Professor Hanns Lochmüller from Children's Hospital of Eastern Ontario Research Institute highlighted results from AMO Pharma's Phase 2 trial with AMO-02 showing improvements in multiple symptom domains in patients with congenital and childhood-onset myotonic dystrophy 1 (CDM-1).
Separate poster presentations review safety and tolerability of AMO-02 and development of first-ever clinician-completed CDM-1 Rating Scale.
LONDON and GOTHENBURG, Sweden, June 13, 2019 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on rare, childhood-onset neurogenetic disorders with limited or no treatment options, today announced the presentation of results from the Phase 2 clinical trial of AMO-02 during the International Myotonic Dystrophy Consortium Meeting in Gothenburg, Sweden. AMO-02 (tideglusib) is an investigational therapy in development at AMO Pharma for the treatment of congenital myotonic dystrophy 1 (CDM-1).
In an oral presentation entitled "Advances in Drug Development in Myotonic Dystrophy", Professor Hanns Lochmüller, neurologist from Children's Hospital of Eastern Ontario Research Institute and lead investigator for the AMO-02 Phase 2 clinical trial, outlined previously reported results from the Phase 2 study of AMO-02 showing that 13 out of 16 patients reported a clinically meaningful response in a range of symptom domains including sleepiness, cognition, autism, muscle function and myotonia, noting that these results can translate into improvements in real world function including speech, motivation and awareness, and activities of daily living. He also noted that results show inhibition of GSK3β with AMO-02 and outlined AMO Pharma's goal to advance the development program for AMO-02 to a pivotal clinical trial to support NDA submission.
"Among the recent advances in research related to myotonic dystrophy, results from the AMO Pharma clinical program for AMO-02 thus far are especially encouraging, indicating the potential for quantifiable improvements in multiple symptom domains," said Professor Lochmüller, adding, "These results together with availability of the first-ever clinician-completed rating scale represent important advances in our understanding of congenital and childhood-onset myotonic dystrophy that will help shape future research and improve the management of patients around the world."
The meeting also included poster presentations highlighting the safety and tolerability of AMO-02 based on results from the Phase 2 trial and a report on the process used to develop a clinician-completed congenital myotonic dystrophy type 1 rating scale. AMO Pharma announced the launch of the new rating scale in April 2019.
"We are especially pleased that this range of data was reviewed during the IMDC meeting, where leaders in DM research and treatment from around the world gather to share insights and perspectives on the future of care," said Joseph P. Horrigan, MD, Chief Medical Officer for AMO Pharma. "Treatment of myotonic dystrophy remains an area of major unmet need in global health, and AMO Pharma is dedicated to advancing promising research that could someday lead to new and better treatment options for patients."
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. AMO-02 is an investigational medicine that has not yet been approved for the treatment of patients anywhere in the world. For more information, please visit the AMO Pharma website at http://www.amo-pharma.com/.
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SOURCE AMO Pharma Limited