LiveKidney.bio announced today the enrollment of its first patient in a Phase I/IIa clinical trial evaluating LK-SC001, the company's proprietary umbilical cord-derived mesenchymal stromal cell (UC-MSC) therapy for lupus nephritis. The open-label study (NCT06737380) is being conducted at the Medical University of South Carolina (MUSC) and represents a significant milestone in the development of novel cell-based therapies for autoimmune kidney diseases.
The trial aims to assess the safety, tolerability, and efficacy of LK-SC001 as a treatment for patients with lupus nephritis and active systemic lupus erythematosus (SLE). This first-in-human study will evaluate whether the subcutaneously administered UC-MSC therapy can improve kidney function and control lupus disease activity.
Addressing a Critical Unmet Need
Lupus nephritis, a serious complication of SLE that affects the kidneys, presents a significant treatment challenge. Current standard therapies show limited efficacy, with only about 40% of patients responding adequately to available options.
"There is a real unmet medical need for innovative, safe and effective treatment for lupus nephritis patients," said Dr. Alon Yaar, CEO of LiveKidney. "The recruitment of the first patient marks a significant milestone for our company and can bring hope to millions of lupus patients worldwide."
Dr. Gary Gilkeson, Professor of Medicine and Associate Dean for Faculty Affairs and Faculty Development at MUSC, highlighted the limitations of existing treatments: "Current standard of care treatment for lupus is limited and effective in only 40% of patients. These therapies do not adequately address the complex immunological nature of the disease."
Novel Mechanism of Action
LK-SC001 utilizes a proprietary formulation of UC-MSCs delivered subcutaneously. The therapy is designed to release bioactive and immunomodulatory compounds that help regulate the immune system and reduce deterioration of kidney function.
The company's approach involves pharmaceutical-grade hydrogel-based formulations that allow for continuous release of therapeutic compounds from the UC-MSCs to the kidneys. This delivery system is intended to trigger anti-inflammatory and anti-fibrotic effects, potentially addressing the underlying pathophysiology of lupus nephritis.
"LK-SC001 offers a new approach designed to improve kidney function and reduce inflammation, addressing a critical gap in current treatment options for affected patients," Dr. Gilkeson explained.
Innovative Delivery Platform
LiveKidney's technology platform represents a potential paradigm shift in the treatment of kidney diseases. The company has developed a proprietary delivery system that enables the subcutaneous administration of UC-MSCs, which may offer advantages over traditional cell therapy approaches.
Dr. Yaar noted that the company's pipeline extends beyond lupus nephritis: "Our pipeline of future products is based on our proprietary delivery platform and is designed to address a wide range of kidney-related diseases."
Trial Design and Objectives
The Phase I/IIa trial will evaluate multiple aspects of LK-SC001's clinical profile:
- Safety and tolerability in patients with lupus nephritis and active SLE
- Preliminary efficacy signals in improving kidney function
- Impact on lupus disease activity
- Pharmacokinetic and pharmacodynamic properties of the subcutaneous formulation
As a first-in-human study, the trial represents a critical step in validating the potential of UC-MSCs as a therapeutic approach for autoimmune kidney diseases.
About LiveKidney
LiveKidney is a clinical-stage biotech company with offices in New York and Tel Aviv. The company focuses on developing cell-based therapies for kidney diseases, with a particular emphasis on preventing progression to kidney failure.
The company's technology leverages umbilical cord-derived mesenchymal stromal cells formulated in proprietary hydrogels. This approach aims to transform treatment paradigms for millions of patients with metabolic syndrome and autoimmune conditions affecting kidney function.
If successful, LiveKidney's platform could offer new hope for patients with limited treatment options and help address the growing global burden of kidney disease.
