GlobalData, a data and analytics company, on Tuesday, said that with a favorable administration route, LUM-201 has the potential to improve low rates of treatment compliance, a significant unmet need in pediatric growth hormone deficiency (PGHD).
According to GlobalData, the release of encouraging interim results from two Phase II trials of LUM-201 has enabled Lumos Pharma to edge closer to its goal of bringing the first orally administered therapy for PGHD to market.
“For decades, the only treatment option available to patients with PGHD has been through injections with recombinant human growth hormone (rhGH). The prospect of an approved oral therapy is an exciting development for patients and their caregivers,” Fiona Chisholm, Managing Analyst at GlobalData, said in a statement.
PGHD is a rare disorder with approximately 14,000 children (aged 0-11 years) diagnosed in the US, according to GlobalData’s epidemiology database.
The disorder is characterised by inadequate secretion of growth hormone from the anterior pituitary gland, which results in stunted growth and requires patients to undergo treatment frequently with daily or weekly injections over many years.
According to key opinion leaders (KOLs) interviewed by GlobalData, this can often lead to poor treatment compliance, and, consequently, suboptimal therapeutic outcomes.
“The burden of injections can be particularly problematic for pediatric populations. Children are often more susceptible to needle phobia than adults, and this can lead to treatment avoidance or resistance. Recently, treatment options for PGHD have improved due to the emergence of the first long-acting rhGHs, which require weekly rather than daily dosing. However, there is still an unmet need for a safe and effective oral therapy,” Chisholm said.
LUM-201 increases the amplitude of endogenous pulsatile growth hormone secretion via potent agonism of the growth hormone secretagogue receptor (GHSR), and, therefore, represents a novel approach to the treatment of PGHD.
“If successful, the therapy is likely to be approved for patients with moderate disease, who account for approximately 60% of the overall PGHD population. In the Phase II trials, the safety and tolerability of LUM-201 was found to be comparable to rhGH and the growth response observed was in line with expectations based on comparisons with historical data from rhGH trials,” the company said.
According to GlobalData’s pipeline product database, it is the only one of eight assets in the late-stage pipeline that is not a rhGH.
“This is an exciting development within the PGHD space. For the first time it appears credible that an oral investigational therapy could demonstrate similar safety and efficacy to rhGH in a PGHD subset. If further data validates this, it will provide Lumos with a unique and compelling marketing strategy for LUM-201,” Chisholm added.