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FDA approvals & regulatory action
The FDA granted accelerated approval to Bristol Myers Squibb’s iberdomide (marketed as Zenbexus) for multiple myeloma patients whose disease has returned or stopped responding to treatment, the...
Regulatory policy – user fees
The FDA outlined changes for the next five-year prescription drug user fee program (PDUFA VIII), including new incentives for domestic manufacturers, faster review timelines for some application...
Bankruptcy M&A in gene therapy
PTC Therapeutics won a bankruptcy auction for Sangamo’s Fabry disease gene therapy program, with the deal structured around a $111 million upfront payment and additional contingent milestones tied...
Oncology trial readouts and phase transitions
Taiho Oncology and Cullinan Therapeutics reported a second Phase 3 win for zipalertinib, clearing the registrational trial’s front-line efficacy goal for EGFR exon 20 insertion–positive lung...
Company finance & fundraising
Silence Therapeutics priced an upsized public offering expected to raise about $175 million to support its short interfering RNA pipeline, including divesiran after a “best-case” outcome in a...
Venture funding for new biotechs
Khartis Therapeutics emerged from stealth with $95 million in total funding to pursue an oral treatment for thyroid eye disease (TED), aiming to compete with established infusion and biologic...
Digital health & AI in clinical operations
A new Tufts Center for the Study of Drug Development analysis concludes that an agentic AI clinical monitoring tool can deliver net financial gains up to $21 million per drug development program...
New diagnostics & biomarker tools
Freenome received FDA breakthrough device designation for its SimpleScreen lung cancer blood test, a multiomic assay combining machine-learning classification with DNA methylation and proteomics....
Clinical development in rare disease
Vaderis Therapeutics closed an oversubscribed Series B financing and simultaneously initiated a global Phase 3 trial, HEROIC, for engasertib (AKT inhibitor) in hereditary hemorrhagic...
Metabolic & cardiovascular drugs – health economics pushback
ICER said AstraZeneca’s newly approved hypertension drug Baxfendy does not meet its cost-effectiveness threshold at current list pricing, according to a draft report. ICER modeled Baxfendy at a...
FDA approvals and removals in oncology and immunology
The FDA granted accelerated approval to Bristol Myers Squibb’s oral iberdomide (marketed as Zenbexus) for multiple myeloma that has returned or become resistant, expanding the agency’s use of...
CAR-T and next-gen cell therapies expand beyond the autologous model
Legend Biotech reported its first profitable quarter, driven by strong demand for its multiple myeloma CAR-T therapy Carvykti, while signaling intensifying competitive pressure from off-the-shelf...
Radiation oncology guideline update for pancreatic cancer
ASTRO updated its radiation therapy guidance for adult pancreatic cancer, replacing the 2019 version and publishing the revised guideline in Practical Radiation Oncology. The change is expected to...
Phase 3 readouts reshape lung cancer competition at EGFR exon 20
Taiho Oncology and Cullinan Therapeutics said their Phase 3 trial of zipalertinib plus chemotherapy cleared a registrational goal in frontline EGFR exon 20 insertion–positive lung cancer. The...
Cyber-physical security finds a path from proximity to onboard aircraft electronics
Researchers challenged a common assumption in aircraft cybersecurity by demonstrating that brief physical access to a Boeing 737’s electronics bay can be leveraged as a potential cybersecurity...
Gene therapy deals after Sangamo bankruptcy auction
PTC Therapeutics won a bankruptcy auction for Sangamo’s Fabry disease gene therapy candidate, taking the program forward with an agreement framed as a near-BLA path. The deal structure combines...
Trial results and trial design signals in rare disease and beyond
Freenome said the FDA granted Breakthrough Device Designation for its SimpleScreen lung cancer screening blood test, positioning the assay for a defined population that is not already receiving...
Clinical development financing and capital markets for RNA and antibodies
Silence Therapeutics priced an upsized public offering tied to its short interfering RNA pipeline after reporting a Phase 2 “best-case” outcome for divesiran in a rare leukemia. The financing...
Venture funding and new startups targeting autoimmune and specialty indications
Khartis emerged from stealth with a $95 million total funding haul, including a $50 million Series B, to advance an oral IGF-1R inhibitor aimed at thyroid eye disease. The company tied its TED...
Regulatory and policy framework shift: FDA user-fee reauthorization
The FDA’s next five-year Prescription Drug User Fee Act (PDUFA VIII) reauthorization includes “America First” provisions alongside faster reviews for some application types and updated ways for...