Tagged News
Revumenib Shows Promise in Phase 1 Trial for KMT2A-Rearranged and NPM1-Mutated Acute Leukemia
• 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.
BriaCell Advances Pivotal Study for Bria-IMT in Metastatic Breast Cancer Following Positive FDA Feedback
• BriaCell Therapeutics has received positive FDA feedback for its pivotal study of Bria-IMT in combination with a checkpoint inhibitor for advanced metastatic breast cancer, potentially accelerating commercialization.
• The FDA has agreed on the eligible patient population—breast cancer patients who have failed available approved therapies—and the primary endpoint of survival improvement compared to physician's choice of treatment.
• BriaCell is also preparing to launch its Bria-OTS personalized treatment program, which matches patients' HLA type with pre-manufactured cells, with dosing expected to begin in the first half of 2023.
Tagrisso Plus Savolitinib Shows Promising 49% Response Rate in EGFR-Mutated Lung Cancer with MET Resistance
• 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
• 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
• 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.
Zanidatamab Plus Chemotherapy Shows Promising Results in Heavily Pretreated HER2-Positive Breast Cancer
• Zanidatamab, a novel HER2-targeted bispecific antibody, demonstrated a 36.4% objective response rate and 86.4% disease control rate when combined with chemotherapy in heavily pretreated HER2-positive breast cancer patients.
• The combination therapy showed a median progression-free survival of 7.3 months, with 42% of patients still on treatment at data cutoff, offering new hope for patients who have progressed after multiple HER2-targeted therapies.
• The treatment was well-tolerated with manageable side effects, primarily low-grade diarrhea, supporting further investigation as a potential new therapeutic option for advanced HER2-positive breast cancer.
Highlighted Clinical Trials:
Jazz Pharmaceuticals
Posted 9/30/2016
Lorlatinib Shows Promising Efficacy in ALK-Rearranged NSCLC with 21.8 Month Median PFS
• 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
• 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.
Novartis's Kymriah: Breakthrough CAR-T Therapy Priced at $475,000 Sparks Value Debate
• Novartis's Kymriah, the first FDA-approved CAR-T cell therapy for pediatric acute lymphoblastic leukemia, demonstrates an impressive 83% remission rate in patients who failed traditional treatments.
• The therapy's $475,000 price tag has ignited debate among healthcare experts, with some defending it as comparable to bone marrow transplants while others argue it's excessive despite its breakthrough status.
• Novartis has implemented innovative value-based pricing models, including outcome-based contracts where payment is contingent upon patient response and indication-specific pricing for future approvals.
D3Bio's Novel KRAS-G12C Inhibitor Shows Promising Results in Phase 1 Cancer Trial
• 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.