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264 stories matching this filter · ← front page

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  • Novo Nordisk halts two IL-6 heart trials as Novartis suffers Phase 3 setbacks; Ionis wins first Alexander disease approval
    Novo Nordisk announced it has ended two late‑stage studies of its investigational IL‑6 inhibitor ziltivekimab, which was being evaluated for cardiovascular disease. The decision follows an earlier…
    BioSpace · 22h agoNegative↗ source
  • Encoded Therapeutics raises $275 million in Series F round for Dravet syndrome program
    Encoded Therapeutics announced closing a Series F financing round that brought in $275 million. The round was led by GV with participation from an undisclosed healthcare investor. The new capital…
    Endpoints · 16h agoDealPositive↗ source
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fda medwatch66
fda recalls64
fda press38
1205
Psychiatry & CNS873
Ophthalmology508
Pain483
Endocrinology & Metabolism483
Infectious Disease451
  • FDA appoints permanent heads of CDER and CBER, adding AI deputy role
    The Trump administration announced permanent appointments to two key FDA centers after a period of turnover. Michael Davis, who had been acting director of the Center for Drug Evaluation and Resea…
    BioPharma Dive · 19h agoRegulatoryPositive↗ source
  • Biotech crossover rounds projected to exceed $4.2 billion in 2026, supporting IPO pipeline
    PitchBook forecasts that biotech crossover rounds could surpass $4.22 billion across roughly 28 rounds in 2026. The surge follows a market bottom after COVID and a rise in investor confidence, wit…
    BioSpace · 1d agoDealPositive↗ source
  • CLINUVEL to cut up to 20% of staff and move headquarters to New York
    CLINUVEL announced a strategic reorganization aimed at focusing resources on the U.S. market. The company will reduce its global workforce by 10% to 20%, affecting up to 21 of its 104 employees.…
    BioSpace · 11h agoLayoffsNegative↗ source
  • Novartis' $12 billion Avidity drug fails Phase 3 trial in myotonic dystrophy
    Novartis reported that its antibody-oligonucleotide conjugate del-desiran did not meet the primary endpoint in the Phase 3 HARBOR study for myotonic dystrophy type 1, showing no significant improve…
    BioSpace · 1d agoNegative readoutNegative↗ source
  • Roivant reports record-breaking Phase 2 results for PH-ILD drug mosliciguat, starts Phase 3
    Roivant announced that its experimental drug mosliciguat met all primary and secondary endpoints in a Phase 2 study in patients with pulmonary hypertension associated with interstitial lung disease…
    BioPharma Dive · 1d agoPositive readoutPositive↗ source
  • Structure Therapeutics reports oral GLP-1 weight-loss results comparable to Wegovy and strong Phase 1 amylin data
    Structure Therapeutics announced that its oral GLP-1 candidate aleniglipron achieved 72-week weight-loss results that BMO Capital Markets said were in line with Novo Nordisk’s oral Wegovy. Particip…
    BioSpace · 1d agoPositive readoutPositive↗ source
  • Novartis' del-desiran misses primary goal in late-stage myotonic dystrophy trial
    Novartis announced that its experimental RNA drug del-desiran did not achieve the primary endpoint in a late-stage study of myotonic dystrophy type 1, a rare muscle‑weakening disease. The trial me…
    BioPharma Dive · 1d agoNegative readoutNegative↗ source
  • BrainChild Bio secures $119M Series A to advance CAR-T therapy for deadly pediatric brain tumor
    BrainChild Bio announced a $119 million Series A financing round aimed at moving its CAR‑T programs for central nervous system cancers in children forward. The company’s lead candidate, BCB-276, w…
    BioSpace · 1d agoDealPositive↗ source
  • BrainChild secures $116M to advance CAR-T therapies for rare pediatric brain tumors
    BrainChild Bio, a Seattle‑based biotech spun out of Seattle Children’s Hospital, announced it has raised $116 million in venture financing. The capital will fund the development of its CAR‑T cell t…
    BioPharma Dive · 1d agoDealPositive↗ source
  • Pharvaris reports injectable-like efficacy from once-daily oral pill for rare genetic disease
    Pharvaris announced that its experimental oral formulation of deucrictibant achieved efficacy comparable to injectable versions in a once-daily pill. The drug is an extended-release version intend…
    Endpoints · 1d agoPositive readoutPositive↗ source
  • Beam Therapeutics reports one-year data from early-stage AATD gene therapy trial
    Beam Therapeutics released new results from its early-stage gene-editing program targeting alpha-1 antitrypsin deficiency (AATD), a rare condition that can damage the lungs and liver. The data com…
    Endpoints · 2d agoPositive readoutPositive↗ source
  • Ionis receives FDA approval for Zanvastro, the first treatment for Alexander disease, as Pfizer partners on new ADC program
    The U.S. Food and Drug Administration approved Zanvastro, marking the first approved medicine for the rare and often fatal neurological disorder Alexander disease. Developed by Ionis Pharmaceutical…
    BioPharma Dive · 5d agoApprovalPositive↗ source
  • Licensing deals with Chinese biotech stay strong as big pharma pours $550M+ into cancer and obesity pipelines
    Western drug developers continue to sign licensing agreements with Chinese biotechnology firms at a steady pace, according to recent data visualizations. More than 100 such deals have been announc…
    BioPharma Dive · 5d agoDealNeutral↗ source
  • Ionis secures FDA approval for zilganersen, its first solo neurology therapy for Alexander disease
    Ionis Pharmaceuticals announced that the U.S. Food and Drug Administration has granted approval to its investigational RNA‑based drug zilganersen for the treatment of Alexander disease, a rare neur…
    Endpoints · 5d agoApprovalPositive↗ source
  • MassBio and SCbio select 10 life science startups for Drive accelerator
    MassBio and SCbio announced that ten early‑stage life‑science companies have been chosen for the fall cohorts of the Drive accelerator program. The selection includes five biotech firms and five te…
    BioSpace · 5d agoPositive↗ source
  • Ultragenyx’s Angelman therapy fails Phase 3, stock drops over 43% and company announces strategic review
    Ultragenyx reported that its investigational Angelman syndrome drug apazunersen did not meet the primary cognitive endpoints in a Phase 3 trial. The lack of efficacy means the therapy will not prov…
    BioSpace · 6d agoNegative readoutNegative↗ source
  • FDA approves Ionis’ Zanvastro as first disease-modifying therapy for Alexander disease
    The U.S. Food and Drug Administration has granted approval to Ionis Pharmaceuticals' antisense oligonucleotide Zanvastro, the first therapy that modifies disease progression for Alexander disease.…
    BioSpace · 6d agoApprovalPositive↗ source
  • Ultragenyx to consider major cost cuts after Angelman drug fails trial
    Ultragenyx announced that its experimental therapy GTX-102 (apazunersen) for Angelman syndrome failed to meet primary and secondary endpoints in a Phase 3 study. The company said there were no dif…
    BioPharma Dive · 6d agoNegative readoutNegative↗ source
  • Revolution Medicines' Rasonque shows 42% response and 16-month survival in NSCLC subgroup
    Data from a subgroup of an earlier trial, described by Truist Securities as the most relevant to Revolution Medicines' ongoing Phase 3 study, reported a 42% overall response rate and a median overa…
    BioSpace · 6d agoPositive readoutPositive↗ source
  • Superluminal Medicines raises $60 million to advance AI-designed MC4R drug for rare obesity forms
    Superluminal Medicines, an AI-driven drug discovery startup, announced a $60 million financing round aimed at moving its first candidate toward clinical testing. The candidate targets the MC4R G p…
    BioPharma Dive · 6d agoDealPositive↗ source
  • ARPA-H allocates up to $125 million to accelerate personalized RNA therapy manufacturing
    ARPA-H announced a new program, called GIVE, that will provide up to $125 million in research funding to develop faster, decentralized manufacturing methods for individualized RNA‑based genetic med…
    BioSpace · 6d agoRegulatoryPositive↗ source
  • Senior FDA official overrides reviewers to grant Replimune accelerated approval for melanoma drug Tudriqev
    FDA's primary review team recommended rejecting Replimune's melanoma therapy Tudriqev, saying the company had not adequately addressed key deficiencies noted in earlier complete response letters.…
    BioSpace · 6d agoRegulatoryNeutral↗ source
  • Ultragenyx's Angelman syndrome drug fails Phase 3 trial, halting development
    Ultragenyx reported that its experimental antisense oligonucleotide designed for Angelman syndrome did not meet primary endpoints in a Phase 3 study. The trial, which enrolled patients with the ra…
    Endpoints · 7d agoNegative readoutNegative↗ source