1. Reblozyl® fails to meet primary endpoint in myelofibrosis anemia but shows other benefits
On July 18, 2025, BMS announced that the Phase 3 INDEPENDENCE results of Reblozyl® (luspatercept-aamt) in combination with JAK inhibitor therapy for adults with myelofibrosis-associated anemia did not meet its primary endpoint of achieving red blood cell (RBC) transfusion independence within any 12-week period in the first 24 weeks of treatment. Myelofibrosis is a rare bone marrow cancer characterized by the scarring (fibrosis) of the bone marrow, which disrupts normal blood cell production. However, the trial showed clinically meaningful secondary benefits, including more patients achieved ≥50% reduction in transfusion burden (and by ≥4 RBC units) and more patients had hemoglobin increases ≥1 g/dL with transfusion independence for ≥12 weeks.
2. Anselamimab misses primary endpoint in AL amyloidosis Phase 3 trial but shows meaningful benefit in patient subgroup
On July 16, 2025, AstraZeneca announced Phase 3 Cardiac Amyloid Reaching for Extended Survival (CARES) results evaluating the effects of anselamimab in patients with Mayo stages IIIa and IIIb light chain (AL) amyloidosis. AL amyloidosis is caused by defective plasma cells in the bone marrow. In AL amyloidosis, abnormal light chain proteins misfold, aggregate and form amyloid fibrils that deposit in tissues and organs.
The primary endpoint was defined as of hierarchical combination of time to all-cause mortality (ACM) and frequency of cardiovascular hospitalisations (CVH). Based on the statistical analysis, the primary endpoint was not achieved. However, in a prespecified patient subgroup, anselamimab showed highly clinically meaningful improvement compared to the placebo arm in survival and cardiovascular-related hospitalisation.
3. Baxdrostat significantly lowers blood pressure in Phase 3 trial for treatment-resistant hypertension
On July 14, 2025, Astra Zeneca showed BaxHTN Phase III trial results investigating the effects of baxdrostat in uncontrolled or treatment-resistant hypertension patients receiving baxdrostat or placebo on top of the standard of care. The trial met its primary endpoint by achieving a significant reduction in systolic blood pressure (SBP) at both 2mg and 1mg doses. Additionally, all secondary endpoints were also achieved.
4. FDA approves prefilled syringe formulation of Shingrix to simplify shingles vaccination
On July 14, 2025, GSK announced that the U.S. FDA has approved Shingrix in a prefilled syringe (Recombinant Zoster Vaccine) for the prevention of shingles (herpes zoster). The current modality consists of two vials which require mixing before injection. The approval of the new prefilled syringe eliminates the need to reconstitute separate vials before administration and allows streamlining the vaccination process for healthcare providers.
5. Oveporexton meets all endpoints in Phase 3 narcolepsy type 1 trials
On July 14, 2025, Takeda announced all primary and secondary endpoints were met in two Phase 3 studies of oveporexton (TAK-861) in narcolepsy Type 1. The primary and secondary endpoints improved the objective and patient-reported improvements in wakefulness, excessive daytime sleepiness, cataplexy, ability to maintain attention, overall quality of life and daily life functions, achieving near normal ranges across the broad range of symptoms investigated. Oveporexton is an oral orexin receptor 2 (OX2R)-selective agonist, complementing the orexin deficiency.
6. MR-139 misses Phase 3 endpoint in blepharitis
On July 18, 2025, Viatris announced that its Phase 3 study results of pimecrolimus 0.3% ophthalmic ointment (MR-139) for blepharitis, an inflammation of the eyelids. The study failed to meet its primary endpoint of complete debris resolution after six weeks. The company is currently assessing next steps for the ophthalmology program, including recently reported positive Phase 3 results for MR-142 in keratorefractive visual disturbances and MR-141 for presbyopia.
7. Enhertu plus pertuzumab has been granted FDA Breakthrough Therapy Designation for first-line HER2+ breast cancer
On July 17 2025, Daiichi Sankyo announced that the FDA has granted Breakthrough Therapy Designation to Enhertu® (fam-trastuzumab deruxtecan-nxki) in combination with pertuzumab for the first-line treatment of adults with unresectable or metastatic HER2-positive breast cancer. The decision is based on the Phase 3 DESTINY-Breast09 trial data showing over three years of median progression-free survival, which is significantly longer compared to the standard of care. This marks Enhertu’s ninth BTD, further reinforcing its role in transforming treatment across multiple HER2-associated cancers.
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