skip navigation

Skip Nav

AIDSinfo Drug Database

AIDSinfo Drugs

Drugs by class



Rintatolimod  Audio icon

Other Names: AMP, Ampligen, Atvogen, poly(I:C12U), polyI:polyC12U, poly(I)-poly(C12U)
Drug Class: Biological Response Modifiers
Molecular Formula: (C10 H13 N4 O8 P)x.(C9 H14 N3 O8 P.C9 H13 N2 O9 P)x
Registry Number: 38640-92-5 (CAS)
Chemical Class: Oligonucleotides
Company: Hemispherx; Rega Institute For Medical Research
Phase of Development: Phase II
Chemical Image:
Click image to enlarge
Molecular Weight: 995.584
(Compound details obtained from ChemIDplus Advanced1 and NIAID Therapeutics Database2)
Patent Version Content


Mechanism of Action: Biological response modifier. Rintatolimod is a synthetic mismatched double-stranded RNA toll-like receptor (TLR) agonist, having specificity for TLR-3. TLR-3, located in the endoplasm of certain immune cells, recognizes and responds to RNA virus infection. TLR-3 activation by natural or synthetic ligands induces cytokine secretion (such as type I interferon, TNF-α, IL-12, and MCP-1).3-5 Rintatolimod has also been described to activate RNase-L against viral RNA transcripts.5 

: Rintatolimod has been studied in patients with metastatic cancer and, in a survey of seven patients, it exhibited a mean plasma half-life of 23 minutes.6 

Resistance: Rintatolimod has exhibited equal in vitro activity against wild-type HIV and HIV resistant to nevirapine, protease inhibitors, or nucleoside analogue reverse transcriptase inhibitors.7 

Dosing in Clinical Trials

Rintatolimod is administered via intravenous (IV) infusion.8,9

Phase II (treatment-experienced with virologic suppression)
  • AMP 720: Study investigating the role of rintatolimod in structured treatment interruption of antiretroviral therapy (ART) 
    Rintatolimod 200- to 400-mg IV infusions administered twice weekly added to structured treatment interruption of ART (up to three structured treatment interruptions over 64 weeks) versus no study drug intervention.8,10

Phase II (treatment-experienced; failing ART)
  • AMP 719: Study investigating the safety and efficacy of adding rintatolimod to continuous ART (no structured treatment interruptions)
    Rintatolimod 200- to 400-mg IV infusions administered twice weekly for 24 weeks (along with ART) versus no study drug added to ART. This study was terminated prior to completion.9

Additional clinical trials of rintatolimod have also been completed. 

Adverse Events

In the AMP 720 study, adverse events associated with rintatolimod were described as generally mild and self-limiting. No adverse effects on lactic acid levels, insulin resistance, or hyperlipidemia were reported in patients receiving rintatolimod alone.10

Drug Interactions

Drug interactions related to rintatolimod use are currently unknown.


1. United States National Library of Medicine. ChemIDplus Advanced. Last accessed on April 16, 2014.

2. National Institute of Allergy and Infectious Diseases (NIAID). NIAID ChemDB, HIV Drugs in Development. Last accessed on April 16, 2014.

3. Engel AL, Holt GE, Lu H. The pharmacokinetics of Toll-like receptor agonists and the impact on the immune system. Expert Rev Clin Pharmacol. 2011 Mar;4(2):275-89. Last accessed on April 16, 2014.

4. Nicodemus CF, Berek JS. TLR3 agonists as immunotherapeutic agents. Immunotherapy. 2010 Mar;2(2):137-40. Last accessed on April 16, 2014.

5. [No authors listed]. Mismatched double-stranded RNA: polyI:polyC12U. Drugs R D. 2004;5(5):297-304. Last accessed on April 16, 2014.

6. Brodsky I, Strayer DR, Krueger LJ, Carter WA. Clinical studies with ampligen (mismatched double-stranded RNA). J Biol Response Mod. 1985 Dec;4(6):669-75. Last accessed on April 16, 2014.

7. Essey RJ, McDougall BR, Robinson WE Jr. Mismatched double-stranded RNA (polyI-polyC(12)U) is synergistic with multiple anti-HIV drugs and is active against drug-sensitive and drug-resistant HIV-1 in vitro. Antiviral Res. 2001 Sep;51(3):189-202. Last accessed on April 16, 2014.

8. Hemispherx Biopharma. The Role of Ampligen in Strategic Therapeutic Intervention (STI) of Highly Active Anti-Retroviral Therapy (HAART): A Multi-Center, Randomized, Controlled Study of Ampligen Potentiation of the HAART-Free Interval. In: Bethesda (MD): National Library of Medicine (US). Registered on May 6, 2002. NLM Identifier: NCT00035893. Last accessed on April 16, 2014.

9. Hemispherx Biopharma. A Multi-Center, Randomized, Controlled Study of the Biological Actions of Ampligen as an Adjunct to HAART in HIV Disease. In: Bethesda (MD): National Library of Medicine (US). Registered on May 3, 2002. NLM Identifier: NCT00035581. Last accessed on April 16, 2014.

10. Blick G, Greiger-Zanlungo P, Strayer DR, Mitchell WM, Carter WA. Interim Results of AMP720: A Phase IIb Prospective, Randomized, Controlled Study Evaluating the Immunomodulatory Role of Ampligen (Poly I:Poly C12U) Against HIV During STI. 2nd International AIDS Society (IAS) Conference on HIV Pathogenesis and Treatment; July 13-16, 2003; Paris, France. Abstract 596. Last accessed on April 16, 2014.

Last Reviewed: April 16, 2014

Last Updated: April 25, 2014

Back to Top