AMO Pharma Presents Positive Interim Analysis of Data from Congenital Myotonic Dystrophy Study at American Neurological Association Annual Meeting

Wednesday, October 18, 2017 Drug News
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LONDON and PHILADELPHIA, Oct. 18, 2017 /PRNewswire/ -- AMO Pharma Limited ("AMO

Pharma"), a privately held biopharmaceutical company focusing on rare, debilitating childhood onset neurogenetic disorders with limited or no treatment options, presented positive interim analysis of data from the first cohort of its study of AMO-02
in congenital myotonic dystrophy. Results, which were presented previously, were reviewed in a poster session during the American Neurological Association's (ANA) 2017 Annual Meeting on October 15th 2017.

"The interim analysis provides strong further support indicating that AMO-02 performs well against multiple endpoints with favorable changes that were statistically significant," said Michael Snape, chief executive officer of AMO Pharma. "We look forward to advancing the development program for AMO-02 and remain committed to bringing a new treatment option to patients affected by congenital myotonic dystrophy patients, which remains a significant area of unmet need in healthcare around the world." 

The presentation, titled "Development of the Clinician Domain-Specific Causes for Concern: Myotonic Dystrophy Rating Scale", includes an updated analysis of data from the first AMO-02 phase IIa study cohort. The 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. Two cohorts of eight subjects (16 total subjects) were planned, with the first cohort receiving 1000mg/day of AMO-02 and the second cohort receiving 400mg/day of AMO-02.

The subjects in the first cohort, six of whom had congenital-onset myotonic dystrophy, underwent once daily oral treatment with 1000mg AMO-02. Data show positive changes related to central nervous system symptoms, autistic features and activities of daily living. Based on results of clinical research thus far, AMO Pharma is positioning AMO-02 to advance to larger multi-site clinical trials in the US, Canada and the UK.

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

About congenital myotonic dystrophyCongenital 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:

  1. difficulty with thinking and problem solving;
  2. weakened muscles; and,
  3. 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.

Contacts  Corporate: Mike Snape, PhDChief Executive OfficerAMO Pharma Ltd.+44 1483 898

Media: Sara Zelkovic Berry & Company Public Relations 212.253.8881

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SOURCE AMO Pharma Limited

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