Disease

Six-month interim data from 17 patients showed BMI reductions, fat mass loss and improved hyperphagia scores, reinforcing the case for Phase 3.

Shattuck Labs priced a $75 million public offering of shares and pre-funded warrants, with proceeds set to advance its DR3 antibody pipeline.

Belite Bio has filed its full NDA for tinlarebant, a once-daily oral therapy that would be the first approved treatment for STGD1.

Bezuclastinib hit a 65% objective response rate in advanced systemic mastocytosis; Cogent plans to file its NDA in June 2026.

DISC-0974 hit major response rates of 50–64% across transfusion subgroups in myelofibrosis, with End of Phase 2 FDA talks targeted for late 2026.

Allogeneic anti-CD7 CAR-T candidate CTD402 posted strong remission rates in adult and paediatric T-cell leukaemia patients across two EHA2026 poster presentations.

Post-hoc MATTERHORN data presented at EHA 2026 show roxadustat achieved transfusion independence in both RS-positive and RS-negative lower-risk MDS patients.

RemIND trial data presented at EAACI 2026 show remibrutinib doubled complete response rates versus placebo in symptomatic dermographism, cold urticaria and cholinergic

The accelerated approval covers children aged eight to 17 recently diagnosed with stage 3 T1D, making Tzield the first disease-modifying therapy in this setting.

Rafael Holdings hit last-patient-last-visit in its pivotal TransportNPC trial and expects to file an NDA for Trappsol Cyclo in the second half

Five datasets presented at EHA 2026 show revumenib achieving high remission and MRD-negativity rates across multiple acute leukaemia subtypes and treatment settings.

Palopegteriparatide sustained multi-organ benefits over five years in hypoparathyroidism, with 82% of patients meeting the multi-component response endpoint.

Updated CaMMouflage phase 1 data show deep, durable responses from a single dose in heavily pretreated, BCMA-naïve multiple myeloma patients.

Early data from 15 heavily pretreated patients show robust B-cell depletion and durable platelet responses, with no serious adverse events reported.

The FDA accepted Roche's sBLA for Tecentriq plus chemotherapy in dMMR/MSI-H stage III colon cancer, with a decision expected by 9 October

ESLIM-02 Phase III data showed a 66% durable response rate versus 15% for placebo, supporting a priority-review NDA filed with China's NMPA.

The MAPKeeper 301 trial will test atebimetinib plus modified chemotherapy in first-line metastatic PDAC, with topline overall survival data expected in mid-2028.

KOMET-007 Phase 1/2 data show ziftomenib plus 7+3 chemotherapy delivered high remission rates and durable responses in newly diagnosed NPM1-mutant AML.

Novartis says its AOC therapy del-brax hit biomarker endpoints in the FORTITUDE study, with Phase III now enrolling 200 FSHD patients.

The UK becomes the first European country to approve a once-daily GLP-1 weight-loss tablet, based on Phase 3 data showing ~14% weight