Patrick Trujillo

Patrick Trujillo asked about FDA clarity on the level of pancreatitis evidence required for a future risk reduction claim for Redemplo in high-risk SHTG patients, discussions around a potential pediatric pathway for FCS, and the target CSF tau knockdown level for clinical proof of concept in the MAPT program.

Answer

James Hamilton, Chief Medical Officer and Head of R&D, and Bruce Given, Chief Medical Officer, clarified that a specific pancreatitis claim for SHTG has not been discussed with the FDA, as physicians and payers generally understand the TG-pancreatitis relationship. They confirmed plans for pediatric work in FCS, pending a weight-based dosing formulation. For MAPT, James Hamilton stated the aim is at least 50-60% CSF tau reduction, based on NHP data showing 75%+ reductions and other ASO data correlating similar reductions with improved tau PET signals.

Patrick Trujillo with H.C. Wainwright inquired about FDA clarity on the level of pancreatitis evidence required for a future risk reduction claim in the high-risk SHTG patient population, discussions regarding a potential pediatric pathway for FCS, and the target level of CSF tau knockdown for clear clinical proof of concept in humans for the ARO-MAPT program.

Answer

James Hamilton, Chief Medical Officer and Head of R&D for Arrowhead Pharmaceuticals, stated that they have not specifically discussed with the FDA what would be required for a pancreatitis risk reduction claim in SHTG, noting that SHASTA V was designed more for international payer requirements. Bruce Given, Chief Medical Officer, confirmed plans for pediatric work in FCS, awaiting a weight-based dosing formulation. James Hamilton added that for ARO-MAPT, they aim for at least 50-60% CSF tau reduction, based on prior data correlating similar reductions with improved tau PET signals.