Prime Medicine, Inc. · NASDAQ:PRME
Prime Medicine — JP Morgan Healthcare Conference 2024
30 slides from Prime Medicine's conference presentation, Jan 8, 2024
Slide 1: Delivering on the promise of Prime Editing
Exhibit 99.1 Delivering on the promise of Prime Editing JP Morgan Healthcare Conference January 2024 1
Slide 2: Forward Looking Statements
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 pro
Slide 3: The age of human therapeutic gene editing is already here
“The age of human therapeutic gene editing isn’t just coming. It’s already here.”* 3 * David Liu, Ph.D., Co-Founder of Prime Medicine
Slide 4: The age of human therapeutic gene editing is already here
“The age of human therapeutic gene editing isn’t just coming. It’s already here.”* 4 * David Liu, Ph.D., Co-Founder of Prime Medicine
Slide 5: The age of human therapeutic gene editing is already here
“The age of human therapeutic gene editing isn’t just coming. It’s already here.”* 5 * David Liu, Ph.D., Co-Founder of Prime Medicine
Slide 6: The age of human therapeutic gene editing is already here
“The age of human therapeutic gene editing isn’t just coming. It’s already here.”* 6 * David Liu, Ph.D., Co-Founder of Prime Medicine
Slide 7: The age of human therapeutic gene editing is already here
“The age of human therapeutic gene editing isn’t just coming. It’s already here.”* 7 * David Liu, Ph.D., Co-Founder of Prime Medicine
Slide 8: Now is our moment: Prime Medicine brings together the right people and technology
Now is our moment: Prime Medicine brings together the right people and the right technology at the right time we are building on decades of progress to deliver the promise of one-time, curative genetic therapies to address the widest spectrum of diseases 8
Slide 9: Now is our moment: Delivering on the promise of Prime Editing
OPERATIONAL EXECUTION BROAD OPPORTUNITY TO ADDRESS LARGE MARKETS Delivering on the promise of Prime Editing Now is our moment: DIFFERENTIATED SAFETY PROFILE Prime Medicine brings together the PLATFORM MODULARITY right people and the right technology at the right time ENTERING THE CLINIC we are build
Slide 10: Consistent Operational Execution Sets Foundation For Transition to Clinical-Stage
Consistent Operational Execution Sets Strong Foundation For Transition to Clinical-Stage Biotech Company in 2024 2020 2021-23 2024+ Platform Platform Translation: Platform Gestation Industrialization Entering the Clinic • Developed high throughput • First IND / CTA, with several • Transferred techno
Slide 11: Prime Editing's Versatility Can Unlock Broad Opportunity Across Diseases
Prime Editing’s Versatility Can Unlock Broad Opportunity Across Wide Spectrum of Diseases Prime Editing is the only gene editing technology with the capability to edit, correct, insert and delete DNA sequences in any target tissue Deletion / Insertion Correction Chronic Granulomatous Disease (CGD) P
Slide 12: PASSIGE Technology Enables Insertion of Gene Sized Sequences
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 double-stranded breaks Non-viral, multiplex- edi
Slide 13: Prime Editing Has Highly Differentiated Safety Profile
Prime Editing Has Highly Differentiated Safety Profile: * No Off-Target Activity Detected in Any Lead Program Prime Medicine uses a comprehensive suite of robust, IND-ready assays to evaluate Prime Editor safety risks Examples from CGD Program that are being used to support IND/CTA filings: No off-t
Slide 14: Platform Modularity Accelerates and De-Risks Ongoing Efforts
Platform Modularity Accelerates and De-Risks Ongoing Efforts and Enables Rapid Generation of New Product Candidates Core components can be readily leveraged to drive pipeline acceleration, efficiency and execution Clinical Regulatory Manufacturing Delivery Off-Target Assays Prime Editors 14
Slide 15: Prime Medicine is Entering the Clinic at the Right Time
Prime Medicine is Entering the Clinic at the Right Time: Evolving Landscape Favors Innovation in Cell and Gene Therapy Positive regulatory interactions in U.S. and globally set stage for near-term clinic entry In 2023, FDA: In 2023, Prime Medicine: Established Office of Therapeutic Products under Dr
Slide 16: Focused Internally on Four Pillars
Prime Medicine is Focused Internally on Four Pillars, Each with Demonstrated High Efficiency, Precise in Vivo Editing Business development can extend reach and impact, bolstering our financial resources and maximizing the potential of Prime Editing Hematology & Business Liver Ocular Neuromuscular Im
Slide 17: Our Pipeline: Aligned to Four Core Modular Platforms
Our Pipeline: Aligned to Four Core Modular Platforms, With Additional Programs Advancing as Potential Partnership Opportunities Strategic pillar Indication Delivery Discovery Lead optimization IND-enabling Phase 1/2 ex vivo Chronic Granulomatous Disease HEMATOLOGY & IMMUNOLOGY Other programs in disc
Slide 18: Advancing PM359 to the Clinic for Chronic Granulomatous Disease
Advancing PM359 to the Clinic for Chronic Granulomatous Disease, A Disease of Significant Unmet Need Rare genetic disease, characterized by defective neutrophil function • Serious life-threatening disease presents in childhood; life expectancy ~40 years phox 1 • Caused by mutation in the p47 protein
Slide 19: With PM359, Prime Medicine is Set to Become a Clinical-Stage Company
With PM359, Prime Medicine is Set to Become a Clinical-Stage Company Poised to Deliver Data in Near-Term PM359 is comprised of autologous hematopoietic stem cells modified ex vivo using Prime Editing Enroll DP Infusion Primary endpoint Mobilize Engraftment Manufacture DHR Read-out Condition ~6 month
Slide 20: Cell Shielding and In Vivo Delivery Has Potential to Expand HSC Platform
Cell Shielding and In Vivo Delivery or Targeting Has Potential to Expand HSC Platform Beyond Rare Diseases Current efforts lay the foundation for wider range of rare and non-rare indications: benign conditioning with CD117 cell shielding enables non-toxic bone marrow transplant Future Non-Viral HSC
Slide 21: Proprietary LNP Platform is Advancing Toward the Clinic for Liver Diseases
Proprietary LNP Platform is Advancing Toward the Clinic for the Treatment of Liver Diseases LNP delivery mechanism shown to precisely correct disease-causing mutations in the liver of NHPs LNP delivered Prime Editors achieved high levels of precise Universal targeted LNP editing in the livers of NHP
Slide 22: Early Data with Proprietary Dual-AAV System in Retinal Diseases
Early Data with Proprietary Dual-AAV System: Key Step Toward Unlocking Opportunities in Retinal Diseases and Larger Indications Proof-of-concept achieved: demonstrated ability to correct pathogenic mutations in the eye with high efficiency and no off-target edits detected In RHO adRP, Prime Editors
Slide 23: Early Data Suggest Potential to Address Neuromuscular Repeat Expansion Diseases
Early In Vitro and In Vivo Data Suggest Potential for Prime Editing To Address Many Neuromuscular Repeat Expansion Diseases Prime Editors offer genetic correction in patient-derived neurons and muscle Friedreich's Ataxia Amyotrophic Lateral Sclerosis Duchenne Muscular Dystrophy Delivery PE restored
Slide 24: Business Development Remains Core Focus
Business Development Remains Core Focus for Building Prime Medicine Prime Medicine will remain active in both sell-side and buy-side business development, with the goal of accelerating our pipeline, bolstering our financial resources, and maximizing the potential of Prime Editing Recent accomplishme
Slide 25: PASSIGE and Multiplex Prime Editing Create Allogeneic CAR-T Cell Product
PASSIGE™ and Multiplex Prime Editing Create Potentially Best-in-Class Allogenic CAR-T Cell Product Modularity of platform has potential to accelerate development of additional CAR-T programs Existing Limitations Prime Editing Solution ✓ >80% integration efficiency to date, aimed at TRAC ꭗ Low payloa
Slide 26: Prime Editors Correct High Unmet Need CF Mutations
Prime Editors Correct “High Unmet Need” CF Mutations, For Example, the Prevalent G542X (null) Mutation Eight hotspot Prime Editors could address the “high unmet need” mutations; These same eight hotspot Prime Editors could address >98% of all CF patients Intestinal organoids swelling assay Prime Edi
Slide 27: Extensive Intellectual Property for Prime Editing Technologies
Prime Medicine Holds Extensive Intellectual Property for Prime Editing Technologies Prime Medicine’s IP includes: • Multiple configurations of RNA-templated gene editing - Prime Editor protein configurations: fusion, separate and split configurations - pegRNA configurations: fusion, split, separate
Slide 28: Key Upcoming Events Will Drive Prime Medicine Forward
Key Upcoming Events will Drive Prime Medicine Forward, Support Our Maturation into a Clinical-Stage Company Summary of key ongoing activities and planned next steps for Prime Medicine in 2024-2025 Hematology & Immunology - Open IND and/or CTA for Phase 1/2 study in Chronic Granulomatous disease in 1
Slide 29: Delivering on the promise of Prime Editing
Delivering on the promise of Prime Editing primemedicine.com 29
Slide 30: Our Pipeline: Aligned to Four Core Modular Platforms
Our Pipeline: Aligned to Four Core Modular Platforms, With Additional Programs as Potential Partnership Opportunities Strategic pillar Indication Delivery Discovery Lead optimization IND-enabling Phase 1/2 Chronic Granulomatous Disease ex vivo HEMATOLOGY & IMMUNOLOGY Fanconi Anemia ex vivo Wilson’s
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