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Iron Deficiency Anemia in Patients Receiving Home Infusion","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Rockwell Medical"},{"company":"BioCryst Pharmaceuticals","sponsor":"OMERS Capital Market","pharmaFlowCategory":"D","amount":"$350.0 million","upfrontCash":"Undisclosed","newsHeadline":"BioCryst Adds $350 Million in New Financing with Royalty Pharma and\u00a0OMERS Capital Markets","therapeuticArea":"Hematology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst 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Details:
BCX9930, significantly increased hemoglobin and reduced transfusions in an ongoing dose-ranging trial in treatment-naïve paroxysmal nocturnal hemoglobinuria patients, and in PNH patients with an inadequate response to C5 inhibitors.
Lead Product(s):
BCX9930
Therapeutic Area: Hematology
Product Name: BCX9930
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
April 08, 2022
Details:
Study results demonstrate that nafamostat had similar efficacy as citrate, while providing lower toxicity and lower costs to the hospital over standard citrate anticoagulation.
Lead Product(s):
Nafamostat
Therapeutic Area: Hematology
Product Name: Niyad
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 29, 2022
Details:
First patient is enrolled for REDEEM-1 Pivotal Trial of BCX9930 on the basis of results from the previous trial which was safe and generally well-tolerated and, showed increased hemoglobin from baseline with 80 percent of patients being transfusion-free over the same period.
Lead Product(s):
BCX9930
Therapeutic Area: Hematology
Product Name: BCX9930
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
January 07, 2022
Details:
Company received feedback from USFDA regarding IND of Triferic (Ferric Pyrophosphate Citrate), designed for the treatment of iron deficiency anemia and maintenance of hemoglobin in patients receiving infusion therapy in the home setting.
Lead Product(s):
Ferric Pyrophosphate Citrate
Therapeutic Area: Hematology
Product Name: Triferic
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
December 14, 2021
Details:
Company enrolled first patient in trial of BCX9930, an oral Factor D inhibitor showed primary endpoint change from baseline in paroxysmal nocturnal hemoglobinuria, as assessed at week 12.
Lead Product(s):
BCX9930
Therapeutic Area: Hematology
Product Name: BCX9930
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 29, 2021
Details:
The funds from these transactions will enable further advancement of BCX9930, BioCryst’s oral Factor D inhibitor, toward filing for registration, expand the development of BCX9930 across multiple indications.
Lead Product(s):
BCX9930
Therapeutic Area: Hematology
Product Name: BCX9930
Highest Development Status: Phase II
Product Type: Small molecule
Partner/Sponsor/Collaborator:
OMERS Capital Market
Deal Size: $350.0 million
Upfront Cash: Undisclosed
Deal Type: Financing
November 22, 2021
Details:
Rockwell Medical is advancing its FPC platform by developing FPC for the treatment of iron deficiency anemia in patients outside of dialysis, who are receiving intravenous medications in the home infusion setting.
Lead Product(s):
Ferric Pyrophosphate Citrate
Therapeutic Area: Hematology
Product Name: Triferic
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 11, 2021
Details:
Ciraparantag is a novel small, water-soluble molecule being investigated for reversal of anticoagulation induced by direct oral anticoagulants (DOACs) or low molecular weight heparin (LMWH).
Lead Product(s):
Ciraparantag
Therapeutic Area: Hematology
Product Name: PER977
Highest Development Status: Phase II
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Norgine
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
October 04, 2021
Details:
Protagonist's rusfertide clinical trials have been placed on clinical hold by the U.S. Food and Drug Administration (FDA). Protagonist is working closely with the FDA to determine next steps for the rusfertide development program.
Lead Product(s):
Rusfertide
Therapeutic Area: Hematology
Product Name: PTG-300
Highest Development Status: Phase II
Product Type: Peptide
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
October 04, 2021
Details:
DisperSol’s active programs include, DST-0509 about to enter Phase 3 for iron overload disorder, DST-2970 in Phase 2 for refractory metastatic prostate cancer and DST-8294 for treatment of clotting disorders.
Lead Product(s):
Deferasirox
Therapeutic Area: Hematology
Product Name: DST-0509
Highest Development Status: Phase II
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Catalent Pharma Solutions
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Collaboration
August 02, 2021
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