ORPH Orphazyme A/S

4.78
-0.04  -1%
Previous Close 4.82
Open 4.87
52 Week Low 4.6
52 Week High 77.77
Market Cap $167,071,712
Shares 34,952,241
Float 34,952,241
Enterprise Value $69,253,163
Volume 679,592
Av. Daily Volume 15,182,228
Stock charts supplied by TradingView

Drug Pipeline

Drug Stage Notes
Arimoclomol
Niemann-Pick disease Type C (NPC)
CRL
CRL
CRL issued June 18, 2021.
Arimoclomol
Amyotrophic Lateral Sclerosis
Phase 3
Phase 3
Phase 3 data trial did not meet primary endpoint - May 7, 2021.
Arimoclomol
Sporadic Inclusion Body Myositis
Phase 2/3
Phase 2/3
Phase 2/3 trial did not meet primary endpoint - March 29, 2021.

Latest News

  1. New Director Dr. Jennifer K. Simpson Added to CytRx Board of Directors

    CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today announced that based on results from its 2021 Annual Meeting of Stockholders (the "Annual Meeting"), both of the Company's Class III director candidates – Joel K. Caldwell, CPA and Jennifer K. Simpson, Ph.D. – have been elected to the Board of Directors (the "Board"). Additional information pertaining to the Company's Annual Meeting results can be found on the Form 8-K filed with the U.S. Securities and Exchange Commission.

    Steven A. Kriegsman, Chairman and Chief Executive…

    New Director Dr. Jennifer K. Simpson Added to CytRx Board of Directors

    CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today announced that based on results from its 2021 Annual Meeting of Stockholders (the "Annual Meeting"), both of the Company's Class III director candidates – Joel K. Caldwell, CPA and Jennifer K. Simpson, Ph.D. – have been elected to the Board of Directors (the "Board"). Additional information pertaining to the Company's Annual Meeting results can be found on the Form 8-K filed with the U.S. Securities and Exchange Commission.

    Steven A. Kriegsman, Chairman and Chief Executive Officer of CytRx, commented:

    "We appreciate our stockholders' participation at this year's Annual Meeting. By electing Mr. Caldwell and Dr. Simpson to the Board, our stockholders have helped to strengthen CytRx's corporate governance. The addition of Dr. Simpson as a new director reflects our commitment to refreshing the Board with diverse perspectives and valuable industry experience."

    Dr. Simpson added:

    "I look forward to working with Mr. Kriegsman and the rest of the Board to advance stockholders' best interests. Given my extensive experience in the biopharmaceutical and oncology sectors, I see opportunities to add value as the Board explores new initiatives and partnership opportunities. I believe CytRx has high-potential assets that can become a source of enduring value for stockholders."

    New Director Biography:

    Jennifer K. Simpson, Ph.D. is the Chief Executive Officer and a Director of Panbela Therapeutics Inc. (NASDAQ:PBLA), a clinical stage drug development company. Shortly after joining, Dr. Simpson led a public financing with an uplist to the NASDAQ exchange. She has more than 13 years' experience in pharmaceutical executive leadership, global marketing and product commercialization. Previously, Dr. Simpson was the Chief Executive Officer and a Director of Delcath Systems Inc. (NASDAQ:DCTH), an interventional oncology company focused on the treatment of primary and metastatic liver cancer. From 2008 to 2012, Dr. Simpson served in various product leadership roles, including Vice President, Global Marketing, Oncology Brand Lead at ImClone Systems, Inc. (a wholly owned subsidiary of Eli Lilly and Company (NYSE: LLY)). While in this role, she led the largest registration program pre-approval in Oncology for a VEGFR-2 monoclonal antibody including initiation and oversight of six phase 3 trials across five indications as well as a robust phase 2 program. Dr. Simpson earned a Ph.D. in Epidemiology from the University of Pittsburgh, a M.S. in Nursing from the University of Rochester, and a B.S. in Nursing from the State University of New York at Buffalo.

    About CytRx

    CytRx Corporation (OTCQB:CYTR) is a biopharmaceutical company with expertise in discovering and developing new therapeutics principally to treat patients with cancer and neurodegenerative diseases. CytRx's most recent advanced drug conjugate, aldoxorubicin, is an improved version of the widely used anti-cancer drug doxorubicin and has been out-licensed to ImmunityBio, Inc. (NASDAQ:IBRX). In addition, CytRx's drug candidate, arimoclomol, was sold to Orphazyme A/S (NASDAQ:ORPH) in exchange for milestone payments and royalties. Orphazyme is developing arimoclomol in Niemann-Pick disease Type C ("NPC") and Gaucher disease. Learn more at www.cytrx.com.

    View Full Article Hide Full Article
  2. ImmunityBio's Receipt of U.S. Patent for Novel Immunotherapy, NANT Cancer Vaccine, Being Studied in Multiple Clinical Trials

    CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today highlighted that ImmunityBio, Inc. (NASDAQ:IBRX) ("ImmunityBio") has been granted a patent by the U.S. Patent and Trademark Office for its proprietary NANT Cancer Vaccine (U.S. Patent 11,071,774). ImmunityBio's investigational treatment for cancer is designed to bolster a patient's own immune response to cancerous cells, augment that response with additional natural killer and T-cell therapies to overcome the cancer's resistance…

    ImmunityBio's Receipt of U.S. Patent for Novel Immunotherapy, NANT Cancer Vaccine, Being Studied in Multiple Clinical Trials

    CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today highlighted that ImmunityBio, Inc. (NASDAQ:IBRX) ("ImmunityBio") has been granted a patent by the U.S. Patent and Trademark Office for its proprietary NANT Cancer Vaccine (U.S. Patent 11,071,774). ImmunityBio's investigational treatment for cancer is designed to bolster a patient's own immune response to cancerous cells, augment that response with additional natural killer and T-cell therapies to overcome the cancer's resistance, and induce long-term T-cell memory to induce remission across multiple tumor types.

    As previously disclosed, CytRx out-licensed global development, manufacturing and commercialization rights for Aldoxorubicin to ImmunityBio in 2017. The Company has an agreement with ImmunityBio that can yield up to $343 million in potential milestone payments and single and double-digit royalties on sales of Aldoxorubicin.

    To study the safety and early efficacy signals across multiple tumor types, ImmunityBio has launched a series of Quantitative Lifelong Trials (QUILT). To date, the vaccine has been studied in more than 100 patients across multiple tumor types, including pancreatic, breast, colorectal, and head and neck cancers. Among these studies is QUILT 88, a Phase 2 trial that includes Aldoxorubicin and is studying the vaccine in metastatic pancreatic cancer patients. Enrollment of Cohort C, which includes Aldoxorubicin and patients who have previously failed two lines of standard-of-care therapy, is expected to be completed in the third quarter of 2021 and an early readout of survival data is expected in the first quarter of 2022.

    ImmunityBio's Founder and Executive Chairman made the following statement this week:

    "We are excited to be developing this orchestrated approach to activate as many elements of the immune system that we can and overcome cancer's ability to evade the immune system. Our hypothesis is that by revealing tumor antigens to the immune system, we activate tumor-specific T cells and targeted natural killer cells to eradicate tumors by what is known as immunogenic cell death. The issuance of the NANT Cancer Vaccine patent is recognition of this innovative approach to therapy that not only potentially provides long-term immune system protection from cancer, it does so with a reduced risk of the toxicity risk that comes with using high-dose chemotherapy and radiation. This closely aligns with the FDA's recent ‘Project Optimist' guidelines for exploring lower doses of therapeutic agents."

    Steven A. Kriegsman, Chairman and Chief Executive Officer of CytRx, commented:

    "CytRx is very encouraged by ImmunityBio's receipt of a U.S. patent for this novel immunotherapy vaccine that has been utilizing Aldoxorubicin in its pancreatic cancer trials. We continue to believe that Aldoxorubicin can be a foundational component of ImmunityBio's innovative cancer treatments."

    About CytRx

    CytRx Corporation (OTCQB:CYTR) is a biopharmaceutical company with expertise in discovering and developing new therapeutics principally to treat patients with cancer and neurodegenerative diseases. CytRx's most recent advanced drug conjugate, Aldoxorubicin, is an improved version of the widely used anti-cancer drug doxorubicin and has been out-licensed to ImmunityBio, Inc. (NASDAQ:IBRX). In addition, CytRx's drug candidate, arimoclomol, was sold to Orphazyme A/S (NASDAQ:ORPH) in exchange for milestone payments and royalties. Orphazyme is developing arimoclomol in Niemann-Pick disease Type C ("NPC") and Gaucher disease. Learn more at www.cytrx.com.

    Forward-Looking Statements

    This press release may contain forward-looking statements. These forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any expected future results, performance, or achievements. Forward-looking statements speak only as of the date they are made and none of CytRx nor its affiliates assume any duty to update forward-looking statements. Words such as "anticipate," "believe," "could," "estimate," "expect," "may," "plan," "will," "would'' and other similar expressions are intended to identify these forward-looking statements. Important factors that could cause actual results to differ materially from those indicated by such forward-looking statements include, without limitation: the timing of, and CytRx's ability to, obtain and maintain regulatory approvals for clinical trials of CytRx's pharmaceutical candidates; the timing and results of CytRx's planned clinical trials for its pharmaceutical candidates; the amount of funds CytRx requires for its pharmaceutical candidates; increased levels of competition; changes in political, economic or regulatory conditions generally and in the markets in which CytRx operates; CytRx's ability to retain and attract senior management and other key employees; CytRx's ability to quickly and effectively respond to new technological developments; CytRx's ability to protect its trade secrets or other proprietary rights, operate without infringing upon the proprietary rights of others and prevent others from infringing on CytRx's proprietary rights; and the impact of the ongoing COVID-19 pandemic on CytRx's results of operations, business plan and the global economy. A discussion of these and other factors with respect to CytRx is set forth in the most recent annual and quarterly reports filed by the CytRx with the SEC, including disclosures under the heading "Risk Factors", and current reports filed since the date of the CytRx's most recent annual report. All forward-looking statements are based upon information available to the CytRx on the date the statements are first published. CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

    View Full Article Hide Full Article
  3. CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today announced the closing of its previously announced sale of its securities pursuant to a securities purchase agreement (the "Purchase Agreement") to a single healthcare-focused institutional investor (the "Investor") for aggregate gross proceeds of approximately $10 million. The Investor is independent of the Company's Board of Directors and management team. The Company intends to use the net proceeds of the offering for working capital purposes.

    Under the terms of the Purchase Agreement, CytRx sold 2,000,000 shares of its common stock at a purchase…

    CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today announced the closing of its previously announced sale of its securities pursuant to a securities purchase agreement (the "Purchase Agreement") to a single healthcare-focused institutional investor (the "Investor") for aggregate gross proceeds of approximately $10 million. The Investor is independent of the Company's Board of Directors and management team. The Company intends to use the net proceeds of the offering for working capital purposes.

    Under the terms of the Purchase Agreement, CytRx sold 2,000,000 shares of its common stock at a purchase price of $0.88 per share for total gross proceeds of approximately $1.76 million in a registered direct offering and 8,240 shares of Series C 10.00% Convertible Preferred Stock (the "Preferred Stock") at a purchase price of $1,000 per share for total gross proceeds of approximately $8.24 million, in a concurrent private placement. The shares of the Preferred Stock are convertible, upon shareholder approval as described below, into an aggregate of up to 9,363,637 shares of common stock at a conversion price of $0.88 per share. The Preferred Stock includes beneficial ownership limitations that preclude conversion that would result in the Investor owning in excess of 9.99% of the Company's outstanding shares of common stock.

    CytRx also issued to the Investor an unregistered preferred investment option (the "Investment Option") that allows for the purchase of up to 11,363,637 shares of common stock for additional gross proceeds of approximately $10 million if the Investment Option is exercised in full. The exercise price for the Investment Option is $0.88 per share. The Investment Option has a term equal to five and one-half years commencing upon the Company increasing its authorized common stock following shareholder approval (the "Authorized Share Increase").

    H.C. Wainwright & Co. acted as exclusive placement agent for the offering.

    As described above, the issuance of the shares of common stock underlying the Preferred Stock and the Investment Option sold in the private placement is subject to the Authorized Share Increase. Pursuant to the Purchase Agreement, the Company must hold a meeting of its stockholders no later than September 25, 2021 to seek shareholder approval.

    The shares of common stock sold in the registered direct offering were offered and sold in the registered direct offering by CytRx pursuant to a "shelf" registration statement on Form S-3 (File No. 333-255431), including a base prospectus, previously filed with, and declared effective by the Securities and Exchange Commission (the "SEC") on July 12, 2021. The registered direct offering was made only by means of a prospectus supplement that forms a part of the registration statement. A final prospectus supplement and an accompanying base prospectus relating to the registered direct offering has been filed with the SEC and is available on the SEC's website located at http://www.sec.gov. Electronic copies of the prospectus supplement and accompanying base prospectus may be obtained from H.C. Wainwright & Co., 430 Park Avenue, New York, NY 10022 or via telephone at (212) 856-5711 or email at .

    The Series C Preferred Stock and Investment Options sold in the private placement and the shares of common stock issuable thereunder were offered pursuant to an applicable exemption from the registration requirements of the Securities Act of 1933, as amended (the "Act"), and have not been registered under the Act, or applicable state securities laws, and may not be offered or sold in the United States absent registration with the SEC or an applicable exemption from such registration requirements.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale, would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About CytRx Corporation

    CytRx Corporation (OTCQB:CYTR) is a biopharmaceutical company with expertise in discovering and developing new therapeutics principally to treat patients with cancer and neurodegenerative diseases. CytRx's most recent advanced drug conjugate, aldoxorubicin, is an improved version of the widely used anti-cancer drug doxorubicin and has been out-licensed to ImmunityBio, Inc. (NASDAQ:IBRX). In addition, CytRx's drug candidate, arimoclomol, was sold to Orphazyme A/S (NASDAQ:ORPH) in exchange for milestone payments and royalties. Orphazyme is developing arimoclomol in Niemann-Pick disease Type C ("NPC") and Gaucher disease. Learn more at www.cytrx.com.

    Forward-Looking Statements

    This press release may contain certain statements relating to future results which are forward-looking statements. These statements are not historical facts, but instead represent only CytRx's belief regarding future events, many of which, by their nature, are inherently uncertain and outside of CytRx's control. Forward-looking statements include those relating to the offering of CytRx's securities, including as to the expected use of proceeds from the offering described above and adverse changes in general economic and market conditions. Forward-looking statements also include statements relating to the potential receipt of EMA and FDA approval of arimoclomol, the CytRx's potential receipt of future milestone and royalty payments from Orphazyme and the achievement of long-term value for the CytRx's stockholders. Such statements involve risks and uncertainties that could cause actual events or results to differ materially from the events or results described in the forward-looking statements, including risks and uncertainties relating to the ability of Orphazyme to obtain regulatory approval for, manufacture and commercialize its products and therapies that use arimoclomol; the results of clinical trials involving arimoclomol; the amount, if any, of future milestone and royalty payments that we may receive from Orphazyme; and other risks and uncertainties described in the most recent annual and quarterly reports filed by the CytRx with the SEC, including disclosures under the heading "Risk Factors", and current reports filed since the date of the CytRx's most recent annual report. All forward-looking statements are based upon information available to the CytRx on the date the statements are first published. The CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

    View Full Article Hide Full Article
  4. CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today announced that it has entered into a securities purchase agreement (the "Purchase Agreement") with a single healthcare-focused institutional investor (the "Investor") for aggregate gross proceeds of approximately $10 million. Under the terms of the Purchase Agreement, CytRx has agreed to sell 2,000,000 shares of its common stock at a purchase price of $0.88 per share for total gross proceeds of approximately $1.76 million in a registered direct offering and 8,240 shares of Series C 10.00% Convertible Preferred Stock (the "Preferred Stock") at a…

    CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today announced that it has entered into a securities purchase agreement (the "Purchase Agreement") with a single healthcare-focused institutional investor (the "Investor") for aggregate gross proceeds of approximately $10 million. Under the terms of the Purchase Agreement, CytRx has agreed to sell 2,000,000 shares of its common stock at a purchase price of $0.88 per share for total gross proceeds of approximately $1.76 million in a registered direct offering and 8,240 shares of Series C 10.00% Convertible Preferred Stock (the "Preferred Stock") at a purchase price of $1,000 per share for total gross proceeds of approximately $8.24 million, in a concurrent private placement. The shares of the Preferred Stock will be convertible, upon shareholder approval as described below, into an aggregate of up to 9,363,637 shares of common stock at a conversion price of $0.88 per share. The Preferred Stock includes beneficial ownership limitations that preclude conversion that would result in the Investor owning in excess of 9.99% of the Company's outstanding shares of common stock.

    CytRx will also issue to the Investor an unregistered preferred investment option (the "Investment Option") that allows for the purchase of up to 11,363,637 shares of common stock for additional gross proceeds of approximately $10 million if the Investment Option is exercised in full. The exercise price for the Investment Option is $0.88 per share. The Investment Option shall have a term equal to five and one-half years commencing upon the Company increasing its authorized common stock following shareholder approval (the "Authorized Share Increase").

    H.C. Wainwright & Co. is acting as exclusive placement agent for the offering.

    CytRx intends to use the net proceeds received from the offering for working capital purposes.

    The registered direct offering and concurrent private placement are expected to close on or about July 15, 2021, subject to the satisfaction of customary closing conditions. The issuance of the shares of common stock underlying the Preferred Stock and the Investment Option sold in the private placement is subject to the Authorized Share Increase.

    The shares of common stock sold in the registered direct offering are being offered and sold in the registered direct offering by CytRx pursuant to a "shelf" registration statement on Form S-3 (File No. 333-255431), including a base prospectus, previously filed with and declared effective by the Securities and Exchange Commission (the "SEC") on July 12, 2021. The registered direct offering is being made only by means of a prospectus supplement that forms a part of the registration statement. A final prospectus supplement and an accompanying base prospectus relating to the registered direct offering will be filed with the SEC and will be available on the SEC's website located at http://www.sec.gov. Electronic copies of the prospectus supplement and accompanying base prospectus may be obtained, when available, from H.C. Wainwright & Co., 430 Park Avenue, New York, NY 10022 or via telephone at (212) 856-5711 or email at .

    The Series C Preferred Stock and Investment Options sold in the private placement and the shares of common stock issuable thereunder are being offered pursuant to an applicable exemption from the registration requirements of the Securities Act of 1933, as amended (the "Act"), and have not been registered under the Act, or applicable state securities laws, and may not be offered or sold in the United States absent registration with the SEC or an applicable exemption from such registration requirements.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale, would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    About CytRx Corporation

    CytRx Corporation (OTCQB:CYTR) is a biopharmaceutical company with expertise in discovering and developing new therapeutics principally to treat patients with cancer and neurodegenerative diseases. CytRx's most recent advanced drug conjugate, aldoxorubicin, is an improved version of the widely used anti-cancer drug doxorubicin and has been out-licensed to ImmunityBio, Inc. (NASDAQ:IBRX). In addition, CytRx's drug candidate, arimoclomol, was sold to Orphazyme A/S (NASDAQ:ORPH) in exchange for milestone payments and royalties. Orphazyme is developing arimoclomol in Niemann-Pick disease Type C ("NPC") and Gaucher disease. Learn more at www.cytrx.com.

    Forward-Looking Statements

    This press release may contain certain statements relating to future results which are forward-looking statements. These statements are not historical facts, but instead represent only CytRx's belief regarding future events, many of which, by their nature, are inherently uncertain and outside of CytRx's control. Forward-looking statements include those relating to the offering of CytRx's securities, including as to the consummation of the offering described above, the expected proceeds from the offering, the intended use of proceeds and the timing of the closing of the offering, which may be affected by, among others, delays in satisfying or failure to satisfy closing conditions for the registered direct offering and the concurrent private placement and adverse changes in general economic and market conditions. Forward-looking statements also includes statements relating to the potential receipt of EMA and FDA approval of arimoclomol, the CytRx's potential receipt of future milestone and royalty payments from Orphazyme and the achievement of long-term value for the CytRx's stockholders. Such statements involve risks and uncertainties that could cause actual events or results to differ materially from the events or results described in the forward-looking statements, including risks and uncertainties relating to the ability of Orphazyme to obtain regulatory approval for, manufacture and commercialize its products and therapies that use arimoclomol; the results of clinical trials involving arimoclomol; the amount, if any, of future milestone and royalty payments that we may receive from Orphazyme; and other risks and uncertainties described in the most recent annual and quarterly reports filed by the CytRx with the SEC, including disclosures under the heading "Risk Factors", and current reports filed since the date of the CytRx's most recent annual report. All forward-looking statements are based upon information available to the CytRx on the date the statements are first published. The CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

    View Full Article Hide Full Article
  5. CytRx Corporation (OTCQB:CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today commented on Orphazyme A/S's (NASDAQ:ORPH) ("Orphazyme") recently announced 24-month interim results of an open-label extension (OLE) trial of arimoclomol for the treatment of Niemann-Pick disease type C (NPC).

    Orphazyme announced 24-month interim results of an OLE trial, providing efficacy and safety data for its investigational treatment arimoclomol in NPC for up to 36 months. The data are featured in a presentation as part of the Parseghian Scientific Conference for NPC Research. The results demonstrate that arimoclomol provided a sustained benefit to study…

    CytRx Corporation (OTCQB:CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today commented on Orphazyme A/S's (NASDAQ:ORPH) ("Orphazyme") recently announced 24-month interim results of an open-label extension (OLE) trial of arimoclomol for the treatment of Niemann-Pick disease type C (NPC).

    Orphazyme announced 24-month interim results of an OLE trial, providing efficacy and safety data for its investigational treatment arimoclomol in NPC for up to 36 months. The data are featured in a presentation as part of the Parseghian Scientific Conference for NPC Research. The results demonstrate that arimoclomol provided a sustained benefit to study participants by reducing NPC progression as measured by the 5-domain NPC Clinical Severity Scale (5D-NPCCSS).

    "Following on the outcomes from the 12-month double-blind phase, which indicated a clinically meaningful effect on disease progression, these longer-term data provide an encouraging picture that arimoclomol could deliver a sustained benefit and consistent safety profile over time," said Marc Patterson, MD, Professor of Neurology, Pediatrics and Medical Genetics, Mayo Clinic Children's Center in Rochester, MN.

    Orphazyme stated arimoclomol demonstrated a consistent safety profile throughout the 36-month treatment period. Adverse events observed during the open label extension phase were similar to those observed in the double-blind phase. A total of 41 patients joined the OLE following the double-blind period; 33 have now completed up to 36 months of treatment.

    Orphazyme also indicated data from the 36-month period support the findings from the 12-month double-blind period, which showed a clinically meaningful difference on the 5-domain NPCCSS, with a significant p-value of 0.046 (previously calculated at p=0.0537).

    Orphazyme CEO Christophe Bourdon stated: "These data provide further evidence of the clinical profile of arimoclomol to treat this population and may support our efforts to pursue regulatory approval to deliver a much-needed option for the NPC community. We continue to evaluate our path forward in the U.S. following the recent FDA response, and our application remains under active review in the European Union."

    Steven A. Kriegsman, Chairman and Chief Executive Officer of CytRx, stated: "We are optimistic about the direction Orphazyme is headed in and look forward to continued developments on the path to regulatory approval for arimoclomol for NPC in Europe and the U.S. Additionally, we are excited by the interim results of the open-label extension trial, which indicated that arimoclomol had a clinically meaningful effect on reducing NPC progression in participants. The data further demonstrates the significance of what arimoclomol can do for society and highlights the importance of focusing on its advancement. We recognize that progress in the biopharmaceutical industry is not always linear, and we believe the actions Orphazyme is taking through its announced restructuring plan will only serve to strengthen the company and streamline its efforts on its drug pipeline progress."

    Orphazyme disclosed that its financial outlook remains unchanged for 2021.

    Orphazyme noted it will provide an update and further information in connection with the publication of its interim report for the first half of 2021, due for release August 24, 2021.

    About CytRx Corporation

    CytRx Corporation (OTCQB:CYTR) is a biopharmaceutical company with expertise in discovering and developing new therapeutics principally to treat patients with cancer and neurodegenerative diseases. CytRx's drug candidate, arimoclomol, was sold to Orphazyme A/S (Nasdaq Copenhagen exchange: ORPHA.CO) in exchange for milestone payments and royalties. Orphazyme is developing arimoclomol in Niemann-Pick disease Type C ("NPC") and Gaucher disease. Learn more at www.cytrx.com.

    About Orphazyme

    Orphazyme is a biopharmaceutical company focused on bringing novel treatments to patients living with life threatening or debilitating rare diseases. Their research focuses on developing therapies for diseases caused by misfolding of proteins including lysosomal storage diseases. Arimoclomol, the company's lead candidate, is in clinical development in Niemann-Pick disease Type C and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme shares are listed on NASDAQ: ORPH. For more information, please visit www.orphazyme.com.

    About Niemann-Pick disease type C

    Niemann-Pick disease type C (NPC) is a rare, genetic, progressively debilitating, and often fatal neurovisceral disease. It belongs to a family known as lysosomal storage diseases and is caused by mutations leading to defective NPC protein. As a consequence, lipids that are normally cleared by the lysosome accumulate in tissues and organs, including the brain, and drive the disease pathology. We estimate the incidence of NPC to be one in 100,000 live births and the number of NPC patients in the United States and in Europe to be approximately 1,800 individuals. There are no approved treatments for NPC in the U.S.

    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, crosses the blood brain barrier, and has been studied in seven Phase 1, four Phase 2 and one pivotal Phase 2/3 clinical trial. Arimoclomol is in clinical development at Orphazyme for the treatment of NPC and Gaucher disease. Arimoclomol has received orphan drug designation for NPC in the US and EU, as well as fast-track designation from the US Food and Drug Administration (FDA) for NPC. In addition, arimoclomol has received breakthrough therapy designation and rare-pediatric disease designation from the FDA for NPC.

    Forward-Looking Statements

    This press release contains forward-looking statements, including statements relating to the potential receipt of EMA and FDA approval of arimoclomol, the Company's potential receipt of future milestone and royalty payments from Orphazyme and the achievement of long-term value for the Company's stockholders. Such statements involve risks and uncertainties that could cause actual events or results to differ materially from the events or results described in the forward-looking statements, including risks and uncertainties relating to the ability of Orphazyme to obtain regulatory approval for, manufacture and commercialize its products and therapies that use arimoclomol; the results of clinical trials involving arimoclomol; the amount, if any, of future milestone and royalty payments that we may receive from Orphazyme; and other risks and uncertainties described in the most recent annual and quarterly reports filed by the Company with the SEC and current reports filed since the date of the Company's most recent annual report. All forward-looking statements are based upon information available to the Company on the date the statements are first published. The Company undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

    View Full Article Hide Full Article
View All Orphazyme A/S News