Latest news with #PrimeMedicine
Yahoo
27-05-2025
- Business
- Yahoo
Citi Downgrades Prime Medicine (PRME) to Neutral, Cuts PT to $1.50 Due to Market Uncertainty
On Tuesday, Citi downgraded Prime Medicine Inc. (NASDAQ:PRME) from a Buy to a Neutral rating while also significantly reducing its price target from $10 to $1.50. Citi said that this adjustment reflects that the company's shares are trading near cash, which shows the market's uncertainty on Prime Medicine's path to potential value inflection. A scientist examining a microchip and circuit board in a hi-tech lab. Despite implementing cost-cutting measures, which include a 25% workforce reduction and prioritizing its liver disease franchise & externally funded programs over Chronic Granulomatous Disease/CGD programs, Prime Medicine's cash reserves are projected to support operations only into H1 2026. This timeline falls short of management's guidance, which anticipates initial in-vivo clinical data for Wilson's disease and Alpha-1 antitrypsin deficiency/AATD in 2027. The company also plans to file an investigational new drug/IND application for these programs in H1 2026 and mid-2026, respectively. Prime Medicine is seeking business development deals to secure non-dilutive capital and extend its financial runway. It continues its cystic fibrosis program, supported by the Cystic Fibrosis Foundation, and collaborates with Bristol Myers Squibb on Prime Edited CAR-T products in hematology, immunology, and oncology. Prime Medicine Inc. (NASDAQ:PRME) is a biotech company that delivers genetic therapies to address the spectrum of diseases by deploying gene editing technology in the US. While we acknowledge the potential of PRME to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than PRME and that has 100x upside potential, check out our report about the cheapest AI stock. READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
Yahoo
19-05-2025
- Business
- Yahoo
Prime Medicine Announces Strategic Restructuring to Focus on Opportunities in Large Genetic Liver Diseases, Cystic Fibrosis, and Partnered Programs Alongside CEO Leadership Transition
-- Initial positive data from Phase 1/2 clinical trial of PM359 in CGD provide clinical proof-of-concept for Prime Editing as a transformative gene editing technology -- -- On-track to file IND and/or CTA for Wilson's Disease and AATD programs in 1H 2026 and mid-2026, respectively; initial clinical data for both expected in 2027 -- -- Allan Reine, M.D., CFO, to succeed Keith Gottesdiener, M.D., as CEO; Jeff Marrazzo, member of the Board of Directors, named Executive Chair -- -- Implementing cost cutting measures to significantly reduce cash needs in advance of key data inflection points -- CAMBRIDGE, Mass., May 19, 2025 (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 a strategic restructuring, including the deprioritization of its Chronic Granulomatous Disease (CGD) programs, as well as a cost and workforce reduction to focus on its liver franchise and programs funded through external partnerships. Prime Medicine is currently advancing in vivo programs to cure two of the largest genetic liver diseases, Wilson's Disease and Alpha-1 Antitrypsin Deficiency (AATD), with initial clinical data from both programs expected in 2027. Prime Medicine will also continue its in vivo Cystic Fibrosis program with support from the Cystic Fibrosis Foundation, and its efforts to develop Prime Edited CAR-T products for hematology, immunology and oncology in partnership with Bristol Myers Squibb. In addition, Prime Medicine will continue to pursue additional business development opportunities to accelerate innovation, ensure the broadest application of Prime Editing, and further bolster its financial resources. Also today, Prime Medicine announced that Keith Gottesdiener, M.D., has decided to step down as Chief Executive Officer (CEO) and a member of the Company's Board of Directors, effective immediately. Allan Reine, M.D., Prime Medicine's Chief Financial Officer (CFO), has been named CEO and member of the Board of Directors and Jeff Marrazzo, member of the Company's Board of Directors, has been named Executive Chair. 'The first-in-human data for PM359 announced today represent a landmark moment for the industry and for our company, demonstrating the unequivocal power of Prime Editing to change patients' lives, with a single dose of PM359 providing potentially curative benefit in a matter of weeks,' said Allan Reine, M.D., Chief Executive Officer of Prime Medicine. 'These data also reinforce the critical importance of ensuring Prime Medicine is positioned to withstand the challenges of the current environment, so that we can one day deliver the tremendous promise of Prime Editing to address a wide spectrum of genetic diseases. I am humbled by the opportunity to lead Prime Medicine through this next chapter, and committed to operating with excellence, efficiency and financial discipline as we focus our efforts internally on our Wilson's Disease and AATD programs, where, in the near-term, we have the potential to cure two of the largest genetic liver diseases.' Pipeline Prioritization: Prime Medicine will focus its internal efforts on the development of in vivo programs for the treatment of Wilson's Disease and AATD, two of the largest genetic liver diseases. Prime Medicine expects to file an investigational new drug (IND) and/or clinical trial application (CTA) for its Wilson's Disease program in the first half of 2026 and for its AATD program in mid-2026; initial clinical data from both programs are expected in 2027. Wilson's Disease: Wilson's Disease is a rare and severe disorder caused by excess copper accumulation in the liver and brain that can lead to liver failure and neurocognitive decline and can be fatal without a liver transplant. There are currently no approved disease-modifying therapies for Wilson's Disease, which affects more than 20,000 people in the United States and European Union. AATD: AATD is a progressive, genetic disorder caused by mutations in the SERPINA1 gene, which can result in both lung- and liver-related symptoms, including shortness of breath, wheezing, chronic cough and frequent chest colds, as well as jaundice, ascites, and cirrhosis. There are currently no disease-modifying or curative treatments approved for the approximately 200,000 people in the United States and European Union with AATD, of which 20,000-30,000 people are currently diagnosed. Many patients with AATD ultimately progress to liver failure or severe lung disease, eventually resulting in premature death. Prime Medicine will also continue its in vivo Cystic Fibrosis program with support from the Cystic Fibrosis Foundation, and its efforts to develop Prime Edited CAR-T products for hematology, immunology and oncology in partnership with Bristol Myers Squibb. As announced in conjunction with initial data for PM359 this morning, Prime Medicine is exploring options for the continued clinical development of PM359 external to the company and ceasing further efforts in X-linked CGD. Prime Medicine believes PM359 has the potential to transform the care of p47phox CGD and is committed to working with urgency to identify an appropriate partner to help ensure this important therapy is delivered to patients. Management and Corporate Update Management Update: The Board of Directors has appointed Dr. Reine, Prime Medicine's CFO, as CEO and member of the Board of Directors, effective immediately. Additionally, Jeff Marrazzo, member of the Board of Directors, has been named as Executive Chair. Dr. Reine is a seasoned executive with more than twenty years' experience in the biotechnology industry. Prior to joining Prime Medicine in 2024, Dr. Reine was CFO at Foghorn Therapeutics and, before that, at Pieris Pharmaceuticals. Previously, Dr. Reine spent fifteen years as a healthcare investor managing various healthcare portfolios primarily focused on biotechnology and pharmaceutical companies. In addition, Dr. Reine started his career in biotechnology investment banking and sell-side research. Dr. Reine also serves as Chairman of the Board of ONK Therapeutics. Mr. Marrazzo joined the Prime Medicine Board of Directors in 2023. Prior to that, he founded and built Spark Therapeutics into the world's first fully integrated, commercial gene therapy company, and successfully orchestrated its $4.8 billion sale to Roche in 2019. Under his leadership, Spark developed and launched LUXTURNA® for a rare blinding disorder, the first U.S. Food and Drug Administration (FDA)-approved gene therapy for a genetic disease in the U.S., developed BEQVEZ™, which was licensed to Pfizer and approved by the FDA for hemophilia B, and advanced multiple programs into Phase 3, including several which received Breakthrough Designation. 'The selection of Allan as Prime Medicine's next CEO is the result of thoughtful succession planning, designed to position the company for long-term success, while maintaining the culture of innovative thinking, scientific rigor and collaboration that has always defined the organization and that will continue to fuel its long-term growth,' said Mr. Marrazzo. 'As a seasoned CFO, former investor and physician, Allan brings a combination of financial and scientific acumen, which will enable him to champion and advance Prime Medicine's pipeline, while ensuring the company can successfully navigate the evolving market conditions. I look forward to working closely together as he assumes this new role.' Mr. Marrazzo continued, 'Since its discovery in 2019, Keith has been an incredible advocate for Prime Editing. He was early to recognize the transformative power of this new technology and built Prime Medicine to enable its rapid development. With the announcement of positive initial data for PM359, and the changes in the company as part of the strategic prioritization, today marks a natural time to transition leadership of Prime Medicine. On behalf of the Board, I thank Keith for his visionary leadership and wish him the very best in his next professional endeavor.' Dr. Gottesdiener will continue to support Prime Medicine as a consultant for a period of one year. Corporate Update: Alongside the pipeline prioritization announced today, Prime Medicine is undertaking cost reduction measures and restructuring its team, including reducing its organizational headcount by approximately 25% percent. These initiatives are designed to significantly decrease Prime Medicine's operating expenses and cash burn, reducing anticipated cash needs by almost half through 2027. Dr. Reine added, 'I am deeply grateful to the employees who will be impacted as part of this restructuring. They all contributed mightily to advancing Prime Editing from concept to clinic, and their efforts across research and development and broader company-building will serve as the foundation for our success for many years to come. Importantly, while today's restructuring positions Prime Medicine to efficiently execute towards potentially transformative inflection points in Wilson's Diseases and AATD, we remain unwavering in our long-term ambition to realize the full promise of Prime Editing for a wide spectrum of diseases.' Also today, Prime Medicine announced that it recently engaged in binding arbitration proceedings with Beam Therapeutics, Inc. regarding the parties' 2019 Collaboration and License Agreement (the 'Agreement') and specific to its AATD program. Prime Medicine is committed to honoring the terms of the Agreement, and confident that it has the rights to pursue AATD under the Agreement. Based on its current plans, Prime Medicine continues to expect that its cash, cash equivalents and investments as of March 31, 2025 will be sufficient to fund its operations and capital expenditure requirements into the first half of 2026. 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: liver, lung, and immunology and oncology. 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 © 2025 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 continued development and advancement of its AATD and Wilson's Disease programs, including the timing of the filing of IND and/or CTA applications in mid-2026 and 1H 2026, respectively, and the timing of initial data for both programs in 2027; the initiation, timing, progress, and results of its research and development programs, preclinical studies and future clinical trials, including the release of data related thereto; the potential of Prime Editing to correct the causative mutations of diseases, including of AATD, Wilson's Disease and CF; the breadth of Prime Editing technology and the implementation of its strategic plans for its business, programs, and technology; the potential of Prime Editing as a transformative gene editing technology and its ability to unlock opportunities across thousands of potential indications; its ability to identify an external partner to deliver PM359 therapy in X-linked CGD to patients; its rights to pursue AATD using its approach in connection with the arbitration proceedings with Beam Therapeutics, Inc.; and its expected cash runway. 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; Prime Medicine's expectations regarding the anticipated timeline of its cash runway and future financial performance; 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 and Media Contacts Gregory DearbornPrime Medicine857-209-0696gdearborn@ Hannah DeresiewiczPrecision
Yahoo
22-03-2025
- Business
- Yahoo
Why Prime Medicine Stock Was Climbing Higher This Week
News of a fresh investigational program was putting some zip into the shares of gene-editing company Prime Medicine (NASDAQ: PRME) across recent trading sessions. According to data compiled by S&P Global Market Intelligence, in reaction, bullish investors were propelling the company's stock over 12% higher week to date as of early Thursday evening. Prime's encouraging news came on Tuesday when the company announced it had launched a preclinical program aimed at treating alpha-1 antitrypsin deficiency (AATD). This is a genetic disorder caused by a mutation in a gene that limits the body's capability to produce a necessary protein and can badly affect the lungs and liver. The ambitious biotech's goal is to file an investigational new drug (IND) and/or clinical trial application (CTA) for this with the U.S. Food and Drug Administration (FDA) in the middle of next year. Prime added that the AATD program leverages its universal liver lipid nanoparticle (LNP) to edit the offending gene. In the press release touting its latest investigational path, the biotech quoted CEO Keith Gottesdiener as saying that it exemplifies our strategy of using our proprietary, modular liver LNP to accelerate the development of new Prime Editors, as well as our ability to leverage learnings, regulatory frameworks and manufacturing synergies to efficiently advance our efforts. A new program aimed at combating a pernicious disorder is usually greeted well by biotech investors, and Prime's news was no different. Now, however, comes the hard part. Many eyes will be on the company as it travels down the path of developing that AATD treatment. There's no guarantee it'll succeed, of course, but it's quite encouraging that it's getting into the fight. Ever feel like you missed the boat in buying the most successful stocks? Then you'll want to hear this. On rare occasions, our expert team of analysts issues a 'Double Down' stock recommendation for companies that they think are about to pop. If you're worried you've already missed your chance to invest, now is the best time to buy before it's too late. And the numbers speak for themselves: Nvidia: if you invested $1,000 when we doubled down in 2009, you'd have $304,759!* Apple: if you invested $1,000 when we doubled down in 2008, you'd have $40,808!* Netflix: if you invested $1,000 when we doubled down in 2004, you'd have $517,445!* Right now, we're issuing 'Double Down' alerts for three incredible companies, and there may not be another chance like this anytime soon.*Stock Advisor returns as of March 18, 2025 Eric Volkman has no position in any of the stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. Why Prime Medicine Stock Was Climbing Higher This Week was originally published by The Motley Fool Sign in to access your portfolio
Yahoo
21-03-2025
- Business
- Yahoo
Why Prime Medicine Stock Is Soaring Today
Shares of Prime Medicine (NASDAQ: PRME) are surging on Thursday. The gene-editing company's stock gained 10.5% as of 2 p.m. ET and was up as much as 17% earlier in the day. The move up comes as the S&P 500 and Nasdaq Composite gained 0.4% and 0.7%, respectively. The clinical-stage biotech unveiled a promising new preclinical program targeting a serious disorder of the lungs and liver. Prime Medicine announced yesterday a new preclinical program for treating alpha-1 antitrypsin deficiency (AATD), a genetic disorder affecting lung and liver function. The announcement included plans to file an Investigational New Drug Application or Clinical Trial Application with the FDA by mid-2026. Prime's universal liver lipid nanoparticle (LNP) technology is at the cutting edge of gene therapy. It will be used to edit the SERPINA1 gene, which when mutated is responsible for the condition. Investors are reacting to the expectations of trials beginning by 2026 as well as compelling early data presented by the company in the release. Though the announcement brings some excitement, Prime Medicine is still a clinical-stage biotech company with substantial development risks ahead. A lot can go wrong in the course of developing new therapies. Even if it all goes according to plan, commercialization is years down the line and there is no guarantee another company won't beat it to the punch. However, the company does have a portfolio of therapies further along in the pipeline and it does have impressive proprietary technology. If you are an investor with a high risk tolerance, Prime could be an interesting addition to your portfolio. Ever feel like you missed the boat in buying the most successful stocks? Then you'll want to hear this. On rare occasions, our expert team of analysts issues a 'Double Down' stock recommendation for companies that they think are about to pop. If you're worried you've already missed your chance to invest, now is the best time to buy before it's too late. And the numbers speak for themselves: Nvidia: if you invested $1,000 when we doubled down in 2009, you'd have $299,339!* Apple: if you invested $1,000 when we doubled down in 2008, you'd have $40,324!* Netflix: if you invested $1,000 when we doubled down in 2004, you'd have $501,530!* Right now, we're issuing 'Double Down' alerts for three incredible companies, and there may not be another chance like this anytime soon.*Stock Advisor returns as of March 18, 2025 Johnny Rice has no position in any of the stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. Why Prime Medicine Stock Is Soaring Today was originally published by The Motley Fool Sign in to access your portfolio
Yahoo
28-02-2025
- Business
- Yahoo
Prime Medicine Reports Full Year 2024 Financial Results and Provides Business Updates
-- On track to report initial data from Phase 1/2 clinical trial of PM359 for p47phox CGD in 2025 -- -- IND-enabling studies ongoing for PM577 for Wilson's Disease; expect to file IND and/or CTA in 1H 2026 -- -- Additional high-value programs advancing through preclinical development -- CAMBRIDGE, Mass., Feb. 28, 2025 (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 full year ended December 31, 2024 and provided a business update. 'Entering 2025 marks the next chapter for Prime Medicine as we look to share initial data for our most advanced product candidate, PM359, in chronic granulomatous disease, the only Prime Editor currently in clinical development,' said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. 'If positive, we believe this readout will be a watershed moment for our company, validating Prime Editing's differentiated safety profile and curative potential.' Dr. Gottesdiener added, 'In parallel, we are advancing our Prime Editors for Wilson's Disease and Cystic Fibrosis. We are particularly encouraged by recent progress in Wilson's Disease, a program that we believe will be a fundamental driver of our long-term growth and remains on-track for clinical entry in 2026. We look forward to sharing new in vivo data from across our preclinical programs in 2025 as we continue to accelerate our pipeline through both internal efforts and additional strategic partnerships.' Prime Medicine's Pipeline: Prime Medicine is advancing a set of high-value programs across its core areas of focus (hematology, immunology and oncology, liver, and lung). These include ex vivo hematopoietic stem cell (HSC) programs for the treatment of p47phox chronic granulomatous disease (CGD) and X-linked CGD; a lipid nanoparticle (LNP) Prime Editor for the treatment of Wilson's Disease; a LNP or adeno-associated virus (AAV) Prime Editors for the treatment of cystic fibrosis (CF); and ex vivo T-cell therapies, which are being developed in collaboration with Bristol Myers Squibb. Anticipated Upcoming Milestones: Chronic Granulomatous Disease (CGD): Announce initial clinical data from Cohort 1 in Phase 1/2 trial of PM359 for p47phox CGD in 2025. The initial readout will include safety and engraftment data, as well as key outcome measures: the rate of Prime Editing in hematopoietic stem cells and the reconstitution of NADPH oxidase activity as measured by the DHR assay. Wilson's Disease: Advance PM577 through investigational new drug (IND)-enabling studies for the treatment of Wilson's Disease patients with the most prevalent Wilson's Disease mutation in the United States. File IND and/or clinical trial application for PM577 in the first half of 2026. Full Year 2024 Financial Results Research and Development (R&D) Expenses: R&D expenses were $155.3 million for the year ended December 31, 2024, as compared to $147.9 million for the year ended December 31, 2023. The increase in R&D expenses was driven by increases in facilities, personnel and clinical expenses related to PM359. General and Administrative (G&A) Expenses: G&A expenses were $50.2 million for the year ended December 31, 2024, as compared to $43.4 million for the year ended December 31, 2023. The increase in G&A expenses was driven by increases in facilities and personnel-related expenses. Net Loss: Net loss was $195.9 million for the year ended December 31, 2024, as compared to $198.1 million for the year ended December 31, 2023. Cash Position: As of December 31, 2024, cash, cash equivalents, investments, and restricted cash were $204.5 million, as compared to $135.2 million as of December 31, 2023. Financial Guidance Based on its current operating plans, Prime Medicine expects that its cash, cash equivalents and investments as of December 31, 2024 will be sufficient to fund its operating expenses and capital expenditure requirements into the first half of 2026. 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 its 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 From time to time Prime Medicine may use its website, our X, formerly Twitter, account (@PrimeMedicine) or its LinkedIn profile at to distribute material information. Its financial and other material information is routinely posted to and accessible on the Investors section of its website, available at Investors are encouraged to review the Investors section of its website because the Company may post material information on that site that is not otherwise disseminated by the Company. Information that is contained in and can be accessed through the Company's website or its social media is not incorporated into, and does not form a part of, this press release. © 2025 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 continued development and advancement of PM359 and PM577, including the ongoing Phase 1/2 trial of PM359 and the timing of the anticipated release of initial clinical data readout for p47phox CGD in 2025, and the timing for completion of IND-enabling studies for PM577; 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 of PM359 to correct the causative mutation of CGD; the potential of Prime Editing to correct the causative mutations of Wilson's Disease and CF; 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, and other rare and non-rare liver indications; the ability to demonstrate, and the timing of, preclinical proof-of-concept in vivo for multiple programs; the further advancement of Prime Editors to maximize their versatility, precision and efficiency; 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 initiation, timing, progress, and results of its research and development programs, preclinical studies and future clinical trials, including the release of data related thereto; 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; 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; the potential of Prime Editing to unlock opportunities across thousands of potential indications; and its expected cash runway . 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; Prime Medicine's expectations regarding the anticipated timeline of its cash runway and future financial performance; 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 Contacts Gregory DearbornPrime Medicine713-503-3364gdearborn@ Hannah DeresiewiczPrecision Media Contact Dan Budwick, 1ABdan@ Condensed Consolidated Balance Sheet Data(unaudited) December 31, (in thousands) 2024 2023 Cash, cash equivalents, and investments $ 190,442 $ 121,665 Total assets 297,508 193,851 Total liabilities 144,359 60,780 Total stockholders' equity 153,149 133,071 Condensed Consolidated Statement of Operations(unaudited) Year Ended December 31, (in thousands, except share and per share amounts) 2024 2023 Revenue: Collaboration revenue — related party $ 1,609 $ — Collaboration revenue 1,374 — Total revenue 2,983 — Operating expenses: Research and development 155,289 147,905 Settlement payment — related party — 13,500 General and administrative 50,161 43,387 Total operating expenses 205,450 204,792 Loss from operations (202,467 ) (204,792 ) Other income: Interest income 3,522 2,811 Accretion (amortization) of investments 3,507 5,677 Change in fair value of short-term investment — related party (485 ) (2,382 ) Other income, net 41 274 Total other income, net 6,585 6,380 Net loss before income taxes (195,882 ) (198,412 ) Benefit from income taxes — 279 Net loss attributable to common stockholders $ (195,882 ) $ (198,133 ) Net loss per share attributable to common stockholders, basic and diluted $ (1.65 ) $ (2.18 ) Weighted-average common shares outstanding, basic and diluted 118,600,381 90,969,327 Sign in to access your portfolio
