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Orphazyme A/S (ORPH)

NasdaqGS - NasdaqGS Real-time price. Currency in USD
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5.51-0.41 (-6.93%)
At close: 3:56PM EDT
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Previous close5.92
Open5.92
Bid5.50 x 1000
Ask5.60 x 1400
Day's range5.46 - 5.92
52-week range5.46 - 14.78
Volume11,134
Avg. volume49,726
Market cap199.156M
Beta (5Y monthly)0.81
PE ratio (TTM)N/A
EPS (TTM)N/A
Earnings dateN/A
Forward dividend & yieldN/A (N/A)
Ex-dividend dateN/A
1y target est18.67
  • Globe Newswire

    Orphazyme announces topline data from pivotal trial of arimoclomol in Amyotrophic Lateral Sclerosis (ALS)

    Orphazyme A/SCompany announcementNo. 14/2021Inside informationCompany Registration No. 32266355 Pivotal trial did not meet primary and secondary endpoints evaluating impact on function and survival Orphazyme remains focused on commercial readiness and potential U.S. approval of arimoclomol for Niemann-Pick disease type C (NPC) in June Copenhagen – May 7, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases, today announced that the ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS. No important safety signals were reported in the trial. Topline data will be presented at the upcoming virtual European Network to Cure ALS (ENCALS) meeting, May 12-14, and complete data from the study will be published later this year. “We are disheartened by these results, as we had hoped arimoclomol might represent a viable new approach against the formidable challenge of this devastating disease. We express our sincere thanks to the investigators, patients and families for their participation and collaboration in our program,” said Thomas Blaettler, MD, Chief Medical Officer, Orphazyme. “With over 18 months of evaluation, this trial represents one of the longest running clinical studies in this category. While unsuccessful, the data generated will contribute meaningfully to the scientific dialogue on this challenging disease. We will apply the invaluable insights from this and other studies to further our pipeline as we continue to pursue the full potential of the heat shock protein response.” The randomized, placebo-controlled Phase 3 trial was conducted among 245 patients at 29 sites in 12 countries in North America and Europe. Participants were randomized (2:1 ratio) to receive either arimoclomol (248 mg three times daily)i or placebo for up to 76 weeks. The primary endpoint was to determine the efficacy of chronic treatment with arimoclomol compared to placebo in participants with ALS as assessed by the combined assessment of function and survival (CAFS). This endpoint was selected to illustrate the overall treatment effect based on survival and the change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. Secondary endpoints included survival, change in ALSFRS-R, and slow vital capacity (SVC). Orphazyme’s applications for arimoclomol (to be branded MIPLYFFATM)ii for Niemann-Pick disease type C (NPC) are under priority review with the U.S. Food and Drug Administration, with an expected PDUFA action date of June 17 2021, as well as with the European Medicines Agency, with an opinion from the Committee for Medicinal Products for Human Use (CHMP) expected later this year. For additional information, please contact: Orphazyme A/S Copenhagen: Anders Vadsholt, CFO, +45 28989055 Chicago: Molly Carey Poarch, Global Media, +1-773-770-6888 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases. The company is harnessing amplification of heat shock proteins (or HSPs) to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases. Arimoclomol, the company’s lead candidate, is in clinical development for Niemann-Pick disease type C (NPC) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA.CO). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of heat shock proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, and has now been studied in 10 phase 1, four phase 2 and three pivotal phase 2/3 trials. Arimoclomol is in clinical development for NPC and Gaucher disease. Arimoclomol has received orphan drug designation (ODD) for NPC in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Arimoclomol is an investigational treatment and has not been approved by the FDA. About ALSAmyotrophic lateral sclerosis (ALS) is a rare, rapidly progressive, and always fatal neurodegenerative disease. Protein misfolding and aggregation in motor neurons are hypothesized to be important contributors to the disease process, which ultimately leads to paralysis of skeletal muscles as well as the muscles that enable breathing. The patient population in Europe and the United States is estimated to be approximately 50,000 patients. Currently, there are only limited treatment options available. Arimoclomol has been granted Orphan Drug Designation (EU and US) for the treatment of ALS. Forward-looking statementThis company announcement may contain certain forward-looking statements, including in respect of the expected PDUFA action date of June 17 2021 for arimoclomol for the treatment of NPC, the potential U.S. approval of arimoclomol in June and the opinion from the Committee for Medicinal Products for Human Use (CHMP) later this year. 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. 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. i Arimoclomol citrate 400 mg three times dailyii MIPLYFFA is a trademark or registered trademark of Orphazyme A/S. The global brand “MIPLYFFA” has received conditional approval from the U.S. Food and Drug Administration; the brand name will be used commercially upon approval for NPC. Attachment 14-2021 Orphazyme announces topline data from pivotal trial of arimoclomol in ALS

  • Globe Newswire

    Orphazyme to present at upcoming investor conferences

    Orphazyme A/SInvestor news No. 05/2021Company Registration No. 32266355 Copenhagen, Denmark and Chicago, IL, USA, April 27, 2021 – Orphazyme A/S [ORPHA.CO (DK); ORPH (US)], a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of neurodegenerative rare diseases, today announced that company management will be participating at the following virtual investor conferences: Redeye Orphan Drugs Seminar Wednesday, April 28, 2021 at 4:15 am EDT/10:15 am CETCorporate presentation by Christophe Bourdon, CEO and Anders Vadsholt, CFOWebcast link: https://www.redeye.se/events/802433/theme-orphan-apr-28th B. Riley Neurosciences ConferenceThursday, April 29, 2021 at 11:00 am EDT/5:00 pm CETCorporate presentation by Christophe Bourdon, CEO and Anders Vadsholt, CFOWebcast link: https://b-riley-neuroscience-conference.events.issuerdirect.com/ For additional information, please contactOrphazyme A/S Anders Vadsholt, CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for Niemann-Pick disease type C (NPC), Amyotrophic Lateral Sclerosis (ALS), and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. 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. 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, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. 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. Attachment 05-2021 Orphazyme to present at upcoming investor conferences

  • Globe Newswire

    New long-term share-based incentive program

    Orphazyme A/SCompany announcement No. 13/2021Company Registration No. 32266355 Copenhagen, Denmark, April 22, 2021 – Orphazyme A/S [ORPHA.CO (DK); ORPH (US)], a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of neurodegenerative rare diseases, has today introduced a new long-term incentive program (the “LTIP”). The LTIP is designed and structured around the concept of retaining members of the Executive Management and other employees of the Group, while also creating an incentive for a positive share price development and corporate performance for the benefit of the Company’s shareholders. The LTIP grants comprise Restricted Share Units (“RSUs”) and Performance Share Units (“PSUs”) which entitle the participants, subject to vesting occurring, to be allocated a number of shares in the Company, equivalent to the number of vested RSUs and/or PSUs, against payment of the par value of each share. The RSUs will have a total vesting period of three years calculated from January 1 or July 1 in the grant year and with one third of the granted RSUs vesting on each January 1 or July 1 in the following three financial years. Vesting of RSUs is not conditional upon achieving any financial or non-financial targets. However, vesting is conditional upon (i) the participant remaining employed with a group member throughout the total vesting period for RSUs or the participant becoming a good leaver during the total vesting period for RSUs, in which case the participant will be entitled to keep any vested RSUs and receive a pro rata allocation, and (ii) the participant having complied in all respects with the general terms and conditions as determined by the Board of Directors. The vested RSUs can only be exercised within four months after the expiration of the total vesting period for RSUs. However, the RSU delivery period may be extended to the next open trading window in certain circumstances. The PSUs will have a total vesting period of three years calculated from January 1 or July 1 in the grant year and with the granted PSUs vesting, in whole or in part, on January 1 or July 1 in the third year following the date of the grant. Vesting of PSUs is conditional upon (i) an increase in the quoted share price of the Company’s shares, (ii) the participant remaining employed with a group member throughout the vesting period for PSUs or the participant becoming a good leaver during the vesting period for PSUs, in which case the participant may be entitled to keep a proportion of the PSUs, and (iii) the participant having complied in all respects with the general terms and conditions as determined by the Board of Directors. Any vested PSUs can only be exercised within four months after the expiration of the vesting period for PSUs. However, the PSU delivery period may be extended to the next open trading window in certain circumstances. Based on the current number of participants in the LTIP, the program and other share-based retention grants are expected to comprise up to 950,000 shares in total. The theoretical fair value of each RSU has been estimated at DKK 58.04 and the theoretical fair value of each PSU under the LTIP has been estimated at DKK 20.02. For additional information, please contact Orphazyme A/S Anders Vadsholt, CFO +45 28 98 90 55 About Orphazyme A/S Orphazyme is a late-stage biopharmaceutical company pioneering the Heat Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for Niemann-Pick disease type C (NPC), Amyotrophic Lateral Sclerosis (ALS), and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). About arimoclomol Arimoclomol is an investigational drug candidate that amplifies the production of Heat Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial. Arimoclomol is in clinical development for NPC, Gaucher Disease, and ALS. Arimoclomol has received orphan drug designation (ODD) for NPC and ALS in the US and EU. Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC and ALS. In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC. Forward-looking statement This company announcement may contain certain forward-looking statements, including in respect of the anticipated commercialization of arimoclomol. 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. 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, including the risk that applicable regulatory authorities fail to approve arimoclomol on the anticipated timeline or at all. 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. Attachment 13-2021 New long-term share-based incentive program