VALENCIA, Spain, Dec. 14, 2022 /PRNewswire/ -- Arthex Biotech S.A., a pre-clinical-stage biotechnology company pioneering innovative microRNA modulating approaches to diseases with high unmet medical needs, today announced that it has met key regulatory milestones in its program to develop ATX-01 in Myotonic Dystrophy Type 1 (DM1), opening a clear path forward to filing an Investigational New Drug (IND) application in the USA and a Clinical Trial Application (CTA) in Europe.
Following a successful pre-IND meeting with the Food and Drug Administration (FDA) in Sept 2021, Arthex filed a request for Scientific Advice from the European Medicines Agency (EMA) on the overall research program for ATX-01 in DM1 and on the planned first in human (FIH) Phase 1/2a clinical trial. The EMA confirmed the non-clinical and chemistry, manufacturing, and controls (CMC) data requirements for a successful CTA, and provided advice on the clinical trial design, as well as the overall development program. Importantly, no red flags or concerns were raised regarding the toxicology or rationale to support a FIH study. Arthex is planning to complete its CTA submission in 2023.
In separate applications, Arthex has obtained Orphan Drug Designation (ODD) from both the FDA and EMA for ATX-01 in the treatment of DM1, a rare disease. ODD status opens the door for significant benefits to the company, including protocol assistance, fee reductions, and some marketing exclusivity if the investigational drug is approved for the designated indication.
Beatriz Llamusí, Chief Executive Officer of Arthex, said "We now have a very good understanding of the requirements to launch the FIH study of ATX-01 in DM1 in both the US and Europe, which represents an important milestone for the company. We look forward to submitting both an IND in the US and CTA in Europe in 2023. In addition, the recognition by both agencies of the Orphan Drug status, allows us to optimise our interactions with regulators and comes with substantial financial incentives. We are delighted and eager to meet future milestones in developing ATX-01 for a community of patients with unmet medical needs."
ATX-01 is an investigational drug compound designed to address myotonic dystrophy, a rare progressive muscle wasting disease, by targeting a microRNA involved in the disease pathogenesis.
About Myotonic dystrophy type 1 (DM1)
Myotonic dystrophy type 1 (DM1) is a highly disabling disease affecting more than one million people worldwide. The condition affects muscles and other tissues (causing respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioural impairment. Most commonly, it manifests during adulthood, although DM1 can develop at birth in a congenital form or during childhood. Although signs and symptoms vary among affected individuals, sadly, with progression of the disease, DM1 patients experience a reduction in the ability to perform activities of daily living. Moreover, patients have a significantly shortened lifespan and there is no approved treatment to slow the progression of the disease.
About Arthex Biotech
Arthex Biotech is a preclinical-stage drug development company focused on improving the lives of patients with high unmet medical needs. Arthex Biotech is developing ATX01 an investigational drug compound designed to address myotonic dystrophy, a rare progressive muscle wasting disease, by targeting a microRNA involved in the disease pathogenesis. Arthex Biotech is also developing a complementary research portfolio focused on anti-microRNAs targeting other disorders. The company maintains its headquarters in Valencia, Spain. Arthex Biotech's investors are Invivo Capital, AdBio Partners, and the Centre for the Development of Industrial Technology (CDTI).