Defactinib

Tempus and Verastem Partner to Develop Companion Diagnostic for First FDA-Approved KRAS-Mutant LGSOC Treatment

2 days ago

• Tempus AI and Verastem Oncology have announced a collaboration to develop a companion diagnostic test for the recently FDA-approved combination therapy of avutometinib and defactinib for KRAS-mutated recurrent low-grade serous ovarian cancer. • The partnership builds on Tempus' confirmatory testing in Verastem's Phase 2 RAMP-201 clinical trial, with their FDA-approved xT CDx assay now being used as an investigational assay in the global Phase 3 RAMP-301 trial. • Low-grade serous ovarian cancer (LGSOC) is a rare form affecting younger women, accounting for 6-10% of serous ovarian cancers, with historically poor response to chemotherapy and limited treatment options.

Shionogi to Acquire Japan Tobacco's Pharmaceutical Subsidiaries for $1.1 Billion

13 days ago

• Shionogi & Co. plans to acquire Japan Tobacco's pharmaceutical subsidiaries Torii Pharmaceutical and Akros Pharma for ¥160 billion ($1.1 billion) to strengthen its global R&D capabilities. • The acquisition, negotiated since early 2024, will proceed through a tender offer beginning June 18 with a share price of ¥6,350 to acquire at least 11.89% of the minority stake. • This strategic move represents significant consolidation in the Japanese pharmaceutical sector and expands Shionogi's market presence amid increasing global competition.

FDA to Deploy AI Assistant Across All Centers by June 30 to Accelerate Scientific Reviews

14 days ago

• FDA Commissioner Martin Makary announced an agency-wide rollout of AI tools to assist with scientific reviews across all 11 FDA centers, with full deployment expected by June 30, 2025. • The AI assistant aims to handle repetitive aspects of regulatory reviews, allowing FDA scientists to focus on critical safety evaluations while potentially accelerating approval timelines for new therapies. • Implementation will be led by FDA's Chief AI Officer Jeremy Walsh and CDER's Strategic Programs Director Sridhar Mantha, with transparency measures including public release of user feedback and performance results.

Novel Drug Combination Overcomes Resistance in BRAF-Mutated Melanoma, Prevents Brain Metastasis

3 months ago

• Researchers have identified a promising combination therapy using FAK inhibitors with RAF-MEK clamps that effectively overcomes treatment resistance in BRAF V600E-mutated melanoma. • The dual-targeting approach not only treats existing brain metastases but also prevents melanoma cells from spreading to the brain, addressing a critical unmet need for patients with limited treatment options. • This oral combination therapy of defactinib (FAK inhibitor) and avutometinib (RAF-MEK inhibitor) could improve accessibility for melanoma patients in rural areas who struggle to access infusion-based immunotherapies.

Verastem Oncology to Present New LGSOC Treatment Data at SGO 2025 Annual Meeting

3 months ago

• Verastem Oncology will present additional analyses from the Phase 2 RAMP 201 trial evaluating avutometinib plus defactinib in recurrent low-grade serous ovarian cancer at SGO 2025, building on their FDA Priority Review submission. • The presentations include new subgroup analyses by KRAS mutational status, supporting the company's ongoing efforts to advance treatment options for RAS/MAPK pathway-driven cancers. • The company's NDA for the avutometinib-defactinib combination therapy has received Priority Review from the FDA with a PDUFA date of June 30, 2025, for KRAS mutant LGSOC patients.

Verastem Oncology Refinances Debt and Advances Avutometinib/Defactinib Combination

4 months ago

• Verastem Oncology secured a new credit facility of up to $150 million and a $7.5 million equity investment with Oberland Capital, refinancing existing debt with Oxford Finance. • A strategic collaboration with IQVIA aims to accelerate the commercial launch of the avutometinib and defactinib combination for recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC). • The FDA granted priority review with a PDUFA date of June 30, 2025, for the NDA of avutometinib and defactinib in recurrent KRAS mutant LGSOC after prior systemic therapy. • Verastem exercised its option to license VS-7375, a KRAS G12D inhibitor, from GenFleet Therapeutics, with preliminary Phase 1 data from China showing promise.

Verastem's Avutometinib and Defactinib Combination Receives FDA Priority Review for Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer

5 months ago

• The FDA has granted Priority Review to Verastem Oncology's NDA for avutometinib combined with defactinib for recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC). • The PDUFA target action date is set for June 30, 2025, with the FDA indicating that an advisory committee meeting is not currently planned. • The NDA is supported by Phase 2 RAMP 201 trial data, demonstrating substantial overall response rates and durable responses in patients with recurrent KRAS mutant LGSOC. • If approved, this combination therapy would be the first FDA-approved treatment specifically for adults with recurrent KRAS mutant LGSOC, addressing a significant unmet need.

DATROWAY® (Datopotamab Deruxtecan) Receives EU Approval for Previously Treated Metastatic HR+/HER2- Breast Cancer

6 months ago

• DATROWAY, a TROP2-directed antibody drug conjugate (ADC), has been approved in the European Union for treating adult patients with unresectable or metastatic HR+/HER2- breast cancer who have received endocrine therapy and at least one line of chemotherapy. • The approval is based on the TROPION-Breast01 phase 3 trial, which showed DATROWAY reduced the risk of disease progression or death by 37% compared to chemotherapy, with a median PFS of 6.9 months versus 4.9 months. • This marks the second ADC approved for breast cancer based on Daiichi Sankyo's DXd technology and the third medicine from their oncology pipeline to receive EU approval, highlighting their commitment to developing innovative cancer treatments.

Verastem Oncology Gears Up for Potential 2025 Launch of Avutometinib Plus Defactinib in KRAS Mutant LGSOC

6 months ago

• Verastem Oncology anticipates a potential FDA decision on avutometinib plus defactinib for recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC) by June 30, 2025. • The company plans for a mid-2025 U.S. commercial launch of the combination therapy in recurrent KRAS mutant LGSOC, pending FDA approval. • Clinical trials for avutometinib plus defactinib in metastatic pancreatic cancer and non-small cell lung cancer are progressing, with data updates expected in 2025. • Verastem is set to file an IND for VS-7375, a KRAS G12D inhibitor, in Q1 2025 and initiate a Phase 1/2a trial in the U.S. by mid-2025.

Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025

7 months ago

• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD. • EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration. • Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option. • Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.

Verastem Oncology Completes FDA Submission for Avutometinib Plus Defactinib in Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer

7 months ago

• Verastem Oncology has completed its New Drug Application (NDA) submission to the FDA for avutometinib plus defactinib to treat recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC). • The NDA is based on Phase 2 RAMP 201 trial data showing a 44% overall response rate and a median progression-free survival of 22 months in patients with KRAS mutant LGSOC. • The FDA previously granted Breakthrough Therapy Designation and Orphan Drug Designation to the combination therapy for LGSOC, and a decision is expected by mid-2025. • If approved, avutometinib plus defactinib would be the first FDA-approved treatment specifically for recurrent KRAS mutant LGSOC, addressing a significant unmet need.

Drug Development Updates