JointStem (autologous adipose stem cell therapy) / K-STEMCELL - LARVOL DELTA

A Phase 2b/3a Study to Evaluate the Efficacy and Safety of JointStem in Patients Diagnosed as Knee Osteoarthritis (clinicaltrials.gov) - P2/3 | N=140 | Recruiting | Sponsor: Nature Cell Co. Ltd. | Phase classification: P2b ➔ P2/3 | Trial completion date: Dec 2024 ➔ Dec 2026 | Trial primary completion date: Jun 2024 ➔ Dec 2026

Head-to-Head • Phase classification • Trial completion date • Trial primary completion date • Immunology • Osteoarthritis • Pain • Rheumatology

Nature Cell stock soars after FDA breakthrough therapy designation (Chosun Biz) - "Nature Cell noted...that its stem cell therapy 'Jointstem' for degenerative joint disease was designated as a breakthrough therapy by the FDA. This is the first of its kind in South Korea. Being designated as a breakthrough therapy allows for intensive support from the FDA, including fast track status, which increases the likelihood of product approval...According to Nature Cell, Jointstem is a stem cell therapy for patients with severe knee osteoarthritis. It is said that a single injection into the knee joint space can result in pain reduction and improvement in joint function, with effects lasting for at least three years through cartilage regeneration."

Breakthrough therapy • Inflammation • Osteoarthritis • Pain

Lack of Systemic Effects from Intra-articular XT-150 for the Treatment of Moderate to Severe Pain Due to OA of the Knee: Safety Results from a Phase 2 Trial (ACR Convergence 2023) - P2 | "Background/Purpose: This study (NCT04124042) was designed to assess the safety, tolerability, and efficacy of XT-150, a non-viral gene therapy, in adult participants with moderate to severe pain due to osteoarthritis (OA) of the knee. Safety data from this study support continued investigation of XT-150 for the management of pain and improvement of physical function in patients with knee OA. The local nature of treatment-related AEs and the absence of treatment-related SAEs support a two-dose regimen of 0.45 mg XT-150 (total dose of 0.9 mg) as safe and well-tolerated for future studies. 1."

Clinical • P2 data • Cardiovascular • Gene Therapies • Immunology • Musculoskeletal Pain • Myocardial Infarction • Osteoarthritis • Pain • Rheumatology • IL10

Efficacy of XT-150, a Novel Non-Viral Gene Therapy Delivering IL-10v, on Moderate to Severe Pain Due to Osteoarthritis of the Knee: Results of a Phase 2 Trial (ACR Convergence 2023) - P2 | "Background/Purpose: Interleukin (IL)-10 is a regulator of cytokine activity and has the potential to restore homeostasis in osteoarthritis (OA) of the knee. These data suggest improvement in pain and function with two doses vs one dose of XT-150 and support the future development of repeat doses of XT-150 for the potential treatment of pain from moderate-to-severe knee OA. 1. Kwilasz AJ, et al."

Clinical • Gene therapy • P2 data • Gene Therapies • Immunology • Osteoarthritis • Pain • Rheumatology • IL10

A Phase 2b/3a Study to Evaluate the Efficacy and Safety of JointStem in Patients Diagnosed as Knee Osteoarthritis (clinicaltrials.gov) - P2b | N=140 | Recruiting | Sponsor: Nature Cell Co. Ltd. | Trial completion date: Dec 2023 ➔ Dec 2024 | Trial primary completion date: Jun 2023 ➔ Jun 2024

Head-to-Head • Trial completion date • Trial primary completion date • Immunology • Osteoarthritis • Pain • Rheumatology

Regulators give final rejection for Nature Cell's JointStem (Korea Biomedical Review) - "The Ministry of Food and Drug Safety (MFDS) has dismissed R Bio's appeal regarding the approval denial for Nature Cell's stem cell therapy, JointStem, to treat severe osteoarthritis....Earlier, the MFDS had requested supplementary data on the efficacy of JointStem during the approval review in April, but rejected the application due to lack of clinical significance. In response, R Bio filed an appeal against the rejection and submitted data on the feasibility of approval, including clinical significance, to the MFDS."

Non-US regulatory • CNS Disorders • Immunology • Osteoarthritis • Pain

Investigator Initiated Trial to Evaluate Cartilage Regeneration by Arthroscopy After JOINTSTEM Administration (clinicaltrials.gov) - P=N/A | N=21 | Not yet recruiting | Sponsor: R-Bio | Trial completion date: Dec 2023 ➔ Dec 2024 | Trial primary completion date: Feb 2022 ➔ Aug 2024

Trial completion date • Trial primary completion date • Immunology • Orthopedics • Osteoarthritis • Pain • Rheumatology

Follow-up Study for Participants of Jointstem Investigator Initiated Trial by Arthroscopy (clinicaltrials.gov) - P=N/A | N=14 | Not yet recruiting | Sponsor: R-Bio | Trial completion date: Dec 2028 ➔ Dec 2029 | Trial primary completion date: Dec 2027 ➔ Dec 2028

Trial completion date • Trial primary completion date • Immunology • Orthopedics • Osteoarthritis • Pain • Rheumatology

Follow-up Study for Participants of Jointstem Phase 3 Clinical Trial (clinicaltrials.gov) - P3 | N=129 | Active, not recruiting | Sponsor: R-Bio | Enrolling by invitation ➔ Active, not recruiting | N=260 ➔ 129

Enrollment change • Enrollment closed • Immunology • Osteoarthritis • Pain • Rheumatology

A Phase 2b/3a Study to Evaluate the Efficacy and Safety of JointStem in Patients Diagnosed as Knee Osteoarthritis (clinicaltrials.gov) - P2b | N=140 | Recruiting | Sponsor: Nature Cell Co. Ltd. | Trial completion date: Dec 2022 ➔ Dec 2023 | Trial primary completion date: Jun 2022 ➔ Jun 2023

Head-to-Head • Trial completion date • Trial primary completion date • Immunology • Osteoarthritis • Pain • Rheumatology

A Phase 2b/3a Study to Evaluate the Efficacy and Safety of JointStem in Patients Diagnosed as Knee Osteoarthritis (clinicaltrials.gov) - P2b; N=140; Recruiting; Sponsor: Nature Cell Co. Ltd.; Not yet recruiting ➔ Recruiting; Trial primary completion date: Dec 2021 ➔ Jun 2022

Enrollment open • Trial primary completion date • Immunology • Osteoarthritis • Pain • Rheumatology

A Phase 2 Study to Evaluate the Efficacy and Safety of JointStem in Treatment of Osteoarthritis (clinicaltrials.gov) - P2; N=28; Active, not recruiting; Sponsor: Nature Cell Co. Ltd.; Trial completion date: Mar 2018 ➔ Mar 2019; Trial primary completion date: Sep 2017 ➔ Mar 2018

Clinical • Trial completion date • Trial primary completion date • Immunology • Osteoarthritis • Pain • MRI

A Phase 2 Study to Evaluate the Efficacy and Safety of JointStem in Treatment of Osteoarthritis (clinicaltrials.gov) - P2; N=28; Completed; Sponsor: Nature Cell Co. Ltd.; Active, not recruiting ➔ Completed

Clinical • Trial completion • Osteoarthritis • Pain • Rheumatology • MRI

A Phase 2 Study to Evaluate the Efficacy and Safety of JointStem in Treatment of Osteoarthritis (clinicaltrials.gov) - P2; N=45; Recruiting; Sponsor: Nature Cell Co. Ltd.; Trial primary completion date: Mar 2017 ➔ Aug 2017

Clinical • Trial primary completion date • Immunology • Osteoarthritis • Pain • Rheumatology • MRI

A Phase 2 Study to Evaluate the Efficacy and Safety of JointStem in Treatment of Osteoarthritis (clinicaltrials.gov) - P2; N=30; Recruiting; Sponsor: Nature Cell Co. Ltd.; N=45 ➔ 30

Clinical • Enrollment change • Immunology • Osteoarthritis • Pain • Rheumatology • MRI

A Phase 2 Study to Evaluate the Efficacy and Safety of JointStem in Treatment of Osteoarthritis (clinicaltrials.gov) - P2; N=28; Active, not recruiting; Sponsor: Nature Cell Co. Ltd.; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Osteoarthritis • Pain • Rheumatology • MRI

A Phase 2 Study to Evaluate the Efficacy and Safety of JointStem in Treatment of Osteoarthritis (clinicaltrials.gov) - P2; N=45; Recruiting; Sponsor: Nature Cell Co. Ltd.

Clinical • New P2 trial • Immunology • Inflammation • Osteoarthritis • Pain • Rheumatology • MRI

Follow-up Study for Participants of Jointstem Investigator Initiated Trial by Arthroscopy (clinicaltrials.gov) - P=N/A; N=14; Not yet recruiting; Sponsor: R-Bio

New trial • Immunology • Orthopedics • Osteoarthritis • Pain • Rheumatology

Investigator Initiated Trial to Evaluate Cartilage Regeneration by Arthroscopy After JOINTSTEM Administration (clinicaltrials.gov) - P=N/A; N=21; Not yet recruiting; Sponsor: R-Bio

New trial • Immunology • Orthopedics • Osteoarthritis • Pain • Rheumatology

Follow-up Study for Participants Jointstem Clinical Trial (clinicaltrials.gov) - P2b; N=11; Completed; Sponsor: R-Bio; Active, not recruiting ➔ Completed; Trial completion date: Dec 2021 ➔ Dec 2020; Trial primary completion date: Jun 2021 ➔ Dec 2020

Clinical • Trial completion • Trial completion date • Trial primary completion date • Immunology • Osteoarthritis • Pain • Rheumatology

A Phase 3 Study to Evaluate the Efficacy and Safety of JointStem in Treatment of Osteoarthritis (clinicaltrials.gov) - P3; N=260; Completed; Sponsor: R-Bio; Active, not recruiting ➔ Completed; Trial primary completion date: Jun 2020 ➔ Dec 2020

Clinical • Trial completion • Trial primary completion date • Immunology • Osteoarthritis • Pain • Rheumatology • MRI

Investigator Initiated Trial to Evaluate the Efficacy and Safety of Intra-articular Injection of ADMSC Comared to Negative Control After High Tibial Osteotomy in the Osteoarthritis Patients. (clinicaltrials.gov) - P=N/A; N=26; Completed; Sponsor: R-Bio; Active, not recruiting ➔ Completed; Trial primary completion date: Feb 2020 ➔ May 2020

Clinical • Trial completion • Trial primary completion date • Immunology • Orthopedics • Osteoarthritis • Pain • Rheumatology • MRI

A Phase 2b/3a Study to Evaluate the Efficacy and Safety of JointStem in Patients Diagnosed as Knee Osteoarthritis (clinicaltrials.gov) - P2b; N=140; Not yet recruiting; Sponsor: Nature Cell Co. Ltd.; Initiation date: Dec 2020 ➔ Mar 2021

Clinical • Head-to-Head • Trial initiation date • Immunology • Osteoarthritis • Pain • Rheumatology

A Phase 2b/3a Study to Evaluate the Efficacy and Safety of JointStem in Patients Diagnosed as Knee Osteoarthritis (clinicaltrials.gov) - P2b; N=140; Not yet recruiting; Sponsor: Nature Cell Co. Ltd.; Trial completion date: Jul 2022 ➔ Dec 2022; Initiation date: Jun 2020 ➔ Dec 2020; Trial primary completion date: Jun 2021 ➔ Dec 2021

Clinical • Head-to-Head • Trial completion date • Trial initiation date • Trial primary completion date • Immunology • Osteoarthritis • Pain • Rheumatology

Follow-up Study for Participants of Jointstem Phase 3 Clinical Trial (clinicaltrials.gov) - P3; N=260; Enrolling by invitation; Sponsor: R-Bio

Clinical • New P3 trial • Immunology • Ocular Inflammation • Ophthalmology • Osteoarthritis • Pain • Rheumatology • MRI