AMO Pharma Initiates Phase 2 Clinical Trial in UK for AMO-02 for Treatment of Congenital and Juvenile Onset Myotonic Dystrophy

Study represents first sponsor-led clinical research program for patients with congenital and juvenile onset myotonic dystrophy

LONDON and PHILADELPHIA, Aug. 10, 2016 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, today announced initiation of a phase 2 clinical study in the U.K. for AMO-02 (tideglusib), the company's investigational therapy in development for the treatment of congenital and juvenile onset myotonic dystrophy. This is the first sponsor-led clinical study evaluating an investigational therapy in this patient group as part of a development program. The clinical study involves a 14-week treatment period to investigate tideglusib in adults with congenital and juvenile onset myotonic dystrophy and includes safety, efficacy and biomarker endpoints.

"Myotonic dystrophy is a devastating disease for which there are no approved treatments. It causes progressive muscle weakness and cognitive and developmental challenges that can be especially severe in younger patients," said Dr. Joseph Horrigan, chief medical officer of AMO Pharma. "We look forward to working with the clinician and patient communities to advance this historic research effort in the months ahead."

"Treatment of myotonic dystrophy remains an area of critical unmet need in global healthcare, and Newcastle is very pleased to be participating in this important new research effort that could bring hope to families around the world," said the principal investigator professor Hanns Lochmuller, chair of experimental myology at Newcastle University Institute for Genetic Medicine. "Our established myotonic dystrophy patient registry and our work with many patient families throughout the U.K. will be an important advantage in our efforts to identify patients who are eligible to participate in this clinical research effort."

Earlier in 2016 AMO Pharma completed discussions with the U.S. Food and Drug Administration (FDA) for a planned investigational new drug (IND) application for AMO-02. AMO is planning additional global studies of AMO-02 in both congenital and adult onset myotonic dystrophy.  Based on FDA feedback, AMO Pharma is currently working to finalize plans to advance its clinical development program.

About U.K. Clinical Trial
For more information on the clinical trial, please visit the European Clinical Trials Database (EudraCT) at https://www.clinicaltrialsregister.eu/ctr-search/trial/2016-000067-16/GB or Clinicaltrials.gov at https://clinicaltrials.gov/ct2/show/NCT02858908.

About Myotonic Dystrophy
Myotonic dystrophy is the most common form of muscular dystrophy, affecting approximately 1 in 10,000 people. It is a progressive disease and the predominance of symptoms such as muscle weakness, intellectual/developmental impairment or cognitive/behavioral changes is determined by a number of factors, including age at onset. There are currently no approved therapies indicated for the treatment of myotonic dystrophy.

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 genetic diseases. AMO Pharma is currently advancing two investigational therapies for treatment of fragile X syndrome and myotonic dystrophy, and is in the process of licensing additional development-stage products in diverse areas including autism and other CNS disorders. For more information, please visit the AMO Pharma website at http://www.amo-pharma.com/.

Contacts

Corporate:
Lisa Wittmer, PhD
Chief Operating Officer
Head, Portfolio Development
AMO Pharma Ltd.
484.431.7991
lisa.wittmer@amo-pharma.com

Media:
Sara Zelkovic
Berry & Company Public Relations
212.253.8881
szelkovic@berrypr.com