Ionis ramps up Angelman syndrome awareness with Lifetime segment

As it kicks off a pivotal phase 3 trial of its investigational treatment for Angelman syndrome (AS), Ionis Pharmaceuticals is laying the groundwork for a potential launch. After spending the last decade developing the drug known as ION582 and building relationships with the AS community, Ionis is now expanding that outreach work to include awareness-raising efforts for a broader audience, a company spokesperson told Fierce Pharma Marketing. To that end, Ionis recently worked with Lifetime to put together a segment on AS for the recurring “Behind the Mystery” series on the cable TV channel’s morning talk show “The Balancing Act.” The 20-minute segment, which aired on Lifetime in November and has since been made available online—and promoted via social media, email communications and conference handouts, per the spokesperson—features two sets of parents, each with a child living with AS, as well as doctors and AS community advocates. Angelman syndrome is a rare genetic neurological disorder that causes developmental delays, intellectual disability and balance issues, with its effects often noticeable before the age of 1. It’s estimated to affect around 500,000 people worldwide, and there is currently no cure. The “Behind the Mystery” episode sought to share not only educational information about the disease, but also the many challenges and triumphs that face families navigating AS diagnosis and treatment. In doing so, its overall goal was to familiarize the general public with AS in hopes of improving early recognition and diagnosis, as it can often be misdiagnosed as more common conditions like cerebral palsy and autism. The company spokesperson said Ionis opted out of “heavily scripting” the show to allow all the participants to tell their stories as authentically as possible. Ionis also reportedly involved the participants “at every stage” of the episode’s development, allowing them to review each cut and share feedback. Since the segment premiered, according to the spokesperson, many members of the AS community have shared with Ionis how meaningful it was to them. And one of the company’s advocacy partners reported that it received requests for more information about AS from 10 families across the U.S. within 24 hours of the episode’s first airing. “By keeping the community’s insights at the heart of what we do, we can celebrate progress and learn from challenges together—building greater awareness and visibility for everyone involved,” Holly Kordasiewicz, Ph.D., senior VP of neurology at Ionis, said in a company post about the segment. “This transparency is essential as we work to advance our science and bring new treatment options closer to patients.” Ionis announced in June that it had dosed the first patient in its phase 3 Reveal study, which is aiming to test the effects of quarterly dosing of ION582 over the course of one year in around 200 children and adults with UBE3A deletion or mutation. A phase 1/2 study of the drug—which was previously part of a Biogen partnership—showed that after six months of treatment, 97% of participants experienced clinically meaningful overall improvements on a scale measuring clinicians’ impressions of AS symptoms like cognition, expressive communication, motor skills and more, Ionis shared in July 2024.