ORPH Orphazyme A/S

12.24
-0.17  -1%
Previous Close 12.41
Open 11.86
52 Week Low 8.89
52 Week High 13.98
Market Cap $426,782,288
Shares 34,867,834
Float 34,867,834
Enterprise Value $346,863,735
Volume 7,039
Av. Daily Volume 10,928
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Latest News

  1. 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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  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 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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  3. 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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  4. 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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  5. 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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