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Therapeutics"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Blarcamesine","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Anavex 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                            Neurotech Pharmaceuticals

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                            Lead Product(s) : Revakinagene Taroretcel

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Neurotech Provides Update on BLA for NT-501 in Macular Telangiectasia

                            Details : NT-501 (revakinagene taroretcel) leverages is a gene therapy which delivers CNTF through an ocular implant, it is designed for the treatment of chronic retinal diseases, such as MacTel.

                            Brand Name : NT-501

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            November 08, 2024

                            Lead Product(s) : Revakinagene Taroretcel

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

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                            Palvella Therapeutics

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                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

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                            Palvella Therapeutics Doses First Patient in SELVA Trial for Lymphatic Malformations

                            Details : Qtorin (rapamycin) is a macrocyclic lactone antibiotic produced by bacteria Streptomyces hygroscopicus. It is being evaluated for the treatment of microcystic lymphatic malformations.

                            Brand Name : Qtorin

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            November 07, 2024

                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

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                            Palvella Therapeutics

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                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : FDA

                            Deal Size : $2.6 million

                            Deal Type : Funding

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                            Palvella Wins $2.6M FDA Grant for Phase 3 Trial in Microcystic Lymphatic Malformations

                            Details : The proceeds will support the ongoing Phase 3 of QTORIN rapamycin anhydrous gel for the treatment of microcystic lymphatic malformations (microcystic LMs).

                            Brand Name : Qtorin

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            October 03, 2024

                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : FDA

                            Deal Size : $2.6 million

                            Deal Type : Funding

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                            04

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Zilganersen

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Zilganersen Fast-Tracked by FDA for Alexander Disease Treatment

                            Details : ION373 (zilganersen) is an investigational antisense oligonucleotide medicine being developed as a potential treatment for people with genetically confirmed Alexander disease.

                            Brand Name : ION373

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            October 01, 2024

                            Lead Product(s) : Zilganersen

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            05

                            Savara

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Savara

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Molgramostim

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Savara Launches Expanded Access Program For Molgramostim in aPAP Patients

                            Details : Molgramostim is an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis.

                            Brand Name : Undisclosed

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            September 27, 2024

                            Lead Product(s) : Molgramostim

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            06

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Ionis Pharmaceuticals

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Zilganersen

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Morgan Stanley

                            Deal Size : $550.3 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Ionis announces pricing of $500.3 million public offering

                            Details : The net proceeds will used to fund the commercial launch company's lead product of ION373 (zilganersen), which is a late-stage clinical program to treat Alexander's disease.

                            Brand Name : ION373

                            Molecule Type : Large molecule

                            Upfront Cash : Undisclosed

                            September 09, 2024

                            Lead Product(s) : Zilganersen

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Morgan Stanley

                            Deal Size : $550.3 million

                            Deal Type : Public Offering

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                            07

                            Rafael Holdings

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Rafael Holdings

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Hydroxypropyl-Beta Cyclodextrin

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Cyclo Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Merger

                            DEALS_DEV arrow-down

                            Rafael Holdings and Cyclo Therapeutics Enter Into a Definitive Merger Agreement

                            Details : The combined company will focus on the development of Trappsol Cyclo (hydroxypropyl-beta cyclodextrin) for the treatment of Niemann-Pick Disease Type C1.

                            Brand Name : Trappsol Cyclo

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            August 22, 2024

                            Lead Product(s) : Hydroxypropyl-Beta Cyclodextrin

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Cyclo Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Merger

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                            08

                            Apellis Pharmaceuticals

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Apellis Pharmaceuticals

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Pegcetacoplan

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Apellis and Sobi Announce Positive Results from Phase 3 Pegcetacoplan Trial in C3G

                            Details : Syfovre (pegcetacoplan injection) targets C3, being investigated for C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis.

                            Brand Name : Syfovre

                            Molecule Type : Peptide

                            Upfront Cash : Not Applicable

                            August 08, 2024

                            Lead Product(s) : Pegcetacoplan

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            09

                            Savara

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Savara

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Molgramostim

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Jefferies

                            Deal Size : $100.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Savara Announces Pricing of $100.0 Million Underwritten Offering of Common Stock

                            Details : The net proceeds will be used to fund the clinical development and approval of molgramostim, which is being evaluated in the treatment of autoimmune pulmonary alveolar proteinosis.

                            Brand Name : Molgradex

                            Molecule Type : Large molecule

                            Upfront Cash : Undisclosed

                            June 28, 2024

                            Lead Product(s) : Molgramostim

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Jefferies

                            Deal Size : $100.0 million

                            Deal Type : Public Offering

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                            10

                            Mirum Pharmaceuticals

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Mirum Pharmaceuticals

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Chenodeoxycholic Acid

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Mirum Submits New Drug Application to FDA for Chenodiol for the Treatment of CTX

                            Details : Chenodal (chenodeoxycholic acid) is an oral cholesterol synthesis inhibitor which is being evaluated for the treatment of cerebrotendinous xanthomatosis.

                            Brand Name : Chenodal

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            June 28, 2024

                            Lead Product(s) : Chenodeoxycholic Acid

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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