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[{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces Publication of Manuscript Evaluating Mosaicism in Fanconi Anemia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protara Therapeutics Receives Rare Pediatric Disease Designation for TARA-002 for the Treatment of Lymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Protara Therapeutics"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protara Therapeutics Provides Comparability and U.S. Regulatory Updates for TARA-002 to Advance in Oncology and Rare Disease Indications","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Protara Therapeutics"},{"orgOrder":0,"company":"Raziel Therapeutics","sponsor":"Fosun Pharmaceutical","pharmaFlowCategory":"D","amount":"$74.0 million","upfrontCash":"$27.0 million","newsHeadline":"Raziel Announces China Market License Agreement with Fosun Pharma for RZL 012","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"ISRAEL","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Raziel Therapeutics"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Cowen","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Protara Therapeutics Announces Proposed Concurrent Public Offerings of Common Stock and Preferred Stock","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Protara Therapeutics"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals to Present Data from its Fanconi Anemia, Leukocyte Adhesion Deficiency-I and Pyruvate Kinase Deficiency Programs at the 62nd American Society of Hematology Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Reports Positive Topline Results from Tesomet Phase 2 Open-Label Extension Study in Hypothalamic Obesity","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"AGC Biologics","sponsor":"Rocket Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AGC Biologics Expands Partnership with Rocket Pharmaceuticals","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"AGC Biologics"},{"orgOrder":0,"company":"Baymedica","sponsor":"InMed Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"InMed Pharmaceuticals Signs Non-Binding Letter of Intent to Acquire BayMedica, a Commercial Manufacturer of Rare Cannabinoids","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Baymedica"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","amount":"$12.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Pharmaceuticals Announces $12 Million Private Placement Priced At-the-Market under Nasdaq Rules","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Results Show Long-Lasting and Holistic Improvements in Children with AADC Deficiency Treated with PTC-AADC Gene Therapy","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"PTC Therapeutics"},{"orgOrder":0,"company":"ChromaDex, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ChromaDex Shares Findings from First Clinical Study on Nicotinamide Riboside (NR) in Children, Highlighting Improvements for Prematurely Aging Patients with Ataxia-Telangiectasia (AT)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"ChromaDex, Inc"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Provides Update on EMA Regulatory Review Timeline For Its AADC Deficiency Gene Therapy Program","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"PTC Therapeutics"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"University of L\u2019Aquila","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"InMed Expands Patent Portfolio with Novel Cannabinoid Analogs and Advances Collaboration Agreement with Leading Cannabinoid Research Expert","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Receives Positive CHMP Opinion for Upstaza\u2122 for the Treatment of AADC Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"PTC Therapeutics"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Pharmaceuticals Announces Commencement of Phase 2 Clinical Trial Investigating Cannabinol (CBN), a Rare Cannabinoid, in the Treatment of Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclerion Therapeutics Provides Additional Positive CY6463 MELAS Clinical Data at a Webinar Hosted by United Mitochondrial Disease Foundation","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Cyclerion Therapeutics"},{"orgOrder":0,"company":"InflaRx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InflaRx Receives FDA Fast Track Designation for Treatment of Ulcerative Pyoderma Gangrenosum","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"GERMANY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"InflaRx"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Update on Phase 2 Clinical Trial Investigating INM-755 Cannabinol Cream for Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Kira Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kira Pharmaceuticals Receives FDA Orphan Drug Designation for KP104, a Bifunctional Antibody Fusion Protein, for the Treatment of Paroxysmal Nocturnal Hemoglobinuria","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Kira Pharmaceuticals"},{"orgOrder":0,"company":"Kira Pharmaceuticals","sponsor":"Perelman School of Medicine","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kira Pharmaceuticals to Present New Preclinical Data on Lead Asset KP104 at the 2022 European Meeting on Complement in Human Disease Conference","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Kira Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","amount":"$6.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Pharmaceuticals Announces Closing of $6 Million Private Placement Priced at a Premium to Market","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Baymedica","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Pharmaceuticals Provides Update on its Core Research and Development Programs and BayMedica Business","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","amount":"$28.0 million","upfrontCash":"Undisclosed","newsHeadline":"Taysha Announces Pricing of Public Offering of Common Stock","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Taysha Gene Therapies"},{"orgOrder":0,"company":"Kira Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kira Pharmaceuticals Announces First Cohort of Patients Dosed in Phase 2 Study of KP104 in Paroxysmal Nocturnal Hemoglobinuria (PNH)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Kira Pharmaceuticals"},{"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":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"NovelMed Therapeutics"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palvella Therapeutics Announces Positive Topline Results from Phase 2 Study of QTORIN 3.9% Rapamycin Anhydrous Gel (QTORIN rapamycin) for the Treatment of Microcystic Lymphatic Malformations, a Serious, Rare Genetic Skin Disease with No FDA-approved Thera","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Palvella Therapeutics"},{"orgOrder":0,"company":"Silk Road Therapies","sponsor":"Soligenix","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Soligenix Enters into Exclusive Option Agreement with Silk Road Therapeutics for Rights to Topical Pentoxifylline Designed to Treat Beh\u00e7et's Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Silk Road Therapies"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$8.0 million","upfrontCash":"$8.0 million","newsHeadline":"Cyclerion Announces Definitive Agreement for Zagociguat and CY3018","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Cyclerion Therapeutics"},{"orgOrder":0,"company":"Tisento Therapeutics","sponsor":"Invus","pharmaFlowCategory":"D","amount":"$81.0 million","upfrontCash":"Undisclosed","newsHeadline":"Tisento Launches with $81 Million from Top-Tier Investor Syndicate and Promising Cyclerion Assets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Tisento Therapeutics"},{"orgOrder":0,"company":"AlgoTherapeutix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AlgoTx Announces First Patients Dosed in US and European Arms of Phase II Clinical Trial of ATX01 in Rare Disease Erythromelalgia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"September 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"AlgoTherapeutix"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Caliway Announces CBL-514 Phase 2 Study Topline Results for Dercum's Disease, Showing 64.5% of Lipomas with Complete Clearance or Dimensions Reduction of Over 50% and Reduced Pain by 4.7","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"TAIWAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Caliway Biopharmaceuticals"},{"orgOrder":0,"company":"Vigil Neuroscience","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vigil Neuroscience Receives Positive Opinion from European Medicines Agency on Orphan Drug Designation for VGL101 for the Treatment of AlSP","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Vigil Neuroscience"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Ligand Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$5.0 million","newsHeadline":"Palvella Therapeutics and Ligand Pharmaceuticals Expand Strategic Partnership to Accelerate Phase 3 Development of QTORIN\u2122 Rapamycin for Microcystic Lymphatic Malformations and Additional High Unmet Need Clinical Indications","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Palvella Therapeutics"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Caliway Closes Oversubscribed Capital Raise and Secures Over $100M to Advance CBL-514 Pivotal Phase 3 Study in Subcutaneous Fat Reduction","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"TAIWAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Caliway Biopharmaceuticals"},{"orgOrder":0,"company":"Longboard Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Longboard Pharmaceuticals Announces Positive Topline Data from the PACIFIC Study, a Phase 1b\/2a Clinical Trial, for Bexicaserin (LP352) in Participants with Developmental and Epileptic Encephalopathies (DEEs)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Longboard Pharmaceuticals"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"USAN Approves Generic Name \u201cRuxoprubart\u201d for NM8074, an Antibody Therapy focused on Complement-Mediated Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"NovelMed Therapeutics"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Caliway Announces FDA Acceptance of CBL-514 IND Application for Placebo-Controlled Phase 2 Study to Treat Dercum's Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"TAIWAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Caliway Biopharmaceuticals"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"The European Commission Grants Orphan Drug Designation to NS-229 for the Treatment of Eosinophilic Granulomatosis with Polyangiitis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"NS Pharma"},{"orgOrder":0,"company":"Amylyx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amylyx Pharmaceuticals Announces Completion of Enrollment in HELIOS, a Phase 2 Study of AMX0035 for the Treatment of Wolfram Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Amylyx Pharmaceuticals"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ruxoprubart (NM8074) Scores FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"NovelMed Therapeutics"},{"orgOrder":0,"company":"Palvella Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palvella Therapeutics Announces U.S. FDA Breakthrough Therapy Designation Granted to QTORIN\u2122 3.9% Rapamycin Anhydrous Gel (QTORIN\u2122rapamycin) for the Treatment of Microcystic Lymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Palvella Therapeutics"},{"orgOrder":0,"company":"Glycomine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Glycomine Announces First Pediatric Patient Dosed in Phase 2 Clinical Study of GLM101, a Potential Treatment for PMM2-CDG","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Glycomine"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Caliway Received U.S. FDA Fast Track Designation for CBL-514 for the Treatment of Dercum's Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"TAIWAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Caliway Biopharmaceuticals"},{"orgOrder":0,"company":"Caliway Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Caliway Announced Orphan Drug Designation Granted to CBL-514 for the Treatment of Dercum's Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"TAIWAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Caliway Biopharmaceuticals"},{"orgOrder":0,"company":"Glycomine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Glycomine Announces Encouraging Efficacy Data from Ongoing Phase 2 Clinical Study in PMM2-CDG","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Glycomine"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Xenon Pharmaceuticals Inc","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xenon Pharmaceuticals Provides Update on Partnered Program with Neurocrine Biosciences","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Neurocrine Biosciences"},{"orgOrder":0,"company":"Hansa Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Imlifidase Demonstrated Positive Safety, Tolerability, and Early Efficacy Outcomes in 15-HMedIdeS-09 Phase 2 Trial in Guillain-Barr\u00e9 Syndrome (GBS)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"SWEDEN","productType":"Large molecule","productStatus":"Approved","date":"December 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Hansa Biopharma"}]

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            GLM101 is a mannose-1-phosphate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG Type Ia.

            Lead Product(s): GLM101

            Therapeutic Area: Rare Diseases and Disorders Product Name: GLM101

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 04, 2024

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            CBL-514 is a potentially first-in-class small-molecule drug, is an injection lipolysis drug that can induce adipocytes apoptosis and lipolysis to reduce subcutaneous adiposity. It is being developed for the treatment of dercum's disease.

            Lead Product(s): CBL-514

            Therapeutic Area: Rare Diseases and Disorders Product Name: CBL-514

            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 03, 2024

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            CBL-514 is a potentially first-in-class small-molecule drug, is an injection lipolysis drug that can induce adipocytes apoptosis and lipolysis to reduce subcutaneous adiposity. It is being developed for the treatment of dercum's disease.

            Lead Product(s): CBL-514

            Therapeutic Area: Rare Diseases and Disorders Product Name: CBL-514

            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 February 14, 2024

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            NM8074 (ruxoprubart) is an alternative pathway (AP) blocker anti-Bb antibody. It is being evaluated for the treatment of paroxysmal nocturnal hemoglobinuria.

            Lead Product(s): Ruxoprubart

            Therapeutic Area: Rare Diseases and Disorders Product Name: NM8074

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 12, 2024

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            Relyvrio (sodium phenylbutyrate and taurursodiol) is an oral, fixed-dose combination. It is a HDAC inhibitor which is being evaluated in phase 2 clinical trials for the treatment of Wolfram syndrome.

            Lead Product(s): Sodium Phenylbutyrate,Tauroursodeoxycholic Acid

            Therapeutic Area: Rare Diseases and Disorders Product Name: Relyvrio

            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 February 08, 2024

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            NS-229 is a potent and selective Janus kinase (JAK) 1 inhibitor. It is being evaluated in phase 2 clinical trials for the treatment of eosinophilic granulomatosis with polyangiitis.

            Lead Product(s): NS-229

            Therapeutic Area: Rare Diseases and Disorders Product Name: NS-229

            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 22, 2024

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            NM8074 (ruxoprubart) is a novel Factor B binder, which is under phase 2 clinical development for the treatment of patients with paroxysmal nocturnal hemoglobinuria.

            Lead Product(s): Ruxoprubart

            Therapeutic Area: Rare Diseases and Disorders Product Name: NM8074

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 08, 2024

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            CBL-514 is a small-molecule drug, is an injection lipolysis drug that can induce adipocyte apoptosis and lipolysis to reduce subcutaneous adiposity. It is being evaluated in preclinical studies for the treatment of Dercum's Disease.

            Lead Product(s): CBL-514

            Therapeutic Area: Rare Diseases and Disorders Product Name: CBL-514

            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 08, 2024

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            LP352 (Bexicaserin) is a potentially best-in-class and highly selective, oral, novel 5-HT2C receptor superagonist, which is under phase 1/2 clinical development for the treatment of Developmental and Epileptic Encephalopathies (DEEs).

            Lead Product(s): Bexicaserin

            Therapeutic Area: Rare Diseases and Disorders Product Name: LP352

            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 02, 2024

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            The net financing will enable Caliway to advance the clinical development of its lead pipeline CBL-514, a small-molecule injectable drug that reduces subcutaneous fat in treatment areas.

            Lead Product(s): CBL-514

            Therapeutic Area: Rare Diseases and Disorders Product Name: CBL-514

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Financing December 16, 2023

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