Drug information

Audio
Pronounce:
Other Names:
VRC-HIVMAB060-00-AB, VRC-HIVMAB080-00-AB, VRC01LS (long-acting form of VRC01)
Drug Class:
Broadly Neutralizing Antibodies
Registry Number:
1412901-55-3 (CAS)
Organization:
NIAID Vaccine Research Center (VRC)
Phase of Development:

VRC01 is in Phase 2 development as a broadly neutralizing antibody for HIV treatment. VRC01 is also being studied for HIV prevention.

(Compound details obtained from ChemIDplus Advanced,1 Science Translational Medicine article,2 Treatment Action Group website,3 and Treatment Action Group Pipeline Report 20194)

 
What is VRC01?What is VRC01?

What is VRC01?

 

VRC01 is an investigational drug that is being studied as a possible strategy to treat and prevent HIV infection. VRC01 belongs to a group of HIV drugs called broadly neutralizing antibodies (bNAbs).3,4

A long-acting form of VRC01, called VRC01LS, is also under study for HIV treatment and prevention.3,4 

To learn how investigational drugs are tested during clinical trials, read the HIVinfo What is an Investigational HIV Drug? and HIV and AIDS Clinical Trials fact sheets.

How do broadly neutralizing antibodies work?How do broadly neutralizing antibodies work?

How do broadly neutralizing antibodies work?

 

Antibodies are proteins that the immune system makes to fight infection. A person with HIV produces specific antibodies against HIV. However, most of these antibodies do not stop HIV from multiplying in the body.5,6

Some people with HIV naturally produce rare types of HIV antibodies called broadly neutralizing antibodies (bNAbs). bNAbs are powerful antibodies that can work against different HIV strains. bNAbs can block HIV from entering healthy cells and activate other immune cells to help destroy infected cells.5,7,8

Researchers are investigating whether giving bNAbs to people with HIV can help them maintain undetectable levels of HIV without the need for daily antiretroviral therapy. Additionally, some bNAbs are being studied because they may be able to reduce the size of the latent HIV reservoir.7,9

Researchers are also trying to find out if giving bNAbs to people who do not have HIV can help protect them from getting the virus.4 This record discusses the study of VRC01 for both HIV treatment and prevention.

Select clinical trials of VRC01Select clinical trials of VRC01

Select clinical trials of VRC01

 

VRC01 for HIV treatment

Study Names: Tatelo Study; NCT03707977
Phase: 1/2
Status: This study is ongoing, but not recruiting participants.
Location: Botswana
Purpose: The purpose of this study is to evaluate the efficacy of VRC01LS and the investigational bNAb 10-1074 in maintaining viral suppression in children who had received early ART treatment.10
Selected Study Results: Results from early parts of the trial presented at CROI 2020 and CROI 2021 showed that treatment with both 10-1074 and VRC01LS (given either individually or in combination) was generally safe. There were no infusion reactions or severe side effects related to bNAb treatment.11,12

Study Names: RV 397; NCT02664415
Phase: 2
Status: This study has been completed.
Location: Thailand
Purpose: The purpose of this study was to evaluate the safety of VRC01 and efficacy of VRC01 in preventing viral rebound in participants undergoing a structured treatment interruption of ART.13
Selected Study Results: Results published in the Lancet HIV journal showed that VRC01, given to individuals who had started ART during acute HIV infection, did not have a significant impact on the number of participants with viral suppression at 24 weeks after treatment interruption of ART. No serious side effects were reported during the study.14

Study Names: (1) HVTN 804/HPTN 095; NCT04801758 and (2) HVTN 805/HPTN 093; NCT04860323
Phase: Not available
Status: These studies are currently recruiting participants.
Location: (1) HVTN 804/HPTN 095 – United States and South America (2) HVTN 805/HPTN 093 – Sub-Saharan Africa
Purpose: The purpose of these studies is to evaluate the ability of the immune system to control viral load levels during a structured treatment interruption of ART in participants who received VRC01 or placebo and acquired HIV while enrolled in the  HVTN 704/HPTN 085 or the HVTN 805/HPTN 093 prevention trials.15,16


For more details on the studies listed above, see the Health Professional version of this drug summary.

Additional studies investigating HIV treatment with VRC01 or VRC01LS have been or are being conducted. Some of these studies include:
  • RV 398 (NCT02591420): A Phase 1 study evaluating the safety and antiviral effects of VRC01 when given alone and when given with ART in adults with early acute HIV infection. This study is currently recruiting participants.17
  • ACTG A5378 (NCT02840474): A Phase 1 trial that evaluated the safety and antiviral effects of VRC01LS and another long-acting bNAb called VRC07 523LS in adults with HIV who had never received ART. This study has been completed.18
  • NCT03831945: A Phase 1 trial that evaluated whether the combination of VRC01 plus 10-1074 could control viral load levels in virologically suppressed adults undergoing structured treatment interruptions of ART. This study has been completed.19
  • IMPAACT 2008 (NCT03208231) and IMPAACT P1115 (NCT02140255): Two Phase 1/2 trials evaluating the use of VRC01 with ART in infants with HIV. NCT03208231 has been completed. NCT02140255 is currently recruiting participants.20,21


VRC01 for HIV prevention

Study Identifiers: (1) HVTN 704/HPTN 085; NCT02716675 and (2) HVTN 703/HPTN 081; NCT02568215
Phase: 2b
Status: These studies have been completed.
Location: (1) HVTN 704/HPTN 085 – United States, South America, and Switzerland (2) HVTN 703/HPTN 081 – Sub-Saharan Africa 
Purpose: The purpose of these studies was to evaluate the safety and efficacy of VRC01 administered every 8 weeks in preventing HIV-1 infection among men and transgender persons who have sex with men and heterosexual women who were at risk of acquiring HIV.22–24
Selected Study Results: Results published in the New England Journal of Medicine found that overall, VRC01 did not significantly protect participants from acquiring HIV in either trial. This overall lack of efficacy was due to a low percentage of VRC01-sensitive virus circulating in the regions where the trials were conducted. When looking at the effectiveness of VRC01 infusions in preventing the acquisition of VRC01-sensitive HIV strains in participants from both trials, VRC01 was 75% effective. The number and severity of side effects was similar across treatment groups.24

What side effects might VRC01 cause?What side effects might VRC01 cause?

What side effects might VRC01 cause?

 

One goal of HIV research is to identify new drugs that have fewer side effects. The following side effects were observed in some of the studies of VRC01 listed above.

Tatelo Study (NCT03707977):

Results from early parts of this Phase 1/2 trial showed that no participants experienced infusion reactions or severe side effects related to single or dual bNAb administration with 10-1074 and VRC01LS.10–12

RV 397 (NCT02664415):

In this Phase 2 study, one participant dropped out of the study because of a severe side effect (hives) that occurred during their first dose of VRC01. Other side effects that occurred with intravenous infusions of either VRC01 or placebo were mild, with the exception of one case of moderate infusion-site bruising in a participant receiving VRC01.13,14

HVTN 804/HPTN 095 (NCT04801758) and HVTN 805/HPTN 093 (NCT04860323):

In these two Phase 2b prevention studies, the number and severity of side effects that occurred during the trials was similar across VRC01 and placebo treatment groups. Also, the percentage of participants experiencing reactogenic side effects to VRC01 infusions was similar across groups.24 (Reactogenic side effects are short-term side effects that occur during or shortly after receiving a vaccine. Examples of reactogenic side effects include injection-site pain or tenderness and fever.)

Because VRC01 is still being studied, information on possible side effects of the drug is not complete. As testing of VRC01 continues, additional information on possible side effects will be gathered.

Where can I get more information about clinical trials studying VRC01?Where can I get more information about clinical trials studying VRC01?

Where can I get more information about clinical trials studying VRC01?

 

More information about VRC01-related research studies is available from ClinicalTrials.gov.

Some clinical trials may be looking for volunteer participants. Your health care provider can help you decide whether participating in a clinical trial is right for you. For more information, visit NIH Clinical Research Trials and You.

ReferencesReferences

References

 

  1. United States National Library of Medicine. ChemIDplus Advanced: VRC01. https://chem.nlm.nih.gov/chemidplus/rn/1412901-55-3. Accessed October 6, 2021
  2. Lynch RM, Boritz E, Coates EE, et al. Virologic effects of broadly neutralizing antibody VRC01 administration during chronic HIV-1 infection. Sci Trans Med. 2015;7(319):319ra206. doi:10.1126/scitranslmed.aad5752
  3. Treatment Action Group website. Research toward a cure trials. https://www.treatmentactiongroup.org/cure/trials/. Accessed October 6, 2021
  4. Jefferys R. HIV vaccines & passive immunization. Treatment Action Group Pipeline Report 2021. https://www.treatmentactiongroup.org/wp-content/uploads/2021/07/pipeline_2021_HIV_vaccine_final.pdf. Accessed October 6, 2021
  5. HIV Vaccine Trials Network (HVTN). Using antibodies for HIV prevention. https://www.hvtn.org/en/science/hiv-vaccine-basics/why-hiv-vaccine1.html. Accessed October 6, 2021
  6. Snow B. The rise of broadly neutralizing antibodies.  AIDS Vaccine Advocacy Coalition (AVAC). Published May 17, 2018. https://www.avac.org/blog/rise-broadly-neutralizing-antibodies. Accessed October 6, 2021
  7. National Institute of Allergy and Infectious Diseases (NIAID). Sustained ART-free HIV remission. https://www.niaid.nih.gov/diseases-conditions/sustained-art-free-hiv-remission. Accessed October 6, 2021
  8. National Institute of Allergy and Infectious Diseases (NIAID). Future directions for HIV treatment research. https://www.niaid.nih.gov/diseases-conditions/future-hiv-treatment. Accessed October 6, 2021
  9. Grobben M, Stuart RA, van Gils MJ. The potential of engineered antibodies for HIV-1 therapy and cure. Curr Opin Virol. 2019;38:70-80. doi:10.1016/j.coviro.2019.07.007
  10. National Institute of Allergy and Infectious Diseases (NIAID). A clinical trial to evaluate the impact of broadly neutralizing antibodies VRC01LS and 10-1074 on maintenance of HIV suppression in a cohort of early-treated children in Botswana (dual bNAb treatment in children). In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). October 12, 2018. NLM Identifier: NCT03707977. https://clinicaltrials.gov/ct2/show/NCT03707977. Accessed October 6, 2021
  11. Capparelli EV, Ajibola G, Maswabi K, et al. Safety and pharmacokinetics of intravenous VRC01LS and 10-1074 in young children. Abstract presented at: Conference on Retroviruses and Opportunistic Infections (CROI); March 8-11, 2020; Virtual. Abstract 465. https://www.croiconference.org/abstract/safety-and-pharmacokinetics-of-intravenous-vrc01ls-and-10-1074-in-young-children/. Accessed October 6, 2021
  12. Capparelli EV, Ajibola G, Maswabi K, et al. Safety and pharmacokinetics of VRC01LS and 10-1074 among children in Botswana. Webcast presented at: Conference on Retroviruses and Opportunistic Infections (CROI); March 6-10, 2021; Virtual. http://www.croiwebcasts.org/console/player/47673?mediaType=slideVideo&. Accessed October 6, 2021
  13. National Institute of Allergy and Infectious Diseases (NIAID). Safety and therapeutic efficacy of the broadly neutralizing HIV-1 specific monoclonal antibody VRC01 during analytic treatment interruption in patients who initiated antiretroviral therapy during early acute HIV infection. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on January 20, 2016. NLM Identifier: NCT02664415. https://clinicaltrials.gov/ct2/show/NCT02664415. Accessed October 6, 2021
  14. Crowell TA, Colby DJ, Pinyakorn S, et al. Safety and efficacy of VRC01 broadly neutralising antibodies in adults with acutely treated HIV (RV397): a Phase 2, randomised, double-blind, placebo-controlled trial. Lancet HIV. 2019;6(5):e297-e306. doi:10.1016/S2352-3018(19)30053-0
  15. HIV Vaccine Trials Network. Antiretroviral analytical treatment interruption (ATI) to assess immunologic and virologic responses in participants who received VRC01 or placebo and became HIV-infected during HVTN 704/HPTN 085. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on March 1, 2021. NLM Identifier: NCT04801758. https://www.clinicaltrials.gov/ct2/show/NCT04801758. Accessed October 6, 2021
  16. HIV Vaccine Trials Network. Antiretroviral analytical treatment interruption (ATI) to assess immunologic and virologic responses in participants who initiated ART in early HIV infection after having received VRC01 or placebo in HVTN 703/HPTN 081. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on April 14, 2021. NLM Identifier: NCT04860323. https://clinicaltrials.gov/ct2/show/NCT04860323. Accessed October 6, 2021
  17. National Institute of Allergy and Infectious Diseases (NIAID). Safety and virologic effect of a human monoclonal antibody (VRC01) administered intravenously to adults during early acute HIV infection. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on October 28, 2015. NLM Identifier: NCT02591420. https://clinicaltrials.gov/ct2/show/NCT02591420. Accessed October 6, 2021
  18. National Institute of Allergy and Infectious Diseases (NIAID). A phase 1, single dose study of the safety and virologic effect of an HIV-1 specific broadly neutralizing human monoclonal antibody, VRC-HIVMAB080-00-AB (VRC01LS) or VRC-HIVMAB075-00-AB (VRC07-523LS), administered intravenously to HIV-infected adults. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered July 19, 2016. NLM Identifier: NCT02840474. https://clinicaltrials.gov/ct2/show/NCT02840474. Accessed October 6, 2021
  19. National Institute of Allergy and Infectious Diseases (NIAID). Combination therapy with VRC-HIVMAB060-00-AB (VRC01) and 10-1074 in HIV-infected individuals undergoing sequential treatment interruptions. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on February 5, 2019. NLM Identifier: NCT03831945. https://clinicaltrials.gov/ct2/show/NCT03831945. Accessed October 6, 2021
  20. National Institute of Allergy and Infectious Diseases (NIAID). Evaluating the safety and antiviral activity of monoclonal antibody VRC01 in HIV-infected infants receiving combination antiretroviral therapy. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered June 30, 2017. NLM Identifier: NCT03208231. https://clinicaltrials.gov/ct2/show/NCT03208231. Accessed October 6, 2021
  21. National Institute of Allergy and Infectious Diseases (NIAID). Very early intensive treatment of HIV-infected infants to achieve hiv remission: a Phase I/II proof of concept study. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on May 13, 2014. NLM Identifier: NCT02140255. https://clinicaltrials.gov/ct2/show/NCT02140255. Accessed October 6, 2021
  22. National Institute of Allergy and Infectious Diseases (NIAID). A Phase 2b study to evaluate the safety and efficacy of VRC01 broadly neutralizing monoclonal antibody in reducing acquisition of HIV-1 infection among men and transgender persons who have sex with men. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on March 17, 2016. NLM Identifier: NCT02716675. https://clinicaltrials.gov/ct2/show/NCT02716675. Accessed October 6, 2021
  23. National Institute of Allergy and Infectious Diseases (NIAID). A Phase 2b study to evaluate the safety and efficacy of VRC01 broadly neutralizing monoclonal antibody in reducing acquisition of HIV-1 infection in women in Sub-Saharan Africa. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on October 1, 2015. NLM Identifier: NCT02568215. https://clinicaltrials.gov/ct2/show/NCT02568215. Accessed October 6, 2021
  24. Corey L, Gilbert PB, Juraska M, et al. Two randomized trials of neutralizing antibodies to prevent HIV-1 acquisition. N Engl J Med. 2021;384(11):1003-1014. doi:10.1056/NEJMoa2031738
 

Last Reviewed: October 6, 2021