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FDA doubles down: agency demands sham‑controlled trial for uniQure’s Huntington’s gene therapy
The FDA reaffirmed its reviewers’ decision requiring a randomized, sham‑controlled study for uniQure’s AMT‑130 Huntington’s gene therapy, rejecting the company’s objections that such a trial would...
Amylin setback: Roche‑Zealand obesity drug falls short of expectations
Mid‑stage data for petrelintide, an amylin analogue developed by Zealand Pharma with Roche, produced a mean placebo‑controlled weight loss of about 9.0% (10.7% absolute at 42 weeks), below...
FDA speeds Tecvayli‑Darzalex approval: rare fast‑track nod for myeloma combo
The FDA granted rapid clearance to Johnson & Johnson’s Tecvayli (pirtobrutinib) combined with Darzalex Faspro (daratumumab) for relapsed or refractory multiple myeloma under a priority‑review...
Kestrel lands IND for oral pan‑KRAS candidate – first‑in‑human study planned
Kestrel Therapeutics received IND clearance from the FDA for KST‑6051, an oral small‑molecule pan‑KRAS inhibitor targeting KRAS‑driven cancers, clearing the way for a first‑in‑human Phase I FALCON...
Tenaya and Alnylam strike $1B+ deal to mine genetic heart‑disease targets
Tenaya Therapeutics and Alnylam Pharmaceuticals entered a research collaboration in which Tenaya will deliver up to 15 novel genetic targets for cardiovascular disease discovery. The agreement...
FDA places partial hold on PepGen’s DM1 mid‑stage trial over preclinical signal
The FDA issued a partial clinical hold on PepGen’s Freedom2‑DM1 Phase II multiple‑ascending‑dose trial in myotonic dystrophy type 1 (DM1), citing concerns in previously submitted preclinical...
Illumina expands Alliance for Genomic Discovery with proteomics add‑on
Illumina announced it will add 50,000 genomes with paired proteomic data to the Alliance for Genomic Discovery (AGD), expanding the consortium’s multi‑omics resource for drug discovery. DeCode...
Perimeter’s Claire wins FDA PMA: AI imaging cleared for breast‑conserving surgery
Perimeter Medical Imaging AI secured FDA premarket approval for Claire, an AI‑enabled wide‑field optical coherence tomography device that provides real‑time imaging of tumor margins during...
FDA’s vaccines chief to depart in April – Prasad exits
Vinay Prasad will leave his role as director of the FDA’s Center for Biologics Evaluation and Research at the end of April, the agency confirmed in multiple reports. Prasad’s tenure included...
FDA doubles down on sham-control for uniQure’s Huntington’s gene therapy
A senior FDA official defended the agency’s reviewers and insisted uniQure must conduct a randomized sham‑controlled trial for AMT‑130, rejecting the company’s claims that the requirement was...
Servier to buy Day One for $2.5B – gains pediatric glioma asset
Servier signed a definitive agreement to acquire Day One Biopharmaceuticals for approximately $2.5 billion in cash, giving the French drugmaker access to Ojemda (tovorafenib), an FDA‑approved...
Petrelintide underwhelms investors – amylin rival trails Lilly’s GLP-1s
Phase 2 data for petrelintide, Roche‑partnered and developed by Zealand Pharma, showed mean placebo‑corrected weight loss of about 9% (10.7% absolute) at 42 weeks—below analyst expectations and...
FDA fast‑tracks J&J myeloma combo under national priority pilot
The FDA granted rapid approval to Johnson & Johnson’s Tecvayli plus Darzalex regimen for previously treated multiple myeloma under the Commissioner’s National Priority Voucher pilot, completing...
Alnylam pays Tenaya to mine up to 15 genetic cardio targets — deal tops $1B
Alnylam and Tenaya Therapeutics entered a research collaboration in which Tenaya will deliver up to 15 novel genetic targets for cardiovascular disease; Alnylam paid $10 million up front and the...
Ketone metabolite BHB sharpens CAR‑T potency in preclinical work; human trials launched
Researchers at the Arc Institute and Stanford reported that supplementation with β‑hydroxybutyrate (BHB) enhanced metabolic fitness and antitumor activity of CAR‑T cells in mouse models, and human...
FDA places partial hold on PepGen’s DM1 Phase 2 trial after re‑examining preclinical data
The FDA put a partial clinical hold on PepGen’s Freedom2‑DM1 Phase 2 study, citing concerns about previously submitted preclinical pharmacology and toxicology data in mice; the agency said the...
Illumina expands Alliance for Genomic Discovery with 50,000 proteomes
Illumina announced it will add 50,000 genomes paired with proteomic data to the Alliance for Genomic Discovery (AGD), broadening the alliance’s dataset and enabling integrated...
Perimeter wins FDA approval for Claire: AI‑guided margin imaging for breast conservation
Perimeter Medical Imaging AI secured FDA premarket approval for Claire, an AI‑powered wide‑field optical coherence tomography (OCT) device that provides surgeons with real‑time, high‑resolution...
Servier expands rare-cancer push: to buy Day One for $2.5B
Servier announced a definitive agreement to acquire Day One Biopharmaceuticals for roughly $2.5 billion, securing the pediatric glioma drug Ojemda and multiple investigational oncology programs....
FDA doubles down: demands sham-controlled trial for uniQure’s Huntington’s gene therapy
A senior FDA official publicly defended the agency’s reviewers and reiterated that uniQure must conduct a randomized sham‑controlled study to support approval of AMT‑130, its intracranial gene...