HD Treatment Tominsersen Discontinued, FDA Approves Atogepant NDA, NurOwn's Promising Results in Progressive MS

This week NeurologyLive covered the discontinuation of the phase 3 GENERATION HD1 study evaluating Huntington disease treatment tominersen, the FDA's acceptance of a new drug application for atogepant, and the positive phase 2 trial data of NurOwn in patients with progressive MS.

Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

The phase 3 GENERATION HD1 study evaluating tominersen, the first agent to successfully target and reduce levels of mutant huntingtin protein in patients with Huntington disease, will discontinue dosing, according to a recent announcement from Roche. The decision, informed by an unblinded Independent Data Monitoring Committee, stems from a pre-planned review of the results of the phase 3 study. Despite no new or emerging safety signals in treatment with tominersen, the recommendation was based on the investigational therapy’s potential benefit/risk profile for study participants. Roche noted that they will still follow participants for safety and clinical outcomes, without the dosing of the investigational agent or placebo. Following complete analysis of the available data from the phase 3 study, the company will present the research to the HD community as well as detail their future plans.

AbbVie announced that the FDA has accepted its new drug application for its investigational agent atogepant for the preventive treatment of migraine in adults with episodic migraine. A regulatory decision is expected to come late in the third quarter of 2021. Atogepant, an orally administered calcitonin gene-related peptide (CGRP), had its NDA supported by a clinical program that included nearly 2500 patients and spanned across the phase 3 ADVANCE study, the pivotal phase 2b/3 study, and the phase 3 long-term safety study. The pivotal phase 3 ADVANCE trial met the primary end point, with those treated in the 10-, 30-, and 60-mg arms experiencing decreases in monthly migraine days (MMD) of 3.69, 3.86, and 4.2 days, respectively, compared with 2.48 days with placebo over the 12-week treatment period.

BrainStorm Cell Therapeutics announced that the company’s phase 2 trial evaluating NurOwn (autologous mesenchymal stromal cells secreting neurotrophic factors [MSC-NTF] cells) met its primary end point of safety in the treatment of progressive multiple sclerosis. The 28-week, open-label trial enrolled 20 patients with primary and secondary progressive MS who were given 3 repeated administrations of NurOwn, each 2 months apart. Additional secondary efficacy data and detailed cerebrospinal fluid and blood biomarker analyses are still underway and will be reported at an upcoming scientific meeting, according to BrainStorm. A total of 18 patients were treated and 16 (80%) completed the study. Procedure-related adverse events (AEs) resulted in 2 patients discontinuing. There were no study deaths or AEs related to MS worsening. "This is an extremely exciting outcome, as it demonstrates the potential of our proprietary cell therapy NurOwn in progressive MS and expands the body of evidence supporting the NurOwn technology platform in neurodegenerative disease,” Chaim Lebovits, chief executive officer, BrainStorm, said in a statement.

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