Defactinib

Verastem Oncology's NDA for avutometinib and defactinib combo, targeting recurrent KRAS mutant LGSOC, received FDA Priority Review with a PDUFA date of June 30, 2025. This potential first-ever FDA-approved treatment for the condition is based on Phase 2 RAMP 201 trial results, showing durable responses and tolerability.

The combination of avutometinib and defactinib showed a 28% ORR in recurrent low-grade serous ovarian cancer patients, with a 93% DCR. KRAS-mutated and wild-type patients had similar ORRs. The FDA chose the combination over monotherapy for further analysis. Verastem Oncology plans to use RAMP-201 and FRAME trial data for potential accelerated approval. The treatment was well-tolerated, with common AEs being mild to moderate.

Combination therapies targeting pancreatic cancer's extracellular stroma and membrane receptors show promise. Hyaluronidase and anti-PD-L1/PD-1 antibodies enhance tumor growth inhibition. IL-6 blockade with anti-PD-L1 improves survival. VDR ligands and TGF-β inhibitors combined with checkpoint inhibitors increase T cell infiltration. CD40 agonists and CXCR4 inhibitors with anti-PD-1 show potential in enhancing anti-tumor immunity. FAK and BTK inhibitors, alongside PARP inhibitors, are being explored for their roles in improving treatment efficacy.

Recent advancements in cancer treatment include psilocybin-assisted psychotherapy for mental health, pembrolizumab with radiotherapy and surgery for sarcoma, dordaviprone for glioma, and avutometinib with defactinib for ovarian cancer. Discussions on patient care strategies, EGFR-targeted therapies, and new drug approvals like zongertinib and tislelizumab highlight progress in oncology.

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2024-2025 saw significant advancements in cancer treatment, including FDA approvals for new therapies, promising clinical trial results, and discussions on treatment strategies for various cancers. Key developments include enasidenib for IDH2-mutated AML, ropeginterferon alfa-2b for essential thrombocythemia, and new drug applications for recurrent glioma and ovarian cancer treatments.