Gemvax & KAEL is showing strong performance. The news that its progressive supranuclear palsy (PSP) treatment, GV1001, has been designated as an orphan drug by the U.S. Food and Drug Administration (FDA) appears to be influencing its stock price.
As of 10:42 a.m. on May 2, the stock was trading at 38,350 won, up 16.21%.
Gemvax explained that the orphan drug designation signifies that GV1001's potential as a PSP treatment has been recognized externally. The company expects that, by utilizing various FDA support programs, it can enhance development efficiency and accelerate the development of the world's first PSP treatment.
The FDA's orphan drug designation program aims to encourage the development of treatments for rare diseases affecting fewer than 200,000 patients in the United States. Once designated as an orphan drug, the benefits include: up to a 25% tax credit on clinical trial costs, exemption from new drug application fees, and seven years of market exclusivity after approval.
Gemvax, which is planning a global phase 3 clinical trial for GV1001 as a PSP treatment, has now received orphan drug designation, allowing it to benefit from various incentives from the clinical stage onward. Last year, the company also received "orphan drug in development" designation from the Ministry of Food and Drug Safety in Korea.
GV1001 is a multi-mechanism drug in which various effects of telomerase, such as anti-aging, antioxidant, and anti-inflammatory properties, are simultaneously exhibited. In particular, by improving the brain's immune environment and alleviating inflammatory responses, the drug is expected to offer treatment potential for neurodegenerative diseases such as Alzheimer's disease and PSP.
Last year, Gemvax completed the first phase 2 clinical trial for PSP in Korea. The results showed good tolerability and a tendency to slow disease progression. In earlier preclinical studies, GV1001 demonstrated statistically significant efficacy in restoring motor and spatial cognitive abilities and in inhibiting tau protein damage.
Currently, in addition to completing a six-month phase 2 clinical trial in Korea, the company is smoothly conducting a 12-month extension trial. Through this, Gemvax plans to verify the long-term efficacy and safety data of 18 months of GV1001 administration in PSP patients.
PSP is an atypical parkinsonian syndrome caused by the degeneration of nerve cells related to the nuclei that control eye movements. Major symptoms include loss of balance, cognitive impairment, and muscle rigidity. Since there is currently no fundamental treatment, the rapid development of a therapy is urgently needed.
A Gemvax official stated, "With GV1001 being designated as an orphan drug by the FDA following its designation in Korea, its potential as a PSP treatment has been recognized both domestically and internationally. As many countries are actively supporting orphan drug development, we will do our utmost to succeed in developing the world's first PSP treatment through more efficient global clinical trials."
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