AMO Therapeutics Announces Presentation of Concordant Analysis of Results of Phase 2 Study of AMO-02 in treatment of myotonic dystrophy

Treatment associated with improvement in cognitive function, fatigue and neuromuscular symptoms; results support continued clinical development.

LONDON and DURHAM, N.C., Oct. 23, 2018 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on rare, debilitating diseases with limited or no treatment options, today announced the presentation of results from a concordant trend analysis of a Phase 2 clinical study of AMO-02 for the treatment of congenital and childhood onset myotonic dystrophy type 1 (DM1). Results were presented today by Michael Snape, Ph.D., chief executive and chief scientific officer of AMO Pharma, in a podium presentation during the 7th International Conference on Neurology and Neuromuscular Diseases in Madrid, Spain.

The analysis was designed to evaluate trends in several within-study assessments and help in the selection of appropriate efficacy measures in future clinical research. Overall, the study results indicated that treatment with AMO-02 was associated with a range of clinical benefits in the majority of subjects at 12 weeks, with the largest benefit seen in patients treated at the 1000 mg/day dose level.  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.

"Standard measures of treatment efficacy are often not well defined for underserved disease states such as DM1," said Dr. Snape. "This concordant trend analysis offers important new insights that can help us better identify the most precise and appropriate measures of efficacy and safety in clinical research involving AMO-02 and potentially other therapies in the years ahead."

The presentation, entitled "The utility of concordant trend analyses in a Phase 2 study in congenital and childhood onset myotonic dystrophy type 1: a case example," highlights results of an analysis of 10 expert-selected efficacy variables used in the Phase 2 study to further confirm the accuracy of previously reported study findings. The results mirrored previous findings showing that most patients treated with AMO-02 experienced significant clinical benefits after 12 weeks of treatment, with a larger response apparent in those treated with 1000 mg/day versus 400 mg/day.

"This analysis provides a more nuanced understanding of the potential for AMO-02 to offer benefits in the treatment of DM1," said Joseph Horrigan, MD, chief medical officer of AMO Pharma. "These results reinforce the decision to advance the clinical development of AMO-02 to a registration-caliber clinical trial for the treatment of DM1."  

AMO Pharma is now advancing development of AMO-02 in the treatment of CDM1 in an ongoing 6-month registration-caliber study. Results from that study will also be assessed based on a clinician-completed rating scale specific to congenital myotonic dystrophy that will serve as a primary outcome measure.

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. For more information, please visit the AMO Pharma website at http://www.amo-pharma.com/.

Contacts 
Corporate:
Mike Snape, PhD
Chief Executive Officer
AMO Pharma Ltd.
+44 1483 898 448
mike.snape@amo-pharma.com

Media:
Kelly Wakelee
Berry & Company Public Relations
212.253.8881
kwakelee@berrypr.com