Recordati Rare Disease has revealed new data from a phase 3 trial of its Cushing’s disease treatment Isturisa.
Results from the LINC 4 study, presented at the Endocrine Society’s Annual Meeting, showed that Isturisa (osilodrostat) provided rapid and sustained normalisation of mean urinary-free cortisol (mUFC) levels.
Cushing’s disease is a rare disorder that occurs when the body produces too many corticosteroid hormones, usually as a result of a tumour on the pituitary gland. That in turn leads to weight gain, fat build-up on the face and susceptibility to bruising.
The normalisation of mUFC levels represents an ‘important’ treatment goal, Recordati said in a statement, by potentially reducing morbidity, improving quality of life and restoring life expectancy of patients with Cushing’s disease towards that of the general population.
Isturisa also met the key secondary endpoint of the LINC 4 trial, with 81% of patients having normal mUFC levels at week 36.
This was accompanied by improvements in cardiovascular and metabolic-related parameters, including systolic and diastolic blood pressure and glycated haemoglobin.
“We are delighted that the positive and statistically significant data from the LINC 4 study have been presented at the Endocrine Society’s Annual Meeting,” said Andrea Recordati, chief executive officer of Recordati.
“This data adds to the robust body of evidence supporting Isturisa as an effective and well-tolerated treatment for patients with Cushing’s disease,” he added.
In March 2020, the US Food and Drug Administration (FDA) approved Isturisa for adult Cushing’s disease patients who can’t be treated with surgery, or for whom surgery is ineffective.
It is the first FDA-approved drug to directly address overproduction of cortisol in Cushing’s disease by blocking the enzyme 11-beta-hydroxylase involved in the synthesis of cortisol, the body’s main stress hormone.
Isturisa was originally developed by Novartis, although Recordati licensed global rights from the company in July 2019 for $390m upfront in a deal that also included rights to Signifor and Signifor LAR that are both based on the somatostatin analogue pasireotide.
The drug is also indicated in the EU for the treatment of adult patients with endogenous Cushing’s syndrome, a rare condition of hypercortisolism that is most commonly caused by Cushing’s disease.
