ORPH Orphazyme A/S

5.8
+0.29  (+5%)
Previous Close 5.51
Open 5.9
52 Week Low 5.4088
52 Week High 14.78
Market Cap $202,722,998
Shares 34,952,241
Float 34,952,241
Enterprise Value $94,252,748
Volume 1,791
Av. Daily Volume 71,031
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Upcoming Catalysts

Drug Stage Catalyst Date
Arimoclomol
Niemann-Pick disease Type C (NPC)
PDUFA priority review
PDUFA priority review
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Drug Pipeline

Drug Stage Notes
Arimoclomol
Amyotrophic Lateral Sclerosis
Phase 3
Phase 3
Phase 3 data trial did not meet primary endpoint - May 7, 2021.
Arimoclomol
Sporadic Inclusion Body Myositis
Phase 2/3
Phase 2/3
Phase 2/3 trial did not meet primary endpoint - March 29, 2021.

Latest News

  1. 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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  2. 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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  3. 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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  4. 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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  5. 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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