Tembexa (brincidofovir oral) / SymBio Pharma, Jazz - LARVOL DELTA

In vivo characterization of preclinical efficacy of brincidofovir against glioblastoma (SNO 2025) - "Brincidofovir (BCV) is lipid-conjugated cidofovir characterized by a lack of nephrotoxicity and superior activity compared to cidofovir...In the combination study, mice were administered temozolomide 2 mg/kg p.o. (TMZ) once daily for 5 days, whole brain radiotherapy (WBRT) of 1.5 Gy for 5 days plus TMZ, or bevacizumab 5 mg/kg i.p. BIW alone or concomitant with BCV 10 mg/kg... BCV monotherapy suppressed tumor growth and prolonged survival in PDX models of GBM and improved the efficacy of the standard of care for GBM."

Preclinical • Brain Cancer • Glioblastoma • Solid Tumor

Synergistic antiviral activity against human adenovirus through combination of itraconazole and brincidofovir. (PubMed, Br J Pharmacol) - "Given the gastrointestinal toxicity associated with brincidofovir, its combination with the host-directed drug itraconazole allowed lower brincidofovir doses to be used to decrease HAdV titres, thereby minimizing adverse drug effects while maintaining antiviral efficacy."

Journal • Gastroenterology • Gastrointestinal Disorder • Infectious Disease

Broad-spectrum antiviral brincidofovir inhibits Epstein-Barr virus and related gammaherpesvirus in human and nonhuman primate cells. (PubMed, J Clin Invest) - "These results suggest that BCV may be a useful antiviral for inhibiting EBV activity in MS patients. Additionally, this work further validates the utility of CalHV-3 in marmosets as a translational model for the investigation of successful EBV-targeting therapeutics."

Journal • CNS Disorders • Epstein-Barr Virus Infections • Multiple Sclerosis

Battle won, war ongoing: A call for mpox vaccination in high-risk dermatologic cases. (PubMed, JAAD Case Rep) - No abstract available

Journal

Lipid-Optimized Sulfone-Bridged Cidofovir Prodrugs as Potent Antivirals Against Orthopoxviruses. (PubMed, J Med Chem) - "Compound 13f exhibited antiviral activity against vaccinia virus (VACV) comparable to that of the reference brincidofovir (BCV), while 13i displayed 8.2-fold enhanced potency. Notably, 13i exhibited a favorable safety profile and significant therapeutic efficacy in both VACV-challenged BALB/c and MPXV-challenged severe combined immunodeficiency mouse models. Through systematic lipid-chain optimization, we identified 13i as a promising antiviral candidate with enhanced efficacy and favorable pharmaceutical properties."

Journal • Genetic Disorders • Immunology • Infectious Disease • Primary Immunodeficiency

In vivo characterization of preclinical efficacy of brincidofovir against glioblastoma (WFNOS 2025) - "Brincidofovir (BCV) is lipid-conjugated cidofovir characterized by a lack of nephrotoxicity and superior activity compared to cidofovir...In the combination study, mice were administered temozolomide 2 mg/kg p.o. (TMZ) once daily for 5 days, whole brain radiotherapy (WBRT) of 1.5 Gy for 5 days plus TMZ, or bevacizumab 5 mg/kg i.p. BIW alone or concomitant with BCV 10 mg/kg... BCV monotherapy suppressed tumor growth and prolonged survival in PDX models of GBM and improved the efficacy of the standard of care for GBM."

Preclinical • Brain Cancer • Glioblastoma • Solid Tumor

Disseminated Mpox Infection in a Vaccinated Kidney Transplant Recipient (KIDNEY WEEK 2025) - "He was on a belatacept-based immunosuppressive regimen and has received Jynneos approximately two years prior to symptom onset...Given the severity of his lesions, he was treated with IV tecovirimat, vaccinia immune globulin (VIVIg), brincidofovir and topical cidofovir...Additional studies are needed to assess the effectiveness of these vaccines in immunocompromised patients. Figure 1: Perioral lesions before treatment (A) and after treatment ~2 months later (B), abdomen (C), right hand (D), and back lesions with typical desquamation (E)"

Clinical • Chronic Kidney Disease • Human Immunodeficiency Virus • Infectious Disease • Renal Disease • Transplantation

Therapeutic Repurposing of Brincidofovir in Non-Hodgkin Lymphoma – Potential Synergy with Immune Checkpoint Blockade (ASH 2024) - "Conclusions : Taken together, these results demonstrate a novel role of BCV in the treatment of lymphoma. The immunogenic effects of BCV suggests potential for combination with PD1/PDL1 and CTLA4 checkpoint immunotherapy."

Checkpoint block • Checkpoint inhibition • IO biomarker • B Cell Lymphoma • B Cell Non-Hodgkin Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Natural Killer/T-cell Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • BCL2 • CCL2 • CXCL9 • GZMB • JAK3 • MYC • PD-L1 • TP63

Re-emergence and clinical management of monkeypox: insights into viral biology, therapeutics, and vaccination. (PubMed, Mol Biol Rep) - "Antiviral therapies such as tecovirimat (TPOXX), cidofovir, and brincidofovir are under consideration, with tecovirimat showing the most clinical promise...Currently approved vaccines include ACAM2000, LC16 KMB, and JYNNEOS (MVA-BN), with JYNNEOS preferred due to its safety profile, especially for immunocompromised individuals and pregnant women. Preventive strategies, public education, and international surveillance are essential to mitigate further outbreaks. Understanding the biology, clinical behavior, and control measures of Mpox is critical for informing future responses and improving global preparedness."

Journal • Review • Fatigue • Infectious Disease • Musculoskeletal Pain • Pain

ADENOVIRUS INFECTION IN ALLOGENEIC TRANSPLANT ADULT PATIENTS WITH POST-TRANSPLANT CYCLOPHOSPHAMIDE (SIE 2025) - "All pts received PTCy in association with sirolimus (31), MMF (28) and ATG (1) as GvHD prophylaxis...Overall, 21 pts received specific antiviral treatment, mainly based on intravenous immunoglobulin and cidofovir. Brincidofovir was administered as compassionate use in 6 pts...We reported AdV mainly in the context of haplo and MUD transplants, myeloablative conditioning regimen, severe aGvHD. In our analysis OS was positively affected by the introduction of AdV weekly monitoring which is warranted for early diagnosis and pre-emptive treatments."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Bone Marrow Transplantation • CNS Disorders • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Transplantation

Brincidofovir in the Era of Mpox (ASTMH 2025) - "BCV moves intracellularly and is phosphorylated to cidofovir-diphosphate (CDV-PP), which then inhibits viral DNA polymerase activity. As reported from published cases, the use of BCV in 5 patients as a first-line treatment of clade II infections showed improved survival outcomes. Based on the mechanism of action of the drug, development of MPXV resistance to the viral DNA polymerase inhibitory activity of BCV is less likely to occur than with tecovirimat (TCV), which targets a viral surface protein. BCV has been demonstrated to be synergistic with TCV in vitro and in vivo in orthopoxviral infections."

Infectious Disease • Pediatrics

Evaluating the advancements and efficacies in pharmacological Mpox treatments: a comprehensive review. (PubMed, Front Pharmacol) - "Prominent reviewed stewards include tecovirimat, brincidofovir, and cidofovir: drugs first developed for smallpox but repositioned for Mpox under expanded access programs. The review endorses increased clinical trial capacities, amended regulatory approaches development of new classes of therapeutics as part of global Mpox response efforts. By combining pharmacological breakthroughs with public health readiness, global health communities can be better equipped to respond to Mpox and other possible orthopox outbreaks of the future."

Journal • Review • Infectious Disease

Mpox: transmission dynamics, treatment, and innovations. (PubMed, Ther Adv Infect Dis) - "Current treatment strategies primarily involve supportive care, with advanced therapeutics such as tecovirimat, cidofovir, and brincidofovir reserved for severe cases. The emergence of drug-resistant strains, the persistence of mpox in vulnerable populations, and the potential for future outbreaks necessitate sustained investment in research and public health infrastructure. By integrating innovative therapeutic approaches, effective preventive measures, and comprehensive outbreak management strategies, the global health community can better address the ongoing threat of mpox and prepare for future public health challenges."

Journal • Review • Infectious Disease

Small molecule direct-acting antivirals for treatment of mpox. (PubMed, Antiviral Res) - "Two small molecule DAAs (tecovirimat and brincidofovir), approved by national regulatory agencies (e.g., U.S. FDA) for the treatment of smallpox based on efficacy in animal models, are in Phase 3 trials for mpox as of mid-2025. Additional DAAs for treatment of mpox are in early development, highlighting a major gap in medical countermeasures. Compound characteristics are identified that should increase the probability of clinical success and durability for mpox and improve the likelihood that they will also be effective against other ORPVs."

Journal • Review • Infectious Disease

The Role of Epstein-Barr Virus in MS: Brincidofovir Inhibits Epstein-Barr Virus and Related Gammaherpesvirus in Human and Nonhuman Primate Cells (ECTRIMS 2025) - "These results suggest that BCV may be a useful antiviral for inhibiting EBV activity in MS patients and could alleviate inflammatory symptoms related to the reactivation of EBV in MS patients. These results also suggest a method for the selection of MS patients for possible inclusion in an anti-herpesvirus clinical trial due to their ability to reactivate EBV. The results of this work, and future clinical trials with BCV, will help further elucidate the pathogenesis of MS and how viral reactivation plays a role in disease etiology."

CNS Disorders • Epstein-Barr Virus Infections • Infectious Disease • Inflammation • Multiple Sclerosis

Tecovirimat Is Active against Various MPXV Strains, while Cidofovir, Brincidofovir, Trifluridine, and Gemcitabine Have No Detectable MPXV-Specific Antiviral Activity. (PubMed, Virus Res) - "The EC50 values of CDV, BCV, TFT, and dFdC against MPXVRTEC/A290V were 18, 1.8, 3.8, and 0.02 µM, respectively; however, the apparent anti-MPXV activity of these four agents was highly associated with their cytotoxicity as they were examined with qualitative and quantitative cell-based-morphometric assays. The data strongly show that none the four agents examined exhibited significant anti-MPXV activity and indicate that effective anti-MPXV agents active against wild-type and drug-resistant variants are urgently needed."

Journal

Mpox infection: A state-of-the-art overview of epidemiological, molecular, and clinical aspects following the 2024 public health emergency. (PubMed, J Infect Public Health) - "Although antiviral therapies, such as tecovirimat, cidofovir, and brincidofovir, remain effective, resistance is emerging among immunocompromised patients. Enhanced diagnostics, including WHO-approved rapid molecular tests, along with preventive strategies such as zoonotic risk reduction and equitable vaccine distribution, are critical. Continued genomic monitoring, enhanced global surveillance and interdisciplinary collaboration are essential to prevent future outbreaks and strengthen global preparedness."

Journal • Review • Infectious Disease

Mpox: disease manifestations and therapeutic development. (PubMed, J Virol) - "Current antiviral agents, including tecovirimat and brincidofovir, have demonstrated uncertain or disappointing efficacy in preclinical and clinical studies, underscoring the urgent need for further therapeutic development. We highlight the importance of innovative experimental models that can authentically replicate mpox disease manifestations and serve as robust platforms for therapeutic testing. Advancing these research efforts is critical for responding to the ongoing mpox emergency and for sustaining preparedness against future poxvirus epidemics."

Journal • Review • Infectious Disease

A Primer on Monkeypox Infection: An Emerging Threat to Global Public Health. (PubMed, Arch Med Res) - "However, antiviral medications, such as tecovirimat, brincidofovir, and cidofovir, as well as vaccinia immune globulin intravenous (VIGIV), are available as therapeutic options. This review emphasizes an overview of the history, etiology, epidemiology, structure, reservoirs, transmission, virus phylogeny, genome organization, clinical cases and symptoms, diagnosis, treatment, and prevention of mpox with recent updates. Therefore, a multifaceted approach is essential and includes improved surveillance, early diagnosis, isolation of index cases, immunization, and adoption of a "One Health" approach to prevent an mpox outbreak."

Journal • Review • Infectious Disease

Recent Progress in the Discovery of Anti-Mpox Agents. (PubMed, Med Res Rev) - "As no specific drugs for mpox are available, tecovirimat and brincidofovir for the treatment of smallpox infection are recommended to treat Mpox infection. The purpose of this review is to provide reference for further development of Mpox virus antiviral drugs by summarizing the resolving Mpox virus target protein structures and its mechanism of action, drugs with potential anti-Mpox virus activity, in vitro and in vivo inhibitory activity detection methods of anti-Mpox virus drugs, and the prevention of drug resistance. We hope to facilitate the discovery of antiviral drugs for MPXV by providing insights into the further development of appropriate anti-Mpox drugs based on currently available viral protein targets."

Journal • Review • Infectious Disease

Functional characteristics of plitidepsin as an antiviral treatment against monkeypox virus infection. (PubMed, Antiviral Res) - "Additionally, the limited efficacy of current smallpox antivirals, such as Tecovirimat and Brincidofovir, alongside growing concerns about the emergency of tecovirimat resistance mutants, underscores the need for new therapeutic options. These findings indicate plitidepsin as a promising candidate for mpox treatment. Further studies are needed to explore its potential as a standalone or combination therapy, supporting clinical evaluation for mpox treatment."

Journal • Infectious Disease • Oncology • EEF1A1

Concurrent Mpox and HSV-1 Proctitis in a Young Male With AIDS: A Case Report of Treatment Failure. (PubMed, Case Rep Infect Dis) - "Despite initial treatment with oral tecovirimat, acyclovir, and antiretrovirals, the patient's condition worsened, requiring readmission. The patient received intravenous tecovirimat, vaccinia immune globulin, and brincidofovir. Sigmoidoscopy revealed extensive rectal and sigmoid lesions that necessitated prolonged hospitalization and pain management. This case emphasizes the complexity of treating severe coinfections in immunocompromised patients, highlighting the need for a multidisciplinary approach and consideration of alternative therapies when standard treatment fails."

Journal • Human Immunodeficiency Virus • Infectious Disease • Pain • CD4

Brincidofovir in the era of Mpox. (PubMed, Expert Rev Anti Infect Ther) - "Brincidofovir is being evaluated in a randomized, double-blind, placebo-controlled trial under the Mpox Study in Africa (MOSA) protocol. Additional studies will assist in clarification of single vs. combination therapy, safety and populations most likely to benefit, as well as the management of treatment complications such as immune reconstitution inflammatory syndrome (IRIS)."

Clinical • Journal • Review • Infectious Disease

Identification of a novel ectromelia virus from rodent: Implications for use as an in vivo infection model for vaccine and antiviral research. (PubMed, Virol Sin) - "Furthermore, ECTV-C-Tan-GD01 exhibited utility in evaluating orthopoxvirus countermeasures: a single dose of vaccinia virus Tiantan (VTT) or non-replicating vaccinia virus Tiantan (NTV) conferred cross-protection, while tecovirimat (ST-246), cidofovir (CDV), and brincidofovir (initially CMX001) significantly reduced viral loads and pathology. This study establishes ECTV-C-Tan-GD01 as a dual-purpose resource for probing orthopoxvirus evolution and advancing therapeutic development."

Journal • Preclinical • Infectious Disease • SEMA7A

Mpox unveiled: Global epidemiology, treatment advances, and prevention strategies. (PubMed, One Health) - "Current treatments rely on therapies designed for smallpox, including antivirals like tecovirimat, brincidofovir, and cidofovir...Diagnosis is based on clinical presentation, confirmed by PCR testing of skin lesions or swabs, while HIV testing is recommended for adults with Mpox. Preventive measures, including isolation and the use of personal protective equipment (PPE), are essential, alongside the use of two available vaccines, Jynneos and ACAM2000, which show partial efficacy against Mpox."

Journal • Review • Human Immunodeficiency Virus • Infectious Disease