Posted: 10 September 2024
Dimerix Limited (ASX: DXB), a biopharmaceutical company with a Phase 3 clinical asset in kidney disease, today announced that the first patient has successfully completed the ACTION3 Phase 3 clinical trial, including the initial follow-up period, and has chosen to enter the global open-label extension (OLE) study. The OLE study is optional for all qualifying patients who have completed the ACTION3 Phase 3 clinical trial and offers a 2 year treatment with DMX-200. The OLE study will evaluate the longer term safety and efficacy of DMX-200 treatment in patients with focal segmental glomerulosclerosis (FSGS) in an open label (unblinded) setting and will run in conjunction with the Company’s existing blinded ACTION3 Phase 3 global clinical trial.
All patients in the OLE study will receive DMX-200, regardless of whether they received drug or placebo during the ACTION3 Phase 3 clinical trial. All patients in the OLE study will continue on the background standard of care blood pressure medication, angiotensin receptor blocker (ARBs) and will be treated for 2 years with DMX-200. Further information on Open Label Extension studies is outlined later in this announcement.
What is an Open Label Extension (OLE) study?
When a promising new drug is being tested for a serious problem, doctors and patients generally want the drug to be available outside the period of the randomised controlled trial. As there is inevitably a delay between the completion of the trial and the potential marketing approval, participants often want to continue treatment. Compassionate use schemes, such as the Special Access Scheme in Australia, which are programs that allow patients to access non-approved medications, are not considered research and as such data cannot be collected. In contrast, a properly designed and conducted OLE study can provide rigorous information on long term safety and tolerability of potential new drugs, as well as enable those patients who participated in the study to remain on drug. This in turn can benefit the licensing application for the drug by providing longer term data that would otherwise not be available until after the licence was approved.
All participants in the open label extension study are given the study drug, regardless of whether they were on drug or placebo during the blinded phase of the Phase 3 study, and so both they and the investigators know they are receiving the active drug (i.e. the open label study is “unblinded”).
What data are collected during DMX-200 Open Label Extension study
Data are collected periodically throughout the study. At the end of the OLE treatment period, the change in urine protein to creatinine ratio (UPCR) and the change in estimated glomerular filtration rate (eGFR), which are markers of drug efficacy, will be evaluated. Further, a comparison between those patients who were given placebo and those patients who received DMX-200 in the ACTION3 Phase 3 trial will also be made at the end of the OLE study.
The initiation of the open label extension study is another important milestone as we advance DMX-200 as a potential treatment to address the significant unmet need of patients with FSGS. The Open Label Extension study will provide us with additional longer term information regarding treatment with DMX-200, and we are pleased to be able to provide the active drug to all patients who complete the ACTION3 Phase 3 trial, irrespective of treatment arm. We are delighted that the investigators, participants and regulatory authorities endorse us moving forward into this important longer term study, supported by the strong safety profile to date of the drug. We acknowledge the FSGS patients and their families who continue to inspire us, as well as our investigators, partners and collaborators who provide invaluable assistance in our product development efforts. Working together with the renal community, we strive towards bringing this potential new and much-needed therapeutic solution for people living with FSGS.” Dr David Fuller, Chief Medical Officer, Dimerix
