The ARTHEx Biotech spin-off, located in the scientific-business area of the University of Valencia Science Park (PCUV) and specialized in the development of targeted RNA therapies for rare genetic diseases, has announced the closure of an expansion of its Series B round to a total of $87 million. The operation has been led by the French fund Bpifrance and has had the support of all current investors, including the Valencian fund Columbus Venture Partners, leader of the initial Series B, which was announced last 2023 and amounted to 42 million. This represents a new capital injection of $45 million. In addition, Columbus recently made another investment to PCUV biotech company Highlight Therapeutics, which is working on the development of a compound for the treatment of basal cell carcinoma.
According to the company, the funds raised will help advance the international clinical development of ATX-01, the company’s most advanced therapy for Type 1 Myotonic Dystrophy (DM1), as well as the preparation of an advanced phase clinical trial for registration. In addition, the funds will support the expansion of its RNA therapy roadmap into other muscular, cardiac and neurological diseases with high unmet medical need as rare diseases.
"The ARTHEx Biotech approach to DM1 has shown convincing results both in vitro and in vivo. The dual mechanism of action of ATX-01 offers real potential to become the reference treatment for the disease. In addition, the company’s management platform has been shown to reach multiple affected tissues, which makes it possible for ATX-01 to treat the disease as a whole and not just muscle symptomatology," the company explains.
In anticipation of the first clinical data to be made public in 2026, Frédéric Legros, CEO of ARTHEx Biotech, says that the Science Park company "is in a strong position to move towards a clinical trial for registration in DM1". For Bpifrance, this is not only the investment that will complete the development of DM1 therapy, but also make precision RNA drugs available to patients with other conditions without cure. "It is very exciting to partner with ARTHEx at this point as the company reaches clinical proof of concept, and we look forward to supporting Frédéric and his team on their way to building a leading precision RNA drug franchise", says Laurent Higueret, deputy director of the Large Venture fund at Bpifrance.
"ARTHEx’s core technology for modulating microRNAs has shown great potential from the outset. In this round, in addition to confirming the vision we had just 2 years ago, we see how the company is building a pipeline capable of addressing the cure for other diseases with an enormous unmet medical need," says José Mesa, partner at Columbus Venture Partners.
ATX-01 is an oligonucleotide designed to inhibit microRNA-23b, which acts as a natural brake on the MBNL protein, key to muscle function. By blocking this microRNA, ATX-01 increases MBNL production and reduces levels of the toxic DMPK RNA responsible for Myotonic Dystrophy Type 1 (DM1). In doing so, it succeeds in restoring muscle function in animal models and offers real hope to patients.
DM1 is the most common form of muscular dystrophy in adults, with an estimated prevalence of between 1 and 11 cases per 100,000 inhabitants according to European clinical studies. It is a genetic disease, progressive and multisystemic, which causes muscle weakness, heart, respiratory and cognitive problems, for which there are currently no approved treatments that change the course of the disease.
ARTHEx Biotech S.L. is a spin-off company of the University of Valencia located in the UV Science Park, focused on the development of a treatment against muscular dystrophy type 1 based on microRNA inhibitors. The proof of concept of the efficacy of these molecules in animal models of the disease was generated at the translational genomics laboratory of the University of Valencia.
The founders of this company are Beatriz Llamusí and Ruben Artero. The business model proposed to complete this project corresponds to the current open innovation model, in which the biotechnology company emerging from a research centre develops products up to clinical phase 2 (efficacy), and then license them to pharmaceutical multinationals with the ability to complete development, more advanced studies and their commercialization. In addition, last June the company was awarded at the Santander X Spain Awards in the category of best startup.
Don’t miss Chapter 8 of our podcast "La Bombilla de Livermore" with Beatriz Llamusí, CSO of ARTHEx Biotech
Source: Valencia Plaza