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327 stories matching this filter · ← front page

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  • AstraZeneca partners with Oracle to boost clinical trial patient recruitment using AI
    AstraZeneca announced a collaboration with data-technology firm Oracle to apply artificial-intelligence tools to clinical-trial recruitment. The non-binding agreement will let the partners explore…
    Fierce Biotech · 8h agoDealPositive↗ source
  • Merck KGaA to acquire Silence Therapeutics for $1.3 billion, expanding siRNA portfolio
    Merck KGaA, the German science and technology conglomerate, announced an agreement to purchase UK‑based Silence Therapeutics. The deal is valued at about $1.3 billion and gives Merck access to Sil…
    Endpoints · 21h agoDealPositive↗ source
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  • Disc spins past FDA rejection with ‘overwhelmingly positive’ phase 3 data for rare disease drug
    A phase 3 trial of Disc Medicine’s bitopertin has hit both co-primary endpoints and all secondary endpoints, positioning the biotech to refile for approval of the once-rejected rare disease drug ca…
    Fierce Biotech · 1d agoApprovalPositive↗ source
  • Merck KGaA to acquire Silence Therapeutics for $1.3 billion, adding Phase-2 RNAi drug for polycythemia vera
    Merck KGaA announced it will purchase UK‑based Silence Therapeutics for about $1.3 billion, paying $20 in cash per share, representing a 130 % premium. The deal gives Merck control of an RNA‑inter…
    BioPharma Dive · 21h agoDealPositive↗ source
  • Disc Medicine reports positive Phase 3 results for bitopertin in erythropoietic porphyria
    Disc Medicine announced that its experimental therapy bitopertin achieved its primary and secondary goals in a Phase 3 trial for erythropoietic porphyria, a rare condition that makes patients extre…
    BioPharma Dive · 18h agoPositive readoutPositive↗ source
  • Disc Medicine's rare sun-sensitivity drug meets all endpoints in Phase 3 trial
    Disc Medicine announced that its experimental therapy for a rare condition that makes sun exposure painful has completed a Phase 3 study. The trial reported success on every primary and secondary…
    Endpoints · 1d agoPositive readoutPositive↗ source
  • Saudi Arabia rolls out biotech partnerships to support its $34.6 billion non‑oil growth plan
    Saudi Arabia’s National Biotechnology Strategy, launched in early 2024, aims to add $34.6 billion to non‑oil GDP by 2040 and create 11,000 jobs by 2030. The plan is now moving beyond policy into co…
    BioPharma Dive · 1d agoPositive↗ source
  • Merck KGaA buys Silence in $1.3B deal after siRNA drug's phase 2 blood cancer win
    Germany’s Merck KGaA has picked up Silence Therapeutics to the tune of $1.3 billion in the wake of the British biotech’s mid-stage success for its challenger to Takeda’s Mimrylo.
    Fierce Biotech · 1d agoDealNeutral↗ source
  • City Therapeutics raises $200 million in IPO to fund next-gen RNAi programs
    City Therapeutics, a biotech focused on RNA interference, completed a $200 million initial public offering, selling about 11.1 million shares at $18 each. The proceeds will back three drug candida…
    BioPharma Dive · 4d agoDealPositive↗ source
  • Ambrose Healthcare acquires AM-Pharma for up to €161 million
    Ambrose Healthcare, a British rare‑disease specialist, announced it has completed the acquisition of Dutch biotech AM‑Pharma. The deal values the company at up to 161 million euros (about $181 mill…
    Fierce Biotech · 4d agoDealNeutral↗ source
  • FDA launches Rare Disease Framework Initiative to overhaul review process
    The FDA announced a new Rare Disease Framework Initiative aimed at redesigning how therapies for rare conditions are regulated. The initiative will move away from a one-size-fits-all model, instea…
    BioPharma Dive · 4d agoRegulatoryPositive↗ source
  • Oxford biotech OMass Therapeutics secures $310 million to advance genetic adrenal disorder program
    Oxford-based OMass Therapeutics announced a $310 million private financing round, one of the largest recent fundraises for a UK biotech. The capital will fund the development of therapies targetin…
    Endpoints · 5d agoDealNeutral↗ source
  • FDA proposes new framework prioritizing consistency over flexibility for rare disease drug reviews
    The FDA announced a shift in its approach to evaluating rare disease therapies, emphasizing a more uniform assessment process. Agency officials explained that the flexibility shown over the summer…
    Endpoints · 5d agoRegulatoryNeutral↗ source
  • FDA launches Rare Disease Framework Initiative to reshape drug development for small patient populations
    The FDA announced a new Rare Disease Framework Initiative aimed at overhauling how drugs and biologics for rare conditions are developed. The initiative will produce a tailored framework that refl…
    fda press · 5d agoRegulatoryPositive↗ source
  • Ultragenyx sells second priority review voucher as Biomea shares tumble after oral GLP-1 data
    Ultragenyx Pharmaceutical, a commercial-stage rare disease company, completed its second sale of a priority review voucher this month. The voucher sale reflects the company's strategy of monetizin…
    Endpoints · 5d agoDealNeutral↗ source
  • iOnctura’s roginolisib shows encouraging phase 1 results in metastatic uveal melanoma
    iOnctura, a spinout of Merck KGaA, reported data from a two‑part phase 1 trial of its PI3K delta inhibitor roginolisib in patients with advanced solid and hematologic cancers, including a cohort of…
    Fierce Biotech · 6d agoPositive readoutPositive↗ source
  • Beam Therapeutics reports early Phase 1/2 data for base editing therapy in GSD Ia, partners with Pfizer and Enodia
    Beam Therapeutics disclosed early results from a Phase 1/2 study evaluating a base editing approach aimed at correcting the genetic defect that causes glycogen storage disease type Ia, a rare metab…
    Endpoints · 6d agoPositive readoutNeutral↗ source
  • Syndax halts development of Niktimvo after Phase 2 failure in IPF trial
    Syndax announced it is stopping work on its experimental drug Niktimvo for idiopathic pulmonary fibrosis (IPF) after the compound did not meet its primary endpoints in a placebo-controlled Phase 2…
    Endpoints · 7d agoNegative readoutNegative↗ source
  • Mirum's Livmarli cuts itching in ultra-rare liver disease trial, showing significant benefit
    Mirum Pharmaceuticals reported that its ileal bile acid transporter inhibitor Livmarli achieved a statistically significant reduction in cholestatic pruritus compared with placebo in a late‑stage s…
    Endpoints · 7d agoPositive readoutPositive↗ source
  • AstraZeneca attributes Phase 3 failure of bone disease enzyme therapy to trial design flaws
    AstraZeneca disclosed that its Phase 3 study of an enzyme replacement therapy for a rare genetic bone disorder did not meet its primary objectives. The company said the shortfall was largely due t…
    Endpoints · 8d agoNegative readoutNegative↗ source
  • Parkinson’s-focused Aspen and bispecific biotech Bambusa plan IPOs
    Parkinson’s disease cell therapy-focused Aspen Neuroscience and bispecific biotech Bambusa Therapeutics are the latest drug developers to eye up a public listing.
    Fierce Biotech · 10d ago↗ source
  • FDA review flags limited efficacy of Revolution's pancreatic cancer drug Rasonque, shares drop 6%
    Shares of Revolution Medicines fell about 6% after the Food and Drug Administration released review documents that suggest its pancreatic cancer therapy Rasonque may not work as well in all patient…
    BioPharma Dive · 10d agoRegulatoryNegative↗ source
  • Fierce Biotech outlines criteria for its 2026 Fierce 15 list, spotlighting top innovators
    The Fierce Biotech editorial team released its 2026 Fierce 15 list and discussed the selection process on a recent episode of "The Top Line" podcast. More than 150 high‑quality entries were review…
    Fierce Biotech · 11d agoPositive↗ source
  • HHS unveils new vaccine injury surveillance plan with NIH clinic and reporting reimbursements
    The Department of Health and Human Services announced a comprehensive initiative to improve monitoring of vaccine-related injuries. The plan includes opening a new clinic at the National Institute…
    BioPharma Dive · 11d agoRegulatoryNeutral↗ source
  • Cadrenal Therapeutics launches strategic review to explore sale or partnership for its late-stage cardiology pipeline
    Cadrenal Therapeutics, a heart-focused biotech, announced a strategic review of its late-stage cardiology pipeline, seeking to offload assets or the entire company. The board is evaluating options…
    Fierce Biotech · 12d agoDealNegative↗ source