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Lung cancer – Phase 3 readout for exon 20 EGFR inhibitor sets up major FDA race
Taiho Oncology and Cullinan Therapeutics said zipalertinib has cleared a registrational Phase 3 endpoint in frontline EGFR exon 20 insertion lung cancer, using an early data check to halt...
Neurodegeneration – CAR-T regimens move toward α-synuclein targeting in Parkinson’s
A preclinical study reported a first CAR-Treg concept aimed at a pathogenic form of α-synuclein in Parkinson’s disease. In two mouse models, engineered regulatory T cells recognizing α-synuclein...
Genie therapy – Sangamo Fabry assets auction accelerates PTC and Lilly entry
PTC Therapeutics and Eli Lilly are preparing to buy pieces of Sangamo Therapeutics’ Fabry disease gene therapy business via a bankruptcy auction, with Deal terms reported as up to $264 million for...
Oncology – Intranasal NEO100 shows Phase 2a endpoint hit in IDH1-mutant glioma
NeOnc Technologies reported Phase 2a results for intranasal NEO100 in recurrent IDH1-mutant high-grade glioma, saying the study met its primary endpoint for six-month progression-free survival....
Rare disease antibodies – AbCellera raises $200M to fund ABCL635 and ABCL365 programs
AbCellera Biologics launched an underwritten public offering aimed at raising $200 million through a mix of common shares and pre-funded warrants. The financing is designed to fund continued...
Regulatory devices – FDA grants Breakthrough Device designation to Freenome’s SimpleScreen lung test
Freenome said the FDA has issued Breakthrough Device designation for its blood-based SimpleScreen lung cancer screening assay. The designation applies to adults ages 50 to 80 with at least a 20...
Capital markets – Epicrispr secures $90M Series C to advance gene-silencing therapy into pivotal trials
Epicrispr Biotechnologies announced an oversubscribed $90 million Series C round to advance EPI-321, its lead epigenetic gene-silencing therapy for facioscapulohumeral muscular dystrophy. The...
Biotech funding and innovation – Khartis Therapeutics emerges with $95M for oral TED IGF-1R inhibitor
Khartis Therapeutics officially emerged from stealth with a $50 million Series B that brought its total funding to $95 million, led by Forge Life Science Partners with Longwood Fund and Lilly Asia...
Clinical pipeline and drug safety – Neurocrine’s Vykat XR faces expert concerns tied to reported deaths and severe events
Experts in Prader-Willi syndrome have issued safety alerts for Neurocrine Biosciences’ Vykat XR, citing deaths and severe adverse events reported to the FDA’s adverse event monitoring systems. The...
Molecular glue and gene editing finance – Sangamo bankruptcy auction also brings Lilly purchase of technology stake
Sangamo’s bankruptcy proceedings are driving deal-making across the Fabry space, with PTC’s winning bid for the program and Eli Lilly receiving a technology purchase for $50 million. The reported...
Lung cancer detection assay validation and regulatory pathway
Lisen Imprinting Diagnostics is working with ChristianaCare to clinically validate its lung cancer detection assay, built around quantifying “loss of imprinting” in ambiguous biopsy cases. The...
EGFR-driven lung cancer: Phase 3 wins and competitive positioning
Taiho Oncology and Cullinan Therapeutics said their EGFR inhibitor zipalertinib cleared a Phase 3 registrational test in the front-line setting for a genetically defined lung cancer population....
FDA regulatory signals for screening: Freenome’s lung blood test
Freenome received FDA breakthrough device designation for its blood-based SimpleScreen lung cancer screening assay. The designation covers the assay’s proposed use in adults ages 50 to 80 with at...
Gene therapy deal-making: PTC to buy Sangamo Fabry program
PTC Therapeutics won an auction-stage deal to acquire portions of bankrupt Sangamo Therapeutics, including Sangamo’s Fabry disease program, for up to $264 million. The transaction structure...
Rare disease financing and late-stage execution: Vaderis HHT Phase 3
Vaderis Therapeutics closed an oversubscribed $152 million Series B financing and launched its global Phase III study HEROIC in hereditary hemorrhagic telangiectasia (HHT). The trial evaluates...
AI-enabled clinical operations ROI for oncology trials
Tufts Center for the Study of Drug Development (CSDD) and Medable reported an eNPV-based analysis indicating Medable’s AI clinical monitoring agent can generate net financial gains of up to $21...
Corporate restructuring and new metaboic pipeline funding: Vogenx IPO
Vogenx Inc. priced an $81.3 million IPO aimed at funding continued development of mizagliflozin, its SGLT1 inhibitor in metabolic disease. The company said the drug is in development for...
Diagnostic technology rollout: N6 Tec expands IconPCR distribution
N6 Tec expanded distribution for its IconPCR instruments and AutoNorm intelligent normalization technology across Europe, the Middle East, and Africa. The company said it extended its partnership...
Gene editing safety and oversight: new report of a DMD editing death in China
China’s gene-editing landscape faced another safety setback as Huidagene Therapeutics disclosed the death of a boy receiving HG-302 gene editing for Duchenne muscular dystrophy (DMD). The...
Neuroendocrine cancer regulation: ITM Isotope receives CRL for ITM-11
FDA issued a complete response letter (CRL) to ITM Isotope Technologies Munich SE for its radiotherapeutic ITM-11 (177Lu-edotreotide) intended to treat gastroenteropancreatic neuroendocrine tumors...