CLINICAL TRIALS PROFILE FOR BRINCIDOFOVIR

All Clinical Trials for brincidofovir

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00793598 ↗	CMX001 in Post-transplant Patients With BK Virus Viruria	Completed	Chimerix	Phase 1/Phase 2	2009-11-01	This was a randomized, double-blind, multiple-dose placebo-controlled study of oral brincidofovir (BCV) in hematopoietic stem cell transplant and renal transplant recipients with BK virus viruria.
NCT00942305 ↗	Study of CMX001 to Prevent/Control Cytomegalovirus Infection in R+ Hematopoietic Stem Cell Transplant Recipients	Completed	Chimerix	Phase 2	2009-10-01	This was a multicenter, randomized, double-blind, placebo-controlled, dose-escalation study of brincidofovir (BCV) administered orally once or twice weekly for up to 11 weeks. Dosing was initiated immediately following engraftment (between Days 14-30 post-transplant) to prevent/control cytomegalovirus (CMV) infection or prevent disease in R+ hematopoietic stem cell transplant (HCT) recipients.
NCT01143181 ↗	Study to Assess Brincidofovir Treatment of Serious Diseases or Conditions Caused by Double-stranded DNA Viruses	Completed	Chimerix	Phase 3	2010-12-01	This was a multicenter, open-label study of oral brincidofovir (BCV) treatment of serious disease or conditions caused by double-stranded DNA (dsDNA) virus(es). Subjects received either a weight-based or a fixed dose of oral BCV once weekly (QW) or twice weekly (BIW) for up to 3 months until clinical disease was resolved or stabilized and/or viral DNA by polymerase chain reaction testing was negative for 4 consecutive weeks, whichever was longer. Under the first protocol amendment, adults and adolescents (≥13 years) received 200 mg or 300 mg BCV BIW (not to exceed 4 mg/kg total weekly dose) depending on the difficulty of treating their disease (i.e., Group 1 or Group 2, respectively), and pediatric subjects (≤12 years) received 4 mg/kg BCV BIW. Under the second protocol amendment, adults and adolescents (≥13 years), regardless of viral infection/disease, had a maximum weekly dose of 200 mg, i.e., 200 mg QW or 100 mg BIW; not to exceed 4mg/kg total weekly dose. Pediatric subjects (≤12 years), regardless of viral infection/disease, had a maximum weekly dose of 4 mg/kg, i.e., 4 mg/kg QW or 2 mg/kg BIW; not to exceed 200 mg.
NCT01241344 ↗	Phase 2 Study to Evaluate Brincidofovir for the Prevention of Adenovirus Disease	Completed	Chimerix	Phase 2	2010-11-01	This study was designed to assess the safety and efficacy of preemptive treatment with oral brincidofovir (BCV), as compared to placebo, for the prevention of adenovirus (AdV) disease in recipients of hematopoietic stem cell transplantation (HCT) with asymptomatic AdV viremia.
NCT01769170 ↗	A Study of the Safety and Efficacy of CMX001 for the Prevention of CMV Infection in CMV-seropositive HCT Recipients	Completed	Chimerix	Phase 3	2013-08-01	This randomized, double-blind, placebo-controlled, parallel group, multicenter study compared the effectiveness of oral brincidofovir (BCV) to placebo for the prevention of cytomegalovirus (CMV) infection in stem cell transplant patients who were CMV seropositive but negative for CMV viremia before starting treatment with BCV.
NCT02087306 ↗	Study to Assess the Safety and Efficacy of Brincidofovir in Treatment of Early Versus Late Adenovirus Infection	Completed	Chimerix	Phase 3	2014-03-01	This was a Phase 3 open-label, non-randomized, multicenter study of oral brincidofovir (BCV) administered twice weekly for the treatment of adenovirus (AdV) infection detected during asymptomatic AdV viremia or during symptomatic AdV infection.

Clinical Trial Conditions for brincidofovir

Condition Name

Condition Name for brincidofovir
Intervention	Trials
Adenovirus	4
Cytomegalovirus Disease	2
Adenovirus Infection	2
Ebola Virus	1

Condition MeSH

Condition MeSH for brincidofovir
Intervention	Trials
Adenoviridae Infections	7
Infections	5
Communicable Diseases	4
Virus Diseases	4

Clinical Trial Locations for brincidofovir

Trials by Country

Trials by Country for brincidofovir
Location	Trials
United States	206
United Kingdom	5
Canada	4
Belgium	3
Germany	3

Trials by US State

Trials by US State for brincidofovir
Location	Trials
California	14
North Carolina	11
Washington	10
Pennsylvania	10
New York	10

Clinical Trial Progress for brincidofovir

Clinical Trial Phase

Clinical Trial Phase for brincidofovir
Clinical Trial Phase	Trials
PHASE1	1
Phase 3	5
Phase 2	8

Clinical Trial Status

Clinical Trial Status for brincidofovir
Clinical Trial Phase	Trials
Completed	9
Terminated	5
Withdrawn	3

Clinical Trial Sponsors for brincidofovir

Sponsor Name

Sponsor Name for brincidofovir
Sponsor	Trials
Chimerix	16
SymBio Pharmaceuticals	4
Food and Drug Administration (FDA)	1

Sponsor Type

Sponsor Type for brincidofovir
Sponsor	Trials
Industry	21
U.S. Fed	1

Brincidofovir Market Analysis and Financial Projection

Last updated: February 8, 2026

What Are the Recent Developments in Brincidofovir Clinical Trials?

Brincidofovir, an oral nucleotide analog antiviral, is under ongoing evaluation due to its potential against various viral infections. The drug received Emergency Use Authorization (EUA) in the U.S. in 2020 for smallpox treatment but was withdrawn from consideration for broader approvals due to mixed clinical results and safety concerns.

Current Clinical Trial Status:

• Phase 2 and 3 Trials: Several studies are ongoing or completed primarily targeting viral diseases such as cytomegalovirus (CMV) and adenovirus in transplant populations.

• Key Pending Data: Results from phase 3 trials evaluating efficacy against CMV in hematopoietic stem cell transplant recipients are awaited, expected to clarify the drug's therapeutic profile.

• Regulatory Interactions: No recent official submissions for approval; discussions with health authorities remain limited.

How Has the Market for Brincidofovir Evolved?

Market Entry and Limitations:

• Originally developed by Chimerix Inc., brincidofovir entered the antiviral market around 2015–2016 with high expectations for broad-spectrum activity.

• Market Challenges: Concerns over safety, particularly gastrointestinal and hepatic adverse events, slowed adoption, and clinical development was not accelerated post-EUA withdrawal.

• Pricing and Access: Cost concerns and limited indications led to minimal market penetration outside clinical settings.

Market Size and Segments:

• The global antiviral market exceeds $30 billion in 2022, with nucleotide analogs comprising a significant segment due to drugs like tenofovir and entecavir.

• Brincidofovir's niche is limited mainly to transplant-associated viral infections, representing an estimated $500 million segment historically, but the current market presence is minimal due to clinical setbacks.

What Projections Exist for Brincidofovir's Future Market Position?

Market Outlook (2023-2030):

Year	Estimated Revenue (USD Million)	Notes
2023	50-75	Limited sales, primarily from ongoing trials
2025	150-200	Potential approval for CMV if phase 3 results positive
2030	300-500	Possible expansion to other viral indications if approved

Factors Influencing Future Growth:

• Clinical Trial Results: Positive data for CMV or other indications can lead to regulatory approval and market re-entry.

• Safety Profile: Improved safety profile compared to earlier data could enhance adoption in transplantation settings.

• Competitive Landscape: Other antivirals like letermovir (approved for CMV prophylaxis) could limit market expansion unless brincidofovir demonstrates clear advantages.

Competitive Analysis:

Drug	Indication	Market Status	Strengths
Brincidofovir	CMV in transplant pts	Under clinical evaluation	Oral administration, broad-spectrum activity
Letermovir	CMV prophylaxis	Approved in US and EU	Well-tolerated, proven efficacy
cidofovir	CMV, adenovirus	Approved (injectable)	Potent antiviral activity

What Regulatory and Developmental Challenges Persist?

• Safety Concerns: Gastrointestinal toxicity and hepatotoxicity observed in trials limit widespread use.

• Limited Indications: Focus on transplant-related viral infections restricts market size.

• Regulatory Hurdles: No recent filings for new indications; further data required for seeking approval.

• Economic Factors: Cost of development and market entry risk remains high due to prior setbacks.

What Are the Key Drivers and Barriers for Brincidofovir?

Drivers:

• Effective oral antiviral option with activity against multiple viruses.

• Potential for use in immunocompromised populations, especially if safety improves.

• Increased awareness of antiviral resistance may renew interest.

Barriers:

• Safety profile issues have limited broader application.

• Competitive landscape with existing approved drugs.

• Limited clinical data supporting expanded indications.

• Regulatory uncertainty post-withdrawal of EUA.

Key Takeaways

• Brincidofovir's development has stagnated since the 2020 EUA withdrawal; ongoing trials focus on CMV in transplant patients.

• Market size remains limited unless new indications are approved and safety concerns mitigated.

• Positive phase 3 trial results could see brincidofovir re-entering the market, particularly in CMV management.

• Competition from approved agents like letermovir poses a barrier unless brincidofovir demonstrates superior efficacy or safety.

• Future growth heavily depends on clinical trial outcomes, regulatory approvals, and developments addressing safety issues.

FAQs

1. Why was brincidofovir withdrawn from the EUA?
Due to safety concerns and mixed clinical data, particularly gastrointestinal and hepatic adverse events, which limited its broader approval and market penetration.

2. What infections is brincidofovir primarily targeting now?
Currently, the focus is on cytomegalovirus (CMV) in transplant recipients, with ongoing clinical trials assessing efficacy.

3. How does brincidofovir compare to other antiviral drugs?
It offers an oral, broad-spectrum antiviral activity but has safety profile limitations compared to approved agents like letermovir, which has demonstrated better tolerability.

4. What are the prospects for brincidofovir's approval in the near future?
Conditional on positive phase 3 trial outcomes and formal regulatory submissions; otherwise, prospects remain uncertain.

5. What factors could enable brincidofovir to regain market relevance?
Demonstration of improved safety, approval for new indications, and potential combination therapies.

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Sources
[1] Chimerix Inc., "Brincidofovir Development Update," 2022.
[2] MarketWatch, "Antiviral Drugs Market Size & Share," 2022.
[3] FDA, "Emergency Use Authorization for Brincidofovir," 2020.