1. 12-Month Study of Arimoclomol Showed a Clinically Meaningful Treatment Effect, Corresponding to a Reduction in Disease Progression

    Highlights Arimoclomol is Under Regulatory Review in Europe, With a Decision Expected in Q4 2021

    Notes Orphazyme Stated it Continues to Evaluate a Path Forward for Arimoclomol in the U.S.

    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 Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") published the results from a Phase 2/3 trial of arimoclomol, an investigational heat-shock protein amplifier, in Niemann-Pick disease type C (NPC) in the peer-reviewed…

    12-Month Study of Arimoclomol Showed a Clinically Meaningful Treatment Effect, Corresponding to a Reduction in Disease Progression

    Highlights Arimoclomol is Under Regulatory Review in Europe, With a Decision Expected in Q4 2021

    Notes Orphazyme Stated it Continues to Evaluate a Path Forward for Arimoclomol in the U.S.

    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 Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") published the results from a Phase 2/3 trial of arimoclomol, an investigational heat-shock protein amplifier, in Niemann-Pick disease type C (NPC) in the peer-reviewed Journal of Inherited Metabolic Disease (JIMD). The online publication is available here.

    The Phase 2/3 trial (NPC-002; ClinicalTrials.gov identifier: NCT02612129), was a prospective, randomized, double-blind, placebo-controlled study. Fifty patients aged 2–18 years were randomized 2:1 to arimoclomol:placebo, stratified by miglustat use. Routine clinical care was maintained. Arimoclomol was administered orally three times daily. The primary endpoint was change in 5-domain NPC Clinical Severity Scale (NPCCSS) score from baseline to 12 months, as described by Mengel et al.1 and Patterson et al2. The 5-domain NPCCSS comprises the domains determined to be most clinically relevant to patients, caregivers, and clinicians: ambulation, cognition, fine motor skills, speech, and swallowing (Cortina-Borja et al.3). A recent validation of the 5-domain NPCCSS shows that a change of 1 point or greater on the total score constitutes a clinically meaningful change for caregivers/patients and physicians (Patterson et al4).

    At 12-months, a significant treatment effect in favor of arimoclomol of −1.40 points (95% CI: −2.76, −0.03; p = 0.046) was observed, corresponding to a 65% relative reduction in annual disease progression. In the prespecified subgroup of patients receiving miglustat as routine care, arimoclomol resulted in stabilization of disease severity with a treatment difference of −2.06 in favor of arimoclomol (p = 0.006). In the pre-specified subgroup of patients ≥4 years of age the mean treatment difference was −1.80 in favor of arimoclomol (p=0.016), corresponding to 82% relative reduction in annual disease progression.

    Arimoclomol was well-tolerated, with adverse events occurring in 88.2% of patients receiving arimoclomol and 75.0% of patients receiving placebo. Fewer patients had serious adverse events with arimoclomol (14.7%) versus placebo (31.3%).

    Orphazyme's Chief Medical Officer stated the following in an announcement this week:

    "We are pleased to share the data from our Phase 2/3 trial in JIMD. NPC is a rare, inherited progressive neurodegenerative disorder with a high unmet medical need for disease-modifying treatment options. This trial demonstrated a statistically significant and clinically meaningful treatment effect of arimoclomol in NPC supported by significant and consistent effects across several disease- and pharmacodynamic biomarkers. We believe these data establish the potential of arimoclomol as an efficacious and well-tolerated disease-modifying treatment for NPC."

    Orphazyme's Chief Executive Officer added the following in an announcement this week:

    "We are committed to serving the NPC community and are working expeditiously to deliver this potential new medicine to patients. Arimoclomol is under regulatory review in Europe, with an anticipated CHMP opinion in Q4 2021, and we continue to evaluate the path forward in the U.S. following the recent FDA response."

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

    "CytRx is encouraged by Orphazyme's published results of its Phase 2/3 trial of arimoclomol in Niemann-Pick disease type C, which exhibited a statistically significant and clinically meaningful treatment effect on reducing disease progression. The biomarker data further suggests arimoclomol can be an effective solution for young patients suffering from NPC, with relatively few serious adverse effects. We look forward to monitoring Orphazyme's pursuit of European regulatory approval for arimoclomol in Q4 2021 and additional developments in the U.S. following the FDA's feedback."

    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 contains 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 statements relating to the potential receipt of EMA and FDA approval of arimoclomol and the CytRx's potential receipt of future milestone and royalty payments from Orphazyme. 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.

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    1 Mengel E, Bembi B, Del Toro M, et al (2020) Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study. Orphanet J Rare Dis 15: 328.

    2 Patterson MC, Lloyd-Price L, Guldberg C, et al (2021) Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale. Orphanet J Rare Dis 16: 79.

    3 Cortina-Borja M, Vruchte D, Mengel E, et al (2018) Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials. Orphanet J Rare Dis 13: 143. doi:110.1186/s13023-13018-10880-13029.

    4 Patterson MC, Lloyd-Price L, Guldberg C, et al (2021) Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale. Orphanet J Rare Dis 16: 79.

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  2. 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 commented on its results for the second quarter ended June 30, 2021. In addition, CytRx recapped corporate developments as well as matters pertaining to its agreements with ImmunityBio, Inc. (NASDAQ:IBRX) ("ImmunityBio") and Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme"). The Company's 10-Q was filed today.

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

    "We took important steps to enhance our capital position and further strengthen our corporate governance during the second quarter. By raising gross proceeds of $10…

    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 commented on its results for the second quarter ended June 30, 2021. In addition, CytRx recapped corporate developments as well as matters pertaining to its agreements with ImmunityBio, Inc. (NASDAQ:IBRX) ("ImmunityBio") and Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme"). The Company's 10-Q was filed today.

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

    "We took important steps to enhance our capital position and further strengthen our corporate governance during the second quarter. By raising gross proceeds of $10 million from our recent financing, we have obtained valuable working capital that can help us maintain stability as we efficiently manage our portfolio of licensing agreements and high-potential assets. We continue to believe in the long-term promise of our licensed drugs and Centurion Biopharma. We look forward to monitoring Orphazyme's pursuit of European regulatory approval for arimoclomol in Q4 2021 and ImmunityBio's Q1 2022 release of Cohort C survival data from its QUILT 88 study, which is a Phase 2 pancreatic cancer trial that includes aldoxorubicin."

    First Quarter Financial Overview

    • CytRx concluded the quarter ended June 30, 2021 with cash on hand of approximately $8.4 million.
    • The Company recorded a net loss of $1.2 million for the quarter ended June 30, 2021, compared to a net loss of $1.3 million for the same period in 2020.
      • General and administrative expenses were $1.2 million for the quarter, compared with $1.4 million for the same period in 2020.

    Recent Developments

    Corporate Highlights

    • Last month, CytRx entered into a securities purchase agreement with a healthcare-focused institutional investor, resulting in 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 for working capital purposes.
    • Last month, Jennifer K. Simpson, Ph.D joined the Company's Board of Directors. Dr. Simpson is the Chief Executive Officer and a Director of Panbela Therapeutics Inc. (NASDAQ:PBLA), a clinical stage drug development company. Shortly after joining Panbela Therapeutics, 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.
    • With respect to Centurion Biopharma, Mr. Kriegsman and Lead Director Louis Ignarro, PhD continued pursuing strategic partnership opportunities to advance clinical testing for the platform's assets. Discussions with prospective partners under confidentiality agreements are ongoing. There are no formal updates to report at this time.
    • CytRx maintains federal and state net operating loss ("NOL") carryforwards of $327.6 million and $252.6 million, respectively, available to offset against future taxable income. Of this amount, $258.3 million of federal NOLs and $252.6 million of state NOLs are unrestricted.

    ImmunityBio Highlights

    • ImmunityBio announced during the quarter that Cohort C of its QUILT 88 study in pancreatic cancer, 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. An early readout of survival data is expected in the first quarter of 2022.

    Orphazyme Highlights

    • Orphazyme announced during the quarter that it received a Complete Response Letter ("CRL") from the Food and Drug Administration ("FDA") following its review of the new drug application for arimoclomol. Orphazyme disclosed that the FDA issued the CRL based on needing additional evidence to further substantiate the validity and interpretation of the 5-domain NPC Clinical Severity Scale and, in particular, the swallow domain. Further, the FDA noted in the CRL that additional data is needed to bolster confirmatory evidence beyond the single phase 2/3 clinical trial to support the benefit-risk assessment of the NDA.
    • Subsequently, Orphazyme announced 24-month interim results of an open-label extension ("OLE") trial of arimoclomol for the treatment of NPC. Orphazyme provided efficacy and safety data for its investigational treatment arimoclomol in NPC for up to 36 months. 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).
    • Orphazyme is expecting prospective European regulatory approval for arimoclomol in the treatment of NPC by the end of 2021.

    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 contains 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 statements relating to the potential receipt of EMA and FDA approval of arimoclomol and the CytRx's potential receipt of future milestone and royalty payments from Orphazyme. 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.

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  3. 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.

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  4. 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.

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  5. 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 placements@hcwco.com.

    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.

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  6. 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 placements@hcwco.com.

    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.

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  7. 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.

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  8. CytRx Corporation (OTCQB:CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today noted that Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") announced it has received a Complete Response Letter ("CRL") from the U.S. Food and Drug Administration ("FDA") following its review of the new drug application for arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C ("NPC").

    Orphazyme's announcement disclosed that the FDA issued the CRL based on needing additional qualitative and quantitative evidence to further substantiate the validity and interpretation of the 5-domain NPC Clinical Severity Scale ("NPCCSS…

    CytRx Corporation (OTCQB:CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today noted that Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") announced it has received a Complete Response Letter ("CRL") from the U.S. Food and Drug Administration ("FDA") following its review of the new drug application for arimoclomol, a heat shock protein amplifier intended for the treatment of Niemann-Pick disease type C ("NPC").

    Orphazyme's announcement disclosed that the FDA issued the CRL based on needing additional qualitative and quantitative evidence to further substantiate the validity and interpretation of the 5-domain NPC Clinical Severity Scale ("NPCCSS") and, in particular, the swallow domain. Further, the FDA noted in the CRL that additional data are needed to bolster confirmatory evidence beyond the single phase 2/3 clinical trial to support the benefit-risk assessment of the NDA.

    A primary endpoint of the phase 2/3 clinical trial was progression in disease severity as measured by the 5-domain NPCCSS. This is a disease-specific measure of disease progression consisting of the five clinically most relevant domains to patients with NPC, caregivers and physicians.

    Orphazyme CEO Christophe Bourdon stated: "We are disheartened by the outcome of the FDA's review, given the urgent need for a new therapeutic option for NPC, but we remain committed to working with the regulators, with the goal of delivering arimoclomol to families managing this challenging disease. We will focus our efforts on pursuing the European regulatory approval, with CHMP opinion expected in Q4 2021 and potential Marketing Authorization in Q1 2022. We are assessing the potential path forward in the U.S. in partnership with the FDA. In the short-term, we will need to reduce our costs substantially and freeze all company efforts not related to clinical and regulatory activities to support approval for NPC."

    Orphazyme disclosed that as stated in its Annual Report 2020, initial outlook for the year was subject to various risks and uncertainties, including but not limited to the timing of regulatory decisions, the success of Orphazyme's commercial efforts and development activities. The outcome of the FDA decision has significant influence on Orphazyme's outlook for full-year 2021. Orphazyme's cash position at year-end 2021 is now expected to be approximately $8 million (previously $56 million).

    Orphazyme noted it will provide an update and further information in the coming weeks.

    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.

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  9. CytRx Corporation (OTCQB:CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today noted that Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") announced that the ORARIALS-01 pivotal trial for arimoclomol in Amyotrophic Lateral Sclerosis ("ALS") did not meet primary and secondary endpoints to show benefit in people living with the disease. According to Orphazyme, no important safety signals were reported in the trial. Topline data will be presented at the upcoming virtual European Network to Cure ALS meeting and complete data from the study will be published later this year.

    Orphazyme's announcement disclosed that the randomized, placebo-controlled…

    CytRx Corporation (OTCQB:CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today noted that Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") announced that the ORARIALS-01 pivotal trial for arimoclomol in Amyotrophic Lateral Sclerosis ("ALS") did not meet primary and secondary endpoints to show benefit in people living with the disease. According to Orphazyme, no important safety signals were reported in the trial. Topline data will be presented at the upcoming virtual European Network to Cure ALS meeting and complete data from the study will be published later this year.

    Orphazyme's announcement disclosed that 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) 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).

    Notably, Orphazyme's applications for arimoclomol (to be branded MIPLYFFA™) for Niemann-Pick disease type C are under priority review with the U.S. Food and Drug Administration, with an expected PDUFA action date of June 17, 2021, and with the European Medicines Agency, an opinion from their Committee for Medicinal Products for Human Use is expected later this year.

    CytRx will continue to provide updates that are relevant to our agreement with Orphazyme.

    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 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, IBM and ALS in the US and EU, as well as fast-track designation from the US Food and Drug Administration (FDA) for NPC, IBM and ALS. 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.

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  10. CytRx Corporation (OTCQB:CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today noted that Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") announced that its phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis ("IBM") did not meet its primary and secondary endpoints. According to Orphazyme, the primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale.

    Orphazyme announced earlier this week that the randomized, placebo-controlled trial was conducted among 150 IBM patients at 12 sites in North America and Europe, in partnership with University…

    CytRx Corporation (OTCQB:CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today noted that Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") announced that its phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis ("IBM") did not meet its primary and secondary endpoints. According to Orphazyme, the primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale.

    Orphazyme announced earlier this week that the randomized, placebo-controlled trial was conducted among 150 IBM patients at 12 sites in North America and Europe, in partnership with University College of London and the University of Kansas. Participants were randomized (1:1 ratio) to receive either arimoclomol citrate (400 mg three times daily) or placebo for up to 20 months. No important safety concerns were detected in the trial. The analysis of the data is continuing and complete findings from the study will be shared in a future scientific forum.

    Orphazyme's interim Chief Executive Officer commented: "We continue to believe in the promise of arimoclomol and heat shock protein science and are fully committed to our mission to deliver new therapies to patients."

    CytRx will continue to provide updates that are relevant to our agreement with Orphazyme.

    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 four indications including Amyotrophic Lateral Sclerosis ("ALS"), Niemann-Pick disease Type C ("NPC"), Gaucher disease and Inclusion Body Myositis ("IBM"). 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 for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. 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 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, Gaucher disease, IBM and ALS. Arimoclomol has received orphan drug designation for NPC, IBM and ALS in the US and EU, as well as fast-track designation from the US Food and Drug Administration (FDA) for NPC, IBM and ALS. 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.

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  11. Orphazyme Announces the Global Brand Name for Arimoclomol – MIPLYFFA – Ahead of Prospective FDA and EMA Approvals

    Expanded its Early Access Program for NPC in the U.S. and Europe as Well as Expectation of European Regulatory Decision by Q4 2021

    CytRx Corporation (OTCQB:CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today highlighted that Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") has released a new investor presentation that outlines its momentum ahead of potential U.S. and European approval of arimoclomol for the treatment of Niemann-Pick disease Type C ("NPC"). The March 2nd presentation can be found under the "Events & Presentations…

    Orphazyme Announces the Global Brand Name for Arimoclomol – MIPLYFFA – Ahead of Prospective FDA and EMA Approvals

    Expanded its Early Access Program for NPC in the U.S. and Europe as Well as Expectation of European Regulatory Decision by Q4 2021

    CytRx Corporation (OTCQB:CYTR) ("CytRx"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today highlighted that Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") has released a new investor presentation that outlines its momentum ahead of potential U.S. and European approval of arimoclomol for the treatment of Niemann-Pick disease Type C ("NPC"). The March 2nd presentation can be found under the "Events & Presentations" section of www.orphazyme.com. CytRx has an agreement with Orphazyme that can yield potential milestone payments and future single and double-digit royalties paid on sales of arimoclomol.

    Items noted in the presentation include:

    • Arimoclomol expected to be first to market in NPC in the U.S.
    • In anticipation of U.S. Food and Drug Administration ("FDA") and European Medicine Agency ("EMA") approval, Orphazyme announced MIPLYFFA as the global brand name for arimoclomol.
    • Orphazyme has expanded its NPC Early Access Program (EAP) in the U.S. and opened similar programs in France and Germany.
    • Orphazyme now expects to receive a decision on European regulatory approval for arimoclomol for NPC by Q4 2021.
    • Arimoclomol for NPC remains under the FDA's priority review with a Prescription Drug User Fee Act target action date of June 17, 2021.
    • Orphazyme has expanded its commercial organization in the U.S. and EU while extending its footprint to support launch readiness.
    • Goal is to position arimoclomol as first-line in NPC globally.
    • Orphazyme projects arimoclomol sales for NPC this year of up to $20 million.

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

    "We are very excited to see Orphazyme announce a global brand name for arimoclomol ahead of potential regulatory approvals in the U.S. and Europe. We are equally enthusiastic about the company's commitment to expanding EAPs and fortifying its global operations in anticipation of commercializing MIPLYFFA. In our view, Orphazyme is taking the right steps to commence future distribution and enhance its engagement with providers, patients and regulators. CytRx looks forward to monitoring Orphazyme's progress as it works to make MIPLYFFA available for NPC and three other rare diseases over time."

    We will continue to provide updates that are relevant to our agreement with Orphazyme. As noted, Orphazyme's March 2nd presentation can be found under the "Events & Presentations" section of www.orphazyme.com.

    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 four indications including Amyotrophic Lateral Sclerosis ("ALS"), Niemann-Pick disease Type C ("NPC"), Gaucher disease and Inclusion Body Myositis ("IBM"). 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 for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. 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 NPC

    Niemann-Pick disease Type C (NPC) is a rare, genetic and progressive disease that impairs the ability of the body to move cholesterol and other fatty substances (lipids) inside the cells. The result is an accumulation of lipids within the body's tissue, including the brain tissue, causing damage to the affected 2 areas. The symptoms upon onset of NPC vary from fatality during the first months after birth to a progressive disorder not diagnosed until adulthood. The disease affects neurologic and psychiatric functions as well as various internal organs. Systemic symptoms of NPC are more common in infancy or childhood and the rate of progression is usually much slower in individuals with onset of symptoms during adulthood. NPC is usually fatal and the majority of individuals with the disease die before the age of 20. NPC has been granted Orphan Drug Designation (EU and U.S.) for the treatment of NPC. It is estimated 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. and only one approved product in Europe called miglustat.

    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, Gaucher disease, IBM and ALS. Arimoclomol has received orphan drug designation for NPC, IBM and ALS in the US and EU, as well as fast-track designation from the US Food and Drug Administration (FDA) for NPC, IBM and ALS. 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.

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  12. Christophe Bourdon's Deep Commercialization Background and Vast International Experience Make Him the Ideal CEO For Orphazyme

    CEO Relocation to Copenhagen Seen as a Positive Sign of the Commercial Potential of Arimoclomol

    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 Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") has announced the appointment of Christophe Bourdon as its new Chief Executive Officer, effective as of April 1, 2021. This appointment comes as Orphazyme awaits potential Food and Drug Administration ("FDA") approval of arimoclomol for the treatment of Niemann-Pick…

    Christophe Bourdon's Deep Commercialization Background and Vast International Experience Make Him the Ideal CEO For Orphazyme

    CEO Relocation to Copenhagen Seen as a Positive Sign of the Commercial Potential of Arimoclomol

    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 Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") has announced the appointment of Christophe Bourdon as its new Chief Executive Officer, effective as of April 1, 2021. This appointment comes as Orphazyme awaits potential Food and Drug Administration ("FDA") approval of arimoclomol for the treatment of Niemann-Pick disease Type C ("NPC"). CytRx has an agreement with Orphazyme that can yield potential milestone payments and future single and double-digit royalties paid on sales of arimoclomol.

    As reported by Orphazyme, Mr. Bourdon has successfully launched a variety of products in demanding environments, making him an ideal individual to lead Orphazyme as it prepares for a potential commercial launch of arimoclomol. He joins from Amgen, Inc., where he has held the role of Senior Vice President, General Manager for the U.S. Oncology Business. He was leading commercialization planning and execution for several products. Previously, Mr. Bourdon was Senior Vice President of Europe, Middle East, Africa and Canada at Alexion Pharmaceuticals Inc. as the company launched two breakthrough ultra-orphan drugs and negotiated payor access across the United Kingdom, Germany, France, Italy and Canada. He holds an MBA from IMD business school (Switzerland) and a BA from ISG (France).

    Orphazyme's Chairman noted the following in a statement issued earlier this week:

    "I am delighted to announce Christophe Bourdon will join our leadership team as Chief Executive Officer starting April 1, 2021. This is an important time for Orphazyme, with numerous near-term milestones that will shape the company's future direction. Our Board conducted an extensive search for a leader who can guide not only our near-term execution, but also align the company around a vision for impact and scale for our long-term growth ambitions."

    Mr. Bourdon added the following:

    "The opportunity to join Orphazyme was compelling for me, not only based on its purposeful mission, but also the incredible near-term opportunities to create impact for patients. It is both exciting and humbling to assume this role at such a pivotal time. Building on the learnings I have gained from my invaluable experience at Amgen and the rare disease experience garnered at Alexion, I look forward to championing this talented team to advance the mission on behalf of our patient communities and deliver value for our shareholders. I am also very much looking forward to relocating to Copenhagen."

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

    "We are very pleased to see Orphazyme appoint a new, highly-qualified Chief Executive Officer at this exciting moment in time. We believe Mr. Bourdon's relocation to corporate headquarters in Copenhagen is a positive sign of Orphazyme's commercial potential for arimoclomol in its other three orphan diseases. Having experienced leadership atop the company is particularly important for potential commercialization of arimoclomol for NPC upon prospective FDA approval this year. In our view, Orphazyme continues to take the right steps to begin future distribution and deepen engagement with providers, patients and regulators across the globe. This year, Orphazyme may also receive a response to its submission for regulatory approval in Europe for arimoclomol to treat NPC."

    We will continue to provide updates that are relevant to our agreement with Orphazyme.

    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 four indications including Amyotrophic Lateral Sclerosis ("ALS"), Niemann-Pick disease Type C ("NPC"), Gaucher disease and Inclusion Body Myositis ("IBM"). 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 for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. 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 NPC

    Niemann-Pick disease Type C (NPC) is a rare, genetic and progressive disease that impairs the ability of the body to move cholesterol and other fatty substances (lipids) inside the cells. The result is an accumulation of lipids within the body's tissue, including the brain tissue, causing damage to the affected 2 areas. The symptoms upon onset of NPC vary from fatality during the first months after birth to a progressive disorder not diagnosed until adulthood. The disease affects neurologic and psychiatric functions as well as various internal organs. Systemic symptoms of NPC are more common in infancy or childhood and the rate of progression is usually much slower in individuals with onset of symptoms during adulthood. NPC is usually fatal and the majority of individuals with the disease die before the age of 20. NPC has been granted Orphan Drug Designation (EU and U.S.) for the treatment of NPC. It is estimated 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. and only one approved product in Europe called miglustat.

    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, Gaucher disease, IBM and ALS. Arimoclomol has received orphan drug designation for NPC, IBM and ALS in the US and EU, as well as fast-track designation from the US Food and Drug Administration (FDA) for NPC, IBM and ALS. 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.

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  13. NantKwest and ImmunityBio's QUILT 88 Trial Shows Novel Combination Immunotherapy, Which Includes Aldoxorubicin, Has More Than Doubled Historic Median Survival Rates in Patients

    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 NantKwest, Inc. (NASDAQ:NK) ("NantKwest") and ImmunityBio, Inc. ("ImmunityBio") have announced that their ongoing Phase 2 clinical trials of a novel combination immunotherapy – which include CytRx's licensed drug aldoxorubicin – for locally advanced or metastatic pancreatic cancer have produced early indications of increased survival rates for patients…

    NantKwest and ImmunityBio's QUILT 88 Trial Shows Novel Combination Immunotherapy, Which Includes Aldoxorubicin, Has More Than Doubled Historic Median Survival Rates in Patients

    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 NantKwest, Inc. (NASDAQ:NK) ("NantKwest") and ImmunityBio, Inc. ("ImmunityBio") have announced that their ongoing Phase 2 clinical trials of a novel combination immunotherapy – which include CytRx's licensed drug aldoxorubicin – for locally advanced or metastatic pancreatic cancer have produced early indications of increased survival rates for patients with no other approved treatment options. Interim results of the three-cohort trials, known as QUILT 88, show median survival rates more than doubled that of the historic rate in patients with advanced metastatic pancreatic cancer (for which no other FDA approved treatment exists).

    According to NantKwest and ImmunityBio, the current trials were based on the original Cancer Moonshot hypothesis and exploratory QUILT trials initiated in 2017. The companies announced this week that the trials appear to validate the theory that by orchestrating natural killer and T-cell therapy, survival rates for individuals afflicted with advanced metastatic pancreatic cancer could be improved without high-dose chemotherapy. The interim trial results disclosed by the companies are as follows:

    • In the Cancer Moonshot QUILT trials of haNK combined with PD-L1 inhibitor avelumab, which were completed in 2019, the median overall survival rate more than doubled (three months historic control versus 8 months in the treatment arm) in the 12-patient trial.
    • A complete remission was achieved when replacing haNK and PD-L1 inhibitor avelumab with PD-L1 t-haNK and four out of five patients who had not yet reached median survival time (three months) are alive 8-16 months since beginning treatment on these expanded protocols.
    • A single-arm Phase 2 trial (QUILT 88, Cohort C) was initiated in October 2020, for which the primary endpoint is overall survival and 15 out of 18 (83%) patients enrolled with second-line or greater pancreatic cancer remain alive to date.
    • Randomized Phase 2 trials (QUILT 88, Cohorts A and B) for first- and second-line metastatic pancreatic cancer are actively enrolling at three sites with more than 50 patients enrolled or being evaluated in QUILT 88 to date.

    Additional information pertaining to QUILT 88 trial details:

    According to NantKwest and ImmunityBio, QUILT 88 is intended to evaluate the comparative efficacy and overall safety of standard-of-care chemotherapy versus standard-of-care chemotherapy in combination with PD-L1 t-haNK, Anktiva (N-803) and aldoxorubicin in subjects with locally advanced or metastatic pancreatic cancer. Each treatment setting, as well as each first- and second-line or later maintenance treatment, will be evaluated independently as Cohort A, Cohort B, and Cohort C, respectively, with A and B having independent experimental and control arms. The trial will initially enroll 298 subjects across all three cohorts. The primary objective of Cohorts A and B is progression-free survival and the objective of Cohort C is overall survival per RECIST V1.1. Secondary objectives include initial safety and additional efficacy measures, including overall response rate, complete response rate, durability of response, disease control rate, and overall survival.

    Cancer Moonshot QUILT trial numbers include QUILT 3.039, 3.060, 3.070, and 3.080.

    Additional information pertaining to QUILT 88 trial sites and enrollment:

    According to NantKwest and ImmunityBio, there are currently three trial sites that have been activated: Hoag Memorial Hospital Presbyterian in Orange County, Calif.; The Chan Soon-Shiong Institute for Medicine in Los Angeles County, Calif.; and Avera McKennan Hospital and University Health Center in Sioux Falls, South Dakota. The latter site will serve patients in the tri-state area (Iowa, Nebraska, and South Dakota). More than 50 patients are currently enrolled in or being evaluated for the trial.

    Additional information pertaining to pancreatic cancer:

    Pancreatic cancer is the fourth leading cause of cancer-related deaths in the U.S., with an estimated 47,050 deaths and 57,600 new cases expected in 2020. It is the 12th most common cancer worldwide, with around 338,000 new cases diagnosed in 2012 (2% of all cancer diagnoses). Pancreatic cancer continues to increase today with no standard of care available for patients beyond second line. A clear unmet medical need exists in these patients facing anticipated survival times that are brief and high levels of existing comorbidities.

    Patrick Soon-Shiong, M.D., Chairman, and Chief Executive Officer of ImmunityBio, issued the following statements earlier this week:

    "For five patients for whom no other treatment was available, we replaced haNK and avelumab with the investigational NK cell therapy PD-L1 t-haNK and were pleased to observe a complete remission in the first patient to receive this combo therapeutic. To date, four out of five of these patients remain alive since beginning treatment. These observations confirmed the promise of our hypothesis that activating the patient's own immune system with low-dose chemo immunomodulation therapy could improve outcomes. On the basis of our initial studies, we initiated our QUILT 88 randomized trials in metastatic pancreatic cancer and are pleased to present today those findings, including Cohort C survival rates. While this data is still early, a doubling of the survival rate is encouraging and warrants further confirmation through QUILT 88."

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

    "We are very encouraged to see that Aldoxorubicin is helping to deliver positive outcomes for pancreatic cancer patients participating in the ongoing QUILT 88 trials. In our view, the interim results announced by NantKwest and ImmunityBio reflect the significant potential of the novel combination immunotherapy being studied. We look forward to monitoring the continuation of QUILT 88 and remain optimistic about the role Aldoxorubicin can play in combating aggressive cancers."

    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 advanced drug conjugate, aldoxorubicin, is an improved version of the widely used anticancer drug doxorubicin and has been out-licensed to ImmunityBio, Inc. In addition, CytRx's other drug candidate, arimoclomol, was sold to Orphazyme (NASDAQ:ORPH) Orphazyme A/S (Nasdaq Copenhagen exchange: ORPHA.CO) in exchange for milestone payments and royalties. Orphazyme is testing arimoclomol in four indications including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM) and have filed an NDA for NPC with a PDUFA date of June 17, 2021. CytRx Corporation's website is www.cytrx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements, including statements relating to the success of the QUILT 88 trial and other types of combination immunotherapy in improving the treatment and outcomes of cancer patients. 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, such as the risks and uncertainties relating to the ability of NantKwest and ImmunityBio to obtain regulatory approval for their products that use aldoxorubicin; the ability of NantKwest or ImmunityBio to continue their planned preclinical and clinical development of their respective development programs and the timing and success of any such continued preclinical and clinical development and planned regulatory submissions; the ability of NantKwest and ImmunityBio to manufacture and commercialize products or therapies that use aldoxorubicin; the amount, if any, of future milestone and royalty payments that we may receive from NantKwest and ImmunityBio; the final results of the QUILT 88 trial and other studies and trials of cancer treatments and therapies that include aldoxorubicin, including combination immunotherapies; and other risks and uncertainties described in the most recent annual and quarterly reports filed by CytRx with the Securities and Exchange Commission and current reports filed since the date of CytRx's most recent annual report. All forward-looking statements are based upon information available to 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.

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  14. 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 provided an update on the U.S. Food and Drug Administration's ("FDA") review of Orphazyme A/S's (NASDAQ:ORPH) ("Orphazyme") New Drug Application ("NDA") for arimoclomol to treat Niemann-Pick Disease Type C ("NPC"). According to Orphazyme, the FDA has extended the review period with a standard extension of three months in order to complete the NDA review. The updated Prescription Drug User Fee Act ("PDUFA") target action date is June 17, 2021.

    Orphazyme disclosed that the FDA has confirmed that the NDA remains under Priority Review. The FDA grants…

    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 provided an update on the U.S. Food and Drug Administration's ("FDA") review of Orphazyme A/S's (NASDAQ:ORPH) ("Orphazyme") New Drug Application ("NDA") for arimoclomol to treat Niemann-Pick Disease Type C ("NPC"). According to Orphazyme, the FDA has extended the review period with a standard extension of three months in order to complete the NDA review. The updated Prescription Drug User Fee Act ("PDUFA") target action date is June 17, 2021.

    Orphazyme disclosed that the FDA has confirmed that the NDA remains under Priority Review. The FDA grants Priority Review to applications for potential therapies that, if approved, could offer a significant improvement in safety or effectiveness, diagnosis, or prevention of serious conditions. Notably, the FDA has confirmed that the extension does not impede eligibility for a Pediatric Rare Disease Priority Review Voucher.

    Arimoclomol previously received the FDA's Fast-Track and Breakthrough Therapy Designations for NPC as well as Orphan Drug and Rare Pediatric Disease Designations. If approved in the U.S., arimoclomol will be the first and only approved medicine for NPC. In November 2020, Orphazyme also submitted a Marketing Authorisation Application to the European Medicines Agency for arimoclomol to treat NPC.

    Orphazyme's U.S. President issued the following statement on December 27, 2020:

    "Orphazyme is working closely with the FDA to support the final review of the new drug application for arimoclomol. There is significant unmet medical need for the treatment of NPC, and we are committed to bringing arimoclomol to patients in the U.S. and Europe as soon as possible."

    Orphazyme's Chief Medical Officer added:

    "We have responded to all FDA information requests and submitted all outstanding information regarding the arimoclomol NDA for NPC. The Phase 3 trials for Amyotrophic Lateral Sclerosis and Inclusion Body Myositis remain on track for read-out in the first half of 2021 and we look forward to providing an update on our progress."

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

    "We believe Orphazyme has taken a number of important steps in 2020 ahead of potential commercialization of arimoclomol for NPC upon prospective FDA approval during the first half of 2021. Orphazyme has strengthened its financial position through a successful capital raise and subsequently established a strong U.S. footprint with new headquarters in Chicago and the addition of more than 30 employees. In our view, Orphazyme is well positioned for future distribution and expanded engagement with providers, patients, regulators and the clinical testing community in 2021. It is also noteworthy that next year, Orphazyme may receive a response to its submission for regulatory approval in Europe for arimoclomol to treat NPC."

    We will continue to provide updates that are relevant to our agreement with Orphazyme.

    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 four indications including Amyotrophic Lateral Sclerosis ("ALS"), Niemann-Pick disease Type C ("NPC"), Gaucher disease and sporadic Inclusion Body Myositis ("sIBM"). 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 for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme shares are listed on Nasdaq (ORPHA). For more information, please visit www.orphazyme.com.

    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.

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  15. Highlights That Potential Merger Can Help Accelerate Current and Future Clinical Programs Utilizing CytRx's Licensed Drug Aldoxorubicin

    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 congratulated ImmunityBio, Inc. ("ImmunityBio") and NantKwest, Inc. (NASDAQ:NK) ("NantKwest") on their proposed merger and announced plans to create a leading immunotherapy and cell therapy company.

    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…

    Highlights That Potential Merger Can Help Accelerate Current and Future Clinical Programs Utilizing CytRx's Licensed Drug Aldoxorubicin

    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 congratulated ImmunityBio, Inc. ("ImmunityBio") and NantKwest, Inc. (NASDAQ:NK) ("NantKwest") on their proposed merger and announced plans to create a leading immunotherapy and cell therapy company.

    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.

    In their announcement, ImmunityBio and NantKwest highlighted that the combined entity will have a broad clinical-stage pipeline – including 13 assets in clinical trials and 11 in Phase 2 to 3 programs – as well as a robust early-stage pipeline to address other difficult to treat cancers. The companies also noted that the combination of NantKwest's Natural Killer cell platform and ImmunityBio's immunotherapy platforms – including albumin-based tumor microenvironment immune modulators (aldoxorubicin) – have already resulted in complete responses in aggressive and late-stage metastatic cancers. To date, complete responses have been noted in patients with second-line or greater metastatic pancreatic cancer, triple-negative breast cancer, head and neck cancer, and Merkel Cell Carcinoma.

    Notably, ImmunityBio and NantKwest recently announced the addition of a third cohort to an ongoing Phase 2 study of a novel combination immunotherapy – which includes aldoxorubicin – for locally advanced or metastatic pancreatic cancer (QUILT-88). As previously outlined in CytRx's October 7, 2020 announcement regarding the successful treatment delivered to former Nevada Senator Harry Reid for his stage IV pancreatic cancer, the randomized, open-label study is evaluating the safety and efficacy of a combination immunotherapy that includes aldoxorubicin, ImmunityBio's IL-15 superagonist Anktiva (N-803), NantKwest's PD-L1 t-haNK and standard of care. The study results will be compared to standard of care chemotherapy for first- and second-line treatment. However, the third-line cohort is a single arm with no comparator and each cohort will be evaluated independently to provide a more precise comparative.

    Dr. Patrick Soon-Shiong, founder and Chief Executive Officer of ImmunityBio, issued the following statement yesterday regarding the proposed merger:

    "We are developing next-generation immunotherapies to defeat cancer and infectious disease. By combining ImmunityBio's immunotherapy platform, which includes the Anktiva IL-15 superagonist, with NantKwest's natural killer cell platform, the merged entity will have a powerful and broad product portfolio that can activate both the innate (natural killer cell and macrophage) and adaptive (T cell) immune systems to create long-term immunological memory. What distinguishes the merged entity is the late stage immunotherapy product pipeline that is designed to eliminate the need for high-dose chemotherapy, improve the outcomes of current CAR T cell therapies, and extend beyond checkpoint inhibitors. With 13 clinical trials across multiple tumor types at Phase I to III and with the combined talent in research, clinical development and manufacturing, the merged entity is poised to be a leader in the immunotherapy space."

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

    "This proposed combination is an exciting development that can help ImmunityBio put even greater energy and resources behind innovative clinical programs, including its Phase 2 study of a novel pancreatic cancer treatment that includes aldoxorubicin. We are encouraged to see that ImmunityBio is presenting aldoxorubicin as one of the cornerstones of its clinical-stage platforms. If approved, this merger can be transformative for providers and patients fighting cancer and rare diseases."

    For more information about the transaction, stakeholders can visit www.immunitybio.com. ImmunityBio's website also notes that its albumin associated anthracycline is designed to release doxorubicin directly into tumor sites with lower side effects (reduced cardiotoxicity and loss of hair).

    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 advanced drug conjugate, aldoxorubicin, is an improved version of the widely used anti-cancer drug doxorubicin and has been outlicensed to ImmunityBio, Inc. In addition, CytRx's other drug candidate, arimoclomol, was sold to Orphazyme (NASDAQ:ORPH) Orphazyme A/S (Nasdaq Copenhagen exchange: ORPHA.CO) in exchange for milestone payments and royalties. Orphazyme is testing arimoclomol in four indications including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM). CytRx Corporation's website is www.cytrx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements, including statements relating to the proposed transaction involving NantKwest and ImmunityBio and their future success in improving the treatment of various diseases and illnesses. 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, such as the risks and uncertainties relating to the ability of NantKwest and ImmunityBio to successfully complete their proposed merger on a timely basis and obtain regulatory approval for their products that use aldoxorubicin; the ability of NantKwest or ImmunityBio to continue their planned preclinical and clinical development of their respective development programs and the timing and success of any such continued preclinical and clinical development and planned regulatory submissions; the ability of NantKwest and ImmunityBio to manufacture and commercialize products or therapies that use aldoxorubicin; the amount, if any, of future milestone and royalty payments that we may receive from NantKwest and ImmunityBio; the results of future studies and trials of cancer treatments and therapies that include aldoxorubicin, including combination immunotherapies; the ability of Orphazyme to obtain regulatory approval for its products that use arimoclomol; the ability of Orphazyme A/S to manufacture and commercialize products or therapies that use arimoclomol; the amount, if any, of future milestone and royalty payments that we may receive from Orphazyme A/S; and other risks and uncertainties described in the most recent annual and quarterly reports filed by CytRx with the Securities and Exchange Commission and current reports filed since the date of CytRx's most recent annual report. All forward-looking statements are based upon information available to 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.

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  16. Highlights Orphazyme's Establishment of U.S. Headquarters in Chicago and Recruitment of More Than 30 U.S. Employees

    Comments That Orphazyme's Investments in the U.S. are Intended to Support Regulatory Efforts and the Prospective Commercialization of Arimoclomol for Niemann-Pick Disease Type C and Possibly Other Rare Diseases in the Future

    CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical firm focused on research and development for the oncology and neurodegenerative disease categories, today highlighted that Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") has announced the expansion of its U.S. presence and workforce ahead of potential Food and Drug Administration ("FDA") approval of arimoclomol for the…

    Highlights Orphazyme's Establishment of U.S. Headquarters in Chicago and Recruitment of More Than 30 U.S. Employees

    Comments That Orphazyme's Investments in the U.S. are Intended to Support Regulatory Efforts and the Prospective Commercialization of Arimoclomol for Niemann-Pick Disease Type C and Possibly Other Rare Diseases in the Future

    CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical firm focused on research and development for the oncology and neurodegenerative disease categories, today highlighted that Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") has announced the expansion of its U.S. presence and workforce ahead of potential Food and Drug Administration ("FDA") approval of arimoclomol for the treatment of Niemann-Pick disease Type C ("NPC"). CytRx has an agreement with Orphazyme that can yield potential milestone payments and future royalties paid on sales of arimoclomol.

    In addition to establishing its U.S. headquarters in Chicago, Orphazyme has already recruited more than 30 U.S. employees. Orphazyme has also named three U.S. leaders to its global executive team:

    • Molly Painter, U.S. President, is heading the launch and commercial operations in the market.
    • Terri Stevens, Chief Business Officer, is responsible for global strategy, corporate development, and business development and licensing.
    • Molly Carey Poarch, Global and U.S. Head of Corporate Communications.

    Orphazyme's U.S. team, which is focused on regulatory review efforts and a first potential commercial launch in 2021, includes legal, commercial, finance, advocacy relations, and regulatory and medical affairs functions.

    Illinois State Senator Mattie Hunter issued the following statement this week:

    "I am excited to welcome Orphazyme, a fresh and innovative biopharmaceutical to Chicago's robust group of health care companies. I believe their methodology will make leeway in the fight against rare neurodegenerative diseases while bringing job growth to the city."

    Orphazyme's Chief Executive Officer delivered the following statement this week:

    "We have established a U.S. headquarters in Chicago to allow us to more closely engage with our partners and community members as we work together to pursue innovation for debilitating neurodegenerative diseases based on our pioneering science. Our U.S. growth will be fueled by the team of industry experts we have been assembling. We are excited to add three key U.S.-based leaders to our corporate executive team as we expand to support our ambition of serving rare disease communities around the world."

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

    "We are very pleased to see Orphazyme establish a strong U.S. footprint by investing in headquarters in Chicago and retaining more than 30 employees ahead of potential commercialization of arimoclomol for NPC upon prospective FDA approval in the first quarter of 2021. In our view, Orphazyme is taking the right steps to begin future distribution and deepen engagement with U.S. providers, patients, regulators, and the clinical testing community in 2021. It is also noteworthy that during the year 2021, Orphazyme may receive a response to its submission for regulatory approval in Europe for arimoclomol to treat NPC. Also, on or before June 30, 2021, Orphazyme is expected to report Phase 3 data on arimoclomol for sporadic Inclusion Body Myositis and Amyotrophic Lateral Sclerosis. We remain optimistic about Orphazyme's continued progress."

    We will continue to provide updates that are relevant to our agreement with Orphazyme.

    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 four indications including Amyotrophic Lateral Sclerosis ("ALS"), Niemann-Pick disease Type C ("NPC"), Gaucher disease and sporadic Inclusion Body Myositis ("sIBM"). 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 for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme shares are listed on Nasdaq (ORPHA). For more information, please visit www.orphazyme.com.

    About NPC

    Niemann-Pick disease Type C (NPC) is a rare, genetic and progressive disease that impairs the ability of the body to move cholesterol and other fatty substances (lipids) inside the cells. The result is an accumulation of lipids within the body's tissue, including the brain tissue, causing damage to the affected 2 areas. The symptoms upon onset of NPC vary from fatality during the first months after birth to a progressive disorder not diagnosed until adulthood. The disease affects neurologic and psychiatric functions as well as various internal organs. Systemic symptoms of NPC are more common in infancy or childhood and the rate of progression is usually much slower in individuals with onset of symptoms during adulthood. NPC is usually fatal and the majority of individuals with the disease die before the age of 20. NPC has been granted Orphan Drug Designation (EU and U.S.) for the treatment of NPC. It is estimated 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. and only one approved product in Europe called miglustat.

    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, Gaucher disease, sIBM and ALS. Arimoclomol has received orphan drug designation for NPC, sIBM and ALS in the US and EU, as well as fast-track designation from the US Food and Drug Administration (FDA) for NPC, sIBM and ALS. 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.

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  17. Applauds Orphazyme's Efforts to Pursue Approval in Europe for Arimoclomol in the Treatment of Niemann-Pick Disease Type C

    Notes That Arimoclomol for Niemann-Pick Disease Type C is Under the FDA's Priority Review With a Target Action Date of March 17, 2021

    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 Orphazyme A/S (ORPHA) (NASDAQ:ORPH) ("Orphazyme") has submitted a Marketing Authorisation Application ("MAA") to the European Medicines Agency ("EMA") for arimoclomol in the treatment of Niemann-Pick disease Type C ("NPC"). CytRx has an agreement with Orphazyme that can yield…

    Applauds Orphazyme's Efforts to Pursue Approval in Europe for Arimoclomol in the Treatment of Niemann-Pick Disease Type C

    Notes That Arimoclomol for Niemann-Pick Disease Type C is Under the FDA's Priority Review With a Target Action Date of March 17, 2021

    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 Orphazyme A/S (ORPHA) (NASDAQ:ORPH) ("Orphazyme") has submitted a Marketing Authorisation Application ("MAA") to the European Medicines Agency ("EMA") for arimoclomol in the treatment of Niemann-Pick disease Type C ("NPC"). CytRx has an agreement with Orphazyme that can yield potential milestone payments and future royalties paid on sales of arimoclomol.

    This submission of an MAA to the EMA comes on the heels of Orphazyme filing a New Drug Application ("NDA") in the United States for arimoclomol in the treatment of NPC. The NDA is currently under Priority Review with the U.S. Food and Drug Administration ("FDA") with a Prescription Drug User Fee Act ("PDUFA") target action date of March 17, 2021. It is also important to note that arimoclomol was previously granted Orphan Drug Designation in Europe and the U.S. and it received the FDA's Fast Track and Breakthrough Therapy Designations for NPC as well as Rare Pediatric Disease Designations in the U.S.

    With respect to the submission of an MAA to the EMA, Orphazyme's Chief Executive Officer delivered the following statement this week:

    "This filing in Europe is a significant milestone for Orphazyme as we work toward our first potential approvals of arimoclomol in major markets. There are few options today that can address the devastating effects of NPC, and we are hopeful we can address an important need for this community. We look forward to working with EMA as they complete their review of our application."

    Steven Kriegsman, Chairman and Chief Executive Officer of CytRx, added:

    "We are pleased that Orphazyme is building momentum and making strong progress in its pursuit of potential approvals for arimoclomol to treat NPC with both the FDA and EMA. Given the lack of treatment options for individuals afflicted with NPC, arimoclomol represents a possible breakthrough for patients with this brutal disease. We are encouraged that Orphazyme has publicly disclosed that it is investing in a strong foundation to underpin arimoclomol's prospective commercialization and distribution for NPC if it receives requisite approvals in March 2021."

    Mr. Kriegsman concluded:

    "It is also noteworthy that during the first half of 2021, Orphazyme will be reporting Phase 2/3 data on arimoclomol for sporadic Inclusion Body Myositis and Phase 3 data for amyotrophic lateral sclerosis. As we have said in the past, arimoclomol is a promising drug that can be a game changer for patients afflicted with NPC and other neurodegenerative diseases."

    CytRx will continue to provide updates that are relevant to our agreement with Orphazyme.

    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 four indications including amyotrophic lateral sclerosis ("ALS"), Niemann-Pick disease Type C ("NPC"), Gaucher disease and sporadic Inclusion Body Myositis ("sIBM"). 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 for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA). For more information, please visit www.orphazyme.com.

    About NPC

    Niemann-Pick disease Type C (NPC) is a rare, genetic and progressive disease that impairs the ability of the body to move cholesterol and other fatty substances (lipids) inside the cells. The result is an accumulation of lipids within the body's tissue, including the brain tissue, causing damage to the affected 2 areas. The symptoms upon onset of NPC vary from fatality during the first months after birth to a progressive disorder not diagnosed until adulthood. The disease affects neurologic and psychiatric functions as well as various internal organs. Systemic symptoms of NPC are more common in infancy or childhood and the rate of progression is usually much slower in individuals with onset of symptoms during adulthood. NPC is usually fatal and the majority of individuals with the disease die before the age of 20. NPC has been granted Orphan Drug Designation (EU and U.S.) for the treatment of NPC. It is estimated 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. and only one approved product in Europe called miglustat.

    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, Gaucher disease, sIBM and ALS. Arimoclomol has received orphan drug designation for NPC, sIBM and ALS in the US and EU, as well as fast-track designation from the US Food and Drug Administration (FDA) for NPC, sIBM and ALS. 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.

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  18. Applauds Orphazyme for Investing in Commercial and Other Pre-Launch Activities in Preparation of Potential FDA Approval of Arimoclomol to Treat Niemann-Pick Disease Type C

    Arimoclomol for Niemann-Pick Disease Type C is Under the FDA's Priority Review With a Target Action Date of March 17, 2021

    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 Orphazyme A/S (ORPHA.CO) (NASDAQ:ORPH) ("Orphazyme") has announced new plans to accelerate commercial and other pre-launch activities during the fourth quarter of 2020 in preparation for potential approval of arimoclomol in Niemann-Pick…

    Applauds Orphazyme for Investing in Commercial and Other Pre-Launch Activities in Preparation of Potential FDA Approval of Arimoclomol to Treat Niemann-Pick Disease Type C

    Arimoclomol for Niemann-Pick Disease Type C is Under the FDA's Priority Review With a Target Action Date of March 17, 2021

    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 Orphazyme A/S (ORPHA.CO) (NASDAQ:ORPH) ("Orphazyme") has announced new plans to accelerate commercial and other pre-launch activities during the fourth quarter of 2020 in preparation for potential approval of arimoclomol in Niemann-Pick disease Type C ("NPC"), which is currently under Priority Review by the U.S. Food and Drug Administration ("FDA") with a target action date of March 17, 2021. CytRx has an agreement with Orphazyme that can yield potential milestone payments and future royalties paid on sales of arimoclomol.

    According to Orphazyme, the organization's reinforced financial position following a successful global stock offering has enabled it to commit additional resources to efforts pertaining to arimoclomol. Orphazyme stated that it will further invest in its Early Access Programs, additional API manufacturing, regulatory affairs and clinical safety activities. Orphazyme also expects continued costs through year-end for the ongoing trials in sporadic Inclusion Body Myositis and amyotrophic lateral sclerosis in order to provide home nursing and direct to patient distribution due to the COVID-19 pandemic. Orphazyme has stated that it wants to be well-situated to commercialize and launch arimoclomol for NPC if it receives FDA approval in 2021.

    Orphazyme's Chief Executive Officer, issued the following statement last week:

    "There is real momentum here at Orphazyme as we move closer to potential approval of arimoclomol in the U.S. in its first indication of NPC and accelerating our preparatory efforts now will help ensure a smooth launch. There are currently no approved products for NPC in the U.S. and arimoclomol has the potential to make a significant difference to patients with this devastating disease, so our team is working expeditiously to ensure we are optimally positioned for a successful launch if approved."

    In September 2020, the FDA accepted Orphazyme's New Drug Application ("NDA") for arimoclomol for NPC, with priority review. The FDA set a target action date of March 17, 2021 under the Prescription Drug User Fee Act, or PDUFA, for completion of its review of the NDA. Orphazyme continues to expect to submit a Marketing Authorization Application, or MAA, to the European Medicines Agency in the second half of 2020.

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

    "We applaud Orphazyme for investing in a strong foundation to underpin arimoclomol's commercialization and distribution for NPC if it receives FDA approval in the first quarter of 2021. It is also noteworthy that during the first half of 2021, Orphazyme will be reporting Phase 3 data on arimoclomol for sporadic Inclusion Body Myositis and for amyotrophic lateral sclerosis. Arimoclomol is a promising drug that can hopefully be a gamechanger for patients afflicted with NPC and other neurodegenerative diseases. We remain very optimistic about the drug's long-term potential."

    We will continue to provide updates that are relevant to our agreement with Orphazyme.

    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 four indications including amyotrophic lateral sclerosis ("ALS"), Niemann-Pick disease Type C ("NPC"), Gaucher disease and sporadic Inclusion Body Myositis ("sIBM"). 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 for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme shares are listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit www.orphazyme.com.

    About NPC

    Niemann-Pick disease Type C (NPC) is a rare, genetic and progressive disease that impairs the ability of the body to move cholesterol and other fatty substances (lipids) inside the cells. The result is an accumulation of lipids within the body's tissue, including the brain tissue, causing damage to the affected 2 areas. The symptoms upon onset of NPC vary from fatality during the first months after birth to a progressive disorder not diagnosed until adulthood. The disease affects neurologic and psychiatric functions as well as various internal organs. Systemic symptoms of NPC are more common in infancy or childhood and the rate of progression is usually much slower in individuals with onset of symptoms during adulthood. NPC is usually fatal and the majority of individuals with the disease die before the age of 20. NPC has been granted Orphan Drug Designation (EU and U.S.) for the treatment of NPC. It is estimated 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. and only one approved product in Europe called miglustat.

    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, Gaucher disease, sIBM and ALS. Arimoclomol has received orphan drug designation for NPC, sIBM and ALS in the US and EU, as well as fast-track designation from the US Food and Drug Administration (FDA) for NPC, sIBM and ALS. 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 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.

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