Study Overview - The HELIOS trial evaluated the safety, tolerability, and efficacy of PB&TURSO in Wolfram Syndrome patients [1, 15] - The study was an open-label Phase 2 trial [1, 16] - The trial included 12 enrolled participants, with 11 in the Per Protocol (PP) population who had genetically confirmed Wolfram Syndrome [16, 26] Efficacy Results - At Week 24, the Per Protocol group (N=11) showed improvement in C-peptide response to Mixed Meal Tolerance Test (MMTT) compared to screening [30, 31] - At Week 24, C-Peptide AUC increased by 16.8 min*ng/mL in ITT population and 20.2 min*ng/mL in Per Protocol population [34] - 10 out of 11 participants in the Per Protocol group maintained or decreased time to peak C-peptide at Week 24 compared to screening [37, 38] - HbA1c levels showed a mean change from baseline of -0.09% in the ITT group and -0.16% in the Per Protocol group at Week 24 [40, 45] - 9 of 11 Per Protocol participants demonstrated reduced or unchanged HbA1c from Screening to the latest available time point [42] - 8 of 11 participants in the Per Protocol group demonstrated improved or stable visual acuity in their best eye from Screening to the latest available timepoint [51] Safety and Tolerability - PB&TURSO was generally well-tolerated [53] - 91.7% of participants (11 out of 12) experienced at least one Treatment Emergent Adverse Event (TEAE) [53] - Diarrhea was the most common TEAE, affecting 50% of participants, but all cases were of mild severity [55]