Positive interim results for potential first oral medication for pediatric GH deficiency
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An interim analysis of two phase 2 trials showed an oral medication was associated with beneficial height velocity outcomes among 50% of participants with pediatric growth hormone deficiency, according to a company press release.
“The take home message is that the response to [ibutamoren] was everything we could have hoped for because there's tremendous data looking at the growth hormone response in the population we're studying, which has moderate growth hormone deficiency,” David B. Karpf, MD, chief medical officer of Lumos Pharmaceuticals and adjunct clinical professor of endocrinology at Stanford University School of Medicine, Palo Alto, California, told Healio. “It really showed between 8.3 and 8.5 centimeters per year of growth in the extensive Lilly-Genesis database.”
The OraGrowtH210 trial was a multisite global trial of orally administered ibutamoren (Lumos; LUM-201) at three different doses compared with a standard dose of 34 µg/kg per day of injectable recombinant human GH. The study’s 66 participants were diagnosed with moderate pediatric GH deficiency.
At 6 months, the mean annualized height velocity was 7.26 cm per year among 11 participants who received ibutamoren 0.8 mg/kg per day, 8.57 cm per year among 10 participants who received ibutamoren 1.6 mg/kg per day, 7.77 cm per year among 10 participants who received ibutamoren 3.2 mg/kg per day and 11.05 cm per year among 10 participants who received the injectable recombinant human GH.
The mean annualized height velocity observed at 6 months in the 1.6 mg/kg per day group met expectations for 8.3 cm per year annualized height velocity observed after 12 months of treatment with recombinant GH in the 20-year phase 4 GeNeSIS database, according to the release.
In interim analysis of OraGrowtH210 at 9 and 12 months, researchers observed durability of the growth response for ibutamoren, although the number of participants was smaller. Over time, the annualized height velocity rate among participants in the recombinant GH arm was more pronounced, from 11.05 cm per year at 6 months to 9.93 cm per year at 12 months compared with 8.57 cm per year at 6 months to 8.14 cm per year at 12 months among participants who received ibutamoren 1.6 mg/kg per day.
Researchers also conducted an interim analysis of OraGrowtH212, a single-site, open-label trial of the pharmacokinetic and pharmacodynamic effects of oral ibutamoren in up to 24 participants with pediatric GH deficiency at 1.6 mg/kg and 3.2 mg/kg per day. At 6 months, annualized height velocity for each arm was comparable to results observed in the OraGrowtH210 trial with durable growth out to 12 months, according to the release.
This growth hormone response in this population of 11.05 cm was completely unexpected because other studies showed growth of 8.3 cm or 8.5 cm, according to Karpf.
“I'm quite sure that because this was an outlier, the imbalance, it tells me two things. One, if I repeated the same study, [the participants assigned to] growth hormone wouldn't have grown that much, and certainly in a large phase 3 study, as we’re planning, I think we will have much better balance of all these factors and are very unlikely to have any imbalance as in this very small population,” Karpf said.
There were no treatment-related serious adverse events and no drop-outs due to such events. Both the OraGrowtH212 and OraGrowtH210 trials demonstrated consistent safety data, according to the release.
“This market so far for the past 40 years, has treated every kid with growth hormone deficiency, whether severe or organic, or moderate and idiopathic, in the same way just by injection,” Karpf said. “This really shows that, for the first time, there can be an oral option for these patients, which will be very favorably looked upon by prescribers, as well as parents and patients.”
For more information:
David B. Karpf, MD, can be reached at dbkarpf@stanford.edu.
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