Dimerix has opened its first paediatric site for ACTION3 in Mexico. Pic: Getty Images.
Health & Biotech
Special Report: Clinical-stage biotech Dimerix has achieved a key milestone with the first paediatric site opening for its ACTION3 Phase 3 clinical trial of lead drug DMX-200 to treat rare kidney disease focal segmental glomerulosclerosis (FSGS).
Dimerix (ASX:DXB) said the first clinical site specialising in paediatric kidney disease was activated today in Mexico, allowing for the active recruitment of adolescent patients into the ACTION3 Phase 3 clinical trial.
DXB said FSGS was one of the leading causes of kidney failure in children, with 20% of all presentations of Nephrotic Syndrome in paediatric patients caused by the disease.
Activation of the Mexico site followed the Independent Data Monitoring Committee (IDMC), and subsequently the US FDA and EMA – as part of DXB’s Paediatric Investigation Plan (PIP) – confirming dosage of DMX-200 to be used in adolescent trial patients aged 12-17 years would be the same as provided to adults (120 mg twice daily).
DXB said the determination was based on reviewing aggregate interim safety and pharmacokinetic data, including simulations in adolescents, from the adult cohort of the ACTION3 Phase 3 trial, taken at the first interim analysis point in March 2024.
Of the ~170 planned ACTION3 clinical sites, ~15 are specialist paediatric kidney centres across the UK, US, Mexico, Brazil, and Argentina, which have been selected to recruit a target population of about 22 adolescent patients with FSGS into the ACTION3 study.
DXB said patients in the “adolescent or paediatric cohort” of the ACTION3 Phase 3 clinical trial would also be blinded and randomised, meaning unknowingly placed on either placebo or DMX-200 and followed for a period of two years.
Should the paediatric cohort succeed, and DMX-200 be approved in adults, DXB may expand its approval for DMX-200 to market it to adolescents in key territories, including the US and Europe.
DXB said 116 patients out of the Part 2 target population of 144 have been randomised into the study with recruitment on track with timing of the interim analysis, expected around mid-2025, remaining unchanged.
The company said it was important to note that recruitment into clinical trials is not linear, and as previously advised, the recruitment rate is expected to increase with the initiation of ~100 new trial sites, bringing the total number of clinical sites to around 170 globally.
DXB said its ability to fund the ongoing ACTION3 Phase 3 clinical study remains strong with cash reserves of $22m at the end of the June quarter.
The company said an anticipated 2024 R&D tax incentive cash rebate of nearly $8m and expected exercise of 1.54 cent options (expiring June 2025), could bring in up to an additional $7.6m.
Furthermore, DXB said its cash position does not include potential milestone payments due under existing licensing arrangements or potential up-front payments from future licensing deals.
The company inked an exclusive license agreement with Taiba for the commercialisation of DMX-200 in several Middle Eastern countries in May.
DXB also signed a license deal with multinational pharmaceutical company Advanz Pharma, in October 2023 covering the European Economic Area, UK, Switzerland, Canada, Australia, and New Zealand.
The company said it continued to focus on licensing in other available territories, including the US, which could represent up to 50% of the global FSGS opportunity, as well as China.
To support the ACTION3 study’s broadening into paediatric patients, expert paediatric nephrologist Dr Howard Trachtman has been appointed to DXB’s medical advisory board.
Trachtman is a graduate of the University of Pennsylvania Medical School and completed a fellowship in paediatric nephrology at the Albert Einstein College of Medicine in New York.
The company said he has had a distinguished career, including roles as chief of the division of paediatric nephrology at Cohen Children’s Medical Center and NYU Langone Health, and has been the Principal Investigator of multiple NIH and industry-sponsored clinical trials specifically in FSGS patients.
His clinical research includes co-authoring more than 195 peer-reviewed articles on kidney disease in the past 15 years.
Additionally, Trachtman is a board member of the Kidney Health Initiative and serves on the editorial boards of several journals, including Kidney360, Glomerular Diseases, and Paediatric Nephrology.
DXB chief medical officer Dr David Fuller was an important step towards providing a potential new treatment for children with FSGS.
“The initiation of the first paediatric site in Mexico marks a new milestone for the ACTION3 study,” he said.
“We are delighted that we can now draw on the expertise and professional networks of Dr Trachtman as we continue to focus on the recruitment goals of this pivotal study.”
Trachtman said FSGS remained an area of huge unmet need with similarly poor outcomes in both children and adults.
“There are still no approved therapies to delay or prevent progression to kidney failure,” he said.
“While DMX-200 remains experimental in this area, it appears very well suited to use in patients of all ages, particularly as it is taken orally and is well tolerated.
“Based on data to date, it is a drug candidate that is certainly worth testing in all patients with FSGS.”
It’s been a good week for DXB, which also announced that it had enrolled the first patient into the Open Label Extension study for DMX-200 after completing the ACTION3 trial.
The OLE study allows patients who have successfully completed the ACTION3 Phase 3 clinical trial, to optionally start or continue a two-year treatment of DMX-200 in an open label (unblinded) setting.
This article was developed in collaboration with Dimerix, a Stockhead advertiser at the time of publishing.
This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.
Health & Biotech
Health & Biotech
Health & Biotech
