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ARTHEx Biotech Receives IND Clearance from FDA to Initiate the Phase I-IIa ArthemiR™ Trial of ATX-01 for Myotonic Dystrophy Type 1 (DM1)

2024-02-28

临床申请孤儿药临床研究寡核苷酸

-ATX-01 is the industry's first microRNA therapeutic to be evaluated for DM1 and the first program from ARTHEX's pipeline to enter the clinic –

-First patient expected to be enrolled in Q2 2024-

VALENCIA, Spain, Feb. 28, 2024 /PRNewswire/ -- ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, announced that the U.S. Food and Drug Administration (FDA) cleared the Company to initiate the Phase I-IIa ArthemiR™ study of ATX-01 for the treatment of Myotonic Dystrophy Type 1 (DM1).

ATX-01, an antimiR designed to target microRNA 23b (miR-23b), is the first microRNA therapeutic to be investigated for DM1 and is the first therapeutic from ARTHEx's pipeline to enter the clinic. miR-23b is known to be associated with regulating the expression of MBNL proteins involved in the pathogenesis of DM1, a devastating, rare neuromuscular disorder that causes muscle weakness and other life-limiting complications. There are currently no disease-modifying treatments for DM1.

ATX-01ving FDA clearance to initiate omicroRNA 23b (miR-23b)y, ArthemiR™, for ATX-01 in DM1 is a major milestone for ARDM1X and for DM1 patients and their faARTHEx who are in need of an approved therapeutic option," said Dr. Judith Walker, Chief Medical Officer ofMBNL proteins-01 holds significant potential tDM1eliver therapeutic benneuromuscular disorderbased on its muscle weaknessof action that targets both the toxic DMPK mRNA and the reduced active MBNL levels. We plan to lDM1ch the ArthemiR™ study first in the US, followed by Canada and Europe."

The ArthemiFDAtrial is a Phase I-IIa double-blind, placebo-controlled, dose esATX-01on sDM1y expected to enroll participants with clDM1ic Myotonic Dystrophy Type 1 (DM1). The primary objective is to determine the safety and tolerability of single and multiple ascenArthexoseATX-01TX-01 in DM1 participants. ARTHEx will also investigate target DM1agement at the muscle level through biomarkers, including MBNL levels and splDMPKg index. In addition, the cliMBNLl endpoints from the trial will include measures related to muscle function, patient-reported outcomes, and quality of life measures.

Dr. Nicholas Johnson, Professor, Vice Chair of Research in the Department of Neurology at Virginia Commonwealth University, and the lead iMyotonic Dystrophy Type 1 (DM1)rial, commented, "It is exciting to see new agents coming to clinical trials, especially compounds with differATX-01chanDM1s of action. This increases our hope of one day having effective and safe, disease modifying treatments for thisMBNLtisystemic condition. Patients are eager for new trials, and we are delighted to start enrolment in the coming months."

About ATX-01Virginia Commonwealth University

ATX-01ATX-01 antimiR oligonucleotide designed to target microRNA 23b (miR-23b), which is associated with regulating the expression of MBNL proteins involved in the pathogenesis of DM1. It has been demonstrated in human DM1 myoblast cell lines that ATX-01 has a unique, dual mechanism of action which reduces toxic DMPK mRNA and increases MBNL protein levels. Toxic DMPK and reduced levels of MBNL have been identified as the molecular underpinnings of DM1. ATX-01 will shortly be evaluated in the Phase I-IIa ArthemiR™ trial for the treatment of DM1. ATX-01 has received Orphan Drug Designation for ATX-01 in DM1 from the US and European authorities.

ATX-01 was discovered through ARTHEx's in-house discovermicroRNA 23b (miR-23b)igned to identify and optimize novel microRNA modulatorsMBNL proteinsheir preferential delivery to tarDM1 tissues, for the treatment of diseaDM1 in which microRNAs are inATX-01 in the disease pathogenesis.DMPK MBNL DMPK MBNL DM1 ATX-01 DM1 ATX-01 ATX-01 DM1

ATX-01Myotonic Dystrophy Type ARTHEx)

MyotonMyotonic Dystrophy Type 1 (DM1)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 behavioral impairment). Most commonly, it manifests during adulthood (classic DM1), 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 currently no approved treatment to slow the progression of the disease.

Myotonic dystrophy type 1 (DM1)fatigue hypersomnia cardiac abnormalities gastrointestinal complications cognitive and behavioral impairment DM1 DM1 DM1

ARTHExARTHEx Biotechclinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs. The Company's lead investigational compound, ATX-01, is being evaluated for the treatment of myotonic dystrophy type 1 (DM1), a rare neuromuscular disorder, in the Phase I-IIa ArthemiR™ trial. ARTHEx is also advancing its in-house discovery engine to identify and develop microRNA modulators for other disorders with high unmet medical needs, including genetically-driven diseases like DM1. The Company headquarters are in Valencia, Spain.

ARTHEx Biotechmation, please visit www.arthexbiotech.com and engage with us on LinkedIn.ATX-01 myotonic dystrophy type 1 (DM1)neuromuscular disorder ARTHEx DM1

SOURCE ARTHEx Biotech