Unicyte AG, a Leader in Stem Cell Therapy Innovation, Advances Lead Program to Phase 2 Clinical Development

— Submits Clinical Trial Application for potentially life-saving therapy for pediatric patients with urea cycle disorders, setting the stage for patient enrollment in H2 2022 —

OBERDORF NW, Switzerland, April 28, 2022 (GLOBE NEWSWIRE) — Unicyte AG, a clinical-stage development company innovating the field of regenerative medicine, today announced the submission of a Clinical Trial Application (CTA) in Europe for a Phase 2 trial (reLiver-2) designed to assess the safety and efficacy of its proprietary human liver stem cell (HLSC) therapy, HLSC-001, in pediatric patients with early-onset urea cycle disorders (UCD). HLSC-001 is being evaluated in these patients initially as a bridging therapy prior to liver transplantation with the option and ultimate goal to provide a curative therapy. The company expects to initiate patient enrollment in the second half of 2022.

“Good outcomes for pediatric patients with UCD remain a challenge today despite the progress achieved in medical care. Brain damage in these patients correlates with the duration, frequency and severity of episodes of high ammonia levels that result from the inability to convert ammonia to urea,” said Prof. Johannes Haeberle, MD, at University Children’s Hospital Zurich, Switzerland. “While liver transplantation can provide curative therapy for patients, it cannot be performed right after birth and new approaches to normalizing ammonia levels prior to transplantation are essential for improving outcomes. HLSC-001 is a promising approach to providing urea cycle function during this critical period and has the potential to improve patient outcomes significantly. The planned Phase 2 trial will provide important insights into how HLSC-001 can be used to advance the care of children with UCD.”

Unicyte’s proprietary HLSC platform uses a novel population of stem cells derived from healthy human adult liver tissue. These cells have been shown to contribute to kidney and liver repair and regeneration in a variety of animal models and have also been shown to differentiate into a variety of pancreatic cell types, including cells that play critical roles in glucose regulation and diabetes. In UCD, HLSCs are injected directly into the patient’s liver, where they are expected to differentiate into urea-producing liver cells (hepatocytes) that can support clearance of ammonia through improved urea cycle activity. A previously completed Phase 1 investigator-initiated clinical trial conducted in three infants demonstrated that HLSC-001 had a very favorable safety profile and showed normal ammonia levels even as the infants’ daily protein intake increased by about 30 percent.

“Unicyte’s unique HLSC platform has significant potential to treat a range of kidney, liver and pancreatic disorders where our products can induce cell regeneration leading to improved organ function. Organ regeneration could catalyze a new era in medicine with significantly improved health outcomes,” said Florian Jehle, CEO of Unicyte. “Submission of the CTA for the Phase 2 UCD study is an important milestone in the development of this technology from cutting-edge science to potentially transformative therapies addressing substantial unmet patient need. Combined with our ongoing Phase 1 trial (reLiver-1), which is designed to provide important insight into UCD characteristics and support endpoint selection for future trials, this Phase 2 trial puts us on track to realize our vision of delivering on the curative potential of regenerative medicine.”

UCDs are a group of rare disorders that result from inherited deficiencies of components that comprise the urea cycle, a key enzymatic pathway required for metabolizing protein that is mediated by cells within the liver. These deficiencies lead to accumulation of high levels of ammonia, which can result in severe illness within hours or days of birth and may lead to irreversible damage if untreated. While UCD may be managed by restricting protein intake, use of ammonia-scavenging medications, and dialysis, curative treatment can only be achieved by liver transplantation, which is not recommended before three months of age due to higher rates of complications and lower survival rate. Unicyte is evaluating HLSC-001 as a bridging therapy that could restore urea cycle activity for three months to two years prior to transplantation or as longer-term therapy for patients who are ineligible for transplant.

The Phase 2 study is designed to evaluate the safety and efficacy of two percutaneous intrahepatic injections of HLSC-001 administered one week apart, with change in urea production from baseline as the primary efficacy endpoint. Safety endpoints include the frequency and severity of adverse events and treatment-emergent adverse events; change from baseline in vital signs, laboratory parameters and developmental and nutrition status; number and types of infections/illness; immunogenicity; and histological assessment of liver tissue at the time of transplant. The trial will be conducted at up to 15 sites throughout Europe and Middle East and will enroll up to 15 patients with UCD. Administration of HLSC-001 may be repeated and/or continued depending on individuals risk assessments, with a minimum of 12 weeks between each administration until the patient undergoes liver transplantation.

About Unicyte AG
Unicyte AG is a regenerative medicine company with a focus on kidney and liver disorders, diabetes, and oncology. Unicyte evolved from a long-term research collaboration of Professor Giovanni Camussi at Italy’s University of Turin and Fresenius Medical Care. Unicyte is headquartered in Oberdorf NW, Switzerland, and is an independent affiliate of Fresenius Medical Care, the world’s leading provider of products and services for individuals with renal diseases.

For more information visit the company’s website at www.unicyte.ch.

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