What is an investigational drug?
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.
What is BMS-986001?
BMS-986001 is an investigational drug for the treatment of HIV infection.
- BMS-986001 is an investigational anti-HIV drug included in the nucleoside reverse transcriptase inhibitor (NRTI) drug class.
- NRTIs prevent HIV from replicating by blocking reverse transcriptase, an HIV enzyme. By preventing HIV from replicating, NRTIs reduce the amount of HIV in the blood.
- BMS-986001 is similar in chemical structure to the FDA-approved NRTI stavudine (brand name: Zerit). However, in vitro studies have found that BMS-986001 not only has greater antiviral activity than stavudine, but also has less toxicity than stavudine and other NRTIs. In addition, research in animals has shown that BMS-986001 is not associated with kidney or bone toxicities.
How are clinical trials of investigational drugs conducted?
Clinical trials are conducted in “phases.” Each phase has a different purpose and helps researchers answer different questions.
- Phase I trials: Researchers test an investigational drug in a small group of people (20–80) for the first time. The purpose is to evaluate its safety and identify side effects.
- Phase II trials: The investigational drug is administered to a larger group of people (100–300) to determine its effectiveness and to further evaluate its safety.
- Phase III trials: The investigational drug is administered to large groups of people (1,000–3,000) to confirm its effectiveness, monitor side effects, compare it with standard or equivalent treatments, and collect information that will allow the investigational drug to be used safely.
An investigational drug must be proven safe and effective in a Phase III clinical trial to be considered for traditional 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.
In what phase of testing is BMS-986001?
BMS-986001 is currently being studied in a Phase II clinical trial.
What have recent studies shown about BMS-986001?
In a Phase Ia-IIb study, four different strengths of BMS-986001 taken once daily were compared with placebo in HIV-infected, treatment-experienced participants who were off therapy for at least 3 months. Study participants did not receive additional anti-HIV drugs 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.)
In this trial, significant reductions in 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.
An additional Phase II study of BMS-986001 in HIV-infected treatment-naive adults is under way.
More information on testing of BMS-986001 is available from these sources:
- BMS-986001 study results presented at the 13th International Workshop on Clinical Pharmacology of HIV Therapy, March 16–18, 2012; abstract O_06:
- BMS-986001 study results presented at the 50th Interscience Conference on Antimicrobial Agents and Chemotherapy, September 12-15, 2010:
- BMS-986001 study results presented at the 19th International AIDS Conference, July 22-27, 2012:
- PubMed abstract:
Where can I get more information about clinical trials studying BMS-986001?
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.
I am interested in participating in a clinical trial of BMS-986001. How can I find more information about participating in a clinical trial?
Clinical trials involve benefits and risks. Before deciding to participate in a clinical trial, talk to your health care provider. For more information on participating in clinical trials, visit NIH Clinical Research Trials and You
Last Reviewed: January 25, 2013
Last Updated: January 25, 2013