e-therapeutics De-risks Clinical Path for GalOmic Candidate ETX-312 with Positive Non-clinical Data
e-therapeutics (OTCQX:ETXPF) has announced significant progress in de-risking its lead candidate ETX-312, a GalOmic siRNA therapy for metabolic dysfunction-associated steatohepatitis (MASH). The company has achieved two major milestones: successful completion of GLP-compliant toxicology studies showing excellent tolerability at high doses, and GMP manufacturing of the clinical trial batch.
The toxicology studies demonstrated that ETX-312 was well tolerated across all dose levels, with no toxicity or adverse findings, supporting a broad therapeutic window. The company remains on track for Clinical Trial Application (CTA) submission in Q4 2025, with productive regulatory interactions supporting their clinical strategy.
e-therapeutics (OTCQX:ETXPF) ha annunciato importanti progressi nella riduzione del rischio del suo candidato principale ETX-312, una terapia GalOmic siRNA per la steatoepatite associata a disfunzione metabolica (MASH). L'azienda ha raggiunto due traguardi fondamentali: il completamento con successo di studi tossicologici conformi a GLP che hanno evidenziato un'ottima tollerabilit脿 a dosi elevate, e la produzione GMP del lotto destinato alla sperimentazione clinica.
Gli studi tossicologici hanno dimostrato che ETX-312 猫 stato ben tollerato a tutti i livelli di dosaggio, senza tossicit脿 o effetti avversi, confermando un ampio margine terapeutico. L'azienda mantiene l'obiettivo di presentare la domanda di sperimentazione clinica (CTA) nel quarto trimestre del 2025, supportata da interazioni regolatorie efficaci che sostengono la strategia clinica.
e-therapeutics (OTCQX:ETXPF) ha anunciado avances significativos en la reducci贸n de riesgos de su candidato principal ETX-312, una terapia GalOmic siRNA para la esteatohepatitis asociada a disfunci贸n metab贸lica (MASH). La compa帽铆a ha alcanzado dos hitos importantes: la finalizaci贸n exitosa de estudios toxicol贸gicos conforme a GLP que mostraron una excelente tolerabilidad a dosis altas, y la fabricaci贸n GMP del lote para ensayos cl铆nicos.
Los estudios toxicol贸gicos demostraron que ETX-312 fue bien tolerado en todos los niveles de dosis, sin toxicidad ni hallazgos adversos, lo que respalda un amplio margen terap茅utico. La empresa sigue en camino para la presentaci贸n de la Solicitud de Ensayo Cl铆nico (CTA) en el cuarto trimestre de 2025, con interacciones regulatorias productivas que respaldan su estrategia cl铆nica.
e-therapeutics (OTCQX:ETXPF)電� 雽�靷� 旮半姤 鞛レ暊 鞐瓣磤 歆�氚╆皠鞐�(MASH) 旃橂毳� 鞙勴暅 GalOmic siRNA 旃橂鞝滌澑 ETX-312鞚� 鞙勴棙 臧愳唽鞐� 鞛堨柎 欷戩殧頃� 歆勳爠鞚� 氚滍憸頄堨姷雼堧嫟. 須岇偓電� 霊� 臧�歆� 欤检殧 鞚挫爼響滊ゼ 雼劚頄堨姷雼堧嫟: 瓿犾毄霟夓棎靹� 鞖办垬頃� 雮挫暯靹膘潉 鞛呾頃� GLP 欷�靾� 霃呾劚 鞐瓣惮鞚� 靹标车鞝� 鞕勲鞕� 鞛勳儊 鞁滍棙鞖� 氚办箻鞚� GMP 鞝滌“鞛呺媹雼�.
霃呾劚 鞐瓣惮 瓴瓣臣 ETX-312電� 氇摖 韴棳霟夓棎靹� 鞛� 瓴帉臁岇溂氅�, 霃呾劚鞚措倶 攵�鞛戩毄鞚� 氚滉铂霅橃 鞎婌晞 雱撿潃 旃橂 氩旍渼毳� 霋冯皼旃暕雼堧嫟. 須岇偓電� 2025雲� 4攵勱赴 鞛勳儊鞁滍棙 鞁犾箔(CTA) 鞝滌稖鞚� 氇╉憸搿� 靾滌“搿矊 歆勴枆 欷戩澊氅�, 鞛勳儊 鞝勲灥鞚� 歆�鞗愴晿電� 攴滌牅 雼龟淡瓿检潣 靸濎偘鞝侅澑 靻岉喌霃� 鞚措（鞏挫瓿� 鞛堨姷雼堧嫟.
e-therapeutics (OTCQX:ETXPF) a annonc茅 des progr猫s significatifs dans la r茅duction des risques de son candidat principal ETX-312, une th茅rapie siRNA GalOmic pour la st茅ato-h茅patite associ茅e 脿 une dysfonction m茅tabolique (MASH). L'entreprise a atteint deux 茅tapes majeures : la r茅ussite des 茅tudes toxicologiques conformes aux normes GLP montrant une excellente tol茅rance 脿 fortes doses, et la fabrication GMP du lot destin茅 脿 l'essai clinique.
Les 茅tudes toxicologiques ont d茅montr茅 que ETX-312 茅tait bien tol茅r茅 脿 tous les niveaux de dose, sans toxicit茅 ni effets ind茅sirables, soutenant ainsi une large fen锚tre th茅rapeutique. L'entreprise reste sur la bonne voie pour le d茅p么t de la demande d'essai clinique (CTA) au 4e trimestre 2025, avec des interactions r茅glementaires productives qui appuient leur strat茅gie clinique.
e-therapeutics (OTCQX:ETXPF) hat bedeutende Fortschritte bei der Risikominderung seines Hauptkandidaten ETX-312, einer GalOmic siRNA-Therapie f眉r metabolisch bedingte Steatohepatitis (MASH), bekannt gegeben. Das Unternehmen hat zwei wichtige Meilensteine erreicht: den erfolgreichen Abschluss von GLP-konformen Toxikologiestudien, die eine ausgezeichnete Vertr盲glichkeit bei hohen Dosierungen zeigten, sowie die GMP-Herstellung der klinischen Pr眉fchargen.
Die Toxikologiestudien zeigten, dass ETX-312 in allen Dosierungsstufen gut vertragen wurde, ohne Toxizit盲t oder unerw眉nschte Befunde, was ein breites therapeutisches Fenster unterst眉tzt. Das Unternehmen bleibt im Zeitplan f眉r die Einreichung des Clinical Trial Application (CTA) im vierten Quartal 2025 und profitiert von produktiven regulatorischen Interaktionen, die die klinische Strategie unterst眉tzen.
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ETX-312 well tolerated at exaggerated dose levels in GLP-compliant toxicology studies
GMP manufacturing of clinical trial batch successfully completed
On track for CTA submission in Q4 2025
LONDON, July 10, 2025 (GLOBE NEWSWIRE) -- e-therapeutics plc, a company integrating computational power and biological data to discover life-transforming RNAi medicines, today announced significant progress on its lead candidate, ETX-312, a GalOmic siRNA therapy for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The Company has successfully de-risked the path to clinical entry for ETX-312 and remains on track to submit a clinical trial application (CTA) in Q4 2025.
ETX-312 was repeatedly administered at doses far exceeding anticipated clinical exposure in GLP-compliant toxicology and toxicokinetic studies. The candidate was well tolerated across all dose levels, with no toxicity or adverse findings observed. These non-clinical results support a broad therapeutic window for ETX-312 and strengthen confidence in the program and the GalOmic platform as it advances toward first-in-human trials.
In parallel, e-therapeutics has successfully completed GMP manufacturing of the clinical batch of ETX-312, supporting the Company鈥檚 operational readiness to progress to first-in-human dosing. Productive interactions with regulatory authorities have provided further support for our clinical strategy and plans.
鈥淩ecent results and progress on ETX-312 represent a pivotal step towards the clinical development of our first GalOmic therapeutic, designed to silence a novel target identified in-house鈥� said Ali Mortazavi, CEO of e-therapeutics. 鈥淭he tolerability profile of ETX-312 provides key de-risking for our program, supporting a Q4 2025 CTA filing. We look forward to advancing ETX-312 to first-in-human studies over the coming months and continuing to execute efficiently to offer a differentiated treatment option to MASH patients.鈥�
About ETX-312
ETX-312 is a GalOmic GalNAc-conjugated small-interfering RNA (GalNAc-siRNA) therapeutic candidate in development as a safe and effective treatment for metabolic dysfunction-associated steatohepatitis (MASH) with potential for a quarterly subcutaneous dosing regimen. In preclinical studies using the Gubra-Amylin NASH diet-induced obese (GAN-DIO) mouse model, administration of ETX-312 led to dramatic reductions in NAFLD Activity Score (NAS), decreased hepatic inflammation, and slowed fibrosis progression, both as monotherapy and in synergistic combination with approved and emerging therapies. ETX-312 is currently progressing through IND-enabling studies, with a regulatory submission planned by the end of 2025.听
About e-therapeutics plc听
e-therapeutics plc ("ETX") uniquely combines computation and RNAi to discover and develop life-transforming medicines.听ETX's proprietary RNAi chemistry platform, GalOmic, enables generation of specific, potent, and durable siRNA therapeutics for effective silencing of novel gene targets in hepatocytes. The cutting-edge HepNet computational platform allows ETX to discover better medicines faster through generation of novel insights and increased automation across all stages of drug development. HepNet encompasses an extensive hepatocyte-specific knowledgebase and a suite of advanced AI-driven approaches which enable identification of novel gene targets, rapid target-indication assessment, and predictive听in silico听siRNA design. The Company has specialist expertise and a robust position in applying computation to biology. Its computational approaches have been extensively validated through generation of data from pipeline programs and successful drug discovery collaborations with biopharma companies, such as Novo Nordisk, Galapagos NV, and iTeos Therapeutics.
Leveraging the combined capabilities of HepNet and GalOmic, ETX is progressing a therapeutic pipeline听of highly differentiated RNAi candidates across a variety of therapeutic areas with high unmet need. The Company has generated positive proof-of-concept data on preclinical assets in metabolic dysfunction-associated steatohepatitis (MASH), dry age-related macular degeneration (dry AMD), haemophilia, heart failure, and cardiometabolic disease, further validating its computationally enhanced approach to research and development. ETX is currently progressing its GalOmic therapies towards the clinic with its most developed assets, ETX-312 for MASH, ETX-148 for bleeding disorders, and ETX-407 for dry AMD, at the IND-enabling stage.
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