{"id":1020587,"date":"2026-11-12T16:14:44","date_gmt":"2026-11-12T21:14:44","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/prime-medicine-reports-third-quarter-2026-financial-results-and-provides-business-updates\/"},"modified":"2026-11-12T16:14:44","modified_gmt":"2026-11-12T21:14:44","slug":"prime-medicine-reports-third-quarter-2026-financial-results-and-provides-business-updates","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/prime-medicine-reports-third-quarter-2026-financial-results-and-provides-business-updates\/","title":{"rendered":"Prime Medicine Reports Third Quarter 2026 Financial Results and Provides Business Updates"},"content":{"rendered":"
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\n — First patient dosed in global Phase 1\/2 clinical trial of PM577a in Wilson disease, the first clinical study of an in vivo Prime Editing therapy from Prime Medicine; initial clinical data expected in 2027 —<\/em>\n <\/p>\n

\n — Received FDA clearance of Investigational New Drug application for PM647 in Alpha-1 Antitrypsin Deficiency; initial clinical data expected in 2027 —<\/em>\n <\/p>\n

\n — Planning for a BLA submission for PM359 in the first half of 2027 —<\/em>\n <\/p>\n

\n — Closed underwritten public offering, including the full exercise of the underwriters\u2019 option to purchase additional shares, raising $143.7 million in gross proceeds —<\/em>\n <\/p>\n

\n — Pro-forma cash, cash equivalents, investments and restricted cash of approximately $209.5 million; cash runway through 2027 —<\/em>\n <\/p>\n

CAMBRIDGE, Mass., Nov. 12, 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 quarter ended September 30, 2026 and provided a business update.<\/p>\n

\u201cPrime Medicine continued to make significant progress this quarter, highlighted by dosing the first patient in our Phase 1\/2 trial of PM577a for Wilson disease, our first clinical study of an in vivo<\/em> Prime Editing therapy,\u201d said Allan Reine, M.D., Chief Executive Officer of Prime Medicine. \u201cHaving demonstrated through our CGD program that Prime Editing can correct disease-causing mutations in humans ex vivo<\/em>, we are now extending that experience in vivo<\/em>, beginning with our liver directed programs. Furthermore, the FDA clearance of our IND for PM647 in Alpha-1 Antitrypsin Deficiency further underscores the versatility of our universal liver LNP platform. In just months, our two wholly owned liver programs have moved into, or been cleared for, clinical development, underscoring both the productivity of our platform and our ability to translate scientific innovation into clinical programs.\u201d<\/p>\n

Dr. Reine continued, \u201cWe view Wilson disease and AATD as the foundation of a liver franchise built on a shared technology platform and development strategy. This expansion has already begun within Wilson disease \u2013 starting with PM577b, our follow-on Prime Editor targeting the R778L mutation \u2013 and we intend to continue expanding our efforts, both within Wilson disease and more broadly. Furthermore, we are encouraged by the FDA\u2019s recently issued draft guidance on individualized genome editing, which, if finalized, could represent an important advance for patients with ultra-rare diseases and create a meaningful opportunity for us to extend Prime Editing therapies to additional diseases. Our recent financing strengthens our balance sheet and provides additional resources needed to advance these programs through key clinical milestones. Looking ahead, we expect 2027 to be a meaningful year for the patient communities we hope to treat, with initial clinical data anticipated from both PM577a and PM647 and a planned BLA submission for PM359.\u201d<\/p>\n

\n Recent Corporate Updates:<\/strong>\n <\/p>\n

\n Wilson Disease (PM577a)<\/strong>\n <\/p>\n

In October 2026, Prime Medicine announced dosing of the first patient in its global Phase 1\/2 clinical trial of PM577a. Also in October 2026, the FDA granted Rare Pediatric Disease designation to PM577 for the treatment of Wilson disease. PM577a targets the ATP7B <\/em>H1069Q mutation, the most prevalent Wilson disease-causing allele in North America and Europe.<\/p>\n

The Phase 1\/2 clinical trial (NCT07748403) is an open-label, global, first-in-human study designed to evaluate the safety, tolerability, and preliminary clinical efficacy of ascending doses of PM577a in adults and adolescents with Wilson disease containing at least one p.H1069Q allele in the ATP7B<\/em> gene. The study is initially enrolling adults who are clinically stable on standard-of-care therapy. Initial clinical data are expected in 2027.<\/p>\n

PM577a is the first program in the PM577 family, a portfolio of Wilson disease medicines designed to address genetically defined patient populations worldwide using a shared LNP delivery platform. A follow-on candidate targeting the R778L mutation, the most prevalent Wilson disease-causing variant in East Asian populations, is in preclinical development. Additional efforts are underway to develop Prime Editors targeting other prevalent mutations.<\/p>\n

\n Alpha-1 Antitrypsin Deficiency (PM647)<\/strong>\n <\/p>\n

In September 2026, the FDA cleared the Company\u2019s IND for PM647, an investigational in vivo<\/em> Prime Editor designed to correct the E342K (Pi*Z) mutation in the SERPINA1<\/em> gene, the most common cause of AATD. The clearance enables PM647 to proceed to clinical study initially in the United States, where approximately 100,000 people carry the PiZZ genotype that PM647 is designed to correct. The IND clearance follows the binding resolution in July 2026 of the Company\u2019s arbitration with Beam Therapeutics, Inc., in which the Tribunal declared that PM647 is within Prime Medicine\u2019s \u201cField\u201d as defined by the parties\u2019 2019 Collaboration and License Agreement.<\/p>\n

The Phase 1\/2 clinical trial will be a global, single-arm, open-label, first-in-human study designed to evaluate the safety, tolerability and preliminary clinical efficacy of ascending doses of a one-time intravenous infusion of PM647 in adults with AATD. The study will initially enroll adult participants with lung-only manifestations of AATD and, upon demonstration of tolerability, will expand to include a separate cohort enrolling adults with significant liver disease, with or without concurrent lung manifestations of AATD. Initial clinical data are expected in 2027.<\/p>\n

\n Chronic Granulomatous Disease (PM359)<\/strong>\n <\/p>\n

PM359 has received Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA. Prime Medicine continues to engage with the FDA on the most efficient path to approval, with a BLA submission planned for the first half of 2027.<\/p>\n

\n Other Pipeline Programs <\/strong>\n <\/p>\n

Prime Medicine continues to advance its in vivo Cystic Fibrosis (CF) program with support from the Cystic Fibrosis Foundation, with the goal of developing potentially curative therapies that address the underlying genetic cause of CF.<\/p>\n

The Company is also advancing Prime Edited ex vivo CAR-T products for hematology, immunology and oncology in partnership with Bristol Myers Squibb. The collaboration brings together Prime Medicine\u2019s Prime Editing and PASSIGE technologies and Bristol Myers Squibb\u2019s cell therapy expertise to develop next-generation therapies for immunological diseases and cancer.<\/p>\n

\n Corporate Financing<\/strong>\n <\/p>\n

In November 2026, Prime Medicine completed an underwritten public offering, including the exercise in full of the underwriters\u2019 option to purchase additional shares, resulting in $143.7 million of gross proceeds, before deducting underwriting discounts and offering expenses. Net proceeds were approximately $134.7 million, after deducting the underwriting discounts and commissions and offering expenses.<\/p>\n

\n Upcoming Milestones<\/strong>\n <\/p>\n