AMO Pharma Announces Activation of Additional Clinical Trial Sites for Pivotal REACH-CDM Study in Congenital Myotonic Dystrophy

Company now enrolling patients at leading research centers in Canada as well as in the United States

LONDON, May 17, 2021 /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 activation of four additional clinical trial sites in Canada and the United States for its pivotal REACH-CDM study evaluating the efficacy and safety of the investigational therapy AMO-02 in the treatment of congenital myotonic dystrophy (CDM1).

"The REACH-CDM study represents an important milestone as the first treatment trial for CDM1, which has no approved treatment," said Dr. Craig Campbell, of Children's Hospital London Health Sciences Centre, London, Ontario.  "We are very pleased to join with these leading research centers in the effort to screen and enroll participants in this important study as quickly as possible."

Dr. Hanns Lochmuller, of Children's Hospital of Eastern Ontario, Ottawa, Ontario said, "We are pleased to see these additional sites now recruiting in this landmark study, particularly given the number of patients affected by CDM1 in Canada."

AMO Pharma announced initiation of the REACH-CDM trial in December 2020, with plans to enroll a total of 56 patients at leading research centers in several countries. The current participating centers are:

• Children's Hospital London Health Sciences Centre (LHSC), London, Ontario, Canada
  Principal Investigator: Dr. Craig Campbell 

• Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada
  Principal Investigator: Dr. Hanns Lochmuller

• Arkansas Children's Hospital, Little Rock, Arkansas, United States
  Principal Investigator: Dr. Aravindhan Veerapandiyan   

• Stanford University Palo Alto, California, United States
  Principal Investigator: Dr. John W Day

• University of Iowa Hospitals and Clinics, Iowa City, United States
  Principal Investigator: Dr. Katherine Mathews

• University of Rochester Medical Center, Rochester, New York, United States
  Principal Investigator: Dr. Bo Hoon Lee  

"We are very pleased that these additional treatment centers are now positioned to enroll patients in our REACH-CDM trial, and we continue to be encouraged by the interest from the CDM1 community as we work to enroll patients at sites in multiple territories," said Dr. Joseph Horrigan, AMO Pharma chief medical officer. "We are grateful to the global team of outstanding clinical investigators who have worked with us to ensure that participants can take part in this study quickly and safely. We look forward to our continued work with them as we advance this clinical program in the months ahead."

Additional information about the REACH-CDM trial is available at www.reachcdm.com.

About AMO-02

AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has potential for use in additional CNS, neuromuscular and other orphan indications. AM0-02 is a clinical stage investigational medicine for the treatment of the severe form of congenital myotonic dystrophy known as DM1 or Steinert disease. AMO-02 has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3β.

About AMO Pharma

AMO Pharma is a biopharmaceutical company 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 and severe childhood onset neurogenetic disorders with limited or no treatment options. In addition to developing AMO-02 for congenital myotonic dystrophy, the company is also progressing AMO-01 as a clinical stage treatment for Phelan-McDermid syndrome and AMO-04 as a clinic-ready potential medicine for Rett syndrome and related disorders. AMO-02, AMO-01 and AMO-04 are investigational medicines that have 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/.

Contacts

Media:
Jenna Urban
Berry & Company Public Relations
jurban@berrypr.com
212 253 8881

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