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Pharma","pharmaFlowCategory":"D","therapeuticArea":"Hematology","country":"IRELAND","productType":"Other Small Molecule","year":"2025","type":"Financing","leadProduct":"Iron Hydroxide Adipate Tartrate","moa":"Ferritin","graph1":"Hematology","graph2":"Phase III","graph3":"Nemysis","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Hematology","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Nemysis \/ Avia Pharma","highestDevelopmentStatusID":"10","companyTruncated":"Nemysis \/ Avia Pharma"}]

Find Hematology Drugs in Phase III Clinical Development

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Hematology

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

                          Product Name : SAR439774

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          April 04, 2023

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Hematology

                          Study Phase : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

                          Product Name : SAR439774

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          July 10, 2022

                          Lead Product(s) : Fitusiran

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : Givosiran Sodium

                          Therapeutic Area : Hematology

                          Study Phase : Phase III

                          Sponsor : Alnylam Pharmaceuticals

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Data from phase 3 studies achieved primary and secondary endpoints for ALN-AT3SC (Fitusiran) prophylaxis demonstrated significant and clinically meaningful improvements in bleed protection across all study populations.

                          Product Name : ALN-AT3SC

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          December 14, 2021

                          Lead Product(s) : Givosiran Sodium

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase III

                          Sponsor : Alnylam Pharmaceuticals

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          04

                          Lead Product(s) : Givosiran Sodium

                          Therapeutic Area : Hematology

                          Study Phase : Phase III

                          Sponsor : Alnylam Pharmaceuticals

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Sanofi will resume fitusiran dosing in ongoing U.S. adolescent and adult clinical studies. Fitusiran is an investigational, small interference RNA therapy in development for the treatment of people with hemophilia A or B, with or without inhibitors.

                          Product Name : ALN-AT3SC

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          December 10, 2020

                          Lead Product(s) : Givosiran Sodium

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase III

                          Sponsor : Alnylam Pharmaceuticals

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          05

                          Lead Product(s) : Givosiran Sodium

                          Therapeutic Area : Hematology

                          Study Phase : Phase III

                          Sponsor : Alnylam Pharmaceuticals

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : The WFH, EHC and NHF have learned of and subsequently confirmed a decision by Sanofi Genzyme to initiate a voluntary sponsor-led global dosing hold on its full clinical development program for fitusiran due to the identification of new adverse events.

                          Product Name : ALN-AT3SC

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          November 05, 2020

                          Lead Product(s) : Givosiran Sodium

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase III

                          Sponsor : Alnylam Pharmaceuticals

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          06

                          Lead Product(s) : Givosiran Sodium

                          Therapeutic Area : Hematology

                          Study Phase : Phase III

                          Sponsor : Alnylam Pharmaceuticals

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Long-term interim results from the Phase 2 open-label extension (OLE) study reinforce fitusiran’s potential to restore hemostatic balance and to lower annualized bleed rates (ABRs) over a period up to 57 months.

                          Product Name : ALN-AT3SC

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          June 19, 2020

                          Lead Product(s) : Givosiran Sodium

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase III

                          Sponsor : Alnylam Pharmaceuticals

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          07

                          Immuno-Oncology 360
                          Not Confirmed
                          Immuno-Oncology 360
                          Not Confirmed

                          Details : The financing aims to fund the clinical development of IHAT (iron hydroxide adipate tartrate), which is being evaluated for the treatment of iron deficiency.

                          Product Name : IHAT

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          March 18, 2025

                          Lead Product(s) : Iron Hydroxide Adipate Tartrate

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase III

                          Sponsor : Avia Pharma

                          Deal Size : Undisclosed

                          Deal Type : Financing

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                          08

                          Immuno-Oncology 360
                          Not Confirmed
                          Immuno-Oncology 360
                          Not Confirmed

                          Details : Anktiva and CAR-NK combination is being evaluated for the reversal of Lymphopenia in Relapsed Pancreatic Cancer.

                          Product Name : Anktiva

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          February 27, 2025

                          Lead Product(s) : Nogapendekin Alfa Inbakicept,CAR-NK

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Immuno-Oncology 360
                          Not Confirmed
                          Immuno-Oncology 360
                          Not Confirmed

                          Details : Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is being developed for adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.

                          Product Name : Pyrukynd

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 08, 2025

                          Lead Product(s) : Mitapivat

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Immuno-Oncology 360
                          Not Confirmed
                          Immuno-Oncology 360
                          Not Confirmed

                          Details : Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator. It is under phase 3 clinical development for the treatment of pateints with sickle cell disease.

                          Product Name : Pyrukynd

                          Product Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 18, 2024

                          Lead Product(s) : Mitapivat

                          Therapeutic Area : Hematology

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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