Conditional Approval Application Upon Confirmation of Clinical Efficacy
GemVax & Kael Kael announced on the 28th that it has completed patient recruitment for the domestic Phase 2 clinical trial of GV1001, a treatment for progressive supranuclear palsy (PSP).
The Phase 2 clinical trial involves 78 PSP patients divided into a placebo group, Test Group 1 (GV1001 0.56mg), and Test Group 2 (GV1001 1.12mg), evaluating the efficacy in improving disease severity and safety over six months. The trial is being conducted under the supervision of Professor Lee Ji-young from the Department of Neurology at Seoul National University College of Medicine, at Seoul National University Hospital, Seoul Metropolitan Government Boramae Medical Center, Samsung Seoul Hospital, Bundang Seoul National University Hospital, and Kyung Hee University Hospital.
This clinical trial is the first PSP clinical trial in Korea. With the enrollment of the last patient, it is expected that all patients will complete dosing by the end of September.
GemVax plans to seek conditional approval for commercialization if the Phase 2 clinical trial results demonstrate efficacy and safety. GV1001 for PSP was designated as a "Rare Drug in Development" by the Ministry of Food and Drug Safety last month. Designation as a rare drug accelerates the review process for conditional approval, and if approved, grants exclusive marketing rights for four years from the date of approval.
PSP belongs to atypical parkinsonism syndromes. "Supranuclear palsy" refers to dysfunction in eye movement due to abnormalities in the nerve nuclei, with PSP characterized by progressively impaired eye movement. Symptoms such as gait disturbance, rigidity, tremor, and cognitive decline accompany PSP, similar to Parkinson's disease, but PSP progresses faster and currently has no fundamental treatment.
The cause of onset is unknown, but PSP shares pathophysiological similarities with Alzheimer's disease, including neuroinflammation and neuronal death caused by tau protein damage.
In preclinical trials using tauopathy transgenic mice (TauP301L0BiFC mice), GemVax confirmed that GV1001 inhibits tau protein hyperphosphorylation and aggregation, improving motor and cognitive functions. The preclinical results attracted attention at international conferences. In August, the GV1001 PSP preclinical paper was selected as a cutting-edge innovation abstract at the International Parkinson and Movement Disorder Society (MDS) conference held in Denmark. In October, at Neuro2023, world-renowned PSP expert Dr. G?nter U. H?glinger identified GV1001 as a new multi-mechanism PSP drug candidate.
GemVax is also conducting a 12-month extension clinical trial for patients who completed the initial trial and wish to participate. Unlike the original trial, the extension is a single-arm study where all participants receive GV1001 1.12mg for 12 months. Through this, GemVax will obtain additional data on the long-term effects of GV1001 administration.
Global PSP clinical trials are also underway. In February, the U.S. Food and Drug Administration (FDA) approved the Phase 2 clinical trial plan for PSP. GemVax has begun preparatory work for the U.S. trial and is preparing to apply for Phase 2 clinical trials in Europe and the United Kingdom.
A GemVax representative explained, "PSP is more difficult to manage symptomatically than Parkinson's disease, and there is currently no drug worldwide that can slow or treat disease progression, resulting in a high unmet medical need." They added, "With the Phase 2 trial nearing completion, the commercialization of the world's first treatment is also approaching."
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