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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the
Securities Exchange Act of 1934
Date of report (Date of earliest event reported):
August 6, 2026
Verastem,
Inc.
(Exact Name of Registrant as Specified in
Charter)
| Delaware |
|
001-35403 |
|
27-3269467 |
(State or Other Jurisdiction
of Incorporation) |
|
(Commission File Number) |
|
(IRS Employer Identification No.) |
| 117 Kendrick Street, Suite 500, Needham, MA |
|
02494 |
| (Address of Principal Executive Offices) |
|
(Zip Code) |
Registrant’s telephone number, including
area code: (781) 292-4200
(Former Name or Former Address, if Changed
Since Last Report)
Check the appropriate box below if the Form 8-K filing is intended
to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| ¨ | Written communications pursuant to Rule 425 under
the Securities Act (17 CFR 230.425) |
| ¨ | Soliciting material pursuant to Rule 14a-12 under
the Exchange Act (17 CFR 240.14a-12) |
| ¨ | Pre-commencement communications pursuant to Rule 14d-2(b) under
the Exchange Act (17 CFR 240.14d-2(b)) |
| ¨ | Pre-commencement communications pursuant to Rule 13e-4(c) under
the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b)
of the Act:
| Title of each class |
|
Trading Symbol(s) |
|
Name
of each exchange on which registered |
| Common stock, $0.0001 par value per share |
|
VSTM |
|
The Nasdaq Capital Market |
Indicate by check mark whether the registrant is an emerging
growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities
Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging
growth company ¨
If
an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for
complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 1.01 Entry into a Material Definitive
Agreement
On August 6, 2026 (the “Amendment Closing
Date”), Verastem, Inc., (the “Company”) entered into Amendment No. 3 (the “Amendment”) to that certain
Note Purchase Agreement, dated as of January 13, 2025 (the “Original Closing Date”), as previously amended by Amendment No.
1 dated as of October 31, 2025 and Amendment No. 2 dated as of March 2, 2026 (as so amended, the “Note Purchase Agreement”)
by and among the Company, certain funds managed by Oberland Capital Management LLC (together with other purchasers party thereto from
time to time, the “Purchasers”) and RGCM SA LLC, as purchaser agent.
The Amendment amends the financing arrangement
under the Note Purchase Agreement by establishing two separate series of senior secured notes: (i) “Initial Notes,” which
are the notes in an initial aggregate principal amount of $75.0 million previously issued on the Original Closing Date in connection
with the first purchase under the Note Purchase Agreement, and (ii) “Revenue Notes,” which are a new series of notes to be
issued in two tranches under the Note Purchase Agreement with an aggregate purchase price of up to $75.0 million (the Initial Notes and
Revenue Notes, together, the “Notes”). The establishment of the Revenue Notes replaces the previous conditional second and
third tranches of $25.0 million principal amount of Initial Notes and $50.0 million principal amount of Initial Notes, respectively.
The Amendment provides that:
| | · | an
initial tranche of $50.0 million in Revenue Notes will be issued on August 28, 2026, subject
to satisfaction of certain customary conditions precedent (the “Second Purchase”);
and |
| | | |
| | · | at
the option of the Company, a second tranche of $25.0 million principal amount of Revenue
Notes may be issued (the “Third Purchase”), at any time on or prior to May 15, 2027,
provided that worldwide net sales of avutometinib and defactinib are at least $40.0 million
for the calendar quarter ended immediately prior to the Third Purchase and subject to certain
other customary conditions precedent. |
The Revenue Notes are a separate series of Notes
which, rather than bearing fixed interest, entitle the Purchasers to quarterly payments (the “Revenue Payments”) equal to
the “Revenue Payment Percentage” of net sales of the Company’s products that combine avutometinib and defactinib for
such fiscal quarter, including any monetary damages recovered from a third party in any action brought for such third party’s infringement
of any intellectual property related to such products, but only to the extent such damages are awarded for lost sales of the product and
are net of specified enforcement expenses and amounts required to be allocated to licensors or sublicensees. The Revenue Payment Percentage
will initially be 4.50%. If the total Revenue Payments are equal to or greater than 100% of the then total purchase price paid by the
Purchasers for all Revenue Notes (the “Funded Amount”) (such condition, the “Test Date Condition”) as of December
31, 2031 (the “Test Date”), the Revenue Payment Percentage will automatically decrease to 1.75% after the Test Date. If the
Test Date Condition is not satisfied by the Test Date, the Purchasers will be entitled to a one-time contingent make-whole payment from
the Company (the “Contingent Make-Whole Payment”) in an amount equal to 100% of the Funded Amount as of the Test Date less
the total Revenue Payments made by the Company as of the Test Date.
The maturity date for the Revenue Notes is June
30, 2033 (the “Revenue Notes Maturity Date”). All of the Revenue Notes may be redeemed prior to the Revenue Note Maturity
Date at the option of the Company, subject to payment of the “Repayment Amount” (as defined in the Note Purchase Agreement).
If the Revenue Notes are redeemed or repaid, the “Repayment Amount” will be: (a) 135% of the Funded Amount if redemption
occurs on or prior to the first anniversary of the date of the Second Purchase (the “Second Purchase Date”) upon a change
of control of the Company; (b) 150% of the Funded Amount if clause (a) does not apply and redemption occurs on or prior to the first
anniversary of the Second Purchase Date upon a sale or exclusive license by the Company of all or substantially all intellectual property
relating to the Primary Product (as defined in the Note Purchase Agreement) within the United States; (c) 150% of the Funded Amount if
clauses (a) and (b) do not apply and the Repayment Amount is paid on or prior to the Test Date and the Test Date Condition has been met;
(d) 165% of the Funded Amount if clauses (a), (b) and (c) do not apply and redemption occurs on or prior to the third anniversary of
the Second Purchase Date; and (e) 185% of the Funded Amount (the “Cap Amount”) if clauses (a), (b) and (c) do not apply and
redemption occurs after the third anniversary of the Second Purchase Date (provided that the Cap Amount decreases to 150% of the Funded
Amount following the Test Date if the Test Date Condition has been met), minus, in each case, total Revenue Payments, any Contingent
Make-Whole Payment, and all payments of original issue discount in respect of the Revenue Notes paid in cash prior to such date.
In the event of any voluntary prepayment of the
Revenue Notes upon the sale or exclusive license (other than a Permitted License (as defined in the Note Purchase Agreement)) of all
or substantially all intellectual property relating to the Primary Product within the United States, a portion of the Repayment Amount
for the Revenue Notes in an amount not to exceed 25% of the Funded Amount may, at the option of the Company, be paid in shares of the
Company’s common stock, subject to certain conditions and limitations. The Purchasers may demand redemption of the Notes prior
to the applicable maturity date in the event of certain change of control events or events of default, subject to payment of the then-applicable
Repayment Amount.
The Company’s obligations under the Note
Purchase Agreement, as amended, continue to be secured by a first-priority security interest (subject to certain permitted liens) on
substantially all of the Company’s and its subsidiaries’ assets, including its intellectual property related to avutometinib
and defactinib, and subject to a negative pledge on the Company’s intellectual property. The Note Purchase Agreement contains no
financial covenants. The Company’s obligations remain subject to customary affirmative and negative covenants, including limitations
on the Company’s ability to dispose of assets, undergo a change of control, merge with or acquire other entities, incur debt, incur
liens, pay dividends or other distributions to holders of its capital stock, repurchase stock and make investments, in each case subject
to certain exceptions.
Until the maturity date of the Initial Notes (the seventh anniversary of
January 13, 2025) (the “Initial Notes Maturity Date”), the Company is obligated to make quarterly Revenue Participation Payments
to the Purchasers equal to the “Revenue Participation Percentage” of net sales of the Company’s products that combine
avutometinib and defactinib during such fiscal quarter, subject to a cap of $100.0 million of such net sales in each fiscal year. Following
the Amendment, the Revenue Participation Percentage is fixed at 1.00% and will not be adjusted in connection with the issuance of additional
Notes. The applicable interest rate and other terms of the Initial Notes remain unchanged as a result of the Amendment. The outstanding
principal amount of the Initial Notes bear interest at a rate per annum equal to the sum of (i) the greater of the Term SOFR (as defined
in the Note Purchase Agreement) and 4.29%, and (ii) 3.71%, subject to adjustment in certain circumstances set forth in the Note Purchase
Agreement and an overall cap of 9.75%, payable quarterly in arrears until the Initial Notes Maturity Date. For the first eight (8) payment
dates on which interest on the Initial Notes is owed and continuing, at the Company’s option, up to 50% of the interest due may
be paid-in-kind and added to the then-outstanding principal balance of the Initial Notes. Upon the occurrence and during the continuance
of an Event of Default (as defined in the Note Purchase Agreement), the then-applicable interest rate on all outstanding Initial Note
obligations may be increased by an additional 5.00%.
A copy of the amendment is attached as Exhibit
10.1 to this Current Report on Form 8-K and is incorporated herein by reference. The foregoing summary of the amendment does not purport
to be complete and is qualified in its entirety by reference to the complete text of the Amendment and the Note Purchase Agreement.
Item 2.03 Creation of a Direct Financial Obligation
or an Obligation under an Off-Balance Sheet Arrangement of a Registrant.
The information set forth
in Item 1.01 of this Current Report on Form 8-K is incorporated by reference into this Item 2.03.
Item 7.01 Regulation FD Disclosure
On August 6, 2026, Verastem,
Inc. posted its updated corporate presentation on its website, a copy of which is furnished hereto as Exhibit 99.1 to this Current Report
on Form 8-K.
Item 9.01 Financial Statements and Exhibits
| Exhibit No. |
|
Description |
| 10.1 |
|
Amendment No. 3 to Note Purchase Agreement, dated August 6, 2026, by
and among Verastem, Inc., RGCM SA LLC and certain funds managed by Oberland Capital Management LLC. |
| 99.1 |
|
Corporate Presentation, dated August 6, 2026 |
| 104 |
|
Cover Page Interactive Data File (embedded within the Inline XBRL document) |
SIGNATURES
Pursuant to the requirements
of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto
duly authorized.
| |
VERASTEM, INC. |
| |
|
|
| Dated: August 6, 2026 |
By: |
/s/ Daniel W. Paterson |
| |
|
Daniel W. Paterson |
| |
|
Chief Executive Officer |
Exhibit 99.1
| 
| Delivering Novel
Therapies for
RAS/MAPK Pathway-Driven Cancers
C O R P O R A T E P R E S E N T A T I O N
A U G U S T 2 0 2 6 |
| 
| 2
FORWARD-LOOKING STATEMENTS
This presentation includes forward-looking statements about, among other things, Verastem Oncology’s (the “Company”) programs and product candidates, strategy, future plans and prospects, including statements related to the approval and
commercialization of AVMAPKI® FAKZYNJA® CO-PACK (avutometinib capsules; defactinib tablets) as a treatment for adult patients with Kirsten rat sarcoma viral oncogene homolog (KRAS) mutant-type (mt) recurrent Low-Grade Serous Ovarian Cancer
(LGSOC), the expected outcome and benefits of collaborations, including with GenFleet Therapeutics (Shanghai), Inc. (GenFleet), including the conduct of a Phase 1/2a study and subsequent studies with respect to VS-7375, the potential of the results of
the RAMP 301 Phase 3 trial to confirm the results of the RAMP 201 study specific to KRAS mutant patients and to expand the indication for AVMAPKI FAKZYNJA CO-PACK regardless of KRAS mutation status, the structure and potential clinical value of our
completed, planned and pending clinical trials, the potential clinical value of various of the Company's clinical trials, including the RAMP 201, RAMP 201J, RAMP 205, RAMP 301 and VS-7375 trials, the timing of commencing and completing trials,
including topline data reports, our interactions with regulators, the timeline and indications for clinical development, regulatory submissions and the potential for and timing of commercialization of our product candidates and potential for additional
development programs involving the Company’s lead compound and the potential market opportunities thereof; and the estimated addressable markets for, and anticipated market opportunities of our drug candidates. The words "anticipate," "believe,"
"estimate," "expect," "may," "plan," "target," "potential," "would," "could," "should," "continue," “can” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying
words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement.
Forward-looking statements are subject to a number of risks and uncertainties including, but not limited to: the assumptions underlying the forward-looking statements; risks related to the development and successful commercialization of our product
candidates; obtaining and maintaining regulatory approvals, including, but not limited to, potential regulatory delays or rejections; the challenges with the commercialization of a new product; our history of operating losses and the possibility that we
may never achieve or maintain profitability; risks associated with meeting the objectives of Verastem's clinical trials, including, but not limited to Verastem's ability to achieve enrollment objectives concerning patient numbers (including an adequate safety
database), outcomes objectives and/or timing objectives for Verastem's trials; any delays or failures enrollment and the occurrence of adverse safety events; our ability to successfully commercialize AVMAPKI FAKZYNJA CO-PACK in the U.S. including our
ability to generate market demand for and acceptance of AVMAPKI FAKZYNJA CO-PACK; the potential inability to raise sufficient capital to fund ongoing operations as currently planned or to obtain financing on acceptable terms or to fund operations
from revenues generated by the sales of AVMAPKI FAKZYNJA CO-PACK; actions or advice of regulatory agencies to maintain regulatory approval of AVMAPKI FAKZYNJA CO-PACK; the impact of current and future healthcare reforms, including those
affecting the delivery of or payment for healthcare products and services; uncertainties related to the activities and initiatives of the current U.S. presidential administration, including regulatory and policy changes that may adversely affect our business;
risks related to our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; decisions by regulatory authorities regarding trial design, labeling and other matters that could affect the timing,
availability or commercial potential of our product candidates; whether preclinical testing of our product candidates and preliminary or interim data from clinical trials will be predictive of the results or success of ongoing or later clinical trials; that the
timing, scope and rate of reimbursement for our product candidates is uncertain; that the market opportunities of our drug candidates are based on internal and third-party estimates which may prove to be incorrect; that third-party payors (including
government agencies) may not reimburse; that there may be competitive developments affecting our product candidates; that data may not be available when expected; that enrollment of clinical trials may take longer than expected; the risks that we will
not satisfy our post-marketing requirements and commitments established and agreed to as part of the FDA's approval of AVMAPKI FAKZYNJA CO-PACK; that our marketed product candidates may cause adverse safety events and/or unexpected
concerns may arise from additional data or analysis, or result in unmanageable safety profiles as compared to their levels of efficacy; that we may not be able to confirm the results from the RAMP 201 study or expand the approved indication for AVMAPKI
FAKZYNJA CO-PACK; that our product candidates may experience manufacturing or supply interruptions or failures; that any of our third-party contract research organizations, contract manufacturing organizations, clinical sites, or contractors, among
others, who we rely on may fail to fully perform; that we face substantial competition, which may result in others developing or commercializing products before or more successfully than we do which could result in reduced market share or market
potential for our product candidates; that we may be unable to successfully initiate or complete the clinical development and eventual commercialization of our product candidates; that the development and commercialization of our product candidates
may take longer or cost more than planned, including as a result of conducting additional studies or our decisions regarding execution of such commercialization; that we may not attract and retain high quality personnel; that we or Pfizer, Inc. may fail to
fully perform under the license agreement covering certain Pfizer FAK inhibitors, including defactinib; that we or Chugai Pharmaceutical Co., Ltd. may fail to fully perform under the avutometinib license agreement; that we or GenFleet may fail to fully
perform under the collaboration and option agreement covering VS-7375 and other assets we may decide to option in; that our total addressable and target markets for our product candidates might be smaller than we are presently estimating; that we
or Secura Bio, Inc. may fail to fully perform under the asset purchase agreement with Secura Bio, Inc., including in relation to milestone payments; that we may not be able to establish new or expand on existing collaborations or partnerships, including
with respect to in-licensing of our product candidates, on favorable terms, or at all; that we may be unable to obtain adequate financing in the future through product licensing, co-promotional arrangements, public or private equity, debt financing or
otherwise; that we may not pursue or submit regulatory filings for our product candidates; that, due to the current presidential administration's significant reduction in the FDA's workforce and potential reductions to the FDA's budget, we may
experience a material impact to the FDA's ability to engage in a variety of activities that may affect our business, including routine regulatory and oversight activities; and that our product candidates may not receive regulatory approval, become
commercially successful products, or result in new treatment options being offered to patients.
Other risks and uncertainties include those identified under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 04,
2026, and in any subsequent filings with the SEC, which are available at www.sec.govand www.verastem.com. The forward-looking statements in this presentation speak only as of the original date of this presentation, and we undertake no obligation to
update or revise any of these statements whether as a result of new information, future events or otherwise, except as required by law. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential
investors, and others should give careful consideration to these risks and uncertainties.
USE OF NON-GAAP FINANCIAL MEASURES
This presentation contains references to our non-GAAP operating expense, a financial measure that is not calculated in accordance with generally accepted accounting principles in the US (GAAP). This non-GAAP financial measure excludes certain
amounts or expenses from the corresponding financial measures determined in accordance with GAAP. 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 this
measure, 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, this non-GAAP financial
measure is 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 this non-GAAP financial measure and the most comparable GAAP financial measure are included in the footnotes to the slides in this presentation on which
such non-GAAP number appears.
THIRD-PARTY SOURCES
Certain information contained in this presentation, including industry and market data and other statistical information, relates to or is based on studies, publications, surveys and other data obtained from third-party sources and the Company’s own
internal estimates and research. While the Company believes these third-party sources to be reliable as of the date of this presentation, it has not independently verified, and makes no representation as to the adequacy, fairness, accuracy or
completeness of, any information obtained from third-party sources. In addition, all of the market data included in this presentation involves a number of assumptions and limitations, and there can be no guarantee as to the accuracy or reliability of
such assumptions.
Disclaimers |
| 
| 3
OUR COMMERCIAL
PRODUCTS
MONOTHERAPY & COMBINATION APPROACHES
RAS: Rat Sarcoma Virus; MAPK: Mitogen-Activated Protein Kinase; RAF: Rapidly Accelerated Fibrosarcoma; MEK: Mitogen-Activated Extracellular Signal-regulated Kinase; FAK: focal adhesion kinase;
KRAS: Kirsten Rat Sarcoma Virus
Please see the full Prescribing Information for more information
OUR FOCUS
To expeditiously develop and deliver transformative therapies that truly change outcomes
for people living with RAS/MAPK pathway-driven cancers.
Avutometinib
RAF/MEK Clamp
Defactinib
FAK Inhibitor
VS-7375
KRAS G12D (ON/OFF)
Inhibitor
Verastem Oncology: Tackling Challenging Cancers with Novel Therapies |
| 
| 4
FDA: Food and Drug Administration
CLINICAL-TO-COMMERCIAL SUCCESS
in bringing novel RAS/MAPK
pathway-targeted therapies
from development to FDA
approval to
commercialization
INNOVATIVE
PIPELINE
with a potential best-in-class KRAS G12D asset
targeting the most
prevalent KRAS mutation in
human cancers
SCALABLE
ORGANIZATION
to maximize future
oncology development
programs and launches
Well Positioned to Deliver Continued Commercial Success and a Potential
Best-in-Class Treatment for Long-term Growth
OUR ADVANTAGE:
4 |
| 
| 5
TARGET
RAS DIRECTLY
TARGET THE
PATHWAY
DOWNSTREAM
TARGET THE PARALLEL
PATHWAY THAT
DRIVES RESISTANCE
First novel/novel
combination therapy,
targeting the
RAS/MAPK pathway,
approved in oncology
Precision Targeting of RAS/MAPK-Driven Cancers Differentiates Our Science
OUR SCIENTIFIC STRATEGY:
EGF: Epidermal Growth Factor; EGRF: Epidermal Growth Factor Receptor; ERK: Extracellular Signal-regulated Kinase; PI3K: Phosphatidylinositol 3-Kinase; AKT: Protein Kinase B; mTOR: Mammalian
Target of Rapamycin; YAP: Yes-Associated Protein; TEAD: Transcriptional Enhanced Associate Domain; MYC: Myelocytomatosis oncogene |
| 
| 6 ICT: investigator choice of treatment; LGSOC: Low-grade Serous Ovarian Cancer; mPDAC: metastatic Pancreatic Ductal Adenocarcinoma; mNSCLC: metastatic Non-small cell lung cancer; mCRC:
metastatic colorectal cancer; FAKi: focal adhesion kinase inhibitor; Gem: Gemcitabine. NabP: nab-paclitaxel; EGFR: epidermal growth factor receptor
Not shown: RAMP201J trial by Japanese Gynecologic Oncology Group sponsored by Verastem.
*GenFleet Therapeutics has an ongoing Phase 1/2 and Phase 3 clinical trials in China with VS-7375, known as GFH375 in China. GenFleet retains greater China rights. Verastem has two undisclosed
assets at discovery phase targeting RAS/MAPK pathway-driven cancers as part of the GenFleet collaboration.
Asset Line of Therapy Phase Upcoming Milestones
Avutometinib, RAF/MEK Clamp + Defactinib, FAKi
Avutometinib
+ Defactinib Recurrent LGSOC
Avutometinib
+ Defactinib vs ICT Recurrent LGSOC Expect to report topline readout of primary
endpoint by mid-2027
VS-7375, oral KRAS G12D (ON/OFF) inhibitor
VS-7375 monotherapy +
various combinations
KRAS G12D-mutated
solid tumors (advanced solid
tumors)
Ongoing enrollment; report update in
October 2026
VS-7375 monotherapy
+ EGFR combination
2L mPDAC; 1L PDAC
combination Expect LPI by end of 2026
VS-7375 monotherapy
2L/3L advanced NSCLC; 2L+
asymptomatic untreated brain
mets
Expect LPI by end of 2026
VS-7375 combinations
w/ EGFR & Chemotherapy
2L+ mCRC; 1L mCRC
combination Expect LPI by end of 2026
VS-7375 combinations 1L mPDAC Expect FPI in 1H 2027
VS-7375 combination 1L advanced NSCLC Expect FPI in 1H 2027
VS-7375 combination 1L mCRC Expect FPI in 1H 2027
Multi-Faceted & Targeted Approaches to Address RAS/MAPK-Driven Cancers
OUR PIPELINE:
RAMP 201: Phase 2 Registration Directed Trial;
Accelerated FDA Approval May 2025
RAMP 301: Phase 3 International Confirmatory Trial
TARGET-D 101: Phase 1/2
dose escalation & expansion
TARGET-D 201: Phase 2 registration directed trial
TARGET-D 202: Phase 2 registration directed trial
TARGET-D 203: Phase 3 registration directed trial
TARGET-D 301: Phase 3 pivotal trial (planned)
TARGET-D 302: Phase 3 pivotal trial (planned)
TARGET-D 303: Phase 3 pivotal trial (planned) |
| 
| 7
✓ Dosed first
patient in
TARGET-D 201
(PDAC)
Registration-Directed Trial
June 2026
• Complete
enrollment across
all three
TARGET-D Phase
2 Trials
• Report an update
on VS-7375
across tumor types
in October 2026
• Q3 earnings call
Mid-2026 2H 2026 End of 2026 1H 2027
• Enroll first
patient in each of
the Phase 3
TARGET-D pivotal
trials (PDAC, CRC,
NSCLC)
• Q4/FY26 & Q1
earnings calls
✓ Dosed first
patient in
TARGET-D 202
(NSCLC)
& TARGET-D 203
(CRC) Registration-
✓ Directed Trials Completed
target enrollment
in TARGET-D 101
PDAC, NSCLC,
and CRC cohorts
Upcoming Catalysts/Milestones
OUR MILESTONES: |
| 
| 8
Commercially Launched
in the U.S. for KRAS-mutated Recurrent LGSOC
FDA APPROVAL DATE: MAY 8, 2025 |
| 
| 9
70% of LGSOC Tumors are Driven by the RAS/MAPK Pathway;
~30% of These Have a KRAS Mutation1,2,3,4
• Avutometinib inhibits MEK kinase activity while
blocking the compensatory reactivation of MEK
by upstream RAF5,6,7
• Blocking RAF and/or MEK activates FAK, a key
mediator of drug resistance8,9
• Defactinib, a FAK inhibitor, inhibits parallel
pathway signaling10,11,12
• Together, avutometinib plus defactinib offer
more complete blockade of the signaling that
drives the growth of RAS/MAPK pathway-dependent tumors
RAF-MEK
Complex
The Combination of Avutometinib and Defactinib
Induces Deeper Inhibition of Tumor Growth
1. AACR Genie v16.1; 2. Cheasley et al., J Pathol 2021; 3. Thomson et al., Gynecol Oncol 2023;
4. Gershenson et al., Gynecol Oncol 2022; 5. Coma et al., AACR 2022; 6. Ishii et al., Cancer Res, 2013;
7. Lito et al., Cancer Cell, 2014; 8. Lubrano et al., AACR 2024; 9. Banerji et al., AACR 2020; 10. Jones et al.,
Invest New Drugs 2015; 11. McNamara et al., Gynecol Oncol 2024; 12. Banerjee et al., ASCO 2023
ERK: Extracellular Signal-regulated Kinase; FAK: Focal Adhesion Kinase; MEK, Mitogen-Activated
Extracellular Signal-regulated Kinase; mTOR: Mammalian Target of Rapamycin; P: Phosphate; PI3K:
Phosphatidylinositol 3-Kinase; RAF: Rapidly Accelerated Fibrosarcoma; RAS: Rat Sarcoma Virus; RhoA: Ras
Homolog Family Member A; RTK: Receptor Tyrosine Kinase; YAP: Yes-Associated Protein. |
| 
| 10
High Unmet Need for an Effective and
Tolerable Therapy in Recurrent LGSOC
• U.S. annual incidence: ~1,000-2,0001 and
prevalence: 6,000-8,0002
• Affects younger women with bimodal peaks
of diagnosis between the ages of 20-30 and
50-60
– Disproportionately impacts health, fertility and
long-term quality of life3,4
• 80-90% of patients will experience a
recurrence5
• Standard of care offers low to moderate
response rates (6-13%)6,7,8
When you get told that you have a recurrence,
the mental load is a lot. You’re thinking, okay,
what did I have to do for treatment the first
time? Now I have to repeat that. And will there
even be something available for me to take for a
second, or a third recurrence?
- Amanda, real patient living with recurrent LGSOC;
diagnosed at 26 with LGSOC
1.Verastem DOF; 2. US Cancer Statistics. Accessed 2024; 3. Slomovitz Gynecol Oncol 2020;
4. Manning-Geist B et al. Clin Cancer Res 2022;28(20):4456-4465; 5. Babaier 2022/p1/para1/ln6,7;
6. Gershenson Gynecol Oncol 2022; 7. Slomovitz Gynecol Oncol 2020; 8. Monk 2020/p3758/table2/footnote-b; 10 |
| 
| 11
Individual patient experiences may vary. Not all patients treated with AVMAPKI® FAKZYNJA® CO-PACK will experience the same results.
Please see the full Prescribing Information for more information
Mary, 52, real patient treated with AVMAPKI FAKZYNJA
CO-PACK for recurrent KRAS-mutated LGSOC
• 2019 - Diagnosed at 48 with Stage
3 KRAS-mutated LGSOC
• Treated with surgery, chemotherapy
and AI; no evidence of disease for
~4 years
• 2023 - LGSOC recurred & enrolled
in the RAMP 201 trial
• Dose interruptions effectively
managed adverse events, allowing
return to full-dose treatment
• Complete response after 2 years
of treatment with AVMAPKI
FAKZYNJA CO-PACK
Real World Experience: From Recurrence to Complete Response with
AVMAPKI FAKZYNJA CO-PACK |
| 
| 12
AVMAPKI FAKZYNJA CO-PACK: Focused Execution Will Drive Sustained Growth
Please see the full Prescribing Information for more information
✓ New patient starts
✓ Repeat prescribing
✓ Depth of prescribing among existing
accounts
✓ Use in earlier-line patients
✓ Increased refills
Consistent
New Patient
Starts
Use at
First or Next
Recurrence
Help
Patients Stay
on Therapy |
| 
| 13
AVMAPKI FAKZYNJA CO-PACK: Steady Revenue Growth Since Launch
$25.1M in Net Product Revenue in Q2’26
Achieved ~$74.5M in AVMAPKI FAKZYNJA CO-PACK
Net Product Revenue Since Launch in May 2025*
Please see the full Prescribing Information for more information
$11.2M
$17.5M
N E T P R O D U C T R E V E N U E *
$18.7M
Q3’25 Q4’25 Q1’26
$25.1M
Q2’26
*Not shown: Q2 2025 not a full quarter ($2.1M); May 8, 2025 FDA approval
Verastem DOF; US dollars in millions. Refer to press release issued on August 6, 2026 for full financial details. |
| 
| 14
RAMP 201: Demonstrated Durable Results Across Various Efficacy Measures in
Heavily Pretreated US & European Patients With and Without a KRAS Mutation
Avutometinib + Defactinib Regimen: Best Overall Response
82% of All Patients Had a Reduction in Target Lesions,
Regardless of KRAS Status1
All Patients1 KRAS mt KRAS wt
ORR % 31% 44% 17%
DoT, mean 14.5 months 18 months 11 months
DoR, median 31 months 31 months 9 months
PFS, median 13 months 22 months 13 months
DCR at 6 or
more months 61% 70% 50%
Discontinuation Rate
Due to AEs 10%
1. RAMP 201 data cut off as of June 30, 2024; 2. RAMP 201 Long-Term Efficacy and Safety August 2025 data cut off presented at SGO 2026; 3. Japan Society of Gynecologic Oncology
ORR: Objective Response Rate; DoT: Duration of Treatment; mDoR: median Duration of Response; mPFS: media Progression-free Survival; DCR: Disease Control Rate; AE: adverse event
Avutometinib + Defactinib Regimen: Best Overall Response
RAMP 201 Long-Term Data Median Follow-Up of 2 Years2
:
• More than half of responders (56%) remain in response at 24 months
• KRAS mt patients: mDoR remains at 31.1 months and mPFS is 19.6 months
• KRAS wt patients: mDoR is now 12 months and mPFS is 12.7 months
• The discontinuation rate due to AEs remains low (12%) even with extensive follow up
New RAMP 201 Data @ SGO 2026 New RAMP 201 Japan Data @ JSGO 2026
RAMP 201J Presented at Japan SGO Meeting July 20263
:
• KRAS mt patients: mDoR remains at 31.1 months and mPFS is 19.6 months
• KRAS wt patients: mDoR is now 12 months and mPFS is 12.7 months
• The discontinuation rate due to AEs remains low (12%) even with extensive follow up
Positive initial data from Japan RAMP 201J study |
| 
| 15
OS
PFS by RECIST v1.1
per INV assessment
ORR
DoR
DCR
Safety
Pharmacokinetics
PROs
a Unless otherwise specified, all tumor
response-based endpoints will be
analyzed using both BICR and INV
assessments
*US FDA analysis plan will evaluate PFS independently in KRAS-mt and KRAS wt LGSOC; BID: twice a day; BIW: twice a week; DCR: disease control rate; DoR: duration of response; INV: investigator;
KRAS: kirsten rat sarcoma virus; MEKi: MEK inhibitor; mt: mutant; PO: per oral; pts, patients; ORR: objective response rate; OS: overall survival; PD: progressive disease; PFS: progression-free survival;
PROs: patient-reported outcomes; RECIST: response evaluation criteria in solid tumors; wt: wild type. BICR: blinded independent central radiological review
• Expect to report topline primary endpoint of PFS by mid-2027
• Similar entry criteria to RAMP 201 patient population: KRAS mt and KRAS wt recurrent LGSOC
• Study sites include the U.S., Canada, UK, Europe, Australia, New Zealand, Japan and South Korea
• Recurrent disease after prior
platinum therapy
• Documented KRAS mutation status
• Measurable disease per RECIST v1.1
• Confirmed LGSOC diagnosis
• Prior MEKi allowed
• Prior bevacizumab allowed
RAMP 301 (GOG-3907/ENGOT-ov81/GTG-UK): NCT06072781
Pegylated Liposomal Doxorubicin
Paclitaxel
Letrozole
Anastrozole
Investigator’s Choice
n = 135
Avutometinib 3.2 mg PO BIW
Defactinib 200 mg BID
3 weeks on, 1 week off
Avutometinib + Defactinib
n = 135
May cross over upon
BICR-confirmed PD
PFS (BICR by RECIST v1.1)
Hierarchical Evaluation of PFS*:
KRAS mutant LGSOC
All recurrent LGSOC
KRAS wt LGSOC
Primary Endpoint:
Secondary Endpointsa
1:1 Randomization
n = 270
Stratification Factors:
• KRAS mutation status
(wt vs. mt)
• Geography (N.
America/EU) vs. ROW
• Number of prior
therapies (1-3 vs.
4 or more)
Inclusion Criteria
RAMP 301: International Phase 3 Confirmatory Trial of
Avutometinib + Defactinib in Recurrent LGSOC |
| 
| 16
U.S.
Secured FDA
Accelerated Approval
in KRAS-mutated
recurrent LGSOC.
2029
2025
RAMP 301:
Topline data expected in
mid-2027.
U.S. Label Expansion
Leverage the RAMP 301
results to confirm the initial
indication and expand the
indication regardless of
KRAS mutation status.
Japan:
Leverage the results from
RAMP 201J & RAMP 301 for
potential approval in both
KRAS mutant and
wild type recurrent LGSOC.
Europe:
Leverage results from
RAMP 301 for potential
approval in both KRAS
mutant and wild type
recurrent LGSOC.
2028
2027
KRAS mt represents ~33% of the population, while the wt represents 67% and therefore there are many more addressable patients.
Potential for Label and Geographic Expansion
AVMAPKI FAKZYNJA CO-PACK Future Commercial Opportunity |
| 
| 17
VS-7375,
Oral KRAS G12D
(ON/OFF) Inhibitor |
| 
| 18
PANCREATIC CANCER
40%
COLORECTAL CANCER
15%
LUNG CANCER
5%
KRAS G12D: The Most Prevalent Mutation in Cancer with Poor Prognosis
and High Unmet Need
Annual U.S. TAM:
~29K
Annual U.S. TAM:
~22K
Annual U.S. TAM:
~10K
CancerMPact Patient Metrics Active Disease calculation for Stage IV patients; TAM: Total Addressable Market; Note: TAM calculation is based on applying biomarker rate to active
disease estimate. Ref: Lee et al Nature, Dec 2022 for epidemiology; 1 McIntyre et al., Cancer Cell 2024, 42:1614-1629 and Hafezi S et al., Int J Mol Sci 2021, 22(19):10219; 2: : Lee J, et
al. J Clin Med. 2020;9(12):3863; 3: Judd J, Abdel Karim N, Khan H, et al. Mol Cancer Ther. 2021;20(12):2577-2584;4
KRAS G12D mutation in pancreatic cancer
correlates with worse outcomes, shorter
survival and a higher risk of progression1
KRAS G12D mutation is a significant driver
in lung cancer, especially among non-smokers, and is linked to poor responses to
SOC3
KRAS G12D mutation in CRC is often linked
to more aggressive tumors2
VS-7375: Opportunity to Address a Large Patient Population Across KRAS G12D-Mutated Cancers |
| 
| 19 Pachter et al., Targeting RAS 2nd edition 2025; Ai et al., ASCO 2025; Li et al., World Conference on Lung Cancer 2025; pERK: phosphorylated Extracellular signal-regulated
Kinase; GEF: Guanine nucleotide exchange factor GAP: GTPase-activating protein
VS-7375: Potential Best-in-Class G12D Inhibitor for Advanced KRAS G12D-Mutated Cancers
D I F F E R E N T I A T E D P R O F I L E V S . O T H E R R A S I N H I B I T O R S
Dual potent inhibition of both ON and OFF states of KRAS G12D
Correlates with better in vivo efficacy and durability vs. ON-only (tricomplex) RAS
inhibitors
High affinity for KRAS G12D with long residence time (18-24hrs)
Correlates with more rapid and durable suppression of pERK signaling vs. zoldonrasib in
tumor cell lines
Selective inhibition of KRAS G12D
Spares T cell proliferation in contrast to RAS-Multi inhibitor (e.g., daraxonrasib), which
impairs T cell proliferation
Once daily dose-proportional oral dosing in patients
Enables exposures in all patients corresponding to maximal tumor regressions across
preclinical models |
| 
| 20
VS-7375: Well-Positioned to Compete as Best-in-Class KRAS G12D Inhibitor in
Pancreatic, Colorectal and Lung Cancer Markets
Potential best-in-class KRAS G12D
(ON/OFF) inhibitor
Differentiated
Profile
Efficacy at both
600 mg QD and
900 mg QD in PDAC,
NSCLC, and CRC
Anti-tumor
Activity Across
Tumors
Low-grade GI
side effects that
attenuate after the
first cycle
Favorable
Tolerability
Profile
Combinable
with anti-EGFR
therapy and SOC
chemotherapy
Broad
Combination
Potential
Potential for
Accelerated
Approval pathway
Defined
Development
Path
PDAC: Pancreatic Ductal Carcinoma; NSCLC: Non-small Cell Lung Cancer; CRC: colorectal cancer; GI: gastrointestinal; EGFR: Epidermal Growth Factor Receptor; GI:
gastrointestinal; QD: daily; SOC: Standard of Care |
| 
| 21
VS-7375: Competitive Profile Emerging Across Pancreatic, Colorectal and
Lung Cancers
AE: adverse event; Gem/NabP: gemcitabine, nab-paclitaxel; 1L: first-line; 1H: first-half
Safety Profile
Significantly Improved
After Cycle 1
Combinability with
SOC Therapies
Multiple Paths
to Registration
• Clinical activity at multiple doses in
pancreatic, colorectal and lung
cancers
• Dose-response efficacy in PDAC
• Potential for chemo-free regimen in PDAC
• Favorable mono and combination
tolerability
• No clinically meaningful drug-related
liver or hematologic toxicity observed
• Primarily low-grade GI AEs that improve
after cycle 1
• No acneiform rash; no stomatitis / muco-sitis observed with VS-7375 monotherapy
• Successfully combined with
cetuximab & full dose and schedule of
Gem/NabP
• Evaluating higher-dose combinations
• Multiple novel combination opportunities
• Three Phase 2 trials underway for
potential Accelerated Approval
• Expect to initiate three Phase 3 trials in
1L setting by 1H 2027
Anti-tumor Activity
Across Tumor Types |
| 
| 22
VS-7375: Enrolled 190+ Patients Across Dose Escalation & Expansion Cohorts
Phase 1/2 Study Evaluating VS-7375, a KRAS G12D (ON/OFF) inhibitor, as
Monotherapy and in Combination, in Patients with KRAS G12D-Mutated Solid Tumors
The study is dosing with meals and using prophylactic antiemetics
NCT07020221; DL: dose level; 2L: second-line; 3L: third-line
Monotherapy
Dose Escalation
&
Expansion
DL2:
600 mg QD
DL3:
900 mg QD
DL1:
400 mg QD
2L+ Tumor
Agnostic
Solid Tumors
2L/3L
NSCLC
2L
PDAC
DL4:
1200 mg QD
Combination
Dose Escalation
&
Expansion
VS-7375 +
Cetuximab
2L+ CRC and
1L, 2L PDAC
VS-7375 + Carbo/
Pemetrexed/Pembro
1L NSCLC
VS-7375 +
Gem/NabP
1L and 2L+
PDAC
Dose-Level Cleared
Ongoing
Completed target enrollment in PDAC, CRC, and NSCLC cohorts in June 2026 |
| 
| 23
900 mg QD of VS-7375 Achieves the Targeted Human AUCss in the Majority of
Patients Corresponding to Maximal Tumor Regression Across Mouse Models
400mg QD 600mg QD 900mg QD
Equivalent exposure to mice at 100 mg/kg
(PR in almost all mice in 4 tumor models)
Equivalent exposure to mice at 10 mg/kg
(Tumor regression in sensitive tumor models)
Equivalent exposure to mice at 30 mg/kg
(Tumor regression in all tumor models)
VSTM DOF June 2026; AUCss: area under the curve at steady state; PR: partial response; KG: kilogram
T A R G E T- D 1 0 1
N=11 N=73 N=24
40 mg QD
60 mg QD
90 mg QD
10
100
1000
10000
TARGET-D 101
VS-7375 (ng*h/mL)
N=11 N=73 N=24 |
| 
| 24
14 patients dosed at 900 mg QD had
elevated levels of CA19-9 at baseline (>37
U/mL) and at least one scheduled on-treatment measurement
– All patients remain on treatment
– Includes 2-4L patients
93% (13/14) of patients showed >50%
reduction in CA19-9
≥50% reduction in CA19-9 has been
correlated with improved PFS and OS for
patients with PDAC1,2
93% of mPDAC Patients Treated with VS-7375 900 mg QD Achieved >50%
Reduction in CA19-9
1NAPOLI-1, Wang-Gillam et al., European Journal of Cancer, 2019; 2ACCORD11/PRODIGE4, Robert et al., Oncology, 2017.
PFS: progression-free survival; OS: overall survival
Data cutoff: June 2026
0 6 12 18 24 30
-100
-50
0
Weeks
% Change in CA19-9
(from baseline)
0101-020
0102-004
0103-007
0104-012
0104-016
0105-021
0105-023
0105-026
0106-016
0110-007
0112-016
0116-003
0116-005
0116-008
On Treatment |
| 
| 25
VS-7375: Compelling Single Agent and Combination Responses in PDAC
Patients, As Early as Week 6
Week 12:
cPR (-47%) (SLD: 80mm)
Response ongoing
Baseline Lesion:
Liver, surgical bed,
peritoneum
(SLD: 151mm)
Week 6:
uPR (-100%) (SLD: 0mm)
Response ongoing
Baseline Lesion:
Mesentery, peritoneum
(SLD: 64mm)
Baseline Lesion:
Pleura, lung,
mediastinum lymph
node
(52mm)
Week 12:
cPR: (-70%) (16mm)
Confirmed Partial Response
at Week 12
Complete Resolution of Target
Lesion at Week 6
Deep and Rapid Response
Achieved at Subtherapeutic Dose
+ Anti-EGFR
VS-7375 900 mg QD Monotherapy in
55 y/o Male with mPDAC
VS-7375 900 mg QD Monotherapy in
79 y/o Female with mPDAC
VS-7375 400 mg QD + Anti-EGFR
in 64 y/o Male with mPDAC
SLD: sum of longest diameter; cPR: confirmed Partial Response; uPR: unconfirmed Partial Response;
Source for all scans: TARGET-D 101 investigators;
See appendix for full details. |
| 
| 26
VS-7375: Deep and Durable Tumor Responses Observed Across Lung and
Colorectal Cancers
Week 12:
SD: (-29.5%) (SLD: 260mm)
Baseline Lesion:
(SLD: 370mm)
CRC: Marked Reduction
of Disease Burden Week 12:
cPR (-49%) (SLD: 35mm)
Baseline Lesion:
(SLD 69mm)
VS-7375 600 mg QD in 77 y/o Female with
Advanced NSCLC
NSCLC: Confirmed Partial Response at Week 6 with
Deepening of Response Through Week 12
VS-7375 600 mg QD + Cetuximab in Heavily Pre-treated 42 y/o Male with mCRC
Source: TARGET-D 101 investigators; See appendix for further details. |
| 
| 27
LFT: Liver function test
VS-7375: Safety Summary: Favorable Tolerability Profile Across
Monotherapy and Combinations
Primarily
low-grade nausea,
vomiting, diarrhea
Nearly all
GI side effects
respond to standard
prophylactic
treatment
No unexpected
AEs and low
rates of
Grade 3 AEs
No clinically-meaningful
cytopenias, LFT
abnormalities,
stomatitis or rash
AE profile does
not show a dose-dependent pattern
Promising longer
term tolerability
data with no
clinically significant
cumulative
toxicities |
| 
| 28 TRAEs: treatment-related adverse events; Neutropenia' and 'Neutrophil count decreased' are grouped as 'Neutropenia’
VSTM: DOF, Data cutoff: June 12, 2026
VS-7375 Well-Tolerated Up To 900 mg; Low-Grade GI AEs Most Common
TRAEs reported in >1 patient
600 mg (N=57) 900 mg (N=25)
Gr. 1
n(%)
Gr. 2
n(%)
Gr. 3
n(%)
Gr. ≥4
n(%)
All Gr.
n(%)
Gr. 1
n(%)
Gr. 2
n(%)
Gr. 3
n(%)
Gr. ≥4
n(%)
All Gr.
n(%) System Organ Class / Preferred Term
Gastrointestinal disorders
Diarrhea 22 (39) 7 (12) 2 (4) 0 31 (54) 9 (36) 3 (12) 0 0 12 (48)
Nausea 20 (35) 8 (14) 1 (2) 0 29 (51) 9 (36) 3 (12) 1 (4) 0 13 (52)
Vomiting 16 (28) 4 (7) 1 (2) 0 21 (37) 5 (20) 1 (4) 0 0 6 (24)
Constipation 5 (9) 0 0 0 5 (9) 0 0 0 0 0
Dyspepsia 4 (7) 0 0 0 4 (7) 1 (4) 0 0 0 1 (4)
Abdominal distension 1 (2) 0 0 0 1 (2) 2 (8) 1 (4) 0 0 3 (12)
Abdominal pain 1 (2) 0 1 (2) 0 2 (4) 1 (4) 0 0 0 1 (4)
Flatulence 1 (2) 0 0 0 1 (2) 2 (8) 0 0 0 2 (8)
General disorders
Fatigue 14 (25) 3 (5) 0 0 17 (30) 5 (20) 3 (12) 0 0 8 (32)
Oedema peripheral 2 (4) 0 0 0 2 (4) 0 0 0 0 0
Investigations
Lipase increased 4 (7) 2 (4) 0 0 6 (11) 0 1 (4) 0 0 1 (4)
Amylase increased 3 (5) 1 (2) 0 0 4 (7) 1 (4) 0 0 0 1 (4)
Alanine aminotransferase increased 2 (4) 0 0 0 2 (4) 1 (4) 0 0 0 1 (4)
Blood and lymphatic system disorders
Neutropenia 2 (4) 3 (5) 1 (2) 0 6 (11) 0 2 (8) 0 0 2 (8)
Anemia 0 4 (7) 0 0 4 (7) 0 2 (8) 0 0 2 (8)
Leukopenia 2 (4) 0 0 0 2 (4) 0 0 0 0 0
Thrombocytopenia 1 (2) 0 0 0 1 (2) 1 (4) 0 0 0 1 (4)
White blood cell count decreased 1 (2) 1 (2) 0 0 2 (4) 0 0 0 0 0
Metabolism and nutrition disorders
Decreased appetite 0 2 (4) 0 0 2 (4) 4 (16) 1 (4) 0 0 5 (20)
Hypomagnesaemia 2 (4) 0 0 0 2 (4) 0 0 0 0 0
Nervous system disorders
Dysgeusia 3 (5) 0 0 0 3 (5) 2 (8) 0 0 0 2 (8)
Dizziness 1 (2) 0 1 (2) 0 2 (4) 0 0 0 0 0
Skin and subcutaneous tissue disorders
Pruritus 3 (5) 0 0 0 3 (5) 0 0 0 0 0
Rash 3 (5) 0 0 0 3 (5) 0 0 0 0 0
Urticaria 0 0 1 (2) 0 1 (2) 1 (4) 0 0 0 1 (4)
Renal and Urinary disorders
Acute kidney injury 0 0 1 (2) 0 1 (2) 0 1 (4) 0 0 1 (4) |
| 
| VSTM: DOF, Data cutoff: June 12, 2026 29
Major TRAEs Largely Attenuated After Cycle 1 on Both VS-7375 600 mg QD
& 900 mg QD
600 mg
(N=51)
900 mg
(N=22)
System Organ Class / Preferred Term Gr. 1
n(%)
Gr. 2
n(%)
Gr. 3
n(%)
Gr. ≥4
n(%)
All Gr.
n(%)
Gr. 1
n(%)
Gr. 2
n(%)
Gr. 3
n(%)
Gr. ≥4
n(%)
All Gr.
n(%)
Gastrointestinal disorders
Diarrhea 6 (12) 4 (8) 2 (4) 0 12 (24) 4 (18) 0 0 0 4 (18)
Nausea 4 (8) 5 (10) 0 0 9 (18) 2 (9) 0 1 (5) 0 3 (14)
Vomiting 7 (14) 3 (6) 0 0 10 (20) 2 (9) 0 0 0 2 (9)
Investigations
Amylase increased 3 (6) 1 (2) 0 0 4 (8) 0 0 0 0 0
Lipase increased 3 (6) 1 (2) 0 0 4 (8) 0 0 0 0 0
Nervous system disorders
Dizziness 1 (2) 0 1 (2) 0 2 (4) 0 0 0 0 0
Dysgeusia 2 (4) 0 0 0 2 (4) 1 (5) 0 0 0 1 (5)
Blood and lymphatic system disorders
Neutropenia 1 (2) 3 (6) 1 (2) 0 5 (10) 0 0 0 0 0
Anemia 0 3 (6) 0 0 3 (6) 0 1 (5) 0 0 1 (5)
Leukopenia 2 (4) 0 0 0 2 (4) 0 0 0 0 0
General disorders
Fatigue 4 (8) 2 (4) 0 0 6 (12) 0 1 (5) 0 0 1 (5)
Metabolism and nutrition disorders
Decreased appetite 0 2 (4) 0 0 2 (4) 1 (5) 0 0 0 1 (5)
Hypomagnesaemia 2 (4) 0 0 0 2 (4) 0 0 0 0 0
TRAEs reported in >1 patient after cycle 1 (≥29 days); only include patients followed up for at least 29 days |
| 
| 30
*All A1 patients with SD allowed to crossover to cetuximab combo if A1 fails to meet efficacy threshold
Maximizing the Opportunity in PDAC Through VS-7375
Monotherapy and EGFR Combination Strategies
R 1:1 randomization
Part A: 2L PDAC
Cohort
A1
Cohort
A2
VS-7375 900 mg
monotherapy
(N=20)
VS-7375 900 mg +
Cetuximab
(N=20)
Cohort
B1
Cohort
B2
VS-7375 900 mg
monotherapy
(N=60)
VS-7375 900 mg +
Cetuximab
(N=60)
Part B: 2L PDAC Expansion
Cohort(s) that meet
efficacy threshold*
Part C: 1L PDAC
VS-7375 900 mg + Cetuximab
(N=25)
Study Population Key Endpoints Next Key Milestone
Part A & B: 2L KRAS
G12D-mutated PDAC
Part C: 1L KRAS
G12D-mutated PDAC
Part A, B, C:
Primary: ORR by BICR
Secondary: DOR
Expect to complete
enrollment by end of
2026
FPD: First patient dosed; LPD: Last patient dosed; EGFR: Epidermal Growth Factor Receptor
FPD in June
2026 |
| 
| 31
Evaluating VS-7375 Monotherapy in Advanced
NSCLC, Including Patients with Brain Metastases
Cohort A:
Confirm
900 mg QD is the
go-forward dose
Part A: 2L/3L NSCLC
VS-7375 900 mg QD
(N=20) Cohort B: Preferred dose of VS-7375
(N=60)
Part B: 2L/3L NSCLC
Part C: 2L/3L/4L
VS-7375 900 mg QD
with asymptomatic untreated brain
metastasis
(N=25)
Study Population Key Endpoints Next Key
Milestone
Parts
A & B:
• Prior treatment with platinum-based chemo and ICI
• At least 1 and no more than 2
prior systemic lines of therapy
Primary: ORR by BICR
Secondary: DOR Expect to
complete
enrollment by
end of 2026
Part C:
Received at least 1 and no more
than 3 prior systemic lines
of therapy
Intracranial ORR by
mRECIST v1.1 by BICR
FPD in July
2026 |
| 
| 32
#No prior TAS-102 (trifluridine and tipiracil) or regorafenib. VEGFi as appropriate.
Advancing a VS-7375 Combination Strategy with
EGFR Inhibitors and Chemotherapy in Metastatic CRC
Cohort
A1:
R
Confirm
900 mg QD is the
go-forward dose
2:1 randomization
Cohort
A2:
Part A: 2L+ CRC
VS-7375 900 + Cetuximab or
Panitumumab
(N=40)
VS-7375 900 mg monotherapy
(N=20)
Cohort B:
Preferred Regimen:
VS-7375 + Cetuximab or
Panitumumab
OR
VS-7375 monotherapy
(N=60)
Part C: 1L CRC
VS-7375 + Cetuximab + mFOLFOX6
(N=30)
Part B: 2L+ CRC
mFOLFOX: modified oxaliplatin, leucovorin, and 5-fluorouracil; VEGFI: vascular endothelial Growth Factor
Study Population Key Endpoints Next Key Milestone
Part
A, B:
Prior SoC: fluoro-pyrimidine, irinotecan,
oxaliplatin, VEGFi#
Primary: ORR by
BICR
Secondary: DOR
Expect to complete
enrollment by end of
2026
Part C: No prior tx for
metastatic disease Safety/Tolerability
FPD in July
2026 |
| 
| 33
VS-7375: Leveraging Potential Best-in-Class Profile to Pursue Parallel
Clinical Development Paths
*Subject to discussions with the FDA.
Potential opportunities based on data from ongoing clinical trials; GNP: gemcitabine and nab-paclitaxel; aNSCLC: advanced non-small cell lung cancer; mCRC: metastatic
colorectal cancer
2L mPDAC mono & EGFR Combo
1L mPDAC EGFR Combo
1L mPDAC EGFR Combo &
GnP Combo*
2L advanced NSCLC Mono
1L advanced NSCLC Brain Mets Mono
1L advanced NSCLC
Chemo Combo*
2L mCRC EGFR Combo
1L mCRC EGFR + Chemo Combo
1L mCRC EGFR + Chemo Combo*
Pancreatic
Cancer
Colorectal
Cancer
Lung
Cancer
Clinical Approaches:
• Earlier lines
• New combinations with PRMT5i,
PanRASi
Regulatory Engagement:
• Breakthrough Therapy
Designations
• Potential Accelerated Approval
Pathway
✓ FDA granted Fast Track Designations in advanced/metastatic PDAC
✓ FDA granted Fast Track Designation in advanced/metastatic NSCLC
Now Next Future |
| 
| RAMP 205:
Avutometinib + Defactinib
+ Standard-of Care in First-Line
Metastatic Pancreatic Cancer |
| 
| 35
RAMP 205: 86% 6-month Overall Survival Rate, Follow-up Continues and
Survival Data Continue to Mature
• 90% of patients presented with metastatic disease (Stage IV) at diagnosis
• At the RP2D (DL1), the majority (83%) of patients experienced tumor shrinkage
• 9 patients remain on therapy, follow up continues
• Adverse events remained generally consistent with previously reported safety and tolerability profile,
with no new safety signals observed
RAMP 205
Dose Level 1
9.8 months median follow-up
GnP in IL mPDAC
NAPOLI-31
N = 29 N= 387
ORR % (n) Confirmed: 52% (15/29) Confirmed: 36.2% (140/387)
6 months PFS % (95% CI) 68% (45, 82) 43% (38, 49)
6 months OS % (95% CI) 86% (47,99) 68% (64,73)
RP2D DL1: Avutometinib 2.4 mg twice weekly (BIW), defactinib 200 mg twice daily (BID) for 3 weeks
on and one week off, and gemcitabine (800 mg/m2
) plus nab-paclitaxel (125 mg/m2
) administered
on Days 1, 8, and 15 of each 28-day cycle.
1. Wainberg Z, Melisi D, Macarulla T et al. NALIRIFOX versus nab-paclitaxel and gemcitabine in treatment-naive patients with metastatic pancreatic ductal
adenocarcinoma (NAPOLI 3): a randomized, open-label, phase 3 trial; The Lancet, 2023; 402, 1272-1281
VSTM DOF: As of June 5, 2026 data cutoff |
| 
| Financials
36 |
| 
| 37
Oberland Finance Credit Facility
• $75M principal of notes outstanding
– Floating interest rate, subject to a floor and a cap
– Interest only payments through January 2031
Oberland Revenue Notes
• Up to $75M in synthetic royalty revenue notes
– $50M funded at closing in August 2026
– $25M available at Company’s option upon quarterly sales of AVMAPKI FAKZYNJA CO-PACK of at least $40M by Q1 2027
– Royalty rate of 4.50%, reduces to 1.75% upon paying previously funded amounts in royalties prior to December 31, 2031
– Maturity Date of June 2033
No financial covenants
Proforma cash of $201.4M with Oberland and COPIKTRA sales milestone, combined with expected product revenue and
future tranche from Oberland facility, expected to extend cash runway into 2H 2027
Financial Summary
($ in millions)
Three Months
Ended June 30, 2026
Net Product Revenue $25.1M
GAAP Operating Expenses $72.8M
Non-GAAP Operating Expenses $69.1M*
As of June 30, 2026
Cash, cash equivalents & short-term investments $136.4M
Pro-Forma cash, cash equivalents & short-term investments $201.4M**
Shares Outstanding 90.5M***
Q2 2026 Financial Highlights
* Three months ended June 30, 2026 GAAP operating expenses of $72.79M less Q2 2026 stock-based compensation expense of $3.43M less amortization of intangible assets of $0.28 = $69.08M
Q2 2026 non-GAAP operating expenses.
** Pro-Forma amount includes receipt of $15M COPIKTRA milestone in July 2026 and $50M upon closing of Oberland royalty facility in August 2026
*** Excludes unexercised pre-funded warrants (9.7M shares upon exercise). |
| 
| EU: European Union ; *Please see full 38
Delivering for Long-term Growth
• Establishing commercial presence with AVMAPKI FAKZYNJA CO-PACK
– Meaningful quarterly growth driven by new patient starts, increased refills and
expanding physician adoption
– RAMP 301: Fully-enrolled Phase 3 confirmatory trial has the potential to expand U.S.
label and can be leveraged for EU/Japan approvals in recurrent LGSOC regardless
of KRAS mutation
• VS-7375 addresses significant opportunity in multiple KRAS G12D solid
tumors with a differentiated profile and compelling anti-tumor activity
– Broad clinical development program advancing VS-7375 across multiple tumor
types with multiple registration pathways
– Three registration-directed Phase 2 trials underway in pancreatic, lung and
colorectal cancers, with three Phase 3 trials planned to initiate in 1H 2027
• Cash runway into 2H of 2027* to see key data inflection points
– AVMAPKI FAKZYNJA CO-PACK franchise will be self-sustaining in 2H 2026, funding
both commercial operations and avutometinib plus defactinib clinical trials |
| 
| THANK YOU! |
| 
| Appendix |
| 
| 41
AVMAPKI FAKZYNJA CO-PACK: Category 2A Recommendation for KRAS-mutated Recurrent LGSOC
NCCN
Category 1
NCCN
Category 2a
NCCN
Category 2b
NCCN
Category 3
General %
Commercial
Payer Coverage
Examples of Clinical
Data in LGSOC and
Current NCCN
Guideline Category
No category 1
recommendation
Hormonal therapy
(e.g., Anastrozole, Letrozole)
& chemotherapy
• 6-13% ORR 4
• 17-30% discontinuation rate due
to AEs4
Trametinib
(2-4% US utilization rate1
)
• 26% ORR by INV assessment, no BICR5
• 36% discontinuation rate due to AEs5
Binimetinib
• Study stopped early due to futility
• 16% ORR by BICR
• 31% discontinuation rate due to
AEs
• Supported by MILO study3
Avutometinib + Defactinib
Combination Therapy
KRAS mt recurrent LGSOC
Please see the full Prescribing Information for more information
General source: NCCN; McGivney Global Advisory research and analysis; L.E.K. research and analysis. NCCN categories of preference: Preferred intervention, Other recommended intervention, Useful in certain circumstances.
High-level of evidence generally means large randomized controlled Phased 3 trials; Pie charts represent coverage by all major commercial players; 1. Data on File; 2. GOG 281 trial Gershenson et al., Lancet 2022; 3. MILO
Study Monk et al., J Clin Oncol 2020; 4. Supported by GOG 281 and MILO studies2,3 ; 5. Supported by GOG 2812
;AEs: adverse events; BICR: Blinded Independent Central Review |
| 
| 42
OB: Orange Book Listed Patent
AVMAPKI FAKZYNJA CO-PACK Patent Portfolio
OB: 7,897,792 Avutometinib COM (expiry 2/9/2027)
OB: 11,400,090 – Avutometinib mono dosing - (expiry 10/29/2038) PTE
OB: 7,928,109 Defactinib COM US (expiry 4/17/2028) PTA PTE (Aug, 2034)
OB: 8,247,411 Defactinib – genus (expiry 4/17/2028)
OB: 11,517,573 A+D combo dosing– (expiry 9/11/2040)
OB: 11,873,296 Avuto polymorph – (expiry 12/29/2042)
PTE (May 2039) |
| 
| 43
GFH375/VS-7375 Confers Single Agent Anti-Tumor Activity in Patients with
Previously Treated PDAC and NSCLC
PDAC
• 58.3% ORR (n=12) in 2L; 40.7% ORR (n=59) in all evaluable pts at 600 mg QD*
• mPFS and mOS has not been reached for 2L PDAC pts. Median follow up time
was 5.65 months.
C -PR confirmed
→ -On treatment
NSCLC
• 68.8% ORR (n=16) at 600 mg QD; 57.7% ORR (n=26) evaluable pts*
• Among the 5 pts with baseline brain metastases, 2 achieved PR
Ongoing Trials by GenFleet in China
• Initiated registrational Phase 3 trial in previously treated KRAS G12D-mutated PDAC at 600 mg QD versus investigator choice of chemotherapy
• Ongoing Phase 1b/2 trial of GFH375 in combination with cetuximab or
chemotherapy. The chemotherapy combination will be conducted 1L PDAC.
• Ongoing Phase 1/2 trial in G12D solid tumors
Manageable Safety Profile
• GFH375 presented a manageable safety profile at 600mg QD in
heavily previously treated KRAS G12D mutant NSCLC & PDAC patients
• NSCLC: 4.2% of patients discontinued treatment due to TRAEs.
Dose intensity = 90%.
• PDAC: 3% of patients discontinued treatment due to TRAEs.
Dose intensity = 93%
All patients
(N=26)
600mg QD
(N=16)
ORR [90% CI] 57.7% [39.8%, 74.2%] 68.8% [41.3%, 89.0%]
DCR [90% CI] 88.5% [72.8%, 96.8%] 93.8% [69.8%, 99.8%]
All PDAC
(N=59)
2L
(N=12)
3L+
(N=47)
ORR
[90%CI] 40.7% [29.87%, 52.22%] 58.3% [31.52%, 81.90%] 36.2% [24.52%, 49.18%]
DCR
[90%CI] 96.6% [89.71%, 99.39%] 100% [77.91, 100%] 95.7% [87.20%, 99.24]
GFH375 is being developed by GenFleet Therapeutics in China. Source of data: GenFleet presentation at WCLC, ESMO and company event in 2025. *Includes confirmed and
unconfirmed responses. QD: once daily; PR: partial response; TRAE: treatment-related adverse events; IL: first-line; 2L: second line; 3L: third-line; |
| 
| 44
Novel Combinations with VS-7375 Induce Dramatic and Sustained Tumor
Regressions in Preclinical Models
Combination of VS-7375 With Daraxonrasib Yields More
Sustained Tumor Regression Than Combination of
Zoldonrasib + Daraxonrasib
0 20 40 60 80
0
500
1000
1500
2000
2500
Days after first dose
Tumor volume
(mm3 ± SEM)
Vehicle VS-7375 50 mg/kg BID
Zoldonrasib 100 mg/kg QD
Daraxonrasib 25 mg/kg QD
VS-7375 + daraxonrasib Zoldonrasib + daraxonrasib
vehicle daraxonrasib
zoldonrasib
zoldonrasib + daraxonrasib VS-7375 VS-7375 + daraxonrasib
KP4 KRAS G12D PDAC model
Verastem data on file; Coma et al., AACR 2026; PRMT5: Protein arginine N-methyltransferase 5
QD: once daily; BID: twice per day
Combination of VS-7375 + PRMT5 Inhibitor Induces Dramatic
Sustained Tumor Regressions in KRAS G12D/MTAP-Del PDAC
Models
P A C X 0 2 0 P a n c r e a t i c C a n c e r M o d e l
All mice (8/8) showed complete responses with
VS-7375 + navlimetostat (PRMT5 inhibitor)
0 20 40 60 80
0
250
500
750
1000
1250
1500
Days after first dose
Tumor volume
(mm3 +/- SEM)
Vehicle VS-7375 50 mg/kg BID
Navlimetostat 100 mg/kg QD
VS-7375 + navlimetostat
vehicle navlimetostat
VS-7375 VS-7375 + navlimetostat
0 20 40 60 80
0
250
500
750
1000
1250
1500
Days after first dose
T
u
m
o
r
v
olu
m
e
(m
m
3
+/- S
E
M)
Vehicle
VS-7375 50 mg/kg BID
Navlimetostat 100 mg/kg QD
VS-7375 + navlimetostat
vehicle navlimetostat
VS-7375
VS-7375
+ navlimetostat |
| 
| 45
Confirmed Partial Response at Week 12 with VS-7375 900 mg QD
Monotherapy in 55 y/o Male with mPDAC
• Prior mFOLFIRINOX (SD for 4 mos)
and Gem/NabP (PD after 2 mos)
• Investigator-reported pain resolution
within 1 week of treatment
(completely off morphine from 100
mg/day)
• No treatment-related AE reported
• Normal CA-19-9 levels at baseline. No
CEA or CA125 measurements
Week 12:
cPR (-47%) (SLD: 80mm)
Response ongoing
Baseline Lesion:
Liver, surgical bed, peritoneum
(SLD: 151mm)
SLD: sum of longest diameter; cPR: confirmed Partial Response; SD: stable disease; mos: months; PD: progressive disease; CEA: carcinoembryonic antigen; CA125: cancer
antigen 125; mFOLFIRINOX: oxaliplatin, irinotecan, leucovorin, and fluorouracil (5-FU)
Source: TARGET-D 101 investigator |
| 
| 46
Complete Resolution of Target Lesion at Week 6 with VS-7375 900 mg QD
Monotherapy in 79 y/o Female with mPDAC
uPR: unconfirmed partial response; G: Grade.; Tx: therapy
Source: TARGET-D 101 investigator
• Prior Gem/NabP (6 mos of Tx) , NALIRI (11
mos of Tx), and FOLFOX (3 mos of Tx)
• Investigator-reported abdominal pain and
distention (caused by ascites) resolved in
2 weeks
• Selected AE: G2 diarrhea (resolved), G2
fatigue, G2 anorexia
• Highly elevated CA-19-9 levels at baseline
with >60% reduction in 3 weeks and
>99% reduction in 6-9 weeks
Week 6:
uPR (-100%) (SLD: 0mm)
Response ongoing
Baseline Lesion:
Mesentery, peritoneum
(SLD: 64mm)
0
4000
8000
12000
16000
20000
0 3 6 9
CA 19-9
(U/mL)
Time on Tx
(weeks)
>99% reduction of CA 19-9 |
| 
| 47 Source: VS-7375 TARGET-D 101 investigator; RT: radiotherapy; TEAE: treatment-emergent adverse event
Baseline Lesion: Pleura, lung,
mediastinum lymph node
(52mm)
7x5mm
9x7mm
Week 6:
cPR (-46%) (28mm)
Week 12:
cPR: (-70%) (16mm)
Deep and Rapid Response Achieved at Subtherapeutic Dose with VS-7375
400 mg QD + Anti-EGFR in 64 y/o Male with mPDAC
• Prior FOLFIRINOX + RT (PR) and
FOLFIRINOX (SD for 3 mos)
• Investigator-reported cough had
resolved within 1 week of treatment
• Selected TEAEs included upper GI
hemorrhage*, anemia*, gastritis*
and rash maculopapular**
0
200
400
600
800
1000
0 3 6 9 12
CA19-9
(U/mL)
Time on Tx
(weeks)
Significant drop in CA19-9
Levels by Week 3
*Unrelated to VS-7375 per investigator **Likely attributed to cetuximab, not VS-7375 |
| 
| 48
Marked Reduction of Disease Burden with VS-7375 600 mg QD + Cetuximab
in Heavily Pre-treated 42 y/o Male with mCRC
Source: TARGET-D 101 Investigator
Week 12:
SD: (-29.5%) (SLD: 260mm)
Baseline Lesion:
(SLD: 370mm)
0
500
1000
1500
2000
2500
3000
3500
4000
4500
0 3 6 9 12
CEA
(ng/mL)
Time on Tx
(weeks)
90% CEA reduction
from baseline
• 6 prior lines of therapy. Exhausted
standard-of-care therapies,
including TAS-102, and received 2
different and sequential
investigational agents through
clinical trials
• Investigator-reported abdominal
distention mostly resolved
• No significant AE except
cetuximab-induced acneiform rash |
| 
| 49
Week 12:
cPR (-49%) (SLD: 35mm)
Baseline Lesion:
(SLD 69mm)
Source: TARGET-D 101 investigator
Confirmed Partial Response at Week 6 with Deepening of Response Through
Week 12 at VS-7375 600 mg QD in 77 y/o Female with Advanced NSCLC
▪ Prior therapy of Pemetrexed-Carbo-Pembro (SD 4 mos)
▪ Patient had an unconfirmed partial
response at week 6 (-34%) that was
confirmed at week 12 (-49%)
▪ Investigator-reported shortness-of-breath
and tumor pain improved within 2 weeks
of dosing
▪ TEAE: diarrhea G3, dose reduced in cycle
3 to 400 mg for 4 weeks and re-escalated
to 600 mg after in cycle 4 |