International Niemann–Pick Disease Alliance

Updates

  1. Orphazyme announces U.S. FDA acceptance and Priority Review of New Drug Application for arimoclomol for Niemann-Pick disease Type C

     If approved, arimoclomol would become the first approved therapy in the U.S. for people with Niemann-Pick disease Type C (NPC) FDA has set target PDUFA action date of March 17, 2021 Copenhagen, Denmark, September 16, 2020 – Orphazyme A/S (ORPHA.CO), a...

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  2. Cyclo Therapeutics Inc. Announces Additional Efficacy Data from its Ongoing Phase I/II Trial using Trappsol® CycloTM Intravenously to Treat Patients with Niemann-Pick Disease Type C1 (NPC1)

    GAINESVILLE, FL – (BusinessWire) – September 8, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, today announced that 3...

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  3. Cyclo Therapeutics Closes $2.8 Million Private Placement Led by Novit LP with $1.0 Million

    GAINESVILLE, FL – (BusinessWire) – August 27, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, today announced that it...

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  4. Orphazyme completes rolling submission of New Drug Application to U.S. FDA for arimoclomol for Niemann-Pick disease Type C

    Orphazyme A/S Investor News                                                                       ...

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  5. Pfrieger’s Digest – 2nd Issue

    “Pfrieger’s Digest” is written by Frank Pfrieger of Niemann-Pick Selbsthiifegruppe (Germany) to provide an overview of the latest advances in Niemann-Pick disease based on recent scientific publications. Frank Pfrieger is group leader at the Institute of Cellular and Integrative Neurosciences in...

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  6. Cyclo Therapeutics’ Clinical Data Webinar Recording Available on Company Website

    GAINESVILLE, FL – (BusinessWire) – May 26, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, issued a press release...

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  7. Cyclo Therapeutics Inc. Announces Positive Top Line Results from Phase I Trial and Interim Analysis of Phase I/II Trial using Trappsol® Cyclo™ Intravenously to Treat Patients with Niemann-Pick Disease Type C1 (NPC1)

    Top line results of Phase I study confirm favorable safety and tolerability of Trappsol® Cyclo™ in patients with NPC1 and demonstrate biological responses for both dose groups. An Unblinded Interim Analysis of Phase I/II study shows encouraging signals in...

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  8. Cyclo Therapeutics to Hold Call for Patients and Investors Wednesday, May 20 at 4:30 pm EDT

    GAINESVILLE, FL – (BusinessWire) – May 19, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, today announced that it...

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  9. Cyclo Therapeutics Announces Positive Feedback from European Medicines Agency on its Pivotal Trial Design for Trappsol® Cyclo™ for Niemann-Pick Disease Type C1

    GAINESVILLE, FL – (Businesswire) – May 18, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical-stage biotechnology company that develops cyclodextrinbased products for the treatment of Niemann-Pick Disease Type C1 (NPC1) and Alzheimer’s Disease, today announced that it received...

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  10. Cyclo Therapeutics Closes $2.0 Million Private Placement

    ALACHUA, FL – (BusinessWire) – April 27, 2020 – Cyclo Therapeutics, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, today announced that it...

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