Name: GV1001
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;

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., 2014Park et al., 2016). The authors attribute these actions to GV1001’s ability to mimic TERT’s antioxidant, anti-apoptotic, and pro-survival functions.

Beneficial effects have been reported in rodent models of hearing loss and macular degeneration (Kim et al., 2018Kim et al., 2018Lee et al., 2020).


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

Last Updated: 27 Jan 2020


No Available Comments

Make a Comment

To make a comment you must login or register.


News Citations

  1. At CTAD, Early Failures and Hints of Success, from Small Trials

Paper Citations

  1. . Gemcitabine and capecitabine with or without telomerase peptide vaccine GV1001 in patients with locally advanced or metastatic pancreatic cancer (TeloVac): an open-label, randomised, phase 3 trial. Lancet Oncol. 2014 Jul;15(8):829-40. Epub 2014 Jun 19 PubMed.
  2. . Anti-cancer effect of GV1001 for prostate cancer; function as a ligand of GnRHR. Endocr Relat Cancer. 2018 Oct 1; PubMed.
  3. . Inhibition of HIV-1 reactivation by a telomerase-derived peptide in a HSP90-dependent manner. Sci Rep. 2016 Jul 1;6:28896. PubMed.
  4. . The novel vaccine peptide GV1001 effectively blocks β-amyloid toxicity by mimicking the extra-telomeric functions of human telomerase reverse transcriptase. Neurobiol Aging. 2014 Jun;35(6):1255-74. Epub 2013 Dec 26 PubMed.
  5. . Neural stem cells injured by oxidative stress can be rejuvenated by GV1001, a novel peptide, through scavenging free radicals and enhancing survival signals. Neurotoxicology. 2016 Jul;55:131-141. Epub 2016 Jun 2 PubMed.
  6. . The Novel Peptide Vaccine GV1001 Protects Hearing in a Kanamycin-induced Ototoxicity Mouse Model. Otol Neurotol. 2018 Sep;39(8):e731-e737. PubMed.
  7. . Novel Peptide Vaccine GV1001 Rescues Hearing in Kanamycin/Furosemide-Treated Mice. Front Cell Neurosci. 2018;12:3. Epub 2018 Jan 19 PubMed.
  8. . A telomerase-derived peptide vaccine inhibits laser-induced choroidal neovascularization in a rat model. Transl Res. 2020 Feb;216:30-42. Epub 2019 Oct 5 PubMed.

External Citations


Further Reading


  1. . Telomerase Biology Associations offer Keys to Cancer and Aging Therapeutics. Curr Aging Sci. 2019 Jun 20; PubMed.
  2. . Telomerase-based cancer therapeutics: a review on their clinical trials. Curr Top Med Chem. 2020 Jan 1; PubMed.