News

Clinical readouts, approvals, and setbacks, financing, dealmaking, platform progress, and reported research across biotech and biopharma, including the companies building AI into biology.

Thu, Oct 15Licensing deal AI drug discovery
AstraZeneca taps insitro's AI platform for liver and heart drug targets
AstraZeneca is paying insitro $40 million upfront, with up to $1.46 billion in potential milestones that bring the deal's headline value to $1.5 billion, to identify drug targets for metabolic liver disease and heart failure. AstraZeneca secures exclusive worldwide rights to advance any successful programs emerging from the alliance, which follows previous discovery partnerships insitro formed with Bristol Myers Squibb, Gilead, and Eli Lilly.
Fierce Biotech, also reported by ReutersEndpoints News (subscription required)
Thu, Oct 15AI drug discovery
Machine learning identifies brain-spliced microexons linked to autism
Researchers from the University of Toronto, the Sloan Kettering Institute, King's College London, and the Francis Crick Institute applied a computational tool named MicroExonator to over 100 terabytes of RNA sequencing data, uncovering more than 1,500 tiny protein-coding segments known as microexons. A trained machine learning model predicted which rare genetic variants disrupt the regulatory code that controls microexon inclusion in brain transcripts. The team found that predicted splicing-altering variants clustered heavily among autistic individuals who previously lacked an identified genetic contributor to their condition.
GEN
Thu, Oct 15Product launch AI drug discovery
Illumina bundles multiomics software and splice-predicting AI into new BioInsight platform
Illumina announced the launch of BioInsight, a unified software suite that combines its multiomics analytics, artificial intelligence tools, and biological datasets. The software includes SpliceA12, an updated genomic AI model that identified 17% more disease-linked splicing variants than competing tools in tests across Genomics England patient cohorts. Splicing alterations misdirect how cells piece together RNA transcripts, making them a major driver of rare inherited disorders, familial cancers, and drug response differences.
GEN

Thu, Oct 15Clinical update
Merck and Moderna plan broader tumor trials ahead of personalized mRNA vaccine filing
Following positive Phase 3 adjuvant melanoma results reported in August, the partners are preparing regulatory submissions for intismeran autogene given alongside Keytruda, an anti-PD-1 antibody.
STAT
Thu, Oct 15Regulatory decision
Revolution Medicines prepares solo European launch for oral RAS inhibitor
The European Medicines Agency's human medicines committee recommended marketing authorization for daraxonrasib, Revolution Medicines' oral RAS inhibitor for pancreatic cancer sold in the United States as Rasonque.
Reuters
Thu, Oct 15Single cell biology
MIT study ties APOE4 nerve-sheath damage to blocked Wnt signaling
Researchers at MIT's Picower Institute found that the Alzheimer's risk variant APOE4 impairs myelination by suppressing Wnt signaling in oligodendrocytes, the brain cells responsible for insulating nerve fibers.
GEN
Thu, Oct 15Financing
OrbiMed closes $3 billion across Asian and Western venture funds
Healthcare investment firm OrbiMed has collected $3 billion across two vehicles, comprising over $1.9 billion for its 10th flagship private investment fund focused on North America and Europe alongside more than $1 billion for its sixth Asia-dedicated partnership.
Fierce Biotech
Thu, Oct 15Trial results
Osivax's universal flu vaccine misses mid-stage trial target with 24% efficacy
France-based Osivax reported that its influenza A candidate OVX836 achieved a vaccine efficacy of 24% in a Phase 2b trial of 2,850 healthy European adults, missing the 50% bar required by the primary endpoint.
Fierce Biotech
Wed, Oct 14Acquisition
Armed Cell buys Lakefront's preclinical CAR-T portfolio for up to $238 million
Lakefront Biotherapeutics, formerly Galapagos, has sold its early-stage CAR-T cell therapy assets to newly formed Belgian biotech Armed Cell Therapeutics for €8 million upfront and up to $230 million in regulatory and commercial milestones.
BioPharma Dive, also reported by Endpoints News (subscription required)
Wed, Oct 14Financing
OMass Therapeutics raises $310 million to advance rare endocrine drug
Bain Capital Life Sciences, Forbion, and Novo Holdings co-led the Series C financing, backed by new investors Janus Henderson, SV Health Investors, and Jefferson Life Sciences alongside Alphabet-backed GV and Syncona.
Fierce Biotech, also reported by Endpoints News (subscription required)
Wed, Oct 14Regulatory decision
Compass Pathways plans cautious psilocybin launch ahead of FDA submission
Compass Pathways plans to complete a rolling FDA submission this quarter for COMP360, a synthetic psilocybin therapy for treatment-resistant depression, ahead of a potential launch early next year.
STAT
Wed, Oct 14Financing AI drug discovery
Iambic Therapeutics raises $150 million in Nasdaq debut
Iambic Therapeutics priced its initial public offering at $16 per share to raise $150 million, selling roughly 9.4 million shares ahead of trading on the Nasdaq under ticker IAM.
BioPharma Dive, also reported by Fierce Biotech
Wed, Oct 14Approval
FDA approves ImmunityBio's Anktiva for papillary bladder cancer
The FDA cleared ImmunityBio's Anktiva, an IL-15 superagonist immunotherapy, for patients with papillary-only non-muscle invasive bladder cancer.
Fierce Pharma, also reported by FDA
Wed, Oct 14Financing
Ultragenyx sells two FDA priority review vouchers for $413 million
Ultragenyx has monetized two rare pediatric disease priority review vouchers for a combined $413 million following two summer gene therapy approvals from the FDA.
Fierce Pharma
Wed, Oct 14Regulatory decision
FDA launches rare disease framework to adapt drug trials for small populations
The Food and Drug Administration launched its Rare Disease Framework Initiative to tailor trial requirements and evidence standards to small patient groups.
FDA, also reported by Endpoints News (subscription required)BioPharma Dive
Wed, Oct 14Trial results
Verastem reports 41% response rate in pancreatic cancer with oral KRAS G12D drug
In an early Phase 1/2 trial, Verastem Oncology reported an overall response rate of 41% among 22 patients with advanced pancreatic ductal adenocarcinoma treated with its daily pill VS-7375.
Fierce Biotech, also reported by BioPharma DiveEndpoints News (subscription required)STAT
Wed, Oct 14Discontinuation
Verdiva cancels Phase 2 trial of weekly oral GLP-1 weight-loss drug
Verdiva Bio has terminated a Phase 2 trial testing its once-weekly oral GLP-1 peptide, VRB-101, as a maintenance treatment for patients switching off injectable weight-loss drugs.
Fierce Biotech
Wed, Oct 14Clinical update
Biomea stock drops 30% after oral GLP-1 shows modest weight loss in Phase 1
Biomea Fusion reported early trial results for BMF-650, its oral small-molecule GLP-1 receptor agonist, showing mean weight reductions between 1.5% and 3.2% across four cohorts at Day 29.
Fierce Biotech, also reported by Endpoints News (subscription required)
Wed, Oct 14Clinical update
Arrowhead's subcutaneous RNA drug cuts brain tau by up to 85%
ARO-MAPT, an experimental RNA interference medicine delivered by simple injection under the skin, lowered tau protein levels by up to 85% in healthy volunteers.
BioPharma Dive, also reported by Endpoints News (subscription required)
Wed, Oct 14Acquisition Clinical AI
Agilent acquires digital pathology developer Proscia for up to $270 million
Agilent Technologies agreed to pay $240 million in cash upfront, plus up to $30 million in milestone payments, to acquire the digital pathology software developer Proscia.
Fierce Biotech
Tue, Oct 13Trial results
iOnctura reports 20.8-month median overall survival in eye cancer trial
The Merck KGaA spinout demonstrated a 20.8-month median overall survival in a 44-patient Phase 1 trial evaluating roginolisib, an oral small-molecule PI3K delta inhibitor, across advanced malignancies including second-line metastatic uveal melanoma.
Fierce Biotech
Tue, Oct 13Licensing deal
Roche strikes $1.2 billion licensing deal for Rona's RNAi obesity candidate
Roche has committed up to $100 million in upfront and near-term payments, as part of a deal worth up to nearly $1.2 billion including $1.06 billion in future milestones, to license an RNA interference candidate targeting ALK7 from China-based Rona Therapeutics.
Fierce Biotech, also reported by BioPharma Dive
Tue, Oct 13Licensing deal Venture round
Gilead licenses solid-tumor T cell engagers from Vir in $1.9 billion pact
Gilead Sciences committed $90 million upfront, comprising $65 million in cash and a $25 million equity investment at a 30% premium, to license three masked T-cell engager therapies engineered using Vir Biotechnology's PRO-XTEN platform.
Fierce Biotech, also reported by Endpoints News (subscription required)
Tue, Oct 13Licensing deal AI drug discovery
Zealand Pharma and Generate partner on AI-designed obesity drugs in $1.2 billion pact
Zealand Pharma and Flagship Pioneering spinout Generate Biomedicines agreed to collaborate on developing treatments for obesity using generative AI, according to Bloomberg News.
Reuters, also reported by Fierce Biotech
Tue, Oct 13Financing Merger
Velaris goes public in Alaunos reverse merger backed by $350 million
Fairmount Funds led a concurrent private placement of roughly $350 million alongside the merger with the former cell-therapy developer Alaunos Therapeutics.
BioPharma Dive
Tue, Oct 13Licensing deal
Pfizer signs $500 million Sec61 protein-degrader deal with Enodia
Enodia Therapeutics has signed a research collaboration and licensing pact worth up to $500 million in upfront cash, milestones, and royalties to develop small-molecule Sec61 inhibitors with Pfizer.
GEN, also reported by Fierce BiotechEndpoints News (subscription required)
Tue, Oct 13Venture round
Souffle Therapeutics raises $135 million for RNA medicines
Juvia Group led the Series B round to advance three siRNA programmes into early clinical testing.
Endpoints News (subscription required)
Tue, Oct 13Discontinuation
Syndax and Incyte drop Niktimvo in lung fibrosis after mid-stage failure
Syndax Pharmaceuticals and Incyte have halted development of their CSF-1R-blocking antibody Niktimvo (axatilimab) for idiopathic pulmonary fibrosis after a Phase 2 trial failed to meet its primary endpoint.
Fierce Pharma
Tue, Oct 13Regulatory setback
Neurocrine faces safety scrutiny following deaths tied to rare disease drug Vykat XR
Physicians and patient advocates raised alarm after Neurocrine Biosciences' Prader-Willi syndrome drug, Vykat XR, was linked to seven patient deaths and nearly 100 hospitalizations since its approval 18 months ago.
STAT (subscription required)
Tue, Oct 13Trial results
BioNTech mRNA cancer vaccine misses Phase 3 progression goal in head and neck cancer
The therapeutic mRNA cancer vaccine BNT113 failed to show a statistically significant improvement in progression-free survival when added to Merck's Keytruda in HPV16-positive head and neck cancer.
Fierce Biotech, also reported by Endpoints News (subscription required)BioPharma Dive

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