GemVax to Announce Full Clinical Results for Progressive Supranuclear Palsy Within Two Weeks

On November 10, GemVax & KAEL announced that it would release the full results of its Phase 2 clinical trial for PSP within two weeks.

Progressive Supranuclear Palsy (PSP) is a rare, intractable disease and one of the degenerative brain disorders for which there is no treatment worldwide. Due to the small number of patients and the rapid progression of the disease, conducting long-term clinical trials is difficult. Securing objective data on the efficacy and safety of the drug over an 18-month period represents a rare long-term research outcome.

GemVax registered its first patient in June 2023 and began the preliminary Phase 2 clinical trial for PSP. Including the extension trial for 67 patients (85.9%) who agreed to continue after completing the initial trial, the company conducted a total of 18 months of PSP clinical trials.

The preliminary trial demonstrated the tolerability of GV1001 at a 0.56mg dose, as well as a trend toward slowing disease progression. In an interim analysis combining six-month data from both the preliminary and extension trials, the company confirmed statistically significant effects compared to an external control group (P<0.05). GemVax explained that the safety of the drug was confirmed in interim analyses for both the preliminary and extension trials.

After the completion of the clinical trials, GemVax has continued to provide additional dosing opportunities to existing trial patients through a compassionate use program. Under a system allowing treatment at the discretion of medical professionals, four individual cases have received support. In the compassionate use program for groups of two to fewer than 25 patients, all 24 available slots have already been filled.

The PSP treatment candidate GV1001 has been designated as an orphan drug and granted Fast Track status by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Ministry of Food and Drug Safety in Korea.

A GemVax representative stated, "Given that the average survival period for patients with the rare disease PSP is five to eight years, the 18-month research results are a valuable achievement that is rarely seen in the global pharmaceutical and biotech industry." The representative added, "We hope to confirm evidence that the drug's effects are sustainable over the long term, as well as its long-term safety profile."

GV1001 is a peptide-based drug candidate that inhibits the accumulation of beta-amyloid and the aggregation of tau proteins in nerve cells, and simultaneously exhibits various effects such as anti-inflammatory and antioxidant actions, as well as mitochondrial protection.