Trial Outcomes & Findings for Study to Evaluate the Clinical Activity and Safety of Oral NX-13 in Moderate to Severe Ulcerative Colitis (NCT NCT05785715)

A total of 159 participants were screened. A total of 81 participants were randomized and received at least 1 dose of study treatment. After completion of induction period, participants were eligible to enter a long-term extension period (LTE). Participants who received NX-13 250 or 750 mg during the induction period continued to receive the same blinded dose during the LTE. Participants who received placebo during the induction period were randomized to receive blinded NX-13 250 or 750 mg.

Study to Evaluate the Clinical Activity and Safety of Oral NX-13 in Moderate to Severe Ulcerative Colitis

The MMS ranges from 0 (normal or inactive disease) to 9 (severe disease) and is calculated as the sum of 3 subscores (stool frequency \[SFS\], rectal bleeding \[RBS\] and finding on endoscopy \[ES\]), each of which ranges from 0 (normal) to 3 (severe disease) with lower scores indicating better health status. A negative change in MMS score indicates improvement.

Treatment-emergent adverse events (TEAEs) were defined as AEs that occurred after the participant received first dose of study treatment or if a pre-existing condition worsens in severity or becomes serious after receiving the first dose of study treatment until 21 days after the date and time of last dose of study drug. A summary of other non-serious AEs and all serious AEs, regardless of causality, is located in the Reported AE section.

SAEs were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of other non-serious AEs and all serious AEs, regardless of causality, is located in the Reported AE section.

Clinical Remission per MMS was defined as achieving MMS \<=2, with RBS =0, SFS \<=1 and not greater than baseline, and ES \<=1. The MMS ranges from 0 (normal or inactive disease) to 9 (severe disease) and is calculated as the sum of 3 subscores (SFS, RBS, and ES), each of which ranges from 0 (normal) to 3 (severe disease). A lower MMS indicates better health status.

The MMS ranges from 0 (normal or inactive disease) to 9 (severe disease) and is calculated as the sum of 3 subscores (SFS, RBS, and ES), each of which ranges from 0 (normal) to 3 (severe disease). A lower MMS indicates better health status.

The Robarts Histopathology Index (RHI) score is used to assess the disease severity. The RHI is defined as the sum of four weighted items from Geboes: Grade 1: lamina propria chronic inflammation; Grade 2B: lamina propria neutrophils; Grade 3: epithelial neutrophils; Grade 5: surface epithelial injury. Total RHI score ranges from 0 (no disease activity) to 33 (severe disease activity) with a higher score indicating more severity.

Clinical Response was defined as \>=2 Points and \>=30% decrease from baseline in MMS with \>=1 point decrease in RBS or RBS \<=1. The MMS ranges from 0 (normal or inactive disease) to 9 (severe disease) and is calculated as the sum of 3 subscores (SFS, RBS, and ES) each of which ranges from 0 (normal) to 3 (severe disease) with lower scores indicating better health status.

Endoscopic Response was defined as ES \<=1. The ES subscore of the MMS ranges from 0 (normal) to 3 (severe disease) with lower scores indicating better health status.

Endoscopic Remission was defined as ES =0. The ES subscore of the MMS ranges from 0 (normal) to 3 (severe disease) with lower scores indicating better health status.

Endoscopic-histologic Mucosal Improvement was defined as ES \<=1 and Geboes score \<2.0. The ES subscore of the MMS ranges from 0 (normal) to 3 (severe disease) with lower scores indicating better health status. The Geboes scoring system is a stepwise ordinal grading system for histological assessment of disease severity in UC. The Geboes score includes 7 histological items (Grade 0 Grade 5) with a total score summed to a continuous variable, ranging from 0 to 22. Lower Geboes scores indicate better health status.

HEMI was defined as ES \<=1 and Geboes score \<3.1. The ES subscore of the MMS ranges from 0 (normal) to 3 (severe disease) with lower scores indicating better health status. The Geboes scoring system is a stepwise ordinal grading system for histological assessment of disease severity in UC. The Geboes score includes 7 histological items (Grade 0 Grade 5) with a total score summed to a continuous variable, ranging from 0 to 22. Lower Geboes scores indicate better health status.

HEMR was defined as ES = 0 and Geboes score \<2.0. The ES subscore of the MMS ranges from 0 (normal) to 3 (severe disease) with lower scores indicating better health status. The Geboes scoring system is a stepwise ordinal grading system for histological assessment of disease severity in UC. The Geboes score includes 7 histological items (Grade 0 Grade 5) with a total score summed to a continuous variable, ranging from 0 to 22. Lower Geboes scores indicate better health status.

Symptomatic remission was defined as RBS = 0 and (i) SFS = 0 or (ii) SFS = 1 with baseline SFS \>2, at Week 12. The MMS ranges from 0 (normal or inactive disease) to 9 (severe disease) and is calculated as the sum of 3 subscores (SFS, RBS, and ES) each of which ranges from 0 (normal) to 3 (severe disease) with lower scores indicating better health status.

Clinical response per partial MMS was defined as achieving \>=1 points and \>=30% decrease from baseline in partial MMS, with \>=1 point decrease in RBS or RBS \<=1. Partial MMS ranges from 0 (normal or inactive disease) to 6 (severe disease) and is calculated as the sum of 2 subscores (SFS and RBS), each of which ranges from 0 (normal) to 3 (severe disease). A lower partial MMS indicates better health status.

Participants rated their abdominal pain on a scale from 0 (no pain) to 10 (worst imaginable pain), with higher scores indicating more pain.

The rectal urgency score is based on the number of times participants must rush to the toilet to have a bowel movement. The score ranges from 0 (2 or fewer events) to 10 (12 or more events), with higher scores representing more severe symptoms.