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ListaMid Cap Copenhagen
Orphazyme är verksamma inom biokemi. Bolaget är specialiserade mot forskning och utveckling av ovanliga sjukdomar där det finns en brist i funktionen hos värmekokproteiner. Tekniken baseras på arimoklomol, som stimulerar produktionen av värmekokproteiner i celler som upplever en ökad grad av stress och toxicitet. Sjukdomar som ämnar behandlas med bolagets produkter innefattar Gauchers sjukdom och amyotrofisk lateral skleros (ALS). Bolaget har sitt huvudkontor i Köpenhamn.


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2020-08-28 07:00:00

Company announcement
No. 45/2020

Orphazyme A/S
Ole Maaløes Vej 3
DK-2200 Copenhagen N
CVR No.: 32266355


Copenhagen, Denmark, August 28, 2020 – Orphazyme A/S (ORPHA.CO), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces its Interim Report First Half 2020 for the period January 1–June 30, 2020.

Pipeline Highlights First Half 2020

  • Reported positive data from 12-month open-label phase 2/3 extension clinical trial in Niemann-Pick disease Type C (NPC) showing sustained effect of arimoclomol in reducing disease progression over two years
  • Announced a U.S. Early Access Program for NPC
  • Initiated rolling submission of New Drug Application (NDA) for arimoclomol with the U.S. Food and Drug Administration (FDA) in NPC, which was subsequently completed in July 2020
  • Reported top-line data from a phase 2 clinical trial in Gaucher disease Type 1 and Type 3 demonstrating marked improvements in key clinical markers following 6 months of treatment with arimoclomol
  • Received U.S. Fast-Track Designation for arimoclomol in Amyotrophic Lateral Sclerosis (ALS)

Financial and Business Highlights First Half 2020

  • For the first six months of 2020, Orphazyme reported a net loss of DKK 251.4 million or DKK 9.88 per share (basic and diluted) compared to a net loss of DKK 163.9 million or DKK 8.20 per share (basic and diluted) for the same period in 2019
  • Research and development expenses for the period totaled DKK 167.0 million compared to DKK 141.7 million for the same period in 2019, as our pipeline continued to advance through late-stage trials
  • General and administrative expenses for the period totaled DKK 78.6 million compared to DKK 23.3 million for the same period in 2019 due to build-up of our commercial organization as well as related support functions in anticipation of the potential approval of arimoclomol in NPC
  • Completed offering of 7,032,937 shares in a directed issue and private placement, raising approximately DKK 745 million in gross proceeds (approximately USD 110 million)
  • As of June 30, 2020, Orphazyme held cash totaling DKK 610.4 million compared to DKK 225.6 million as of June 30, 2019 and DKK 123.6 million as of December 31, 2019

Subsequent Events

  • Completed rolling submission of NDA for arimoclomol with U.S. FDA in NPC
  • Announced confidential submission of draft registration statement for a potential registered public offering of American Depositary Shares in the United States
  • Announced collaboration with The Michael J. Fox Foundation on Parkinson’s disease research, joining its Research Tools Consortium which brings together experts from the medical community and industry to identify and develop new tools to address unmet research needs in Parkinson’s disease

“During the first half of the year, we made significant progress in our efforts to bring our first product candidate to market and in July completed the submission of a rolling New Drug Application (NDA) to the FDA for arimoclomol to NPC. We are pleased to be able to already provide patients in the U.S. access to this much needed treatment for NPC through an Early Access Program, which we initiated during Q1. We continue to work at pace in building a highly specialized commercial organization in anticipation of potential approval in NPC in the U.S. and in preparing to submit a marketing application for NPC to regulatory authorities in Europe during the second half of this year”, said Kim Stratton, Chief Executive Officer of Orphazyme. “Our belief that arimoclomol has pipeline-in-a-product potential was also further underlined during the first half of this year with the phase 2 data readout supporting further studies in Gaucher disease and the award by the U.S. regulators of Fast-Track Designation for arimoclomol for the treatment of ALS. Also importantly, the first half of this year saw a boost to our efforts in building a strong international biopharmaceutical company that can maximize the value of arimoclomol, with a successful private placement raising gross proceeds of DKK 745 million (~USD 110 million). We believe the next twelve months hold significant potential for Orphazyme as we near the anticipated commercial launch of arimoclomol, if approved, in its first indication and as we look forward to data readouts from pivotal stage clinical trials testing arimoclomol in ALS and sporadic Inclusion Body Myositis (sIBM) during H1 2021.

The company maintains its 2020 outlook as announced on February 28, 2020. Net operating expenses are anticipated to be in the range of DKK 500–550 million and our cash position at year-end 2020 is anticipated to be greater than DKK 300 million.


Conference Call

Orphazyme will host an investor call at which Chief Executive Officer, Kim Stratton, and Chief Financial Officer, Anders Vadsholt, will present the Interim Report First Half 2020. The presentation will be followed by a Q&A session.

The call will be held on Friday, August 28, 2020 at 2.00 PM CET/8.00 AM ET.

Dial-in details:

  • Denmark: +45 3272 8042
  • United States: +1 6315 107 495
  • Standard International: +44 (0) 2071 928000
  • United Kingdom: +44 (0) 8445718892
  • France: +33 (0) 176700794
  • Netherlands: +31 (0) 207143545
  • Sweden: +46 (0) 850692180

Event Title: Orphazyme Interim Report First Half 2020

Confirmation code: 5555608

The presentation will also be available via webcast: https://edge.media-server.com/mmc/p/guyn7ubp

After the call, the presentation will be available via the webcast link above.


For additional information, please contact

Orphazyme A/S

Anders Vadsholt, CFO                   +45 28 98 90 55


About Orphazyme A/S 
Orphazyme is a biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is focused on developing therapies for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Gaucher Disease, sporadic Inclusion Body Myositis (sIBM), and Amyotrophic Lateral Sclerosis (ALS). The Denmark-based company is listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit www.orphazyme.com. 

This company announcement may contain certain forward-looking statements, including in respect of the timing of the company’s clinical trials and the results thereof, anticipated regulatory approvals of the company’s product candidates, the company’s anticipated operating performance and financial position, and the proposed offering of the company’s securities in the United States. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this company announcement about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control, including adverse developments in the company’s clinical program, effects of the global COVID-19 pandemic, technical and scientific developments in the indications that the company’s product candidates are designed to treat, regulatory developments; the proposed offering of the company’s securities in the United States remains subject to review by the U.S. Securities and Exchange Commission (SEC) and the Nasdaq Stock Market in the United States (Nasdaq). These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could”, and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance, or achievements to be materially different from the expected results, performance, or achievements expressed or implied by such forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. This announcement is not an offer of securities for sale in the United States; no securities of the company may be offered or sold in the United States absent registration or an exemption from registration; any public offering of securities to be made in the United States by the company will be made by means of a prospectus that may be obtained from the company and that will contain detailed information about the company and management, as well as financial statements. As previously disclosed, the company has confidentially submitted a draft registration statement on Form F-1 to the SEC relating to a potential registered public offering of the company’s securities in the United States.