expression in both research and therapeutic settings. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Trilink Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Trilink Thematic and that your personal data will be used as described in their Privacy Policy

MIAMI, FL / ACCESSWIRE / December 23, 2024 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), or the "Company", an emerging leader in age-reversal science, today announced significant findings from its copper binding studies with Telomir-1. This is a promising development as the company explores Telomir-1 for the treatment of Wilson's disease and other copper related disorders. The announcement marks an important step forward in Telomir's mission to address critical unmet medical needs while continuing to explore transformative therapies.Key FindingsRecent preclinical in vitro studies conducted by Recipharm and Smart Assays have demonstrated Telomir-1's exceptional binding affinity for copper ions, highlighting its ability to selectively interact with and regulate copper metabolism. Additionally, Telomir-1's capability to bind and exchange key ions, including copper, iron, and zinc, emphasizes its unique profile and positions it as a promising candidate for further research. These findings form a strong foundation for investigating Telomir-1's potential in addressing Wilson's disease, a rare genetic disorder characterized by excessive copper accumulation that can result in significant damage to the liver, brain, and other vital organs.Wilson's Disease: A Critical Unmet NeedWilson's disease is a rare genetic disorder that affects approximately 1 in 30,000 individuals worldwide. The condition is caused by mutations in the ATP7B gene, which impair the body's ability to properly regulate copper metabolism. As a result, copper accumulates to toxic levels, particularly in the liver and brain, leading to severe and potentially life-threatening complications, including liver failure, neurological damage, and psychiatric disorders. Left untreated, Wilson's disease is fatal.Current treatments for Wilson's disease, such as chelating agents (e.g., penicillamine and trientine) and zinc therapy, are primarily aimed at reducing copper levels in the body. While these treatments can be effective, they come with significant drawbacks, including:Chelating agents often cause severe side effects, including kidney damage, bone marrow suppression, and gastrointestinal issues. Additionally, these drugs require lifelong adherence, which can be burdensome for patients.Zinc therapy works by preventing copper absorption in the gut but can result in side effects like anemia, gastrointestinal discomfort, and reduced effectiveness over time.The limitations of current therapies underscore the urgent need for new treatment options that address the root cause of Wilson's disease while minimizing side effects. Telomir-1's copper binding properties hold the potential to offer a safer, more effective alternative that directly targets the underlying mechanisms of the disease. The designation of Wilson's disease as a rare and life-threatening condition also positions it as a candidate for orphan drug designation, which could accelerate development timelines and provide additional regulatory and financial incentives."These recent groundbreaking results on copper binding and interaction showcase Telomir-1's potential to address a significant unmet need in Wilson's disease by enabling beneficial regulations at the underlying causes rather than just managing the symptoms of the disease," said Dr. Itzchak Angel, Chief Scientific Advisor at Telomir. "This is a pivotal moment as we work to bring transformative therapies to patients who need them most."Update on Previous Osteoarthritis StudyEarlier this year, Telomir partnered with Midwest Veterinary Services to launch a planned large-scale study on osteoarthritis in dogs. While only the safety phase of the study was completed-showing no adverse effects-the Company chose to discontinue the study. This decision was based on the requirement to induce osteoarthritis in the dogs, a practice that conflicted with the new management's commitment to the Company's mission and values. Instead, the Company is now in discussions with a well-known veterinary institution to design a new study that will recruit elderly dogs who naturally suffer from osteoarthritis. As such, Telomir is committed to obtaining an INAD (Investigational New Animal Drug) application for pets while progressing toward an IND (Investigational New Drug) application for humans.Expanding Research and DevelopmentBuilding on these results, Telomir is planning and advancing studies to expand the therapeutic potential of Telomir-1. Key ongoing initiatives include:Wilson's Disease Study: Investigating Telomir-1's effects on copper regulation in preclinical models.Type 2 Diabetes Studies: After the success achieved with the zebrafish model, Telomir is currently conducting a rat diabetic model to further strengthen the data on Telomir-1's efficacy in reversing key metabolic parameters.Progeria Research: Examining Telomir-1's impact on accelerated aging and telomere function.Cancer Models: Exploring anti-cancer applications using xenograft studies.Alzheimer's Disease: Investigating Telomir-1 for its potential to mitigate cognitive decline and neurodegeneration associated with Alzheimer's.Metal Toxicity: Exploring Telomir-1's role in addressing metal toxicity, which occurs when metals like copper, iron, or lead accumulate to harmful levels in the body. Metal toxicity can disrupt cellular function, lead to oxidative stress, and contribute to chronic and age-related diseases. By regulating metal levels, Telomir-1 aims to restore cellular balance and reduce associated health risks.DNA Methylation Analysis: Investigating Telomir-1's role in modulating DNA methylation rates, which influence gene expression and play a key role in aging and disease progression.DNA methylation, the addition of chemical groups to DNA, can disrupt normal gene function when dysregulated, contributing to various chronic and age-related diseases. By addressing these epigenetic changes, Telomir-1 aims to restore healthy gene expression patterns.Clinical Progress and Future MilestonesBuilding on our preclinical success demonstrating the potential for age reversal, improved longevity, and health span, as well as the reversal of key Type 2 diabetes parameters, the Company is committed to unlocking the full potential of Telomir-1 to revolutionize the treatment of age-related diseases by addressing their root causes. The possibilities are vast, and we believe this drug holds extraordinary promise to potentially transform the pharmaceutical industry.As we continue to gather scientific data and delve deeper into the molecular mechanisms of action of Telomir-1, we are positioning ourselves to identify the most impactful indication for our initial IND application. The Company is currently working on the optimization of manufacturing processes to enable an affordable and scalable approach. This will support the production of GMP-grade quantities necessary for IND-enabling studies and clinical trials. The Company plans to carry out the IND-enabling safety studies in Q2 2025, targeting IND submission by Q4 2025, with first-in-human clinical trials anticipated in the first half of 2026. The company also plans to secure an INAD for pets, paving the way for Telomir-1's dual application in both human and veterinary medicine.Market PotentialThe global anti-aging drugs market is experiencing significant growth, driven by increasing demand for treatments that address age-related diseases and promote longevity. In 2024, the market was valued at approximately USD 91.05 billion and is projected to reach USD 160.24 billion by 2031, growing at a compound annual growth rate (CAGR) of 7.32% during this period. This expansion reflects a rising consumer focus on health and wellness, advancements in medical research, and a growing aging population seeking solutions to enhance quality of life. (Verified Market Research)Telomir-1 represents a novel approach by targeting the root causes of age-related diseases rather than simply managing symptoms. By developing therapies that extend health span and enhance quality of life, the Company seeks to redefine patient care and make a meaningful impact in this rapidly growing multi-billion-dollar market.Leadership Perspective"As we look to 2025, Telomir remains dedicated to its mission," said Erez Aminov, Chairman and CEO of the Company. "We are pleased to have secured additional financing at a 20% premium with no warrants, providing the resources needed to continue advancing Telomir-1's development while focusing on creating value for our shareholders."About Telomir Pharmaceuticals, Inc.Telomir Pharmaceuticals, Inc. (Nasdaq:TELO) is a pre-clinical stage pharmaceutical company seeking to lead development in several areas, including age-reversal science. Telomir is focused on the development of Telomir-1, a novel small molecule designed to lengthen the DNA's protective telomere caps, which are crucial in the aging process. Telomir's goal is to explore the potential of Telomir-1 starting with ongoing research in animals and then in humans.Telomeres are the protective end caps of a chromosome made up of DNA sequences and proteins. As humans age, telomeres shorten, with metal reactivity accelerating the process, which presents humans and pet animals with an increased chance of contracting a number of degenerative and age-related diseases. Telomir's goal is to develop and gain regulatory approval for Telomir-1, proposed to be dosed orally, with the broader aim of promoting longevity and enhancing overall quality of life.The Nobel Assembly at Karolinska Institute (Sweden) awarded the Nobel Prize in Physiology or Medicine in 2009 for the discovery of how chromosomes are protected by telomeres and the enzyme telomerase.Telomir-1 is in preclinical development and has not yet been tested in humans. There is no assurance that Telomir-1 will proceed through development or will ultimately receive FDA approval for marketing.Cautionary Note Regarding Forward-Looking StatementsThis press release, statements of Telomir Pharmaceuticals' management or advisors related thereto, and the statements contained in the news story linked in this release contain "forward-looking statements," which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding (i) the anticipated benefits of the preclinical testing results described herein, (ii) anticipated timelines and subject matter for additional preclinical and clinical testing of Telomir-1 and (iii) the potential therapeutic benefits of Telomir-1 generally.Any forward-looking statements in this press release are based on Telomir's current expectations, estimates and projections only as of the date of this release and are subject to a number of significant risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the potential use of the data from our studies, our ability to develop and commercialize Telomir-1 for specific indications and safety of Telomir-1. These and other risks concerning Telomir's programs and operations are described in additional detail in its Annual Report on Form 10-K for the fiscal year ended December 31, 2023, which is on file with the SEC. Telomir explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.Telomir's lead compound, Telomir-1, is being investigated for its potential to address a range of conditions, including Type 2 diabetes, Wilson's disease, progeria, Alzheimer's disease, and cancer. The company's innovative approach aims to treat the root causes of disease, offering a promising path forward in improving patient care.For more information, visit www.telomirpharma.com.Contact InformationHelga Moya [email protected](786) 396-6723SOURCE: Telomir Pharmaceuticals, Inc.

-- Presented initial in vivo data for universal liver-targeted LNP and Wilson’s Disease program at ESGCT; initiated IND-enabling activities for Wilson’s Disease program in 4Q 2024 and remains on track to file IND and/or CTA in 1H 2026 -- -- Announced strategic research collaboration and license agreement with Bristol Myers Squibb; received $110 million upfront, with potential for more than $3.5 billion in milestone payments -- -- On track to report initial data from Phase 1/2 clinical trial of PM359 for p47phox CGD in 2025 -- -- Unveiled strategically focused pipeline, prioritizing set of high value programs, each with a clearly defined path to value creation and the potential to accelerate development of follow-on programs -- -- Pro-forma cash, cash equivalents and investments as of September 30, 2024, expected to be sufficient to fund planned operations into 1H 2026 -- CAMBRIDGE, Mass., Nov. 12, 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 reported financial results for the third quarter ended September 30, 2024 and provided a business update. “In October, we presented the first in vivo preclinical data from our Wilson’s Disease program, demonstrating that Prime Editors can efficiently correct pathogenic mutations, without introducing safety concerns or detectable off-target edits,” said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. “These data are meaningful both for our efforts in Wilson’s Disease – a devasting condition that is believed to affect more than 20,000 people in the United States and Europe, and for which there are currently no approved disease-modifying therapies – and for our efforts in liver disease programs more broadly. Our Wilson’s Disease program leverages our proprietary, universal LNP platform, and these interim results suggest that our LNP may successfully deliver increased potency, as well as an improved safety profile and biodistribution, compared to other commonly used LNPs in development. We look forward to advancing our Wilson’s Disease program toward an IND and/or CTA filing in the first half of 2026.” Dr. Gottesdiener continued, “Also in recent months, we announced our decision to strategically prioritize a set of high value programs, each with a clear path to value inflection and the potential to unlock multiple follow-on opportunities. This includes our wholly owned efforts in CGD and Wilson’s Disease, as well as our efforts in cell therapy and cystic fibrosis, which we are advancing with support from Bristol Myers Squibb and the Cystic Fibrosis Foundation, respectively. We believe partnerships like these will continue to play a critical role as we build Prime Medicine, allowing us access to best-in-class expertise and non-dilutive capital and enabling us to more rapidly realize the full potential of Prime Editing.” Recent Business Updates Pipeline Updates In September 2024, Prime Medicine unveiled its strategically focused pipeline and plans to prioritize a set of high value programs, each targeting a disease with well-understood biology and a clearly defined clinical development and regulatory path. Each prioritized program is also intended to serve as a beachhead, in that the modularity of the Prime Editing platform is expected to allow Prime Medicine to generate follow-on candidates rapidly and efficiently. The prioritized programs sit across Prime Medicine’s core areas of focus (hematology, immunology and oncology, liver, and lung), and include ex vivo hematopoietic stem cell (HSC) programs for the treatment of p47phox chronic granulomatous disease (CGD) and X-linked CGD; ex vivo T-cell therapies; a lipid nanoparticle (LNP) Prime Editor for the treatment of Wilson’s Disease; and LNP or adeno-associated virus (AAV) Prime Editors for the treatment of cystic fibrosis (CF). In order to pursue these prioritized high value programs as rapidly as possible, the Company is identifying partnership opportunities to advance its other programs, including those for neurological diseases, cell therapy, ocular diseases and hearing loss. Hematology, Immunology and Oncology: In the third quarter of 2024, Prime Medicine initiated its global Phase 1/2 clinical trial of PM359 in the United States. The Phase 1/2 clinical trial is a multinational, first-in-human trial designed to assess the safety, biological activity and preliminary clinical activity of PM359 in adult and pediatric study participants. Prime Medicine expects to report initial clinical data from the Phase 1/2 trial in 2025. Liver: At the European Society for Gene and Cell Therapy (ESGCT) 31st Annual Congress (October 22-25, 2024), Prime Medicine presented data from multiple studies showcasing the potential of its proprietary, universal LNP platform to precisely deliver Prime Editors to correct disease-causing mutations in the liver, including in Wilson’s Disease. Prime Medicine’s universal LNP contains a GalNAc-targeting ligand (GalNAc-LNP), a validated mechanism for liver-specific delivery of gene editors. Highlights included: Preclinical data demonstrated that the delivery of Prime Editors using a GalNAc-LNP (GalNAc-LNP Prime Editor) resulted in increased potency, as well as improved safety profile and biodistribution, compared to traditional LNPs that have gone into the clinic.In vivo proof-of-concept data from Prime Medicine’s GalNAc-LNP Prime Editor for the treatment of Wilson’s Disease, demonstrated up to 80% precise correction of the H1069Q mutation and restoration of ATP7B mRNA to wild-type levels in a humanized mouse model, as well as precise editing of liver cells in non-human primates (NHPs), with up to 51% precise editing with a surrogate H1069Q Prime Editor. In preclinical studies to-date, Prime Medicine observed significant reductions of copper accumulation in the livers of humanized mice. In both mouse and NHP studies, no detectable off-target edits or unintended edits at the target site were observed. Prime Medicine is completing the final stages of lead optimization and recently initiated investigational new drug (IND)-enabling activities for its Wilson’s Disease program, with an IND and/or clinical trial application (CTA) filing expected in the first half of 2026. Prime Medicine will present on its Wilson’s Disease program at the American Association for the Study of Liver Diseases (AASLD), to be held on November 15-19, 2024. Longer term, the Company expects to use its universal LNP across all liver disease programs, allowing more rapid and cost-efficient expansion into follow-on rare and non-rare liver indications. Corporate Updates In September 2024, Prime Medicine entered into a strategic research collaboration and license agreement with Bristol Myers Squibb to develop and commercialize multiple Prime Edited ex vivo T-cell therapies. Under the terms of the agreement, Prime Medicine received a $55 million upfront payment and a $55 million equity investment from Bristol Myers Squibb. Prime Medicine is also eligible to receive more than $3.5 billion in milestones, including up to $1.4 billion in development milestones and more than $2.1 billion in commercialization milestones, along with royalties on net sales. Third Quarter 2024 Financial Results Research and Development (R&D) Expenses: R&D expenses were $40.3 million for the three months ended September 30, 2024, as compared to $41.0 million for the three months ended September 30, 2023.General and Administrative (G&A) Expenses: G&A expenses were $14.1 million for the three months ended September 30, 2024, as compared to $10.5 million for the three months ended September 30, 2023. The increase in G&A expenses was driven by personnel expenses, primarily related to an increase in non-cash stock-based compensation expense.Net Loss: Net loss was $52.5 million for the three months ended September 30, 2024, as compared to $50.7 million for the three months ended September 30, 2023.Cash Position: As of September 30, 2024, pro-forma cash, cash equivalents, investments and restricted cash were $244.6 million, which includes the $55 million equity investment from Bristol Myers Squibb received in September 2024 and the $55 million up-front consideration received in October 2024. Cash, cash equivalents, investments, and restricted cash were $189.6 million, as compared to $135.2 million as of December 31, 2023. Financial GuidanceBased on its current operating plans, Prime Medicine expects that its pro-forma cash, cash equivalents and investments as of September 30, 2024 will be sufficient to fund its operating expenses and capital expenditure requirements into the first half of 2026. About Prime MedicinePrime 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 StatementsThis 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 the release of updated data and filing of an IND and/or CTA application in the first half of 2026; the potential for its modular universal LNP platform to precisely deliver Prime Editors, correct disease-causing mutations in the liver, and deliver transformative treatments for Wilson’s Disease, GSD1b, and other rare and non-rare liver indications; the modular universal LNP platform’s ability to be used repeatedly to generate candidates that offer an improved safety profile and biodistribution compared to other LNPs in development; the anticipated maturation into a clinical-stage company by bringing PM359 into clinical development in 2024 with initial clinical data from the ongoing Phase 1/2 clinical trial of PM359 expected in 2025; the collaboration with Bristol Myers Squibb and the intended and potential benefits thereof, including the receipt of potential milestone and royalty payments from commercial product sales, if any; the safety profile, tolerability, and durability of its universal LNP; 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 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; its continued development and optimization of various non-viral and viral delivery systems; its ability to demonstrate superior off-target profiles for Prime Editing programs; the expansion of Prime Editing’s therapeutic potential and the creation of value through strategic business development to extend the reach and impact of Prime Editing to areas beyond Prime Medicine’s current core areas of focus; exploring business development opportunities that could accelerate existing work and the benefits thereof; 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: uncertainties related to Prime Medicine’s product candidates entering clinical trials; 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; Prime Medicine’s ability to identify and enter into future license agreements and collaborations; and general economic, industry and market conditions. 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, 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 ContactGregory DearbornPrime Medicine713-503-3364gdearborn@primemedicine.com Hannah DeresiewiczPrecision AQ212-362-1200hannah.deresiewicz@precisionaq.com Media ContactDan Budwick, 1ABdan@1ABmedia.com Condensed Consolidated Balance Sheet Data(unaudited) (in thousands) September 30,2024 December 31,2023Cash, cash equivalents, and investments 175,527 121,665Total assets 332,784 193,851Total liabilities 143,280 60,780Total stockholders’ equity 189,504 133,071 Condensed Consolidated Statement of Operations(unaudited) Three Months EndedSeptember 30,(in thousands, except share and per share amounts) 2024 2023 Collaboration revenue $209 $— Operating expenses: Research and development $40,340 $40,967 General and administrative 14,101 10,492 Total operating expenses 54,441 51,459 Loss from operations (54,232) (51,459)Other income: Accretion (amortization) of investments 885 1,769 Interest income 697 410 Change in fair value of short-term investment — related party 215 (1,579)Other income, net (83) 43 Total other income, net 1,714 643 Net loss before income taxes (52,518) (50,816)Benefit from income taxes — 108 Net loss attributable to common stockholders $(52,518) $(50,708)Net loss per share attributable to common stockholders, basic and diluted $(0.44) $(0.55)Weighted-average common shares outstanding, basic and diluted 119,764,270 91,846,835