Discussing phase 3 data of Panzyga for PANS, with Michael Daines, MD

Michael O. Daines, MD, division chief of Pediatric Allergy and Immunology at the University of Arizona College of Medicine and principal investigator of the phase 3 trial evaluating Panzyga for pediatric acute-onset neuropsychiatric syndrome (PANS), emphasized the potential clinical benefit of the treatment despite narrowly missing its primary efficacy endpoint in a recent phase 3 study.

"We chose a primary endpoint of something called a CY-BOCS, which is a validated scoring system for looking at OCD," said Daines. "There was a clinically relevant improvement in children that were treated with Panzyga in this study, but it did not reach statistical significance, because there was also some improvement in the placebo."

The randomized, double-blind, placebo-controlled trial assessed the safety and efficacy of Panzyga (immune globulin intravenous, human - ifas) 10% in 71 pediatric patients aged 6 to 17 years with moderate-to-severe PANS. Participants received 3 infusions of Panzyga or placebo over 2 days every 3 weeks for a total of 9 weeks, followed by a double-blind, crossover extension period.

While CY-BOCS (Children's Yale-Brown Obsessive-Compulsive Scale) was selected as the primary endpoint because of the central role OCD symptoms play in diagnosing PANS, the improvement observed in the Panzyga group, though greater than in placebo, did not achieve statistical significance (P = 0.072).

"Many times in clinical studies, when you start bringing kids in and observing them and looking at them and doing things with them, they get better just from being part of a clinical study," Daines noted.

However, the trial's secondary endpoint—the Clinical Global Impression-Improvement (CGI-I) scale—showed a statistically and clinically significant improvement (P = 0.017).

"This is a broader tool that looks at essentially how sick the child is," said Daines. "It’s also going to include those minor criteria... and there was a clinically relevant and strongly significant improvement in the CGI in the children that were receiving treatment."

Looking ahead, Daines highlighted the need for continued research, and pointed to the need for studies of other immune-modulatory treatments that may offer lower costs or improved safety, as well as the importance of identifying a biomarker for more efficient diagnosis.

"There are a lot of diseases that don't have biomarkers... but because it is a relatively complex assessment... if we had a simple blood test or microbiome test... that could help us get to a diagnosis quicker," he added.

Reference:

Octapharma releases results for phase 3 superiority study of pediatric acuteonset neuropsychiatric syndrome. Octapharma. Press release. May 27, 2025. Accessed June 19, 2025. https://www.octapharma.com/news/press-release/2025/octapharma-releases-results-phase3-superiority-study-pediatric-acuteonset-neuropsychiatric-syndrome