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  • FDA questions efficacy of Capricor's Duchenne muscular dystrophy cell therapy ahead of advisory committee
    FDA reviewers have raised concerns about the efficacy of Capricor's cell therapy for Duchenne muscular dystrophy as the agency prepares for an advisory committee meeting. The agency said it could n…
    Endpoints · 5d agoRegulatoryNegative↗ source
  • FDA says Capricor's DMD cell therapy failed to meet Phase 3 endpoints
    The FDA briefing documents released before an advisory committee meeting state that the Phase 3 trial of Capricor's deramiocel did not achieve its pre-specified primary and secondary efficacy endpo…
    BioSpace · 5d agoRegulatoryNegative↗ source
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  • MapLight's schizophrenia drug fails primary endpoint in Phase 2, sending stock down over 60%
    MapLight Therapeutics saw its shares tumble more than 60% after a mid‑stage trial showed that its once‑daily schizophrenia candidate did not meet the primary efficacy endpoint, even though the over…
    Endpoints · 5d agoNegative readoutNegative↗ source
  • Lilly, AbbVie and others pursue in-vivo CAR-T, but safety concerns loom
    Analysts at H.C. Wainwright say safety will be the decisive factor for in‑vivo CAR‑T therapies, noting that convenience and efficacy alone will not win over investors. In‑vivo CAR‑T aims to genera…
    BioSpace · 5d agoNeutral↗ source
  • Summit Therapeutics reports positive Phase 3 data for lung cancer drug ivonescimab, highlighting regulatory hurdles for US approval
    Summit Therapeutics and its Chinese partner Akeso announced that their PD-1/VEGF antibody ivonescimab showed positive results in a Phase 3 trial for non-small cell lung cancer in Western patients.…
    BioSpace · 5d agoPositive readoutPositive↗ source
  • FDA advisory panel lifts restrictions on six wellness peptides
    The FDA's advisory committee concluded its two-day meeting by voting to remove the existing limits on six widely used health and wellness peptides. Committee members said the vote overrode earlier…
    Endpoints · 5d agoRegulatoryPositive↗ source
  • MapLight’s schizophrenia pill hits primary endpoint but falls short of challenging BMS’s Cobenfy
    MapLight Therapeutics reported results from its Phase 2 ZEPHYR trial of the oral muscarinic agonist ML-007C-MA in schizophrenia. The trial met its primary endpoint, showing a statistically signifi…
    BioSpace · 5d agoPositive readoutNeutral↗ source
  • Gossamer Bio regains rights to lung disease asset from Chiesi after Phase 3 failure, eyes FDA submission
    Gossamer Bio announced that it has taken back the rights to its inhaled pulmonary hypertension candidate from partner Chiesi after the drug failed to meet its primary endpoint in a Phase 3 trial.…
    BioSpace · 5d agoNegative readoutNeutral↗ source
  • MapLight’s schizophrenia drug misses primary endpoint in Phase 2, shares tumble over 60%
    MapLight Therapeutics reported results from a mid‑stage Phase 2 trial of its experimental schizophrenia therapy. The study evaluated a once‑daily dosing regimen and failed to meet the predefined p…
    Endpoints · 5d agoNegative readoutNegative↗ source
  • RA Capital launches rare disease company Oak Hill Bio onto Nasdaq via SPAC
    Rare disease biotech Oak Hill is headed to the Nasdaq through a SPAC merger with RA Capital-sponsored Research Alliance Corporation III (RACC) to fund a rare genetic disease candidate.
    Fierce Biotech · 5d agoDealNeutral↗ source
  • AstraZeneca has no plans to ramp up dealmaking in wake of Wainua failure
    AstraZeneca remains "very much on track" to hit its 2030 goal of reaching $80 billion in revenue despite the recent late-stage failure of Wainua in ATTR-cardiomyopathy, CEO Pascal Soriot said durin…
    BioSpace · 5d agoDealNeutral↗ source
  • Argenx deepens immuno expertise with $2.2B Forte Biosciences buy
    The star of the takeover is Forte Biosciences' investigational antibody therapy, FB102, which has shown early potential in vitiligo and celiac disease.
    BioSpace · 5d agoDealNeutral↗ source
  • STAT+: RA Capital backs upstart rival to Ultragenyx and Ionis in rare brain disease
    Oak Hill Bio, a new company backed by RA Capital and other investors, will take on Ultragenyx and Ionis in developing an Angelman syndrome medicine.
    STAT · 5d agoDealNeutral↗ source
  • Argenx to pick up autoimmune biotech Forte Biosciences for $2.2B
    Argenx will spend $2.2 billion to buy Forte Biosciences and its anti-CD122 monoclonal antibody that’s under investigation for autoimmune diseases. The Belgian-Dutch pharma said Monday it will pay $…
    Endpoints · 5d agoDealNeutral↗ source
  • STAT+: Argenx to buy Forte Biosciences for $2.2B
    Argenx plans to buy Forte Biosciences for $2.2 billion, expanding its pipeline of drugs that could treat multiple autoimmune diseases.
    STAT · 5d agoDealNeutral↗ source
  • Argenx acquires Forte for $2.2B to get hands on phase 2-stage vitiligo drug
    Argenx is spending $2.2 billion to absorb Forte Biosciences for its phase 2-stage vitiligo and celiac disease drug.
    Fierce Biotech · 5d agoDealNeutral↗ source
  • Labcorp unveils direct-to-consumer genetic health panel covering 163 genes and 100+ conditions
    Labcorp announced the launch of a new direct-to-consumer test called the Marker by Labcorp Genetic Health Panel. The panel examines 163 genes to assess risk for more than 100 hereditary conditions…
    Fierce Biotech · 5d agoPositive↗ source
  • Sarepta names former AbbVie R&D exec Michael Severino as next CEO
    After leaving Tessera Therapeutics earlier this month, Michael Severino will take over Sarepta Therapeutics as the biotech awaits two key regulatory decisions in Duchenne muscular dystrophy.
    BioSpace · 5d agoRegulatoryNeutral↗ source
  • STAT+: Sarepta names Michael Severino, former Tessera CEO, as new chief executive
    The beleaguered Sarepta Therapeutics named Michael Severino, who previously led Tessera Therapeutics, as its new CEO.
    STAT · 5d agoRegulatoryNeutral↗ source
  • STAT+: Pharmalittle: We’re reading about a U.S. peptide panel’s votes, Amgen scuffling with the FDA, and more
    An FDA panel recommended that compounding pharmacies be allowed to make two more peptides, a step toward RFK Jr.'s goal to make them more available
    STAT · 5d agoRegulatoryNeutral↗ source
  • STAT+: Ahead of hearing, FDA questions the efficacy of Capricor’s Duchenne drug
    Ahead of a hearing, the FDA raised questions about the benefits of a Duchenne treatment from Capricor Therapeutics, another twist for the rare disease drug.
    STAT · 5d agoRegulatoryNeutral↗ source
  • A trend that isn’t fading, ultra-processed baby foods
    An FDA advisory panel serves some peptide wins, a measles record, and more health news from Morning Rounds
    STAT · 5d agoRegulatoryNeutral↗ source
  • Opinion: How midwives like me can help fight medical misinformation
    “I have come to believe that changing a mind is rarely the point. Planting a seed is,” writes nurse-midwife Mara Evans.
    STAT · 5d agoRegulatoryNeutral↗ source
  • STAT+: As some medical schools embrace RFK Jr.’s nutrition push, they deftly sidestep MAHA priorities
    Even as some medical schools heed health secretary Robert F. Kennedy Jr.’s call for more nutrition education, several told STAT that they plan to steer clear of topics that aren’t…
    STAT · 5d agoRegulatoryNeutral↗ source
  • Opinion: E-bikes, e-scooters are a burgeoning pediatric public health emergency
    “Emergency departments across America report dramatic increases in serious injuries from e-bikes and e-scooters,” write CHOP’s J. Todd R. Lawrence and Madison A. Kesler.
    STAT · 5d agoRegulatoryNeutral↗ source