Investigational oral therapy increases height velocity for children with GH deficiency
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Key takeaways:
- Daily ibutamoren was noninferior to recombinant human GH at 12 months for children with moderate GH deficiency.
- No serious adverse events were reported, and no adverse events led to discontinuation of the drug.
An oral medication under investigation for treating pediatric GH deficiency boosted annualized height velocity at 6 and 12 months for children, according to topline results from two phase 2 trials.
As Healio previously reported, ibutamoren (LUM-201, Lumos Pharma) increased annualized height velocity by 8.6 cm at 6 months for children with idiopathic pediatric growth hormone deficiency in interim data presented at the Pediatric Endocrine Society Annual Meeting. In new topline data from the phase 2 OraGrowtH210 dose-finding trial and the OraGrowtH212 Pharmacokinetic/Pharmacodynamic trial, ibutamoren met all primary and secondary endpoints with no safety signals identified, according to an industry press release.
In the OraGrowtH210 trial, 82 youths with moderate pediatric GH deficiency were randomly assigned to receive either 0.8 mg/kg, 1.6 mg/kg or 2.4 mg/kg of ibutamoren, or 34 µg/kg of recombinant human GH daily. Children receiving 1.6 mg/kg of ibutamoren had an annualized height velocity of 8.2 cm per year at 6 months compared with 10 cm per year for those receiving recombinant human GH. At 12 months, the 1.6 mg/kg dosing group had an annualized height velocity of 8 cm per year compared with 9.7 cm per year with human GH. According to the press release, the 1.6 mg/kg dose met expectations for growth and were within 2 cm per year margin for noninferiority compared with recombinant human GH.
In the OraGrowtH212 trial, 24 children who previously did not receive treatment for pediatric GH deficiency received either 1.6 mg/kg or 3.2 mg/kg of ibutamoren daily. According to the topline results, participants reached an expected annualized height velocity with only 20% of the GH concentration observed when using injectable recombinant human GH. Ibutamoren also raised circulating GH levels to normal physiological ranges compared with recombinant human GH, which elevated GH level 4 to 5 times higher than a typical healthy child.
“These findings not only align with historical growth expectations on therapy, but also underscore the distinct advantage of LUM-201’s unique pulsatile mechanism,” Ron Rosenfeld, MD, chairman of the clinical and scientific advisory board for Lumos Pharma, said in the release. “Demonstrating the ability to achieve expected growth with oral LUM-201 while exposing patients to only 20% of the growth hormone compared to daily recombinant human GH injections is a significant scientific breakthrough that has the potential to revolutionize the approach to treating children with moderate growth hormone deficiency.”
Data at 18 and 24 months were available for 10 participants in participated in one of the two trials. Combined data from children who received either 1.6 mg/kg or 3.2 mg/kg showed participants has sustained annualized height velocities from 12 to 24 months without a consider decline in growth velocity.
According to the release, there were no treatment-related serious adverse events reported in the trial. No participants discontinued treatment due to adverse events. There were also no safety concerns after an analysis of laboratory values, adverse event data and electrocardiogram readings.
According to the release, Lumos Pharma will finalize plans for a phase 3 trial in the first half of 2024.
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