Adarx to use $200M in financing to advance its clinical programs
Funding to support testing of mRNA therapy for HAE, other diseases
Adarx Pharmaceuticals will use $200 million in oversubscribed Series C financing to further develop its clinical programs — including ADX-324, its treatment candidate for hereditary angioedema (HAE) — the clinical-stage biotechnology company announced.
The funding round was led by Bain Capital Life Sciences and TCGX, an investment firm focused on innovative drug discovery. Also participating were a number of new and existing investors.
In addition to ADX-324, Adarx will use the proceeds from the financing to advance its experimental therapy ADX-038 for multiple complement mediated disorders. The funding will generally support development of the company’s novel mRNA silencing or editing therapeutic candidates for a broad range of conditions.
“The strong support from our new and existing investors is a testament to the significant progress we have made in advancing next generation RNA therapeutics,” Zhen Li, PhD, president and CEO of Adarx Pharmaceuticals, said in a company press release.
ADX-324 is now being tested in a Phase 1 clinical trial (NCT05691361) involving HAE patients and healthy volunteers.
Adarx trial testing ADX-324 now underway in Australia
The trial, which is assessing the safety and pharmacological properties of ADX-324, has two parts. In the first part, healthy people will receive up to six doses of ADX-324, administered as a single subcutaneous or under-the-skin injection. In the second part, a dose to test in HAE patients will be chosen based on part one results.
The first group of participants — the healthy volunteers — was dosed in January. Trial recruitment continues at the single South Australian study site.
“The early Phase 1 clinical data strongly supports the potential of ADX-324 to be the best-in-class treatment for patients suffering from hereditary angioedema,” Li said.
“With this financing, we are well-positioned to achieve our clinical milestones for multiple clinical programs and to build a leading company in RNA therapeutics,” Li added.
HAE is caused by mutations that result in the excessive production of bradykinin, a signaling molecule that triggers the swelling attacks that characterize the disorder. ADX-324 aims to mitigate swelling by reducing bradykinin levels. Specifically, ADX-324 is designed to impede the production of a protein called kallikrein, which plays an instrumental role in bradykinin production.
When a protein in produced, the genetic code that houses the instructions for building that protein is copied from a cell’s DNA to a temporary molecule called messenger RNA (mRNA). The cell’s ribosomes — protein-producing machinery components — then use that mRNA molecule as a template for protein construction.
As a small interfering RNA, ADX-324 can prime mRNA molecules with a certain genetic sequence for destruction. ADX-324 is specifically designed to target the mRNA for prekallikrein, a protein precursor to kallikrein.
The early Phase 1 clinical data strongly supports the potential of ADX-324 to be the best-in-class treatment for patients suffering from hereditary angioedema.
“We are impressed by the progress that Adarx has made in developing RNA therapeutics and their recent clinical data,” said Chen Yu, PhD, managing partner of TCGX, adding, “We believe that their technology and platform have the potential to transform the treatment of a wide range of diseases. We are excited to partner with the Adarx team to help bring their medicines to patients.”
Around the time ADX-324 first entered the clinic, Adarx announced the close of a $46 million Series B-1 financing round, to be used to further develop its pipeline of experimental treatments focused on genetic, cardiometabolic, and central nervous system disorders. The company now is developing nine active programs.
“Adarx has developed a unique siRNA platform that we believe has the potential to generate differentiated clinical data from several programs over the near and long-term,” said Ricky Sun, PhD, partner at Bain Capital Life Sciences.
“We share the company’s commitment to targeting diseases across therapeutic areas where there is significant unmet medical need and look forward to supporting the advancement of their platform technology and growing drug development pipeline, with the ultimate goal of bringing important new treatments to patients,” Sun said.