Rick Hawkins, Chief Executive Officer at Lumos Pharma, discusses the OraGrowth212 Clinical Trial testing LUM-201 (ibutamoren) to treat pediatric growth hormone deficiency (PGHD).
Transcription:
We have a global trial up and running. There are 80 patients total in the trial. There are four arms of the trial, three different doses of our drug (ibutamoren) versus the standard dose of growth hormone. The three doses of the drug start with 0.8 mg/kg/day. That’s the highest dose that’s been used before this trial.
The safety profile of the drug is outstanding, so the FDA allowed us to double the dose to 1.6 in a second arm of 20 patients. Then the third arm allowed us to quadruple the dose to 3.2 mg/kg/day. All patients will be on drug for an extended period of time, actually several years. But the endpoint we’re looking at is catch-up growth or growth at six months annualized.
We’re going to report top-line results on a full 20 patients in each one of those arms in those cohorts on drug for six months annualized in the fourth quarter of this year. But we’ll have a very robust data set because there’s going to be at least 12 patients in each arm who will be on drug for a year, then a smaller number of patients on for 18 months, and a smaller number of patients that will be able to look at endpoint of height velocity at two years. That is a very robust data set for a phase II-B study.
We wanted to look at the data in the study with only 10 patients we did in the study. A number of months ago, we looked at them and had an interim look at the data. We reported that a number of months ago, and we’re quite pleased with the results.
To get some reference about how these patients grow with moderate growth hormone deficiency, what we looked at is some databases that have been kept for a number of years by large companies like Pfizer and Eli Lilly and Genentech and others.
When we apply our diagnostic review and selection process to pull out those moderate patients and know that they still have an intact pituitary, then we know that those moderate patients on growth hormone grow about 8.3-8.6 centimeters a year. When we had our patients, when we looked at that interim look with just six patients in each cohort, we found that they grew 8.6 centimeters. We were pleased with that.
Now, there was a bit of an imbalance in the growth hormone arm. Everyone knows that the younger patients have the greatest catch-up growth, and there was an imbalance by over 10 months as a younger group of patients in the growth hormone arm, so that arm grew better. But as we got to full enrollment, that balance returned in terms of not just age, but any number of other factors that allows us to predict that we’re going to have a good outcome in this study.
In addition to that, this study I just described has been highly de-risked in an important way. That is, we can choose patients who still have an intact pituitary, who meet what we call predictive enrichment markers positive patients by giving them a dose of our drug before they start the study, take a blood draw an hour later, and then measure two growth factors that have been measured in this space for a very long time.
One is just their growth hormone that they produce. The other one is a secondary growth factor called IGF-1, insulin-like growth factor 1. If growth hormone is at least 5 nanograms per ml or the IGF-1 levels are at least 30 or more, then we know that’s a patient that still has a functioning pituitary, but they don’t make enough growth hormone, and that our drug will help patients. We de-risked this whole program by only selecting those patients prior to them entering the study with that predictive enrichment marker strategy.
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