Lumos Pharma announced new analyses of data from its Phase 2 OraGrowtH210 and OraGrowtH212 clinical trials, presented at the 62nd Annual European Society for Paediatric Endocrinology Meeting (ESPE 2024). The data demonstrate sustained growth in children with moderate Pediatric Growth Hormone Deficiency (PGHD) treated with oral LUM-201 for up to 24 months. These findings support the advancement of LUM-201 into a Phase 3 clinical trial next year.
Sustained Growth with LUM-201
Updated combined data from the Phase 2 OraGrowtH210 and OraGrowtH212 trials showed that treatment with LUM-201 resulted in a significant increase in Annualized Height Velocity (AHV) at six months. Specifically, the combined 1.6 and 3.2 mg/kg/day doses led to an AHV of 7.8 cm/yr at 6 months (N=66), compared to a baseline AHV of 4.6 cm/yr (N=37).
Growth rates were sustained out to 24 months, with AHVs of 7.3 cm/yr (N=65) at 12 months, 7.5 cm/yr (N=36) at 18 months, and 7.3 cm/yr (N=20) at 24 months. These results indicate a consistent growth response over the two-year treatment period.
Impact on IGF-1 and IGFBP-3 Levels
The trials also demonstrated significant increases in Insulin-like Growth Factor 1 (IGF-1) and Insulin-like Growth Factor-Binding Protein 3 (IGFBP-3) levels from baseline to 24 months. IGF-1 levels increased from 13.1 nmol/L at baseline to 25.5 nmol/L at 12 months and 29.2 nmol/L at 24 months. Similarly, IGFBP-3 levels increased from 133.5 nmol/L at baseline to 179.6 nmol/L at 12 months and 186.2 nmol/L at 24 months.
Correlation of Pulsatile GH Secretion and Growth
Further analysis revealed a relationship between the pulsatile growth hormone (GH) profiles induced by LUM-201 and the growth and IGF-1 responses. Data from the OraGrowtH212 trial indicated that greater 6-month AHV was associated with an earlier periodicity of augmented pulsatile GH secretion. This suggests that both the amount and pattern of GH secretion are crucial for growth and IGF-1 responses to LUM-201.
"The new analyses and updated data from our Phase 2 OraGrowtH210 and OraGrowtH212 Trials presented at ESPE this week demonstrate sustained growth on LUM-201 in moderate Pediatric Growth Hormone Deficiency (PGHD) to 24 months, as well as the correlation of LUM-201’s pulsatile mechanism of action (MOA) to growth," said John C. McKew, PhD, President and Chief Scientific Officer of Lumos Pharma. "We are encouraged by these data and look forward to advancing oral LUM-201 in a Phase 3 clinical trial in moderate PGHD next year."
