ARTHEx Biotech obtains 42 million euros to advance treatment of myotonic dystrophy

29/05/2023
  • The company located in the Science Park of the University of Valencia has just announced the closing of a financing round of 42 million euros to progress with its compound ATX-01, a new drug for the treatment of myotonic dystrophy type 1 (DM1)
  • ARTHEx expects to initiate the clinical trial in the second half of 2023. The round is led by the venture capital firm Columbus Venture Partners

ARTHEx Biotech S.L., a clinical-stage biotechnology company located at the University of Valencia Science Park (PCUV), is focused on the development of innovative drugs through microRNA modulation. The company's lead investigational compound -ATX-01- is progressing towards its clinical phase for the treatment of myotonic dystrophy type 1 (DM1), a rare disease that causes progressive muscle wasting.

The funding obtained will now be used to advance the development of the compound, a drug designed against microRNA 23b in a clinical trial (Phase I/IIa) for the treatment of this devastating and rare neuromuscular disorder which, in addition to muscle weakness, causes various disabling complications in sufferers. There are currently no treatments capable of modifying the course of DM1, which affects at least 40,000 people in the U.S. and 70,000 in Europe.

The Series B financing round, led by the venture capital firm Columbus Venture Partners, included the participation of the European Innovation Council (EIC), Hadean Ventures, Sound Bioventures and previous investors Invivo Capital, AdBio Partners and CDTI.

"This funding allows us to translate the scientific project for which the company was founded into the clinic, and we have tremendous confidence in our approach to realize the new therapeutic potential of microRNAs," Beatriz Llamusí, chief scientific officer (CSO) and co-founder of ARTHEx Biotech

"This funding allows us to translate the scientific project for which the company was founded into the clinic, and we have tremendous confidence in our approach to realize the new therapeutic potential of microRNAs," said Beatriz Llamusí, chief scientific officer (CSO) and co-founder of ARTHEx Biotech.

José Mesa, partner at Columbus Venture Partners, added that "ARTHEx's foundational microRNA modulation technology shows great promise for identifying new forms of treatment, blocking or activating key targets that trigger disease. We believe we have a tremendous opportunity to develop a new therapy in DM1, an orphan disease with a significant number of patients worldwide."

Frédéric Legros, Chairman of the Board of ARTHEx and new CEO of the spin-off, said he was "excited to lead ARTHEx Biotech alongside a successful executive team, board of directors and syndicate of investors, who share our commitment to developing innovative medicines for disabling diseases. This will be a pivotal year as we prepare to initiate the ArthemiR™ Phase I/IIa study with lead compound ATX-01 for DM1 in the second half of 2023."

"We believe we have a tremendous opportunity to develop a new therapy in DM1, an orphan disease with a significant number of patients worldwide," Columbus Venture Partners partner José Mesa

Origin of the spin-off

Arthex Biotech is, since 2020, a spin-off of the Universitat de València born at the end of 2019 from the Translational Genomics research group headed by Professor of Genetics Rubén Artero. His team previously discovered a new therapeutic target for DM1 and invented molecules that allowed to recover symptoms of the disease. The invention was patented and the proof of concept, in an animal model, was published in the journal Nature Communications. UV subsequently licensed the patent to ARTHEx for exploitation.

ARTHEx is also working on drug discovery to identify and develop microRNA modulators for other disorders with high unmet medical needs, including genetic diseases such as DM1. The headquarters of this spin-off company of the Universitat de València is located in the Parc Científic of the academic institution.