Horizon Scraps Actimmune Clinical Program in FA as Trial Failure Becomes Evident

Horizon Pharma has dropped its entire Actimmune (interferon gamma-1b) clinical development program in Friedreich’s ataxia following the failure of a Phase 3 trial which aimed to show that the treatment improves outcomes of the disease. The results are disappointing for patients with a disease lacking any effective treatment.

The STEADFAST trial (NCT02415127), which Horizon pursued in collaboration with Friedreich’s Ataxia Research Alliance (FARA) Collaborative Clinical Research Network (CCRN), had recruited 92 patients. Participants were randomized to receive either Actimmune or a placebo three times a week for 26 weeks.

The hope from the trial’s start was replaced with despair as it became obvious that the study did not prove its main point: that Actimmune could provide a significant change in the modified Friedreich’s Ataxia Rating Scale. The rating scale measures disease progression based on examinations of factors such as speech, ability to swallow, upper and lower limb coordination, gait and posture.

The analysis also showed that the treatment was ineffective in improving secondary study goals, including daily living activities and neurological outcomes.

“A well-designed, rigorous study like STEADFAST would not have been possible without the extraordinary drive of the Friedreich’s ataxia community, particularly the people who enrolled in the study, the clinical trial investigators and the Friedreich’s Ataxia Research Alliance,” Timothy P. Walbert, chairman, president, and CEO of Horizon Pharma, said in a statement.

“While the results were not what we hoped for, this is the very reason why research and development is important — to find answers that may help inform future research,” he said.

The decision to shut down the Actimmune development for Friedreich’s ataxia was reached in agreement with study partners at FARA. Both FARA and the study’s principal investigator will continue to be involved in further data analysis, in hopes that the study may provide information to aid further research.

“FARA’s mission is to drive research to develop therapies that will treat and cure Friedreich’s ataxia, and we remain passionately committed to that mission so that one day soon patients and caregivers impacted by this devastating disease will have effective treatment options,” said Ronald J. Bartek, co-founder and founding president of FARA.

“We want to extend our sincere appreciation to all of the patients, patient families and investigators who were a part of this study, as well as Horizon for collaborating with us so impressively on this important research,” Bartek added.

Patients who are currently enrolled in the ongoing extension study (NCT02797080) are asked to contact their study site coordinator for further information.