AMO Pharma Participates in Workshop on Central Nervous System Involvement in Progression and Treatment of Myotonic Dystrophy During Myotonic Dystrophy Foundation Annual Conference


LONDON and PHILADELPHIA, Sept. 16, 2019 /PRNewswire/ -- 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 Joe Horrigan, MD, AMO Pharma's chief medical officer, presented an update on the company's research targeting use of CNS therapies in the treatment of myotonic dystrophy (DM) during a full-day workshop on CNS involvement in DM held during the Myotonic Dystrophy Foundation Annual Conference in Philadelphia, PA on September 13, 2019.

During the workshop, experts in research and clinical care examined the natural history of DM1, DM2 and congenital myotonic dystrophy (CDM) and the underlying scientific premise for targeting therapy development to address the CNS phenotype, a key contributor to overall burden of disease. Through platform presentations and focused discussion sessions, participants also worked to identify targetable CNS phenotypes, evaluate the current knowledge of the underlying disease mechanisms and molecular targets, and discuss what biomarkers and outcome measures might best demonstrate the clinical effectiveness of drugs and biologics in patients living with DM1, DM2 and CDM.

"Increasingly, leaders in research are better understanding the role that the central nervous system plays in both the progression and potentially the treatment of myotonic dystrophy," said Dr. Horrigan.  "We were very pleased to join in this important meeting and share an update on our progress in development of AMO-02 and the applications of the first ever clinician-completed rating scale to assess features of congenital myotonic dystrophy type 1 (CDM1).

In mid-2018, AMO Pharma announced results from a concordant trend analysis of a Phase 2 clinical study of AMO-02 for the treatment of congenital and childhood onset DM1. Study results indicated that treatment with AMO-02 was associated with a range of clinical benefits.  Strongest improvements were recorded in cognitive functioning, fatigue, neuromuscular symptoms and ability to perform daily tasks of living. Assessments of efficacy included clinician-completed and caregiver-completed rating scales.

In April 2019, AMO Pharma announced presentation of a new clinician-completed rating scale to assess features of CDM1. The fit-for-purpose scale identifies 11 observable characteristics of CDM1 and rates each on a four-point Likert scale using information from natural history studies, consultation with therapeutic area experts and feedback from regulatory agencies. An evaluation of the scale's use in clinical research found it has potential to effectively assess the CDM1 phenotype in a low-burden manner, is change-sensitive, can detect treatment effects and may provide useful outcome measure for future clinical trials.

"In terms of both our understanding of the role of CNS phenotype in the burden of DM and the ability to assess and quantify changes in observable characteristics of the disease in patients, we have made significant progress in recent years," said Michael Snape, chief executive officer of AMO Pharma.  "We are very encouraged by the results of our development program for AMO-02 thus far and look forward to advancing this important clinical program in the months ahead."

About congenital myotonic dystrophy
Congenital myotonic dystrophy (CDM1) is a genetic disease that typically presents at birth. People living with CDM1 typically experience weakened muscles, difficulty with thinking and problem solving, and speech, hearing and vision difficulties. Patients are at risk of early death. 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.

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. AMO-02 is an investigational medicine that has not yet been approved for the treatment of patients anywhere in the world.  For more information, please visit the AMO Pharma website at


Mike Snape, PhD
Chief Executive Officer
AMO Pharma Ltd.
+44 1483 898 448

Bill Berry
Berry & Company Public Relations

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SOURCE AMO Pharma Limited


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