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Vorasidenib Shows Promise as First Targeted Therapy for IDH-Mutant Low-Grade Gliomas

Targeted TherapyDrug ApprovalOncology
  • Vorasidenib, the first targeted therapy developed specifically for brain cancer, more than doubled progression-free survival in patients with recurrent grade 2 glioma carrying IDH1/IDH2 mutations, extending the time without disease progression from 11.1 months to 27.7 months.
  • The international INDIGO trial involving 331 patients demonstrated that vorasidenib delayed the need for chemotherapy and radiation by nearly 17 months compared to placebo, with 85.6% of patients going 18 months before requiring next treatment.
  • The drug showed excellent tolerability with limited adverse effects, offering a new treatment option for younger patients typically in their 30s and 40s who face cognitive deficits from standard radiation and chemotherapy treatments.
  • Results from this phase 3 study, published in the New England Journal of Medicine and presented at ASCO, are expected to establish a new standard of care for IDH-mutant low-grade gliomas pending FDA approval.

Revumenib Shows Breakthrough Results in Advanced Acute Myeloid Leukemia Trial

Targeted Therapy
  • Revumenib, a novel menin inhibitor, achieved complete remission in 30% of heavily pretreated acute myeloid leukemia patients in the phase 1 AUGMENT-101 trial.
  • The drug works by disrupting the menin-MLL1 protein complex, reprogramming leukemia cells back into normal blood cells or causing them to die.
  • Twelve patients who achieved remission successfully underwent stem cell transplants, with nine remaining in remission at last analysis.
  • The treatment targets specific genetic alterations (KMT2A rearrangements and NPM1 mutations) found in 30-40% of AML cases, potentially benefiting up to 50% of all acute myeloid leukemias.

Revumenib Shows Promise in Phase 1 Trial for KMT2A-Rearranged and NPM1-Mutated Acute Leukemia

OncologyTargeted Therapy
  • Revumenib, a first-in-class menin inhibitor, demonstrated a 30% complete remission rate in heavily pretreated patients with KMT2A-rearranged or NPM1-mutated acute leukemia, with 78% achieving undetectable measurable residual disease.
  • The oral therapy works by disrupting the menin-KMT2A interaction, downregulating key leukemogenic genes and promoting differentiation of leukemic cells, addressing a critical unmet need for these poor-prognosis genetic subtypes.
  • While QT interval prolongation was the most common treatment-related adverse event (53%), the phase 1 trial established recommended phase 2 dosing with manageable safety profile, supporting further development of this targeted therapy.

Tagrisso Plus Savolitinib Shows Promising 49% Response Rate in EGFR-Mutated Lung Cancer with MET Resistance

Targeted TherapyOncologyPrecision Medicine
• Preliminary results from the SAVANNAH Phase II trial demonstrated that Tagrisso (osimertinib) plus savolitinib achieved a 49% objective response rate in EGFR-mutated NSCLC patients with high levels of MET overexpression who progressed on Tagrisso.
• MET was identified as the most common resistance biomarker in EGFR-mutated lung cancer, with 62% of patients screened showing MET overexpression and/or amplification after progression on Tagrisso.
• The combination therapy showed the highest response rate (52%) in patients with high MET levels who had not received prior chemotherapy, potentially offering a less toxic alternative to the current standard of chemotherapy after targeted therapy failure.

Cullinan Oncology and Taiho Pharmaceutical Forge $275M Strategic Collaboration for EGFR Inhibitor CLN-081/TAS6417

Targeted TherapyOncologyPrecision Medicine
  • Taiho Pharmaceutical will acquire Cullinan Pearl for $275 million upfront plus up to $130 million in regulatory milestones, gaining exclusive global rights to CLN-081/TAS6417 outside the U.S.
  • CLN-081/TAS6417 is an oral, irreversible EGFR inhibitor targeting exon 20 insertion mutations in non-small cell lung cancer, which affect approximately 2-3% of NSCLC patients globally.
  • The companies will jointly develop and co-commercialize the drug in the U.S. with equal profit sharing, while Taiho will commercialize in territories outside the U.S. and China.

HUTCHMED to Present Key Clinical Data for Multiple Cancer Therapies at ASCO 2025

Targeted TherapyOncology
  • HUTCHMED will showcase new data from several studies at the 2025 ASCO Annual Meeting, including promising results from the SACHI Phase III trial of savolitinib plus osimertinib in EGFR-mutant NSCLC with MET amplification.
  • The Phase I study of ranosidenib (HMPL-306), a dual IDH1/2 inhibitor, demonstrated favorable tolerability and 100% disease control rate in lower-grade glioma patients, showing potential for this novel targeted therapy.
  • Fruquintinib combination therapy showed clinically meaningful responses in advanced endometrial cancer patients with pMMR status, with an objective response rate of 37% in serous carcinoma subgroup and durable efficacy regardless of prior chemotherapy exposure.

Study on Savolitinib Combined With Osimertinib in Treatment of Advanced NSCLC With MET Amplification

NCT05015608Active, Not RecruitingPhase 3
Hutchison Medipharma Limited
Posted 11/22/2021

Lorlatinib Shows Promising Efficacy in ALK-Rearranged NSCLC with 21.8 Month Median PFS

Real World EvidenceOncologyTargeted Therapy
  • A real-world study of lorlatinib in ALK-rearranged non-small cell lung cancer demonstrated a median progression-free survival of 21.8 months, significantly higher than in previous trials.
  • The objective response rate was 43% with disease control achieved in 94% of patients, while 81% of patients with brain metastases showed objective response for intracranial lesions.
  • Patients experiencing adverse events, particularly hypercholesterolemia and edema, had significantly better outcomes than those without side effects, suggesting a potential correlation between drug exposure and efficacy.

Pertuzumab Regimen Shows Modest Survival Benefit in HER2-Positive Early Breast Cancer

Monoclonal AntibodyOncologyDrug ApprovalTargeted Therapy
• The APHINITY trial's 6-year analysis reveals that adjuvant pertuzumab with trastuzumab plus chemotherapy improved overall survival by 0.9% in HER2-positive early breast cancer patients, though not reaching statistical significance.
• Patients with node-positive disease showed the most significant benefit from the pertuzumab regimen, with a 4.5% absolute improvement in invasive disease-free survival at 6 years compared to placebo.
• The pertuzumab combination maintained a favorable cardiac safety profile with severe cardiac events occurring in less than 1% of patients, supporting its continued use in high-risk HER2-positive early breast cancer.

FDA Grants Breakthrough Therapy Designation to Novel Triplet Combination for BRAF-Mutant Metastatic Colorectal Cancer

Targeted Therapy
  • The FDA has granted breakthrough therapy designation to the triplet combination of encorafenib, binimetinib, and cetuximab for treating BRAF V600E-mutant metastatic colorectal cancer after 1-2 prior treatments.
  • The designation is based on promising data from the BEACON CRC trial showing a 48% overall response rate and 62% one-year overall survival rate.
  • This represents a significant advancement for patients with BRAF-mutant mCRC, who currently have no approved regimens specifically targeting their mutation.
  • The breakthrough designation will expedite development and regulatory review of this combination therapy for patients with limited treatment options.

Study of Encorafenib + Cetuximab Plus or Minus Binimetinib vs. Irinotecan/Cetuximab or Infusional 5-Fluorouracil (5-FU)/Folinic Acid (FA)/Irinotecan (FOLFIRI)/Cetuximab With a Safety Lead-in of Encorafenib + Binimetinib + Cetuximab in Patients With BRAF V600E-mutant Metastatic Colorectal Cancer

NCT02928224CompletedPhase 3
Pfizer
Posted 10/13/2016

D3Bio's Novel KRAS-G12C Inhibitor Shows Promising Results in Phase 1 Cancer Trial

OncologyPrecision MedicineTargeted Therapy
  • A multinational study led by Chinese University researchers found that D3S-001, a mainland-developed KRAS-G12C inhibitor, demonstrated significant efficacy with over 70% of patients experiencing tumor shrinkage or disappearance.
  • The novel compound from D3Bio inhibits KRAS-G12C mutations at a faster rate and potentially longer duration than existing treatments, according to Dr. Herbert Loong from CUHK's Department of Clinical Oncology.
  • Researchers are planning phase 3 trials with the goal of positioning D3S-001 as a first-line treatment option for patients with KRAS-G12C-driven cancers of the lung, pancreas, and colon.

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