Rochem International Inc Rochem International Inc

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Corporation"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. Food & Drug Administration Grants Priority Review of TAK-755 for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Takeda Pharmaceutical"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Govorestat (AT-007) Receives Orphan Medicinal Product Designation from the EMA for Treatment of SORD Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Applied Therapeutics"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Pharmanovia","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Stealth BioTherapeutics Enters into Exclusive Licensing Agreement with Pharmanovia to Commercialize Elamipretide for the Treatment of Barth Syndrome in Europe and Key Global Territories","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Peptide","graph2":"Stealth Biotherapeutics"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Rafael Holdings","pharmaFlowCategory":"D","amount":"$5.0 million","upfrontCash":"Undisclosed","newsHeadline":"Cyclo Therapeutics Announces Execution of Definitive Agreement with Rafael Holdings, Inc. Providing for $5.0 Million of Additional Funding Via a Private Placement","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Data Show Roches Subcutaneously Administered Crovalimab Achieved Disease Control and was Well-Tolerated in People with Paroxysmal Nocturnal Haemoglobinuria (PNH)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"F. Hoffmann-La Roche"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Danicopan as Add-on to Ultomiris or Soliris Improved Haemoglobin Levels and Maintained Disease Control in Patients with PNH Experiencing Signs or Symptoms of Clinically Significant Extravascular Haemolysis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai Files New Drug Application in Japan for Crovalimab for Paroxysmal Nocturnal Hemoglobinuria","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Chugai Pharmaceutical"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio NPC Phase III Successful Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"IntraBio"},{"orgOrder":0,"company":"Savara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Savara Completes Enrollment of Pivotal Phase 3 IMPALA-2 Trial of Molgramostim Nebulizer Solution (Molgramostim) in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Savara"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AG10 (Acoramidis) is an Investigational, Orally-Administered Small Molecule Designed to Potently Stabilize Tetrameric Transthyretin (TTR)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Rafael Holdings","pharmaFlowCategory":"D","amount":"$5.0 million","upfrontCash":"Undisclosed","newsHeadline":"Cyclo Therapeutics Announces Closing of $5.0 Million Private Placement with Rafael Holdings, Inc.","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"August 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Hercules Capital","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"X4 Pharmaceuticals Closes $115 Million Loan Facility with Hercules Capital","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"X4 Pharmaceuticals"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Announces First Patient Enrolled in Phase III Clinical Trial of Leniolisib for the Treatment of APDS in Japan","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"Approved","date":"August 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharming"},{"orgOrder":0,"company":"Genentech","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Accepts Application for Genentech\u2019s Crovalimab for the Treatment of PNH, a Rare Life-Threatening Blood Condition","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Genentech"},{"orgOrder":0,"company":"Applied Molecular Transport","sponsor":"Cyclo Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Cyclo Therapeutics, Inc. and Applied Molecular Transport Inc. 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            The net proceeds will be used in the anticipated launch of AG10 (acoramidis HCl) as the potential backbone of therapy for transthyretin amyloid cardiomyopathy (ATTR-CM).

            Lead Product(s): Acoramidis Hydrochloride

            Therapeutic Area: Rare Diseases and Disorders Product Name: AG10

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Blue Owl Capital

            Deal Size: $500.0 million Upfront Cash: $500.0 million

            Deal Type: Financing January 18, 2024

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            Pyrukynd (mitapivat sulfate) is a pyruvate kinase activator indicated for the treatment of hemolytic anemia. It is under phase 3 clinical development for the treatment of non-transfusion-dependent alpha- or beta-thalassemia.

            Lead Product(s): Mitapivat

            Therapeutic Area: Rare Diseases and Disorders Product Name: Pyrukynd

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 03, 2024

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            Joenja (leniolisib) is an oral, selective PI3K-delta inhibitor which inhibits the signalling pathways that lead to increased production of PIP3, hyperactivity of the downstream mTOR/AKT pathway, and to the dysregulation of B and T cells in patients with APDS.

            Lead Product(s): Leniolisib

            Therapeutic Area: Rare Diseases and Disorders Product Name: Joenja

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 10, 2023

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            INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme replacement therapy in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton.

            Lead Product(s): rhENPP1-Fc

            Therapeutic Area: Rare Diseases and Disorders Product Name: INZ-701

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 10, 2023

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            ANX005 is a a monoclonal antibody (mAb) designed to fully block C1q and the entire classical complement pathway with the aim of preserving functioning synapses to slow or halt neurodegeneration. It is beig evaluated for GBS autoimmune disoder.

            Lead Product(s): ANX005

            Therapeutic Area: Rare Diseases and Disorders Product Name: ANX005

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 10, 2023

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            Chenodiol (chenodiol) tablets is a naturally occurring bile acid, which is investigated for the treatment of adult patients with cerebrotendinous xanthomatosis.

            Lead Product(s): Chenodeoxycholic Acid

            Therapeutic Area: Rare Diseases and Disorders Product Name: Chenodal

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 02, 2023

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            INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme replacement therapy in development for the treatment of ENPP1 Deficiency and ABCC6 Deficiency (PXE, pseudoxanthoma elasticum).

            Lead Product(s): INZ-701

            Therapeutic Area: Rare Diseases and Disorders Product Name: INZ-701

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 26, 2023

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            The combined company will focus on advancing Cyclo Therapeutics’ pivotal Phase 3 global study evaluating Trappsol Cyclo (hydroxypropyl beta cyclodextrin), an orphan drug designated product in the United States and Europe, for Niemann-Pick Disease Type C1 (NPC1).

            Lead Product(s): Hydroxypropyl-Beta Cyclodextrin

            Therapeutic Area: Rare Diseases and Disorders Product Name: Trappsol Cyclo

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Cyclo Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Merger September 21, 2023

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            RG6107 (crovalimab) is an investigational, novel anti-C5 recycling monoclonal antibody designed to block the complement system which plays a vital part of the innate immune system that acts as the body’s first line of defence against infection and treatment of PNH.

            Lead Product(s): Crovalimab

            Therapeutic Area: Rare Diseases and Disorders Product Name: RG6107

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Chugai Pharmaceutical

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 06, 2023

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