Breakthrough Hope for Rare Brain Disorder: Gemvax Pursues Groundbreaking Treatment for Progressive Supranuclear Palsy
▲ Gemvax & Kael CI (Photo = Provided by Gembax & Kael)
[메디컬투데이=최유진 기자] Gemvax & KAEL received the topline results of the domestic phase 2a clinical trial of GV1001 for patients with progressive supranuclear palsy (PSP) and confirmed its development potential as a PSP treatment, but its effectiveness is still unpredictable.
Clinical results showed effectiveness in the GV1001 0.56mg administration group. In particular, it was confirmed to have high potential as a treatment in the progressive supranuclear palsy-Richardson syndrome (PSP-RS) type patient group.
The GV1001 phase 2a clinical trial was conducted in Korea on 78 PSP patients, administering placebo, 0.56mg of GV1001, and 1.12mg each for 6 months. In addition, efficacy and safety evaluation variables were collected to assess the potential of GV1001 as a PSP treatment and to serve as a basis for determining the possibility of proceeding to follow-up clinical trials.
The change in PSP rating scale score, calculated as the least squares mean using the MMRM estimation method, which is the primary evaluation variable, worsened by 2.14 points in the GV1001 0.56mg treatment group over 6 months, while the placebo group worsened by 4.10 points, resulting in 48% disease progression in the treatment group. Delayed results were obtained.
In addition, in the simple average analysis performed as a post-ad-hoc sensitivity analysis, the GV1001 0.56 mg treatment group worsened by 1.35 points, while the placebo group worsened by 4.36 points, delaying disease progression by 70% in the treatment group.
The typical case of PSP that is usually clinically referred to is the PSP-RS type, which accounts for the majority of all PSP patients. Compared to other PSP types, the disease progresses faster and the average survival time is shorter.
When the scope of patients in the clinical trial was narrowed down to PSP-RS type, the least square mean value worsened by 1.88 points in the GV1001 0.56 mg treatment group, while the placebo group worsened by 4.45 points, resulting in a 58% delay in disease progression in the treatment group. .
In addition, in the simple mean value analysis performed as a post-ad-hoc sensitivity analysis, the GV1001 0.56 mg treatment group worsened by 0.25 points, while the placebo group worsened by 5.19 points, delaying disease progression by 95% in the treatment group.
Like Parkinson’s disease, PSP is accompanied by symptoms such as gait disturbance, stiffness, tremors, and cognitive decline, but it is a degenerative disease that progresses faster and has no fundamental cure.
This type shows typical PSP symptoms, including early falls, early cognitive dysfunction, vertical gaze disturbance, and postural instability. Tau accumulation is more severe than other types of PSP, and large areas such as the cerebellum, dentate nucleus, pontine nucleus, frontal lobe, and parietal lobe are involved.
A Gemvax official said, “The number of participants was very small, 78 people, and it was difficult to confirm effectiveness as the clinical trial was conducted for a short 6 months. However, since it is a rare disease, recruitment was difficult, and securing objectivity was difficult compared to other diseases. “It is true,” he said.
He added, “We have confirmed the possibility of developing a treatment for acute paralysis through clinical trials, and since the data was good, we are planning to enter global Chapter 3.”
Medical Today Reporter Choi Yu-jin (gjf256@mdtoday.co.kr)
[저작권자ⓒ 메디컬투데이. 무단전재-재배포 금지]