Peptide education
Brilacidin
PMX-30063 · PMX63
Brilacidin is an investigational non-peptide molecule designed to mimic aspects of host-defense peptides. Researchers have studied it as an intravenous drug and as an oral rinse.
In the main comparison of a randomized COVID-19 study, median time to sustained recovery was 13 days with five doses of brilacidin and 12 days with pooled placebo, both alongside standard care.
Overview
Quick answer
Laboratory activity, results in hospitalized patients, and a registered skin-infection study are different kinds of evidence. An exact FDA active-ingredient search found no match for brilacidin.
Is brilacidin approved in the United States?
An openFDA exact active-ingredient search for brilacidin returned no match. The search has name and database limits and does not establish approval status outside the United States.
Is it a peptide?
It is a non-peptide molecule designed to mimic properties of host-defense peptides, rather than a peptide sequence.
Did the COVID-19 trial show faster recovery?
No significant difference was reported for the main comparison of five-dose brilacidin with pooled placebo. Both groups received standard care.
What did the skin-infection trial find?
The registry has no posted outcomes. The sponsor separately reported early responses among treated patients assessed at 48 to 72 hours. The study was not powered for statistical comparisons.
What did the mucositis study report?
In the registry's modified analysis, severe oral mucositis occurred in 9 of 21 participants using brilacidin rinse and 15 of 25 using placebo rinse. The analysis included 46 of 61 randomized participants who received at least one rinse dose and 55 to 72 Gy of radiation.
Evidence
Evidence snapshot
Median sustained recovery time with five-dose IV brilacidin versus pooled placebo, in the registry's main comparison.
Severe mucositis in the modified analysis of rinse versus placebo, limited to participants who received a rinse dose and 55 to 72 Gy of radiation.
The registry has no posted results; the sponsor separately reported early responses in assessed treated patients.
Viral inhibition was observed in cells, not established as clinical benefit.
Claims
Common claims vs evidence
| Claim | Human evidence | Mechanistic evidence | Verdict |
|---|---|---|---|
| A laboratory antiviral result proves benefit in people with COVID-19. | In the randomized COVID-19 study, the five-dose group did not recover faster than pooled placebo on the main comparison. | A cell-culture study observed SARS-CoV-2 inhibition under laboratory conditions. | Laboratory activity and clinical outcomes are distinct. |
| The skin-infection trial registry proves brilacidin works as an antibiotic. | The registry describes a randomized phase 2 study comparing three brilacidin schedules with daptomycin, but it has no posted results. The sponsor separately reported early response in assessed treated patients. | The small study was not powered to establish equivalence or superiority. | |
| Oral rinse results establish the effects of intravenous brilacidin. | The mucositis trial studied a rinse in people receiving chemoradiation; the COVID-19 and skin studies used intravenous formulations in different populations. | Formulation, route, and indication must be assessed separately. |
Bottom line
Main takeaway
Brilacidin is investigational; an exact FDA active-ingredient search found no match under that name.
The rinse and IV studies address different conditions and formulations.
The COVID-19 registry separates three-dose and five-dose groups, while the mucositis endpoint uses a modified analysis population.
Identity
What it is
Brilacidin is a non-peptide defensin mimetic. It has been investigated for bacterial skin infection, oral mucositis, and COVID-19.
An openFDA exact active-ingredient search for brilacidin found no match; the search does not cover every name or jurisdiction.
Use context
Routes, doses, and cycle patterns
The registered studies used different preparations and schedules for different conditions.
Human studies and product labels
Acute bacterial skin infection study
- Purpose
- Early clinical response after treatment
- Context
- Study-reported phase 2 design
- Route
- Intravenous
- Amount
- 0.6 mg/kg once in one group, 0.8 mg/kg once in another
- Frequency
- Single dose in those two groups
A third group received 0.6 mg/kg on day 1 and 0.3 mg/kg on days 2 and 3. The comparator received daptomycin. The registry has no posted outcomes; the sponsor separately reported early response results.
Oral mucositis study
- Purpose
- Severe mucositis during chemoradiation for head and neck cancer
- Context
- Study-reported phase 2 regimen
- Route
- Oral rinse
- Amount
- 3 mg/mL solution
- Frequency
- Three times daily
- Duration
- Seven weeks
The modified analysis required at least one rinse dose and a cumulative radiation dose of 55 to 72 Gy.
Hospitalized COVID-19 study
- Purpose
- Time to sustained recovery
- Context
- Study-reported phase 2 regimen
- Route
- Intravenous infusion
- Frequency
- Three or five daily doses alongside standard care
The registry reports the three-dose and five-dose groups separately against pooled placebo.
What varies
- Formulation: a rinse and an IV infusion do not have the same route or study population.
- Evidence: the skin registry has no posted results; the sponsor separately reported outcomes for assessed patients.
Research
Research evidence
ClinicalTrials.gov contains posted results for a COVID-19 trial and an oral mucositis trial. The skin-infection registry has no posted results, but the sponsor separately reported early responses and adverse events. Laboratory SARS-CoV-2 findings are separate.
| Study / evidence area | Population | Design | Product context | Main outcome | Limitations | Weight |
|---|---|---|---|---|---|---|
| COVID-19 phase 2 | 120 hospitalized participants with COVID-19 | Randomized, double-blind, placebo-controlled study | IV brilacidin or placebo alongside standard care | Median sustained recovery was 13 days in the five-dose group versus 12 days with pooled placebo. | Small study; the three-dose group was analyzed separately. | Limited |
| Oral mucositis phase 2 | Patients receiving chemoradiation for head and neck cancer | Randomized, double-blind, placebo-controlled study | Brilacidin rinse versus placebo rinse | Severe mucositis occurred in 9 of 21 versus 15 of 25 in the modified analysis. | The modified analysis included 46 of 61 randomized participants after applying rinse and radiation-dose criteria. No comparison test for this endpoint was posted in the registry. | Limited |
| Acute bacterial skin infection phase 2 | 215 participants with acute bacterial skin and skin structure infections | Randomized, double-blind, active-comparator study | Three IV brilacidin regimens versus IV daptomycin | At 48 to 72 hours, the sponsor reported success in 47 of 51 assessed recipients of 0.6 mg/kg brilacidin, 46 of 48 receiving 0.8 mg/kg, 51 of 52 on the three-day brilacidin regimen, and 45 of 48 receiving daptomycin. | These are sponsor-reported outcomes for assessed treated patients, not all randomized participants. The study was not powered for statistical comparisons, and the registry has no posted outcomes. | Limited |
Cautions
Safety and unknowns
- The COVID-19 registry reported reduced sensation in 16 of 44 people in the five-dose brilacidin group and none of 60 in pooled placebo; adverse events do not by themselves establish causation.
- A sponsor protocol reports numbness or tingling in 31 of 53, 33 of 53, and 39 of 53 participants across the three skin-study brilacidin groups, versus 4 of 50 receiving daptomycin.
- Serious adverse events occurred in both arms of the mucositis study. Small trials cannot characterize uncommon or long-term harms reliably.
Product quality
Product identity and quality
The trials used defined research formulations for IV infusion or oral rinse.
Formulation and route
Findings from an oral rinse cannot be assumed to apply to an IV formulation.
Mechanism
How it is proposed to work
Brilacidin was designed to mimic some actions of host-defense peptides.
A laboratory study observed inhibition of SARS-CoV-2 in cell culture; this does not determine patient outcomes.
FAQ
Common questions
Is brilacidin a peptide drug?
It is a non-peptide molecule designed to mimic aspects of host-defense peptides.
Did it shorten recovery in the COVID-19 study?
The registry's main five-dose comparison did not show faster sustained recovery than pooled placebo.
Details
Technical details
Sources
References
- 1.
ClinicalTrials.gov. Phase 2 brilacidin COVID-19 study and posted results
NCT04784897
- 2.
ClinicalTrials.gov. Phase 2 brilacidin skin infection study registration
NCT02052388
- 3.
Cellceutix SEC filing. Sponsor-reported phase 2b skin infection trial outcomes
Sponsor-reported results for treated patients assessed at the early response visit.
- 4.
ClinicalTrials.gov. Sponsor protocol summarizing prior skin infection trial safety
NCT04784897
The COVID-19 study protocol summarizes sponsor-reported safety in the earlier skin infection study.
- 5.
ClinicalTrials.gov. Phase 2 brilacidin oral rinse study and posted results
NCT02324335
- 6.
PubMed. Brilacidin inhibition of SARS-CoV-2 in cell culture
doi:10.3390/v13020271 PMID:33572467
- 7.
FDA. Drugs@FDA search page for brilacidin
An openFDA exact active-ingredient query for brilacidin found no match. The link opens the FDA search page, not a saved result; names and database coverage are limited.