restem-L / Restem - LARVOL DELTA

Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD) (clinicaltrials.gov) - P1 | N=16 | Recruiting | Sponsor: Restem, LLC. | N=24 ➔ 16

Enrollment change • Muscular Dystrophy

Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD) (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: Restem, LLC. | Not yet recruiting ➔ Recruiting

Enrollment open • Muscular Dystrophy

IIMPACT: Safety and Efficacy of ULSC on Disease Severity and Steroid Tapering in Participants With Dermatomyositis/ Polymyositis (DM/PM), Also Known as Idiopathic Inflammatory Myopathy (IIM) (clinicaltrials.gov) - P2/3 | N=40 | Not yet recruiting | Sponsor: Restem, LLC.

New P2/3 trial • Dermatomyositis • Immunology • Myositis

Rationale and Study Design for a Phase 2, Double-blinded, Randomized, Dose-repeating, Cross-over Study to Assess Safety and Efficacy of Allogeneic Umbilical Cord Lining Stem Cells on Disease Severity and Steroid Tapering in Participants with Idiopathic Inflammatory Myopathy (EULAR 2025) - P1 | "The infusion was temporarily paused, 150 mg intravenous hydrocortisone administered, and the infusion re-initiated with no recurrence...Average prednisone dosage decreased by 35% at 6 months, when excluding data from one subject (a non-responder by TIS) who had a flare treated with corticosteroids at that time point. Taken together, the overall human experience with the intravenous infusion of ULSC has demonstrated an absence of adverse outcomes attributable to this investigational product, and in the context of DM/PM, has demonstrated early evidence of clinical improvement in multiple participants using complementary outcome measures, in the context of progressive reduction in steroid dosing. A phase 2 trial has been designed that will further evaluate safety and efficacy endpoints."

Clinical • P2 data • Dermatomyositis • Metabolic Disorders • Muscular Dystrophy • Myositis • Pain

RESTEM Presents Phase 1 Data of its ULSC Program in Polymyositis/Dermatomyositis at ACR Convergence 2024 (GlobeNewswire) - P1 | N=10 | NCT04723303 | "'The treatment was well tolerated and led to clinically meaningful and durable improvements in the TIS in 78% of patients...Based on the promising results from Phase 1 we look forward to advancing the study to Phase 2/3 in 1Q 2025.'...Efficacy data showed that 6 of 9 patients met the criteria for a moderate improvement with a total improvement score (TIS) of ≥40% - a composite outcome of six core measures of myositis activity within 6 months of the infusion; The criterion for clinically significant improvement, TIS of ≥20%, was met by 7 out of 9 subjects; The average TIS was sustained in the 30-35% range over a 12-month period; Manual Muscle Testing (MMT8) showed a significant improvement in strength between baseline and Month 6 of nearly 10 points, from 59 ± 4 at baseline to 68 ± 4 at Month 6 (mean ± SEM), p< 0.001."

New P2/3 trial • P1 data • Dermatomyositis • Myositis

RESTEM Provides Corporate Update and Announces Key Business Objectives for 2025 (GlobeNewswire) - "Expected Milestones in 2025: Advance Restem-L in IIM to a potentially registrational, randomized, double-blinded Phase 2/3 trial in Q1 2025...Initiate Phase 2 clinical trial in rheumatoid arthritis patients who are refractory to standard of care in the second half of 2025."

New P2 trial • New P2/3 trial • Dermatomyositis • Myositis • Rheumatoid Arthritis

RESTEM Receives FDA Fast Track Designation for Restem-L for Idiopathic Inflammatory Myopathy (GlobeNewswire) - "RESTEM...today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for Restem-L, the Company’s umbilical cord outer lining stem cells (ULSCs) program for the treatment of Polymyositis and Dermatomyositis (PM/DM), a rare autoimmune disease, now defined by the FDA as Idiopathic Inflammatory Myopathy (IIM)...'The recently presented Phase 1 data demonstrated that treatment with our Restem-L led to 78%.'....'We look forward to advancing the Restem-L program development and initiating IIMPACT, a potentially registrational Phase 2/3 clinical trial in IIM in Q1 2025.'"

Fast track • New P2/3 trial • Dermatomyositis • Inflammation

Safety and Efficacy Data from a Phase I Trial of Umbilical Lining-Derived Stem Cells (ULSC) in Adult Dermatomyositis/Polymyositis (ACR Convergence 2024) - P1 | "Umbilical cord-derived mesenchymal stromal cells, a classification of cells that includes umbilical cord lining stem cells (ULSC) may be associated with a younger cellular phenotype with minimal expression of HLA-DR after activation, which is expected to correlate with reduced immunogenic potential...The infusion was temporarily paused, 150 mg intravenous hydrocortisone administered, and the infusion re-initiated with no recurrence... Taken together, the overall human experience with the intravenous infusion of ULSC has demonstrated an absence of adverse outcomes attributable to this investigational product, and in the context of DM/PM, has demonstrated early evidence of clinical improvement in multiple participants using complementary outcome measures, in the context of progressive reduction in steroid dosing."

Clinical • P1 data • Dermatomyositis • Immunology • Metabolic Disorders • Muscular Dystrophy • Myositis

Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM) (clinicaltrials.gov) - P1 | N=10 | Completed | Sponsor: University of Florida | Active, not recruiting ➔ Completed | Trial primary completion date: Mar 2023 ➔ Dec 2023

Stroma • Trial completion • Trial primary completion date • Dermatomyositis • Immunology • Myositis

Umbilical Cord Lining Stem Cells (ULSC) in Patients With COVID-19 ARDS (clinicaltrials.gov) - P1/2 | N=17 | Completed | Sponsor: Restem, LLC. | Active, not recruiting ➔ Completed | Trial completion date: Feb 2024 ➔ May 2023 | Trial primary completion date: Dec 2023 ➔ May 2023

Trial completion • Trial completion date • Trial primary completion date • Infectious Disease • Novel Coronavirus Disease • Respiratory Diseases

Umbilical Cord Lining Stem Cells (ULSC) in Patients With COVID-19 ARDS (clinicaltrials.gov) - P1/2 | N=17 | Active, not recruiting | Sponsor: Restem, LLC. | Trial completion date: Jul 2023 ➔ Feb 2024 | Trial primary completion date: Apr 2023 ➔ Dec 2023

Trial completion date • Trial primary completion date • Infectious Disease • Novel Coronavirus Disease

Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM) (clinicaltrials.gov) - P1 | N=10 | Active, not recruiting | Sponsor: University of Florida | Trial completion date: Apr 2023 ➔ Apr 2024

Stroma • Trial completion date • Dermatomyositis • Immunology • Myositis

Umbilical Cord Lining Stem Cells (ULSC) in Patients With COVID-19 ARDS (clinicaltrials.gov) - P1/2 | N=17 | Active, not recruiting | Sponsor: Restem, LLC. | Trial completion date: Dec 2022 ➔ Jul 2023 | Trial primary completion date: Nov 2022 ➔ Apr 2023

Trial completion date • Trial primary completion date • Infectious Disease • Novel Coronavirus Disease

Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM) (clinicaltrials.gov) - P1 | N=10 | Active, not recruiting | Sponsor: University of Florida | Recruiting ➔ Active, not recruiting | N=22 ➔ 10

Enrollment change • Enrollment closed • Stroma • Dermatomyositis • Immunology • Myositis

Umbilical Cord Lining Stem Cells (ULSC) in Patients With COVID-19 ARDS (clinicaltrials.gov) - P1/2 | N=17 | Active, not recruiting | Sponsor: Restem, LLC. | Recruiting ➔ Active, not recruiting | N=60 ➔ 17 | Trial completion date: Dec 2021 ➔ Dec 2022 | Trial primary completion date: Oct 2021 ➔ Sep 2022

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Infectious Disease • Novel Coronavirus Disease

Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM) (clinicaltrials.gov) - P1 | N=22 | Recruiting | Sponsor: University of Florida | Trial completion date: Apr 2022 ➔ Apr 2023 | Trial primary completion date: Apr 2022 ➔ Apr 2023

Trial completion date • Trial primary completion date • Dermatomyositis • Immunology • Myositis

Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM) (clinicaltrials.gov) - P1; N=9; Recruiting; Sponsor: University of Florida; Initiation date: Mar 2021 ➔ Aug 2021

Clinical • Trial initiation date • Dermatomyositis • Immunology • Myositis

Umbilical Cord Lining Stem Cells (ULSC) in Patients With COVID-19 ARDS (clinicaltrials.gov) - P1/2; N=60; Recruiting; Sponsor: Restem, LLC.; Trial primary completion date: Oct 2020 ➔ Oct 2021

Clinical • Trial primary completion date • Infectious Disease • Novel Coronavirus Disease

Umbilical Cord Mesenchymal Stem Cells Used to Successfully Treat 3 Critically Ill COVID-19 Patients - "'The remarkable ability for these cells to mitigate inflammatory processes holds great promise for COVID-19 patients as well as for people with many other illnesses,' said Dr. Koehne....'These patients have improved their lung status much more quickly than patients treated with other experimental therapies,' added Dr. Koehne....Specialists at HWCOM spearheaded this collaborative effort between academia, Baptist Health and RESTEM."

Media quote

Healthcare worker recovers from coronavirus with help of stem cells - "Doctors at Baptist Health South Florida teamed up RESTEM, a bio-technology company, to treat four coronavirus patients through intravenous infusions. Dr. Guenther Koehne...said, 'We got immediate approval, with the strong support of the FDA.'"

Media quote

New Stem Cell Therapy for COVID-19 Finds Success in Clinical Trial at Baptist Health (YouTube) - "In a clinical trial being conducted under an emergency approval from the Food and Drug Administration (FDA)...physicians Guenther Koehne, M.D., Ph.D...safely administered the new treatment to patients through intravenous infusions of experimental umbilical cord lining-derived stem cells (ULSC)."

Video

Umbilical Cord Lining Stem Cells (ULSC) in Patients With COVID-19 ARDS (clinicaltrials.gov) - P1/2; N=60; Recruiting; Sponsor: Restem, LLC.

Clinical • New P1/2 trial • Infectious Disease • Novel Coronavirus Disease • PCR