Rallybio to begin RLYB116 confirmatory clinical PK/PD study in Q2

Rallybio (RLYB) announced results of new biomarker characterization analyses and data from the recently completed manufacturing process enhancements for RLYB116, the company’s nce-weekly, small volume, subcutaneously injected C5 inhibitor in development for the treatment of patients with complement-mediated diseases. Based on this recently completed work, Rallybio believes that RLYB116 has the potential to be a best-in-class C5 inhibitor and intends to initiate a confirmatory clinical pharmacokinetic/pharmacodynamic or PK/PD study in the second quarter of 2025. “Recently completed efforts to better understand the disconnect between the compelling preclinical potency data of RLYB116 and the results of the Phase 1 study indicate that RLYB116 achieved greater complement inhibition in the Phase 1 study than initially reported,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. …Additionally, Rallybio completed manufacturing process enhancements in the third quarter of 2024 that are expected to further improve the tolerability of RLYB116. Based on the results of enhanced analytical techniques, including mass spectrometry, process enhancements have successfully further purified the RLYB116 drug substance. As a result, RLYB116 is expected to have a favorable tolerability profile at doses at and above those evaluated in the Phase 1 MAD study. Rallybio plans to initiate a RLYB116 confirmatory clinical PK/PD study in the second quarter of 2025 to demonstrate improved tolerability as well as complete and sustained complement inhibition. This single-blind multiple ascending dose study will evaluate a 4-week treatment duration that will include two cohorts of 8 participants each. It is planned that Cohort 1 will evaluate weekly dosing of 150 mg and Cohort 2 will evaluate weekly dosing of 225 mg. Follow-up will continue for 10 weeks after the conclusion of treatment.