Synonyms: RIAVAXTM, Tertomotide
Therapy Type: Immunotherapy (active) (timeline)
Target Type: Other (timeline)
Condition(s): Alzheimer's Disease
U.S. FDA Status: Alzheimer's Disease (Phase 2)
Company: GemVax & KAEL Bio
Approved for: Pancreatic cancer (Korea)
Originally developed as a cancer vaccine, GV1001 is a 16-amino-acid peptide comprising a sequence from the human enzyme telomerase reverse transcriptase (TERT). Most cancers highly express TERT, and immunization with GV1001 aims to activate the immune system to recognize and kill cancer cells. GV1001 is approved in Korea for immunotherapy of pancreatic cancer, but failed to show efficacy in two international pancreatic cancer trials (Middleton et al., 2014; Clinicaltrials.gov).
The peptide reportedly penetrates cells, interacts with HSP70 and HSP90, and displays direct anti-cancer and anti-viral activity independent of an immune response (Kim et al., 2018; Kim et al., 2016).
In non-cancer cells, GV1001 has anti-inflammatory and antioxidant activity. In rat neuronal stem cells, GV1001 blocked Aβ oligomer-induced toxicity, and increased survival of cells exposed to oxidative stress (Park et al., 2014; Park et al., 2016). The authors attribute these actions to GV1001’s ability to mimic TERT’s antioxidant, anti-apoptotic, and pro-survival functions.
In 2017, GemVax and Kael began a Phase 2 trial to assess safety and efficacy of GV1001 in people with mild to moderate Alzheimer’s disease at 11 sites in Korea. The study enrolled 90 clinically diagnosed patients, who were randomized equally to receive four weekly subcutaneous injections of 0.56 mg or 1.12 mg of GV1001, or placebo, followed by biweekly injections for 24 weeks, for a total of 14 injections. The primary outcome at 24 weeks was change on the Severe Impairment Battery; secondary outcomes included the Korean-MMSE and standard measures of cognition and function, i.e. the CDR-SB, Neuropsychiatric Inventory, Global Deterioration Scale, ADCS-Activities of Daily Living and Clinician Interview-Based Impression of Change.
The company presented top-line trial results at CTAD 2019 (Dec 2019 conference news). The study met its primary endpoint, with vaccinated participants holding steady on the SIB (0.12 percent drop) whereas those on placebo dropped by 7.23 points. A dose response was not shown; results for secondary endpoints were not shown. Adverse events were similar across all three groups.
A second Phase 2 trial is registered, but had not started recruiting as of January 2020. To be conducted in the U.S., it has the same number of participants, inclusion criteria, and dosing plan as the Korean trial. The primary outcome will be the number of patients who improve on the ADAS-Cog; secondary outcomes are 24-week changes in similar measures of cognition and function as the prior study.
For details on GV1001 trials, see Clinicaltrials.gov.
Last Updated: 27 Jan 2020
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