X
[{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Announces Achievement of Target Patient Dosing in HOPE-B Pivotal Trial of AMT-061 in Hemophilia B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$450.0 million","newsHeadline":"CSL Behring Agrees to Acquire Novel Late-Stage Gene Therapy Candidate for Hemophilia B Patients from uniQure","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Announces Clinical Data Presentations at the 62nd American Society of Hematology (ASH) Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Presents Late-Breaking Data from the HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"D","amount":"$2,050.0 million","upfrontCash":"$450.0 million","newsHeadline":"UniQure Announces Closing of Commercialization and License Agreement With CSL Behring","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Announces First Patient Enrolled in Pediatric Clinical Trial of Leniolisib","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharming"},{"orgOrder":0,"company":"NewAmsterdam Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NewAmsterdam Pharma Doses First Patient in Phase 3 BROOKLYN Clinical Trial Evaluating Obicetrapib in Patients with Heterozygous Familial Hypercholesterolemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"NewAmsterdam Pharma"},{"orgOrder":0,"company":"NewAmsterdam Pharma","sponsor":"Frazier Lifesciences Acquisition Corporation","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NewAmsterdam Pharma Showcases Leadership in Cardiometabolic Disease Treatment in Multiple Presentations at ESC Congress 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"NewAmsterdam Pharma"},{"orgOrder":0,"company":"NewAmsterdam Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NewAmsterdam Pharma Completes Enrollment in Pivotal Phase 3 BROOKLYN Clinical Trial Evaluating Obicetrapib in Patients with Heterozygous Familial Hypercholesterolemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"NewAmsterdam Pharma"},{"orgOrder":0,"company":"NewAmsterdam Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NewAmsterdam Pharma Completes Enrollment in Pivotal Phase 3 BROADWAY Clinical Trial Evaluating Obicetrapib in Patients with Heterozygous Familial Hypercholesterolemia and\/or Established Atherosclerotic Cardiovascular Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"NewAmsterdam Pharma"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
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Details:
TA-8995 (obicetrapib) is a next-generation, non statin, oral once-daily, low-dose CETP inhibitor having the potential for lowering LDL-C. It is currently being investigated in combination with ezetimibe as an adjunct to high-intensity statin therapy.
Lead Product(s):
Obicetrapib
Therapeutic Area: Genetic Disease Product Name: TA-8995
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableJuly 25, 2023
Details:
TA-8995 (obicetrapib) is a next-generation, oral, low-dose CETP inhibitor that NewAmsterdam is developing to potentially overcome the limitations of current LDL-lowering treatments.
Lead Product(s):
Obicetrapib
Therapeutic Area: Genetic Disease Product Name: TA-8995
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableApril 24, 2023
Details:
Leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor. It is being investigated in children with activated phosphoinositide 3-kinase delta syndrome (APDS).
Lead Product(s):
Leniolisib
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableFebruary 21, 2023
Details:
Emerging data reinforce combination lipid-lowering therapy as first-line strategy for very high-risk patients, supporting the development of TA-8995 (obicetrapib) as adjunct to high-intensity statins.
Lead Product(s):
Obicetrapib
Therapeutic Area: Genetic Disease Product Name: TA-8995
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator:
Frazier Lifesciences Acquisition Corporation
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableAugust 29, 2022
Details:
BROOKLYN evaluates TA-8995 (obicetrapib) as an adjunct to diet and maximally tolerated lipid lowering therapy in patients requiring additional lowering of low-density lipoprotein cholesterol (LDL-C).
Lead Product(s):
Obicetrapib
Therapeutic Area: Genetic Disease Product Name: TA-8995
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableJuly 28, 2022
Details:
Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable FIX expression, assessed at 18 months following a single dose of AMT-061(etranacogene dezaparvovec).
Lead Product(s):
Etranacogene Dezaparvovec
Therapeutic Area: Genetic Disease Product Name: AMT-061
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
CSL Behring
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableDecember 09, 2021
Details:
uniQure announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec expired on May 5, 2021, and the agreement became fully effective on May 6, 2021.
Lead Product(s):
Etranacogene dezaparvovec
Therapeutic Area: Genetic Disease Product Name: AMT-061
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
CSL Behring
Deal Size: $2,050.0 millionUpfront Cash: $450.0 million
Deal Type: Licensing AgreementMay 06, 2021
Details:
UniQure's comprehensive investigation showed that AMT-061 (etranacogene dezaparvovec) is very unlikely to have contributed to the HCC in our patient. The company is looking forward to announcing top-line 52-week data from the HOPE-B pivotal trial later this quarter.
Lead Product(s):
Etranacogene dezaparvovec
Therapeutic Area: Genetic Disease Product Name: AMT-061
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableApril 26, 2021
Details:
The number of bleeds requiring treatment decreased by 91 percent after the one-time administration of etranacogene dezaparvovec, with 87 percent of patients reporting no such bleeds after dosing.
Lead Product(s):
Etranacogene dezaparvovec
Therapeutic Area: Genetic Disease Product Name: AMT-061
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableDecember 08, 2020
Details:
Company to present long-term follow-up data from its hemophilia B gene therapy studies, including two years of follow-up on the Phase IIb clinical trial of etranacogene dezaparvovec (AMT-061) and up to five years of follow-up from the Phase I/II clinical trial of AMT-060.
Lead Product(s):
Etranacogene dezaparvovec
Therapeutic Area: Genetic Disease Product Name: AMT-061
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableNovember 04, 2020
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