Copenhagen, Denmark, March 15, 2018Orphazyme 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, www.orphazyme.com.

Business progress in 2017

Priority                  √              Targeted milestone

sIBM                       √              Initiate Phase II/III trial

sIBM                       √              Assume sponsorship of Phase II/III trial         

ALS                                       Conduct ‘end of Phase II’ meeting with FDA

ALS                         √              Prepare design for Phase II/III trial

NPC                       √              Complete patient enrollment in Phase II/III trial

NPC                       √              Rare Pediatric Disease Designation from FDA

Financing             √              Venture financing to expand clinical development program

Financing             √              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

MDKK                                                   2018 guidance                  2017 actual result           2017 guidance**

Operating loss                                   (245) – (275)                       (126)                                      (125) – (135)

Cash position at year-end*          >350                                      632                                         -

*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


Orphazyme Annual Report 2017