[Edaily Reporter KIM JI-WAN ] SAMSUNG PHARMACEUTICAL(001360)announced on the 30th that it has submitted an application for a Phase 3 clinical trial in Korea for “GV1001,” a treatment for progressive supranuclear palsy (PSP), to the Ministry of Food and Drug Safety.

This clinical trial will be conducted as a multicenter, randomized, double-blind, placebo-controlled, parallel-group, prospective Phase 3 clinical trial.

The study plans to evaluate the efficacy in improving disease severity and the safety of GV1001 by administering 0.56 mg of the drug subcutaneously to patients with the Richardson syndrome (PSP-RS) subtype of progressive supranuclear palsy.

This trial will be conducted at 7 to 9 clinical trial sites in South Korea (tentative), including Boramae Medical Center in Seoul, with a total of 204 patients.

The primary efficacy endpoint is the change in the total score on the “Progressive Supranuclear Palsy Rating Scale (PSPRS)” after 48 weeks of GV1001 administration compared to baseline.

Prior to this, SAMSUNG PHARMACEUTICAL submitted an application for conditional marketing authorization for GV1001, a PSP treatment, to the Ministry of Food and Drug Safety (MFDS) last January, and the application is currently under review. Conditional marketing authorization is a system that grants priority market approval for treatments of life-threatening severe or rare diseases based on Phase 2 clinical trial results, subject to the submission of Phase 3 clinical trial results after launch.

The company plans to objectively demonstrate the drug’s efficacy and safety through this Phase 3 clinical trial, regardless of whether conditional marketing authorization is granted. Furthermore, the initiation of this clinical trial is a proactive measure to prevent any delays in submitting results due to a potential delay in trial commencement following approval, should conditional marketing authorization be granted.

Progressive supranuclear palsy (PSP) shares some symptoms with Parkinson’s disease but progresses more rapidly and does not respond well to existing Parkinson’s treatments, making the development of a fundamental cure an urgent priority. Although various studies have been conducted worldwide to develop treatments, no commercially available therapy currently exists.

GV1001 demonstrated excellent safety in a 24-week Phase 2 clinical trial conducted in Korea. In particular, a positive trend toward slowing the progression of the disease was observed in the group of patients with PSP-RS who received low doses. Additionally, it was designated as an orphan drug in the development stage by the Ministry of Food and Drug Safety in 2024.

A SAMSUNG PHARMACEUTICAL spokesperson stated, “PSP is a rare, intractable disease for which there are currently no suitable treatment options, causing immense suffering for patients and their families,” adding, “We will do our utmost to definitively prove the drug’s efficacy and safety through this Phase 3 clinical trial and to bring the world’s first PSP treatment to market, thereby offering new hope to patients.”