Prime Medicine Reports Full Year 2025 Financial Results and Provides Business Updates

-- On track to file IND and/or CTA for Wilson Disease and AATD programs in 1H 2026 and mid-2026, respectively; initial clinical data for both expected in 2027 --

-- Ongoing engagement with FDA for PM359 in CGD; plan to submit BLA following final alignment --

-- Prime Medicine reported cash, cash equivalents, investments, and restricted cash of $191M providing cash runway into 2027 --

CAMBRIDGE, Mass., March 03, 2026 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today reported financial results for the full year ended December 31, 2025 and provided a business update.

“We are shaping the future of genetic medicine by advancing a platform that is rapidly emerging as the predominant gene editing technology. With Prime Editing, we have the opportunity to permanently and safely correct disease-causing mutations across a broad range of indications, supported by our comprehensive IP estate,” said Allan Reine, M.D., Chief Executive Officer of Prime Medicine. “With this foundation, our vision is bold and unwavering: to strategically deliver on the promise of Prime Editing and ensure patients have access to transformative therapies capable of delivering durable, and potentially lasting cures. This begins with PM359, our ex vivo Prime Edited autologous HSC product for CGD, for which we announced breakthrough data in 2025, which we believe supports an accelerated approval in the United States.”

Dr. Reine continued, “We are also intensely focused on R&D execution. We are progressing toward key regulatory milestones for our two liver‑focused programs – in Wilson Disease and Alpha-1 Antitrypsin Deficiency – including planned IND and CTA submissions and the initiation of Phase 1/2 clinical trials. In parallel, we continue to generate compelling preclinical data across our portfolio, including for our program for Cystic Fibrosis; to further optimize our modular delivery and manufacturing approaches; and to explore additional collaborations that could expand the reach of our platform. These near‑ and mid‑term milestones position us to deliver important additional clinical validation of Prime Editing, as we accelerate our path toward bringing meaningful, life‑changing therapies to patients.”

Prime Medicine’s Pipeline:

Prime Medicine is advancing in vivo gene editing programs aimed at treating two of the most significant genetic liver disorders: Wilson Disease (WD) and Alpha‑1 Antitrypsin Deficiency (AATD). Prime Medicine anticipates submitting an investigational new drug (IND) and/or clinical trial application (CTA) for its anchor WD program (targeting the H1069Q mutation) in the first half of 2026, and plans to leverage the modularity of the Prime Editing platform to subsequently advance follow-on programs targeting other mutations, which collectively address a majority of WD patients. Prime Medicine expects to file an IND and/or CTA for its AATD program in mid-2026, and to report initial clinical data from both WD and AATD programs in 2027.

Following positive proof-of-concept data from the first two patients treated in its Phase 1/2 study of PM359 for the treatment of Chronic Granulomatous Disease (CGD), Prime Medicine is actively working to ensure this transformative therapy is available for patients in need.

Prime Medicine is also progressing an in vivo Cystic Fibrosis (CF) program with support from the Cystic Fibrosis Foundation, and anticipates generating preclinical proof of concept data in 2026. Additionally, its efforts to develop Prime Edited CAR-T products for hematology, immunology and oncology continue in partnership with Bristol Myers Squibb.

Recent Business Updates:

• Prime Medicine continues to engage with the U.S. Food and Drug Administration (FDA) to explore ways to make PM359 available to patients with CGD. Based on recent interactions, Prime Medicine believes clinical data generated to-date may be sufficient to support an accelerated approval of PM359. The Company is working towards final alignment with the FDA, and intends to submit a Biologics License Application (BLA).
• In December 2025, Prime Medicine announced the publication of Phase 1/2 clinical data with PM359 in the New England Journal of Medicine (NEJM). The data, which were also presented in a poster session at the 67
  th
  American Society of Hematology (ASH) Annual Meeting, showed rapid neutrophil and platelet engraftment, as well as durable restoration of NADPH oxidase activity and early clinical benefit, without any safety concerns.

Full Year 2025 Financial Results

• Research and Development (R&D) Expenses: R&D expenses were $160.6 million for the year ended December 31, 2025, as compared to $155.3 million for the year ended December 31, 2024. The increase in R&D expenses is driven primarily by license and intellectual property costs and facility related expenses, offset by Prime Medicine’s strategic focus on advancing its in vivo liver franchise, deprioritization of its CGD program, and a reduction in R&D personnel resulting from the workforce reduction.
• General and Administrative (G&A) Expenses: G&A expenses were $52.3 million for the year ended December 31, 2025, as compared to $50.2 million for the year ended December 31, 2024. The increase in G&A expenses was driven by increases in professional and consultant fees.
• Net Loss: Net loss was $201.1 million for the year ended December 31, 2025, as compared to $195.9 million for the year ended December 31, 2024.
• Cash Position: As of December 31, 2025, cash, cash equivalents, investments, and restricted cash were $191.4 million, as compared to $204.5 million as of December 31, 2024.

Financial Guidance

Based on its current operating plans, Prime Medicine expects that its cash, cash equivalents and investments as of December 31, 2025 will be sufficient to fund its operating expenses and capital expenditure requirements into 2027.

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 its core areas of focus: hematology, immunology and oncology, liver and lung. Across each core area, Prime Medicine is focused initially on a set of high value programs, each targeting a disease with well-understood biology and a clearly defined clinical development and regulatory path, and each expected to provide the foundation for expansion into additional opportunities. Over time, the Company intends to maximize Prime Editing’s broad and versatile therapeutic potential, as well as the modularity of the Prime Editing platform, to rapidly and efficiently expand beyond the diseases in its current pipeline, potentially including additional genetic diseases, immunological diseases, cancers, infectious diseases, and targeting genetic risk factors in common diseases, which collectively impact millions of people. For more information, please visit www.primemedicine.com.

© 2026 Prime Medicine, Inc. All rights reserved. PRIME MEDICINE, the Prime Medicine logos, and PASSIGE are trademarks of Prime Medicine, Inc. All other trademarks referred to herein are the property of their respective owners.

Condensed Consolidated Balance Sheet Data
(unaudited)
		December 31,
(in thousands)			2025			2024
Cash, cash equivalents, and investments		$	177,680		$	190,442
Total assets			342,733			297,508
Total liabilities			221,865			144,359
Total stockholders’ equity			120,868			153,149

Condensed Consolidated Statement of Operations
(unaudited)
		Year Ended December 31,
(in thousands, except share and per share amounts)			2025				2024
Revenue:
Collaboration revenue — related party		$	4,586			$	1,609
Collaboration revenue			46				1,374
Total revenue			4,632				2,983
Operating expenses:
Research and development			160,636				155,289
General and administrative			52,346				50,161
Total operating expenses			212,982				205,450
Loss from operations			(208,350	)			(202,467	)
Other income:
Interest income			4,149				3,522
Accretion (amortization) of investments			2,479				3,507
Change in fair value of short-term investment — related party			432				(485	)
Other income, net			148				41
Total other income, net			7,208				6,585
Net loss attributable to common stockholders		$	(201,142	)		$	(195,882	)
Net loss per share attributable to common stockholders, basic and diluted		$	(1.35	)		$	(1.65	)
Weighted-average common shares outstanding, basic and diluted			148,758,527				118,600,381