An investigational drug is one that is under study and is not approved by the U.S. Food and Drug Administration (FDA) for sale in the United States. Medical research studies are conducted to evaluate the safety and effectiveness of an investigational drug. These research studies are also called clinical trials. Once an investigational drug has been proven safe and effective in clinical trials, FDA may approve the drug for sale in the United States.
BMS-986001 is an investigational drug that is being studied for the treatment of HIV infection.
BMS-986001 belongs to a class (group) of HIV drugs called nucleoside reverse transcriptase inhibitors (NRTIs).2 NRTIs block an HIV enzyme called reverse transcriptase. (An enzyme is a protein that starts or increases the speed of a chemical reaction.) By blocking reverse transcriptase, NRTIs prevent HIV from multiplying and can reduce the amount of HIV in the body.
BMS-986001 is similar in chemical structure to the FDA-approved NRTI stavudine (brand name: Zerit). However, in vitro studies have shown that BMS-986001 may be more effective than stavudine and may be less toxic than stavudine and other NRTIs. 4,5,6 (In vitro studies are studies done in test tubes or other laboratory equipment and not on animals or humans.) Research in animals has shown that BMS-986001 is not associated with kidney or bone toxicities.7
Clinical trials are conducted in phases. Each phase has a different purpose and helps researchers answer different questions.8
In most cases, an investigational drug must be proven safe and effective in a Phase III clinical trial to be considered for approval by the FDA for sale in the United States. Some drugs go through the FDA’s accelerated approval process and are approved before a Phase III clinical trial is complete. After a drug is approved by the FDA and made available to the public, researchers track its safety in Phase IV trials to seek more information about the drug’s risks, benefits, and optimal use.8
BMS-986001 is currently being studied in a Phase IIb clinical trial.2
In a 10-day Phase Ib-IIa study, four different strengths of BMS-986001 taken once daily were compared with placebo in HIV-infected participants who had taken HIV medicines before entering the study (treatment-experienced) but who were off HIV medicines for at least 3 months. (A placebo is an inactive drug that is identical in appearance to the active drug being studied.) Study participants did not receive additional HIV medicines as part of a background regimen. (A background regimen is a combination of drugs that are not being studied as the investigational drug[s] in the clinical trial, but are being given to help control a participant’s HIV infection.)9,10
In this study, significant reductions in the amount of HIV in the blood (viral load) were seen after 10 days of treatment with each of the BMS-986001 doses studied. In terms of safety, BMS-986001 was generally well tolerated.9,10
A Phase IIb study of BMS-986001 is under way in HIV-infected adults who had never taken HIV medicines before entering the study (treatment-naive).11
In the 10-day Phase Ib-IIa study discussed under the previous question, side effects were reported by participants but no clear pattern of side effects was seen.9
Because BMS-986001 is still being studied, information on possible side effects of the drug is not complete. As testing of BMS-986001 continues, additional information on possible side effects will be gathered.
More information about BMS-986001-related research studies is available from the AIDSinfo database of ClinicalTrials.gov study summaries. Click on the title of any trial in the list to see the ClinicalTrials.gov trial summary and more information about the study.
Participating in a clinical trial can provide benefits. For example, a volunteer participant can benefit from new research treatments before they are widely available. Participants also receive regular and careful medical attention from a research team that includes doctors and other health professionals. However, clinical trials may also involve risks of varying degrees, such as unpleasant, serious, or even life-threatening side effects from the treatment being studied.8
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.
1. United States National Library of Medicine. ChemIDplus Advanced.
2. National Institute of Allergy and Infectious Diseases (NIAID). NIAID ChemDB, HIV Drugs in Development.
3. Bristol-Myers Squibb: Press Releases. Bristol-Myers Squibb and Oncolys BioPharma Enter Global Licensing Agreement for Investigational HIV Compound. Accessed March 29, 2013.
4. Dutschman GE, Grill SP, Gullen EA, et al. Novel 4'-substituted stavudine analog with improved anti-human immunodeficiency virus activity and decreased cytotoxicity. Antimicrob Agents Chemother. 2004 May;48(5):1640-6.
5. Yang G, Dutschman GE, Wang CJ, et al. Highly selective action of triphosphate metabolite of 4'-ethynyl D4T: a novel anti-HIV compound against HIV-1 RT. Antiviral Res. 2007 Mar;73(3):185-91.
6. Wang F, Flint O. The HIV NRTI BMS-986001 does not degrade mitochondrial DNA in long term primary cultures of cells isolated from human kidney, muscle and subcutaneous fat. Abstract presented at: 19th International AIDS Conference; July 22-27, 2012; Washington, DC. Abstract TUPE042.
7. Guha M, Pilcher G, Moehlenkamp J, et al. Absence of renal and bone toxicity in non-clinical studies of BMS-986001, a nucleoside reverse transcriptase inhibitor (NRTI) of human immunodeficiency virus (HIV). Abstract presented at: 19th International AIDS Conference; July 22-27, 2012; Washington, DC. Abstract TUPE041.
8. National Institutes of Health (NIH). NIH Clinical Research Trials and You.
9. Cotte L, Dellamonica P, Raffi F, et al. A Phase-Ib/IIa Dose-Escalation Study of OBP-601 (4’-ethynyl-d4T, Festinavir) in Treatment-Experienced, HIV-1-Infected Patients. Abstract presented at: 50th Interscience Conference on Antimicrobial Agents and Chemotherapy (ICAAC); September 12-15, 2010; Boston, MA. Abstract H-933.
10. Hwang C, Zhu L, Chan H, et al. Antiviral activity, exposure-response, and resistance analyses of monotherapy with the novel HIV NRTI BMS-986001 in ART-experienced subjects. Abstract presented at: 13th International Workshop on Clinical Pharamcology of HIV Therapy; March 16-18, 2012; Barcelona, Spain. Abstract O_06.
11. Bristol-Myers Squibb. A Phase IIb Randomized, Controlled, Partially Blinded Clinical Trial to Investigate Safety, Efficacy and Dose-response of BMS-986001 in Treatment-naive HIV-1-infected Subjects, Followed by an Open-label Period on the Recommended Dose. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on December 7, 2011. NLM Identifier: NCT01489046. Last accessed April 3, 2013.
Last Reviewed: May 29, 2013
Last Updated: May 29, 2013