AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on rare, debilitating childhood onset neurogenetic disorders with limited or no treatment options, today announced the presentation of final data from the company's phase 2 proof-of-concept study of AMO-02, an investigational therapy for treatment of congenital and childhood onset myotonic dystrophy type 1. Results were presented by Joseph Horrigan MD, AMO Pharma's chief medical officer, in a poster session and podium presentation during the Muscular Dystrophy Association Conference on March 13 and 14, 2018 in Arlington, Virginia.

In the study, AMO-02 was shown to be safe and well tolerated, with no early discontinuations or dose adjustments required. Most patients treated with AMO-02 also showed clinical benefits including improvements in cognitive function, levels of fatigue and the ability to perform daily tasks. There was a demonstrated greater response in patients who received 1000mg/day of AMO-02 versus the 400mg/day of AMO-02 dose. Results also showed co-occurring autism symptoms improved in several AMO-02 patients.

"These significant data are an important step in the development of AMO-02 as a potentially safe and effective treatment option for many patients living with congenital and childhood onset myotonic dystrophy type 1," said Michael Snape, MD, chief executive officer of AMO Pharma. "We look forward to advancing the clinical development program for AMO-02 and are grateful to the clinicians, caregivers and patients who participated in this landmark trial."

Myotonic dystrophy type 1 is a rare, genetic, life-threatening neuromuscular disorder that causes impairment in muscle function, cognition and quality of life. The phase 2 proof-of-concept study was designed to evaluate the safety and tolerability of AMO-02 in 16 patients, ages 13 to 34 years old, living with myotonic dystrophy type 1. Patients received either an oral dose of placebo, 1000mg/day of AMO-02 or 400mg/day of AMO-02, over a 14-week treatment period. AMO-02 is an investigational medicine which is not yet approved for the treatment of myotonic dystrophy. Results were presented in a podium presentation entitled "A Phase 2 Study of AMO-02 in Congenital and Childhood Onset Myotonic Dystrophy Type 1." The most informative assessments of efficacy were clinician-completed and caregiver-completed rating scales, which revealed large treatment-associated effect sizes, these effects being statistically significant across the period of treatment with AMO-02.

"As the first clinical trial devoted to this patient population, these results are especially encouraging," said Dr. Horrigan. "We look forward to continuing to expand our research and further evaluate the efficacy of AMO-02 in larger multi-site clinical trials in the US, Canada and the UK."

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. 

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