LAUSR

Abstract Disclosure: E. Brincks: None. A. Parikh: Lumos Pharma, Inc. M. Quesnot: Lumos Pharma, Inc. C. Smith: Lumos Pharma, Inc. R. Christopher: Lumos Pharma, Inc. J. McKew: Lumos Pharma, Inc. P. Pitukcheewanont: Lumos Pharma, Inc. A. Bruchey: Lumos Pharma, Inc.. Objectives: The primary objectives of this study were to compare the bioavailability of the current tablet and capsule formulations of LUM-201, to determine the effect of food on the bioavailability of the new capsule formulation (filled with mini-tablets), and to assess the safety and tolerability of the new capsule formulation. The secondary objectives included evaluating the PD responses (specifically, serum GH levels) to the new capsule formulation as well as the PK of the major metabolite of LUM-201 (M8) in the fed versus fasted state. Methods: Part 1 was a 4-sequence/4-treatment/4-period crossover study determining relative bioavailability of current tablets compared to new capsule formulations and administrations. Part 2 was a 2-sequence/2-treatment/2-period crossover study to evaluate the new capsule formulation’s bioavailability in fed versus fasted conditions. Comparisons are reported as Dose-Normalized AUC0-inf (DNAUC0-inf) and Dose-Normalized Cmax (DNCmax). Results: The new capsule formulation showed bioequivalence to the tablet formulation in terms of DNAUC0-last and DNAUC0-inf. Compared with the tablet formulation, significant decreases in DNCmax were observed with 85.3mg whole capsule (14.1% decrease), with 85.3mg capsule contents in a dosing cup (20.4% decrease), or 85.3mg capsule contents in banana puree (14.4% decrease). In the food effect study, significant increases in exposures were noted for Cmax under fed conditions (64.4% increase) and AUC0-inf (17.8% increase). No significant effect of food was observed for the PK parameters of the metabolite M8. In terms of pharmacodynamics, serum growth hormone (GH) concentrations increased similarly across all treatment groups, demonstrating the expected PD response to LUM-201. Regarding food effect with LUM-201, the fed state reached Cmax 30 min later than the fasted state (Cmax of fed = 14.10 ng/mL; Cmax of fasted = 22.18 ng/mL). Importantly, serum GH reached similar Cmax values by 1.5 h after dose regardless of fed state. Additionally, serum insulin-like growth factor 1 (IGF-1) concentrations remained stable over time. Safety and tolerability of LUM-201 were consistent with the established safety profile; no SAEs were reported. Conclusions: The new LUM-201 capsule formulation produces PK and PD comparable to the current tablet formulation and offers children & their caretakers a more flexible and user-friendly oral capsule formulation. No significant differences in PK or PD were observed in comparisons of fasted versus fed state. Thus, regardless of formulation or administration method, the immediate GH release stimulated by LUM-201 results in similar GH Cmax values. Presentation: Sunday, July 13, 2025