Infant Bacterial Therapeutics (IBT) Study Update summary

Study design and endpoints

• The Phase III study enrolled 2,153 premature infants over five years to assess IBP-9414, targeting NEC and sustained feeding tolerance (SFT) as primary endpoints, but neither reached statistical significance (NEC: 8.7% vs 10.2%, p=0.24; SFT: 16 vs 17 days, p=0.07).

• The primary endpoint for confirmed NEC showed a 14% risk reduction (relative risk 0.86, p=0.24).

• All-cause mortality was a key secondary endpoint, showing a statistically significant 27% risk reduction (6.2% vs 8.5%, relative risk 0.73, p=0.036–0.04), equating to 23 lives saved.

• No significant reduction was observed in days in hospital or other secondary endpoints related to reimbursement.

Safety and subgroup analysis

• No safety signals or increased risk of sepsis were observed, including no cases of Lactobacillus reuteri in the blood.

• Late-onset sepsis rates were similar between treatment and placebo groups, and early-onset sepsis was excluded from the study.

• Subgroup analyses did not reveal any regions or patient groups with detrimental effects; mortality benefit was consistent across subgroups.

• The 95% confidence interval for mortality did not cross one, supporting the robustness of the mortality benefit.

Regulatory and commercialization outlook

• Despite missing primary endpoints, the significant mortality reduction and strong safety profile are seen as compelling for regulatory submission.

• Regulatory precedent exists for drug approval based on secondary endpoints when medical need and safety are clear.

• The company plans to proceed with regulatory filings and production preparations, maintaining financial stability for continued development (SEK 272 million as of Q2).

• Label discussions with authorities will consider NEC, SFT, and potentially mortality, referencing similar precedents like Curosurf.