The company will present an update on the pivotal REACH-CDM clinical study currently underway to assess the efficacy and safety of the investigational therapy AMO-02 in the treatment of congenital myotonic dystrophy (CDM1). The update will cover timelines and answer questions from the DM community on this program.
AMO Pharma Limited (“AMO Pharma”), a privately held biopharmaceutical company focusing on rare, childhood onset neurogenetic disorders with limited or no treatment options, today announced that the company will participate in the “Meet the DM Drug Developers” webinar series sponsored by the Myotonic Dystrophy Foundation (MDF) on Friday, March 5, 2021 beginning at 3 PM EST/noon PST.
“AMO is very pleased to be participating in this webinar series sponsored by the MDF to bring important information about advances in research to patients, caregivers, clinicians and others who are interested in progress in the effort to develop new treatments for congenital myotonic dystrophy,” said Dr. Joseph Horrigan, AMO Pharma chief medical officer. “We look forward to updating on our progress in the REACH-CDM trial, which has commenced screening of patients at trial sites in Arkansas and California, and which is positioned to build new levels of momentum in the weeks ahead as more sites are up and running and enrolling patients.”
In August 2020, AMO Pharma published the results of a Phase 2 study of AMO-02 (tideglusib) in the treatment of childhood onset DM1, with data presented in the peer-reviewed journal Pediatric Neurology. In January 2021, the company announced the initiation of the REACH-CDM clinical study to assess the efficacy and safety of AMO-02, a dual action investigational mRNA modulating/GSK3ß kinase inhibitor, in the treatment of CDM1. For information about the REACH-CDM trial click here.
“We are especially pleased to be participating in this program with the MDF just after Rare Disease Day 2021, which works to build broader awareness of rare diseases such as DM1,” Dr. Horrigan said to Prittle Prattle New . “We look forward to sharing our update with members of the DM1 community and to answering questions about this important research effort.”
Through the MDF Meet the DM Drug Developers program, leading biotechnology, pharmaceutical and academic researchers working to develop treatments or cures for myotonic dystrophy are invited to share updates about their progress and answer questions for members of the myotonic dystrophy community. To register for the AMO Pharma update on March 5, 2021, visit https://www.myotonic.org/meet-dm-drug-developers-amo-pharma.
About AMO-02
AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has potential for use in additional CNS, neuromuscular and other orphan indications. AM0-02 is a clinical stage investigational medicine for the treatment of the severe form of congenital myotonic dystrophy known as DM1 or Steinert disease. In cellular and animal models of DM1 as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to increase. AMO-02 is an inhibitor that has been shown to normalise levels of GSK3ß in transgenic models and in ex vivo tissue samples in patients with DM1 and to reduce levels of the mRNA that is pathogenic for DM1.
About AMO Pharma
AMO Pharma is a 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 congenital myotonic dystrophy, 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. For more information, please visit the AMO Pharma website at http://www.amo-pharma.com/.
This article was shared to Prittle Prattle News as a Press Release.
By PR Newswire
