South Korean biopharmaceutical company GemVax & Kael announced the results from its Phase 2 clinical trial of a potential drug for progressive supranuclear palsy Richardson’s syndrome (PSP-RS, a PSP subtype). The trial demonstrated the drug’s safety and tolerability, with the company noting that its data support proceeding to a Phase 3 trial.

GV1001 is Safe and Tolerable in People with PSP-RS

In PSP, a disorder traditionally associated with movement-based symptoms, the immune cells of the central nervous system (called microglia) are thought to contribute to the progression of the disease. In addition to being involved in neuroinflammation, microglia can spread dysfunctional tau and a-betta proteins that accumulate into toxic aggregations. GemVax & Kael’s experimental drug, GV1001, is designed to target microglial dysfunction in PSP and other tauopathies like Alzheimer’s.

According to the company, GV1001 was safe and well-tolerated by participants throughout the 72-week trial period. Participants experienced no severe adverse effects, and any side effects were minor in nature. There was no mortality risk associated with GV1001.

While the efficacy of the drug was not the primary goal (or “endpoint”) of the trial, the company shared data that may indicate a slowdown in disease progression. Participants in the study were regularly assessed using the PSP-rating scale, a widely used clinical measure where increasing scores reflect worsening symptoms. People in the test group experienced a slower progression of PSP, with symptoms worsening by 5.61 points compared to external control groups, whose symptoms progressed by 10.66 points. Please see below for important details on which groups were compared.

The efficacy data are more promising compared to earlier interim results, which were less clear. Based on the data from the current study and prior trials, GemVax & Kael plans to proceed with an international Phase 3 study of GV1001.

Study Used External Control Groups from Other PSP Trials

GemVax & Kael used external data from three other trials for their Phase 2 study, a practice in some stages of the clinical research process for rare diseases like PSP. This methodology is used due to the difficulty of having to have a placebo group in the trial (people who do not receive the active treatment).

Using external control groups requires careful consideration to ensure that the data aligns with the study’s goals. Scientists at the South Korean company used special analytical methods to enhance the objectivity of their results. Regulators, however, typically require internal placebo group data for Phase 3 trials.

Thanks to the availability of external data, however, the trial was able to generate promising results that support the company’s progression to the next stage of the clinical trial process.

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