LucaVax (tertomotide) / GemVax & KAEL, Samsung Pharm - LARVOL DELTA

A human telomerase reverse transcriptase-derived peptide GV1001 rescues neurodegeneration in a mouse model of Alzheimer disease. (PubMed, Exp Mol Med) - "Furthermore, we revealed that GV1001 facilitated microglial migration and amyloid β phagocytosis in an mTORC2-dependent manner. Collectively, our work demonstrates the amyloidolytic effects and the relevant in-depth signaling mechanism of GV1001 in microglia, suggesting GV1001 as a promising disease-modifying therapeutic agent for AD."

Journal • Preclinical • Alzheimer's Disease • CNS Disorders • TERT

GV1001: repurposing a telomerase-derived peptide for neurological therapeutics. (PubMed, Expert Opin Investig Drugs) - "Phase 3 confirmation, biomarker-driven patient stratification, and combination therapy investigations represent critical development priorities. Successful development may help validate multi-mechanism approaches in neurodegeneration, potentially catalyzing paradigm shifts from reductionist single-target strategies."

Journal • Review • Alzheimer's Disease • Cardiovascular • CNS Disorders • Depression • Immunology • Inflammation • Multiple Sclerosis • Oncology • Psychiatry

GV1001, hTERT Peptide Fragment, Prevents 5-Fluorouracil-Induced Mucositis by Inhibiting Mitochondrial Damages. (PubMed, Cells) - "GV1001 markedly mitigated these 5-FU-induced mitochondrial defects. Taken together, GV1001 mitigates CIM by most likely preserving mitochondrial integrity and function, supporting its potential as a strategy to prevent cancer chemotherapy-associated mucosal injury in patients."

Journal • Gastrointestinal Disorder • Hematological Disorders • Leukopenia • Metabolic Disorders • Mucositis • Oncology • TERT

GV1001 Reprograms CD47 Immune Checkpoint to Restore Macrophage Antitumor Activity in Oral Squamous Cell Carcinoma. (PubMed, Int J Mol Sci) - "GV1001 administered alone or in combination with cisplatin produced antitumor effects. Collectively, these findings demonstrate that GV1001 functions as a potent immunomodulatory anticancer peptide that downregulates CD47 expression and restores macrophage-mediated tumor clearance, highlighting its potential as a therapeutic strategy for OSCC."

Journal • Oncology • Oral Cancer • Squamous Cell Carcinoma • CD47 • TERT

The telomerase reverse transcriptase-derived peptide GN11 alleviates glycolysis and cellular senescence in the fibrotic kidney by inhibiting the TGF-β1/Smad3/lncRNA Oip5-os1/miR-30c-5p/Rock2 axis. (PubMed, Biochem Pharmacol) - "In this study, we discovered that GV1001(GV), a peptide fragment derived from human telomerase reverse transcriptase (hTERT), can inhibit the unilateral ureteral obstruction (UUO)-induced renal fibrosis...These findings suggest that GN11 has promising potential as a candidate molecule for the treatment of renal fibrosis and other types of organ fibrosis. Moreover, our research provides valuable insights and a scientific foundation for the development of novel therapeutic agents against renal fibrosis."

Journal • Chronic Kidney Disease • Fibrosis • Immunology • Nephrology • Renal Disease • MicroRNA 30c • ROCK2 • SMAD3 • TERT • TGFB1

GV1001 reduces pathological 4R tau and functional deficits in models relevant to progressive supranuclear palsy. (PubMed, Sci Rep) - No abstract available

Journal • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

GV1001 Subcutaneous for the Treatment of Moderate to Severe Alzheimer's Disease(AD) (clinicaltrials.gov) - P3 | N=750 | Not yet recruiting | Sponsor: Samsung Pharmaceutical Co., Ltd. | Trial completion date: Jun 2028 ➔ Jul 2031

Trial completion date • Alzheimer's Disease • CNS Disorders

Samsung Pharmaceutical announced on the 20th that it has applied for conditional approval of GV1001, a treatment for progressive supranuclear palsy (PSP), to the Ministry of Food and Drug Safety. [Google translation] (HIT News)

Korea filing • Progressive Supranuclear Palsy

Biomarkers. (PubMed, Alzheimers Dement) - "This study highlights the potential of monitoring immunomodulatory strategies to address the complex interplay between chronic neuroinflammation and protein aggregation in ND. If successful, these findings could inform the development of novel therapeutic approaches for AD and related disorders, bridging the gap between preclinical research and clinical application."

Biomarker • Journal • Alzheimer's Disease • CNS Disorders • Inflammation

Transcriptomic and functional annotation datasets for GV1001 peptide treatment in an experimental autoimmune encephalomyelitis mouse model. (PubMed, Data Brief) - "The dataset includes six tables detailing DEG lists, functional enrichment results, cell-type mapping, and microglial gene modulation. These data are valuable for researchers investigating neuroinflammation, glial cell biology, and peptide-based therapeutics, and can be reused for comparative analyses or hypothesis generation in central nervous system (CNS) disease models."

Journal • Preclinical • CNS Disorders • Immunology • Inflammation • Multiple Sclerosis

GemVax & KAEL announced on the 5th that it has signed a technology transfer agreement with Samsung Pharmaceutical on the right to clinical development and commercialization of four major Asian countries for the progressive supranuclear palsy (PSP) treatment 'GV1001.' (Maeil Business Newpaper) - "The technology transfer was aimed at South Korea, Japan, India, and Indonesia, with a total contract size of 220 billion won. Under the deal, GemVax will receive 11.5 billion won in advance payment from Samsung Pharmaceutical and 208.5 billion won in technical fees (milestone) for each phase of product approval. Technology usage fees (royalty) based on sales are set separately...Based on this, GemVax is preparing for a phase 3 clinical trial of PSP Global. PSP treatment GV1001 has been designated as a fast-track and rare drug by the U.S. Food and Drug Administration (FDA), and the European Medicines Agency (EMA) has also been designated as a rare drug, providing various benefits and support during global clinical trials."

Licensing / partnership • New P3 trial • Progressive Supranuclear Palsy

GV1001, an hTERT-Derived Peptide, Prevents Cisplatin-Induced Nephrotoxicity by Preserving Mitochondrial Function. (PubMed, Cells) - "Collectively, these findings demonstrated that GV1001 might protect kidney from cisplatin through maintaining mitochondrial structure and function and suppressing downstream injury cascades in renal epithelial cells. By directly targeting the mitochondrial mechanisms underlying cisplatin toxicity, GV1001 represents as a promising therapeutic strategy to mitigate CIN and improve the safety of cisplatin-based chemotherapy."

Journal • CNS Disorders • Renal Disease

Administration of GV1001 for the Treatment of Progressive Supranuclear Palsy Who Completed Study GV1001-PSP-CL2-011 (clinicaltrials.gov) - P2 | N=67 | Completed | Sponsor: GemVax & Kael | Active, not recruiting ➔ Completed | Trial completion date: Dec 2025 ➔ Sep 2025

Trial completion • Trial completion date • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

Evaluation of the Safety, Tolerability and PK Characteristics of GV1001 in Healthy Subjects (clinicaltrials.gov) - P1 | N=59 | Completed | Sponsor: GemVax & Kael | Recruiting ➔ Completed | Trial completion date: Dec 2025 ➔ Aug 2025

Trial completion • Trial completion date • CNS Disorders

GV1001, a tertomotide-derived peptide, alleviates motor dysfunction and extends survival in ALS (SOD1-G93A) transgenic mice (ALS-MND 2025) - "Notably, low-dose GV1001 not only improved neuromuscular performance but also modulated critical disease-associated pathways. Furthermore, the successful completion of a Phase 2a clinical trial in PSP reinforces its potential as a disease-modifying therapy for neurodegenerative diseases, warranting further clinical evaluation in ALS."

Preclinical • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders • Inflammation • Movement Disorders • Progressive Supranuclear Palsy • TERT

Gemvax's 'GV1001' fails to meet cognitive improvement target in Alzheimer's global phase 2 trial. [Google translation] (HIT News) - "The company announced that it had achieved statistical significance in the secondary endpoint (QoL-AD) that asks about the quality of life of patients, and that it had also confirmed strengths in safety, and that it would proceed with a global phase 3 design based on this....However, in the primary endpoint, ADAS-cog11, there was no significant difference compared to the placebo. According to the figures presented by Gemvax, the least squares mean change in ADAS-cog11 at week 52 was 4.14 points in the 0.56 mg GV1001 group, 4.31 points in the 1.12 mg group, and 4.30 points in the placebo group. The P value indicating statistical significance was 0.8989 in the 0.56 mg group and 0.9918 in the 1.12 mg group, failing to reach the significance level."

New P3 trial • P2 data • Alzheimer's Disease

Therapeutic effects of telomerase-derived peptide GV1001 in experimental autoimmune encephalomyelitis: Inhibiting neuroinflammation and promoting remyelination. (PubMed, Biochem Pharmacol) - "Co-culture assays confirmed that GV1001 blunts microglial neurotoxicity and facilitates remyelination during inflammation. Collectively, the data indicate that GV1001 modulates microglial activity to attenuate neuroinflammation and foster spinal cord repair, underscoring its promise as a multitarget therapy for inflammatory demyelinating diseases such as multiple sclerosis."

Journal • CNS Disorders • Immunology • Inflammation • Multiple Sclerosis • Oncology • Solid Tumor • IGF1 • PRMT1 • TERT

Multi-tracer PET monitoring of an immunomodulatory therapy in 4R tauopathy: Evaluating a novel drug's impact on glial function and protein pathology (EANM 2025) - "Anesthesia was induced with 3.0% isoflurane via mask at 3.5 L/min and maintained at 1.5% during imaging. Moreover, the results highlight the potential utility of MAO-B PET imaging as a translational biomarker for monitoring in vivo treatment effects. Collectively, these data provide supportive evidence for the continued evaluation of GV1001 as a candidate therapeutic for tauopathies such as PSP."

Immunomodulating • Alzheimer's Disease • Anesthesia • CNS Disorders • Inflammation • NEFL

GemVax & KAEL announced on the 23rd that it has completed the administration of a phase 2 extended clinical trial of "GV1001", a treatment for progressive supranuclear palsy (PSP). (Maeil Business Newpaper) - "According to GemVax, 67 patients participated in the extended clinical trial, completing the process of recent dosing and last observation."

Trial status • Progressive Supranuclear Palsy

GemVax & KAEL announced on the 22nd that it has applied for Breakthrough Therapy Designation (FDA) for GV1001, a treatment for progressive supranuclear palsy (PSP). (Maeil Business Newpaper) - "Gembax has proven its potential as a therapeutic agent by confirming the drug resistance of GV1001 and its tendency to slow the progression of the disease in clinical trials prior to phase 2 PSP. In addition, statistically significant effects compared to external controls were confirmed in the interim results analyzed by summing up the 6-month data from the preceding and extended clinical trials."

Breakthrough therapy • Progressive Supranuclear Palsy

GV1001 Subcutaneous(SC) for the Treatment of Mild to Moderate Alzheimer's Disease (AD) (clinicaltrials.gov) - P2 | N=199 | Completed | Sponsor: GemVax & Kael | Active, not recruiting ➔ Completed | Trial completion date: Mar 2026 ➔ Apr 2025

Trial completion • Trial completion date • Alzheimer's Disease • CNS Disorders

Gemvax Presents Results of Imaging Biomarker Study on 'GV1001' at International Conference [Google translation] (HIT News) - "Gemvax & KAEL (hereinafter Gemvax, 082270) has proven the potential of 'GV1001', which is being developed as a treatment for progressive supranuclear palsy (hereinafter PSP), to control neuroinflammation and tau pathology using positron emission tomography (PET) imaging...The study results confirmed that GV1001 can reduce neuroinflammation and tau pathology in a mouse model of tauopathy....'While astroglial activation was observed widely in the brains of the control group, it was limited to localized areas within the brain in the GV1001-administered group. Microglial activation was also reduced by GV1001 administration, and differences were particularly observed in the hippocampus and cortex regions.'"

Preclinical • Progressive Supranuclear Palsy

A randomized, active-controlled, multicenter, phase 3 clinical trial to evaluate the efficacy and safety of GV1001 in patients with benign prostatic hyperplasia. (PubMed, Prostate Int) - "No patients underwent prostate surgery or minimally invasive procedures during the study. GV1001 exhibited corresponding efficacy and tolerability, providing evidence of amelioration in urinary symptoms among patients with BPH in comparison to the use of 5 mg finasteride."

Journal • P3 data • Benign Prostatic Hyperplasia

Anti-depressant effects of a human telomerase-derived peptide GV1001 in an animal model of chronic restraint stress. (PubMed, Behav Brain Res) - "GV1001 efficiently reduced the increased serum corticosterone level and suppressed activation of the hypothalamic CRH neurons, suggesting the modulation of the hypothalamus-pituitary-adrenal axis. Collectively, our findings provide evidence for a novel antidepressant efficacy of GV1001."

Journal • Preclinical • CNS Disorders • Cognitive Disorders • Depression • Mood Disorders • Psychiatry • TERT

GemVax's GV1001 gets orphan drug status in Europe for progressive supranuclear palsy (Chosun Biz) - "GemVax announced on the 29th that its progressive supranuclear palsy (PSP) treatment drug 'GV1001' has been designated as an orphan medicinal product (OMP) by the European Medicines Agency (EMA)."

Orphan drug • Progressive Supranuclear Palsy