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[{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Ligand Pharmaceuticals","pharmaFlowCategory":"D","amount":"$3.0 milion","upfrontCash":"$2.0 milion","newsHeadline":"Ligand Earns $3 Million Milestone Payment from Palvella Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Petrichor","pharmaFlowCategory":"D","amount":"$37.7 million","upfrontCash":"Undisclosed","newsHeadline":"Palvella Therapeutics Announces Series D Financing of Up to $37.7 Million to Accelerate Late-Stage Development and Support Commercialization of Novel Therapies for Serious, Rare Genetic Skin Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palvella Therapeutics Announces Pipeline Update on QTORIN\u2122 3.9% Rapamycin Anhydrous Gel (QTORIN\u2122 Rapamycin) for Serious, Rare Genetic Skin Diseases with No FDA-approved Therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palvella Therapeutics Announces Positive Topline Results from Phase 2 Study of QTORIN 3.9% Rapamycin Anhydrous Gel (QTORIN rapamycin) for the Treatment of Microcystic Lymphatic Malformations, a Serious, Rare Genetic Skin Disease with No FDA-approved Thera","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2023","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palvella Therapeutics Announces Planned Pivotal Phase 3 Study Design of QTORIN\u2122 3.9% Rapamycin Anhydrous Gel (QTORIN\u2122 rapamycin) for the Treatment of Microcystic Lymphatic Malformations and Topline Results from Phase 2b CODY Study in Gorlin Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palvella Therapeutics Reports Topline Results from Pivotal Phase 3 VAPAUS Study of QTORIN\u2122 3.9% Rapamycin Anhydrous Gel (QTORIN\u2122 rapamycin) for the Treatment of Pachyonychia Congenita","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Ligand Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$5.0 million","newsHeadline":"Palvella Therapeutics and Ligand Pharmaceuticals Expand Strategic Partnership to Accelerate Phase 3 Development of QTORIN\u2122 Rapamycin for Microcystic Lymphatic Malformations and Additional High Unmet Need Clinical Indications","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"December 2023","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palvella Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to QTORIN\u2122 3.9% Rapamycin Anhydrous Gel (QTORIN\u2122rapamycin) for the Treatment of Microcystic Lymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II"}]

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            Development Status

            Details:

            The partnership aims to accelerate Phase 3 development of Qtorin™ rapamycin for the treatment of Microcystic Lymphatic Malformations (Microcystic LMs).

            Lead Product(s): Sirolimus

            Therapeutic Area: Rare Diseases and Disorders Product Name: Qtorin

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Ligand Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: $5.0 million

            Deal Type: Partnership December 01, 2023

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

            Lead Product(s): Sirolimus

            Therapeutic Area: Rare Diseases and Disorders Product Name: Qtorin

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 16, 2023

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            Qtorin (rapamycin) a topical mTOR inhibitor, which is being investigated for the treatment of microcystic lymphatic malformations and in individuals with basal cell carcinomas (BCCs) in gorlin syndrome (GS).

            Lead Product(s): Sirolimus

            Therapeutic Area: Genetic Disease Product Name: Qtorin

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 20, 2023

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            Qtorin (rapamycin), a topical mTOR inhibitor and the lead product candidate from the QTORIN™ platform, is being investigated in Phase 3 clinical trial for the treatment of Pachyonychia Congenita (PC).

            Lead Product(s): Sirolimus

            Therapeutic Area: Genetic Disease Product Name: Qtorin

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 20, 2023

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            Details:

            Qtorin™ (rapamycin) is the lead product candidate from Palvella’s QTORIN™ platform and is initially under evaluation for for the treatment of individuals with Pachyonychia Congenita and Microcystic LM, as well as for the prevention of BCCs in individuals with GS.

            Lead Product(s): Sirolimus

            Therapeutic Area: Rare Diseases and Disorders Product Name: Qtorin

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 09, 2023

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            Details:

            Qtorin™ (rapamycin) is the lead product candidate from Palvella’s QTORIN™ platform and is initially under evaluation for for the treatment of individuals with Pachyonychia Congenita and Microcystic LM, as well as for the prevention of BCCs in individuals with GS.

            Lead Product(s): Sirolimus

            Therapeutic Area: Genetic Disease Product Name: Qtorin

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 21, 2023

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            Details:

            Proceeds from the financing will be used to advance the development QTORIN rapamycin anhydrous gel for the treatment of Pachyonychia Congenita, treatment of Microcystic Lymphatic Malformations, and for the prevention of Basal Cell Carcinomas in Gorlin Syndrome.

            Lead Product(s): Sirolimus

            Therapeutic Area: Genetic Disease Product Name: QTORIN

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Petrichor

            Deal Size: $37.7 million Upfront Cash: Undisclosed

            Deal Type: Series D Financing January 05, 2023

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            Details:

            The milestone payment comes as a result of a development funding and royalties agreement to advance their lead product candidate PTX-022 (QTORIN™ 3.9% rapamycin anhydrous gel) for the treatment of pachyonychia congenita.

            Lead Product(s): Sirolimus

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Recipient: Ligand Pharmaceuticals

            Deal Size: $3.0 milion Upfront Cash: $2.0 milion

            Deal Type: Agreement May 28, 2020

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