Initiation of Phase II Clinical Trial for Regenerative Medical Products Using Allogeneic Mesenchymal Stem Cells for Severe COVID-19 Pneumonia Patients

Background

Reports on COVID-19 have indicated that approximately 20% of cases progress to severe pneumonia. This severity has been associated with a phenomenon called "cytokine storm," in which the immune cells of infected individuals uncontrollably release cytokines while fighting the virus. Therefore, it is believed that treatment for severe COVID-19 should not only focus on eliminating the virus but also on addressing the cytokine storm. Mesenchymal stem cell therapy using allogeneic mesenchymal stem cells has been explored worldwide, with multiple reports suggesting its potential effectiveness in treating severe COVID-19 cases. Mesenchymal stem cells are believed to possess anti-inflammatory properties through various mechanisms, making them a promising treatment option for patients who do not respond to approved COVID-19 therapies. ROHTO Pharmaceutical is committed to advancing research in this area to establish a mesenchymal stem cell-based treatment approach for COVID-19 and future infectious disease management.

About ADR-001

ROHTO Pharmaceutical's investigational product, "ADR-001," is a cell-based therapy composed of allogeneic adipose tissue-derived stem cells. ADR-001 is cultured using a proprietary serum-free medium designed to maximize the potential of adipose tissue-derived stem cells without the inclusion of animal-derived materials, reducing the risk associated with animal-origin viral infections. Adipose tissue is known to contain numerous mesenchymal stem cells, and its collection is relatively non-invasive, with excess tissue often available from surgeries. This makes it easily accessible for transplantation with allogeneic adipose tissue-derived cells, offering a rapid supply for eligible patients.
Following our ongoing clinical trial for patients with liver cirrhosis※, ROHTO Pharmaceutical has proceeded with the Phase I clinical trial of ADR-001 to assess its safety in severe COVID-19 pneumonia patients. As a result of the Phase I trial confirming a certain level of safety, we have planned a Phase II trial with efficacy as the primary endpoint. This trial will include an ADR-001 treatment group and a placebo group, with a comparative study between the two.※A Phase I/II Trial of ADR-001 in Subjects with Cirrhosis.

Clinical Trial Overview

Trial Name: Phase II Clinical Trial of ADR-001 for Severe Pneumonia Caused by SARS-CoV-2 Infection

• Target Disease: Severe pneumonia caused by SARS-CoV-2 infection
• Trial Design: Randomized, placebo-controlled, double-blind, intergroup comparative trial
• Primary Endpoint: Efficacy
• Administration Method: Intravenous infusion
• Planned Enrollment: 20 participants
• Scheduled Trial Duration: June 2021 to September 2022