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65 Diseases | | 6 Products |
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- || Fintepla (low-dose fenfluramine) / UCB
New P1 trial: A Study to Assess Drug-drug Interaction of ZX008 in Healthy Male and Female Study Participants (clinicaltrials.gov) - Nov 7, 2024
P1, N=22, Not yet recruiting, Sponsor: UCB BIOSCIENCES, Inc.
- | Nayzilam (midazolam intranasal) / UCB
Retrospective data, Journal: Return to full baseline functionality after repeated intermittent use of midazolam nasal spray in patients with seizure clusters: Post hoc analysis of an open-label extension trial. (Pubmed Central) - Nov 4, 2024
P3
In almost all patients, RTFBF was observed within 24
- | Xiflam (tonabersat) / InflammX
Journal: Development and validation of stability-indicating HPLC method for assay of tonabersat in pharmaceutical formulations. (Pubmed Central) - Oct 29, 2024
No chemical degradation was observed in either formulation on storage. Thus, the method was sensitive, specific, and suitable for stability testing of tonabersat in pharmaceutical formulations.
- | ZEMBRACE SymTouch (sumatriptan succinate) / Tonix
Journal: Select autosomal dominant DFNA11 deafness mutations activate Myo7A in epithelial cells. (Pubmed Central) - Sep 30, 2024
Our findings reveal that Myo7A is regulated by specific IQ motifs within its lever arm, and that this regulation can function at least partially independent of its tail sequence. Importantly, we demonstrate that many of the DFNA11 deafness mutations reported in patients activate Myo7A targeting, providing a potential explanation for the autosomal dominant genetics of this form of deafness.
- | Vagifem (estradiol) / Novo Nordisk, Estrofem (estradiol oral) / Novo Nordisk, Divigel (estradiol) / Sawai Pharma
New P4 trial: Determining Dose Equivalence Between Oral and Transdermal Estrogen Treatment in Women With Turner Syndrome (clinicaltrials.gov) - Aug 8, 2024
P4, N=50, Recruiting, Sponsor: Aarhus University Hospital
- Xcopri (cenobamate) / SK Bio, Nayzilam (midazolam intranasal) / UCB
VAGAL NERVE STIMULATOR-INDUCED SLEEP DISORDERED BREATHING IN A PATIENT WITH REFRACTORY SEIZURES (Convention Center Exhibit Hall: Poster Area 5) - Jul 31, 2024 - Abstract #CHEST2024CHEST_5654;
This case report elucidates several novel aspects, informed by the provided data. VNS benefits in patients with refractory seizures but careful observation and monitoring is recommended with any patients with preexisting sleep disordered breathing.
- | Xiflam (tonabersat) / InflammX
Journal: Tonabersat suppresses priming/activation of the NOD-like receptor protein-3 (NLRP3) inflammasome and decreases renal tubular epithelial-to-macrophage crosstalk in a model of diabetic kidney disease. (Pubmed Central) - Jul 6, 2024
Using a model of DKD, we report for the first time that high glucose and inflammatory cytokines trigger aberrant Cx43 hemichannel activity, events that instigate NLRP3-induced inflammation in RPTECs and epithelial-to-macrophage crosstalk. Recapitulating observations previously reported in diabetic retinopathy, these data suggest that Cx43 hemichannel blockers (i.e., Tonabersat) may dampen multi-system damage observed in secondary complications of diabetes.
- || padsevonil (UCB0942) / UCB
PK/PD data, Preclinical, Journal: Overview of preclinical and clinical studies investigating pharmacokinetics and drug-drug interactions of padsevonil. (Pubmed Central) - Jun 27, 2024
P1
The studies presented helped in understanding padsevonil disposition and risks of DDIs, which would inform dosing and prescribing. https://www.clinicaltrials.gov/identifiers are NCT04131517, NCT03480243, NCT03695094, NCT04075409.
- | Xiflam (tonabersat) / InflammX
New P2 trial: Study of Xiflam (clinicaltrials.gov) - May 31, 2024
P2, N=15, Recruiting, Sponsor: Inflammx Therapeutics Inc
- Xiflam (tonabersat) / InflammX
Featured Presentation: Historical Perspective on Migraine Treatment* (Grand Ballroom 5-9) - Apr 29, 2024 - Abstract #AHS2024AHS_111;
The invention of a human migraine provocation model showed possibilities in the nitric oxide triggered cascade (not yet exploited) and strongly supported the development of CGRP antagonistic drugs. The same model has suggested many novel targets for drug development in migraine, most notably PACAP(validated) and potassium channels
- Nayzilam (midazolam intranasal) / UCB
Real-world use and associated health care resource utilization among patients with epilepsy receiving rescue midazolam nasal spray () - Apr 9, 2024 - Abstract #AMCP2024AMCP_117;
Compared with PwE who received 1 MDZ-NS, those who received 2 or more MDZ- NS fills may have had more severe epilepsy, as shown by a greater likelihood of having more than 2 prior/concomitant ASMs and higher epilepsy-related HCRU and costs. Overall, MDZ-NS was a minor contributor to pharmacy costs.
- | Xiflam (tonabersat) / InflammX
Preclinical, Journal: Integrating and optimizing tonabersat in standard glioblastoma therapy: A preclinical study. (Pubmed Central) - Mar 19, 2024
The study suggests tonabersat's potential to reduce tumor invasiveness, providing a new avenue for GB treatment. In conclusion, this preclinical investigation highlights tonabersat's potential as an effective adjuvant treatment for GB, and its established safety profile from clinical trials in migraine treatment presents a promising foundation for further exploration.
- | Journal: Drugs for menopausal symptoms. (Pubmed Central) - Mar 2, 2024
In conclusion, this preclinical investigation highlights tonabersat's potential as an effective adjuvant treatment for GB, and its established safety profile from clinical trials in migraine treatment presents a promising foundation for further exploration. No abstract available
- | Xiflam (tonabersat) / InflammX
Preclinical, Journal: Tonabersat Significantly Reduces Disease Progression in an Experimental Mouse Model of Multiple Sclerosis. (Pubmed Central) - Dec 27, 2023
MOG EAE mice showed clinical signs of MS, but MOG EAE mice treated with tonabersat retained behavior closer to normal. These data suggest that clinical trial phase IIb-ready tonabersat may merit further investigation as a promising candidate for MS treatment.
- ZEMBRACE SymTouch (sumatriptan succinate) / Tonix
Sumatriptan Induced Ischemic Colitis (Exhibit Hall) - Jul 29, 2023 - Abstract #ACG2023ACG_700;
For any individual on medication for migraine experiencing abdominal symptoms, a high index of suspicion needed for sumatriptan related ischemic colitis. Figure: Sumatriptan induced Ischemic Colitis - Gross, Microscopic and Imaging pictures
- | Xiflam (tonabersat) / InflammX
Enrollment open: AN: Evaluation of Tonabersat for DME (clinicaltrials.gov) - Jun 18, 2023
P2, N=128, Recruiting, Sponsor: Jaeb Center for Health Research
Figure: Sumatriptan induced Ischemic Colitis - Gross, Microscopic and Imaging pictures Not yet recruiting --> Recruiting
- | ONZETRA Xsail (sumatriptan intranasal) / Otsuka, Tosymra (intranasal sumatriptan) / Sawai Pharma, Neurelis, ZEMBRACE SymTouch (sumatriptan succinate) / Sawai Pharma
Journal: Comparison table: Triptans. (Pubmed Central) - Jun 6, 2023
Not yet recruiting --> Recruiting No abstract available
- | Journal: Drugs for migraine. (Pubmed Central) - Jun 6, 2023
No abstract available No abstract available
- |||| Diastat (diazepam rectal gel) / Bausch Health, Valtoco (diazepam intranasal) / Neurelis, Nayzilam (midazolam intranasal) / UCB
Compliance: I am certain this will improxve compliance with Epi first! Nayzilam and Valtoco have done that for seizures--vs using Diastat rectally. Least invasive med! (Twitter) - May 12, 2023
- || Nayzilam (midazolam intranasal) / UCB
DTC ad spotted in my feed today on Nayzilam (intranasal midaz). Concerned re: abuse potential. @drchadb are you hearing talk about pub health risks here? (Twitter) - Mar 24, 2023
- Valtoco (diazepam intranasal) / Neurelis, Nayzilam (midazolam intranasal) / UCB
Application Of Plan-Do-Study-Act Method In Seizure Rescue Medication Practices: A Single Pediatric Neurology Clinic Quality Improvement Study (Both in-person and online) - Mar 12, 2023 - Abstract #AAN2023AAN_1985;
There was an increase in prescribing trend of Valtoco and Nayzilam noted in the PDSA2 cohort...Conclusions Our study showed improvement of seizure rescue medication prescription practices in PDSA2 compared to PDSA1 cycles. The role of educational session for fellows, attending physicians and patient family regarding the timing of rescue medication, appropriate options, and correct dosing after the PDSA1 cycle was significant.Effective strategy of implementing systematic and rigorous standards in PDSA cycles sequentially could improve the prescription rate to 100% for convulsive seizures and consequently result in reduction in number of ED visits for breakthrough seizures and status epilepticus admissions.
- | Xiflam (tonabersat) / InflammX
Preclinical, Journal: Orally Delivered Connexin43 Hemichannel Blocker, Tonabersat, Inhibits Vascular Breakdown and Inflammasome Activation in a Mouse Model of Diabetic Retinopathy. (Pubmed Central) - Mar 2, 2023
However, the tonabersat treatment in the inflammatory NOD mice significantly reduced macrovascular abnormalities, hyperreflective foci, sub-retinal fluid accumulation, vascular leak, inflammation, and inflammasome activation. These findings suggest that tonabersat may be a safe and effective treatment for DR.
- | Xiflam (tonabersat) / InflammX
New P2 trial: AN: Evaluation of Tonabersat for DME (clinicaltrials.gov) - Feb 14, 2023
P2, N=128, Not yet recruiting, Sponsor: Jaeb Center for Health Research
- | ZEMBRACE SymTouch (sumatriptan succinate) / Sawai Pharma
Journal, Next-generation sequencing: Identification of novel missense mutation related with non-syndromic sensorineural deafness, DFNA11 in korean family by NGS. (Pubmed Central) - Jan 12, 2023
These findings suggest that tonabersat may be a safe and effective treatment for DR. This study is the first to report pathogenic mutations within MYO7A gene in Korean family and our data would facilitate diagnosing the primary cause of hereditary hearing loss in Korean.
- Diastat (diazepam rectal gel) / Bausch Health, Nayzilam (midazolam intranasal) / UCB
Baseline Characteristics and Antiseizure Medication Use of Patients with Epilepsy in the Year Prior to Initiating Midazolam Nasal Spray, Diazepam Rectal Gel, or Diazepam Nasal Spray (Hall B, Level 3) - Nov 29, 2022 - Abstract #AES2022AES_1133;
New users of MDZ-NS and DZP-NS showed generally similar baseline characteristics, but were older with more prior/concomitant ASMs vs the DZP-RG cohort. Baseline characteristics and prior/concomitant ASM use were influenced by approved patient age and BZP prescribing information, with adults most commonly receiving MDZ-NS.
- ||| Nayzilam (midazolam intranasal) / UCB
P3 data, Journal: Psychosocial outcomes of repeated treatment of seizure clusters with midazolam nasal spray: Results of a phase 3, open-label extension trial. (Pubmed Central) - Nov 22, 2022
P3
Limitations of this exploratory analysis include the open-label trial design and that these questionnaires have not been directly validated in epilepsy to identify clinically important changes; however, this does not mean these findings are not clinically meaningful. Overall, MDZ-NS is a socially acceptable drug device for outpatient treatment of seizure clusters that has the potential to improve quality of life and overall independence.
- | Xiflam (tonabersat) / InflammX
Journal: Blocking connexin-43 hemichannel-mediated ATP release reduces communication within and between tubular epithelial cells and medullary fibroblasts in a model of diabetic nephropathy. (Pubmed Central) - Nov 19, 2022
Tonabersat-sensitive hemichannel-mediated ATP release enhances TGFβ1-driven heterotypic cell-cell interaction and favours myofibroblast activation. The data supports the potential benefit of Cx43 inhibition in reducing tubulointerstitial fibrosis in late-stage diabetic nephropathy.
- || Nayzilam (midazolam intranasal) / UCB
Review, Journal: Midazolam nasal spray to treat intermittent, stereotypic episodes of frequent seizure activity: pharmacology and clinical role, a comprehensive review. (Pubmed Central) - Oct 21, 2022
Many studies have indicated quick cessation of seizures with Nayzilam compared to rectal diazepam, which has been the standard of care treatment. Nayzilam has been proven to be safe and effective for acute seizures in children, deeming it a revolutionary alternative in times where intravenous administration is not possible.
- | SSKI (potassium iodide) / Sawai Pharma
Journal: Dentin Discoloration and Pulpal Ion Concentrations Following Silver Diamine Fluoride and Potassium Iodide Treatment. (Pubmed Central) - Oct 18, 2022
The application of KI after SDF treatment could significantly reduce the dentin staining. The pulpal fluoride concentration in the groups using SDF-KI was lower compared to the ones using SDF only, whereas there was no difference observed in iodide and silver among the groups with treatment.
- | ZEMBRACE SymTouch (sumatriptan succinate) / Sawai Pharma
Biomarker, Journal, Next-generation sequencing: Novel compound heterozygous synonymous and missense variants in the MYO7A gene identified by next-generation sequencing in a Chinese family with nonsyndromic hearing loss. (Pubmed Central) - Sep 29, 2022
We reported a novel missense (c.5994G > T) and identified, for the first time, a novel pathogenic synonymous (c.2904G > A) variant within MYO7A in a patient with DFNB2. These findings enrich our understanding of the MYO7A variant spectrum of DFNB2 and can contribute to accurate genetic counseling and diagnosis of NSHL patients.
- | ZEMBRACE SymTouch (sumatriptan succinate) / Sawai Pharma
Journal, Heterogeneity: Clinical Heterogeneity Associated with MYO7A Variants Relies on Affected Domains. (Pubmed Central) - Apr 24, 2022
MYO7A variants are associated with DFNA11, a subtype of ADHL...Audiological differences among individuals correspond to specific domains which contain the variants. Therefore, appropriate rehabilitation is needed, particularly for patients with late-onset familial hearing loss.
- | Xiflam (tonabersat) / InflammX
Journal: Cx43 hemichannels contribute to astrocyte-mediated toxicity in sporadic and familial ALS. (Pubmed Central) - Apr 21, 2022
SignificanceOur results demonstrate that connexin 43 hemichannels are the conduits for amyotrophic lateral sclerosis (ALS) astrocyte-mediated motor neuron toxicity and disease spread, acting as a common mechanism that can target both familial ALS and sporadic ALS populations. Furthermore, our present work provides proof of principle that tonabersat, as a drug already studied in clinical trials for other indications, could serve as a potential ALS therapeutic.
- | Xiflam (tonabersat) / InflammX
Journal: Blocking connexin43 hemichannels prevents TGF-β2 upregulation and epithelial-mesenchymal transition in retinal pigment epithelial cells. (Pubmed Central) - Apr 5, 2022
All of these changes were significantly reduced with tonabersat treatment, which also prevented HG + Cyt-induced TGF-?2 release. In conclusion, Cx43 hemichannel block with tonabersat attenuated both TGF-?2 release and RPE EMT under disease-mimicking conditions, offering the potential to ameliorate the progression of EMT-associated retinal diseases.
- Fintepla (low-dose fenfluramine) / Zogenix
Serotonin and Focal Epilepsy: A Therapeutic Role? (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_3922;
P2
Design/ We performed a pilot trial of PRX-00023 (Nalutozan), a high affinity 5HT1A agonist , in patients with focal epilepsy that showed promise in trials for anxiety and depression (Investigator initiated IND 110522; NCT01281956)...Subjects on benzodiazepines, phenobarbital, or phenytoin were excluded... Our two small pilot trials do not provide any suggestion that 5HT1A agonists have a treatment role in focal epilepsy.
- || Nayzilam (midazolam intranasal) / UCB
Clinical, PK/PD data, Review, Journal: Clinical pharmacokinetic and pharmacodynamic profile of midazolam nasal spray. (Pubmed Central) - Feb 9, 2022
However, coadministration of MDZ-NS with moderate or strong CYP3A4 inhibitors should be avoided, and MDZ-NS should be used with caution when coadministered with mild CYP3A4 inhibitors, as these may result in prolonged MDZ effects owing to a decrease in plasma clearance. Taken together, the PK and PD properties of MDZ-NS, with a short t that translates into rapid CNS PD effects of sedation and psychomotor impairment, demonstrate rapid CNS penetration and onset of action, supporting its use for acute treatment of seizure clusters.
- | Divigel (estradiol) / Sawai Pharma
New P4 trial, Combination therapy, Monotherapy: Comparative Study to Evaluate the Efficacy and Safety of the Fixed-dose Combination of Estradiol / Dydrogesterone in Perimenopausal Women (clinicaltrials.gov) - Dec 13, 2021
P4, N=150, Recruiting, Sponsor: Scientific Research Institute of Public Health, Russian Federation
- Diastat (diazepam rectal gel) / Bausch Health, Valtoco (diazepam intranasal) / Neurelis, Nayzilam (midazolam intranasal) / UCB
Use of New Intranasal Benzodiazepines at a Typical Adult Epilepsy Center () - Dec 12, 2021 - Abstract #AES2021AES_1459;
Slightly more than half of the patients who were prescribed the medication had used it. The overall satisfaction of the two medications was comparable, with 81% and 79% of the patients who were either satisfied or very satisfied with the results.
- || Divigel (estradiol) / Sawai Pharma
Clinical, Journal: Thin endometrium problem in IVF programs. (Pubmed Central) - Oct 14, 2021
Estrogens improve the state of the endometrium and increase pregnancy rates in cases of thin endometrium in in vitro fertilization programs. The use of transdermal estrogens (Divigel, Orion Corporation, Finland) ensures an adequate increase in endometrial thickness and significantly lower estrogen doses.
- | Nayzilam (midazolam intranasal) / UCB
Preclinical, Journal: Evaluation of in vitro models for assessment of human intestinal metabolism in drug discovery. (Pubmed Central) - Sep 15, 2021
Significance Statement We found that cellular models of the human gut (permeabilized enterocytes and cryopreserved intestinal mucosa) offer an alternative to and potential advantage over intestinal microsomes in studies of drug metabolism, particularly for low clearance compounds and alternative pathways (e.g. sulfation, hydrolases, and flavin-containing monooxigenases). The predictivity of human f for common CYP- and UGT-substrates based on the Q model is still limited, however, and appropriate further evaluation is recommended.
- | tonabersat (USL260) / Sawai Pharma
Preclinical, Journal: Tonabersat Inhibits Connexin43 Hemichannel Opening and Inflammasome Activation in an In Vitro Retinal Epithelial Cell Model of Diabetic Retinopathy. (Pubmed Central) - Sep 15, 2021
It also prevented the release of proinflammatory cytokines IL-1β, VEGF, and IL-6. Tonabersat therefore has the potential to reduce inflammasome-mediated inflammation in diabetic retinopathy.
- || ZEMBRACE SymTouch (sumatriptan succinate) / Sawai Pharma
Clinical, Journal: Auditory Outcome after Cochlear Implantation in Children with DFNB7/11 Caused by Pathogenic Variants in TMC1 Gene. (Pubmed Central) - Jul 24, 2021
Tonabersat therefore has the potential to reduce inflammasome-mediated inflammation in diabetic retinopathy. Our results do not support the variable auditory outcome reported in the literature, which may be affected by several social and environmental factors and by the genetic background.
- || Review, Journal: Acute Treatment of Migraine: What has Changed in Pharmacotherapies? (Pubmed Central) - Jun 22, 2021
These 5-HT receptor agonists and CGRP receptor antagonists did not cause vasoconstriction, offering advantages over the current mainstay of specific acute migraine treatment. Overall, these new agents have expanded the available acute therapies for migraine treatment and will likely change the strategy with which we treat patients with migraine in the future.
- | tonabersat (USL260) / Sawai Pharma
Journal: Connexin43 hemichannel block inhibits NLRP3 inflammasome activation in a human retinal explant model of diabetic retinopathy. (Pubmed Central) - Apr 27, 2021
Furthermore, we showed that by blocking connexin43 hemichannels with the hemichannel modulator, tonabersat we were able to prevent NLRP3 inflammasome complex aggregation, Müller cell activation, as well as release of pro-inflammatory cytokines and VEGF. This further supports the possible use of connexin43 hemichannel blockers as potential new therapies for DR.
- | tonabersat (USL260) / Sawai Pharma
Journal: Differential Action of Connexin Hemichannel and Pannexin Channel Therapeutics for Potential Treatment of Retinal Diseases. (Pubmed Central) - Apr 20, 2021
The analysis showed that treatment with tonabersat alone was better than probenecid alone at restoring retinal function in the light-damaged retina model. The results assist in the interpretation of the differential action of connexin hemichannel and pannexin channel therapeutics for potential treatment of retinal diseases.
- | ZEMBRACE SymTouch (sumatriptan succinate) / Sawai Pharma
Journal: The p.R206C Mutation in MYO7A Leads to Autosomal Dominant Nonsyndromic Hearing Loss. (Pubmed Central) - Mar 31, 2021
The recurrent p.R206C variant in MYO7A is pathogenic and is likely in a mutation hot spot or due to a founder effect. Reports of such rare variants in multiple patients or families may facilitate exploitation of its pathogenicity.
- Nayzilam (midazolam intranasal) / UCB
[VIRTUAL] Return to Full Baseline Functionality After Repeated Intermittent Use of Midazolam Nasal Spray in Patients With Seizure Clusters: Post-Hoc Analysis of an Open-Label Extension Trial () - Mar 18, 2021 - Abstract #AAN2021AAN_893;
P3
The vast majority of patients returned to FBF within 24 h of intermittent administration of one or two MDZ-NS doses, with a similar median time to return to FBF. These return to FBF data further support the favorable profile of repeated intermittent MDZ-NS use in patients with SCs.
- Nayzilam (midazolam intranasal) / UCB
[VIRTUAL] Treatment Satisfaction, Anxiety Level, and Confidence About Traveling With Midazolam Nasal Spray in Patients With Seizure Clusters: Phase III, Open-Label Extension Trial () - Mar 18, 2021 - Abstract #AAN2021AAN_890;
P3
Patients perceived MDZ-NS as a favorable therapy: perceived effectiveness, side-effects, convenience, and global satisfaction showed improvement from Baseline. This is supported by improvement in anxiety level and consistently high confidence about traveling with MDZ-NS in patients and caregivers over repeated intermittent use.
- | tonabersat (USL260) / Sawai Pharma
Preclinical, Journal: Connexin Hemichannel Block Using Orally Delivered Tonabersat Improves Outcomes in Animal Models of Retinal Disease. (Pubmed Central) - Feb 5, 2021
Inflammation was also reduced and retinal electrical function restored. Tonabersat regulates assembly of the inflammasome (NLRP3) through Connexin43 hemichannel block, with the potential to reduce inflammation, restore vascular integrity and improve anatomical along with some functional outcomes in retinal disease.
- | Botox (onabotulinumtoxin A) / GSK, AbbVie
Journal: Drugs for Migraine. (Pubmed Central) - Jan 23, 2021
Tonabersat regulates assembly of the inflammasome (NLRP3) through Connexin43 hemichannel block, with the potential to reduce inflammation, restore vascular integrity and improve anatomical along with some functional outcomes in retinal disease. No abstract available
- | PRX-5 / Sawai Pharma
Journal: Peroxiredoxins are involved in the pathogenesis of multiple sclerosis and neuromyelitis optica spectrum disorder. (Pubmed Central) - Jan 21, 2021
Spinal cords from EAE mice showed remarkable PRX5 staining, especially in CD45+ infiltrating cells. In conclusion, PRX5 and PRX6 may play a role in the pathogeneses of MS and NMOSD.