Dimerix (DXB) CEO Nina Webster had encouraging news to share regarding the status of DXB's ACTION3 trial at the 17th annual Bioshares Biotech Summit. The pivotal Phase III trial is a randomised, double-blind, placebo-controlled trial of the efficacy and safety of DMX-200 for the treatment of Focal Segmental Glomerulosclerosis (FSGS), a rare form of kidney disease.
Webster emphasised that FSGS has a "really poor prognosis", with no approved treatments yet available and even transplant being rather ineffective, with 60% of transplant patients experiencing reoccurring FSGS. Left untreated, FSGS leads to kidney failure within five years after the onset of proteinuria. The disease affects children as young as two years of age, with Dimerix now including children 12 years and older into its study.
DXB is the "first mover" in the FSGS space, with DMX-200 the "only treatment in a Phase III clinical trial anywhere in the world". Dimerix's competitors, including giants Vertex Pharmaceuticals and Boehringer Ingelheim, are "a number of years behind", only just commencing Phase II trials.
The ACTION3 trial is composed of three parts, with interim data set to be released after the conclusion of Parts 1 and 2. Due to DMX-200's orphan drug status, Dimerix is eligible to seek accelerated marketing approval prior to the conclusion of the study given that the Part 2 interim data is positive.
Webster lauded the trial as a "truly global study", with part 1 of the study currently in 70 sites across 11 countries and Part 2 set to include a further six countries. The most important of these additions is China, the biggest market globally for kidney disease.
Webster stated that Dimerix had been in contact with the Chinese regulatory body, who considered the trial to be acceptable for approval given the high unmet need. If Dimerix can ensure that 10% of the trial cohort is Chinese, the drug will be eligible for approval in China without any supplementary trials.
Webster reported that all 72 patients involved in Part 1 of the study have been recruited and randomised to receive drug or placebo. Patient data collection is scheduled to conclude February 26th 2024, with the first interim data outcome set for the 15th of March 2024.
With approval very much a possibility within the next few years, the company is searching for potential partners. Despite tough macroeconomic conditions, Webster claimed that in terms of partnership and licensing deals things are "business as usual", citing some "very lucrative deals in the kidney disease space" such as AstraZeneca's acquisition of Cincor Pharma this year for US$1.8 billion. Also of particular relevance is one of Dimerix's competitors, Chinook Therapeutics, which Novartis announced in June this year it would acquire for US$3.2B (now completed). Chinook's lead kidney disease treatment program is in a Phase III study.
Webster summarised some of the recent deals in the kidney disease space for novel drug candidates. Since May last year, seven global deals have been completed with upfront payments of US$10 million (on market), US$15 million (Phase II), US$30 million (preclinical), US$55 million (in Phase III), and US$70 million (Phase I), including one undisclosed upfront payment deal (preclinical).
The company is considering both global and regional partners, with Webster noting that while global partners are convenient, giving DXB one partner through which they can distribute DMX-200 universally, regional partners are often "more specialist in the territory they're working in, particularly in orphan disease where it is quite a unique challenge with regulatory agencies". She also posits that smaller partners are more likely to "have a greater focus on the product because it becomes one of their key pipeline opportunities".
Webster was quick to assert that Dimerix is more than capable of taking DMX-200 through to an NDA, but that a partner would help greatly to "get the product to the patient". Beyond distribution services, the company is looking for partners with local regulatory and marketing expertise.
Webster highlighted the importance of working with the right partner: "The relationship we're looking for is not a quick transaction, it's a long-term, 10-to-20-year relationship." Reflecting on the company's progress so far in locking down a licensing deal, she stressed that "building up a champion in the other company has been critical over the last 12 months."
Webster said that the company is in detailed contract negotiations with potential partners for DMX-200 with non-binding term sheets currently on the table. Dimerix is capitalised at $31.5M with a cash position of $8M at the conclusion of the financial year.
Bioshares recommendation: Speculative Buy Class A