[{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Baclofen","moa":"GABA B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Not Applicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"FRANCE","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Baclofen","moa":"GABA B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Not Applicable"},{"orgOrder":0,"company":"Charcot Marie Tooth Association","sponsor":"Pharnext","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Collaboration","leadProduct":"Baclofen","moa":"PMP22 gene overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Charcot Marie Tooth Association","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Charcot Marie Tooth Association \/ Pharnext","highestDevelopmentStatusID":"10","companyTruncated":"Charcot Marie Tooth Association \/ Pharnext"},{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Baclofen","moa":"PMP22 gene overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Not Applicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Alpha Blue Ocean","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2021","type":"Financing","leadProduct":"Baclofen","moa":"PMP22 gene overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Alpha Blue Ocean","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Alpha Blue Ocean"},{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Baclofen","moa":"PMP22 gene overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Not Applicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Baclofen","moa":"PMP22 gene overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Not Applicable"},{"orgOrder":0,"company":"Amneal Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Baclofen","moa":"PMP22 gene overexpression","graph1":"Musculoskeletal","graph2":"Approved","graph3":"Amneal Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Granule","sponsorNew":"Amneal Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amneal Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Baclofen","moa":"PMP22 gene overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Not Applicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Baclofen","moa":"PMP22 gene overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Not Applicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Neovacs","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2022","type":"Financing","leadProduct":"Baclofen","moa":"GABA B receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Neovacs","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Neovacs"}]

Find Clinical Drug Pipeline Developments & Deals for Sorbitol API

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01

SCOPE Summit
Not Confirmed
SCOPE Summit
Not Confirmed

Details : The financing by Neovacs will support Pharnext operations and cash requirements, in particular the Phase III PREMIER pivotal study assessing its PTX3003 (baclofen) candidate in CMT1A, a rare debilitating peripheral neuropathy with currently no existing a...

Brand Name : PTX3003

Molecule Type : Small molecule

Upfront Cash : Undisclosed

October 03, 2022

Lead Product(s) : Baclofen,Naltrexone,Sorbitol API

Therapeutic Area : Genetic Disease

Highest Development Status : Phase III

Sponsor : Neovacs

Deal Size : $20.0 million

Deal Type : Financing

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02

SCOPE Summit
Not Confirmed
SCOPE Summit
Not Confirmed

Details : PXT3003 (baclofen) completed an international Phase III trial with positive topline results for the treatment of CharcotMarie-Tooth disease type 1A (‘CMT1A’) and benefits from orphan drug status in Europe and the United States.

Brand Name : PXT3003

Molecule Type : Small molecule

Upfront Cash : Not Applicable

September 13, 2022

Lead Product(s) : Baclofen,Naltrexone Hydrochloride,Sorbitol API

Therapeutic Area : Genetic Disease

Highest Development Status : Phase III

Sponsor : Not Applicable

Deal Size : Not Applicable

Deal Type : Not Applicable

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03

SCOPE Summit
Not Confirmed
SCOPE Summit
Not Confirmed

Details : PXT3003 (baclofen) is a novel fixed-dose synergistic combination of baclofen, naltrexone and sorbitol formulated as an oral solution given twice a day for the treatment of Charcot-Marie-Tooth disease type 1A.

Brand Name : PXT3003

Molecule Type : Small molecule

Upfront Cash : Not Applicable

September 12, 2022

Lead Product(s) : Baclofen,Naltrexone Hydrochloride,Sorbitol API

Therapeutic Area : Genetic Disease

Highest Development Status : Phase III

Sponsor : Not Applicable

Deal Size : Not Applicable

Deal Type : Not Applicable

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04

SCOPE Summit
Not Confirmed
SCOPE Summit
Not Confirmed

Details : LYVISPAH is bioequivalent to oral baclofen tablets and provides flexible, a skeletal muscle relaxant, is well established for the treatment of spasticity and spinal cord injuries.

Brand Name : Lyvispah

Molecule Type : Small molecule

Upfront Cash : Not Applicable

June 01, 2022

Lead Product(s) : Baclofen,Naltrexone Hydrochloride,Sorbitol API

Therapeutic Area : Musculoskeletal

Highest Development Status : Approved

Sponsor : Not Applicable

Deal Size : Not Applicable

Deal Type : Not Applicable

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05

SCOPE Summit
Not Confirmed
SCOPE Summit
Not Confirmed

Details : New data from the ongoing Open-Label Phase III Extension Study of PXT3003 (baclofen), the PLEO-CMT-FU trial, suggest good safety profile and continuous treatment effect of PXT3003 measured on the Overall Neuropathy Limitation Scale after 5 years of total...

Brand Name : PXT3003

Molecule Type : Small molecule

Upfront Cash : Not Applicable

May 16, 2022

Lead Product(s) : Baclofen,Naltrexone Hydrochloride,Sorbitol API

Therapeutic Area : Genetic Disease

Highest Development Status : Phase III

Sponsor : Not Applicable

Deal Size : Not Applicable

Deal Type : Not Applicable

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06

SCOPE Summit
Not Confirmed
SCOPE Summit
Not Confirmed

Details : PXT3003 is a novel fixed-dose synergistic combination of baclofen, naltrexone and sorbitol formulated as an oral solution given twice a day used in Charcot-Marie-Tooth Disease Type 1A.

Brand Name : PXT3003

Molecule Type : Small molecule

Upfront Cash : Not Applicable

October 18, 2021

Lead Product(s) : Baclofen,Naltrexone Hydrochloride,Sorbitol API

Therapeutic Area : Genetic Disease

Highest Development Status : Phase III

Sponsor : Not Applicable

Deal Size : Not Applicable

Deal Type : Not Applicable

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07

SCOPE Summit
Not Confirmed
SCOPE Summit
Not Confirmed

Details : This financing agreement for up to 81 million euros will enable Pharnext to strengthen its cash position and continue to develop its pivotal Phase III clinical trial of PXT3003, the PREMIER trial, in Charcot-Marie-Tooth disease type 1A (CMT1A).

Brand Name : PXT3003

Molecule Type : Small molecule

Upfront Cash : Undisclosed

June 10, 2021

Lead Product(s) : Baclofen,Naltrexone,Sorbitol API

Therapeutic Area : Genetic Disease

Highest Development Status : Phase III

Sponsor : Alpha Blue Ocean

Deal Size : $96.0 million

Deal Type : Financing

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08

SCOPE Summit
Not Confirmed
SCOPE Summit
Not Confirmed

Details : The new results announced today continue to show sustained treatment benefits for CMT1A patients treated with PXT3003 at High Dose (‘HD’) in the PLEO-CMT-FU Period 2 trial with a data readout at 54 months of total trial time (double-blind + open-labe...

Brand Name : PXT3003

Molecule Type : Small molecule

Upfront Cash : Not Applicable

April 28, 2021

Lead Product(s) : Baclofen,Naltrexone,Sorbitol API

Therapeutic Area : Genetic Disease

Highest Development Status : Phase III

Sponsor : Not Applicable

Deal Size : Not Applicable

Deal Type : Not Applicable

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09

SCOPE Summit
Not Confirmed
SCOPE Summit
Not Confirmed

Details : The primary objective of this collaboration is to identify and validate potential treatment responsive CMT1A biomarkers that could be further explored in future clinical studies, in particular the upcoming Phase III study of PXT3003, Pharnext’s lead dr...

Brand Name : PXT3003

Molecule Type : Small molecule

Upfront Cash : Undisclosed

September 03, 2020

Lead Product(s) : Baclofen,Naltrexone,Sorbitol API

Therapeutic Area : Genetic Disease

Highest Development Status : Phase III

Sponsor : Pharnext

Deal Size : Undisclosed

Deal Type : Collaboration

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10

SCOPE Summit
Not Confirmed
SCOPE Summit
Not Confirmed

Details : PXT3003 is a novel drug candidate for the treatment of CMT1A and has been granted both Orphan Drug Designation and Fast Track Designation by the US Food and Drug Administration.

Brand Name : Undisclosed

Molecule Type : Small molecule

Upfront Cash : Not Applicable

June 10, 2020

Lead Product(s) : Baclofen,Naltrexone Hydrochloride,Sorbitol API

Therapeutic Area : Neurology

Highest Development Status : Phase III

Sponsor : Not Applicable

Deal Size : Not Applicable

Deal Type : Not Applicable

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