Drug development company Dimerix (ASX: DXB) emerged from a trading halt today with top-line results from its phase 2a ACTION study into DMX-200 for treatment of focal segmental glomerulosclerosis (FSGS), a rare kidney disorder that often leads to end-stage kidney failure.
Dimerix received orphan drug designation for DMX-200 in November 2018 in both the US and Europe for the treatment of FSGS, after repurposing the drug based on preliminary non-clinical data which showed a reduction in the number of podocytes lost and an improvement in proteinuria, the presence of excess proteins in the urine.
The preliminary results were described as “very impressive” by Dimerix’s medical advisory board chair Dr Hiddo Heerspink, who also confirmed that future studies were already being planned.
Meanwhile, the study investigator and head of the Melbourne Renal Research Group, Associate Professor David Packham, said the top-line results were “tremendous and very encouraging” for all FSGS sufferers.
Given the phase 2 level of the study, the primary endpoint was safety, measured by the number and severity of adverse events when using DMX-200 compared to a placebo in patients with FSGS who were already receiving 300mg of high blood pressure drug irbesartan, also known as Avapro under its brand name.
More specifically, the study was a double-blind, randomised, placebo-controlled, crossover study designed to evaluate both safety and “preliminary signs of efficacy”.
Each patient in the study received DMX-200 for 16 weeks preceded or followed by 16 weeks of placebo.
Dimerix reported that preliminary safety findings indicated that DMX-200 was “generally safe and well-tolerated”, with no variation in the incidence or severity of adverse events between while being treated with DMX-200 or the placebo.
According to Dimerix, six out of seven patients (86%) demonstrated a reduction in proteinuria as a result of treatment compared to a placebo, with an average 29% change from baseline in 24-hour proteinuria compared to placebo following treatment with DMX-200.
Moreover, two out seven (29%) of patients achieved more than a 40% reduction in proteinuria as a result of the study.
“I believe that the results of this phase 2a FSGS study, together with the positive outcomes of the company’s prior phase 2a study in chronic kidney disease, further validates Dimerix’ lead candidate, DMX-200, in sclerotic kidney diseases,” said Dr Hiddo explained.
“The positive signals suggest that treatment with DMX-200 may indeed result in clinically meaningful improvements in kidney function when added to the standard of care in patients with FSGS,” he said.
Dimerix noted that its study results were of a short duration which meant they weren’t statistically significant, although the study did allow the company to “derive maximum insight from a small number of patients, while retaining the ability for a flexible number of patients to complete the study”.
As such, Dimerix said the study delivered encouraging data, which meant further studies would be in development to investigate DMX-200 and FSGS.
“FSGS patients today face poor outcomes with limited medical options, and we continue to progress our proposed development pathway forward that could deliver the first approved pharmacologic treatment to the FSGS community,” Dimerix chief executive officer and managing director Dr Nina Webster said.
“In the meantime, we will soon be reporting on the larger diabetic kidney disease phase 2 study that we hope will further support the growing evidence of DMX-200 efficacy in treating kidney diseases,” she added.