Category: Today’s Highlights

bluebird bio Receives Approval for its Gene Therapy SKYSONA™ for Young People with Early CALD Without a Matched Sibling Donor

bluebird bio Receives EC Approval for  bluebird bio (BLUE) announced that the European Commission (EC) has granted marketing authorization of SKYSONA™ (elivaldogene autotemcel, Lenti-D™) one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen . . . This content is for paid subscribers. Please click here to subscribe or here to …

Why Cytokinetics’ Stock Price Soared in a Bad Market Day

Cytokinetics Positive Topline Results of CK-274 Cytokinetics (CYTK) announced positive topline results of Phase 2 clinical trial of CK-274 - an investigational cardiac myosin inhibitor in development for the potential treatment of hypertrophic cardiomyopathy (HCM). The results inform dose selection. They also support the progression of CK-274 to a planned Phase 3 registrational clinical trial, expected to start before . . . This content is …

Important Steps Forward Taken by Vir Biotechnology and Separately by Compugen

Vir Biotechnology Vir Biotechnology (VIR) announced the dosing of the first patient in the Phase 2 MARCH (Monoclonal Antibody siRNA Combination against Hepatitis B) trial to evaluate VIR-2218 together with VIR-3434 for the treatment of patients with chronic hepatitis B virus (HBV) infection – a combination designed to achieve a functional cure. VIR-2218 is an investigational . . . This content is for paid subscribers. …

Halozyme Announced Janssen Received U.S. FDA Approval of DARZALEX FASPRO®. See Also: IVERIC bio News

Halozyme Therapeutics Announced US FDA Approval for Janssen Biotech's DARZALEX FASPRO® Halozyme Therapeutics (HALO) announced that Janssen Biotech, Inc. (Janssen) received U.S. FDA approval of DARZALEX FASPRO® (daratumumab + hyaluronidase-fihj) in combination with pomalidomide and dexamethasone (Pd) for adult patients with multiple myeloma who have received at least one prior line of therapy, including . . . This content is for paid subscribers. Please click here to …

IVERIC bio Receives FDA Agreement Under an SPA for GATHER2 Phase 3 Clinical Trial of Zimura®

IVERIC bio GATHER2 Trial of Zimura® for Geographic Atrophy  IVERIC Bio (ISEE) announced that it received written agreement from the U.S. FDA under a Special Protocol Assessment (SPA) for the overall design of GATHER2, the pivotal clinical trial of IVERIC product Zimura® (avacincaptad pegol) for the treatment of . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Vir Biotechnology: Promising Clinical Data from Ongoing Trials of VIR-2218 and VIR-3434 for Chronic HBV Infection  

Vir Biotechnology Announced Positive Safety Findings from its Ongoing Trials for Chronic HBV Vir Biotechnology (VIR) announced new data from its ongoing Phase 2 clinical trials of VIR-2218 and ongoing Phase 1 studies of VIR-3434 in patients with chronic hepatitis B virus (HBV) infection. The results comprised positive safety findings in addition to a reduction in hepatitis . . . This content is for paid …

Halozyme Therapeutics Good News About Approvals. See Also: Arrowhead Pharmaceuticals Good Trial Results for Liver Diseases  

Halozyme Therapeutics Approvals for DARZALEX®SC Halozyme Therapeutics (HALO) announced that the European Commission (EC) has granted Janssen Pharmaceutical Companies of Johnson & Johnson two marketing authorizations for the subcutaneous formulation of daratumumab, known as DARZALEX® SC in the European Union.  The first authorization relates to the use of DARZALEX® SC in combination with bortezomib . . . This content is for paid subscribers. Please click here to …

Jounce Therapeutics Inc Achieves First Milestone in Exclusive License Agreement with Gilead Sciences for JTX-1811

Jounce Therapeutics Inc: US FDA Cleared IND Application for JTX-1811 Targeting CCR8 Jounce Therapeutics Inc (JNCE) - a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for its product JTX-1811, an anti-CCR8 antibody.

The U.S. FDA Approves Vertex Pharmaceuticals TRIKAFTA for Children with Cystic Fibrosis Aged 6 to 11 with Certain Mutations

Vertex Pharmaceuticals Receives FDA Approval for TRIKAFTA® Vertex Pharmaceuticals (VRTX) announced the U.S. FDA approved the expanded use of TRIKAFTA® to include children with cystic fibrosis (CF) ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive . . . This content is for paid subscribers. Please click here to …

Intellia Therapeutics Gene Editing Product NTLA-2001 – A Dream on its Way to Becoming Reality

Focus on Intellia Therapeutics Upcoming Presentation for NTLA-2001 Data to offer insight into safety and pharmacodynamics of NTLA-2001, the first-ever systemically administered in vivo CRISPR therapy candidate Late-breaking abstract selected for oral presentation on June 26 Intellia Therapeutics NTLA-2001 for People Living with hATTR-PN Intellia Therapeutics (NTLA) announced that Phase 1 . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

BridgeBio Pharma Has Promising News

BridgeBio Pharma Granted Fast Track Designation for Encaleret BridgeBio Pharma (BBIO) announced the U.S. FDA granted Fast Track designation for Encaleret – a treatment of autosomal dominant hypocalcemia (ADH1), on World Hypoparathyroidism Awareness Day, an annual global awareness event meant to support people who are living with ADH1 and with other types of hypoparathyroidism. ADH1 is a rare, genetic form of . . . This …

Annovis Bio Inc: Very Encouraging Trial Results in Neurodegenerative Diseases

Annovis Bio Inc and Neurodegenerative Diseases Annovis Bio Inc (ANVS), a clinical-stage drug platform company. This firm's focus is the treatment of neurodegenerative diseases. Annovis Bio Inc announced today new results from a double-blind, placebo-controlled study of its product ANVS401, the Company's lead . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Compugen: First Quarter Financial Results, Accomplishments and Future Plans

Compugen Updated Trial Data  Updated data from COM701 Phase 1 combination and monotherapy studies to be presented at the American Society of Clinical Oncology (ASCO) 2021 Annual Meeting Milestone rich 2021 to include data readouts from ongoing COM701 triple combination study and COM902 monotherapy study in Q4 2021 Initiation of Phase 1b cohort expansion study of COM701 with Opdivo® in Q2 2021 and Phase 1 …

Editas Medicine: Business Highlights and Financial Results Reported

Editas Medicine Reporting Editas Medicine (EDIT) reported business highlights and financial results for the first quarter of 2021. The reporting started with James C. Mullen, Chairman, President and Chief Executive Officer, Editas Medicine stating that Editas’ team is making tremendous progress towards discovering, developing, and manufacturing novel genome editing medicines, including excellent forward momentum this quarter for the firm’s two . . . This content …