iECURE Secures Approval from U.K. Medicines & Healthcare Products Regulatory Agency to Expand the OTC-HOPE Study of ECUR-506
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Insights
The recent approval by the U.K. Medicines & Healthcare products Regulatory Agency (MHRA) for the expansion of the OTC-HOPE study into the United Kingdom marks a significant milestone for iECURE, Inc. This authorization for a Phase 1/2 clinical trial of ECUR-506, a gene editing-based therapy for Ornithine Transcarbamylase (OTC) deficiency, suggests a potential shift in the treatment paradigm for this inherited metabolic disorder. Traditionally, severe cases of OTC deficiency may require a liver transplant, which comes with the burden of lifelong immunosuppression and associated risks.
From a medical research perspective, the development of ECUR-506 is notable for its use of meganuclease-based in vivo gene insertion technology. This could represent a major advancement in gene therapy, as it aims to correct the genetic defect at its source. The ability of ECUR-506 to enable children to produce functional OTC enzyme could lead to a durable treatment solution, reducing or eliminating the need for liver transplants in this patient population. The focus on safety and tolerability in the clinical trial, along with secondary objectives to assess pharmacokinetics and efficacy, is crucial for determining the viability of this treatment. Additionally, the exploratory endpoints will provide insight into the long-term impact on patients' quality of life and developmental outcomes.
The approval from the MHRA to conduct clinical trials in the U.K. is a positive development for iECURE, Inc. and could have implications for the company's financial health. Investors are often attentive to such regulatory milestones as they can be indicative of a company's research and development progress. The expansion of the OTC-HOPE study into additional geographies suggests a strategic approach to regulatory engagement and patient recruitment, which can be critical for the timely completion of clinical trials.
While the immediate financial impact may not be significant, as clinical trials are typically costly and the path to revenue generation is long, the long-term potential for ECUR-506 could be substantial if the therapy proves to be safe and effective. The company's stock price could see fluctuations based on the outcomes of the trial phases and the anticipation of market entry. It is also worth noting that the success of ECUR-506 could position iECURE, Inc. as a leader in gene editing therapies for liver disorders, potentially opening the door for future investment and partnerships.
The gene therapy market is rapidly evolving and treatments for rare diseases such as OTC deficiency are particularly significant due to the high unmet need and often limited treatment options. The approval by the MHRA not only adds credibility to iECURE, Inc.'s research but also signals to the market that gene editing therapies are gaining regulatory traction. As a market research analyst, it's important to consider the competitive landscape and the potential market size for ECUR-506.
Given that OTC deficiency is a rare condition, the market for ECUR-506 is likely to be niche with a relatively small patient population. However, treatments for rare diseases often come with premium pricing due to the complexity of development and the value they provide in addressing unmet needs. If ECUR-506 is successful, it could command a significant price point and lead to substantial revenue for iECURE, Inc. Understanding the prevalence of OTC deficiency and the current standard of care will be essential in forecasting the potential market penetration and revenue generation for ECUR-506.
ECUR-506 is an investigational gene editing-based therapy for the treatment of Ornithine Transcarbamylase (OTC) deficiency
“ECUR-506 is the first ever clinical meganuclease-based in vivo gene insertion program, and as we continue progressing ECUR-506 towards first-in-human dosing, we are assured by the confidence instilled in this program by both the MHRA and the TGA,” said Joe Truitt, Chief Executive Officer of iECURE. “Throughout 2024, we will continue to be in close contact with other regulatory bodies as we seek to expand the OTC-HOPE study into additional geographies. We are in the process of ensuring sites are prepared to enroll patients and anticipate site initiations in the upcoming months.”
The OTC-HOPE study is a Phase 1/2 first-in-human study in newborn males with genetically confirmed neonatal onset OTC deficiency. It is designed primarily to assess the safety and tolerability of up to two dose levels of ECUR-506 following intravenous administration of a single dose. Secondary objectives are to assess the pharmacokinetics and efficacy of ECUR-506. In addition, exploratory endpoints will assess disease-specific biologic markers, developmental milestones and quality of life.
“OTC deficiency is a serious inherited metabolic disorder and in most severe cases, liver transplant is required,” said Gabriel M. Cohn, MD, Chief Medical Officer of iECURE. “ECUR-506, if approved, offers hope to families afflicted by this life-threatening condition as an alternative treatment approach. Through gene editing, we believe ECUR-506 can enable these children to durably produce functional OTC enzyme thereby providing lasting clinical benefit and eliminating the need for liver transplant and the lifelong immunosuppressive treatments required to prevent organ rejection.”
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) capsids, 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.1 The cut in the PCSK9 site serves as the insertion site for the OTC gene, providing a potential path to permanent expression of a healthy gene. ECUR-506 is being studied in the OTC-HOPE study, the first clinical meganuclease-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 OTC deficiency and will test up to two dose levels of ECUR-506. The study is enrolling baby boys aged 24 hours to seven months 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.
About iECURE
iECURE is a clinical-stage gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need. We believe our approach has the potential to replace and restore the function of a dysfunctional gene, regardless of mutation, by knocking-in a healthy copy of that gene to offer durable gene expression and long-term, potentially curative, therapeutic benefit. Our management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. We intend to leverage our team’s core strength in research and development strategy to identify what we believe to be the most suitable target and modality for our product candidates to address particular liver diseases. We are collaborating with the University of Pennsylvania’s Gene Therapy Program (GTP) led by James M. Wilson, M.D., Ph.D., to utilize GTP’s world-class translational expertise and infrastructure, which has helped generate our initial pipeline of potential product candidates. For more information, visit https://iecure.com and follow on LinkedIn.
About Precision BioSciences & ARCUS®
Precision BioSciences, Inc. is an advanced gene editing company dedicated to improving life (Nasdaq: DTIL) with its novel and proprietary ARCUS® genome editing platform that is designed to differ 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 where no adequate treatments exist. For more information about Precision BioSciences, visit www.precisionbiosciences.com.
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[1] iECURE has licensed the ARCUS® nuclease from Precision BioSciences for four gene insertion programs including OTC, CTLN1 and PKU.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240306494160/en/
Investors:
David Garrett
dgarrett@iecure.com
Media:
Janine Bogris
Evoke Canale
janine.bogris@evokegroup.com
Source: iECURE, Inc.
FAQ
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