[Edaily Reporter KIM JI-WAN ] GV1001, a new drug candidate from GemVax & KAEL Co.,Ltd.(082270)that is raising expectations for conditional approval in the field of progressive supranuclear palsy (PSP)—for which there are currently no approved treatments—has also demonstrated significant efficacy in preclinical trials for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease).

Having demonstrated signs of slowing disease progression in a Phase 2a clinical trial for PSP and improvements in motor function and extended survival in ALS animal models, the compound is drawing attention for its potential as a platform drug capable of targeting various neurodegenerative diseases with a single compound.

“Targeting Common Pathogenic Mechanisms Simultaneously”… Differentiation Through a Multi-Mechanism New Drug

During the Research and Development (R&D) session at the “Chairman Nam Kyung-pil Inauguration and Vision Proclamation Ceremony” held at the Korea Exchange on the 17th, details regarding GV1001’s mechanism of action, PSP clinical results, and ALS preclinical findings were successively unveiled. The company is accelerating its strategy to expand indications by pursuing conditional approval for PSP while simultaneously preparing for global confirmatory clinical trials and follow-up development for ALS.

Lee Seok-jun, CEO of GemVax & KAEL Co.,Ltd., stated, “We are developing GV1001 not as a treatment for a single indication, but as a platform drug applicable to various neurodegenerative diseases,” adding, “Based on a multi-mechanism approach that targets common pathogenic factors, we plan to continuously expand indications starting with PSP, followed by ALS and Alzheimer’s disease.”

GV1001 refers to a 16-amino acid peptide derived from a key protein of telomerase, which was the subject of the 2009 Nobel Prize in Physiology or Medicine. GV1001 is characterized by a multi-mechanism of action that simultaneously modulates common pathogenic factors of neurodegenerative diseases, such as protein aggregation, mitochondrial dysfunction, and neuroinflammation.

GemVax & KAEL Co.,Ltd. is currently developing the drug with PSP as its top priority indication. It has completed global clinical trials for Alzheimer’s disease and is preparing to enter clinical trials for ALS. In particular, PSP is considered a representative rare neurodegenerative disease, with approximately 400,000 patients worldwide but no approved treatments.

The company has secured Fast Track designation from the U.S. Food and Drug Administration (FDA) and Orphan Drug Designation (ODD) in Korea, the U.S., and Europe. After signing a domestic licensing agreement with its affiliate, SAMSUNG PHARMACEUTICAL(001360), the company is proceeding with the conditional approval process this year.

GemVax & KAEL Co.,Ltd. is also steadily building its research capabilities. There are 69 in-house research papers related to GV1001, and 213 papers citing the research by external researchers. The company holds a total of 479 registered and pending intellectual property (IP) rights worldwide, of which 355 are patents. Although the original substance patent has expired, the company’s strategy is to establish market entry barriers based on use patents for indications such as PSP and Orphan Drug Designation exclusivity.

PSP Phase 2a Clinical Trial: “Slows Disease Progression by Half”

Professor Lee Jae-hong of Asan Medical Center in Seoul stated, explained, “Progressive supranuclear palsy (PSP) is a representative degenerative brain disease caused by the combined effects of abnormal 4R tau protein accumulation and chronic neuroinflammation.” He added, “The average survival period after onset is only 6 to 7 years, and symptoms such as eye movement disorders, repeated falls, gait disturbances, and swallowing difficulties rapidly worsen; however, to date, there are no approved treatments that fundamentally slow the progression of the disease.”

The 4R tau protein normally supports nerve cells, but when it accumulates abnormally, it damages nerve cells and causes degenerative brain diseases such as PSP.

He assessed that “GV1001 is demonstrating the potential to alter the course of the disease itself through a mechanism that simultaneously modulates neuroinflammation and tau pathology.”

In animal studies, GV1001 improved motor function as measured by the rotarod test, and positron emission tomography (PET) scans showed a reduction in signals reflecting brain inflammation. Professor Lee noted that since GV1001 has also been shown to inhibit astrocyte activation and reduce neuroinflammation, it has the potential to slow disease progression beyond merely alleviating symptoms.

Professor Moon Hyung-sik of the University of Southern California (USC) presented the results of a Phase 2a exploratory clinical trial for PSP conducted in South Korea, stating, “Although it was a small-scale study, we identified meaningful signals indicating that GV1001 can slow the rate of disease progression.” A total of 78 participants took part in this clinical trial—25 in the low-dose group, 28 in the high-dose group, and 25 in the placebo group—and the study lasted 24 weeks.

Analysis of the primary endpoint, the Progressive Spastic Paralysis Rating Scale (PSPRS), showed that the low-dose group had a score increase approximately 2 points lower than the placebo group. Although statistical significance was not achieved, this result can be interpreted as a roughly 50% delay in the rate of disease progression. Notably, when patients with rapid-progressing RS were analyzed separately, the difference from the placebo group widened further over time.

Professor Moon emphasized, “These results suggest that GV1001 has the potential to evolve into a ‘disease-modifying therapy’ that alters the natural course of the disease itself, rather than simply being a medication that alleviates symptoms.”

Improvements in Cognitive Function Also Confirmed

Positive results were also observed in

cognitive function

. The MoCA-K, a secondary endpoint, is a representative cognitive function test that comprehensively evaluates memory, attention, language skills, visuospatial abilities, and executive function. In this assessment, the low-dose group recorded improved scores compared to the placebo group.

Professor Moon explained, “These results demonstrate that GV1001 can be expected to slow not only the decline in motor function but also that in cognitive function.”

Safety was also favorable. The incidence of adverse events related to the test drug showed no significant difference from the placebo group, and no new safety concerns were identified in the one-year extension study.

The results of the extension study were also encouraging. Compared to an external control group, the PSPRS score in the GV1001 treatment group deteriorated by only 3.3 points over 72 weeks. In contrast, previous external studies reported a deterioration of 10.7 points in approximately 52 weeks.

Professor Moon stated, “Through this exploratory clinical trial, we confirmed not only safety but also the appropriate dosage and signs of efficacy,” adding, “We have secured sufficient evidence to proceed to a confirmatory clinical trial.”

Preclinical ALS Study

Shows

Over 20% Increase in Survival Rate… “Preparing to Enter Clinical Trials”

The potential of GV1001 was also confirmed

in

preclinical studies on amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease).

Professor Ryu Hoon of the Korea Institute of Science and Technology (KIST) explained, “ALS is a fatal neurodegenerative disease in which motor neurons gradually degenerate, leading to muscle paralysis and eventually the loss of respiratory function,” adding, “Given that current treatment options are very limited, there is an urgent need to develop new therapies that can slow disease progression or extend survival.”

The research team evaluated the efficacy of GV1001 using a SOD1 mutant mouse model.

Professor Ryu explained, “Subcutaneous injection of GV1001 resulted in significant improvements in gait analysis and motor function, and therapeutic effects were confirmed even at relatively low doses.”

The most notable finding was the effect on survival. According to the presentation, the low-dose GV1001 group showed a survival rate increase of more than 20% compared to the control group, and the average survival period was extended by approximately 20 days.

Professor Ryu commented, “The 20-day extension in survival observed in the mouse model is a highly significant result considering the animals’ lifespan,” adding, “While this cannot be directly extrapolated, the data suggests the potential for survival extension on the order of several years when applied to humans.”

Meanwhile, GemVax & KAEL Co.,Ltd. is currently accelerating preparations for global confirmatory clinical trials for GV1001, alongside the PSP conditional approval process. The company is continuing close consultations with regulatory authorities following the U.S. FDA’s Fast Track designation. GemVax plans to sequentially disclose its future approval strategy and follow-up clinical trial plans.