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molecule","productStatus":"Approved","date":"December 2023","year":"2023","type":"Partnership","leadProduct":"Sirolimus","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Palvella Therapeutics","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Ligand Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Palvella Therapeutics \/ Ligand Pharmaceuticals"},{"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":"","productType":"Small molecule","productStatus":"Approved","date":"November 2023","year":"2023","type":"Not Applicable","leadProduct":"Sirolimus","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Palvella Therapeutics","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"Gel","sponsorNew":"Palvella Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Palvella Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Concept Medical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Concept Medical Receives FDA IDE Approval for Magictouch AVF Indication","therapeuticArea":"Nephrology","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"Approved","date":"April 2024","year":"2024","type":"Not Applicable","leadProduct":"Sirolimus","moa":"","url1":"","url2":"","graph1":"Nephrology","graph2":"IND Enabling","graph3":"Concept Medical","amount2":0,"therapeuticAreaNew":"Nephrology","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Nephrology","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Concept Medical \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Concept Medical \/ Not Applicable"},{"orgOrder":0,"company":"Concept Medical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Concept Medical Announces Enrollment of First Patient In \"MAGICAL-ISR\" IDE Study in the US","therapeuticArea":"Cardiology\/Vascular Diseases","highestDevelopmentStatus":"Approved","country":"","productType":"Small molecule","productStatus":"Approved","date":"April 2024","year":"2024","type":"Not Applicable","leadProduct":"Sirolimus","moa":"","url1":"","url2":"","graph1":"Cardiology\/Vascular Diseases","graph2":"Approved","graph3":"Concept Medical","amount2":0,"therapeuticAreaNew":"Cardiology\/Vascular Diseases","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Cardiovascular Diseases","productSubType":"Antibiotic","amount2New":0,"dosageForm":"Implant","sponsorNew":"Concept Medical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Concept Medical \/ Not Applicable"},{"orgOrder":0,"company":"Emtora Biosciences","sponsor":"Biodexa Pharmaceuticals","pharmaFlowCategory":"D","amount":"$41.5 million","upfrontCash":"Undisclosed","newsHeadline":"Biodexa Licenses eRapa\u2122 for Familial Adenomatous Polyposis Treatment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"","productType":"Small molecule","productStatus":"Approved","date":"April 2024","year":"2024","type":"Licensing Agreement","leadProduct":"Sirolimus","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Emtora Biosciences","amount2":0.040000000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Emtora Biosciences \/ Biodexa Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Emtora Biosciences \/ Biodexa Pharmaceuticals"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Ladenburg Thalmann","pharmaFlowCategory":"D","amount":"$7.0 million","upfrontCash":"Undisclosed","newsHeadline":"Biodexa Announces $7 Million of Gross Proceeds from Warrant Exercises","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"","productType":"Small molecule","productStatus":"Approved","date":"May 2024","year":"2024","type":"Private Placement","leadProduct":"Sirolimus","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0.01,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.01,"dosageForm":"","sponsorNew":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Biodexa Reports Positive Phase 2 Results of 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Find Clinical Drug Pipeline Developments & Deals for DSSTox_CID_3582

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                          01

                          Lead Product(s) : PENAO,Sirolimus

                          Therapeutic Area : Oncology

                          Study Phase : Phase I/ Phase II

                          Sponsor : PENAO Pty Ltd

                          Deal Size : Undisclosed

                          Deal Type : Agreement

                          Details : This next-generation adenine nucleotide translocase (ANT) inhibitor, PENAO, was shown to be safe and well-tolerated in a phase I clinical trial conducted in Australia where encouraging efficacy data was observed.

                          Brand Name : PENAO

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          August 18, 2021

                          Lead Product(s) : PENAO,Sirolimus

                          Therapeutic Area : Oncology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : PENAO Pty Ltd

                          Deal Size : Undisclosed

                          Deal Type : Agreement

                          Aurigene

                          02

                          AAPS 2024
                          Not Confirmed
                          AAPS 2024
                          Not Confirmed

                          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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                          03

                          AAPS 2024
                          Not Confirmed
                          AAPS 2024
                          Not Confirmed

                          Details : The proceeds will be used to fund company's upcoming Phase 3 registrational study of eRapa, a proprietary oral tablet formulation of rapamycin, in Familial Adenomatous Polyposis.

                          Brand Name : eRapa

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          September 16, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : CPRIT

                          Deal Size : $17.0 million

                          Deal Type : Funding

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                          04

                          AAPS 2024
                          Not Confirmed
                          AAPS 2024
                          Not Confirmed

                          Details : The net proceeds will be used to advance the clinical development of eRapa (rapamycin), also known as sirolimus. It is being evaluated for treating Familial Adenomatous Polyposis.

                          Brand Name : eRapa

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          July 19, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Ladenburg Thalmann & Co

                          Deal Size : $5.0 million

                          Deal Type : Public Offering

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                          05

                          AAPS 2024
                          Not Confirmed
                          AAPS 2024
                          Not Confirmed

                          Details : eRapa is a proprietary oral capsule formulation of rapamycin (mTOR inhibitor), also known as sirolimus. It is being developed for treating Familial Adenomatous Polyposis.

                          Brand Name : eRapa

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 11, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          AAPS 2024
                          Not Confirmed
                          AAPS 2024
                          Not Confirmed

                          Details : eRapa is a proprietary oral capsule formulation of rapamycin (mTOR inhibitor), also known as sirolimus. It is being developed for treating Familial Adenomatous Polyposis.

                          Brand Name : eRapa

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 24, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          AAPS 2024
                          Not Confirmed
                          AAPS 2024
                          Not Confirmed

                          Details : Agreement will focus on the development of novel topical rapamycin (sirolimus) formulations as potential treatments for a number of rare and orphan diseases.

                          Brand Name : Sirolimus

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          June 12, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : University College Cork

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          08

                          AAPS 2024
                          Not Confirmed
                          AAPS 2024
                          Not Confirmed

                          Details : The proceeds will be used to develop, manufacture, commercialize and advance the clinical potential of eRapa, an oral tablet of rapamycin and an mTOR inhibitor, for familial adenomatous polyposis.

                          Brand Name : eRapa

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          May 22, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Ladenburg Thalmann

                          Deal Size : $7.0 million

                          Deal Type : Private Placement

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                          09

                          AAPS 2024
                          Not Confirmed
                          AAPS 2024
                          Not Confirmed

                          Details : Under the agreement, Biodexa gains rights to develop and commercialize eRapa, a proprietary oral rapamycin formulation and mTOR inhibitor for the treatment of Familial Adenomatous Polyposis.

                          Brand Name : eRapa

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          April 26, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II

                          Sponsor : Biodexa Pharmaceuticals

                          Deal Size : $41.5 million

                          Deal Type : Licensing Agreement

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                          10

                          AAPS 2024
                          Not Confirmed
                          AAPS 2024
                          Not Confirmed

                          Details : Initiated pivotal clinical study to demonstrate safety and effectiveness of the MagicTouch (sirolimus) coated balloon for complex obstructive coronary disease like Coronary Artery Disease.

                          Brand Name : MagicTouch

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 19, 2024

                          Lead Product(s) : Sirolimus

                          Therapeutic Area : Cardiology/Vascular Diseases

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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