Matt Hoffman

The chief executive officer of Alder Biopharmaceuticals detailed where the intravenous anti-CGRP treatment will fit into the marketplace and how the migraine prevention therapy will make an impact in treating patients.

A review of newly implemented primary end points—pain freedom and most bothersome symptom freedom at 2 hours—used in 8 clinical trials may help to guide future migraine researchers in the design of acute treatment trials.

The designation was granted by the FDA based on the results of the largest study ever conducted with monotherapy in NMOSD, which were favorable for the anti-CD19 antibody.

In addition to efficacy end points, the WEB system was markedly safer compared to current treatments, with no primary safety end points occurring between 30 days and 1 year.

Data from the STR1VE trial have reinforced the findings from the phase 1 START trial, including inclinations of prolonged survival as well as milestone achievement never seen in the natural history of SMA.

Alder Biopharmaceuticals’ monoclonal antibody was recently submitted in a BLA to the FDA and is on pace to hit the newly crowded preventive migraine market in early 2020.

MediciNova will be moving forward with a phase 2b/3 clinical trial of the ALS therapy, also known as MN-166, in 150 patients. If successful, its data will be used to support an NDA.

Patients with multiple sclerosis who were exposed to teriflunomide during pregnancy experienced similar risks of major birth defects and spontaneous abortions to the general population’s rates of 2% to 4% and 15% to 20%, respectively.

The scale includes 11 observable characteristics of CDM1 and was established through the merging of experiences and advice from patients, caregivers, therapeutic experts, and the FDA.

The Aquestive Therapeutics product, marketed as Exservan, was previously granted an orphan drug designation in January 2018. Its NDA is supported by a number of studies which sought to confirm its bioequivalence to its reference listed drug.

The study also added to a growing base of findings which suggest that patients who have failed preventive medications prior to trial participation have lower placebo responses, which could have implications for clinical trial design.

Data from the EVOLVE-1 and EVOLVE-2 phase 3 clinical trials suggest that galcanezumab (Emgality, Eli Lilly) can maintain ≥50% responder rates equivalent to that of placebo while reducing more monthly migraine days for 5 months after halting treatment.

Results of a recent real-world study at 2 MS centers have uncovered misdiagnosis rates of 17% and 19%, with patients facing an average time of misdiagnosis of 4 years during which they may be subject to numerous risks due to improper treatment.

Over the course of 2 years, findings revealed that serum NfL levels were significantly higher in patients with evidence of disease activity compared to those with no evidence of disease activity as well as 35 age-matched healthy controls.

The chief scientific officer of the Parkinson’s Foundation spoke about the developing interest in genetic forms of Parkinson, the potential to target therapies in that direction, and how a genetic treatment is the next big stage for the field.

The PEGASUS principal investigator and Translational Neurology Head of the Interdisciplinary Brain Center at Massachusetts General Hospital and Harvard Medical School spoke about the Amylyx Pharmaceuticals product’s potential in Alzheimer.

Pembrolizumab resulted in a decrease in the detection of programmed cell death protein 1 on lymphocytes in both cerebrospinal fluid and peripheral blood, with 5 of the 8 patients displaying clinical improvement or stabilization.

Amylyx Pharmaceuticals announced the trial of the 2-drug combination is seeking to enroll 100 patients with late-stage mild cognitive impairment or dementia due to Alzheimer, with plans to complete the study in 2020.

The chief scientific officer at the Parkinson's Foundation spoke about the need to increase awareness about the proper care of patients with Parkinson disease.

The International Headache Society has issued a number of recommendations for the proper design of trials for the prevention of pediatric migraine, hoping to address the challenges which are unique to pediatric patient populations.

Data from a national cohort has shown a 48% rate of confirmed symptomatic fractures, which the authors suggested was expected to be a large underestimation of the real frequency in the DMD patient population.

According to Kyowa Kirin, the investigational selective adenosine A 2A receptor antagonist has had its PDUFA action date set for August 27, 2019.

The MEK 1/2 inhibitor was previously granted an orphan drug designation by the FDA in February 2018 and resulted in partial responses in 72% of patients in the phase 2 SPRINT trial.

The professor of neurology at Harvard Medical School discussed differentiating pediatric MS from other CNS disorders, as well as the process of treating and managing these patients.

Experts say that while CBD and THC may have potential in multiple sclerosis and epilepsy, research on the endocannabinoid system is in its infancy—and the literature isn’t perfect.

The EMD Serono product was approved based on data which displayed a significant decrease in the number of relapses experienced by patients with MS who had ≥1 relapse in the previous year, compared to placebo.

The study author, a pediatric neurologist at Baylor College of Medicine and director of the Blue Bird Circle Rett Center at Texas Children’s Hospital, spoke about the investigational Rett syndrome treatment’s potential going forward.

The neurologist at Cleveland Clinic’s Mellen Center provided his insight into the clinical evidence for the selective sphingosine 1-phosphate receptor modulator.

With the lack of a gold standard diagnostic test, a study has shown through latent class analysis that cerebrospinal fluid immunoglobulin G may be a practical tool for determining the onset of MS in patients, particularly those who tested negative under the 2005 and 2010 McDonald criteria.

Of the 5 syndrome-specific efficacy measurements, trofinetide showed statistical benefits for patients with Rett syndrome on the Rett Syndrome Behavior Questionnaire, Clinical Global Impression Scale-Improvement, and the RTT Clinician Domain Specific Concerns-Visual Analog Scale.