Phase 2 Trial Evaluating LUM-201 for Treatment of Growth Hormone Deficiency

[madman]

Pisit “Duke” Pitukcheewanont, MD, Chief Medical Officer for Lumos Pharma, discusses a phase 2 trial evaluating LUM-201 (ibutamoren) for treatment of growth hormone deficiency.

Pediatric growth hormone deficiency (PGHD) is a rare condition characterized by insufficient amounts of growth hormone produced by the pituitary gland. GHD can be acquired but is often congenital, with possible mutations in the POU1F1/Pit1 , PROP1 GHRH and GH1 genes.


Signs and symptoms may include:

- Short stature
- Slow growth or absence of growth
- Absent or delayed sexual development during puberty
- Headaches


Ibutamoren is an investigational, once-daily, oral small molecule that targets growth hormone (GH) secretion.

Based on the phase 2 OraGrowtH212 clinical trial, researchers evaluated growth hormone pulsatility data obtained at baseline and six months following treatment with ibutamoren. A significant increase over baseline in key endpoints such as GH secretion, pulsatile GH, and total GH was observed. A combined analysis of both dosing cohorts revealed that at six months, GH secretion was 3.3 ±1.8 to 4.4 ±2.1 μg/kg/day compared to 5.0 ±1.3 μg/kg/day derived from published data in normal children. These results suggest ibutamoren restores GH secretion, endogenous GH pulsatile secretion, and serum IGF-1 concentrations levels to approximately normal.

Results from an evaluation of pulsatile GH profiles and growth response to ibutamoren were also presented in a late-breaking poster. Data from the phase 2 clinical trial pulse assessments at day 1 (D1) and six months (M6) were analyzed to recognize relationships between D1 characteristics, D1 height velocity, 6M annualized height velocity (AHV) and interpuls, pulsatile, and total GH secretion at D1 and M6. Results from the analysis showed an increase in all endpoints from D1 to M6. Restoring GH secretion with ibutamoren results in both an increase in the overall amount of GH, and pattern alteration of the pulse profile.


Chapters:

Growth Hormone Deficiency Overview 00:00
Current Management 3:33
LUM-201 Poster Presentations 4:26
Next Steps 10:49