Something good looks to be in the pipeline for pediatric growth hormone deficiency (PGHD) patients. A company involved in the development of drugs for conditions that are less common and uncared for has announced its acquisition of an experimental molecule that may help boost endogenous growth hormone.
Lumos Pharma revealed towards the end of September that it has obtained from Ammonett Pharma LLC license for LUM-201. This is an investigational oral treatment that supposedly can help to improve the natural production of human growth hormone (HGH) in the body of patients with deficiency issues.
The standard treatment for people with medically low levels of growth hormone is somatropin, also known as recombinant HGH (rHGH). This is a synthetic form of the natural substance that patients take through the use of injections.
Somatropin comes with certain safety issues, however, partly because it is synthetic.
This makes LUM-201 an interesting proposition. It simply enhances the ability of the pituitary gland to secrete HGH naturally. This mechanism has the potential to make it more preferable for people who currently take daily somatropin injections.
The clinical stage biopharmaceutical company revealed plans to commence a Phase IIb trial in people with pediatric growth hormone deficiency in 2019. The oral investigational molecule would be compared to daily rHGH injections.
“The Lumos investors and the entire Lumos team are extremely excited for the opportunity to rapidly advance this oral candidate for PGHD patients,” Lumos CEO Rick Hawkins said. “Many patients will potentially appreciate an alternative to injections.”
PGHD, a disorder that leads to abnormal linear growth, results from poor secretion of growth hormone by the pituitary gland. It is usually suspected when children exhibit stunted growth velocity and short stature.
Diagnosis is made in around 1 in every 4,000 children in America. This typically happens around the age of 7.
Growth hormone treatment should ideally commence immediately following diagnosis and must continue for years. This is to enable affected children to attain their maximum height potential.
But the idea of taking injections daily is not one that sits well with many children, with this adversely impacting adherence and treatment efficacy. This makes LUM-201 all the more interesting.
“Lumos is the perfect company to advance this treatment to approval,” Michael Turner, an endocrinologist at the University of Virginia and an Ammonett Pharma executive, said. “It has excellent rare disease drug development expertise and resources.”
LUM-201 has been granted Orphan Drug Designation in both America and the EU.