Tiago and Philip at the Newcastle Centre with Dregan on his last day of the AMO-O2 trial.


Cure DM is pleased to be in close contact with AMO, bringing up to date information to the community, straight from the horses mouth. We often see so much information on line that we dont know what to believe, everything we publish here has come direct from AMO or has been double checked with them to ensure accuracy.

If you have any questions or queries, you can contact us (curedm@outlook.com) and we will put it to the team. Alternatively you can always contact AMO directly, via their website here: http://www.amo-pharma.com/amo_02.htm


AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has potential for use in additional CNS, neuromuscular and oncology indications.

AM0-02 is positioned to enter clinical stage development for the treatment of the severe form of congenital myotonic dystrophy known as DM1 or Steinert disease.

In cellular and animal models of DM1 and Duchenne muscular dystrophy, as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to increase. Inhibitors of GSK3ß have been shown to correct the activity of regulatory proteins, such as CUGBP1 in animal models of DM1. AMO-02 is an inhibitor of GSK3ß that has demonstrated pre-clinical efficacy in transgenic models and reversal of muscle cell deficits in ex vivo tissue samples in patients with DM1.

Inhibition of increased levels of GSK3ß is known to reverse cognitive and behavioral deficits in transgenic models of syndromic autism, a developmental disorder characterized by social communication deficits and repetitive behaviors.            


After the positive findings from the recently completed trial, AMO is preparing to begin the next stage, which is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1)

You can read more on the study via the Clinical Trials page here:

We will keep you updated as we hear more via this page.


22 Dec 2020

AMO Pharma Announces Initiation of Pivotal REACH-CDM Clinical Trial for AMO-02 in Treatment of Congenital Myotonic Dystrophy


Exciting update for the AMO-02 REACH-CDM trial, in a time of uncertainty in the world.

Dr. Aravindhan Veerapandiyan,of Arkansas Children's Hospital, said “We are extremely excited to start enrolling patients for REACH-CDM, a pivotal therapeutic trial in the space of congenital myotonic dystrophy.

"The REACH-CDM study was designed based on the results of positive Phase 2 data that were recently published in the peer-reviewed journal Pediatric Neurology. In prior research, AMO-02 has been shown to access brain, muscle and other tissues and reduce the effect of DMPK expansion repeat in mRNA that is the pathological basis for congenital myotonic dystrophy."

What does this mean for the UK site? AMO are still keen to bring the trial to the UK community, and are still in discussions with the UK sites and the MHRA. It may feel dissapointing to those who are unable to enrol at this point, but things are moving on, and it is a positive step that reseacrh has begun at other sites. Whilst our UK Kids may not be able to take part at this moment in time, research is ongoing, and that is the most important thing.

Update 29th April 2019

Please email us (Emma@cmmd.uk) to receive more detail on plans for further studies that AMO Pharma has shared with Cure DM - We are in close contact with AMO Pharma who are presently fund raising to enable their planned next clinical study to take place and will update when we hear more from the company

Alternatively Mike Snape will be happy to receive emails directly on mike.snape@amo-pharma.com


AMO Pharma recently presented information on the development of the outcome measure that will be used in the next planned study that was agreed with regulators and clinical researchers in the field - there is a link on their website here:

News Release - April 16, 2019

AMO Pharma Announces Launch of First Clinician-Completed Rating Scale for Congenital Myotonic Dystrophy Type 1

"This is a major milestone for researchers looking to enhance our understanding of this disorder, drawing insight from the experiences of other patients and caregivers in a reproducible manner and incorporating advice from the FDA," said Dr. Horrigan. "We look forward to broadening the scope of this scale so that we may better address the current and future needs of this largely under served community."

You can read more via the press release Here!


News Release - October 29, 2018

AMO Pharma Reports Positive Data from Investigator-Led TIDE Study of AMO-02 in Autism Spectrum Disorder

Treatment associated with improvement in social withdrawal, repetitive behaviors, daily living skills, memory and sleep quality.

This Phase 2 study was conducted at three Canadian clinical trial facilities and assessed the safety and efficacy of AMO-02, a novel orally available GSK3 beta inhibitor, in adolescents with ASD between the ages of 12 and 18 years-old (n = 83). The once-daily treatment for the core symptoms of ASD was found generally safe and well-tolerated, with adverse event rates that were generally similar between tideglusib and placebo. There were no treatment-associated serious adverse events.

You can read more via the press release here.

News Release - October 23, 2018

AMO Therapeutics Announces Presentation of Concordant Analysis of Results of Phase 2 Study of AMO-02 in treatment of myotonic dystrophy

Treatment associated with improvement in cognitive function, fatigue and neuromuscular symptoms; results support continued clinical development.

You can read more on via press release here!



AMO Pharma Announces Initiation of Planned Pivotal Clinical Trial for Myotonic Dystrophy Following $35m Fund Raise  


Trial to assess AMO-02 in treatment of congenital myotonic dystrophy to begin patient enrollment in early 2020.

Drug trial to treat Congenital and Juvenile onset Myotonic Dystrophy - RECRUITING NOW!

The study is recruiting participants from the UK who are between the ages of 16 and 45 and have congenital or juvenile-onset myotonic dystrophy type 1 i.e. experienced symptoms before the age of 12.

More information can be found on clinicaltrials.gov.

We are OVER THE MOON to announce the FIRST drug trial for adults with Congenital/juvenile DM.

It is taking place at the John Walton Muscular Dystrophy Research Centre in Newcastle, UK, and is sponsored by the pharmaceutical company Amo Pharma.

The purpose of this study is to investigate the safety of a drug called tideglusib (AMO-02). Tideglusib inhibits an enzyme called glycogen synthase kinase 3ß (GSK3ß), which is over-active in people with myotonic dystrophy type 1. Preclinical research has shown that inhibiting GSK3ß can increase muscle strength and decrease myotonia.

In addition to safety, the study will also investigate the efficacy (i.e. potential benefits) and the pharmacokinetics (blood levels across time) of tideglusib.

The study is recruiting participants from the UK who are between the ages of 16 and 45 and have congenital or juvenile-onset myotonic dystrophy type 1 i.e. experienced symptoms before the age of 12. Participants must also be able to walk independently and meet a number of other criteria;

If you are interested in taking part in the trial, please get in touch with the trial sub-investigators: Dr. Nikoletta Nikolenko on 07870 517410 or email: Nikoletta.nikolenko@newcastle.ac.uk or Dr. Tiago Gomes on 0191 241 8989 or email: Tiago.Gomes@ncl.ac.uk