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

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

GV1001, a GemVax & KAEL (Gembax) treatment for progressive supranuclear palsy (PSP), has been approved for use for therapeutic purposes (Maeil) - "According to Gembax on the 21st, the Ministry of Food and Drug Safety (hereinafter referred to as the Ministry of Food and Drug Safety) approved the use of GV1001 for treatment purposes in two or more PSP patients on the 7th. The approval was announced earlier this month as attention was focused on the possibility of GV1001, which has acquired both rare drug and fast-track designations from the U.S. Food and Drug Administration (FDA), for therapeutic use (EAP) in the U.S....The approved content from Gembax is for patients with 2 or more and less than 25, and can be used for therapeutic purposes in patients wishing to receive additional administration after the PSP domestic phase 2 clinical trial of GV1001 and the ongoing extended clinical trial, depending on the medical staff's judgment."

Korea approval • Progressive Supranuclear Palsy

Gemvax, Progressive Supranuclear Palsy Treatment GV1001… FDA Fast Track Designation [Google translation] (Hankyung) - "Gemvax Ankael announced on the 7th that its progressive supranuclear palsy (PSP) treatment 'GV1001' received Fast Track designation from the U.S. Food and Drug Administration (FDA)."

Fast track • Progressive Supranuclear Palsy

GemVax & KAEL wins FDA orphan drug designation for PSP treatment (Korea Biomedical Review) - "GemVax & KAEL said Friday that GV1001, its treatment for progressive supranuclear palsy (PSP), has received orphan drug designation (ODD) from the U.S. FDA....GemVax & KAEL is preparing for a global phase 3 clinical trial of GV1001. GV1001 is a multi-mechanism drug that leverages various effects of telomerase, including anti-aging, antioxidant, and anti-inflammatory properties."

Orphan drug • Progressive Supranuclear Palsy

GV1001, hTERT Peptide Fragment, Prevents Doxorubicin-Induced Endothelial-to-Mesenchymal Transition in Human Endothelial Cells and Atherosclerosis in Mice. (PubMed, Cells) - "In conclusion, GV1001 appears to prevent doxorubicin-induced atherosclerosis by safeguarding vascular endothelial cells from mitochondrial dysfunction, inflammation, and phenotypic changes. These findings suggest the potential of GV1001 as a therapeutic agent to mitigate the long-term cardiovascular side effects associated with doxorubicin treatment in humans."

Journal • Preclinical • Alzheimer's Disease • Atherosclerosis • Cardiomyopathy • Cardiovascular • CNS Disorders • Metabolic Disorders • Oncology

[Featured Stock] Gemvax, for Proving the Effect of Suppressing Side Effects of 'GV1001' Anticancer Drug [Google translation] (Nate) - "On this day, Gemvax announced that a paper that recently clarified the possibility of GV1001 preventing the side effects of anticancer drugs was published in 'Cells'...In this study, we confirmed the possibility that GV1001 can reduce the side effects of chemotherapy and improve the quality of life of chemotherapy patients by preventing vascular damage caused by doxorubicin. The research related to this paper was conducted by the research team at the UCLA School of Dentistry in the United States...According to the paper, GV1001 effectively inhibited endothelial-mesenchymal transition (EndMT) caused by oxidative stress, inflammatory response, and mitochondrial dysfunction induced by doxorubicin. In addition, GV1001 was shown to reduce systemic and vascular inflammation, lipid accumulation, and macrophage infiltration caused by doxorubicin in apolipoprotein E (ApoE)-deficient mice."

Preclinical • Oncology

Gembax Presents PSP Phase 2a Topline Results at International Conference Poster [Google translation] (Medipana) - P2a | N=78 | NCT05819658 | Sponsor: GemVax & Kael | "Parkinson's Disease and Movement Disorders (7th TIC-PDMD)' held in Taipei, Taiwan from the 22nd to the 24th and presented the phase 2a clinical data of the PSP treatment GV1001...In a Phase 2a clinical trial of PSP, Gemvax said in a poster that GV1001 0.56 mg delayed disease progression by 48% compared to placebo, and that 75% of PSP patients with PSP-RS delayed disease progression by 95%...Gemvax is well underway in its global Phase 2 clinical trial for Alzheimer's disease in the U.S. and seven European countries and expects to complete the clinical trial dosing in the first half of next year and confirm the results."

P2a data • Trial status • Alzheimer's Disease • CNS Disorders • Progressive Supranuclear Palsy

Gemvax, GV1001 Phase 3 Clinical Trial Outlines Revealed…Targeting FDA Product Approval [Google translation] (Nate) - "The groundwork for the success of the Phase 3 clinical trial of Gemvax's (082270) progressive supranuclear palsy (PSP) treatment 'GV1001' has been completed. Gemvax has meticulously aimed for FDA product approval of GV1001 in terms of clinical scale, usage, and clinical subject selection. According to Gemvax on the 22nd, the Phase 3 clinical trial of GV1001 for PSP is planned to be conducted as a multinational clinical trial in the US, Korea, etc. around the fourth quarter of next year. The Phase 2b clinical trial will be omitted."

New P3 trial • Progressive Supranuclear Palsy

GemVax Announces Topline Results from Phase 2a Progressive Supranuclear Palsy Clinical Trial at Neuro2024 (PRNewswire) - P2a | N=78 | NCT05819658 | Sponsor: GemVax & Kael | "GemVax & KAEL Co., Ltd...announced that topline results of a Phase 2a clinical trial...of GV1001, an investigational peptide drug for the treatment of progressive supranuclear palsy ('PSP'), were presented at 'Neuro2024: The PSP and CBD International Research Symposium' in Toronto, Canada, at 4:45 p.m. local time on 24th October....Many PSP-RS type patients in the treatment group experienced symptom stabilization or even improvement during the clinical period. When calculated as responder rate based on the percentage of patients whose PSP Rating Scale scores improved or remained stable after six months of treatment compared to baseline, 58.33% of PSP-RS type patients in the 0.56 mg GV1001 group showed improvement or stabilization....The safety profile of GV1001 in the Phase 2a PSP Clinical Trial was consistent with prior safety data."

P2a data • Alzheimer's Disease • CNS Disorders

GemVax & KAEL’s GV1001 fails to show effects in P2a trial of progressive supranuclear palsy (Korea Biomedical Review) - P2a | N=78 | NCT05819658 | Sponsor: GemVax & Kael | "Results showed that the mean change in PSP-rating scale total score (LS Mean) from baseline to 24 weeks post-treatment for each treatment arm was +2.14 points in the GV1001 0.56 mg arm, +6.46 points in the GV1001 1.12 mg arm, and +4.10 points in the control arm. The difference in PSP-rating scale total score change between treatment groups was -1.96 points between GV1001 0.56 mg and control and +2.36 points between GV1001 1.12 mg and control, with no statistically significant differences observed...GemVax & KAEL plans to receive a complete study report (CSR) in the first half of 2025...In a separate release on the same day, GemVax & KAEL argued that the LS mean, calculated using the MMRM estimator, showed a 2.14-point worsening in the GV1001 0.56 mg arm over six months compared to a 4.10-point worsening in the placebo arm, resulting in a 48 percent delay in disease progression in the GV1001 arm."

P2a data • CNS Disorders • Progressive Supranuclear Palsy

Administration of GV1001 for the Treatment of Progressive Supranuclear Palsy Who Completed Study GV1001-PSP-CL2-011 (clinicaltrials.gov) - P2 | N=67 | Active, not recruiting | Sponsor: GemVax & Kael | Recruiting ➔ Active, not recruiting | Trial completion date: Jul 2025 ➔ Dec 2025 | Trial primary completion date: Dec 2024 ➔ Nov 2025

Enrollment closed • Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy