• TED-A9 is high-purity midbrain dopaminergic progenitor cells derived from human embryonic stem cells (hESCs) under rigorous GMP conditions
• Investigational cell therapy with TED-A9 completed transplantation of 12 participants in a Phase I/IIa clinical trial in February 2024
• No major safety issues in 3 low dose participants (first low dose cohort) through one year
• 3 low dose participants (first low dose cohort) showed clinical improvement with cell survival and engraftment after 12 months

SEOUL, South Korea: #ClinicalTrials--S.BIOMEDICS (KOSDAQ: 304360) announced positive one-year assessment data from the first 3 participants (first low dose cohort) of Phase I/IIa clinical trial for Parkinson’s disease with TED-A9, hESC (human embryonic stem cell)-derived midbrain dopaminergic progenitors. The data demonstrates that 3 participants showed safety and efficacy of TED-A9 in a one-year follow-up.

The clinical trial was conducted at Severance Hospital of Yonsei University in South Korea led by Prof. Jin-Woo Chang, a neurosurgeon and Prof. Phil Hyu Lee, a neurologist.

For the 3 participants who received initial low dose (3.15 million cells), MRI and CT scans after one year revealed no adverse effects related to the cell transplantation or surgery.

Moreover, the MDS-UPDRS Part III (off) evaluation, which objectively measures motor functions, showed a mean decrease of 12.7 points, from baseline 61.7 to 49.0 one-year post treatment. The MDS-UPDRS Part III (off) evaluation indicated significant improvement in their motor abilities. Improvement was also observed in symptoms such as wearing off and freezing of gait.

DAT brain imaging (FP-CIT-PET), conducted one-year post transplantation, revealed an increase in dopamine transporters (DAT), suggesting the potential engraftment of dopamine neurons, which correlated with improvements in the patients' Parkinson's symptoms.

“Although this clinical evaluation only targets the first 3 low-dose patients, but not all 12 subjects, no adverse issues related to transplant surgery or cell safety were observed in one-year post transplantation. Importantly, the clinical results demonstrated very promising efficacy,” said Prof. Dong-Wook Kim at Yonsei University College of Medicine and CTO of S.BIOMEDICS. “The results are believed to align closely with the findings from our preclinical in vitro and in vivo studies. We are excited that TED-A9 could be a fundamental treatment that directly replaces dopaminergic neurons lost in patients with Parkinson's disease.”

Assessment data of further participants (first high dose cohort) will be announced in September or October of 2024 after a one-year follow-up.

About TED-A9 and Phase I/IIa clinical trial

TED-A9 is an investigational cell therapy designed to replace ventral midbrain-specific dopaminergic cells lost in patients with Parkinson's disease. These ventral midbrain-specific dopaminergic cells are derived from hESCs by exclusively utilizing small molecules only. TED-A9 represents a significant advancement in the field, offering highly purified dopamine cells derived from hESCs. Through surgical procedure, these hESC-derived dopaminergic progenitor (precursor) cells (TED-A9) are transplanted to three segments of the putamen; the anterior, middle, and posterior sections, with three tracks per each putamen. Bilateral putamina received cell transplantation in a single surgical procedure, with cells injected at three points within each track. After transplantation, the expectation is that the transplanted dopaminergic progenitor cells will mature into dopaminergic neurons which will supply the dopamine that Parkinson’s patients are lacking, restoring the motor function of patients.

The Phase I/IIa clinical trial is conducted on 12 participants who have been diagnosed with Parkinson's disease for more than 5 years and exhibited motor complications such as wearing-off, freezing of gait or dyskinesia. The participant’s age was between 50 and 75 years old. TED-A9 were administered to 6 participants in the low-dose group (3.15 million cells) and to another 6 participants in the high-dose group (6.30 million cells).

An initial cohort of three patients was enrolled at a low dose to assess initial safety, including dose-limiting toxicity (DLT) evaluation, over a period of up to 3 months post-transplantation. There were no safety concerns within this timeframe. Thus, an additional 3 patients were enrolled at a high dose for evaluation over another 3-month period post-transplantation. As no safety issues arose during this extended period, the clinical trial continued by adding three further patients to each of the low-dose and high-dose groups, totaling 12 patients. The final participant was administered with TED-A9 on February 2024.

The primary objective of the Phase I/IIa trial is to assess the safety and exploratory efficacy of TED-A9 transplantation over two years post-transplant. Safety will be monitored for additional 3 years, making it a total 5 years.

About S.BIOMEDICS

Established in 2005, S.BIOMEDICS is at the forefront of stem cell therapy, focusing on regenerative medicine powered by data-driven biology. Based on two core platform technologies, S.BIOMEDICS currently develops seven cell therapy programs, targeting non-curable diseases. Its leading programs are under clinical stage accelerating the journey of cell medicine as shown below:

• TED-A9: Ventral midbrain-specific dopaminergic progenitor cells derived from hESCs for Parkinson’s disease (Phase 1/2a)
• TED-N: PSA-NCAM-positive neural progenitor cells derived from hESCs for spinal cord injury (Phase 1/2a)
• FECS-AD: 3D MSC spheroids for critical limb ischemia (Phase 1/2a)

For more information about S.BIOMEDICS, visit www.sbiomedics.com. S.BIOMEDICS is listed on the Korea Exchange (KOSDAQ: 304360) and is also the founder and controller of S.THEPHARM (www.sthepharm.com), a corporation specializing in anti-aging products such as HA-Filler. More Information about the Phase 1/2a clinical trial for Parkinson’s disease is available at ClinicalTrials.gov (NCT05887466).

Fonte: Business Wire