XEN496 for KCNQ2 Epileptic Encephalopathy, the State of Acute Stroke Care, and Ocrelizumab's Success

September 15, 2018

Neurology News Network for the week of September 15, 2018.

This week, Neurology News Network covered the recent addition of XEN496 to the Xenon Pharmaceuticals ion channel pipeline, Tudor Jovin's thoughts on the state of acute stroke care, and a conversation with Fred Lublin about the success of ocrelizumab in primary progressive multiple sclerosis. (Transcript below.)

Matt: Welcome to Neurology News Network. I’m Matt Hoffman.

Jenna: And I’m Jenna Payesko.

Recently, Xenon Pharmaceuticals announced the introduction of XEN496 for the treatment of KCNQ2 epileptic encephalopathy into clinical development. The therapy is a Kv7 potassium channel modulator with ezogabine as its active ingredient. This expansion of the company’s ion channel pipeline was a result of feedback it received from the FDA, and the initiation of a phase III clinical trial is now being planned for mid-2019.

To date, no drug has been studied and approved for this specific indication. The president of the KCNQ2 Cure Alliance, Jim Johnson, said that the group is excited about the announcement and supports the development of new and better therapies for this unmet need.

Matt: Acute stroke care has undergone massive changes in the past few years, mostly thanks to confirmatory data regarding the success of thrombectomy in patients with large vessel occlusion.

For Tudor Jovin, MD, the assistant professor of neurology and neurosurgery at the University of Pittsburgh School of Medicine, and co-director of the Center for Endovascular Therapy at the University of Pittsburgh Medical Center, the most important facet of that improved care has been the assessment of the treatment windows for these patients. He noted in a conversation with NeurologyLive that this new understanding has led to guideline adjustments and has had a real impact on the treatment of patients who experience an acute stroke.

Jenna: In 2017, the FDA approved ocrelizumab for the treatment of primary progressive multiple sclerosis, marking a turning point in the disease’s history, and launching treatments into a new era. For Fred Lublin, MD, the director of the Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Sinai Medical Center, it was a centennial event.

Matt: Although, despite that excitement and its success in its clinical trial program, Lublin told us that there is still much to learn about using ocrelizumab moving forward.

Jenna: There’s much to learn about the progressive treatments in general. But, he was also excited by what’s in the pipeline for progressive disease.

Matt: Specifically, he was excited about siponimod, an oral selective S1P1 modulator that has also shown modest effects on secondary progressive disease in recent clinical data, upping its potential as a progressive therapy.

Jenna: To discuss all of this, Lublin sat with NeurologyLive in an interview. Let’s take a look.

Fred Lublin, MD: We don't yet have a complete understanding of who might be the best individuals to treat with [ocrelizumab]. The effect was very modest—it looked like maybe if you were younger, you did better, if you had any activity, maybe you did better. But the data are not there yet to really answer that question, so as we move forward, we're learning how best to use it, and hopefully, additional clinical trials of similar agents will give us additional information .

Matt: For more on these stories, and for more direct access to expert insight, head to neurologylive.com. This has been Neurology News Network. Thanks for watching.