Argo Biopharmaceutical Co. Ltd. and its RNAi work drew Novartis AG back to the table for a third time, as the companies entered a potential $5.2 billion deal involving cardiovascular-focused assets, including a right to first negotiation for BW-00112, an angiopoietin-like protein 3-targeting siRNA in phase II testing in severe hypertriglyceridemia.
Summit Therapeutics Inc.’s presentation for bispecific antibody ivonescimab in non-small-cell lung cancer could draw some extra attention at next month’s World Conference on Lung Cancer meeting thanks to promising overall survival data from partner Akeso Pharmaceuticals Inc.’s China study of the PD-1/VEGF candidate.
A few years after it was founded with the aim of taking RNA therapies to the next level, Arnatar Therapeutics Inc. emerged from stealth, disclosing a $52 million series A round raised in 2024 as well as U.S. FDA orphan and rare pediatric disease designations for ART-4, an antisense oligonucleotide candidate targeting the root cause of Alagille syndrome.
With a few tweaks to the protocol, Rocket Pharmaceuticals Inc.’s phase II trial testing RP-A501 in Danon disease is expected to resume following the lifting of the clinical hold, issued by the U.S. FDA in May in response to the death of a patient in the pivotal gene therapy study.
It looks like Biogen Inc.’s Nrf2 activator, Skyclarys (omaveloxolone), will maintain its status as the sole therapy approved for treating patients with Friedreich’s ataxia (FA), at least for now. The U.S. FDA asked for another “adequate and well-controlled study” in the complete response letter (CRL) issued to PTC Therapeutics Inc. for 15-lipoxygenase inhibitor vatiquinone. The agency said “substantial evidence of efficacy was not demonstrated.”
Adding another name to an impressive roster of partners assembled over the past few years, Skyhawk Therapeutics Inc. inked a neurology-focused deal with Merck KGaA aimed at discovering small-molecule RNA-targeted drugs that could be worth more than $2 billion.
Adding another name to an impressive roster of partners assembled over the past few years, Skyhawk Therapeutics Inc. inked a neurology-focused deal with Merck KGaA aimed at discovering small-molecule RNA-targeted drugs that could be worth more than $2 billion.
Boehringer Ingelheim GmbH’s zongertinib enters the market as the first oral HER2-targeted therapy for patients with non-small-cell lung cancer, following an accelerated approval by the U.S. FDA. Branded Hernexeos, the drug is cleared for use in adults with unresectable disease or whose tumors have HER2 tyrosine kinase domain activating mutations and who have received prior systemic therapy.
Jazz Pharmaceuticals plc is prepped and ready for launch following U.S. FDA accelerated approval of dordaviprone as the first systemic therapy for H3 K27M-mutant diffuse glioma. Branded Modeyso, the orally administered first-in-class imipridone was acquired via Jazz’s acquisition earlier this year of Chimerix Inc. in a deal valued at about $935 million.
