Rick Hawkins, Chief Executive Officer at Lumos Pharma, discusses treating pediatric growth hormone deficiency (PGHD).
Transcription:
Most of the patients with pediatric growth hormone deficiency or PGHD, have a moderate form of the disease. They don’t make enough growth hormone, they are of short stature, but it’s a mild to moderate case. Or all the way to the full spectrum of patients who just can’t make, store, or release growth hormones.
That’s a severe form of the disease that those patients usually are the shortest stature. They get diagnosed earliest. Once again, the standard care is daily ingestion for 78 years of their lives. There’s some innovation that has taken place by three companies: Pfizer, Novo Nordisk, and Ascendis, who have a weekly injection of a recombinant growth hormone. But you still have to take an injection for these kids. They’re not too pleased with that to a large extent.
The diagnosis is fairly straightforward, and it varies a bit from one part of the world to the other. But pediatricians are accustomed to following patients and looking at their growth patterns. If they fall below a standard for their age and sex, then they will typically get referred to a pediatric endocrinologist, a specialist in this space who then does some additional testing, and you can show up then with the diagnosis of PGHD and get treated with growth hormone.
Once you get the diagnosis, and diagnosis can fall into two categories. Those moderate patients who still have an intact pituitary axis can make some growth hormone. That is roughly two-thirds of the patients, it’s idiopathic. No one knows the cause, but that’s where most of the patients fall.
Roughly a third of the patients, though, have a severe form of the disease, and that could be from head trauma, where the stalk of the pituitary is broken or severed, or radiation because they’ve received radiation to the pituitary for a tumor, or a number of other genetic causes or unknown causes that will cause patients to present with a severe form of the disease.
Those patients with a severe form of the disease should be treated with recombinant growth hormone, because our drug really works in a unique or different way. It’s broadly called a growth hormone secretagogues. Small molecules are then designed to go directly to the receptor in the pituitary that agonizes or stimulates the pituitary to make more growth hormone.
The natural way that we all make growth hormone is that we make it 23-25 times a day or release it from the pituitary. Once we release enough, there’s a natural feedback loop in natural physiology to shut down the further release of growth hormone when we release enough. That biology is very well understood at this stage. Once again, we release this growth hormone 23-25 times a day. All our drug does is increase the amplitude of each one of those pulsatile releases of growth hormone. It brings patients back to natural physiology.
That’s important because most pediatric endocrinologists want to restore a patient to their own natural production of growth hormone. That’s exactly what this drug does. Whereas the injectable products are exogenous of this growth hormone, endocrine feedback loop. You give a dose of growth hormone, there’s a peak, and then it slowly tailors off by the end of the day. Or the long-acting growth hormones, the formula must be, there’s a couple of days a week in order to maintain high enough levels of growth hormone in these long-acting formulations, where you get a couple of standard deviations over normal levels of a growth factor called IGF-1. It’ll take a few years to understand the full effect of that.
Whereas we say, once again, within the natural physiology with our drug, once you produce enough of your own growth hormone, then there’s that feedback loop that sheds down to further production. You can’t get these high levels of IGF-1 and excursions of two to three standard deviations over normal levels that you do get with the long-acting products.
To learn more about growth hormone deficiency and other endocrine disorders, visit checkrare.com/diseases/endocrine-disorders/