Dimerix’s plans for China to be involved in its Phase 3 Focal Segmental Glomerulosclerosis (FSGS) trial have been given a boost after an update from the Chinese regulatory agency.

Dimerix (ASX:DXB) has announced that the National Medical Products Administration (NMPA) in China has confirmed that its FSGS single Phase 3 design is appropriate to support trials and potential registration in the country.

DXB’s Phase 3 global trial is titled Angiotensin II Type 1 Receptor (AT1R) & Chemokine Receptor 2 (CCR2) Targets for Inflammatory Nephrosis – or ACTION3 for short.

The NMPA reviewed a dossier that summarised DXB’s proposed ACTION3 clinical program and its supporting data in the form of non-clinical studies, the manufacturing and process controls and all existing Phase 1 and Phase 2 renal clinical data accumulated to date.

The written response from the NMPA acknowledged that the overall ACTION3 design was acceptable for China.

The NMPA said that DXB may not be required to conduct any further clinical studies before proceeding with the ACTION3 study in the Chinese population.

US$12.6 billion addressable market for rare kidney disease

FSGS is a rare disease attacks the kidney’s filtering units causing irreversible scarring, leading  to permanent damage and eventual end-stage kidney failure, requiring dialysis or transplantation.

For those fortunate enough to receive a kidney transplant, 60% of patients have recurrent FSGS after the first kidney transplant.

FSGS affects both adults and children as young as two years old.  There are presently no drugs specifically approved for FSGS anywhere in the world, leading to limited treatment options and poor prognosis.

The total global FSGS market was valued at US$12.6 billion in 2022 with a CAGR of 8.2%, driven by ~220,000  FSGS  sufferers  across  the  seven  major  markets and  premium  orphan  drug pricing.

DMX-200 has previously received Orphan Drug Designation with the FDA, EMA, and UK, allowing potential fast track of commercialisation if successful.

ACTION3 Trial offers hope to sufferers

ACTION3 is a pivotal multi-centre, randomised, double-blind, placebo-controlled trial of the efficacy and safety of lead drug DMX-200 in patients with FSGS who are receiving a stable dose of an angiotensin II receptor blocker (ARB).

The trial is actively recruiting across clinical sites globally, including in Taiwan and Hong  Kong.

Once the ARB dose is stable, patients aged 18 to 80 years will be randomised to receive either DMX-200 (120mg capsule twice daily) or placebo.

The trial broadened to 12 to 80 years following the first successful interim analysis.   In January the US confirmed inclusion of paediatric adolescent patients aged 12 to 17 was appropriate in the current global trial, recognising its appropriate safety profile.

China could be a key market

CEO and managing director Dr Nina Webster said the response from the NMPA is great news for DXB.

She said whilst the US has the advantage of high orphan pricing for FSGS treatment, China has many more patients with the disease and it could be a very key market for DXB.

“With over 100,000 patients estimated to be diagnosed in China, and no approved treatments, there is very high unmet medical need for new therapeutic options for this disease, which makes studies like this one critical,” she said.

“This formal response from the NMPA is the first step towards ensuring that the proposed FSGS development program meets with Chinese regulatory expectations for clinical trial and marketing approval, in addition to that feedback we have already received from the US and Europe.”

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.