Odyssey Therapeutics (ODTX) investor relations material

Trial design and efficacy

• Open-label Phase IIa enrolled 61 patients with moderate-to-severe UC across 18 sites in seven countries; 56 were evaluable at Week 12 (15 at 10 mg BID, 41 at 20 mg BID). The primary endpoint was met, with mean modified Mayo score reduction of 2.4 points (p<0.001).

• At 20 mg BID, Week 12 clinical remission, clinical response and symptomatic remission were 27%, 61% and 49%, respectively; at 10 mg BID, rates were 20%, 40% and 27%.

• At 20 mg BID, symptomatic remission rose from 21% at Week 2 to 37% at Week 8 and 49% at Week 12, with no plateau during induction.

• Clinical remission at 20 mg BID was 29% in advanced-therapy-naïve and 23% in experienced patients, and 16% in patients with MES 3 disease (n=19).

• Combined Phase I pharmacology and Phase IIa findings support 20 mg BID as the projected lowest maximally effective dose; the proof-of-concept study had no concurrent placebo control, and results may change before database lock.

Biomarkers and transcriptomic findings

• At 20 mg BID, hs-CRP and neutrophils significantly decreased, serum albumin increased, and fecal calprotectin trended lower, supporting reduced local and systemic inflammation.

• Paired biopsies showed reductions in RIPK2-responsive genes and inflammatory pathways, including IL-23, TNF/NF-κB, TL1A, AP-1 and T-cell signaling.

• Fibrosis-associated signatures decreased while epithelial homeostasis and mucosal-healing signatures increased; remitters and responders showed concordant improvements in transcriptional profiles, histology and mucosal inflammation.

• OD-001 downregulated 29 of 34 genes in a published mucosal signature associated with anti-TNF non-response; transcriptomic effects also overlapped with anti-IL-23 response patterns.

Safety and tolerability

• OD-001 was generally well tolerated over 12 weeks, with no serious adverse events, serious infections, treatment-related discontinuations or liver function abnormalities.

• Three adverse events in the 20 mg BID group were considered drug-related: two fevers and one headache. Grade 3 anemia in the 10 mg BID group was considered unrelated and resolved with iron supplementation without discontinuation.