ORPHAZYME'S PHASE II/III TRIAL IN SPORADIC INCLUSION BODY MYOSITIS FULLY ENROLLED

· Study completion expected by end 2020
· Results expected in H1 2021


Copenhagen, April 23, 2019
– Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced that its Phase II/III trial of arimoclomol for the treatment of sporadic Inclusion Body Myositis (sIBM) is fully enrolled. Performance of interim analysis and study completion expected in H1 2020 and by end 2020, respectively. Results are expected in H1 2021.

Anders Hinsby, Chief Executive Officer, said: “We are truly delighted about the strong support for this trial from the patient community and clinicians, which has been paramount to achieving enrollment at an impressive rate. Sporadic Inclusion Body Myositis is a relentlessly progressive and debilitating disease with no current treatment options available and we cannot waste any time in completing this trial in order to evaluate the potential of arimoclomol as a treatment for this disease”.

The Phase II/III trial of arimoclomol for sIBM is a 150-patient, 20-month, randomized, double-blinded, placebo-controlled trial at 11 centers of excellence in the US and one in the UK. Orphazyme has initiated an open-label extension trial to which patients from the Phase II/III trial may enroll and continue treatment.