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6 Diseases |  |
3 Trials |
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3 Trials |  |
392 News |  |
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- || Review, Journal: Update on Antiseizure Medications 2025. (Pubmed Central) - Feb 3, 2025
Since the most recent version of this article was published, one new antiseizure medication, ganaxolone, has been approved by the US Food and Drug Administration (FDA), and the indications of some approved medications were expanded...These agents include lamotrigine, oxcarbazepine, levetiracetam, topiramate, zonisamide, and lacosamide...Lacosamide, pregabalin, and eslicarbazepine have undergone successful trials of conversion to monotherapy for focal epilepsy...Knowledge of antiseizure medication pharmacokinetics, efficacy, and tolerability profiles facilitates the choice of appropriate antiseizure medication therapy for patients with epilepsy. Rational antiseizure medication combinations should avoid antiseizure medications with unfavorable pharmacokinetic interactions or pharmacodynamic interactions related to mechanism of action.
- || Ztalmy (ganaxolone oral) / Marinus
Review, Journal: Ganaxolone: A Review in Epileptic Seizures Associated with Cyclin-Dependent Kinase-Like 5 Deficiency Disorder. (Pubmed Central) - Jan 28, 2025
While somnolence and sedation are related to the dose of ganaxolone, they appear early in treatment and may decrease with continued therapy. Thus, although current evidence is somewhat limited,
- | Ztalmy (ganaxolone oral) / Marinus, Xcopri (cenobamate) / SK Bio, Fintepla (low-dose fenfluramine) / UCB
Journal: Antiseizure Medications: Advancements, Challenges, and Prospects in Drug Development. (Pubmed Central) - Jan 27, 2025
The review also discusses the development of new potential ASMs, including modifying existing ASMs to improve efficacy and tolerability. Furthermore, we expound on the modulation of ?- aminobutyric acid type A receptor (GABAAR) as a strategy for the treatment of epilepsy and the identification of a GABAAR agonist, isoguvacine, as a potential ASM.
- | Ztalmy (ganaxolone oral) / Marinus
Journal: LPM682000012, a Synthetic Neuroactive Steroid That Ameliorates Epileptic Seizures by Downregulating the Serpina3n/NF-?B Signaling Pathway. (Pubmed Central) - Nov 27, 2024
The dose-dependent sedative effects of LPM682000012 and Ganaxolone in normal rats were evaluated, which revealed that they both dose-dependently alleviated acute epileptic seizure in the pentylenetetrazol (PTZ)-mediated seizure model...In summary, this report demonstrated that the novel neurosteroid GABAA PAM LPM682000012 activated the synaptic and extra-synaptic GABAA receptors and alleviated KA-induced neuronal loss and synaptic remodeling, potentially by down-regulating the Serpina3n/NF-?B signaling pathways. The results provide evidence that LPM682000012 is a potential anti-seizure pharmacotherapy candidate for epilepsy and warrants further research.
- Ztalmy (ganaxolone oral) / Marinus
Neurosteroid Protection of Nerve Agent-induced Long-term Seizures and Neurological Dysfunction in a Pediatric Rat Model (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_2312;
Ganaxolone was administered 40 minutes post-GD exposure, standalone, or with standard anticonvulsant midazolam. These results demonstrate the neuroprotective effect of the neurosteroid GX in the GD exposure model, which has implications for mitigating epileptic seizures and long-term neuropsychiatric dysfunction associated with nerve agent exposure.
- Ztalmy (ganaxolone oral) / Marinus
Long-term Safety and Efficacy of Ganaxolone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex During the Extension Phase of an Open-Label Phase 2 Study (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_2269;
P2
Ganaxolone was generally well-tolerated with a safety profile consistent with previous clinical studies. A Phase 3, placebo-controlled study of ganaxolone for the treatment of seizures associated with TSC is ongoing.
- Ztalmy (ganaxolone oral) / Marinus
Ganaxolone in Patients with CDKL5 Deficiency Disorder Under Age 2: A Single-center Experience (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_2168;
Some of the patients entered honeymoon after initiating treatment with ganaxolone, nevertheless the reason for honeymoon in patients with CDD is not well understood. Early genetic diagnosis allows for early initiation of the medication.
- Ztalmy (ganaxolone oral) / Marinus
A Phase I, Single Dose Study of Ganaxolone Pharmacokinetics in Adult Subjects with Normal and Impaired Hepatic Function (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_2024;
Exposure to ganaxolone increased with severity of hepatic impairment. Dose adjustment is required in patients with severe hepatic impairment.
- Ztalmy (ganaxolone oral) / Marinus
Long-term Neuroprotective Effects of Neurosteroid Therapy Following Acute Soman Exposure-induced Status Epilepticus in Pediatric Rats (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_1784;
Postnatal day 21 male rats (P21) were subjected to GD and treated with GX in addition to midazolam at 40 minutes post-exposure... These results demonstrate the long-term neuroprotective effects of GX in mitigating neurologic dysfunction, neurodegeneration, and neuroinflammation in pediatric GD model.
- Ztalmy (ganaxolone oral) / Marinus
Effect of Ganaxolone in Patients with Features of Lennox-gastaut Syndrome: Post-hoc Analysis from a Clinical Trial in CDKL5 Deficiency Disorder (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_1618;
Safety and tolerability were comparable between groups. Although there are limitations in diagnostic confirmation, the results suggest that ganaxolone could be safe and effective in treating patients with LGS.
- Ztalmy (ganaxolone oral) / Marinus
Case Report: Good outcome in Adult Patient with Super-refractory Status Epilepticus treated with Multimodal Therapy and Emergency Use Ganaxolone (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_1508;
We describe a patient with SRSE who achieved a good outcome by hospital discharge despite early poor prognostic indicators. Multidisciplinary coordination and early aggressive management of SRSE through multimodal therapy - including antiseizure medications, immunotherapy, and eIND ganaxolone
- Zulresso (brexanolone) / Sage Therapeutics, Ztalmy (ganaxolone oral) / Marinus
Neuroactive Steroids Induce Sex Specific Effects on Intracellular Signaling via Activation of Membrane Progesterone Receptors (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_1272;
Additionally, the high potency of NAS for mPRs is at concentrations similar to endogenous ALLO in the brain and is below demonstrated plasma levels of FDA approved NAS treatments. Therefore, this work emphasizes the importance of understanding mPR signaling in the brain and the ability of mPRs to modulate neuronal inhibition to better develop future novel therapies.
- Ztalmy (ganaxolone oral) / Marinus
A Study of Ganaxolone in Seizures Associated With Tuberous Sclerosis Complex: A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study (TrustTSC) (South Hall H - Level 1 - LACC) - Nov 26, 2024 - Abstract #AES2024AES_868;
P3
Therefore, this work emphasizes the importance of understanding mPR signaling in the brain and the ability of mPRs to modulate neuronal inhibition to better develop future novel therapies. This global, placebo-controlled trial is designed to generate rigorous clinical evidence on the safety and efficacy of ganaxolone for the treatment of seizures associated with TSC.
- ||||| Ztalmy (ganaxolone oral) / Marinus
Trial completion: TrustTSC: Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy (clinicaltrials.gov) - Nov 5, 2024
P3, N=128, Completed, Sponsor: Marinus Pharmaceuticals
This global, placebo-controlled trial is designed to generate rigorous clinical evidence on the safety and efficacy of ganaxolone for the treatment of seizures associated with TSC. Active, not recruiting --> Completed
- | Ztalmy (ganaxolone oral) / Marinus
Journal: Ganaxolone Therapy After Preterm Birth Restores Cerebellar Oligodendrocyte Maturation and Myelination in Guinea Pigs. (Pubmed Central) - Oct 8, 2024
Importantly, we showed that neurosteroid replacement therapy returns cerebellar development and behavior toward a term-like phenotype. Therefore, ganaxolone may reduce the vulnerability of the cerebellum to postnatal challenges arising from preterm birth.
- | Ztalmy (ganaxolone oral) / Marinus
Trial completion date, Trial primary completion date: Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old (clinicaltrials.gov) - Sep 20, 2024
P3, N=20, Not yet recruiting, Sponsor: Marinus Pharmaceuticals
Therefore, ganaxolone may reduce the vulnerability of the cerebellum to postnatal challenges arising from preterm birth. Trial completion date: Dec 2024 --> Mar 2027 | Trial primary completion date: Dec 2024 --> Dec 2026
- || Ztalmy (ganaxolone oral) / Marinus
Review, Journal: An overview of ganaxolone as a treatment for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder. (Pubmed Central) - Sep 20, 2024
Ganaxolone is a neuroactive 3?-methylated synthetic analogue of the potent agonist of gamma-aminobutyric acid type A receptors, allopregnanolone. Ganaxolone is the only drug that has been studied in a robust randomized controlled trial and been proven to be effective in this population.
- || Ztalmy (ganaxolone oral) / Marinus, Zurzuvae (zuranolone) / Sage Therapeutics, Biogen, sepranolone (UC1010) / Asarina Pharma
Review, Journal: The Emerging Role of Neurosteroids: Novel Drugs Brexanalone, Sepranolone, Zuranolone, and Ganaxolone in Mood and Neurological Disorders. (Pubmed Central) - Sep 2, 2024
Ganaxolone is the only drug that has been studied in a robust randomized controlled trial and been proven to be effective in this population. This
- Ztalmy (ganaxolone oral) / Marinus
Positive allosteric modulators of GABAAreceptors expressed in oligodendroglial cells promote their survival in vitro (MCP Hall A) - Aug 22, 2024 - Abstract #Neuroscience2024Neuroscience_12321;
Here, in OLs maintained in vitro the GABAAR activity was potentiated using ganaxolone (GX) and N-butyl-?-carboline-3-carboxylate (?-CCB) two allosteric positive modulators of the receptor and studied their effects on cell survival to obtain information related with the mechanisms involved...Thus, our results indicated that both Gx and ?-CCB promoted the survival of oligodendroglial cells through the activation of the GABAAR. This would explain at least in part, the positive effects that GABAAR positive modulators such as ?-CCB have on myelination process observed in vivo.
- | Ztalmy (ganaxolone oral) / Marinus
Journal: Ganaxolone (Ztalmy) for CDKL5 deficiency disorder. (Pubmed Central) - Aug 13, 2024
This would explain at least in part, the positive effects that GABAAR positive modulators such as ?-CCB have on myelination process observed in vivo. No abstract available
- ||| Ztalmy (ganaxolone oral) / Marinus
Journal, Adverse events: Caregiver Perspective of Benefits and Side Effects of Anti-Seizure Medications in CDKL5 Deficiency Disorder from an International Database. (Pubmed Central) - Aug 10, 2024
Our findings underscore the necessity of ongoing research to optimise treatment strategies, considering both the efficacy of seizure control and the potential for adverse effects. The study also points to the need for future investigations into the therapeutic potential of emerging treatments such as ganaxolone and the unresolved efficacy of cannabis products in seizure management.
- || Ztalmy (ganaxolone oral) / Marinus
Review, Journal: Epilepsy with PCDH19 mutation: polypharmacy as a consequence of the complexity and diversity of pathogenesis mechanisms (Pubmed Central) - Aug 8, 2024
The need for polypharmacy (in most cases) confirms the diversity of mechanisms involved in the pathogenesis of the disease and makes the prospects for the development of effective and rational treatment regimens very vague. Cautious optimism is caused only by attempts at relatively specific treatment with ganaxolone.
- || Clinical, Journal: Comparison Table: Some oral antiseizure medications. (Pubmed Central) - Jul 29, 2024
Cautious optimism is caused only by attempts at relatively specific treatment with ganaxolone. No abstract available
- || Review, Journal: Drugs for epilepsy. (Pubmed Central) - Jul 29, 2024
No abstract available No abstract available
- | Ztalmy (ganaxolone oral) / Marinus
Journal: Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trial. (Pubmed Central) - Jul 28, 2024
The total change in QOL score for children in the ganaxolone group was 2.6 points (95%CI -1.74,7.02) higher (improved) than in the placebo group but without statistical significance. Along with better seizure control, children who received ganaxolone had improved behavioural scores in select domains compared to placebo.
- || Zulresso (brexanolone) / Sage Therapeutics, Ztalmy (ganaxolone oral) / Marinus, Zurzuvae (zuranolone) / Sage Therapeutics, Biogen
Review, Journal: Understanding and treating postpartum depression: a narrative review. (Pubmed Central) - Jun 28, 2024
Newer neurosteroids such as ganaxolone, valaxanolone, and lysaxanolone are currently under development, but also esketamine and psychedelics are promising potential treatments...PPD is therefore increasingly understood as, at least partially, independent from major depressive disorder. Specific and individualized treatments including pharmacological and non-pharmacological therapies are progressively being introduced in the routine clinical practice.
- | Journal: Conventional and novel anti-seizure medications reveal a particular role for GABAA in a North Sea progressive myoclonus Epilepsy Drosophila model. (Pubmed Central) - Jun 6, 2024
This suggests that GABAA could be a potentially important target in the treatment of NS-PME. Consequently, these findings add rationale to the exploration of the clinical effect of ganaxolone in NS-PME and other progressive myoclonus epilepsies.
- |||| Ztalmy (ganaxolone oral) / Marinus
Enrollment closed, Trial completion date, Trial primary completion date: TrustTSC: Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy (clinicaltrials.gov) - May 13, 2024
P3, N=128, Active, not recruiting, Sponsor: Marinus Pharmaceuticals
Consequently, these findings add rationale to the exploration of the clinical effect of ganaxolone in NS-PME and other progressive myoclonus epilepsies. Recruiting --> Active, not recruiting | Trial completion date: Feb 2025 --> Oct 2024 | Trial primary completion date: Jan 2024 --> Oct 2024
- || Ztalmy (ganaxolone oral) / Marinus
Retrospective data, Review, Journal: A systematic review and meta-analysis on efficacy and safety of Ganaxolone in epilepsy. (Pubmed Central) - Apr 12, 2024
Ganaxolone presents itself as a viable therapeutic option for refractory epilepsy, showing efficacy in reducing seizure frequency and exhibited a favorable safety profile. CRD42023434883.
- || Review, Journal: A Neuroanatomic and Pathophysiologic Framework for Novel Pharmacological Approaches to the Treatment of Post-traumatic Stress Disorder. (Pubmed Central) - Apr 1, 2024
Indeed, investigations and drug development are proceeding in a number of these alternative, non-serotonergic pathways in an effort to improve the management of PTSD. In this manuscript, the authors introduce novel and emerging treatments for PTSD, including drugs in various stages of development and clinical testing (BI 1358894, BNC-210, PRAX-114, JZP-150, LU AG06466, NYV-783, PH-94B, SRX246, TNX-102), established agents and known compounds being investigated for their utility in PTSD (brexpiprazole, cannabidiol, doxasoin, ganaxolone, intranasal neuropeptide Y, intranasal oxytocin, tianeptine oxalate, verucerfont), and emerging psychedelic interventions (ketamine, MDMA-assisted psychotherapy, psilocybin-assisted psychotherapy), with an aim to examine and integrate these agents into the underlying pathophysiological frameworks of trauma-related disorders.
- | Ztalmy (ganaxolone oral) / Marinus, Belviq (lorcaserin) / Eisai
Journal: Using artificial intelligence to identify drugs for repurposing to treat l-DOPA-induced dyskinesia. (Pubmed Central) - Mar 13, 2024
In conclusion, although our data do not support the repurposing of lorcaserin, acamprosate or ganaxolone per se for LID, we demonstrate value of an in silico approach to identify candidate molecules which, in combination with an in vivo screen, can facilitate clinical development decisions. The present study adds to a growing literature in support of this paradigm shifting approach in the repurposing pipeline.
- Ztalmy (ganaxolone oral) / Marinus
Effect of Concomitant Antiseizure Medications on the Safety and Efficacy of Ganaxolone for the Treatment of Seizures Associated with CDKL5 Deficiency Disorder (CDD): Findings from the Phase 3 MARIGOLD Study (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_3954;
P3
However, these results may be confounded by baseline differences in patient characteristics in concomitant ASM subgroups and varying placebo responses. Safety findings were comparable with different concomitant ASMs.
- Ztalmy (ganaxolone oral) / Marinus
Analysis of Real-world Characteristics of Patients with CDKL5 Deficiency Disorder Enrolled for Ganaxolone Treatment (Colorado Convention Center | Exhibit Hall B-E) - Mar 8, 2024 - Abstract #AAN2024AAN_3450;
These data provide insights into real-world utilization of ganaxolone in the treatment of CDD-associated seizures, including use in patients with highly refractory epilepsy. Such data may inform clinical decision-making in the treatment of CDD.
- | Ztalmy (ganaxolone oral) / Marinus
Preclinical, Journal: Early IGF-1 receptor inhibition in mice mimics preterm human brain disorders and reveals a therapeutic target. (Pubmed Central) - Mar 5, 2024
Pharmacological enhancement of GABAergic tonic inhibition by the U.S. Food and Drug Administration-approved drug ganaxolone rescued functional/behavioral alterations in mice. Establishing an unprecedented mouse model of prematurity, our work dissects the mechanisms at the core of abnormal behaviors and identifies a readily translatable therapeutic strategy for preterm brain disorders.
- | Ztalmy (ganaxolone oral) / Marinus
Journal: Cortical Tonic Inhibition Gates the Expression of Spike-and-Wave Discharges Associated with Absence Epilepsy. (Pubmed Central) - Feb 3, 2024
These results, together with previous findings, suggest that deviations of tonic inhibition either above or below an optimal set point can contribute to absence epilepsy. Returning the thalamocortical system to this set point may provide a novel treatment for refractory absence epilepsy.
- | Ztalmy (ganaxolone oral) / Marinus
Journal: Long-term treatment with ganaxolone for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder: 2-year open-label extension follow-up. (Pubmed Central) - Jan 15, 2024
P3
Sustained reductions in MMSF at 2?years in the OLE support the efficacy of ganaxolone in seizures associated with CDD. Safety findings in the OLE were consistent with the double-blind phase.
- || Zulresso (brexanolone) / Sage Therapeutics, Ztalmy (ganaxolone oral) / Marinus, Zurzuvae (zuranolone) / Sage Therapeutics, Shionogi, Biogen
Review, Journal: Novel neurosteroid therapeutics for post-partum depression: perspectives on clinical trials, program development, active research, and future directions. (Pubmed Central) - Dec 11, 2023
Finally, we describe new and future directions in neurosteroid therapeutics, including the development of an oral agent, zuranolone, and the IV and oral formulations of ganaxolone. Ultimately, the hope is that these novel neurosteroid therapeutics will provide fast-acting treatment for these impairing disorders and improve our understanding of the underlying mechanisms of depressive disorders.
- || Ztalmy (ganaxolone oral) / Marinus, soticlestat (TAK-935) / Takeda, Fintepla (low-dose fenfluramine) / UCB, Nippon Shinyaku
Review, Journal: Genetic Background of Epilepsy and Antiepileptic Treatments. (Pubmed Central) - Nov 29, 2023
On the contrary, the same drugs may aggravate seizures related to loss-of-function (LoF) variants of the same genes. Hence, knowledge of gene mutation-treatment response relationships facilitates more favorable selection of drugs for anticonvulsant therapy.
- | Ztalmy (ganaxolone oral) / Marinus
Journal: Neurosteroids as Novel Anticonvulsants for Refractory Status Epilepticus and Medical Countermeasures for Nerve Agents: A 15-Year Journey to Bring Ganaxolone from Bench to Clinic. (Pubmed Central) - Nov 18, 2023
Our pioneering use of neurosteroids for RSE and nerve agents, led us to develop ganaxolone as a novel anticonvulsant and neuroprotectant with significantly improved neurological outcomes. This article describes the bench-to-bedside journey of bringing neurosteroid therapy to patients, with ganaxolone leading the way.
- | Ztalmy (ganaxolone oral) / Marinus, gaboxadol (OV101) / Ovid Therapeutics, Angelini Group, Healx
Journal: Search for Novel Therapies for Essential Tremor Based on Positive Modulation of ?6-Containing GABA Receptors. (Pubmed Central) - Oct 30, 2023
Compound 6 and ketamine failed to display this profile, likely from off-target effects. Selective ?6 modulators hold promise as tremor therapy.
- || Review, Journal: Pharmacological diversity amongst approved and emerging antiseizure medications for the treatment of developmental and epileptic encephalopathies. (Pubmed Central) - Sep 1, 2023
Rufinamide, fenfluramine, stiripentol, cannabidiol and ganaxolone are antiseizure medications (ASMs) with diverse mechanisms of action that have been approved for treating specific DEEs...Stiripentol is licensed for treating seizures associated with Dravet syndrome in patients taking clobazam and/or valproate...Greater understanding of the causes of DEEs has driven research into the potential use of other novel and repurposed agents. Putative ASMs currently in clinical development for use in DEEs include soticlestat, carisbamate, verapamil, radiprodil, clemizole and lorcaserin.
- ||| Ztalmy (ganaxolone oral) / Marinus
Trial completion date, Trial primary completion date: Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy (clinicaltrials.gov) - Aug 31, 2023
P3, N=169, Enrolling by invitation, Sponsor: Marinus Pharmaceuticals
Putative ASMs currently in clinical development for use in DEEs include soticlestat, carisbamate, verapamil, radiprodil, clemizole and lorcaserin. Trial completion date: Dec 2024 --> Jun 2027 | Trial primary completion date: Dec 2024 --> Jun 2027
- || Ztalmy (ganaxolone oral) / Marinus, Xcopri (cenobamate) / SK Bio, Angelini Group, Endo
Review, Journal: New GABA-Targeting Therapies for the Treatment of Seizures and Epilepsy: I. Role of GABA as a Modulator of Seizure Activity and Recently Approved Medications Acting on the GABA System. (Pubmed Central) - Aug 21, 2023
We also review the available data for the two most recently approved antiseizure medications that act, at least in part, through GABAergic mechanisms, namely cenobamate and ganaxolone. Differences in the mode of drug discovery, pharmacological profile, pharmacokinetic properties, drug-drug interaction potential, and clinical efficacy and tolerability of these agents are discussed.
- Ztalmy (ganaxolone oral) / Marinus
Effect of ganaxolone on behaviours in children with the CDKL5 Deficiency Disorder (South Hall 2, 2nd Floor) - May 29, 2023 - Abstract #EPNS2023EPNS_339;
Non-seizure outcomes can contribute to the success of anti-seizure interventions. Along with better seizure control, children who received ganaxolone had improved behavioural scores in select domains compared to those receiving placebo.
- | Ztalmy (ganaxolone oral) / Marinus
Journal: Alcohol and Ganaxolone Suppress Tremor via Extra-Synaptic GABA Receptors in the Harmaline Model of Essential Tremor. (Pubmed Central) - May 26, 2023
This study with a mouse model found that two specific kinds of GABA receptor subunits were needed for alcohol to work. As receptors with both these subunits are found mainly in cerebellum, this work suggests this is where alcohol acts to suppress tremor.
- | Ztalmy (ganaxolone oral) / Marinus
Journal: Complex Metabolism of the Novel Neurosteroid, Ganaxolone, in Humans. A Unique Challenge for MIST Assessment. (Pubmed Central) - May 15, 2023
Significance Statement Studies on the metabolism of GNX in humans revealed a complex array of products that circulated in plasma, the two major components of which were formed via an unexpected multi-step pathway. Complete structural characterization of these (disproportionate) human metabolites required extensive in vitro studies, along with contemporary mass spectrometry, NMR spectroscopy, and synthetic chemistry efforts, which served to underscore the limitations of traditional animal studies in predicting major circulating metabolites in man.
- || Ztalmy (ganaxolone oral) / Marinus
Clinical, P2 data, Journal: Phase 2, placebo-controlled clinical study of oral ganaxolone in PCDH19-clustering epilepsy. (Pubmed Central) - Mar 14, 2023
P2
Ganaxolone was generally well tolerated and led to a greater reduction in the frequency of PCDH19-clustering seizures compared to placebo; however, the trend did not reach statistical significance. Novel trial designs are likely needed to evaluate the effectiveness of antiseizure treatments for PCDH19-clustering epilepsy.
- || Review, Journal: Novel antidepressant drugs: Beyond monoamine targets. (Pubmed Central) - Feb 27, 2023
The renaissance of psychedelic drugs and the emergence of preliminary positive clinical trial results with psilocybin, Ayahuasca, 5-methoxy-N,N-dimethyltryptamine (5-MeO-DMT), and lysergic acid diethylamide (LSD) may pave the way towards establishing this class of drugs as effective therapies for MDD, TRD and other neuropsychiatric disorders. Going beyond the monoamine targets appears to be an effective strategy to develop novel antidepressant drugs with superior efficacy, safety, and tolerability for the improved treatment of MDD and TRD.
- | Ztalmy (ganaxolone oral) / Marinus
Journal: Ganaxolone for management of seizures associated with CDKL5 deficiency disorder. (Pubmed Central) - Dec 16, 2022
Going beyond the monoamine targets appears to be an effective strategy to develop novel antidepressant drugs with superior efficacy, safety, and tolerability for the improved treatment of MDD and TRD. No abstract available
- | Ztalmy (ganaxolone oral) / Marinus
FDA event, Journal: Isobolographic analysis of adjunct Antiseizure activity of the FDA-approved cannabidiol with neurosteroids and benzodiazepines in adult refractory focal onset epilepsy. (Pubmed Central) - Dec 10, 2022
Isobolographic analysis shows the combination regimens of CBD + ganaxolone and CBD+ midazolam exerted combination index of 0.313 and 0.164, indicating strong synergism for seizure protection, with little to no toxicity. Together, these results demonstrate the therapeutic potential of CBD monotherapy and as an adjunct therapy for adult focal refractory epilepsy in combination with GABAergic ASMs.