Prime Medicine, Inc. · NASDAQ:PRME
Prime Medicine — Investor Presentation (January 2025)
38 slides from Prime Medicine's investor presentation, Jan 13, 2025
Slide 1: Delivering on the promise of Prime Editing
1 Delivering on the promise of Prime Editing January 2025 Corporate Presentation
Slide 2: Forward-looking statements
2 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 operat
Slide 3: Advancing Prime Editing to change the course of how diseases are treated
3 We are advancing Prime Editing to change the course of how diseases are treated. We aim to provide safe, effective and curative treatments, which offer lifelong benefit to patients.
Slide 4: Rapid Progress Since Inception: On the Cusp of Clinical Validation
4 Platform Validation Clinical ValidationPlatform Discovery Sustained Growth and Commercial Success Rapid Progress Since Inception: Prime Medicine is on the Cusp of Clinical Validation and Sustainable, Long-Term Value Creation 2020 - 2023 2024 - 2026 Advance first Prime Editors into the clinic. Focu
Slide 5: 2024 Marked a Year of Significant Accomplishment
5 CORPORATE CGD = chronic granulomatous disease; CF = cystic fibrosis 2024 Marked a Year of Significant Accomplishment Executing Against Prime Medicine’s Strategic Priorities PIPELINE PLATFORM Matured into clinical-stage company: initiated Phase 1/2 trial of PM359 in p47phox CGD Advanced next wave o
Slide 6: Entering a New Era of Gene Editing: Generating Clinical Data
6 Prime Medicine is Entering a New Era of Gene Editing: Generating Clinical Data for Multiple Programs, Leveraging Platform Modularity Secure multiple additional strategic partnerships to accelerate our pipeline and bolster our financial resources 2025 2026 2027+ PM359 for p47phox CGD: Announce init
Slide 7: Prime Editing Can Edit, Correct, Insert and Delete DNA Sequences
7 We Believe Prime Editing is the Only Gene Editing Technology That Can Edit, Correct, Insert and Delete DNA Sequences in Any Target Tissue REPLACEMENT Prime Editor Deletion / Insertion Correction p47 Chronic Granulomatous Disease (CGD) Point Mutation Correction Wilson’s Disease Targeted Full Gene I
Slide 8: Prime Editing Has Highly Differentiated Safety Profile
8 No detectable double strand breakage No detectable off-target deletions, chromosomal translocations or rearrangements No detectable off-target edits No detectable bystander edits 1Analysis of edited CD34+ cells from CGD program: Targeted in vitro Analysis of 550 potential off-target sites of off-t
Slide 9: Our Focused Strategy: Near-Term, High Value Potential
9 Our Focused Strategy: Pursue Opportunities with Near-Term, High Value Potential, Where Prime Editing Offers Differentiated Approach Prioritized Programs Meet Six Criteria: 1 Unmet medical need 2 Commercial potential 3 Pipeline and platform modularity 4 Clinical development pathway 5 Regulatory con
Slide 10: Platform Modularity Accelerates and De-Risks Ongoing Efforts
10 Prime Editing Platform Modularity Accelerates and De-Risks Ongoing Efforts, Enables Rapid Generation of New Product Candidates Prime Editors Manufacturing Delivery Off-Target Assays Nonclinical Regulatory Clinical
Slide 11: Our Pipeline: Aligned to Core Modular Platforms
11 Modular Platform Indication Delivery Discovery Lead optimization IND- enabling Phase 1/2 HEMATOLOGY, IMMUNOLOGY & ONCOLOGY p47phox Chronic Granulomatous Disease (CGD) ex vivo X-linked CGD (with PASSIGE ) ex vivo Ex vivo CAR-T1 (with PASSIGE ) ex vivo LIVER Wilson’s Disease LNP Undisclosed Program
Slide 12: Hematology, Immunology and Oncology
12| Hematology, Immunology and Oncology
Slide 13: Chronic Granulomatous Disease: p47phox CGD and XCGD
13Confidential | Chronic Granulomatous Disease p47phox CGD and XCGD
Slide 14: Advancing Prime Editors for CGD, a Disease of Significant Unmet Need
14HSCT = hematopoietic stem cell transplant; GvHD = graft vs. host disease Advancing Prime Editors for Chronic Granulomatous Disease (CGD), A Disease of Significant Unmet Need Rare genetic disease, characterized by defective neutrophil function • Serious life-threatening disease presents in childhoo
Slide 15: Prime Medicine's CGD Franchise Covers Vast Majority of Patient Population
15 Current Status Initial data from Phase 1/2 clinical trial expected in 2025 Preclinical development ongoing Rapidly Advancing IND cleared in April 2024, within 30 days of submission Leveraging modular elements of PM359 program to inform and accelerate advancement Targeted Mutations delGT mutation
Slide 16: PM359: Preclinical Data Support Advancement for CGD
16 • Full immune system reconstitution by Prime Edited LT-HSCs • Edited LT-HSC derived neutrophils had normal enzymatic activity (NADPH oxidase) HSC = hematopoietic stem cell; LT-HSC = long term HSC; DHR = dihydrorhodamine; * normalized to healthy donor control. 1Minimal threshold for patient benefi
Slide 17: PM359: Clinical Trial Enrolling, Initial Clinical Data in 2025
17 Cohort 2: n=2-3, ages 12-17 Cohort 3: n=2-3, ages 6-11 Cohort 1: n=2-3, age ≥ 18 DP = drug product; GMP = good manufacturing practice; QC = quality control; IND = investigational new drug *Pending alignment with FDA PM359: Clinical Trial Enrolling, Initial Clinical Data in 2025 PM359 is comprised
Slide 18: PASSIGE has Potential to Treat X-linked CGD
18 PASSIGE has Potential to Treat X-linked CGD (XCGD) ’All in one’ delivery of PASSIGE reagents for CYBB gene replacement in CD34+ cells has potential to treat >90% of X-linked CGD patients PASSIGE demonstrated high multiplex editing efficiency Potential synergies to accelerate leveraging p47phox CG
Slide 19: Ex vivo CAR-T BMS Collaboration
19Confidential | Ex vivo CAR-T BMS Collaboration
Slide 20: Strategic License and Broad Collaboration Agreement with Bristol Myers Squibb
20 • $110 million upfront • >$3.5 billion in potential milestones, including: ‒ $185 million in preclinical milestones ‒ $1.2 billion in development milestones ‒ More than $2.1 billion in commercial milestones ‒ Royalties on net sales • Multiple targets in immunological diseases and cancer, beyond r
Slide 21: CAR-T: PASSIGE and Multiplex Prime Editing Foundation of BMS Collaboration
21TRAC = T-cell receptor alpha constant; Data presented at ASH, December 2022, ASGCT, May 2023 and ASH, December 2023 CAR-T: PASSIGE and Multiplex Prime Editing is the Foundation of Prime Medicine’s Collaboration with BMS Platform modularity has potential to accelerate development of additional CAR-
Slide 22: Efficiently Multiplex Edit CAR-T Cells
22 Maintained efficiency with five multiplex edits B2M = b2 microglobulin TRAC = T-cell receptor alpha constant Beyond Precisely Inserting a Chimeric Antigen Receptor, We Can Simultaneously and Efficiently Multiplex Edit CAR-T Cells Prime Editors can be multiplexed to introduce multiple genomic modi
Slide 23: Liver
23| Liver
Slide 24: Advancing Prime Editors for Wilson's Disease Using Universal LNP
24 • Disease severity and opportunity ‒ Common liver and systemic disease presenting in teens to 20s ‒ Leads to liver failure, neurocognitive decline and premature death ‒ Greater than 20,000 patients in US and Europe, 30-50% harboring H1069Q mutation ‒ R778L is the predominant mutation in Asian pop
Slide 25: Prime Editor Efficiently Corrects the H1069Q Mutation In Vivo
25 Prime has initiated IND-enabling activities planning for a H1’26 IND and/or CTA application(s) Prime Editor Efficiently Corrects the H1069Q Mutation In Vivo Editing efficiency and copper reduction in fully humanized homozygous p.H1069Q ATP7B mouse model Efficient correction of the H1069Q transver
Slide 26: Up to 51% Precise Editing with Universal LNP in NHP
26 Up to 51% Precise Editing Achieved with Universal LNP-Formulated Surrogate H1069Q Prime Editor In Vivo (NHP) ➢ Well-tolerated with no acute reactions, clinical observations, or body weight changes ➢ Transient LFT elevations ➢ No change in platelets, coagulation or blood count ➢ No change in blood
Slide 27: Proprietary LNP-formulated Prime Editor Multi-Component Drug Product
27 Proprietary LNP–formulated Prime Editor is a Complex Multi-Component Drug Product Designed to Support Current and Future Liver Programs • Control particle size and stability • Stealth coating reduces serum interactions and increases half-life PEG Lipids • Stabilize and improve LNP pharmacokinetic
Slide 28: Lung
28| Lung
Slide 29: Prime Editing Could Benefit More Than 93% of People with CF
29 We believe Prime Editing-based approaches could eventually benefit more than 93% of all people with CF • Disease severity and opportunity ‒ Progressive, genetic disease that affects the lungs, pancreas and other organs, leading to premature death ‒ Impacts close to 40,000 people in the United Sta
Slide 30: Parallel Prime Editing Approaches to CF: Hotspot and PASSIGE
30*Exons in gray introns in black; SA = splice acceptor; 3’ UTR = 3’ untranslated region; attP = complimentary recognition sequence for attb Parallel Prime Editing Approaches to CF: Hotspot and PASSIGE In 2024, Prime Medicine entered into agreement with CF Foundation for up to $15 million to support
Slide 31: Significant Progress Pursuing Five High Unmet Need Hotspot Mutations
31 Efforts towards in vivo delivery to humanized mice and large animals using Prime’s LNP ongoing • We believe primary human lung progenitor data most predictive of in vivo efficacy • Comprehensive suite of assays in development to enable selection of development candidate and advance to IND enablin
Slide 32: Corporate
Corporate Delivering on the promise of Prime Editing
Slide 33: Business Development Will Continue to Play a Critical Role
33 Prime Medicine plans to remain active in sell-side business development, with the goal of accelerating our pipeline, bolstering our financial resources Beyond BMS, Business Development Will Continue to Play a Critical Role in Building Prime Medicine Within Our Core Outside Our Core Access Enablin
Slide 34: Extensive, Foundational Intellectual Property for Prime Editing
34 Prime Medicine Holds Extensive, Foundational Intellectual Property for Prime Editing Technologies • Multiple configurations of RNA-templated gene editing - Prime Editor protein configurations: fusion, separate and split configurations - pegRNA configurations: fusion, split, separate and engineere
Slide 35: Prime Medicine is the Leader in Gene Editing
35*Including $55M equity investment received in September 2024 and $55M up-front consideration received in October 2024 from BMS Prime Medicine is the Leader in Gene Editing Positioned to Create Sustainable Value Through Pipeline Execution and External Partnerships The Leader in Prime Editing Pote
Slide 36: Appendix
Appendix Delivering on the promise of Prime Editing
Slide 37: Prime Editing Designed with a Wide Range of Genome Editing Capabilities
37BP = base pair; KB = kilobase Prime Editing is Designed with a Wide Range of Genome Editing Capabilities Flexibility to select right approach for each indication based on editing need Prime Editing Approach Small edits (e.g., all 12 bp swaps, 1-bp to 20-bp ins or del, combinations thereof) Mid-siz
Slide 38: PASSIGE Technology Enables Insertion of Gene Sized Sequences
38*Not part of Prime Medicine’s current pipeline PASSIGE Technology Enables Prime Editing to Insert Gene Sized Sequences Precisely, Potentially Addressing Large Markets PASSIGE: Prime-Assisted Site-Specific Integrase Gene Editing: One step non-viral multi-kilobase-size gene editing approach with no
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