Matt Hoffman

Following the receipt of a refusal to file letter from the FDA, Zogenix has resubmitted its application for fenfluramine hydrochloride (Fintepla), seeking approval for the treatment of seizures associated with Dravet syndrome.

The clinical development of ND0612 includes 2 doses; a low dose for mid-stage Parkinson patients experiencing loss of motor control on oral therapy, and a high dose for severe disease in which oral drugs are no longer effective.

Acorda Therapeutics’ Inbrija, an inhalation powder formulation of levodopa, was associated with no significant increases in troublesome dyskinesia for patients being treated with carbidopa/levodopa.

Data of amantadine (Gocovri, Adamas) extended-release tablets suggested that the therapy is, in fact, safe and tolerable with a unique pharmacokinetic profile, and results in significant improvements in dyskinesia for patients with Parkinson disease.

Acadia Pharmaceuticals’ pimavanserin significantly improved Hamilton Depression Rating Scale scores for patients with Parkinson disease with depressive symptoms in an 8-week, open-label study.

Cerevel Therapeutics’ highly selective dopamine D1/D5 agonist showed significant reductions in UPDRS-III scores for those with early-stage Parkinson disease and is planned to enter phase 3 in 2020.

PML incidence in a cohort of more than 6000 patients have decreased by 23% each year since risk guidelines and new serologic testing were introduced.

Based on datasets from 2 national registries and the European Interferon Beta Pregnancy Registry, the EMA’s Committee for Medicinal Products for Human Use has given a positive opinion to remove the contraindications, which would allow the Biogen agents to be used by pregnant and breastfeeding women with multiple sclerosis.

The analyses focus on the abuse potential, efficacy in high-burden patients with narcolepsy, and safety data for Harmony Biosciences’ histamine H3 receptor-antagonist/inverse agonist, pitolisant.

Analysis suggests that high-intensity aerobic exercise intervention, when gamified and delivered in an at-home fashion, can attenuate the symptoms of Parkinson disease while maintaining good adherence.

Data to be presented at the 2019 European Pediatric Neurology Society Congress suggest Zolgensma (onasemnogene abeparvovec-xioi) is associated with major milestone gains in SMA type 1.

A prospective, cross-sectional assessment suggests that patients with DMD can be adequately assessed for cognition in a brief period of time using the NIHTB‐CB, and additionally suggested a cognitive vulnerability in mothers who were carriers of DMD mutation.

Further analysis of the SHINE trial suggest nusinersen is linked to 6-year maintained improvement in patients with SMA. Biogen has announced a new global trial of a 50 mg loading dose in a broader cohort.

In a time where more than half of all patients with acute stroke and most patients with severe stroke arrive at the hospital via emergency medical services, a study suggests mobile stroke units are better equipped to accurately triage patients.

Mallinckrodt’s repository corticotropin injection was shown to have a lower cost per patient response compared to other late-line treatments for multiple sclerosis relapses, costing an estimated $148,528 less per response.

The GeNeuro agent showed marked reductions in brain atrophy in the thalamus and cerebral cortex through a long-term extension period treating patients with multiple sclerosis.

Impel NeuroPharma's nasal dihydroergotamine mesylate acute migraine therapy INP104 has shown excellent pharmacokinetic data, and is set to be assessed in a 24-week trial with a planned 28-week extension.

The director of the Sagol Center for Hyperbaric Medicine and Research spoke about how hyperbaric oxygen therapy may offer new hope in improving cognition in patients who have had a stroke.

According to new data from STRIVE, 75.4% of those treated with natalizumab achieved overall No Evidence of Disease Activity status by year 4, and no patients had incidence of progressive multifocal leukoencephalopathy.

After showing significant benefits in delayed disease progression in patients with secondary progressive MS, new data suggests siponimod can delay the time to wheelchair dependence by 4 years.

The global head of neuroimmunology at Genentech spoke about the wealth of data being presented on its anti-CD20 monoclonal antibody ocrelizumab, as well as the success thus far in trials of its NMOSD agent, satralizumab.

Study coauthor and professor of neurology and ophthalmology at the University of Colorado provides insight into phase 3 study results of satralizumab, presented at ECTRIMS 2019.

New phase 3 data from the OPTIMUM head-to-head trial show that 20-mg ponesimod resulted in a significantly lower annualized relapse rate in adults with multiple sclerosis compared to 14-mg teriflunomide at 108 weeks.

New decade-long data have suggested that multiple sclerosis treatment with dimethyl fumarate is associated with stable EDSS scores ≤3, and a low number of patients with confirmed disability progression.

New data from clinical trials of ocrelizumab showed that the anti-CD20+ B cell therapy lowered serum NfL levels, and that the NfL levels offered prognostic value for disease progression in MS.

Data from the phase 3 SUNBEAM trial of the sphingosine 1-phosphate receptor modulator have suggested that the Celgene treatment lowers annualized relapse rates and reduces the rate of cortical gray matter loss, improving cognition measures, compared to IFN-ß1a.

The guidelines recommend that patients with MS receive their recommended vaccines, including that for yearly influenza. Additionally, they noted that no evidence exists that suggests vaccination increases the risk of MS exacerbation.

Ra Pharmaceuticals’ macrocyclic peptide inhibitor of complement component 5 is expected to enter phase 3 of its development in the latter half of 2019 after success in a phase 2 trial of its potential in myasthenia gravis.

The founder and CEO of NeuroEM Therapeutics discussed the potential of transcranial electromagnetic treatment and shared insight into the advantages it may hold over the traditional treatment route.

The study, expected to complete in early 2021, will measure change from baseline in daily on time without troublesome dyskinesia. The study design will be presented at the International Congress of Parkinson's Disease and Movement Disorders.