GemVax Applies for IND for GV1001 Phase 2 Clinical Trial in Progressive Supranuclear Palsy Treatment

[Asia Economy Reporter Jang Hyowon] GemVax & Kael announced on the 29th that it has applied to the Ministry of Food and Drug Safety for approval of the domestic Phase 2a clinical trial plan (IND) for GV1001, a treatment for progressive supranuclear palsy (PSP).

The clinical trial applied for this time is a multicenter, randomized, double-blind, placebo-controlled, parallel-design, prospective Phase 2a clinical trial to evaluate the efficacy and safety of subcutaneous administration of GV1001 at 0.56mg or 1.12mg in patients with progressive supranuclear palsy.

The trial will be conducted over six months with 75 patients at Seoul Boramae Hospital and four other domestic hospitals. The primary efficacy endpoint is the change in the total score of the progressive supranuclear palsy rating scale after six months of GV1001 administration compared to baseline.

Progressive supranuclear palsy is an atypical Parkinsonian syndrome and is known as the most malignant neurodegenerative disease among Parkinsonian disorders. The cause of progressive supranuclear palsy is not clearly identified, and there is currently no approved treatment.

GemVax has conducted research for over three years leading up to the application for the Phase 2 clinical trial of progressive supranuclear palsy and confirmed significant symptom improvement in patients through the compassionate use approval (emergency clinical) system. GV1001 previously secured effective data in a successful Phase 2 clinical trial for Alzheimer's disease and demonstrated efficacy in preclinical trials conducted by leading domestic research institutions.

In preclinical trials, GV1001 showed equivalent and statistically significant efficacy compared to control substances in motor function, spatial cognition, and novel object recognition tests, and brain tissue analysis confirmed its inhibitory effect on tau phosphorylation in key brain lesion areas of progressive supranuclear palsy.

The estimated number of progressive supranuclear palsy patients is about 20,000 in the United States alone and approximately 260,000 worldwide. Since there is no approved treatment to date, if the treatment effect is proven, GV1001 could become the first treatment, creating a high-value therapeutic market. Additionally, as progressive supranuclear palsy is the most malignant form of Parkinson's disease, success in this Phase 2 clinical trial could increase the possibility of conquering Parkinson's disease.

A GemVax official stated, “Progressive supranuclear palsy progresses about three times faster and causes more severe disability than relatively common neurodegenerative diseases such as Alzheimer's disease or Parkinson's disease, and there is no treatment, so the development of a treatment is urgently needed. Based on experimental evidence from preclinical trials and the results of the preceding Phase 2 clinical trial for Alzheimer's disease in Korea, we expect to confirm meaningful effects in this progressive supranuclear palsy clinical trial.”

He added, “GV1001, which has proven efficacy in a Phase 2 clinical trial for Alzheimer's disease, one of the representative neurodegenerative diseases, could demonstrate efficacy and safety in the Parkinsonism spectrum including Parkinson's disease through this clinical trial, confirming its potential across the treatment spectrum of neurodegenerative diseases.”