#Spain #Valencia #ARTHEx Biotech #ATX-01 #RNA Medicines

ARTHEx Biotech Expands Series B Financing to $87 Million

ARTHEx Biotech, a clinical-stage biotech company known for its innovative work in developing targeted RNA medicines, has announced a significant boost to its Series B financing, now totaling $87 million. This round, which was led by the French national investment bank Bpifrance, also saw the participation of existing shareholders including AdBio Partners, CDTI Innovación, and Columbus Venture Partners among others.

The capital raised in this financing round will primarily support the advancement of ARTHEx's lead program, ATX-01, which focuses on treating Myotonic Dystrophy Type 1 (DM1). This condition is characterized by muscle weakness and is caused by the upregulation of microRNA-23b (miR-23b). ATX-01 is designed as an anti-miR oligonucleotide, targeting this microRNA and ultimately aiming to restore normal functioning of muscle cells. The funding will also facilitate broader clinical trials that have begun with the Phase I/IIa ArthemiRTM study.

Understanding ATX-01 and its Mechanism

ATX-01 functions by increasing the production of MBNL proteins, which are critical for normal muscle cell operation. In DM1 patients, the natural activity of MBNL proteins is inhibited by high levels of miR-23b. Notably, ATX-01 addresses this challenge by inhibiting miR-23b, promoting an increase in free MBNL levels while also reducing harmful DMPK mRNA levels, resulting in corrected splicing abnormalities common in DM1.

The extension financing is not only pivotal for ATX-01's development but also aimed at expanding ARTHEx's overall pipeline, which includes various other targeted RNA medicines that address high unmet needs in muscular, central nervous system (CNS), and cardiac conditions.

Support and Anticipation from Investors

Bpifrance has expressed strong optimism regarding ATX-01's potential, with Laurent Higueret, Deputy Director at Bpifrance's Large Venture Fund, stating that the results indicate ATX-01 could significantly alter the therapeutic landscape for DM1 patients. Additionally, Benoit Barteau, Investment Director at Bpifrance, has highlighted the dual action mechanism of ATX-01 as a promising feature that can address the disease comprehensively rather than merely alleviating symptoms.

The management team at ARTHEx, led by Chairman and CEO Frédéric Legros, believes this funding milestone is critical for propelling ATX-01 towards a registrational study, with hopes of demonstrating the drug's clinical potential. Legros emphasized that the company is well-positioned to enhance its pipeline, which aims to tackle numerous serious medical conditions with evident drug delivery challenges.

Clinical Development and Regulatory Designations

In preclinical models, including human DM1 myoblast cell lines and various mice models, ATX-01 has showcased significant molecular improvements alongside functional benefits. The ArthemiR™ study, which is currently active, has received necessary approvals, including Orphan Drug Designation from both the US FDA and European authorities, as well as Rare Pediatric Disease Designation from the FDA. This regulatory support further underscores the urgency and potential impact of ARTHEx's developments in the field of biotechnology.

With this new round of financing, ARTHEx Biotech is set to strengthen its commitment to advancing RNA medicines, positioning itself as a leader in the innovative treatment landscape for rare genetic disorders.