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

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

GV1001 Subcutaneous(SC) for the Treatment of Progressive Supranuclear Palsy (PSP) (clinicaltrials.gov) - P2 | N=78 | Completed | Sponsor: GemVax & Kael | Recruiting ➔ Completed | Trial completion date: May 2024 ➔ Oct 2024 | Trial primary completion date: Dec 2023 ➔ Oct 2024

Trial completion • Trial completion date • Trial primary 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=56 | Recruiting | Sponsor: GemVax & Kael | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders

Evaluation of the Safety, Tolerability and PK Characteristics of GV1001 in Healthy Subjects (clinicaltrials.gov) - P1 | N=56 | Not yet recruiting | Sponsor: GemVax & Kael

New P1 trial • CNS Disorders

Gemvax Announces Phase 2 Topline Progressive Supranuclear Palsy at Canadian Academic Conference in October [Google translation] (Hankyung) - "Gemvax & KAEL announced on the 25th that it has confirmed its participation as a signature sponsor in the international research symposium 'Neuro2024' on progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD). In addition, it plans to introduce the domestic PSP phase 2 clinical trial topline results and hold a related briefing session at this conference...The global PSP clinical trial is currently in preparation after receiving approval for the phase 2 clinical trial plan from the U.S. Food and Drug Administration (FDA) in February."

P2 data • CNS Disorders • Progressive Supranuclear Palsy

Gemvax, October PSP Phase 2 Clinical Trial Topline Announcement… Strong Expectation of 20 Trillion Won Sales Guaranteed [Google translation] (Money Today) - "Gemvax...is on a strong trend after news that it will announce the topline of the phase 2 clinical trial for progressive supranuclear palsy (PSP) indication for its neurodegenerative disease treatment 'GV1001' in Canada in October...Gemvax plans to apply for conditional approval to the Ministry of Food and Drug Safety once the phase 2 clinical trial results are out. It is also separately planning a global phase 3 clinical trial."

P2 data • CNS Disorders • Progressive Supranuclear Palsy

Gemvax ’Will Develop New Drugs for Brain Diseases Without Treatment’ [Google translation] (Hankyung) - "Gemvax is currently conducting domestic phase 2 clinical trials targeting 78 PSP patients...'We will complete administering the drug to the last patient participating in phase 2 clinical trials this month,' and 'The clinical topline will come out within the year or early next year, which will determine the possibility of it being the first-in-class new drug in the series.'"

Enrollment status • P2 data • CNS Disorders • Progressive Supranuclear Palsy

The Cell-Penetrating Peptide GV1001 Enhances Bone Formation via Pin1-Mediated Augmentation of Runx2 and Osterix Stability. (PubMed, Biomolecules) - "GV1001 demonstrated protective effects against bone loss in OVX mice by upregulating osteogenic differentiation via the Pin1-mediated protein stabilization of Runx2 and Osterix. GV1001 could be a potential candidate with anabolic effects for the prevention and treatment of osteoporosis."

Journal • Oncology • Osteoporosis • Rheumatology • PIN1 • RUNX2

Beyond lecanemab: Examining Phase III potential in Alzheimer's therapeutics. (PubMed, PCN Rep) - "The paper highlights the US Food and Drug Administration's approval of aducanumab (Aduhelm) and lecanemab (Leqembi), emphasizing the developmental status of new treatments...The review focuses on amyloid removal (donanemab), tau protein mitigation (E2814), drug repositioning (Semaglutide, GV1001), and disease-modifying small molecules (fosgonimeton, hydralazine, masitinib). However, Gantenerumab and Solanezumab, unsuccessful in Phase III, are not covered. While the future approval status remains uncertain, we hope these drugs will offer beneficial therapeutic effects for potential dementia patients."

Journal • P3 data • Alzheimer's Disease • CNS Disorders • Dementia

hTERT Peptide Fragment GV1001 Prevents the Development of Porphyromonas gingivalis-Induced Periodontal Disease and Systemic Disorders in ApoE-Deficient Mice. (PubMed, Int J Mol Sci) - "GV1001 also suppressed the accumulation of AD biomarkers in the brains of mice with periodontal disease. Overall, these findings suggest that GV1001 holds promise as a preventive agent in the development of atherosclerosis and AD-like conditions associated with periodontal disease."

Journal • Preclinical • Alzheimer's Disease • Atherosclerosis • Cardiovascular • CNS Disorders • Dental Disorders • Dyslipidemia • Oncology • Periodontitis • APOE

GV1001 Subcutaneous(SC) for the Treatment of Mild to Moderate Alzheimer's Disease (AD) (clinicaltrials.gov) - P2 | N=180 | Active, not recruiting | Sponsor: GemVax & Kael | Trial primary completion date: Sep 2025 ➔ Apr 2025

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

GV1001 Subcutaneous(SC) for the Treatment of Mild to Moderate Alzheimer's Disease (AD) (clinicaltrials.gov) - P2 | N=180 | Active, not recruiting | Sponsor: GemVax & Kael | Recruiting ➔ Active, not recruiting | Trial completion date: Sep 2024 ➔ Mar 2026 | Trial primary completion date: Jul 2023 ➔ Sep 2025

Enrollment closed • Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders

Samsung Pharm OKed for simplified phase 3 Alzheimer's study of GV1001 (Korea Biomedical Review) - "Samsung Pharm has received approval from the Ministry of Food and Drug Safety (MFDS) to change the investigative new drug (IND) application for its phase 3 clinical trial of GV1001 for Alzheimer's disease....The MFDS accepted Samsung Pharm's application to simplify GV1001's dosing regimen, approving a change in the protocol from two 0.56 mg and 1.12 mg arms to a single 1.12 mg arm. With the unification of the dose groups, the number of patients will fall from 936 to 750. With this approval, Samsung Pharm will begin a phase 3 clinical trial to evaluate the safety and efficacy of GV1001. The trial will enroll 750 patients with moderate to severe Alzheimer's disease to determine superiority over placebo using the Severe Impairment Battery (SIB) and Clinician's Interview-Based Impression of Change-plus (CIBIC-plus) after 24 weeks (six months) of treatment with 1.12 mg of GV1001, according to the company."

New P3 trial • Alzheimer's Disease • CNS Disorders