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Press Release

Orphazyme announces Annual Report 2017

Posted on: 15 Mar 18

Orphazyme A/S
Company announcement                                                                                                                  

No. 03/2018                                                                                                                                        
Company Registration No. 32266355                                                                                                                                                                                            


Copenhagen, Denmark, March 15, 2018 – Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced its Annual Report for 2017. Below is a summary from the report of Orphazyme’s business progress, financial performance for the year, and guidance for 2018. The full report is attached as a PDF file and can furthermore be found on the Company's website,

Business progress in 2017

PriorityTargeted milestone
  • Initiate Phase II/III trial
  • Assume sponsorship of Phase II/III trial
  • Conduct ‘end of Phase II’ meeting with FDA
  • Prepare design for Phase II/III trial
  • Complete patient enrollment in Phase II/III trial
  • Rare Pediatric Disease Designation from FDA
  • Venture financing to expand clinical development program
  • Prepare and complete Initial Public Offering

All milestones were achieved.


Financial performance
The financial result is in line with the guided outlook for 2017. The net result for 2017 was a loss of DKK 126.2 million compared to DKK 57.9 million in 2016. The increase is primarily due to increased research and development activities and costs associated with the IPO.

Research & development costs totaled DKK 99.0 million in 2017 compared to DKK 55.8 million in 2016. The increase was mainly due to the on-going NPC Phase II/III trial, preparation costs for the Phase II trial in Gaucher, a Phase II/III trial in ALS, IND transfer costs for the sIBM study, and arimoclomol manufacturing costs.

General and administrative expenses increased from DKK 7.7 million in 2016 to DKK 32.0 million in 2017, mainly due to IPO-related costs and the expanded management and additional staff.

The net financials totaled an expense of DKK 0.7 million in 2017 compared to an income of DKK 0.1 million in 2016, primarily reflecting exchange rate gains.

As of December 31, 2017, Orphazyme had cash and cash equivalents of DKK 631.7 million compared to DKK 14.3 million as of December 31, 2016. The increase reflects the net proceeds from the IPO of DKK 557.4 million and capital increases of DKK 156.8 million prior to the IPO.


Guidance 2018

MDKK2018 guidance2017 actual result2017 guidance**
Operating loss(245) – (275)(126)(125) – (135)
Cash position at year-end*>350632 -

*Cash, cash equivalents, and marketable securities

**Prospectus, November 2017


Anders Hinsby, Chief Executive Officer, said: “We continue to progress in accordance with our plans. This year, we are looking forward to reporting the trial results from our Phase II/III trial in NPC, which are anticipated in Q3 2018, and to initiate our ALS Phase II/III trial. By the end of 2020, our ambition is to have completed three potential registration trials with arimoclomol, with the first possible marketing authorization as early as 2020.”


Conference call and webcast

The call will be held today at 11.00 AM CET.

Dial-in details:

  • Denmark: +45 35 15 80 49
  • United Kingdom: +44 (0)330 336 9105
  • United States: +1 646 828 8156

Event Title: Orphazyme Annual Report 2017
Confirmation code: 7829588


[PDF file attached.]


For additional information, please contact

Orphazyme A/S

Anders Hinsby, CEO                             +45 31 44 31 39
Anders Vadsholt, CFO                         +45 28 98 90 55


This announcement contains forward-looking statements. Such statements are subject to risks and uncertainties, as various factors, many of which are beyond the control of Orphazyme A/S, may cause actual developments and results to differ materially from the expectations expressed in this report.

About Orphazyme A/S
Orphazyme is a Danish biotech company listed on Nasdaq Copenhagen (ticker: ORPHA.CO) with a late-stage drug pipeline, developing new treatment options for orphan protein-misfolding diseases. The company was founded in 2009 based on early scientific discovery in heat-shock proteins. The company is headquartered in Copenhagen and currently has 40 employees. The lead candidate arimoclomol is in development as a potential treatment for four orphan diseases; two protein-aggregation diseases, sporadic Inclusion Body Myositis (sIBM) and Amyotrophic Lateral Sclerosis (“ALS”), and two lysosomal storage diseases, Niemann-Pick Type C (NPC) and Gaucher disease. For more information, please visit


03-2018 Orphazyme announces Annual Report 2017

Orphazyme Annual Report 2017


Last updated on: 15/03/2018

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