GemVax Seeks Expanded Access for Mild Alzheimer’s for Planned GV1001 US Trial

GemVax & KAEL has filed for expanded access of its U.S. Food and Drug Administration (FDA) investigational new drug (IND) application for GV1001, its experimental treatment for Alzheimer’s disease.

The company is seeking to include additional investigational groups in its clinical development program.

The filing comes following positive results of a Phase 2 clinical trial (NCT03184467) of the therapeutic candidate, conducted in Korea.

“It gives us the confidence to expand the range of indications targeted with GV1001 to include mild (not severe) Alzheimer’s disease and cognitive impairment and potentially develop a new treatment for dementia,” Kim Sang-Jae, chairman of GemVax, said in a press release.

The expanded access would extend to a second Phase 2b trial (NCT03959553) in the U.S. Expected to launch in 2021, that planned trial would involve patients with mild cognitive impairment and mild symptoms caused by Alzheimer’s.

GV1001 was originally developed as a vaccine for cancer, but failed to show efficacy in the treatment of pancreatic cancer in two clinical trials.

The active ingredient of GV1001 is a peptide comprised of 16 amino acids, the building blocks of proteins, that is identical to an amino acid sequence from the enzyme telomerase reverse transcriptase (TERT).

Many cancers express higher levels of TERT, so the GV1001 treatment was designed to prime the immune system to recognize the TERT sequence, to better recognize and eliminate cancerous cells that express TERT.

Though GV1001 failed as a treatment for cancer, the researchers noted that non-cancerous cells showed lower levels of inflammation and oxidation in the presence of GV1001.

Given this effect, the researchers pivoted to investigating whether GV1001 may act as a treatment for patients with other conditions, including Alzheimer’s disease, in which inflammation is known to play a role.

In the Korean Phase 2 clinical trial, 90 patients with Alzheimer’s were given either 0.56 mg of GV1001, 1.12 mg of GV1001, or a placebo for comparison. The treatment is administered subcutaneously (under the skin) every four weeks for the first four doses, followed by every two weeks until week 24 of the study, for a total of 14 doses.

Throughout the roughly six-month period, the researchers monitored patient progress using a number of established tests. The primary goal of the study was a comparison of Severe Impairment Battery (SIB) scores from zero to 24 weeks.

The SIB is a neuropsychological test to determine the severity of symptoms, typically given to those who are already in an impaired state.

The scores were stable for patients receiving GV1001, with only a 0.1% decrease by 24 weeks, the researchers found. However, among the placebo group, the scores decreased by 7.23 points by the same time point.

Further, two secondary measures, the Neuropsychiatric Inventory (NPI) test and the Alzheimer’s Disease Cooperative Study-Activities of Daily Living (ADCS-ADL) scale, each showed significant improvements in the GV1001 groups compared with the placebo.

“The CSR [clinical study report] analysis, showing both improvement in SIB scores in patients with Alzheimer’s disease and significant results in NPI and ADCS-ADL scores is highly promising,” Sang-Jae said.

GemVax had previously registered the planned Phase 2b clinical trial in the U.S., investigating GV1001 for patients with moderate-to-severe Alzheimer’s, but the initiation of the trial was delayed due to the COVID-19 pandemic.