Merck KGaA Will Pay $1.3 Billion for Silence Therapeutics, Buying Its Way Into Haematology With One Phase 3-Ready siRNA
Merck KGaA paid an 82% premium for one asset and a new therapy area, pricing divesiran’s Phase 2 effect as close to settled.
RNA
Novartis $7.8 Billion Abogen Bet Rests on Efficacy Data From Three Patients
Up to $7.8 billion for an mRNA engager with efficacy data from three patients, 32 days after Novartis halted its CAR-T.
NICE Backs Tryngolza for FCS, Giving Sobi a Monthly Successor to Its Own Waylivra
Sobi is moving its FCS franchise to a less frequent, better-tolerated drug, on broader NICE terms than the one it replaces.
Roche Claims Best-in-Class Potential for Sefaxersen Without Disclosing How Much It Cut Proteinuria
Sefaxersen hits the same target as Novartis’s Fabhalta. Roche withheld the one number that would show which drug does more.
A Custom ALS Drug Took Three Years to Build and One Year to Normalise One Man’s Neurofilament
Built in about three years for one mutation, it normalised a biomarker in one man. A larger cohort sits outside the paper.
The First Phase 3 Win for an mRNA Cancer Therapy Arrives Without Its Numbers
The first Phase 3 win for an mRNA cancer therapy. Statistical significance was reported, effect size was not, and overall survival is still open.
RiboX’s China Trial Data Bought It an Accelerated Dose Titration and a Subcutaneous Route
RiboX filed its Chinese investigator-initiated data with the IND. FDA granted faster escalation and a subcutaneous route on the strength of it.
One Trial Miss Cut AstraZeneca’s Cushion Above $80bn to $800m
A drug worth $1.9bn of 2030 revenue cost AstraZeneca £19bn in a day, leaving roughly $800m of headroom above its $80bn goal with the risky readouts still ahead.