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Verastem Oncology Reports First Quarter 2026 Financial Results and Highlights Recent Business Updates
AVMAPKI® FAKZYNJA® CO-PACK net product revenue of
Appointed
Initiated Phase 2 registration-directed trials “VS-7375 TARGET-D Clinical Program” in 2L PDAC, 2L/3L NSCLC and 2L+ CRC
VS-7375 TARGET-D 101 early data update expected in the first half of 2026; mature data update expected in the second half of 2026
Ended Q1 2026 with
Company to host a conference call and webcast today at
“As we mark the one-year anniversary of the FDA-approval of AVMAPKI FAKZYNJA CO-PACK for KRAS-mutated recurrent low-grade serous ovarian cancer, I am incredibly proud of the progress we have made to deliver a meaningful impact for people living with this rare cancer,” said
First Quarter 2026 and Recent Updates
AVMAPKI® FAKZYNJA® CO-PACK (avutometinib capsules; defactinib tablets)
-
AVMAPKI FAKZYNJA CO-PACK generated net product revenue of
$18.7 million for the first quarter of 2026. - In April, the Company announced the launch of a new healthcare professional and patient marketing campaign, Reimagine Recurrent Low-Grade Serous Ovarian Cancer (LGSOC), to drive awareness of AVMAPKI FAKZYNJA CO-PACK.
-
In April, the Company announced new two-year median follow up data from the Phase 2 RAMP 201 trial that demonstrated durable benefit of avutometinib plus defactinib across both KRAS mutant and KRAS wild-type recurrent LGSOC patients, with discontinuation rates consistent with the primary analysis, presented at the
Society of Gynecologic Oncology 2026 Annual Meeting on Women’s Cancers. A new exposure-response analysis further demonstrated that the approved dose and schedule of avutometinib plus defactinib achieve the optimal therapeutic effect. -
In February, the Company announced updated data for RAMP 201J in
Japan evaluating the combination in patients with LGSOC with or without a KRAS mutation. As of a data cutoff ofJanuary 30, 2026 , a confirmed overall response rate (ORR) of 38% (6/16) was achieved. Patients with a KRAS mutation achieved a confirmed ORR of 57% (4/7) and KRAS wild-type patients achieved a confirmed ORR of 22% (2/9). The safety profile was consistent with previously reported data outside ofJapan .
Expected Key Milestones:
- Report a topline readout of the primary endpoint in the RAMP 301 trial in mid-2027.
-
Continue to pursue regulatory paths for potential expansion into
Europe andJapan .
VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor in Advanced Solid Tumors
- Today, the Company provided an update on its ongoing VS-7375 clinical trials, including branding the trials as the VS-7375 TARGET-D Clinical Trial Program. In the ongoing TARGET-D 101 (VS-7375-101) Phase 1/2 dose escalation, dose expansion and combination-evaluation trial, the Company is now evaluating the 1200 mg daily dose (QD). The Company is also evaluating VS-7375 900 mg QD in combination with cetuximab. The Company expects to finish enrollment across the various expansion cohorts and combination cohorts with chemotherapies in the near term.
- In addition, the Company shared updated pharmacokinetic (PK) data that showed the 900 mg QD dose achieves target plasma levels of VS-7375 and provides clear separation from the 600 mg QD dose.
-
The Company has initiated three Phase 2 registration-directed trials, including:
- TARGET-D 201 to evaluate VS-7375 at 900 mg QD both as monotherapy and in combination with cetuximab in patients with second-line pancreatic ductal carcinoma (PDAC). The study is also evaluating VS-7375 and cetuximab in the first-line PDAC setting.
- TARGET-D 202 to evaluate VS-7375 at 900 mg QD in patients with advanced non-small cell lung cancer (NSCLC) who have received one to two prior lines of therapy. The study is also evaluating VS-7375 in NSCLC patients with asymptomatic untreated brain metastases.
- TARGET-D 203 to evaluate VS-7375 at 900 mg QD as both monotherapy and in combination with EGFR inhibitors, including cetuximab or panitumumab, in previously treated colorectal cancer (CRC). The study will also evaluate VS-7375 in combination with chemotherapy in the first-line setting in patients with metastatic CRC.
-
In March, the Company announced that several late-breaking and regular abstracts were selected for presentation at the
American Association for Cancer Research (AACR) Annual Meeting. The abstracts included preclinical data demonstrating sustained tumor regressions with VS-7375 in combination with PRMT5 inhibitors in MTAP-deleted KRAS G12D mutant pancreatic cancer and improved efficacy in comparison to ON-only KRAS G12D or pan-RAS inhibitors. -
In March, the Company reported an update on its progress with VS-7375, highlighting progress with its dose-escalation and dose-expansion trial, TARGET-D 101, a PK analysis, and a safety update, which demonstrated VS-7375 was generally well-tolerated across all monotherapy dose levels evaluated to date. Patients (n=23) receiving VS-7375 at either 400 mg QD, 600 mg QD or 900 mg QD with a mean duration of therapy of 1.6 months (0.7-5.6), reported no drug related liver function test abnormalities. As of the
January 30, 2026 data cutoff, there was no drug-related neutropenia greater than Grade 2 and rates of nausea, vomiting and diarrhea remained lower than those reported by the Company’s partner inChina . The Company also reported that theU.S. Food and Drug Administration (FDA) requested the Company develop separate Phase 2 trial protocols for their trials in PDAC, NSCLC and CRC. - In January, the Company reported progress from its ongoing TARGET-D 101 trial, with multiple dose levels cleared without dose-limiting toxicities or major toxicities. At that time, monotherapy cohorts and dose escalation combination cohorts were initiated across various KRAS G12D-mutated solid tumors.
The Company shared multiple updates from GenFleet Therapeutics, its partner developing VS-7375, known as GFH375 in
-
In April, GenFleet announced that GFH375 was granted Breakthrough Therapy Designation (BTD) in
China for patients with KRAS G12D-mutated metastatic pancreatic cancer who have received at least one prior systemic therapy. -
In March, GenFleet announced that GFH375 was granted BTD in
China for patients with KRAS G12D-mutated NSCLC who have received prior systemic therapy.
Expected Key Milestones:
- Report early data from the TARGET-D 101 trial in 1H 2026.
- Report an update on the TARGET-D 101 trial in 2H 2026.
- Announce first patient initiated in the TARGET-D 201, TARGET-D 202, and TARGET-D 203 clinical trials in mid-2026.
RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in 1L Metastatic Pancreatic Cancer
Expected
- Report an update on the safety and efficacy of the RAMP 205 expansion cohort with at least six months of follow-up on all patients in Q2 2026.
Corporate Updates
-
The Company announced today the appointment of
Daniel Lyons as chief commercial officer. Prior to joining,Mr. Lyons served as the Senior Vice President, Head ofRare Tumors International and Global Head of Market Access atSpringWorks Therapeutics .
First Quarter 2026 Financial Results
Verastem Oncology ended the first quarter of 2026 with cash, cash equivalents, and investments of
Net product revenue for the three months ended
Total operating expenses for the 2026 Quarter were
Research & development expenses for the 2026 Quarter were
Selling, general & administrative expenses for the 2026 Quarter were
Net loss (GAAP basis) for the 2026 Quarter was
Non-GAAP adjusted net loss for the 2026 Quarter was
Conference Call and Webcast
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
Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company’s GAAP financial statements, because it provides greater transparency and period-over- period comparability with respect to the Company’s operating performance and can enhance investors’ ability to identify operating trends in the Company’s business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company’s operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months ended
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 (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.
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. In
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
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.
Forward-Looking Statements
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 anticipated timing for the IND application for VS-7375/GFH375, the expected outcome and benefits of the Company’s collaboration with
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Verastem Oncology |
|||||
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Condensed Consolidated Balance Sheets |
|||||
|
(in thousands) |
|||||
|
|
|
||||
|
Cash, cash equivalents, and investments |
$ |
181,678 |
$ |
204,990 |
|
|
Accounts receivable, net |
|
10,158 |
|
|
8,813 |
|
Inventory |
|
2,287 |
|
|
1,833 |
|
Grants receivable |
200 |
200 |
|||
|
Prepaid expenses and other current assets |
8,581 |
7,577 |
|||
|
Right-of-use asset, net |
2,793 |
491 |
|||
|
Intangible assets, net |
|
16,146 |
|
|
16,426 |
|
Restricted cash and other assets |
5,965 |
6,112 |
|||
|
Total assets |
$ |
227,808 |
$ |
246,442 |
|
|
Current Liabilities |
66,845 |
72,268 |
|||
|
Long term debt |
73,120 |
76,330 |
|||
|
Vendor financing arrangement, long-term |
|
3,750 |
|
|
5,000 |
|
Lease liability, long-term |
|
2,316 |
|
|
— |
|
Warrant liability |
— |
35,647 |
|||
|
Stockholders’ equity |
81,777 |
57,197 |
|||
|
Total liabilities and stockholders’ equity |
$ |
227,808 |
$ |
246,442 |
|
|
Verastem Oncology |
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|
Condensed Consolidated Statements of Operations |
||||||
|
(in thousands, except per share amounts) |
||||||
|
(unaudited) |
||||||
|
Three months ended |
||||||
|
2026 |
2025 |
|||||
|
Revenue: |
||||||
|
Product revenue, net |
|
$ |
18,671 |
|
$ |
— |
|
Total revenue |
|
18,671 |
|
— |
||
|
Operating expenses: |
||||||
|
Cost of sales – product |
|
|
2,772 |
|
|
— |
|
Cost of sales – intangible amortization |
|
|
279 |
|
|
— |
|
Research and development |
38,218 |
29,152 |
||||
|
Selling, general and administrative |
|
22,299 |
|
15,044 |
||
|
Total operating expenses |
|
63,568 |
|
44,174 |
||
|
Loss from operations |
|
(44,897) |
|
(44,174) |
||
|
Other expense |
(61) |
(40) |
||||
|
Interest income |
|
1,297 |
|
960 |
||
|
Interest expense |
|
(382) |
|
(192) |
||
|
Loss on debt extinguishment |
|
|
— |
|
|
(1,826) |
|
Change in fair value of warrant liability |
9,323 |
(2,416) |
||||
|
Change in fair value of Notes |
|
|
(1,871) |
|
|
(4,415) |
|
Net Loss |
$ |
(36,591) |
$ |
(52,103) |
||
|
Net loss per share—basic |
$ |
(0.37) |
$ |
(0.96) |
||
|
Net loss per share—diluted |
$ |
(0.46) |
$ |
(0.96) |
||
|
Weighted average common shares outstanding used in computing: |
|
|
||||
|
Net loss per share – basic |
98,292 |
54,173 |
||||
|
Net loss per share – diluted |
|
|
99,148 |
|
|
54,173 |
|
Verastem Oncology |
||||||
|
Reconciliation of GAAP to Non-GAAP Financial Information |
||||||
|
(in thousands, except per share amounts) |
||||||
|
(unaudited) |
||||||
|
Three months ended |
||||||
|
2026 |
2025 |
|||||
|
Net loss reconciliation: |
||||||
|
Net loss (GAAP basis) |
|
$ |
(36,591) |
|
$ |
(52,103) |
|
Adjust: |
|
|||||
|
Stock-based compensation expense |
|
2,046 |
|
1,788 |
||
|
Amortization of acquired intangible assets |
|
279 |
— |
|||
|
Non-cash interest, net |
|
|
(119) |
|
|
30 |
|
Change in fair value of warrant liability |
|
(9,323) |
2,416 |
|||
|
Non-cash change in fair value of Notes |
|
186 |
|
3,115 |
||
|
Loss on debt extinguishment |
|
— |
|
1,826 |
||
|
Severance and other |
|
842 |
|
— |
||
|
Adjusted net loss (non-GAAP basis) |
$ |
(42,680) |
$ |
(42,928) |
||
|
|
|
|
|
|
|
|
|
Reconciliation of net loss per share |
|
|
|
|
|
|
|
Net loss per share – basic (GAAP basis) |
|
$ |
(0.37) |
|
$ |
(0.96) |
|
Adjust per basic share |
|
|
|
|
|
|
|
Stock-based compensation expense |
|
0.02 |
|
|
0.03 |
|
|
Amortization of acquired intangible assets |
|
|
— |
|
|
— |
|
Non-cash interest, net |
|
— |
|
|
0.01 |
|
|
Change in fair value of warrant liability |
|
(0.09) |
|
|
0.05 |
|
|
Non-cash change in fair value of Notes |
|
— |
|
|
0.06 |
|
|
Loss on debt extinguishment |
|
— |
|
|
0.03 |
|
|
Severance and other |
|
0.01 |
|
|
— |
|
|
Adjusted net loss per share – basic (non-GAAP basis) |
$ |
(0.43) |
|
$ |
(0.79) |
|
|
Weighted average common shares outstanding used in computing net loss per share—basic |
|
|
98,292 |
|
|
54,173 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20260507004786/en/
For Investor and Media Inquiries:
Senior Vice President, Corporate Communications,
Investor Relations & Patient Advocacy
investors@verastem.com or
media@verastem.com
Source: Verastem Oncology