This phase 2 exploratory study was designed to examine the safety and tolerability of two doses of AMO-02 in adults with congenital and juvenile onset myotonic dystrophy. The study is also examining signals of efficacy and the potential utility of several outcome measures. Two cohorts of eight subjects (16 total subjects) were planned, with the first cohort receiving 1000mg/day AMO-02 and the second cohort receiving 400mg/day AMO-02. This update concerns the data obtained from the first 1000mg/day AMO-02 cohort and will be presented at the American Neurological Association's (ANA) 2017 Annual Meeting October 15th 2017.
In cohort one, subjects with an average age of 22 years, six of whom had congenital-onset myotonic dystrophy, underwent once daily oral treatment with 1000mg AMO-02. Treatment was generally safe and well tolerated, and no subjects discontinued treatment due to adverse events or other issues. The data show multiple endpoints with favorable changes that were statistically significant. The changes seen primarily related to central nervous system symptoms, autistic features and activities of daily living. Although some analyses are still underway, including an examination of the relationship between blood levels of AMO-02 and clinical response, AMO Pharma has been encouraged to progress forward with a subsequent, larger multi-site study in children and adolescents to be conducted in the US, Canada and the UK.
"AMO Pharma would like to thank the families taking part in this study, as well as the staff at Newcastle and all our partners in this work. Without their contribution we would not have been able to gather these data and we truly appreciate all the efforts that they have made. I am very proud that we can report encouraging preliminary data for families affected by congenital myotonic dystrophy, for whom no sponsor led studies have previously been conducted. We are now working to complete the second cohort of this study and finalize planning of our next study in children and adolescents with this condition. We look forward to making further announcements regarding our next steps," said Michael Snape, chief executive officer of AMO Pharma.
"We have been pleased to conduct this study," said Prof Hanns Lochmuller, principal investigator for the study. "This has been a neglected area and so it is important to expand our knowledge of clinical study methodologies in this patient population. The participants, their families and caregivers came from across the UK and were recruited through the joint efforts of Newcastle University and Hospital Trust, the UK patient registry and the patient organizations MDUK and MDSG, showing that efficient recruitment can be achieved and trials conducted in this rare disease."
About AMO Pharma
AMO Pharma is a biopharmaceutical company incorporated in February of 2015. The co-founder, Dr. Michael Snape, has extensive experience in senior scientific and operational roles in both large pharma and biotech companies spanning more than twenty five years, and has brought together a targeted and experienced senior management team with a proven track record of success in all phases of product development and acquisition. The company is 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, debilitating childhood onset neurogenetic disorders with limited or no treatment options. For more information, please visit the AMO Pharma website at http://www.amo-pharma.com/.
About congenital myotonic dystrophy
Congenital myotonic dystrophy (CDM1) is a genetic disease that presents itself at birth. Patients born with CDM1 might have deformed feet, might not be able to breathe or feed properly and might be considered 'floppy' as an infant due to delayed development of muscles. It can affect many parts of the body and can lead to significant physical and cognitive impairment and, in some cases, early death. The most challenging symptoms of CDM1 include:
- difficulty with thinking and problem solving;
- weakened muscles; and,
- speech, hearing and vision difficulties.
Diagnosis is confirmed though a diagnostic evaluation or genetic test. There are currently no approved therapies for CDM1 and patients are typically treated with drugs to address different symptoms of the disease. Many patients also receive support through special education and speech and physical therapy.
Mike Snape, PhD
Chief Executive Officer
AMO Pharma Ltd.
+44 1483 898 448
Berry & Company Public Relations
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SOURCE AMO Pharma Limited