CAMBRIDGE -
In 2024, we expect to bring the first-ever Prime Editing-based product candidate to patients, while continuing to strengthen our modular Prime Editing platform and advance our next wave of programs across a range of target tissues, said
Recent Business Updates
Chronic Granulomatous Disease (CGD)
In
At the
Broader Pipeline and Prime Editing Platform
Also at the ASGCT Meeting and at the 3rd Annual LNP Formulation and Process Development Summit (
Additional preclinical data from Prime Medicine s Rhodopsin (RHO)-mediated Retinitis Pigmentosa (RHO-RP) program, supporting Prime Medicine s ability to correct multiple mutations in the RHO gene and showing that the correction of pathogenic mutations in humanized mouse models resulted in preservation of photoreceptors.
A presentation on the development and characterization of Prime Medicine s off-target assays, which collectively have supported the observed specificity and minimal, if any, off-target activity of Prime Editing.
Corporate
In
I have devoted my career to advancing new therapies to treat some of the most challenging, intractable diseases and I am now excited to work with Prime Medicine in its mission to develop next-generation gene-editing therapeutics, said
Anticipated Upcoming Milestones
Prime Medicine expects the following activities and next steps to drive Prime Medicine forward and support the Company s maturation into a clinical-stage company: Hematology and Immunology: Announce initial clinical data from the Phase clinical trial of PM359 in CGD in 2025.
Advance Shielded Hematopoietic Stem Cell (HSC) and Immunotherapy Pairs (SCIP) technology, establish proof-of-concept in HSC and immunotherapy and identify first clinical program(s) with this approach in 2024.
Advance differentiated CAR-T program, using PASSIGE technology, into lead optimization.
Liver
Continue to advance preclinical studies for three liver programs and initiate IND-enabling activities for at least one in 2024, leading to an IND and/or clinical trial application (CTA) in the second half of 2025 or first half of 2026.
Ocular
Nominate development candidate for RHO-RP program and initiate IND-enabling activities in 2024.
Neuromuscular
Continue to advance Friedreich s Ataxia and advance one other program into lead optimization in 2024.
In large animal studies, establish adeno-associated virus (AAV) delivery platform and route of administration for neuromuscular programs in 2024.
First Quarter 2024 Financial Results
Research and Development (R&D) Expenses: R&D expenses were
General and Administrative (G&A) Expenses: G&A expenses were
Net Loss: Net loss was
Cash Position: As of
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 core areas of focus: hematology and immunology, liver, lung, ocular and neuromuscular. Across each core area, Prime Medicine s initial focus is on genetic diseases with a fast, direct path to treating patients, and those with high unmet need not currently addressable using other gene editing approaches. Over time, the Company intends to maximize Prime Editing s broad and versatile therapeutic potential to expand beyond the genetic diseases in its initial pipeline, potentially including immunological diseases, cancers, infectious diseases, and targeting genetic risk factors in common diseases, which collectively impact millions of people.
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 potential of PM359 to correct the causative mutation of CGD; the anticipated maturation into a clinical-stage company by bringing PM359 into clinical development in 2024 with initial clinical data expected in 2025; the initiation, timing, progress, and results of its research and development programs, preclinical studies and future clinical trials, and the release of data related thereto; the potential for Prime Editors to repair genetic mutations and offer curative genetic therapies for a wide spectrum of diseases; the potential of Prime Editors to reproducibly correct disease-causing genetic mutations across different tissues, organs and cell types, and the capacity of its PASSIGE technology to edit CAR-T cells for the treatment of certain cancers and immune diseases; its continued development and optimization of various non-viral and viral delivery systems; its ability to demonstrate superior off-target profiles for Prime Editing programs; certain activities and next steps to support the Company s maturation into a clinical-stage company, including opening IND and/or CTA applications, clinical data expectations, establishing proof of concept, advancing programs into lead optimization, advancing preclinical studies and initiating IND-enabling activities, nominating development candidates, and establishing delivery platform and rout of administration; 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; the modularity of the Prime Editing platform 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 and the potential of Prime Editing to unlock opportunities across thousands of potential indications. 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 and general economic, industry and market conditions, including rising interest rates, inflation, and adverse developments affecting the financial services industry. 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
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