Health and Pharma

05/09/2026

has granted designation to RZ-001, Rznomics’ investigational trans-splicing ribozyme-based for . The designation is supported by promising Phase 1b/2a safety and preliminary response data presented at .
https://healthandpharma.net/fda-rmat-rz001-rna-therapy-hepatocellular-carcinoma-rznomics

05/09/2026

has approved ( ) for adults with advanced, unresectable or metastatic NRG1 fusion+ after prior systemic therapy. In the Phase 2 eNRGy trial, the confirmed ORR was 36.8% among 19 evaluable patients, with responses lasting 2.8 to 12.9 months. The approval marks the first targeted treatment option for this rare, biomarker-defined cholangiocarcinoma population with high unmet need.
https://healthandpharma.net/fda-approves-bizengri-zenocutuzumab-cholangiocarcinoma

05/08/2026

has granted Fast Track Designation to -1-201 for unresectable or metastatic triple-negative after standard therapy failure or intolerance. The investigational TCR bispecific, now in Phase 1/2 testing, uses an engineered TCR and anti-CD3 domain to redirect T cells toward -selective epitopes in advanced .
https://healthandpharma.net/fda-fast-track-designation-rptr1-201-triple-breast-cancer

05/08/2026

has granted Fast Track designation to , also known as , for unresectable or metastatic cutaneous . Dubodencel is a first-in-class, patient-derived, double-loaded dendritic cell . It combines lysate and amplified tumor-derived mRNA prepared from a patient’s own tumor specimen, aiming to stimulate a broad anti-tumor immune response.
https://healthandpharma.net/fda-designation-dubodencel-doc1021-cutaneous-melanoma

05/07/2026

RNase H2 may represent a new therapeutic vulnerability in triple-negative .
A new study from MD Anderson, published in Cell Reports Medicine, found that cells may rely on RNase H2 to survive high levels of DNA replication stress. When researchers inhibited , cells accumulated more DNA damage, growth was suppressed, and innate immune signals were activated.
The findings also suggest possible synergy with ATR inhibitors, , and immune checkpoint blockade, supporting further investigation of combination strategies.
https://healthandpharma.net/rnase-h2-exposes-triple-breast-cancer

05/07/2026

has accepted sND Application for ( ) aiming to update its label with longer-term efficacy data in advanced ROS1+ non-small cell .
In updated TRUST-I data, TKI-naïve patients achieved a median duration of response of 49.7 months and median progression-free survival of 49.6 months, suggesting more than four years of sustained clinical benefit. In TRUST-II, TKI-pretreated patients had a median duration of response of 19.4 months.
https://healthandpharma.net/fda-taletrectinib-ibtrozi-snda-ros1-lung-cancer

05/07/2026

New PHERGain and PHERGain-2 data presented at strengthen the rationale for chemotherapy de-escalation in selected patients with early HER2+ . showed that about one-third of patients avoided , with nearly 90% remaining relapse-free five years after surgery. In PHERGain-2, 59.6% achieved pCR after chemotherapy-free - , while quality of life was largely preserved.
https://healthandpharma.net/esmo-breast26-phergain-chemotherapy-free-survival-breast-cancer

05/06/2026

A new pan-cancer analysis suggests that mutation may help identify tumors more likely to benefit from immune checkpoint blockade. In a study of 2,930 patients across 11 types, was associated with significantly improved overall survival after , with a 31% lower risk of death compared with ALK wild-type tumors.

Multi-omics analysis also showed that ALK-mutant tumors may have a more immune-active, “hot” tumor phenotype, including:
• Higher tumor mutation burden
• Increased neoantigen abundance
• Greater immune-cell infiltration
• Higher T-cell and B-cell receptor diversity
• Upregulated immune checkpoint expression, including PD-1, PD-L1, and CTLA-4

The findings are clinically interesting because they distinguish ALK mutation from ALK rearrangement, a context in which single-agent have shown limited activity in ALK-rearranged lung .
https://healthandpharma.net/cancer-alk-mutation-immunotherapy-checkpoint-inhibitors

05/04/2026

’s advisory committee has endorsed plus abiraterone and ADT as a treatment option with a favorable benefit-risk profile for patients with PTEN-deficient metastatic hormone-sensitive . In CAPItello-281, the Truqap combination reduced the risk of radiographic progression or death by 19% and extended median rPFS to 33.2 months versus 25.7 months.
If approved, it could become the first targeted treatment option for this high-risk prostate subtype.
Read the full article, link in comments:>

FDA’s advisory committee has endorsed Truqap plus abiraterone and ADT as a treatment option with a favorable benefit-risk profile for patients with PTEN-deficient metastatic HS prostate cancer. In CAPItello-281, the combination reduced radiographic progression or death by 19%

05/03/2026

The FDA has approved (vepdegestrant) for adults with ESR1-mutated ER+/HER2- advanced or metastatic after progression on at least one line of endocrine therapy.

This is a landmark decision for and drug development: is the first -approved therapy, introducing targeted protein degradation into routine care.

In the Phase 3 VERITAC-2 trial, vepdegestrant significantly improved outcomes versus fulvestrant in patients with ESR1-mutated disease:
✅ 43% reduction in risk of progression or death
✅ Median PFS: 5.0 vs 2.1 months
✅ ORR: 19% vs 4%
✅ Oral treatment option compared with intramuscular fulvestrant

The approval also includes Guardant360 CDx as a companion diagnostic to identify eligible patients with ESR1 mutations.
This approval offers a new targeted option for a clinically challenging population where endocrine resistance remains a major barrier.

Read more on our website.
https://healthandpharma.net/fda-vepdegestrant-veppanu-breast-cancer

05/02/2026

SUNRISE-II has begun evaluating , an ultrasound-guided HIFU system, in patients with unresectable . The Stanford-led feasibility trial will enroll 10 patients to assess safety, tolerability, and preliminary efficacy before first-line chemotherapy. The anesthesia-free approach may offer a less invasive local treatment option in a where five-year survival remains about 13%.
https://healthandpharma.net/suizenji-hifu-ablation-pancreatic-cancer