Tideglusib

-Four years of study data highlight continued favorable safety profile for AMO-02 -FDA meeting planned in Q4 2025 to discuss potential route to NDA submission for congenital myotonic dystrophy LONDON, Sept. 15, 2025 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held clinical-stage specialty biopharmaceutical company focusing on rare genetic disorders with limited or no treatment options, today announced a preliminary analysis of safety outcomes from the company's ongoing REACHCDM-X open-label extension (OLE) study of AMO-02 for the treatment of congenital and childhood-onset myotonic dystrophy type 1 (DM1). "We are pleased to share new findings that reflect the experience of patients treated with AMO-02 for almost four full years in our REACHCDM-X open-label extension study. These results include new insights indicating that AMO-02 continues to have an advantageous safety profile, and we were especially encouraged by the low rate of hospitalizations observed during the study," said Mike Snape, Chief Executive Officer at AMO Pharma. "We look forward to meeting with the FDA later this year to review the most recent data and our plans to advance AMO-02 for the treatment of congenital myotonic dystrophy as quickly as possible." Key findings from the REACHCDM-X OLE study of AMO-02 include: AMO-02 has been generally safe and well-tolerated over four years of study. As of August 2025, 45 participants remain on treatment, including 20 who have received AMO-02 for more than three years. A total of 14 participants discontinued treatment, with only one withdrawal due to an adverse event (elevated liver enzymes). Most adverse events were mild or moderate, with respiratory infections and gastrointestinal events being the most common. These are consistent with the known natural course of congenital and childhood-onset DM1. Across more than 151 patient-years of treatment exposure, participants treated with AMO-02 experienced a hospitalization rate of only 0.14 events per patient per year. No deaths or cardiovascular events have been reported. In a 10-meter walk/run test, a secondary endpoint in the trial, participants aged 10 years and older showed little or no decline over one year of treatment. AMO Pharma has submitted these additional data for AMO-02 to the U.S. Food and Drug Administration (FDA) and plans to submit to Health Canada and the UK Medicines and Healthcare products Regulatory Agency (MHRA). AMO Pharma is also scheduled to meet with the FDA in Q4 2025 to discuss the development plan for AMO-02 in the treatment of congenital myotonic dystrophy including assessments on the use of real-world evidence to support a regulatory submission. AMO Pharma has also submitted a protocol for a Phase 3 study in adult-onset myotonic dystrophy to the FDA and will share updates on this review, as well as on the outcomes of the upcoming FDA meeting and ongoing regulatory discussions. AMO Pharma is also planning meetings with UK MHRA and Health Canada. "We are very encouraged by these data, which significantly expand our understanding of the safety and potential clinical benefit of AMO-02 in congenital myotonic dystrophy," said Dr. Snape. "For families affected by congenital and childhood-onset DM1, this disorder is often associated with life-threatening complications. AMO Pharma is encouraged by these data, and we look forward to discussions with regulatory health agencies about the potential to evaluate real-world evidence outcomes that are highly relevant for this community, and may shape the nature of future investigations." The REACHCDM-X study, which began in August 2021 as an open-label extension of the double-blind REACHCDM trial, is the largest and longest-running interventional study ever conducted in congenital and childhood-onset myotonic dystrophy. The study has also expanded to include participants with childhood-onset DM1 up to 45 years of age and offers an optional extended access component, allowing patients to continue on long-term therapy. About AMO Pharma AMO Pharma is a clinical-stage specialty biopharmaceutical company working to identify and advance promising therapies for the treatment of serious and debilitating diseases in patient populations with significant areas of unmet need, including rare and severe childhood onset neurogenetic disorders with limited or no treatment options. In addition to developing AMO-02 for DM1, the company is also progressing AMO-02 as a clinical stage treatment for Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC). AMO-01 is being investigated for treatment of Phelan-McDermid syndrome and AMO-04 as a clinic-ready potential medicine for Rett syndrome and related disorders. AMO-02, AMO-01 and AMO-04 are investigational medicines that have not yet been approved for the treatment of patients anywhere in the world. Advice provided to AMO Pharma by regulators is under the condition that any scientific advice given is not legally binding with regards to any future application for the product concerned. Furthermore, advice cannot be taken as indicative of any future agreed position. For more information, please visit the AMO Pharma website at . Media Contact Holly Stevens CG Life [email protected] Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2 proof-of-concept trial with AMO Pharma investigative therapy AMO-02 now underway at 17 sites across Canada; 120 patients expected to enroll LONDON, Sept. 8, 2025 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held clinical-stage specialty biopharmaceutical company focusing on rare genetic disorders with limited or no treatment options, today announced a license agreement with Population Health Research Institute (PHRI), a joint global health research institute of McMaster University and Hamilton Health Sciences, and Venca Research Inc. The agreement will support the development and potential manufacturing, and commercialization of AMO-02 in arrhythmogenic right ventricular cardiomyopathy (ARVC). Recruitment is now underway with the first patient randomized in the PHRI TaRGET Phase 2 proof-of-concept clinical trial evaluating AMO-02 for the treatment of ARVC. The study will be the largest clinical trial of a therapeutic ever conducted in this rare, heritable cardiomyopathy. "AMO Pharma is committed to advancing and supporting research that can lead to new first-in-class disease-modifying therapies for serious, life-threatening conditions such as ARVC," said Mike Snape, Chief Scientific Officer at AMO Pharma. "The recent publication in JACC: Basic to Translational Science reinforces the growing body of evidence supporting glycogen synthase kinase-3 beta (GSK3β) inhibition as a potential treatment strategy for ARVC and the broader ACM spectrum, which is central to our approach with AMO-02. We look forward to partnering with PHRI to advance this landmark precision cardiology program, which has the potential to change the treatment landscape for patients living with ARVC by targeting the underlying disease biology. This collaboration builds on extensive prior research highlighting AMO-02's mechanism of action indicating the potential to correct abnormal GSK3β activity in the heart, and its established unique profile as a safe and well-tolerated GSK3β inhibitor." ARVC is a rare, inherited cardiomyopathy that can lead to heart failure, malignant ventricular arrhythmias, and sudden cardiac death. It is most often caused by genetic mutations in desmosomal genes that trigger abnormal activation of the enzyme GSK3β in cardiac cells. ARVC is a subtype of arrhythmogenic cardiomyopathy (ACM), a broader condition that can also affect the left ventricle or both ventricles. "In preclinical studies, insertion of human ARVC gene mutations into mice reproduced all the major signs and symptoms of the disease," said Dr. Jason Roberts, principal investigator of the study and PHRI scientist. "When studied in these models, AMO-02 reduced arrhythmias and cardiomyopathy, and both prevented progression and reversed existing disease features. These findings strongly underscore its potential to address the underlying drivers of ARVC and potentially offer a much-needed therapeutic option for patients." The TaRGET study is a randomized, double-blind, placebo-controlled trial evaluating AMO-02 in patients with genotype-positive ARVC. The primary endpoint is the change from baseline in mean premature ventricular contractions per 24 hours, measured by seven-day Holter monitoring compared to placebo at six months of treatment. Secondary endpoints include right ventricular strain on echocardiography, frequency of implantable cardioverter-defibrillator therapies, and episodes of sustained ventricular tachycardia. Under the terms of the agreement, AMO Pharma will work closely with PHRI on regulatory interactions and study oversight. First data from the trial are expected in the second quarter of 2027. About AMO Pharma AMO Pharma is a clinical-stage specialty biopharmaceutical company working to identify and advance promising therapies for the treatment of serious and debilitating diseases in patient populations with significant areas of unmet need, including rare and severe childhood onset neurogenetic disorders with limited or no treatment options. In addition to developing AMO-02 for DM1, the company is also progressing AMO-01 as a clinical stage treatment for Phelan-McDermid syndrome and AMO-04 as a clinic-ready potential medicine for Rett syndrome and related disorders. AMO-02, AMO-01 and AMO-04 are investigational medicines that have not yet been approved for the treatment of patients anywhere in the world. Advice provided to AMO Pharma by regulators is under the condition that any scientific advice given is not legally binding with regards to any future application for the product concerned. Furthermore, advice cannot be taken as indicative of any future agreed position. For more information, please visit the AMO Pharma website at . Media Contact Holly Stevens CG Life [email protected] SOURCE AMO Pharma Limited WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED