NeuroNATA-R (lenzumestrocel) / CoreStem - LARVOL DELTA

Corestem Chemon’s stock plunges after failed P3 ALS drug trial (Korea Biomedical Review) - P3 | N=115 | ALSummit (NCT04745299) | Sponsor: Corestemchemon, Inc. | "The trial aimed to assess the therapeutic efficacy of Neuronata-R by comparing the joint rank score—an integrated analysis of function and survival duration—between the treatment and control groups at the 12-month mark. However, the company announced that no statistically significant differences were observed. Similarly, the comparison of therapeutic efficacy between the treatment and control groups at the six-month mark also failed to achieve statistical significance...The official stressed that the final clinical study report (CSR) is expected in March, and the company will continue further analyses to derive additional insights."

P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

Evaluate the Safety of Neuronata-R® Inj. Suspended With HypoTHermosol® FRS (HTS-FRS) in Patients With ALS (clinicaltrials.gov) - P1 | N=8 | Completed | Sponsor: Corestemchemon, Inc.

New P1 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

CorestemChemOn Completes Phase 3 Clinical Trial of ’Neuronata Al’, a New Drug for Lou Gehrig’s Disease…Challenges FDA Approval [Google translation] (eDaily) - "CorestemChemOn (166480) announced on the 2nd that the last administration of the phase 3 clinical trial of 'Neuronata R' (ingredient name Lenzumestrocel), a stem cell treatment for Lou Gehrig's disease (amyotrophic lateral sclerosis), was successfully completed. With the completion of the last administration and the end of the clinical trial, the company can now begin analyzing the clinical results of the 126 patients who participated in the phase 3 clinical trial that has continued for three years. The official announcement of the final results of the clinical trial is expected in the first quarter of next year, but the company's position is that key data could be received as early as this year."

P3 data • Trial status • Amyotrophic Lateral Sclerosis • CNS Disorders

ALSummit: Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients With ALS (clinicaltrials.gov) - P3 | N=115 | Active, not recruiting | Sponsor: Corestemchemon, Inc. | Trial primary completion date: Oct 2023 ➔ Dec 2024

Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders • CCL4 • IL10 • IL6 • TGFB1 • TNFA

A Propensity-Score-Matched Control Surveillance Study of Intrathecal Autologous Bone Marrow-Derived Mesenchymal Stem Cells (Neuronata-R®) Treatment in Patients with ALS to Assess the Long-Term Survival Benefits (ALS-MND 2023) - "The findings highlight lenzumestrocel as a promising autologous BM-MSC therapy for ALS. mhkim@csco.co.kr"

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

ALSummit: Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients With ALS (clinicaltrials.gov) - P3 | N=115 | Active, not recruiting | Sponsor: Corestem, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Amyotrophic Lateral Sclerosis • CNS Disorders

“Mesenchymal stem cell hopes for treatment of Lou Gehrig’s disease” Focus on Corestem Chemon’s ‘Neuronata-R’ (Yakup Shinmoon) - "On the 12th of last month, Corestemchemon published in the international academic journal 'Frontiers in aging neuroscience', 'Long-term survival benefits of intrathecal autologous bone marrow-derived mesenchymal therapy Neuronata-R in Lou Gehrig's disease'. stem cells (Neuronata-R®: lenzumestrocel) treatment in ALS: Propensity-score-matched control, surveillance study). Neuronata-R is a mesenchymal stem cell-based treatment for Lou Gehrig's disease....As a result, their overall predicted survival time was about 5.9 years (70.79 months). However, the average survival time actually observed was confirmed to be about 9.9 years (118.8 months). It can be seen that mesenchymal stem cell treatment had an effect on extending the survival period by about 4 years."

Clinical data • Amyotrophic Lateral Sclerosis • CNS Disorders

Long-term survival benefits of intrathecal autologous bone marrow-derived mesenchymal stem cells (Neuronata-R®: lenzumestrocel) treatment in ALS: Propensity-score-matched control, surveillance study. (PubMed, Front Aging Neurosci) - "Additionally, there were no serious adverse drug reactions found during the safety assessment, lasting a year after the first administration. The results of the present study showed that lenzumestrocel treatment had a long-term survival benefit in real-world ALS patients."

Journal • Amyotrophic Lateral Sclerosis • CNS Disorders

Efficacy and safety of Lenzumestrocel (Neuronata-R® inj.) in patients with amyotrophic lateral sclerosis (ALSUMMIT study): study protocol for a multicentre, randomized, double-blind, parallel-group, sham procedure-controlled, phase III trial. (PubMed, Trials) - P3 | "Lenzumestrocel is the orphan cell therapy product for ALS conditionally approved by the South Korea Ministry of Food and Drug Safety (MFDS). This ALSUMMIT protocol was developed for the adoption of enrichment enrolment, add-on design, and consideration of ethical issues for the placebo group."

Clinical • Clinical protocol • Journal • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

P3 study of Corestem's stem cell-derived Lou Gehrig's treatment proceeds smoothly (Korea Biomedical Review) - “Corestem said that its phase 3 clinical trial for Neuronata-R, a stem cell-derived Lou Gehrig's treatment, is proceeding smoothly as the company has registered more than 50 percent of patients participating in the trial….According to the company, the patient registration has already reached 52 percent in a mere three months since the company registered the first patient on Feb. 24.…'The company plans to complete the final patient administration by 2023, collect and analyze all data, and apply for a BLA to the FDA in 2024.'"

BLA • Enrollment status • Trial completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

ALSummit: Evaluatiton the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients With ALS (clinicaltrials.gov) - P3; N=115; Recruiting; Sponsor: Corestem, Inc.

Clinical • New P3 trial • Amyotrophic Lateral Sclerosis • CNS Disorders • Complement-mediated Rare Disorders

Core System Approves Phase III Clinical Plan for ALS Treatment The upper limit is reached [Google translation] (The Korea Economic Daily) - “Core System…announced on the 24th that the clinical trial phase 3 of Neuronata-Alju, a treatment for Lou Gehrig's disease (amyotrophic lateral sclerosis), has been approved by the US Food and Drug Administration (FDA). Corestem will begin phase 3 clinical trials in Korea and the US in Korea if the Korea Phase 3 clinical trial application is accepted.”

FDA event • New P3 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Coretem, Neuronata-Alju US FDA Phase 3 clinical trial plan pending [Google Translation] (eDaily) - "Corestem...announced on the 20th that it was notified of the US FDA's Phase 3 clinical trial plan clinical hold, Neuronata-Alju, a stem cell treatment. The company said that the clinical trial plan was held pending due to insufficient data related to characterization, manufacturing, and quality control ('CMC')....'According to US regulations and guidance, FDA will give an opinion on approval within 30 days after reviewing the complaints that have been filed.'"

Trial suspension

"Similarly the below could be a mass trial for 5,000 UK MND sufferers prior to FDA / EMA approval. A collaboration between the companies and NHS. #NurOwn #cuATSM #Tregs #NeuroNataR #BMMSCs https://t.co/61M2RvOVKn" (@CureMND)

European regulatory • FDA event

Corestem applies for FDA meet on Lou Gehrig's drug (Korea Biomedical Review) - “Corestem said Monday that it has applied for a pre-investigational new drug (Pre-IND) application consultation with the U.S. Food and Drug Administration regarding Neuronata-R Injection, the company's treatment for Lou Gehrig's disease...The company will meet with FDA officials in July to do the consultation."

IND