お知らせ • Jun 24

Verastem Oncology Demonstrates Clinical Activity of Vs-7375 in Target-D 101 Phase 1/2 Clinical Trial in Patients with Advanced Kras G12d-Mutated Solid Tumors

Verastem Oncology announced positive preliminary data from the ongoing TARGET-D 101 Phase 1/2 clinical trial evaluating VS-7375, an investigational oral KRAS G12D (ON/OFF) inhibitor, with best-in-class potential, in patients with advanced KRAS G12D-mutated solid tumors. The data demonstrate encouraging clinical activity along with a favorable safety and tolerability profile across multiple dose levels and tumor types, including metastatic pancreatic ductal carcinoma (mPDAC), metastatic colorectal cancer (mCRC), and advanced non-small cell lung cancer (NSCLC). In the TARGET-D 101 trial, dose-escalation is ongoing at 1200 mg once daily (QD). In updated pharmacokinetic (PK) data, the 900 mg QD dose continues to achieve target plasma levels of VS-7375 and provides clear separation from the 600 mg QD dose. VS-7375 demonstrated anti-tumor activity at multiple dose levels, including 400 mg QD, 600 mg QD and 900 mg QD both as monotherapy and in combination with anti-EGFR therapy, across multiple KRAS G12D-driven tumors, including mPDAC, mCRC and advanced NSCLC. In addition, patient follow-up continues to mature across both monotherapy and combination cohorts. Promising clinical activity observed at 900 mg QD monotherapy in previously treated mPDAC, with evidence of dose-dependent anti-tumor activity between 600 mg QD and 900 mg QD. 93% (13/14) of heavily pretreated (2L-4L) patients with mPDAC receiving 900 mg QD monotherapy achieved greater than 50% reduction in the tumor marker CA19-9. All 14 evaluable patients had elevated baseline CA19-9 levels (>37 U/mL) and at least one scheduled on-treatment CA19-9 assessment. All patients remain on treatment. Preliminary data suggest the combination with the anti-EGFR antibody cetuximab is associated with deeper and more rapid tumor reductions, even at a subtherapeutic VS-7375 dose of 400 mg QD. Combination cohorts in previously treated mPDAC demonstrate good combinability with standard-of-care chemotherapy, gemcitabine plus Nab-paclitaxel (Gem/NabP). VS-7375 600 mg QD in combination with full-dose Gem/NabP has been DLT-cleared, and enrollment is ongoing with 900 mg QD plus full-dose Gem/NabP. Among patients with mPDAC who had received at least one prior therapy (2L+), 7 of 20+ patients enrolled at the 600 mg QD dose level and 1 of 20+ patients enrolled at the 900 mg QD dose level had completed at least six months of follow-up. In the mCRC cohort, promising preliminary efficacy was observed with full dose cetuximab at both the 600 mg QD and 900 mg QD dose levels of VS-7375. VS-7375 900 mg QD in combination with full dose cetuximab was DLT-cleared in May 2026, with no overlapping toxicities observed to date. Additional patients will be enrolled at this dose level in the TARGET-D 203 Phase 2 registration-directed mCRC trial. Follow-up remains early in the mCRC cohort, with no patients out of 20+ at 600 mg QD in combination with full dose cetuximab having more than six months of follow-up. In the advanced NSCLC cohort, promising preliminary efficacy was observed at 600 mg QD monotherapy. Follow-up remains early in the NSCLC cohort, with only one out of 20+ patients at 600 mg QD having more than six months of follow-up. The 900 mg QD dose level in advanced NSCLC will be studied in the registration-directed TARGET-D 202 Phase 2 study. Across monotherapy and combination cohorts in TARGET-D 101, VS-7375 continued to demonstrate a favorable and manageable safety profile, consistent with prior observations and supported by increasing patient exposure and longer follow-up. As of the June 12, 2026 data cutoff, VS-7375 monotherapy has demonstrated a favorable and manageable safety profile at both the 600 mg QD (n=57) and 900 mg QD (n=25) dose levels. Treatment-related adverse events (TRAEs) were primarily low-grade nausea, vomiting and diarrhea, which generally diminished over time, with substantially reduced incidence after cycle 1 dosing. The vast majority of the gastrointestinal (GI) side effects were effectively managed with standard supportive care measures, with only 1 reported Grade 3 case of nausea at the 900 mg QD dose that resolved in 4 days after optimization of anti-emetic agents. A very low frequency of rash was observed in either the 600 mg QD or 900 mg QD dose level and no rash above Grade 1. TRAEs occurring in more than one patient were largely confined to the first treatment cycle and attenuated substantially thereafter among patients with at least 29 days of follow-up receiving VS-7375 at both the 600 mg QD (n=51) and 900 mg QD (n=22) dose levels. No unexpected adverse events (AEs) were observed, and rates of Grade 3 AEs remained low. Importantly, no clinically meaningful cytopenias or liver function abnormalities were reported at either the 600 mg QD or 900 mg QD dose level. The limited dose-response relationship observed for gastrointestinal AEs is consistent with a localized irritant effect rather than systemic toxicity. Emerging longer-term follow-up data are encouraging, with no clinically significant cumulative toxicities observed to date. Verastem and Erasca, Inc. announced their intent to enter into an agreement to evaluate VS-7375, Verastem’s potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, in combination with ERAS-0015, Erasca’s potential best-in-class oral pan-RAS molecular glue, across KRAS G12D mutant solid tumor models.

お知らせ • Dec 31

Verastem Oncology Announces FDA Acceptance and Priority Review of New Drug Application for Avutometinib in Combination with Defactinib for the Treatment of Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer

Verastem Oncology announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) under the accelerated approval pathway for avutometinib, an oral RAF/MEK clamp, in combination with defactinib, an oral FAK inhibitor, for the treatment of adult patients with recurrent low-grade serous ovarian cancer (LGSOC), who received at least one prior systemic therapy and have a KRAS mutation. The NDA, which was completed in October 2024, has been granted Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of June 30, 2025. In addition, the FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application. There are currently no FDA-approved treatments specifically for LGSOC, a rare and distinct ovarian cancer that differs from high-grade serous ovarian cancer in both its biology and how it responds to treatment. Priority Review is granted by the FDA for treatments that offer, if approved, significant improvements over available options or that provide a treatment option where no adequate or approved therapy currently exists. The filing was based on a primary analysis of the Phase 2 RAMP 201 clinical trial that evaluated the combination of avutometinib and defactinib in patients with recurrent LGSOC. The results were presented in an oral presentation at the International Gynecologic Cancer Society (IGCS) Annual Global Meeting in October 2024 and demonstrated that the combination of avutometinib plus defactinib resulted in a substantial overall response rate confirmed by blinded independent central review, with responses that were typically durable, and that the combination was generally well-tolerated in patients with recurrent KRAS mutant LGSOC. The NDA also includes supportive data from the FRAME Phase 1 trial, the first study conducted with the combination therapy in recurrent LGSOC. The Company is currently enrolling patients with recurrent LGSOC regardless of KRAS mutation status for RAMP 301, an international Phase 3 trial, which will serve as a confirmatory study for the initial indication and has the potential to support an expanded indication regardless of KRAS mutation status. RAMP 201 (ENGOTov60/GOG3052) (NCT04625270) is an adaptive, two-part multicenter, parallel cohort, randomized, open-label Phase 2 registration-directed trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). The first part of the study (Part A) determined the selection of the go-forward regimen, which was the combination of avutometinib and defactinib versus avutometinib alone, based on overall response rates. The expansion phases of the trial (Parts B and C) are evaluating the safety and efficacy of the go-forward regimen of avutometinib 3.2 mg twice weekly and defactinib 200 mg twice daily. The Part D portion of the trial is evaluating a low dose of avutometinib in combination with defactinib to inform individualized dose reduction. Avutometinib is an oral RAF/MEK clamp that potentially inhibits MEK1/2 kinase activities and induces inactive complexes of MEK with ARAF, BRAF, and CRAF, potentially creating a more complete and durable anti-tumor response through maximal RAS/MAPK pathway inhibition. In contrast to currently available MEK-only inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of the MEK-only inhibitors. Defactinib is an oral, selective inhibitor of focal adhesion kinase (FAK) and proline-rich tyrosine kinase-2 (Pyk2), the two members of the focal adhesion kinase family of non-receptor protein tyrosine kinases. FAK and Pyk2 integrate signals from integrin and growth factor receptors to regulate cell proliferation, survival, migration, and invasion. FAK activation has been shown to mediate resistance to multiple anti-cancer agents, including RAF and MEK inhibitors. Verastem Oncology is currently conducting clinical trials with avutometinib with and without defactinib in RAS/MAPK-driven tumors as part of its Raf And Mek Program or RAMP. Verastem is currently enrolling patients and activating sites for RAMP 301 (GOG-3097/ENGOT-ov81/NCRI) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC). Verastem was granted Priority Review and a Prescription Drug User Fee Act (PDUFA) date of June 30, 2025, for its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA), for the investigational combination of avutometinib and defactinib in adults with recurrent KRAS mutant LGSOC who received at least one prior systemic therapy. Verastem initiated a rolling NDA in May 2024 to the FDA and completed its NDA submission in October 2024. The FDA granted Breakthrough Therapy Designation for the treatment of patients with recurrent LGSOC after one or more prior lines of therapy, including platinum-based chemotherapy, in May 2021. Avutometinib alone or in combination with defactinib was also granted Orphan Drug Designation by the FDA for the treatment of LGSOC. Verastem Oncology has established a clinical collaboration with Amgen to evaluate LUMAKRAS™ (sotorasib) in combination with avutometinib and defactinib in both treatment-naïve patients and in patients whose KRAS G12C mutant non-small cell lung cancer progressed on a G12C inhibitor as part of the RAMP 203 trial (NCT05074810). Verastem has received Fast Track Designation from the FDA for the triplet combination in April 2024.

インタレスト・カバレッジ・レシオ	n/a
現金	US$136.35m
エクイティ	US$49.99m
負債合計	US$156.53m
総資産	US$206.51m

データ	最終更新日（UTC時間）
企業分析	2026/09/09 04:25
終値	2026/09/09 00:00
収益	2026/06/30
年間収益	2025/12/31

パッケージ	データ	タイムフレーム	米国ソース例
会社財務	10年	損益計算書キャッシュ・フロー計算書貸借対照表	SECフォーム10-K SECフォーム10-Q
アナリストのコンセンサス予想	+プラス3年	予想財務アナリストの目標株価	アナリストリサーチレポート Blue Matrix
市場価格	30年	株価配当、分割、措置	ICEマーケットデータ SECフォームS-1
所有権	10年	トップ株主インサイダー取引	SECフォーム4 SECフォーム13D
マネジメント	10年	リーダーシップ・チーム取締役会	SECフォーム10-K SECフォームDEF 14A
主な進展	10年	会社からのお知らせ	SECフォーム8-K

アナリスト	機関
Matthew Cross	Alliance Global Partners
James Molloy	Alliance Global Partners
Matthew Venezia	Alliance Global Partners

Verastem バランスシートの健全性

財務の健全性 基準チェック /36

主要情報

財務の健全性に関する最新情報

Verastem, Inc. Auditor Raises 'Going Concern' Doubt

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Recent updates

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Consensus revenue estimates increase by 14%, EPS downgraded

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New major risk - Financial position

New minor risk - Financial position

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New minor risk - Share price stability

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Insufficient new directors

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RAS And MAPK Cancer Pipeline Will Drive Long Term Upside Potential

President recently sold US$74k worth of stock

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Consensus EPS estimates fall by 13%

Verastem Oncology Announces Encouraging Preliminary Data from Ongoing Phase 1/2A Dose Escalation Trial of Vs-7375, an Oral Kras G12d (On/Off) Inhibitor, in Patients with Kras G12d Mutant Solid Tumors

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Verastem Oncology Announces Updated Data from Partner GenFleet Therapeutics' Phase 1/2 Monotherapy Study in China of GFH375 (VS-7375) in Advanced KRAS G12D Mutant Pancreatic Ductal Adenocarcinoma

Independent Director notifies of intention to sell stock

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Consensus revenue estimates increase by 20%

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Verastem Oncology Grants Fast Track Designation for VS-7375 for the Treatment of KRAS G12D-mutated Locally Advanced or Metastatic Pancreatic Cancer

Consensus revenue estimates decrease by 11%

Verastem: The Market Is Ignoring The Progress

Verastem Oncology Announces Publication of the Primary Results from the Phase 2 RAMP 201 Trial of Avutometinib in Combination with Defactinib in Patients with Recurrent Low-Grade Serous Ovarian Cancer in the Journal of Clinical Oncology

Consensus revenue estimates fall by 28%

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New minor risk - Profitability

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Price target increased by 18% to US$15.75

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Verastem Oncology Provides a Clinical Update for RAMP 203 Trial in Advanced KRAS G12C Mutant Non-Small Cell Lung Cancer

Verastem Oncology Completes Rolling NDA Submission to the FDA for Avutometinib Plus Defactinib as A Treatment for Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer

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財務の健全性基準チェック /36