Copenhagen, Denmark, March 1, 2019 – Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced its Annual Report for 2018. Below is a summary from the report of Orphazyme’s business progress, financial performance for the year, and guidance for 2019. The full report is attached as a PDF file and can furthermore be found on the Company's website www.orphazyme.com.

Anders Hinsby, Chief Executive Officer, said: “2018 was a very important year for Orphazyme, with the initiation of two clinical trials and the successful completion of our Phase II/III clinical trial in Niemann-Pick disease Type C (NPC). For almost 10 years, we have now been steadfast in our mission to develop a treatment option for patients suffering from this severely debilitating disease, and we are now closer than ever to achieving this goal. We are looking forward to 2019, during which we will start the preparations to bring arimoclomol to approval for NPC”.

Business progress in 2018

Priority Targeted milestone

ALS √ Initiate Phase III trial in Q3

sIBM √ ·    Enroll patients in both USA and Europe
ꟷ Complete Phase II/III trial enrollment by year-end

NPC √ Phase II/III top-line results in Q3

Gaucher √ Initiate Phase II trial in Q2
ꟷ Complete trial enrollment by year-end

Financial performance

The operating loss of DKK 231.6 million was below the expected operating loss range of DKK 245-275 million. The difference to the outlook for 2018 was mostly due to slower patient enrollment in our clinical trials.

Research and development expenses totaled DKK 196.5 million in 2018 compared to DKK 99.0 million in 2017. The increase was mainly due to the on-going NPC trial, the ramp-up of the sIBM Phase II/III trial, the initiation of the Phase II trial for Gaucher disease in June, and the initiation of the Phase III trial for ALS in August.

General and administrative expenses totaled DKK 35.1 million in 2018 compared to DKK 32.0 million in 2017. The increase is mainly due to increased employee costs, the initiation of pre-commercial activities as well as increased investor relations activities.

As of December 31, 2018, Orphazyme had cash DKK 394.7 million compared to DKK 631.7 million as of December 31, 2017. The decrease in cash results from the increase in research and development spend, as described above.

Guidance 2019

MDKK 2019 guidance 2018 actual result 2018 guidance
Operating loss (315) – (345) (232) (245) – (275)
Cash position at year-end >50 395 >350


Conference call and webcast

The call will be held today at 11.00 AM CET.

Dial-in details:

·       Denmark: +45 32 72 80 42

·       France: +33 (0) 176 700 794

·       Netherlands: +31 (0) 207 143 545

·       Sweden: +46 (0) 850 692 180

·       United Kingdom: +44 (0) 844 571 88 92

·       United States: +1 63 15 107 495


Event Title: Orphazyme Annual Report 2018

Confirmation code: 1127327


The presentation will also be available via webcast: https://edge.media-server.com/m6/p/et2opupq