Gemvax & Kael, a Kosdaq-listed peptide drug specialist, said on Tuesday that it has submitted a phase 2 investigational new drug (IND) application to the U.S. FDA for GV1001, targeting the treatment of progressive supranuclear palsy (PSP).

PSP is an atypical Parkinson's syndrome characterized by rapid disease progression and is one of the most severe forms of Parkinson's.

The trial will evaluate the efficacy and safety of subcutaneous administration of 0.56 mg or 1.12 mg of GV1001 for 12 months in 150 patients with PSP and will be conducted at 30 to 40 sites in the U.S.

The primary efficacy endpoint is the change in total score on the PSP Rating Scale (mPSPRS) after 12 months of GV1001 treatment from baseline.

According to market research firm Mordor Intelligence, the global market for neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, and PSP is expected to grow from $51.45 billion in 2023 to $72.63 billion by 2028, at a CAGR of 7.14 percent during the forecast period.

Gemvax has been conducting a phase 2 clinical trial of GV1001 in 75 patients at five Korean institutions, including Seoul National University Hospital, after receiving IND approval from the Ministry of Food and Drug Safety in March.

In October, Günter U. Höglinger, a renowned researcher in the field of PSP at Ludwig-Maximilians University in Germany, introduced GV1001 as a new drug candidate for PSP with a novel mechanism at Neuroscience 2023.

Gemvax recently completed an orphan drug application for GV1001 with the Korea Orphan & Essential Drug Center, and if the phase 2 study in Korea is successful, commercialization will be possible with a conditional new drug application for GV1001.

"While the U.S. and European phase 2 clinical trials of Alzheimer's disease are progressing smoothly, PSP, a rare disease, is also challenging global clinical trials, increasing the possibility of global commercialization of GV1001, a treatment for neurodegenerative diseases," a Gemvax official said.

In addition to the U.S. FDA, Gemvax is preparing to file for a phase 2 clinical trial in PSP with the European Medicines Agency (EMA).