Verastem Oncology Reports Third Quarter 2025 Financial Results and Highlights Recent Business Updates
Achieved AVMAPKI™ FAKZYNJA™ CO-PACK net product revenue of
VS-7375 cleared first two monotherapy dose levels with no dose-limiting toxicities reported; no nausea, vomiting or diarrhea greater than Grade 1 were observed
Enrollment initiated for VS-7375 in combination with cetuximab in patients with advanced KRAS G12D mutant solid tumors, including colorectal cancer
Ended Q3 2025 with
Company to host a conference call and webcast today at 8:00 a.m. ET
"Our performance in Q3, which was the first full quarter since our accelerated approval and launch of AVMAPKI FAKZYNJA CO-PACK, exceeded expectations with net revenue of over
Third Quarter 2025 and Recent Updates
AVMAPKI™ FAKZYNJA™ CO-PACK (avutometinib in combination with defactinib)
-
Achieved net product revenue of
$11.2 million - Prescriptions for patients are being received from both academic and community centers, including both repeat prescriptions from physicians prescribing to multiple patients and refills for individual patients.
- There has been broad payer coverage and reimbursement since launch.
Avutometinib and Defactinib Combination in Low-Grade Serous Ovarian Cancer (LGSOC)
- In the ongoing Phase 3 RAMP 301 confirmatory trial, planned enrollment of the targeted 270 patients was completed a full quarter early.
- A pre-planned Interim Analysis (IA) by an Independent Data Monitoring Committee (IDMC) was conducted for RAMP 301, and the IDMC recommended a modest one-time increase in enrollment. Based on the current total enrollment achieved to date, an additional 29 patients will be added across KRAS mutation status. The Company remains blinded to the IA results.
-
Preliminary safety and efficacy data from the Phase 2 RAMP 201J trial in
Japan was accepted as an E-Poster (EP228/ #371) at the International Gynecologic Cancer Society (IGCS) 2025 Annual Meeting. In the published abstract, with a data extract date of April 11, 2025, no dose limiting toxicities were observed, and avutometinib and defactinib drug exposure levels were comparable to those observed in the global RAMP-201 study. Additional data, including efficacy (response rates) and updated safety will be available on November 5, 2025, when the embargo lifts.
Key Milestone:
- Expect to complete patient enrollment of the IDMC recommended increase in Q1 2026.
VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor, in Advanced Solid Tumors
-
Announced a preliminary update on the Phase 1/2a monotherapy dose escalation trial of VS-7375 in patients with previously treated advanced KRAS G12D mutant solid tumors on Oct. 23, 2025.
-
In the study, VS-7375 cleared both the 400 mg daily (QD) and the 600 mg QD monotherapy doses with no dose-limiting toxicities (DLTs) observed. At the two dose levels evaluated in the
U.S. cohort, no nausea, vomiting, or diarrhea greater than Grade 1 were reported. In addition, no new safety signals have been observed relative to earlier data presentations in both pancreatic ductal adenocarcinoma (PDAC) and non-small cell lung cancer (NSCLC) by our partner, GenFleet Therapeutics, in its ongoing Phase 1/2 clinical study inChina evaluating VS-7375 (known as GFH375). The Company’s dose escalation study continues with evaluation of the monotherapy 900 mg QD dose level. - Of the five efficacy evaluable patients in the VS-7375-101 study with at least one scan, four out of five patients have had a tumor reduction and are still on treatment. The remaining patients receiving either the 400 mg QD or 600 mg QD doses have not yet reached their first response assessment.
-
In the study, VS-7375 cleared both the 400 mg daily (QD) and the 600 mg QD monotherapy doses with no dose-limiting toxicities (DLTs) observed. At the two dose levels evaluated in the
- The Company also announced it has initiated patient enrollment for the first dose escalation combination cohort evaluating VS-7375 with cetuximab in patients with advanced solid tumors, including colorectal cancer.
-
Announced updated data from partner GenFleet Therapeutics’ Phase 1/2 study of GFH375 in
China that was featured in a late-breaking oral presentation at the European Society for Medical Oncology (ESMO) Congress on October 19, 2025.-
Among 59 heavily pre-treated patients with PDAC who received one or more prior lines of therapy, an overall response rate (ORR) of
41% was achieved at the monotherapy recommended Phase 2 dose (RP2D) of 600 mg QD. A disease control rate (DCR) of96.7% (57/59) was also reported with the majority of patients (91.5% ) experiencing a reduction in target lesions. -
Overall survival (OS) observed at month four was
92.2% . The median OS was not reached as of the data cutoff, with a median follow-up time of 5.65 months. The median progression-free survival (mPFS) was 5.52 months with a median follow-up time of 5.65 months and a 4-month PFS rate of78.2% . At evaluation, 31 (47% ) patients were still on treatment with the longest duration of treatment eclipsing one year (367 days). The safety profile in PDAC patients was consistent with the previously reported data at recent medical congresses.
-
Among 59 heavily pre-treated patients with PDAC who received one or more prior lines of therapy, an overall response rate (ORR) of
-
Announced updated data from GenFleet’s Phase 1/2 study of GFH375 in
China that was featured in a mini oral presentation at the IASLC 2025 World Conference on Lung Cancer (WCLC) on September 8, 2025.-
At the RP2D of 600 mg QD, the ORR was
68.8% (11/16) (both confirmed and unconfirmed) and the DCR was93.8% (15/16). Among the 26 evaluable patients with NSCLC treated across all dose levels, the ORR was57.7% (15/26) (both confirmed and unconfirmed) and the DCR was88.5% (23/26).
-
At the RP2D of 600 mg QD, the ORR was
-
GenFleet shared the following additional analyses on Oct. 27, 2025, from previously presented data at recent medical congresses evaluating GFH375 in both advanced KRAS G12D mutant PDAC and NSCLC:
-
In a subgroup analysis, 12 patients with 2L PDAC at 600 mg QD achieved an ORR of
58.3% and a DCR of100% . In the 3L+ setting, 47 PDAC patients receiving 600 mg QD achieved an ORR of36.2% and a DCR of95.7% . In the 2L subgroup, the mPFS and mOS have not been reached. An additional analysis of gastrointestinal disorders, hematological toxicities, and liver enzyme abnormalities in 2L+ patients with PDAC (n=66) at 600 mg QD showed no adverse events Grade ≥3 occurred at rates above8.0% . -
In an analysis of pre-treated patients with NSCLC at 600 mg QD, the four-month PFS rate was >
75% and the mPFS has not been reached. The median follow-up time was 4.2 months.
-
In a subgroup analysis, 12 patients with 2L PDAC at 600 mg QD achieved an ORR of
- GenFleet also shared that the first patient has been dosed in a Phase 1b/2 study of GFH375 combined with cetuximab or chemotherapy for advanced solid tumors on October 22, 2025.
Key Milestones:
- Plan to initiate the dose escalation cohorts in combination with chemotherapy for PDAC and with chemotherapy plus anti-PD-1 for NSCLC in Q4 2025.
- Plan to report an interim safety and efficacy update on the Phase 1/2a trial of VS-7375 in 1H 2026.
- Expect to select the RP2D and plan to initiate monotherapy expansion cohorts in advanced PDAC, NSCLC, and other KRAS G12D-mutated solid tumors in 1H 2026.
- Expect to select the RP2D and plan to initiate combination expansion cohorts in CRC, PDAC, and NSCLC in 1H 2026.
- Plan to engage with the FDA to discuss our development path forward, including potential registration-directed clinical trials in PDAC and NSCLC in 1H 2026.
RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in First-Line Metastatic PDAC
- Completed enrollment in the RAMP 205 expansion cohort in Q3 2025.
Key Milestone:
- Expect to report an update on the safety and efficacy of the RAMP 205 expansion cohort with 29 patients at the RP2D in 1H26.
RAMP 203: Avutometinib Plus Defactinib in Combination with a KRAS G12C Inhibitor in NSCLC
- Patients continue to be evaluated in both the doublet and triplet combination cohorts of the study.
Key Milestone:
- Report an interim update on the safety and efficacy results in RAMP 203 from both the doublet and triplet combinations in Q4 2025.
Third Quarter 2025 Financial Results
Net product revenue for the three months ended September 30, 2025 (the “2025 Quarter”) was
Total operating expenses for the 2025 Quarter were
Research & development expenses for the 2025 Quarter were
Selling, general & administrative expenses for the 2025 Quarter were
Net loss (GAAP basis) for the 2025 Quarter was
For the 2025 Quarter, non-GAAP adjusted net loss was
Verastem Oncology ended the third quarter of 2025 with cash, cash equivalents and investments of
Conference Call and Webcast
Verastem will host a conference call and webcast today at 8:00 a.m. ET to review the third quarter 2025 financial results and recent business updates. To access the conference call, please dial (888) 596-4144 (
Use of Non-GAAP Financial Measures
To supplement Verastem Oncology’s condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in
About AVMAPKI and FAKZYNJA Combination Therapy
AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.
The
AVMAPKI FAKZYNJA CO-PACK
Indication
AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.
This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Important Safety Information
Warnings and Precautions
- Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.
- Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.
- Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.
- Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.
- Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.
Adverse Reactions
The most common (≥
Drug Interactions
- Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
- Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
- Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.
- Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.
Use in Specific Populations
- Lactation: Advise not to breastfeed.
- Fertility: May impair fertility in males and females.
Click here for full Prescribing Information.
About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor
VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 is the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. Verastem initiated VS-7375-101, a Phase 1/2a clinical trial, in June of 2025 in the
About the GenFleet Therapeutics Collaboration
The collaboration with GenFleet Therapeutics aims to advance three oncology discovery programs related to RAS/MAPK pathway-driven cancers. The collaboration provides Verastem with an exclusive option to obtain a license for each of the three compounds in the collaboration after the successful completion of pre-determined milestones in a Phase 1 trial. Verastem selected VS-7375 (also known as GFH375), an oral KRAS G12D (ON/OFF) inhibitor, as its lead program in December 2023 and the license for VS-7375 that was exercised in January 2025 is the first one from this collaboration. The licenses would give Verastem development and commercialization rights outside the GenFleet markets of mainland
About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets AVMAPKI™ FAKZYNJA™ CO-PACK in the
Forward-Looking Statements Notice
This press release includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,” and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of commercialization of product candidates, the conduct of the Phase 1/2a study for VS-7375/GFH375, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics (
Verastem Oncology |
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Condensed Consolidated Statements of Operations |
||||||||||||||||
(in thousands, except per share amounts) |
||||||||||||||||
(unaudited) |
||||||||||||||||
|
|
Three months ended September 30, |
|
Nine months ended September 30, |
||||||||||||
|
|
2025 |
|
2024 |
|
2025 |
|
2024 |
||||||||
Revenue: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Product revenue, net |
|
$ |
11,242 |
|
|
$ |
— |
|
|
$ |
13,379 |
|
|
$ |
— |
|
Sale of COPIKTRA license and related assets |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
10,000 |
|
Total revenue |
|
|
11,242 |
|
|
|
— |
|
|
|
13,379 |
|
|
|
10,000 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cost of sales - product |
|
|
1,670 |
|
|
|
— |
|
|
|
1,988 |
|
|
|
— |
|
Cost of sales - intangible amortization |
|
|
290 |
|
|
|
— |
|
|
|
418 |
|
|
|
— |
|
Research and development |
|
|
28,989 |
|
|
|
24,754 |
|
|
|
82,925 |
|
|
|
60,523 |
|
Selling, general and administrative |
|
|
21,008 |
|
|
|
12,276 |
|
|
|
56,702 |
|
|
|
32,843 |
|
Total operating expenses |
|
|
51,957 |
|
|
|
37,030 |
|
|
|
142,033 |
|
|
|
93,366 |
|
Loss from operations |
|
|
(40,715 |
) |
|
|
(37,030 |
) |
|
|
(128,654 |
) |
|
|
(83,366 |
) |
Other expense |
|
|
(37 |
) |
|
|
(77 |
) |
|
|
(186 |
) |
|
|
(131 |
) |
Interest income |
|
|
1,182 |
|
|
|
831 |
|
|
|
2,964 |
|
|
|
3,181 |
|
Interest expense |
|
|
(319 |
) |
|
|
(1,148 |
) |
|
|
(723 |
) |
|
|
(3,416 |
) |
Loss on debt extinguishment |
|
|
— |
|
|
|
— |
|
|
|
(1,826 |
) |
|
|
— |
|
Change in fair value of preferred stock tranche liability |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
4,189 |
|
Change in fair value of warrant liability |
|
|
(55,881 |
) |
|
|
13,457 |
|
|
|
(37,977 |
) |
|
|
13,457 |
|
Change in fair value of Notes |
|
|
(2,748 |
) |
|
|
— |
|
|
|
(10,153 |
) |
|
|
— |
|
Net loss |
|
$ |
(98,518 |
) |
|
$ |
(23,967 |
) |
|
$ |
(176,555 |
) |
|
$ |
(66,086 |
) |
Net loss per share—basic and diluted |
|
$ |
(1.35 |
) |
|
$ |
(0.60 |
) |
|
$ |
(2.73 |
) |
|
$ |
(2.11 |
) |
Weighted average common shares outstanding used in computing net loss per share—basic and diluted |
|
|
73,157 |
|
|
|
40,258 |
|
|
|
64,561 |
|
|
|
31,350 |
|
Verastem Oncology |
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Condensed Consolidated Balance Sheets |
|||||||
(in thousands) |
|||||||
(unaudited) |
|||||||
|
September 30,
|
December 31, 2024 |
|||||
|
|
|
|
||||
Cash & cash equivalents |
$ |
137,706 |
|
|
$ |
88,818 |
|
Accounts receivable, net |
|
6,716 |
|
|
|
— |
|
Inventory |
|
1,794 |
|
|
|
— |
|
Grant receivable |
|
200 |
|
|
|
200 |
|
Prepaid expenses and other current assets |
|
7,640 |
|
|
|
5,943 |
|
Property and equipment, net |
|
20 |
|
|
|
32 |
|
Right-of-use asset, net |
|
730 |
|
|
|
1,405 |
|
Intangible assets, net |
|
16,705 |
|
|
|
— |
|
Other assets |
|
5,341 |
|
|
|
5,140 |
|
Total assets |
$ |
176,852 |
|
|
$ |
101,538 |
|
|
|
|
|
|
|
||
Current Liabilities |
$ |
59,712 |
|
|
$ |
30,973 |
|
Long term debt |
|
78,124 |
|
|
|
40,724 |
|
Vendor financing arrangement, long-term |
|
6,250 |
|
|
|
— |
|
Lease liability, long-term |
|
— |
|
|
|
535 |
|
Accrued expenses, long-term |
|
— |
|
|
|
— |
|
Warrant liability |
|
48,292 |
|
|
|
58,199 |
|
Stockholders’ (deficit) |
|
(15,526 |
) |
|
|
(28,893 |
) |
Total liabilities, and stockholders’ (deficit) |
$ |
176,852 |
|
|
$ |
101,538 |
|
Verastem, Inc. |
||||||||||||||||
Reconciliation of GAAP to Non-GAAP Financial Information |
||||||||||||||||
(in thousands, except per share amounts) |
||||||||||||||||
(unaudited) |
||||||||||||||||
Three months ended
|
Nine months ended
|
|||||||||||||||
2025 |
|
2024 |
|
2025 |
|
2024 |
||||||||||
Net loss reconciliation |
|
|
||||||||||||||
Net loss (GAAP basis) |
|
$ |
(98,518 |
) |
|
$ |
(23,967 |
) |
|
$ |
(176,555 |
) |
|
$ |
(66,086 |
) |
Adjust: |
||||||||||||||||
Stock-based compensation expense |
|
|
2,178 |
|
|
|
1,935 |
|
|
|
7,379 |
|
|
|
5,323 |
|
Non-cash interest, net |
— |
|
201 |
|
(62 |
) |
(212 |
) |
||||||||
Change in fair value of preferred stock tranche liability |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(4,189 |
) |
Change in fair value of warrant liability |
55,881 |
|
(13,457 |
) |
37,977 |
|
(13,457 |
) |
||||||||
Non-cash change in fair value of Notes |
|
|
1,105 |
|
|
|
— |
|
|
|
5,670 |
|
|
|
— |
|
Loss on debt extinguishment |
— |
|
— |
|
1,826 |
|
— |
|
||||||||
Severance and Other |
|
|
— |
|
|
|
10 |
|
|
|
— |
|
|
|
619 |
|
Adjusted net loss (non-GAAP basis) |
|
$ |
(39,354 |
) |
|
$ |
(35,278 |
) |
|
$ |
(123,765 |
) |
|
$ |
(78,002 |
) |
|
|
|
|
|
|
|||||||||||
Reconciliation of net loss per share |
||||||||||||||||
Net loss per share – diluted (GAAP basis) |
|
$ |
(1.35 |
) |
|
$ |
(0.60 |
) |
|
$ |
(2.73 |
) |
|
$ |
(2.11 |
) |
Adjust per basic share |
||||||||||||||||
Stock-based compensation expense |
|
|
0.03 |
|
|
|
0.05 |
|
|
|
0.11 |
|
|
|
0.17 |
|
Non-cash interest, net |
— |
|
— |
|
— |
|
(0.01 |
) |
||||||||
Change in fair value of preferred stock tranche liability |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(0.13 |
) |
Change in fair value of warrant liability |
0.76 |
|
(0.33 |
) |
0.59 |
|
(0.43 |
) |
||||||||
Non-cash change in fair value of Notes |
|
|
0.02 |
|
|
|
— |
|
|
|
0.08 |
|
|
|
— |
|
Loss on debt extinguishment |
— |
|
— |
|
0.03 |
|
— |
|
||||||||
Severance and Other |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
0.02 |
|
Adjusted net loss per share – diluted (non-GAAP basis) |
$ |
(0.54 |
) |
$ |
(0.88 |
) |
$ |
(1.92 |
) |
$ |
(2.49 |
) |
||||
Weighted average common shares outstanding used in computing net loss per share—diluted |
|
|
73,157 |
|
|
|
40,258 |
|
|
|
64,561 |
|
|
|
31,350 |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20251104228194/en/
For Investor and Media Inquiries:
Julissa Viana
Vice President, Corporate Communications,
Investor Relations & Patient Advocacy
investors@verastem.com or
media@verastem.com
Source: Verastem Oncology
