Prime Medicine, Inc.
-- New preclinical information successful Friedrich’s ataxia and cystic fibrosis provided further proof-of-concept for Prime Editing’s quality to execute restoration of familial relation --
-- New preliminary information analyses demonstrated nary detected off-target enactment successful Prime Edited Wilson’s illness cells --
-- New information leveraging PASSIGE exertion demonstrated quality to integrate kilobase-sized DNA successful quality T cells --
-- Additional LNP and AAV transportation information demonstrated businesslike successful vivo transportation of Prime Editing to assorted people tissues successful rodents --
-- Multiple catalysts expected successful 2023, including archetypal improvement campaigner information successful 1Q and further successful vivo information successful 2H --
-- Strong firm position, with robust intelligence spot presumption and currency to money operations into 2025 --
CAMBRIDGE, Mass., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology institution committed to delivering a caller people of differentiated one-time curative familial therapies, contiguous provided an update connected caller advancements of its Prime Editing exertion and advancement crossed its archetypal pipeline of eighteen programs, and outlined its strategical priorities and expected milestones for 2023.
“Since our inception, we person focused connected gathering the indispensable instauration to maximize Prime Editing’s therapeutic potential, identifying and progressing a divers archetypal pipeline, introducing optimizations to widen the scope of our Prime Editing technology, and investing successful the CMC and transportation capabilities that volition yet beryllium required to present our investigational therapies to patients,” said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. “Today, we are pleased to denote accomplishments crossed our portfolio and platform, including caller preclinical proof-of-concept information successful Friedrich’s ataxia and cystic fibrosis showing restoration of familial function; caller information analyses detecting nary off-target editing successful Prime Edited Wilson’s illness cells; the archetypal presumption of our quality to usage PASSIGE exertion to precisely insert a full cistron into quality T cells; and further optimization of our lipid nanoparticle and adeno-associated microorganism transportation platforms. Together, these updates reenforce our content successful Prime Editing arsenic an highly powerful, differentiated technology, with the imaginable to present one-time, curative familial therapies to code a wide spectrum of diseases. As we participate 2023, we look guardant to progressing our astir precocious programs toward the clinic, portion continuing to optimize our exertion and physique the interior organization, civilization and expertise indispensable to conscionable our ambitious goals.”
Recent Business Updates
Pipeline
Prime Medicine is advancing a strategical pipeline of eighteen programs. The institution is initially focused connected indications with the accidental for the fastest, astir nonstop way to the session and method occurrence successful humans, arsenic good arsenic indications that cannot beryllium treated utilizing different cistron editing approaches. In vivo studies are progressing crossed Prime Medicine’s portfolio and, successful caller months, the institution has established preclinical proof-of-concept and expanded information information successful a assortment of people tissues and indications. Today, Prime Medicine is announcing caller preclinical information for respective programs:
Friedrich’s Ataxia (FRDA), a multisystem, autosomal recessive neurodegenerative upset affecting the cardinal and peripheral tense systems, arsenic good arsenic the bosom and different organs. FRDA is caused by GAA-repeat nucleotide series expansions successful intron 1 of the FXN cistron encoding the frataxin protein, which plays important roles successful mitochondria. In preclinical studies, Prime Medicine is utilizing its exertion to precisely region the GAA pathological repeats astatine the FXN gene, restoring Frataxin macromolecule look and sensory neuron relation successful diligent dorsal basal ganglia.
Today, Prime Medicine announced caller preclinical information demonstrating that Prime Editing-mediated removal of pathological repeats in vitro results successful correction of hypermethylation astatine the FXN gene, restoring familial relation backmost to wild-type levels. The institution believes these information besides enactment the valuation of Prime Editing for the imaginable attraction of different repetition enlargement diseases, galore of which grounds hypermethylation arsenic a cardinal diagnostic of the underlying pathogenesis.
Cystic Fibrosis (CF), a progressive lung illness characterized by the accumulation of heavy mucus lung secretions, which artifact patients’ airways, starring to inflammation, lung corruption and, ultimately, lung failure. CF is caused by nonaccomplishment of relation mutations successful the CFTR gene, which reduces chloride and bicarbonate transport to the epithelial lumen, resulting successful thickened secretions, blocked ducts and bronchioles and secondary infections. F508del and mutations astatine 7 further hotspots, including G542X, are recovered successful 98 percent of patients.
Today, Prime Medicine shared archetypal preclinical information showing greater than 70 percent precise editing of the G542X mutational hotspot in vitro, arsenic good arsenic functional restoration of swelling and CFTR relation successful patient-derived intestinal organoids. The G542X mutation is not addressed by presently marketed therapies. The institution believes these information show that precise familial correction by Prime Editing has the imaginable to execute implicit phenotypic rescue.
Off-target Safety Data: Prime Medicine is progressing a broad suite of assays to measure the imaginable off-target enactment of its Prime Editors. Because it does not make double-stranded breaks and requires 3 “edit checks,” oregon places wherever determination indispensable beryllium a lucifer betwixt the exertion and people DNA successful bid to implicit an edit, Prime Editing occurs with precocious specificity, debased indel rates and minimal to nary off-target activity, resulting successful perchance greatly improved information and tolerability. Today, Prime Medicine shared preclinical information analyses from 2 programs – caller information successful Wilson’s illness (WD) and an incremental update connected antecedently reported information successful Chronic Granulomatous Disease (CGD) – which unneurotic further enactment Prime Editing arsenic a highly circumstantial and predictable cistron editing tool.
In WD, a devastating uncommon illness of the liver, Prime Medicine announced the results of a preliminary off-target investigation successful induced pluripotent stem compartment (iPSC)-derived hepatocytes, which detected nary guide-dependent Prime Editing enactment crossed 170 identified imaginable off-target sites.
In CGD, a uncommon illness that causes recurrent, debilitating infections successful children, Prime Medicine shared the results of a preliminary off-target investigation successful CD34+ cells, which detected nary guide-dependent Prime Editing enactment crossed 550 identified imaginable off-target sites.
Prime Editing Platform
Since it was archetypal described successful 2019, Prime Medicine’s level squad has continued to optimize its PASSIGE (Prime Assisted Site-Specific Integrase Gene Editing) technology. PASSIGE combines Prime Editing with an integrase oregon site-specific recombinase enzyme to alteration the instauration of large-sized cargo into the genome arsenic a imaginable one-time therapy. This attack further increases the versatility of Prime Editing and broadens the scope of imperishable genomic edits that Prime Editing tin marque to perchance dainty disease, including the quality to insert, delete oregon invert gene-sized pieces of DNA.
Today, Prime Medicine announced caller preclinical information utilizing PASSIGE successful a one-step, non-viral process, which resulted successful an astir 60 percent precise insertion of a 3.5 kilobase transgene astatine a azygous targeted tract successful superior quality T cells.
CMC and Delivery
Prime Medicine is investing successful interior chemistry, manufacturing, and controls (CMC) improvement and transportation capabilities successful bid to physique the foundational competencies indispensable to present its pipeline programs to patients arsenic the institution prepares to participate the clinic. In caller months, Prime Medicine has continued to beforehand its lipid nanoparticle (LNP) and adeno-associated microorganism (AAV) transportation platforms, demonstrating and disclosing for the archetypal clip today:
Additional proof-of-concept information for LNP transportation of Prime Editors to rodent liver, including the archetypal in vivo objection of the instauration of a precisely edited halt codon successful the PCSK9 gene, resulting successful greater than 40 percent editing and greater than 90 percent simplification successful serum PCSK9 protein. Prime Medicine is utilizing PCSK9 arsenic a exemplary strategy for processing its modular LNP transportation level to the liver.
New information demonstrating that dual AAV transportation to the cardinal tense strategy achieves precocious ratio transduction successful murine models, with a precocious level of precise editing successful transduced cells. Specifically, utilizing intracerebroventricular delivery, Prime Editing precisely edited astir 90 percent of transduced cortical cells and, utilizing section administration, Prime Editing precisely edited astir 80 percent of transduced neurons.
Anticipated Upcoming Milestones
Prime Medicine expects the pursuing activities and adjacent steps to thrust the Prime Editing level forward:
Pipeline
Nominate archetypal improvement campaigner for CGD successful 1Q 2023.
Initiate investigational caller cause (IND)-enabling studies successful CGD successful 2023.
Expand preclinical proof-of-concept in vivo, including sharing information from in vivo rodent studies and ample carnal studies successful respective programs successful 2H 2023.
Share in vitro preclinical information successful further liver, oculus and neuromuscular programs.
First IND filing expected arsenic aboriginal arsenic 2024 and further IND filings anticipated successful 2025.
Platform
Continue to make and optimize non-viral and viral transportation systems and stock further proof-of-concept information from in vivo rodent and ample carnal studies successful 2H 2023.
Further show superior “off-target” profiles for Prime Editing programs.
Expand Prime Editing utilizing proprietary recombinase and/or retrotransposon technologies for caller and existing programs.
Financial Guidance
Based connected its existent operating plans, Prime Medicine expects that its cash, currency equivalents and short-term investments arsenic of September 30, 2022, unneurotic with the astir $200 cardinal successful gross proceeds raised done its archetypal nationalist offering successful October 2022, volition beryllium capable to money its anticipated operating expenses and superior expenditure requirements into 2025.
About Prime Medicine
Prime Medicine is simply a biotechnology institution committed to delivering a caller people of differentiated, one-time, curative familial therapies to code the widest spectrum of diseases. The institution is deploying Prime Editing technology, a versatile, precise, businesslike and wide cistron editing technology, which is designed to marque lone the close edit astatine the close presumption wrong a gene. With the imaginable to repair astir 90 percent of known disease-causing familial mutations crossed galore organs and compartment types, medicines based connected Prime Editing could connection a one-time curative familial therapeutic enactment to a wide acceptable of patients.
Cautionary Note Regarding Forward Looking Statements
This property merchandise contains forward-looking statements wrong the meaning of the Private Securities Litigation Reform Act of 1995, arsenic amended, including, without limitation, implied and explicit statements astir Prime Medicine’s beliefs and expectations regarding: the initiation, timing, advancement and results of its probe and improvement programs, preclinical studies and aboriginal objective trials; its quality to demonstrate, and the timing of, preclinical proof-of-concept successful vivo for aggregate programs; its quality to beforehand immoderate merchandise candidates that Prime Medicine whitethorn place and successfully implicit immoderate objective studies, including the manufacture of immoderate specified merchandise candidates; its quality to rapidly leverage programs wrong its archetypal people indications and to advancement further programs to further make its pipeline; the timing of its regulatory filings, including its investigational caller cause applications submissions; the implementation of its strategical plans for its business, programs and technology; and our estimates of our expenses, superior requirements, and needs for further financing arsenic good arsenic our currency runway into 2025. The words “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “expect,” “estimate,” “seek,” “predict,” “future,” “project,” “potential,” “continue,” “target” and akin words oregon expressions are intended to place forward-looking statements, though not each forward-looking statements incorporate these identifying words.
Any forward-looking statements successful this property merchandise are based connected management’s existent expectations and beliefs and are taxable to a fig of risks, uncertainties and important factors that whitethorn origin existent events oregon results to disagree materially from those expressed oregon implied by immoderate forward-looking statements contained successful this property release, including, without limitation, risks associated with: uncertainties related to the authorization, initiation and behaviour of preclinical and different improvement requirements for imaginable merchandise candidates, including uncertainties related to regulatory approvals; risks related to the results of preclinical studies oregon objective studies not being predictive of aboriginal results successful transportation with aboriginal studies; the scope of extortion Prime Medicine is capable to found and support for intelligence spot rights covering its Prime Editing technology; Prime Medicine’s quality to place and participate into aboriginal licence agreements and collaborations; and wide economic, manufacture and marketplace conditions, including rising involvement rates and inflation. These and different risks and uncertainties are described successful greater item successful the conception entitled “Risk Factors” successful Prime Medicine’s astir caller Quarterly Report connected Form 10-Q, arsenic good arsenic immoderate consequent filings with the Securities and Exchange Commission. In addition, immoderate forward-looking statements correspond Prime Medicine’s views lone arsenic of contiguous and should not beryllium relied upon arsenic representing its views arsenic of immoderate consequent date. Prime Medicine explicitly disclaims immoderate work to update immoderate forward-looking statements. No representations oregon warranties (expressed oregon implied) are made astir the accuracy of immoderate specified forward-looking statements.
Investor Contact
Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
hannah.deresiewicz@sternir.com
Media Contact
Dan Budwick, 1AB
dan@1ABmedia.com
English (US)