A rare joint tumor gets another approved pill

Five things to know

1. Must readFDA approved EMD Serono’s Pimjetmi (pimicotinib) for symptomatic tenosynovial giant cell tumor. In the Phase 3 MANEUVER, 56% responded at week 25 vs 3.2% on placebo, per FDA. It’s another pill aimed at CSF1R for this rare joint tumor; how long control lasts once dosing stops isn’t yet known.Match NCT05804045 registers objective response rate from baseline to week 25, by central read on RECIST 1.1, as the primary; the approval reports response at week 25. 94 enrolled; active, not recruiting; pr#
2. Must readPepGen reported splicing correction in myotonic dystrophy type 1. Mean correction was 46.5% in 6 patients on drug vs 6.8% on placebo pooled from earlier cohorts. The registered primary is safety, the numbers are small, and a US partial clinical hold still applies.Partial NCT06667453 registers safety (adverse events through day 112) as the only primary; splicing, hand-opening time, grip and walking are secondaries. The release reports one cohort of a trial listed at 24#
3. Must readAt ACR, two CD19 cell therapies reported lupus responses. Fate reported responses in its first 3 extrarenal lupus patients given no conditioning chemotherapy; Autolus reported complete renal responses in 6 of 9. Both readouts are early, open label and small.#
4. Know itMirum reports its HDV antibody brelovitug met the primary endpoint of the Phase 3 AZURE-4. 59% and 44% on two doses vs 0% on delayed treatment, as reported by RTTNews; we haven’t seen Mirum’s own release.#
5. Know itNovartis’s ianalumab held its Sjögren’s gains at two years. ESSDAI improved 7.5 points at week 108 in patients on drug throughout, per Novartis, which says an FDA decision is expected in 2026.#

Bright spot

In a 40-week randomized trial in Nature Medicine, people on tirzepatide who added resistance exercise lost the same weight but about 1.1 kg less lean mass, and their strength rose.