Core Insights - Prime Medicine, Inc. reported a quarterly loss of $0.32 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.28, representing an earnings surprise of -14.29% [1] - The company generated revenues of $1.23 million for the quarter ended September 2025, missing the Zacks Consensus Estimate by 75.35%, compared to revenues of $0.21 million a year ago [2] - The stock has increased approximately 43.2% since the beginning of the year, outperforming the S&P 500's gain of 14.3% [3] Financial Performance - Over the last four quarters, Prime Medicine has only surpassed consensus EPS estimates once [2] - The current consensus EPS estimate for the upcoming quarter is -$0.26 on revenues of $8.95 million, and for the current fiscal year, it is -$1.25 on revenues of $7.63 million [7] Industry Context - The Medical - Biomedical and Genetics industry, to which Prime Medicine belongs, is currently ranked in the top 37% of over 250 Zacks industries, indicating a favorable outlook compared to the bottom 50% [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact investor sentiment [5] Future Outlook - The sustainability of Prime Medicine's stock price movement will largely depend on management's commentary during the earnings call and the subsequent revisions of earnings estimates [3][4] - The estimate revisions trend for Prime Medicine was mixed ahead of the earnings release, resulting in a Zacks Rank 3 (Hold) for the stock, indicating expected performance in line with the market [6]

Revenue Generation - The company has not generated any revenue from product sales to date and does not expect to do so in the foreseeable future, with revenues primarily from research collaboration and license agreements [100]. - The company has not generated any revenue from product sales and does not expect to do so until successful completion of clinical development and regulatory approval [127]. Research and Development Expenses - Research and development expenses for the three months ended September 30, 2025, increased by $3.7 million to $43.99 million compared to $40.34 million in the same period of 2024 [108]. - Research and development expenses for the nine months ended September 30, 2025, totaled $125.93 million, an increase of $4.74 million from $121.19 million in 2024 [113]. - Personnel expenses for research and development decreased by $5.99 million for the nine months ended September 30, 2025, primarily due to workforce reductions [113]. - The company anticipates an increase in research and development expenses as it advances product candidates and conducts clinical trials [103]. Clinical Trials and Product Development - The company plans to file IND and/or CTA for Wilson's Disease in the first half of 2026 and for AATD in mid-2026, leveraging its modular technology [97]. - The Phase 1/2 trial for PM359 in chronic granulomatous disease showed promising results, with the first patient achieving 71% dihydrorhodamine positivity by Day 60 [99]. - The company is focused on advancing its in vivo liver franchise, targeting Wilson's Disease and Alpha-1 Antitrypsin Deficiency [97]. Financial Position and Cash Flow - As of September 30, 2025, the company had cash, cash equivalents, and investments totaling $227.0 million, including restricted cash [120]. - The company entered into an Open Market Sale Agreement with Jefferies LLC to issue and sell shares of common stock for up to $300.0 million [121]. - In August 2025, the company issued 43,700,000 shares of common stock at a public price of $3.30 per share, resulting in net proceeds of approximately $138.4 million [122]. - For the nine months ended September 30, 2025, the net cash used in operating activities was $125.3 million, compared to $139.1 million for the same period in 2024 [123]. - The company reported a net loss of $155.1 million for the nine months ended September 30, 2025 [124]. - Net cash provided by financing activities for the nine months ended September 30, 2025, was $151.0 million, driven primarily by stock issuances [126]. - The company expects to require additional funding to support ongoing research and development activities and potential commercialization expenses [128]. - The company believes its existing cash and investments will be sufficient to fund operations into 2027, but acknowledges the possibility of exhausting resources sooner [128]. Other Income and Expenses - General and administrative expenses decreased by $2.9 million to $11.21 million for the three months ended September 30, 2025, compared to $14.10 million in 2024, primarily due to a reduction in personnel expenses [110]. - The company reported a total other income of $3.39 million for the three months ended September 30, 2025, compared to $1.71 million in 2024, driven by changes in fair value of short-term investments [111]. Risk Factors - The company is exposed to interest rate risk related to its investment portfolio, which totaled $227.0 million as of September 30, 2025 [141].

Exhibit 99.1 Prime Medicine Reports Third Quarter 2025 Financial Results and Provides Business Updates -- New preclinical data for PM577 in Wilson's Disease (WD) to be presented at AASLD; on-track to file IND and/or CTA in H1'26, with initial clinical data expected in 2027 -- -- Nominated PM647 as development candidate for Alpha-1 Antitrypsin Deficiency (AATD); on-track to file IND and/or CTA in mid- 2026, with initial clinical data expected in 2027 -- -- Strengthened leadership team with the appointment of ...

Core Insights - Prime Medicine is advancing its pipeline with new preclinical data for PM577 in Wilson's Disease and has nominated PM647 for Alpha-1 Antitrypsin Deficiency, with both expected to file IND and/or CTA applications in the coming years [1][2][3] Pipeline Developments - The company is focused on liver-targeted programs for Wilson's Disease and Alpha-1 Antitrypsin Deficiency, with IND filings anticipated in H1 2026 for AATD and H1 2027 for WD, and initial clinical data expected in 2027 [3][4] - Prime Medicine is also developing a Cystic Fibrosis program and exploring collaborations, including a partnership with Bristol Myers Squibb for Prime Edited CAR-T products [4][11] Leadership and Corporate Updates - Matthew Hawryluk, Ph.D., M.B.A., has been appointed as Chief Business Officer to enhance business development and strategic collaborations [1][10] - A virtual KOL event is scheduled for November 12, 2025, to discuss the company's strategy for Wilson's Disease [1][10] Financial Performance - For Q3 2025, Prime Medicine reported R&D expenses of $44.0 million, up from $40.3 million in Q3 2024, primarily due to increased license and intellectual property costs [10][20] - General and Administrative expenses decreased to $11.2 million from $14.1 million year-over-year, attributed to workforce reductions [10][20] - The net loss for Q3 2025 was $50.6 million, slightly improved from a net loss of $52.5 million in Q3 2024 [10][20] - As of September 30, 2025, the company had cash and investments totaling $213.3 million, sufficient to fund operations into 2027 [8][18]

Core Insights - Prime Medicine, Inc. has appointed Matthew Hawryluk, Ph.D., M.B.A. as Chief Business Officer to lead corporate and business development initiatives, corporate strategy, and alliance management [1][2][4] - The company aims to expand the reach of its Prime Editing technology through strategic partnerships and collaborations, which are considered a core pillar of its strategy [2][4] - Dr. Hawryluk brings nearly two decades of experience in the biotechnology industry, having previously held leadership roles at AIRNA Corporation and Gritstone bio, Inc., where he led significant financing and collaborations [2][3] Company Overview - Prime Medicine is focused on delivering a new class of one-time curative genetic therapies using its proprietary Prime Editing platform, which allows precise and efficient gene editing [5][6] - The company is advancing a diversified portfolio of investigational therapeutic programs targeting liver, lung, immunology, and oncology diseases, with plans to expand into additional genetic and immunological diseases, cancers, and infectious diseases [6][7] - Prime Editing technology has the potential to repair various genetic mutations and is designed to minimize unwanted DNA modifications, unlocking opportunities across thousands of potential indications [5][6]

Core Insights - Prime Medicine, Inc. is set to host a virtual event on November 12, 2025, to discuss its liver disease franchise, particularly focusing on Wilson's Disease [1][2] - The company plans to file an investigational new drug (IND) and/or clinical trial application (CTA) for PM577, targeting H1069Q-driven Wilson's Disease, in the first half of 2026, with initial clinical data expected in 2027 [1][8] Company Overview - Prime Medicine is a biotechnology company focused on developing a new class of one-time curative genetic therapies using its proprietary Prime Editing platform, which allows precise and efficient gene editing [4][5] - The company is advancing a diversified portfolio of investigational therapeutic programs in core areas such as liver, lung, immunology, and oncology, targeting diseases with well-understood biology [5] Strategic Focus - The upcoming event will feature presentations from the management team and Dr. Michael Schilsky, highlighting the treatment landscape for Wilson's Disease, which currently lacks a curative therapy [2][8] - Prime Medicine aims to leverage the modularity of its Prime Editing platform to accelerate the development of multiple Prime Editors for Wilson's Disease and potentially other diseases [8][9] Market Opportunity - The company identifies a significant global market opportunity for Wilson's Disease treatment, noting that current standard-of-care options are limited by compliance, cost, and tolerability [8] - Prime Editing technology has the potential to provide durable cures by precisely addressing the genetic causes of Wilson's Disease, which could impact millions of patients [8][9]

Core Insights - Prime Medicine, Inc. is set to host a virtual event on November 12, 2025, to discuss its liver disease franchise, particularly focusing on Wilson's Disease [1][2] - The company plans to file an investigational new drug (IND) and/or clinical trial application (CTA) for PM577, targeting H1069Q-driven Wilson's Disease, in the first half of 2026, with initial clinical data expected in 2027 [1][9] Company Overview - Prime Medicine is a biotechnology company focused on developing one-time curative genetic therapies using its proprietary Prime Editing platform, which allows precise and efficient gene editing [4][5] - The company aims to address a range of genetic mutations across various tissues and diseases, potentially unlocking thousands of indications [4][5] Strategic Focus - Prime Medicine is concentrating on high-value programs in liver, lung, and immunology and oncology, with a clear clinical development path for each [5] - The company intends to leverage the modularity of its Prime Editing platform to accelerate the development of therapies for Wilson's Disease and other genetic conditions [8][9] Market Opportunity - There is currently no curative therapy for Wilson's Disease, and existing treatments face challenges related to compliance, cost, and tolerability [8] - Prime Editing has the potential to provide a durable cure for Wilson's Disease by precisely addressing its genetic causes [8][9]

Summary of the Genetic Medicines Conference Call Industry Overview - The conference focused on the genome editing industry, highlighting advancements in technologies and programs related to gene editing therapies [1][2] - Key players in the industry include Arbor Biotechnologies, Editas Medicine, Prime Medicine, Yultec Therapeutics, and GenEdit Bio [2] Core Points and Arguments Technological Advancements - The genome editing field has evolved to include various technologies such as nuclease editing, base editing, and prime editing, with discussions on how these technologies will coexist and evolve [15][19] - Companies are focusing on differentiating their products based on efficacy and safety profiles, with a consensus that multiple technologies can coexist in the ecosystem [18][19] Clinical and Commercial Challenges - The need for differentiation in product offerings is emphasized, particularly in the context of rare diseases where the patient pool is limited [50] - The commercial viability of one-time gene editing therapies is debated, with a focus on the importance of demonstrating transformational efficacy to ensure market success [50][51] - Companies are encouraged to consider the economic arguments for one-and-done therapies, highlighting the potential for significant patient benefits [50][51] Delivery Mechanisms - Delivery methods are critical to the success of gene editing therapies, with discussions on the importance of optimizing both the delivery vehicle and the editing technology [28][32] - LNPs (lipid nanoparticles) are currently the leading delivery technology for in vivo applications, particularly for liver-targeted therapies [31][40] - Companies are exploring alternative delivery methods, including non-viral approaches, to enhance accessibility and reduce costs [59] Lessons Learned from Early Therapies - The importance of focusing on clinical significance and commercial viability from the early stages of development is highlighted [55] - Companies are learning from the first wave of therapies, particularly regarding the need for rapid development and differentiation in a competitive landscape [26][27] Other Important Insights - The discussion included the potential for gene editing to revolutionize treatment for various diseases, with a focus on the need for a robust business model to support these innovations [61] - The evolving payer landscape is expected to adapt to the new technologies, potentially leading to multiple approvals and launches in the coming years [63][64] - The conference concluded with optimism about the future of gene editing therapies, emphasizing the importance of demonstrating both technical success and commercial viability [64]

Summary of Prime Medicine Conference Call - October 14, 2025 Company Overview - **Company**: Prime Medicine (NasdaqGM:PRME) - **Focus**: Development of novel gene and cell therapies utilizing a prime editing platform, which is described as a next-generation gene editing technology that is both versatile and safe [2][4][5] Core Technology and Advantages - **Prime Editing**: - Makes single-stranded breaks in DNA, avoiding double-stranded breaks, which reduces off-target effects and chromosomal rearrangements [5][6] - Capable of writing new genetic code directly into the genome, allowing for permanent changes [6][7] - Can address various mutations, including transition, transversion, and frameshift mutations [7][8] Pipeline Focus - **Current Pipeline**: - Focused on liver diseases, specifically: - **Wilson's Disease**: Expected to enter the clinic in the first half of 2026 [8][9] - **Alpha-1 Antitrypsin Deficiency**: Anticipated IND filing in mid-2026 [9][10] - Future programs include cystic fibrosis, with a goal to treat over 90% of patients [10][11] Discontinued Programs - **Chronic Granulomatous Disease (CGD)**: - Program discontinued due to limited patient population (approximately 1,000 patients in the US) and commercial viability concerns despite strong initial clinical data [12][14] Market Opportunity - **Wilson's Disease**: - Estimated patient population in the US: 10,000 to 11,000 [28] - Potential to treat over 60% of patients with various mutations, leading to a global market opportunity exceeding 20 billion dollars [30][29] Clinical Development and Data Expectations - **IND Filing**: - Guidance for IND filing for Wilson's Disease in the first half of 2026, with clinical data expected in 2027 [31][50] - **Trial Design**: - Biomarkers for Wilson's Disease include copper PET scans and ceruloplasmin levels to assess treatment efficacy [35][36] Competitive Landscape - **Alpha-1 Antitrypsin Deficiency**: - Acknowledgment of competition in the space, but confidence in prime editing's ability to provide a best-in-class therapy due to its endogenous control mechanism [37][38] Delivery Mechanism - **Lipid Nanoparticle (LNP) Technology**: - Universal liver LNP has shown high hepatocyte editing efficiency and is expected to facilitate rapid progression of multiple programs [42][44] - Potential for scalability beyond liver applications, with ongoing exploration of delivery methods for other organs [44][45] Collaboration and Partnerships - **Strategic Collaborations**: - Emphasis on the importance of partnerships for funding and expanding the reach of prime editing technologies [46][48] - Successful collaboration with BMS in ex vivo CAR T therapies, securing $110 million upfront [48] Financial Position - **Cash Runway**: - Approximately $260 million at the end of Q2 2025, expected to sustain operations into 2027 [50] Key Catalysts - **Upcoming Milestones**: - Initiation of clinical programs for Wilson's Disease and Alpha-1 Antitrypsin Deficiency in 2026, with significant data readouts anticipated in 2027 [50]

Core Insights - Several biotech companies experienced significant after-hours stock gains due to clinical trial updates, funding announcements, and upcoming product showcases Group 1: Prime Medicine Inc. (PRME) - Shares rose 8.14% in after-hours trading, reaching $5.05 after a regular session close of $4.67, which was up 16.46% [2] - The company reported a narrowed net loss of $52.6 million or $0.41 per share on revenue of $1.12 million, highlighting early clinical data from its CGD program [3] - Upcoming catalysts include planned IND filings for Wilson's Disease and Alpha-1 Antitrypsin Deficiency in 2026, with initial clinical data expected in 2027 [4] Group 2: Meihua International Medical Technologies Co. Ltd. (MHUA) - Shares increased by 7.55% in after-hours trading, reaching $0.4444 after a regular session close of $0.4132, which was down 2.62% [4] - The company is set to report its quarterly earnings on September 18, 2025 [5] Group 3: Elutia Inc. (ELUT) - Shares rose 10.17% in after-hours trading, reaching $1.30 after closing at $1.18, which was down 3.28% [5] - Elutia announced clinical data showing its biologic envelopes significantly ease reoperations for cardiac implantable electronic devices, reducing procedural difficulty by over 40% [6] - The company will exhibit its EluPro antibiotic-eluting BioEnvelope at the Vizient Innovative Technology Exchange on September 17, 2025 [6] Group 4: Aligos Therapeutics Inc. (ALGS) - Shares increased by 2.79% in after-hours trading, reaching $9.20 after closing at $8.95, which was down 4.48% [7] - Aligos presented six preclinical studies at the 2025 International HBV Meeting, including new data on its capsid assembly modulator and a novel ASO strategy targeting hepatitis delta virus [8] - Upcoming catalysts include continued enrollment and data updates from the B-SUPREME study and a scheduled earnings report on November 5, 2025 [9] Group 5: Bionano Genomics Inc. (BNGO) - Shares fell 35.74% to $1.78 before rebounding 6.74% in after-hours trading to $1.90 [10] - The decline followed a $10 million public offering priced at $2.00 per share, expected to close around September 17, 2025 [11] - Bionano plans to use the funds for working capital and general corporate purposes, with recent publications highlighting the utility of its Optical Genome Mapping in cancer biomarker analysis [11] Group 6: Cognition Therapeutics Inc. (CGTX) - Shares climbed 6.21% in after-hours trading, reaching $1.71 after closing at $1.61, which was up 4.55% [12] - The company’s lead candidate, zervimesine, reached 75% enrollment in its Phase 2 START study for early Alzheimer's disease, backed by the National Institute on Aging [13] - Cognition recently closed a $30 million registered direct offering to support Phase 3 development [13]