Small Molecules

24 Oct 2022 Positive Topline Data Announced from Mirum’s LIVMARLI Phase 3 MARCH Study in Progressive Familial Intrahepatic Cholestasis (PFIC)
24 Oct 2022 Quizartinib Granted Priority Review in the U.S. for Patients with Newly Diagnosed FLT3-ITD Positive Acute Myeloid Leukemia
24 Oct 2022 Novartis investigational oral monotherapy iptacopan demonstrates clinically meaningful superiority over anti-C5 treatment in Phase III APPLY-PNH study
23 Oct 2022 Merck’s PREVYMIS™ Demonstrates Efficacy in Phase 3 Study for Prevention of Cytomegalovirus Disease in Adults After Kidney Transplantation
21 Oct 2022 U.S. Food and Drug Administration (FDA) Accepts Supplemental New Drug Application for CAMZYOS® (mavacamten) in Symptomatic Obstructive Hypertrophic Cardiomyopathy to Reduce the Need for Septal Reduction Therapy
20 Oct 2022 STORM Therapeutics to present new data on its lead program STC-15 at the 34th EORTC-NCI-AACR Symposium and the 37th SITC Meeting
20 Oct 2022 Orionis Biosciences Secures $55 Million Financing to Support Advancement into Clinic
18 Oct 2022 Prelude Therapeutics Receives FDA Clearance of IND for PRT3789, a Potent and Selective First-in-Class SMARCA2 Protein Degrader
18 Oct 2022 Avilar Therapeutics to Present New In Vivo Proof-of-Concept Data for ATAC Extracellular Protein Degraders at Upcoming Drug Discovery Conferences
18 Oct 2022 PTC Provides Update on Ongoing Global PIVOT-HD Trial For PTC518
18 Oct 2022 1ST Biotherapeutics, Inc., Announces Positive Phase I Data of c-Abl Inhibitor in Neurodegeneration
18 Oct 2022 POINT Biopharma Publishes Preclinical Data for Actinium-Labelled PNT2001, a Next-Generation PSMA Ligand, at EANM’s Annual Congress
17 Oct 2022 Enanta Pharmaceuticals Doses First Subject in a Phase 1 Clinical Study of EDP-323, its Novel, Oral L-Protein Inhibitor in Development for the Treatment Respiratory Syncytial Virus
17 Oct 2022 BMF-219 Enters the Clinic for KRAS Solid Tumors
17 Oct 2022 Bayer expands development program for elinzanetant with Phase III study in breast cancer patients with vasomotor symptoms caused by endocrine therapy
15 Oct 2022 BridgeBio Pharma Presents 12-month Results from Phase 2 Study of BBP-418 in Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i)
12 Oct 2022 Novo Seeds Portfolio Company NMD Pharma Reports Positive Top-Line Phase I/IIa Data in Myasthenia Gravis
12 Oct 2022 Vertex Advances Program Targeting Alpha-1 Antitrypsin Deficiency
12 Oct 2022 Accutar Biotechnology Announces First Patient Dosed in China with AC0682 in Phase 1 Study in ER-Positive Breast Cancer
12 Oct 2022 DICE Therapeutics Announces Positive Topline Data from Phase 1 Clinical Trial of Lead Oral IL-17 Antagonist, DC-806, for Psoriasis
12 Oct 2022 Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)
10 Oct 2022 Sciwind Biosciences Announces Initiation of Dosing in Phase 1 Clinical Trial Evaluating XW014, an oral small molecule GLP-1 receptor agonist for the Treatment of Obesity and Type 2 Diabetes
10 Oct 2022 Merck Announces Positive Top-line Results from Pivotal Phase 3 STELLAR Trial Evaluating Sotatercept for the Treatment of Adults with Pulmonary Arterial Hypertension (PAH)
05 Oct 2022 Enanta Pharmaceuticals Initiates a Phase 2b Clinical Study of EDP-938 in High-Risk Adults with Respiratory Syncytial Virus
05 Oct 2022 KalVista Pharmaceuticals Announces Termination of KVD824 Phase 2 KOMPLETE Trial for Prophylactic Treatment of Hereditary Angioedema

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