FDA approves a drug that may spare rectal surgery

Five things to know

1. Must readFDA gave Jemperli accelerated approval for rectal cancer that may not need surgery.It covers untreated stage II and III dMMR or MSI-H rectal cancer. In the single-arm AZUR-1 trial, 47% of 116 centrally reviewed patients had a complete response lasting 12 months; lasting benefit still has to be confirmed.Partial AZUR-1 (NCT05723562) registers sustained complete clinical response at 12 months by central review, at 18 months, N=154. FDA's notice reports that measure in 116 evaluable patients (47%); the press an#
2. Must readMerck KGaA agreed to buy Silence Therapeutics for about $1.3B in cash.The price is $20.00 per ADS, a 130% premium, for a company led by divesiran, a Phase 2 siRNA in polycythemia vera. One deal is a price point for RNAi, not a trend.#
3. Must readRoche licensed Hansoh's ADCs for $400M upfront.Hansoh gets $110M more on the first anniversary and potentially more than $15B in milestones for programs that are still preclinical; it keeps Greater China.#
4. Know itRapport's RAP-219 beat placebo in acute bipolar mania, narrowly.Mania scores improved 3.1 points more than placebo at week 3 in 253 patients (p=0.045), with more agitation on drug, Rapport says.Match NCT07046494 registers YMRS change from baseline to week 3, N=253; the release reports that endpoint at that timepoint in 253 patients.#
5. Know itEyePoint's wet AMD implant missed; it is cutting about 53% of staff.LUCIA didn't show non-inferiority on vision against aflibercept, and the release gives no visual acuity numbers.Partial NCT06683742 registers average change in BCVA at weeks 52 and 56, the endpoint the release reports as missed. The registry lists 400 participants; the release says 475.#

Bright spot

A French team profiled 423 newly diagnosed myeloma tumors in Nature Medicine and built a model that predicted, with an area under the curve of 0.77, which patients would clear detectable disease after four-drug induction. It still needs testing in practice.