Delivering on the promise of Prime Editing Corporate Presentation January 2026 1 Forward Looking Statements This presentation contains forward-looking statements of Prime Medicine, Inc. ("Prime", "we" or "our") within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements contain information about our current and future prospects and our operations, which are based on currently available information. All statements other than statements of historica ...

Core Insights - Prime Medicine Inc. (NASDAQ:PRME) is recognized as a promising biotech penny stock, with LifeSci Capital initiating coverage and assigning an Outperform rating along with a $6 price target, emphasizing its innovative gene editing treatments for high unmet medical needs [1][2] Group 1: Company Overview - Prime Medicine Inc. focuses on delivering genetic therapies through gene editing technology to address a wide range of diseases in the US [3] Group 2: Clinical Developments - The company announced Phase 1/2 clinical data for its investigational therapy PM359, published in the New England Journal of Medicine, demonstrating the first-in-human safety and efficacy of Prime Editing [1] - In the clinical trial involving PM359, two patients (one adult and one pediatric) with chronic granulomatous disease (CGD) showed rapid neutrophil and platelet engraftment, achieving 69% and 83% dihydrorhodamine-positive neutrophils by Day 30, significantly exceeding the 20% threshold for clinical benefit [2] - Both participants in the trial have remained free of new CGD-related complications, indicating successful long-term gene correction in hematopoietic stem cells [2] Group 3: Market Potential - LifeSci Capital expressed optimism regarding the commercial and clinical opportunities for Prime Medicine, particularly in the treatment landscapes of Wilson's disease and alpha-1 antitrypsin deficiency (AATD) [1]

Prime Medicine, Inc. (NASDAQ:PRME) is among the 12 Best Genomics Stocks to Invest In. Prime Medicine Announces NEJM Publication Of PM359 Data Prime Medicine, Inc. (NASDAQ:PRME) stated on December 7, 2025, that the New England Journal of Medicine has published Phase 1/2 clinical results for PM359, its experimental autologous hematopoietic stem cell therapy for p47phox chronic granulomatous condition. Prime Editing for p47-phox Chronic Granulomatous Disease is a publication that presents preliminary data f ...

Group 1: Biotech Sector Overview - The healthcare sector has become the top-performing sector over the last 3 months as investors rotate away from high-valuation tech and AI stocks [1] - The biotech sub-sector is highlighted as particularly interesting for 2026, with the XBI (SPDR S&P Biotech ETF) serving as a proxy for small and mid-cap companies [1] - A historic shift in sentiment is observed, with the XBI achieving a rare streak of 6 consecutive months of positive returns [1] Group 2: Market Dynamics and Investor Sentiment - Investors are moving towards healthcare due to concerns over concentrated tech positions and favorable valuations in healthcare [1] - Clarity around drug pricing is identified as a major positive catalyst for pharmaceutical and biotech companies [1] - A surge in M&A activity is noted, with over 20 deals worth more than $500 million in 2025 alone [1] Group 3: Small-Cap Market Insights - The small-cap trade is expected to continue, but leadership within this category is set to change, focusing back on earnings growth [2] - The recent rally in the Russell index has been led by speculative areas, including biotech companies, which currently do not produce earnings [2] - Approximately 30% of trading volume is in stocks priced under $5, indicating young investors' interest in speculative names [2] Group 4: Biotech Penny Stocks - Rezolute Inc. (NASDAQ:RZLT) is identified as a top biotech penny stock with an average upside potential of 68.54% [7] - Prime Medicine Inc. (NASDAQ:PRME) is also highlighted, with an average upside potential of 71.43% and recent positive clinical data [11] - Both companies are involved in innovative treatments, with Rezolute focusing on congenital hyperinsulinism and Prime Medicine on gene editing therapies [10][14]

Core Insights - Prime Medicine, Inc. has published Phase 1/2 clinical data for PM359, an investigational autologous hematopoietic stem cell product for p47phox chronic granulomatous disease (CGD), in the New England Journal of Medicine [1][2] - The data indicates rapid neutrophil and platelet engraftment, durable restoration of NADPH oxidase activity, and early clinical benefits without safety concerns, marking the first-in-human demonstration of Prime Editing's safety and efficacy [2][3] Company Overview - Prime Medicine is focused on developing a new class of one-time curative genetic therapies using its proprietary Prime Editing platform, which aims to make precise edits in genes while minimizing unwanted modifications [4] - The company is advancing a diversified portfolio of therapeutic programs targeting liver, lung, immunology, and oncology diseases, with plans to expand into additional genetic and immunological diseases, cancers, and infectious diseases [5] Clinical Trial Details - The Phase 1/2 trial involved two patients with a history of CGD complications, both of whom showed significant improvements, including 69% and 83% dihydrorhodamine-positive neutrophils by Day 30, exceeding the 20% threshold for clinical benefit [6] - Both patients remained free of new CGD-related complications post-infusion, with one patient stopping mesalamine treatment without flare-ups, and the other showing decreased levels of fecal calprotectin [6]

Core Insights - Prime Medicine is advancing its pipeline with new preclinical data for PM577 in Wilson's Disease (WD) to be presented at AASLD, with plans to file an IND and/or CTA in the first half of 2026 and initial clinical data expected in 2027 [1][4][5] - The company has nominated PM647 as a development candidate for Alpha-1 Antitrypsin Deficiency (AATD), also on track to file an IND and/or CTA in mid-2026, with initial clinical data anticipated in 2027 [1][5] - The leadership team has been strengthened with the appointment of Matthew Hawryluk, Ph.D., M.B.A. as Chief Business Officer [1][4] Pipeline Developments - Prime Medicine is focused on in vivo programs targeting Wilson's Disease and Alpha-1 Antitrypsin Deficiency, with IND and/or CTA filings expected in 2026 and initial clinical data in 2027 [5][6] - The company is also advancing a Cystic Fibrosis program supported by the Cystic Fibrosis Foundation and developing Prime Edited CAR-T products in partnership with Bristol Myers Squibb [6] Financial Performance - For the quarter ended September 30, 2025, R&D expenses were $44.0 million, up from $40.3 million in the same period of 2024, primarily due to license and intellectual property costs [10][12] - General and Administrative expenses decreased to $11.2 million from $14.1 million year-over-year, attributed to reduced personnel costs [10][12] - The net loss for the quarter was $50.6 million, compared to a net loss of $52.5 million in the prior year [10][12] Cash Position - As of September 30, 2025, the company reported cash, cash equivalents, and investments totaling $213.3 million, which is expected to fund operations into 2027 [10][21]

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 - 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]