idebenone / Generic mfg. - LARVOL DELTA

A mitochondria-targeted idebenone derivative W1a mitigates cerebral ischemia-reperfusion injury by preserving mitochondrial integrity and suppressing neuroinflammation. (PubMed, Bioorg Chem) - "To overcome these challenges, we synthesized W1a, a novel IDE derivative conjugated to the mitochondria-targeting peptide SS-20, designed to enhance mitochondrial localization and pharmacodynamic activity. Moreover, W1a inhibited microglial and astrocytic activation, further supporting its anti-inflammatory efficacy. Collectively, our findings establish W1a as a potent neuroprotective agent that targets mitochondrial dysfunction and neuroinflammation drivers of CIRI pathology, offering a promising therapeutic strategy for ischemic stroke."

Journal • Cardiovascular • Inflammation • Ischemic stroke • Metabolic Disorders • Reperfusion Injury

Polymer-lipid hybrid nanoparticles incorporating green coffee extract: enhancing treatment for hepatic steatosis and fibrosis through metabolomic insights. (PubMed, RSC Adv) - "The encapsulation efficiency (55%) and release kinetics of GCE-PLNs were optimized for sustained bioavailability over 24 h. Metabolomic and chemoinformatic analyses identified 1942 metabolites, including GCE-derived bioactive compounds with structural similarities to therapeutic agents like mesalamine, idebenone, and cysteamine, highlighting potential antifibrotic and hepatoprotective effects. In vitro assays, using HepG2 cells, demonstrated that GCE-PLNs modulate the expression of steatosis- and fibrosis-related genes, such as PLIN1, CPT1A, ACTA2, and COL1A1, in HepG2 and HHSC-N cellular models, suggesting reduced lipid accumulation and fibrotic activity. These findings suggest that GCE-PLNs may represent a promising nanotechnological strategy for the treatment of MASLD."

Journal • Fibrosis • Hepatology • Immunology • Metabolic Disorders • Metabolic Dysfunction-Associated Steatohepatitis • Metabolic Dysfunction-Associated Steatotic Liver Disease • ACTA2 • COL1A1 • CPT1A

Advanced therapies for inherited optic neuropathies. (PubMed, Eye (Lond)) - "Clinical trials for LHON have demonstrated the efficacy of idebenone, an oral neuroprotective agent, and gene replacement therapy using allotopic gene expression...In this review, we have critically appraised a range of therapeutic strategies, including gene editing and stem cell-based optic nerve regeneration, with a discussion of the barriers to translation. Future studies focussing on understanding genetic heterogeneity, disease variability and optimising patient selection for clinical trials are essential to improve patient management and fast track transformative therapies for IONs."

Journal • Review • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis

A Clinical Study of the Efficacy of Idebenone in the Treatment of iRBD Into Synucleinopathies (clinicaltrials.gov) - P2 | N=28 | Active, not recruiting | Sponsor: Ruijin Hospital | Recruiting ➔ Active, not recruiting | N=142 ➔ 28

Enrollment change • Enrollment closed • Mental Retardation • Psychiatry • REM Sleep Behavior Disorder

IT TAKES TWO TO TANGO: potential novel therapies for autosomal dominant optic atrophy. (PubMed, Front Ophthalmol (Lausanne)) - "In this paper, we review the current and future investigational therapies for ADOA, including the use of intravitreal injections of antisense oligonucleotides through Targeted Augmentation of Nuclear Gene Output (TANGO), CRISPR-based therapy, genetic editing, gene replacement approaches, and idebenone, a small-molecule mitochondrial modulator...Increased awareness of therapeutics for ADOA will allow for patient counseling regarding treatment access via clinical trials and for underscoring the importance of genetically testing family members, who may be incidentally identified with ADOA in a timely manner for newly available therapies. While patients with ADOA typically have poor visual prognoses, there are increasing promising therapies with the potential for preserving and improving visual function."

Journal • Review • Gene Therapies • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain

Three-dimensional multiscale and multimodal X‑ray neuroimaging: an advanced technique to quantify potential neuroprotective strategies for retinal ganglion cell degeneration in an experimental mouse model of glaucoma (Neuroscience 2025) - "Here we used this technique, along immunohistochemistry, to evaluate the potential neuroprotective effects of JNJ16259685, a negative allosteric modulator of mGlu1 metabotropic glutamate receptors, and of Idebenone, a synthetic analogue of coenzyme Q10 with antioxidant activity, in the DBA/2J mice, a genetic experimental model of glaucoma associated with neurodegeneration...These data are also used to quantitatively characterize the retina layers and the effects of drug treatments at different time points. Results will be presented."

Metastases • Preclinical • Alzheimer's Disease • CNS Disorders

Idebenone alleviates rotenone-induced mitochondrial dysfunction through mitophagy and mitochondrial biogenesis in muscle cells. (PubMed, Cell Signal) - "Remarkably, treatment with idebenone reversed these effects, and pharmacological inhibition of PGC1A abolished the protective effects of idebenone on mitochondrial biogenesis and function. Our findings suggest that idebenone ameliorates rotenone-induced apoptosis, oxidative stress, and mitochondrial damage in C2C12 cells, supporting its potential therapeutic role in the treatment of skeletal muscle atrophy."

Journal • Metabolic Disorders • Muscular Atrophy • PINK1 • PPARGC1A • SQSTM1

Efficacy and safety of idebenone tablets in patients with post-stroke cognitive impairment: a real-world study. (PubMed, Front Neurol) - "However, the observational, single-arm design without a control group means that observed improvements cannot be definitively attributed to the intervention alone. Further randomized controlled trials are warranted to confirm these findings and optimize treatment protocols."

Journal • Real-world evidence • Alzheimer's Disease • Cardiovascular • Cognitive Disorders • Hypertension • Ischemic stroke

Two journeys, one diagnosis: exploring the clinical outcomes of twins with congenital myopathy. (PubMed, BMC Neurol) - "Clinical exome sequencing confirmed the RYR1 mutation, and an antioxidant supplement, idebenone, was introduced. Despite sharing the same variant, the twins presented discordant clinical phenotypes with varying degrees of muscle impairment and other complications. This case highlights the critical role of epigenetic in modulating disease expression, suggesting personalized therapeutic strategies are paramount even in siblings sharing the same homozygous variant."

Clinical data • Journal • CNS Disorders • Myositis • RYR1

Neuroprotective effects of idebenone combined with borneol via the dopamine signaling pathway in a transgenic zebrafish model of Parkinson's disease (PubMed, Sheng Wu Yi Xue Gong Cheng Xue Za Zhi) - "The combination showed a better therapeutic effect compared to the IDE monotherapy group. This study reveals the protective mechanism of IDE combined with BO on the central nervous system for the first time, which provides an important experimental basis and theoretical reference for clinical combination strategy in PD treatment."

Journal • Behavior Disorders • CNS Disorders • Mental Retardation • Movement Disorders • Parkinson's Disease • Psychiatry

REPURPOSING MITOCHONDRIA PROTECTIVE TARGETS FOR ADJUVANT THERAPY IN INFLAMMATORY BOWEL DISEASES (UEGW 2025) - "Aims & Cellular toxicity of the drugs Acipimox, AICAR, ALCAR, Bezafibrate, Epi-743, Idebenone, Metformin, SS-31 and TUDCA was assessed in the intestinal epithelial cell line Mode-K and murine wild-type (WT) small intestinal (SI) organoids. Selected drugs targeting mitochondrial functions restored seeding efficiency, differentiation, and stemness of intestinal organoids from inflamed tissue sections of TNFΔARE mice, TNFΔARE pigs, and CD patients. These findings clearly support the potential of metabolic drug repurposing for adjuvant IBD therapy and indicate putative risks for Metformin."

Clinical • Crohn's disease • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammation • Inflammatory Bowel Disease • Metabolic Disorders • IL10

OPA1 MUTATION PRESENTING AS ETHAMBUTOL-INDUCED OPTIC NEUROPATHY (WCN 2025) - "Patient was continued on idebenone 900 mg/day... Although the cause of EtON is uncertain, it has long been hypothesized that mitochondria play a vital role in its etiology. Genetic evaluation before starting ethambutol may identify some of the at-risk individuals (not currently feasible). In the presence of atypical features among patients with suspected EtON, underlying mitochondrial etiology should be evaluated for and prompt treatment should be initiated."

CNS Disorders • Pain

Genetic and Physical Study of Childhood Nerve and Muscle Disorders (clinicaltrials.gov) - P=N/A | N=9300 | Recruiting | Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) | N=5650 ➔ 9300

Enrollment change • Genetic Disorders • Muscular Dystrophy • Myositis

Establishment of human Leber's hereditary optic neuropathy model using iPSC-derived retinal organoids. (PubMed, Front Cell Neurosci) - "Finally, treatment with idebenone, the only approved therapeutic agent for LHON, improved RGC numbers in LHON-RO...Furthermore, a comprehensive analysis of the LHON-RO model revealed impaired mitophagy, suggesting novel therapeutic targets for LHON. Thus, the LHON-RO model offers a valuable platform for studying LHON pathogenesis and evaluating treatments."

Journal • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain

Successful treatment of congenital sideroblastic anemia with low-dose decitabine in a patient with NDUFB11 gene mutation (c.276_278del): A case report. (PubMed, Curr Res Transl Med) - "We proposed that epigenetic factors might play a role in pathogenesis in patients with c.276_278del (p.F93del) mutation in NDUFB11 gene and low dose decitabine may be a novel treatment in CSA patients with c.276_278del (p.F93del) mutation in NDUFB11 gene."

Journal • Anemia • Cardiomyopathy • Cardiovascular • CNS Disorders • Hematological Disorders • Metabolic Disorders • Ophthalmology • Orthopedics • NDUFB1

First Case in Lithuania of an Autosomal Recessive Mutation in the DNAJC30 Gene as a Cause of Leber's Hereditary Optic Neuropathy. (PubMed, Genes (Basel)) - "This case highlights the importance of considering arLHON as a possible diagnosis for patients with optic neuropathy, because the phenotype of arLHON appears to be identical to that of mtLHON and cannot be distinguished by clinicians."

Journal • Infectious Disease • Inflammation • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain • Vasculitis • DNAJB1

Comparative Antioxidant Protection of Cochlear Hair Cells from Ototoxins. (PubMed, Molecules) - "Neither mitochonic acid nor SQ-29548 was protective against cisplatin. The results indicate that seratrodast and idebenone provide HC protection from gentamicin and cisplatin due to their free radical scavenging properties, but protection from cisplatin was less effective, presumably due to its different mechanism of ototoxicity."

Clinical • Journal • Oncology

ROAR-DIGAP: A Widely Inclusive, Largely Virtual Pilot Trial Utilizing DIGAP (Deep Integrated Genomics Analysis Platform) To Personalize Treatments (clinicaltrials.gov) - P2 | N=50 | Completed | Sponsor: Duke University | Active, not recruiting ➔ Completed

Trial completion • Amyotrophic Lateral Sclerosis • CNS Disorders

Oxidative Stress and Antioxidant Therapies in Friedreich's Ataxia. (PubMed, Cells) - "Among the antioxidant treatments tested in FRDA patients, only omaveloxolone and, to a lesser extent, idebenone (particularly for cardiac hypertrophy) have shown some efficacy. Therefore, these drugs may be useful in treating FRDA and are likely candidates for future clinical trials. Future studies investigating oxidative stress and antioxidant therapies in FRDA should adopt a prospective, multicenter, long-term, double-blind design."

Journal • Review • Ataxia • Friedreich ataxia • Metabolic Disorders • Movement Disorders

Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis (clinicaltrials.gov) - P1/2 | N=45 | Completed | Sponsor: Stanford University | Active, not recruiting ➔ Completed

Trial completion • Hepatology • Immunology • Metabolic Dysfunction-Associated Steatohepatitis

Infographic: therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: the LEROS nonrandomized controlled trial. (PubMed, Eye (Lond)) - No abstract available

Journal • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Ocular Inflammation • Ophthalmology • Optic Neuritis

Outcomes of idebenone therapy for Leber hereditary optic neuropathy in a cohort of patients from Wales. (PubMed, Eye (Lond)) - "The present cohort demonstrates evidence of CRR in a high proportion of patients reaching 27 months of treatment. Further follow-up and a larger cohort of patients will provide further insight into the real-world efficacy of idebenone in LHON."

Journal • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Ocular Inflammation • Ophthalmology • Optic Neuritis

Idebenone: Clinical Potential Beyond Neurological Diseases. (PubMed, Drug Des Devel Ther) - "Interestingly, emerging evidence suggests that idebenone may also have beneficial effects beyond neurological conditions through disrupting mitochondrial membrane potential, inducing mitochondrial apoptosis, promoting mitophagy attenuating ferroptosis, reducing reactive oxygen species and lipid peroxidation, etc. This study aims to comprehensively review the clinical potential of idebenone in various fields, including cancers (such as breast cancer, melanoma, glioblastoma, neuroblastoma, hepatocellular carcinoma, prostatic carcinoma and pancreatic carcinoma), cardiovascular diseases (including atherosclerosis, hypertension, myocardial infarction and heart failure), diabetes mellitus, liver diseases, urogenital diseases, sepsis, and other diseases. The findings highlight the potential of idebenone as a promising therapeutic option for the prevention and management of these condition, which need to be validated in more clinical trials."

Journal • Review • Atherosclerosis • Brain Cancer • Breast Cancer • Cardiovascular • CNS Disorders • Congestive Heart Failure • Diabetes • Glioblastoma • Heart Failure • Hepatocellular Cancer • Hepatology • Hypertension • Infectious Disease • Melanoma • Metabolic Disorders • Myocardial Infarction • Neuroblastoma • Oncology • Pancreatic Cancer • Prostate Cancer • Septic Shock • Solid Tumor

Mitochondrial Diseases: Molecular Pathogenesis and Therapeutic Advances. (PubMed, MedComm (2020)) - "In parallel, metabolic modulators (e.g., coenzyme Q10, idebenone, EPI-743) aim to restore bioenergetics, and mitochondrial replacement technologies and transplantation are being explored. Addressing these issues through multidisciplinary research and clinical translation holds promise for transforming mitochondrial disease management and improving patient outcomes. By bridging the understanding of mitochondrial dysfunction with advanced therapeutic interventions, this review aims to shed light on effective solutions for managing these complex disorders."

Journal • Review • Gene Therapies • Genetic Disorders • Metabolic Disorders • Transplantation

Metformin may alter the course of Leber's hereditary optic neuropathy: a case report. (PubMed, Front Med (Lausanne)) - "In this study, we report the case of a 48-year-old woman diagnosed with LHON (m.11778G>A/MT-ND4) and type 2 diabetes mellitus who experienced visual field improvement following metformin treatment after 26 months of progressive vision loss unresponsive to idebenone, nicotinamide adenine dinucleotide (NAD+), and hormone replacement therapy (HRT). Our findings offer an intriguing perspective on LHON management but require more investigations, particularly on the molecular effects of metformin on the mitochondrial function in LHON patients."

Journal • Diabetes • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain • Type 2 Diabetes Mellitus