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[{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst Begins Proof Of Concept Trial In PNH Patients With Oral Factor D Inhibitor, BCX9930","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Pluri","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pluristem Announces End of Patient Enrollment in its Phase I Hematology Study","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"ISRAEL","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Pluri"},{"orgOrder":0,"company":"eXIthera Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"eXIthera Announces Publication of EP-7041 Data Demonstrating Safe Antithrombotic Activity in ECMO","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"eXIthera Pharmaceutical"},{"orgOrder":0,"company":"Omeros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Omeros\u2019 OMS906 Data Presented at Complement-based Drug Development Summit","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Omeros"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First Subject Dosed in CANbridge Pharmaceuticals CAN106 Trial for Treatment of Complement Dysregulation Diseases in Singapore","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Veralox Therapeutics","sponsor":"Hatteras Venture","pharmaFlowCategory":"D","amount":"$16.6 million","upfrontCash":"Undisclosed","newsHeadline":"Veralox Therapeutics Announces Closing Of $16.6 Million Series A Financing And Announces New Board Members","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Veralox Therapeutics"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge Pharmaceuticals CAN106 Investigational New Drug Application Approved in China for Paroxysmal Nocturnal Hemoglobinuria (PNH)","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Disc Medicine Initiates Phase 1 Clinical Study of DISC-0974","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Disc medicine"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zydus Cadila Seeks DCGI Approval for Novel Oral Pill to Treat Anaemia in Kidney Patients","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"INDIA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Zydus Lifesciences"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NovelMed's Complement Alternative Pathway Specific Anti-Bb Antibody (NM8074) for Rare Diseases Achieves a Major Milestone","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"NovelMed Therapeutics"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge Reports Positive Top-Line CAN106 Phase 1 Data Receives Approval to Treat Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in Phase 1b\/2 Trial in China Results Suggest Complete Blockade of Complement Function CAN106 Safe and Well-Tolerated","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Disc Medicine Announces Presentation of Clinical Data from First-in-Human Phase 1 Study of DISC-0974 at European Hematology Association (EHA) 2022 Congress","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Disc medicine"},{"orgOrder":0,"company":"Keros Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Keros Therapeutics Announces Preliminary Topline Results from its Ongoing Phase 1 Clinical Trial Evaluating KER-012 in Healthy Volunteers","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Keros Therapeutics"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge to Present CAN106 Phase 1 Data at the European Hematology Association 2022 Congress","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Disc Medicine Presents Positive Results from Phase 1 Clinical Study of DISC-0974 in Healthy Volunteers at the 2022 EHA Annual Congress","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Disc medicine"},{"orgOrder":0,"company":"Clover Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Clover Doses First Patient with SCB-219M in Phase 1 Trial for Chemotherapy-Induced Thrombocytopenia","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Clover Biopharmaceuticals"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"X4 Pharmaceuticals Announces New Positive Phase 1b Data Supporting Mavorixafor\u2019s Broad Potential in Chronic Neutropenia (CN)","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"X4 Pharmaceuticals"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
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Results of the Phase 1b clinical trial suggested that X4P-001 (mavorixafor) could be the first oral treatment for CN disorders and has the potential to reduce or replace injectable G-CSF, the current standard of care.
Lead Product(s):
Mavorixafor
Therapeutic Area: Hematology Product Name: X4P-001
Highest Development Status: Phase I Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableSeptember 27, 2022
Details:
In pre-clinical studies, SCB-219M has demonstrated an extended serum half-life and favorable pharmacokinetics /pharmacodynamics (PK/PD) profile that supports weekly dosing possibility of the drug.
Lead Product(s):
SCB-219M
Therapeutic Area: Hematology Product Name: SCB-219M
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableJune 14, 2022
Details:
Data demonstrated for DISC-0974 robust increases in serum iron and improvements in markers of erythropoiesis including statistically significant increases of hemoglobin at the highest dose.
Lead Product(s):
DISC-0974
Therapeutic Area: Hematology Product Name: DISC-0974
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableJune 10, 2022
Details:
KER-012 is designed to bind to and inhibit the signaling of TGF-ß ligands that suppress bone growth, including activin A and activin B. KER-012 is being developed for the treatment of PAH and for the treatment of disorders associated with bone loss.
Lead Product(s):
KER-012
Therapeutic Area: Hematology Product Name: KER-012
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableMay 18, 2022
Details:
Poster to highlight data on safety, tolerability, pharmacokinetics, and effects on hepcidin and measures of iron metabolism of DISC-0974, designed to suppress hepcidin production by inhibiting the hemojuvelin from phase 1 study in healthy volunteers.
Lead Product(s):
DISC-0974
Therapeutic Area: Hematology Product Name: DISC-0974
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableMay 16, 2022
Details:
CAN106 is a long-acting, recombinant human mAB that specifically binds to and neutralizes C5 in the complement system, C5 is a validated mechanism of action in numerous hematology, nephrology, and neurology diseases, for treatment of multiple complement-mediated disorders.
Lead Product(s):
CAN106
Therapeutic Area: Hematology Product Name: CAN106
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableMay 12, 2022
Details:
CAN106 is a novel, long-acting recombinant human monoclonal antibody. CAN106 has demonstrated a favorable PK/PD profile, safety, and tolerability, indicating that CAN106 has the potential to effectively inhibit C5 in patients with certain complement-mediated diseases.
Lead Product(s):
CAN106
Therapeutic Area: Hematology Product Name: CAN106
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableFebruary 07, 2022
Details:
NM8074 is expected to be therapeutically effective across a broad range of rare and common complement-mediated diseases, including hemolytic disorders, renal disorders, ocular disorders, neurological disorders, and inflammatory disorders.
Lead Product(s):
NM8074
Therapeutic Area: Hematology Product Name: NM8074
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableJanuary 31, 2022
Details:
The study demonstrated the noninferiority of Desidustat compared to Epoetin in the treatment of anemia in patients with CKD who were on dialysis. Desidustat is also under clinical trials for Cancer Chemotherapy Induced Anemia (CIA).
Lead Product(s):
Desidustat
Therapeutic Area: Hematology Product Name: Undisclosed
Highest Development Status: Phase I Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableNovember 23, 2021
Details:
This Phase 1 study will be a single-ascending dose design in healthy volunteers to assess the safety and tolerability of DISC-0974 as well as to investigate the effects of inhibiting HJV on hepcidin and iron parameters.
Lead Product(s):
DISC-0974
Therapeutic Area: Hematology Product Name: DISC-0974
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not ApplicableUpfront Cash: Not Applicable
Deal Type: Not ApplicableJuly 28, 2021
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