PUBLICATION OF MANUSCRIPT RELATED TO PHASE II TRIAL RESULTS FOR ARIMOCLOMOL IN PATIENTS WITH SOD1 ALS

Copenhagen, January 24, 2018 – Orphazyme A/S, a Danish biotech company listed on Nasdaq Copenhagen (TICKER: ORPHA.CO), with a late-stage, orphan-drug pipeline, today announced that a manuscript reporting the Phase II trial results for arimoclomol in patients with SOD1 Amyotrophic Lateral Sclerosis (ALS) was published in the peer-reviewed clinical journal Neurology®. The manuscript highlights arimoclomol’s favorable safety profile and provides data on efficacy that supports the continued development of arimoclomol in a Phase II/III trial. 

The manuscript, which is lead-authored by primary investigator Michael Benatar, MD and PhD from the University of Miami, confirms that arimoclomol is safe and well-tolerated at a dosage of 200mg three times a day for up to 12 months.

Additionally, secondary endpoints looking at the efficacy of arimoclomol all favored arimoclomol. The trial was not powered to demonstrate efficacy, but trends of benefit were consistently observed across the full range of pre-specified efficacy outcome measures, including survival, function (ALSFRS-R), vital capacity, as well as the combined assessment of function and survival (CAFS).  

Michael Benatar, MD and PhD, primary investigator says: In addition to providing evidence of the safety and tolerability of arimoclomol 200mg three times daily over a 12-month period, the consistency of the preliminary efficacy data across a range of pre-specified endpoints, and in the only pre-specified subgroup, strongly supports further development of arimoclomol in ALS.

As previously communicated, Orphazyme intends to conduct a Phase II/III trial to support the application for a marketing authorization in ALS. The trial is planned to be initiated in H2 2018.

A link to the article is available on www.orphazyme.com in the Media section.