AMO Pharma presented positive interim analysis of data from the first cohort of its study of AMO-02 in congenital myotonic dystrophy.
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LONDON and PHILADELPHIA, Oct. 18, 2017 /PRNewswire/ -- 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 About congenital myotonic dystrophy
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 Media: SOURCE AMO Pharma Limited |
