Precision BioSciences Announces Complete Clinical Response in First Infant Dosed by Partner iECURE in Ongoing Phase 1/2 Clinical Trial in Ornithine Transcarbamylase (OTC) Deficiency
Precision BioSciences (DTIL) announced positive clinical data from its partner iECURE's Phase 1/2 OTC-HOPE study. The first infant treated with ECUR-506, which uses Precision's ARCUS® nuclease technology, achieved a complete clinical response that was maintained from three to six months post-treatment.
The treatment was generally well-tolerated, with only asymptomatic transaminitis occurring at four weeks, which resolved within four weeks using immunosuppressive therapy. After twelve weeks, the patient no longer required ammonia scavenger medication and could increase protein intake to age-appropriate levels, with ammonia levels remaining normal.
iECURE expects to complete enrollment in 2025 and provide complete data in first half of 2026. The study is being conducted across the UK, US, Australia, and Spain. Separately, Precision's ELIMINATE-B trial for chronic hepatitis B is ongoing with Phase 1 data expected in 2025.
Precision BioSciences (DTIL) ha annunciato dati clinici positivi dallo studio Phase 1/2 OTC-HOPE del suo partner iECURE. Il primo neonato trattato con ECUR-506, che utilizza la tecnologia nuclease ARCUS® di Precision, ha raggiunto una risposta clinica completa che è stata mantenuta da tre a sei mesi dopo il trattamento.
Il trattamento è stato generalmente ben tollerato, con unicamente trasaminasi asintomatiche che si sono verificate dopo quattro settimane, risolte entro quattro settimane con terapia immunosoppressiva. Dopo dodici settimane, il paziente non aveva più bisogno di farmaci per lo scarto dell'ammoniaca e ha potuto aumentare l'assunzione di proteine a livelli appropriati per l'età, con livelli di ammoniaca che sono rimasti normali.
iECURE prevede di completare l'arruolamento nel 2025 e fornire dati completi nella prima metà del 2026. Lo studio è condotto nel Regno Unito, negli Stati Uniti, in Australia e in Spagna. Separatamente, il trial ELIMINATE-B di Precision per l'epatite B cronica è in corso con dati della fase 1 attesi nel 2025.
Precision BioSciences (DTIL) anunció datos clínicos positivos del estudio Phase 1/2 OTC-HOPE de su socio iECURE. El primer bebé tratado con ECUR-506, que utiliza la tecnología de nucleasa ARCUS® de Precision, logró una respuesta clínica completa que se mantuvo de tres a seis meses después del tratamiento.
El tratamiento fue generalmente bien tolerado, con solo transaminasas asintomáticas que ocurrieron a las cuatro semanas, las cuales se resolvieron en cuatro semanas con terapia inmunosupresora. Después de doce semanas, el paciente ya no necesitaba medicamentos para eliminar el amoníaco y pudo aumentar la ingesta de proteínas a niveles apropiados para su edad, manteniendo los niveles de amoníaco dentro de lo normal.
iECURE espera completar la inscripción en 2025 y proporcionar datos completos en la primera mitad de 2026. El estudio se está llevando a cabo en el Reino Unido, Estados Unidos, Australia y España. Por separado, el ensayo ELIMINATE-B de Precision para la hepatitis B crónica está en curso, con datos de fase 1 esperados para 2025.
프리시전 바이오사이언스 (DTIL)는 파트너 iECURE의 Phase 1/2 OTC-HOPE 연구에서 긍정적인 임상 데이터를 발표했습니다. ECUR-506으로 치료받은 첫 번째 영아가 프리시전의 ARCUS® 핵산 효소 기술을 사용하여 완전한 임상 반응을 달성했으며, 이는 치료 후 3개월에서 6개월 동안 유지되었습니다.
치료는 일반적으로 잘 견디며, 치료 4주 후 비증상성 트랜사미나제가 발생했으나 면역억제 치료를 통해 4주 이내에 해결되었습니다. 12주 후, 환자는 더 이상 암모니아 스캐빈저 약물이 필요하지 않았고 나이에 적합한 수준으로 단백질 섭취를 늘릴 수 있었으며, 암모니아 수치도 정상으로 유지되었습니다.
iECURE는 2025년에 등록을 완료하고 2026년 상반기에 완전한 데이터를 제공할 것으로 기대하고 있습니다. 이 연구는 영국, 미국, 호주 및 스페인에서 진행되고 있습니다. 별도로, 프리시전의 만성 간염 B 치료를 위한 ELIMINATE-B 시험이 진행 중이며, 2025년에는 Phase 1 데이터가 예상됩니다.
Precision BioSciences (DTIL) a annoncé des données cliniques positives de l'étude Phase 1/2 OTC-HOPE de son partenaire iECURE. Le premier nourrisson traité avec ECUR-506, qui utilise la technologie nuclease ARCUS® de Precision, a obtenu une réponse clinique complète qui a été maintenue de trois à six mois après le traitement.
Le traitement a généralement bien été toléré, avec seulement des transaminases asymptomatiques survenant à quatre semaines, qui se sont résolues en quatre semaines grâce à une thérapie immunosuppressive. Après douze semaines, le patient n'avait plus besoin de médicaments pour éliminer l'ammoniac et a pu augmenter son apport en protéines à des niveaux adaptés à son âge, les niveaux d'ammoniac restant normaux.
iECURE prévoit de finaliser l'inscription en 2025 et de fournir des données complètes au premier semestre de 2026. L'étude est menée au Royaume-Uni, aux États-Unis, en Australie et en Espagne. Par ailleurs, l'essai ELIMINATE-B de Precision pour l'hépatite B chronique est en cours, avec des données de phase 1 attendues pour 2025.
Precision BioSciences (DTIL) hat positive klinische Daten aus der Phase 1/2 OTC-HOPE-Studie ihres Partners iECURE bekannt gegeben. Das erste behandelte Kleinkind mit ECUR-506, das die ARCUS® Nuklease-Technologie von Precision verwendet, erreichte eine vollständige klinische Reaktion, die von drei bis sechs Monaten nach der Behandlung aufrechterhalten wurde.
Die Behandlung war im Allgemeinen gut verträglich, wobei nach vier Wochen nur asymptomatische Transaminasen auftraten, die innerhalb von vier Wochen mit immunsuppressiver Therapie behoben wurden. Nach zwölf Wochen benötigte der Patient keine Medikamente zur Ammoniakreduktion mehr und konnte die Proteinaufnahme auf altersgerechte Werte erhöhen, während die Ammoniakwerte normal blieben.
iECURE plant, die Rekrutierung bis 2025 abzuschließen und im ersten Halbjahr 2026 vollständige Daten bereitzustellen. Die Studie wird im Vereinigten Königreich, in den USA, Australien und Spanien durchgeführt. Separat läuft die ELIMINATE-B-Studie von Precision zur chronischen Hepatitis B, dessen Phase 1-Daten für 2025 erwartet werden.
- Complete clinical response achieved in first treated patient
- Treatment allowed discontinuation of ammonia scavenger medication
- Patient achieved normal ammonia levels and age-appropriate protein intake
- Response maintained through six-month follow-up period
- Asymptomatic transaminitis occurred requiring immunosuppressive therapy
Insights
The Phase 1/2 clinical trial results for ECUR-506 represent a groundbreaking achievement in gene editing therapeutics. The complete clinical response observed in the first infant patient, maintained for six months post-treatment, validates the ARCUS platform's capability to perform precise gene insertion. The successful protein liberalization and normalized ammonia levels without scavenger medication demonstrate meaningful clinical efficacy.
The safety profile appears manageable, with only transient asymptomatic transaminitis that resolved with immunosuppressive therapy. This favorable benefit-risk profile is important for pediatric applications. The trial's expansion across multiple countries and expected completion in 2025 suggests strong regulatory confidence and potential accelerated development pathway.
For context, OTC deficiency treatment options have been to dietary restrictions and medications, with liver transplantation as a last resort. A one-time gene editing treatment that enables normal protein metabolism could revolutionize the standard of care.
These results mark a significant milestone for Precision BioSciences and the broader gene editing sector. The successful demonstration of ARCUS technology in a human patient validates the platform's commercial potential beyond preclinical studies. This could catalyze increased investor interest in DTIL's pipeline and partnership opportunities.
The licensing agreement with iECURE provides DTIL with potential milestone payments and royalties while reducing development costs and risks. With a market cap of
From a competitive standpoint, success in gene insertion therapy positions ARCUS favorably against other editing platforms primarily focused on gene knockout approaches. The addressable market for genetic disorders requiring gene insertion represents a substantial commercial opportunity.
- Treatment with ECUR-506 resulted in a complete clinical response from three months post exposure to the end of study (six months post exposure)
- ECUR-506 was generally well tolerated with no significant clinical safety concerns
- Insertion of a functional OTC gene through ARCUS in vivo gene editing may provide lasting clinical benefit for children with OTC deficiency who are in dire need of effective treatments
“Congratulations to iECURE for these exciting results and initial clinical validation in the first infant dosed in their OTC-HOPE trial. These results showcase that a complete clinical response can be achieved through in vivo gene editing for children born with this devastating genetic disease,” said Michael Amoroso, Chief Executive Officer of Precision BioSciences. “The data increases our confidence in the therapeutic potential of ARCUS as a novel in vivo gene editing approach for patients who have been and plan to be treated in clinical trials by Precision and partners.”
Treatment with ECUR-506, which employs an ARCUS® nuclease licensed from Precision BioSciences, was generally well tolerated in this infant with no significant clinical safety concerns apart from asymptomatic transaminitis at four weeks. The asymptomatic transaminitis was managed with immunosuppressive therapy and resolved within four weeks. Twelve weeks after a single dose of ECUR-506, ammonia scavenger medication was discontinued and mean daily protein intake was increased to age-appropriate levels. Protein liberalization was well tolerated, and the subsequent mean ammonia level remained within normal limits and was reduced compared to the mean pretreatment level. The response was maintained through six months and indicates that a complete response was achieved in this patient.
The OTC-HOPE study is ongoing in the
Precision’s ELIMINATE-B trial for PBGENE-HBV is ongoing in chronic hepatitis B in
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, Precision’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases such as chronic hepatitis B where no adequate treatments exist.
About ECUR-506
iECURE’s approach to gene editing for its initial programs, including OTC deficiency, relies on the delivery of two adeno-associated virus (AAV) vectors, each carrying different payloads. ECUR-506 comprises two vectors, an ARCUS® nuclease vector targeting gene editing in the well-characterized PCSK9 gene locus and a donor vector that inserts the desired functional OTC gene. iECURE has licensed the ARCUS nuclease for ECUR-506 from Precision BioSciences. The cut in the PCSK9 site serves as the insertion site for the OTC gene, providing a potential path to permanent expression of a functional gene. ECUR-506 is being studied in the OTC-HOPE study, the first clinical ARCUS-based in vivo gene insertion program.
About the OTC-HOPE Study
The OTC-HOPE study is a Phase 1/2 first-in-human clinical trial of ECUR-506 in baby boys with genetically confirmed neonatal onset OTC deficiency and will test multiple dose levels of ECUR-506. The study is enrolling newborn males up to seven months of age at screening who are diagnosed with severe neonatal onset OTC deficiency and meet certain other criteria. The primary objective is to assess the safety and tolerability of intravenous administration of a single dose of ECUR-506. It will also assess the pharmacokinetics and efficacy of ECUR-506 administration and the potential effects of ECUR-506 on disease-specific biologic markers, developmental milestones and quality of life. The main study will occur in a series of stages over a 10-month period, including screening, stabilization, dosing eligibility, study drug administration, and six-month follow-up. Upon completion of the OTC-HOPE study, participants transition to the 14.5 year long term follow up study (ECUR-LTFU). For more information, visit https://OTC-HOPE.com.
About OTC Deficiency
OTC deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for the detoxification of ammonia. Individuals with OTC deficiency can build up excessive levels of ammonia in their blood potentially resulting in devastating consequences, including irreversible neurological damage, coma and death. The severe form of the condition emerges shortly after birth and is more common in boys than girls. The only corrective treatment for early onset severe OTC deficiency is a liver transplant. Currently available medical therapies do not correct the disease and do not eliminate the risk of life-threatening symptoms or crises.
About iECURE
iECURE is a clinical-stage gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion for the treatment of liver disorders with significant unmet need. iECURE believes their approach has the potential to restore the function of a dysfunctional gene, regardless of mutation, by knocking-in a functional copy of that gene to offer durable gene expression and long-term, potentially curative, therapeutic benefit. iECURE’s management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. iECURE intends to leverage their team’s core strength in research and development strategy to identify what they believe to be the most suitable target and modality for their product candidates to address particular liver diseases. For more information, visit https://iecure.com and follow on LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy, benefit, and therapeutic potential of our and our partners’ product candidates and gene editing approaches (including PBGENE-HBV); the suitability of ARCUS nucleases for gene insertion and/or restoration of function, gene elimination other gene editing approaches; the expected timing of regulatory processes; expectations about our and our partners’ operational initiatives, strategies, further development of our and our partners’ programs; expectations about achievement of key milestones; and anticipated timing of our and our partners’ clinical data. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “target,” “will,” “would,” or the negative thereof and similar words and expressions.
Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ or other licensees’ ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the
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Investor and Media Contact:
Naresh Tanna
Vice President of Investor Relations
Naresh.Tanna@precisionbiosciences.com
Source: Precision BioSciences, Inc.
FAQ
What were the results of iECURE's first patient treated with ECUR-506 using DTIL's ARCUS technology?
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What therapeutic benefits did the first patient achieve with ECUR-506 using DTIL's technology?
