Rating scale positioned to deliver meaningful benefit for clinical research presented at 2019 MDA Clinical and Scientific Conference in Orlando, FL
LONDON and DURHAM, North Carolina, April 16, 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 a new clinician-completed rating scale to assess features of congenital myotonic dystrophy type 1 (CDM1). An overview of the new rating scale was presented on Monday, April 15th in a poster presentation titled, "The Development of the Clinician-Completed Congenital Myotonic Dystrophy Type 1 Rating Scale (CDM1-RS)" led by Joseph Horrigan MD, AMO Pharma's chief medical officer, during The Muscular Dystrophy Association (MDA) Clinical & Scientific Conference at the Hyatt Regency Orlando.
"Standard assessment tools should be used to follow FDA guidance and capture all of the characteristic symptoms of a condition to be useful in clinical trials. To that end, the CDM1-RS rating scale has been evolved from the well validated MDHI and CCMDHI measures developed by professor Chad Heatwole and professor Nicholas Johnson in the hope it can become a widely accepted clinician instrument for CDM1," said Michael Snape, chief executive officer of AMO Pharma. "The availability of scale to assess CDM1 can expand our understanding of this disorder, streamline the diagnostic process and could provide essential insights to guide future clinical research."
CDM1 is a genetic disease caused by a large tri-nucleotide expansion repeat adjacent to the DMPK gene. Symptoms usually present shortly after birth and include difficulty with thinking and problem solving, speech impairment, symptoms of autism and weakened muscles. There are currently no approved therapies for CDM1. Patients are often treated with a combination of medical interventions designed to address individual symptom as they arise.
The fit-for-purpose scale identifies 11 observable characteristics of CDM1 and rates each on a four-point Likert scale, using information from natural history studies, consultation with therapeutic area experts and feedback from regulatory agencies. An evaluation of the scale's use in clinical study found it has potential to effectively assess the CDM1 phenotype in a low-burden manner, is change-sensitive, can detect treatment effects and may provide useful outcome measure for future clinical trials.
"This is a major milestone for researchers looking to enhance our understanding of this disorder, drawing insight from the experiences of other patients and caregivers in a reproducible manner and incorporating advice from the FDA," said Dr. Horrigan. "We look forward to broadening the scope of this scale so that we may better address the current and future needs of this largely underserved community."
This clinician-completed rating scale will serve as the primary outcome measure in a Pharma Phase 2/3 registration-caliber study in children and adolescents with CDM1 being planned by AMO Pharma. It is also being validated in an ongoing natural history study in children and adolescents with CDM1.
About congenital myotonic dystrophy
Congenital myotonic dystrophy (CDM1) is a genetic disease that typically presents at birth. People living with CDM1 typically experience difficulty with thinking and problem solving. They may also experience speech difficulties, symptoms of autism and weakened muscles. 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. 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/.
Mike Snape, PhD
Chief Executive Officer
AMO Pharma Ltd.
+44 1483 898 448
Berry & Company Public Relations
SOURCE AMO Pharma Limited