Ionis Pharmaceuticals announces pivotal patient trial of antisense therapy following aggressive joint research effort led by the nonprofits The ALS Association and Project ALS.

Research funded by The ALS Association and Project ALS has resulted in this week’s announcement by Ionis Pharmaceuticals of FUSION, a Phase 3 clinical trial of ION363 (also known as jacifusen), a novel antisense oligonucleotide (ASO) for ALS patients with a mutation in the fused in sarcoma (FUS) gene.

In February 2020, the two ALS nonprofits joined forces to support a pilot clinical research program—led by Neil Shneider, MD, PhD, director of the Eleanor and Lou Gehrig ALS Center at Columbia University Irving Medical Center—which allowed several patients with this rare and aggressive form of ALS to receive treatment with jacifusen.

“The Ionis trial of ION363 is evidence that by working together at a very early stage, nonprofits that are passionate about treating and curing ALS can pay forward more and better opportunities for patients,” said Kuldip Dave, PhD, Vice President of Research, The ALS Association. “This pilot program with Project ALS is part of a larger strategy toward providing treatments for all genetic forms of ALS, including rare types.”

The ION363 trial has its deepest roots in Valentine’s Day, 2019, when Jaci Hermstad, a 25-year-old Iowa woman, was diagnosed with FUS-ALS, eight years after losing her identical twin sister to the same disease at age 17. The Hermstad family contacted Project ALS and Dr. Shneider, who had been investigating the potential of ASOs in treating patients with FUS-ALS, to ask if there were any therapies that might help Jaci.

“The Hermstads’ story became an instant magnet for so many talented and caring people, from Ionis and Dr. Shneider, an expert in the FUS gene, to volunteer regulatory experts, manufacturers, and academic advisers,” said Valerie Estess, Project ALS director of research. “Jaci’s courage—and the teamwork she inspired—can now pay off for all patients with FUS-ALS.

Funding from the ALS Association and Project ALS made it possible for Dr. Shneider and his team at Columbia to offer jacifusen to ten additional FUS-ALS patients over the last two years while tracking safety data on the drug and disease-relevant biomarkers in parallel. “The ALS Association and Project ALS came together at a critical moment, allowing us to learn more about the safety and potential efficacy of jacifusen,” said Dr. Shneider, who will lead the Phase 3 trial. “I believe that their steadfast support of this early-stage clinical research provided Ionis with the data needed to further develop jacifusen in the current pivotal trial.”

“Jaci Hermstad’s spirit inspired us from the start,” said C. Frank Bennett, PhD, Ionis’ chief scientific officer and franchise leader for neurological programs. “Ionis will take this program forward because everyone involved in this experience shares the same goal: better medicine for ALS and other devastating neurological illnesses.”

Read Ionis Pharmaceuticals’ statement to the community for additional details of the phase 3 trial HERE.