Verastem Oncology Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Business Updates
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Insights
The recent announcement by Verastem Oncology regarding the initiation of the Phase 3 RAMP 301 trial for the avutometinib and defactinib combination addresses an urgent need in the treatment of recurrent low-grade serous ovarian cancer (LGSOC). LGSOC is a relatively rare and indolent subtype of ovarian cancer, often characterized by a poor response to conventional chemotherapy. The combination therapy, if successful, could offer a new line of treatment for patients who have limited options.
The Fast Track designation granted by the FDA for the avutometinib and sotorasib combination for KRAS G12C-mutant non-small cell lung cancer (NSCLC) is significant. This mutation is present in a subset of NSCLC patients and is associated with a poor prognosis. Fast Track status can expedite the review process, potentially bringing a new therapy to market more quickly.
It is important to note that the efficacy of these treatments must be demonstrated in larger, confirmatory trials and safety profiles must be well-characterized. The anticipation of multiple data readouts in 2024 will be critical in determining the potential impact of these therapies on patient care.
From a financial perspective, Verastem Oncology's progress in advancing its pipeline is a positive indicator for investors. The initiation of the Phase 3 trial and the planned New Drug Application (NDA) submission for the avutometinib and defactinib combination could lead to a significant milestone payment and future revenue streams upon approval and commercial launch. The market for ovarian cancer treatments is competitive, but the potential approval of a new therapy for LGSOC can capture a niche market segment.
The Fast Track designation for another of their drug combinations could potentially lead to quicker revenue generation, given the expedited review process. However, investors should be aware of the inherent risks in biotech investing, as clinical trial outcomes are uncertain and can significantly impact the company's valuation.
It is also worth monitoring the company's burn rate and cash reserves as they prepare for potential commercial launch in 2025. The cost of clinical trials and the ramp-up for commercialization can be substantial and the company may need additional capital infusions or partnerships to sustain operations.
Looking at the market dynamics, Verastem Oncology's strategic focus on RAS/MAPK driven cancers taps into a growing segment of the oncology market. The specificity of targeting genetic mutations like KRAS G12C and G12D positions the company in a favorable niche that is gaining attention for personalized medicine approaches. The collaboration with GenFleet Therapeutics on the oral KRAS G12D inhibitor GFH375 (VS-7375) indicates a proactive approach in pipeline expansion and diversification.
Given the competitive landscape, the company's success will depend not only on clinical outcomes but also on effective marketing and sales strategies post-approval. The patient and healthcare professional programs initiated by Verastem Oncology suggest an awareness of the importance of stakeholder engagement in the oncology field.
Furthermore, the anticipation of multiple data readouts in 2024 could serve as catalysts for the company's stock price, making it an interesting year for potential investors and current stakeholders. The company's ability to meet its clinical development timelines and manage regulatory interactions will be key factors in maintaining investor confidence.
Initiated confirmatory Phase 3 RAMP 301 trial evaluating avutometinib and defactinib combination in recurrent low-grade serous ovarian cancer; on track to submit rolling NDA for accelerated approval in H1 2024; preparations underway for potential commercial launch in 2025
Granted FDA Fast Track designation for avutometinib and sotorasib combination for the treatment of KRAS G12C-mutant non-small cell lung cancer
Announced oral KRAS G12D inhibitor GFH375 (VS-7375) as lead program in discovery and development collaboration with GenFleet Therapeutics
Multiple data readouts evaluating combination therapies in low-grade serous ovarian cancer, non-small cell lung cancer and pancreatic cancer anticipated in 2024
“In 2023, we made significant progress toward expanding the opportunities for avutometinib combination therapies across RAS/MAPK driven cancers and I am extremely proud of the team’s accomplishments,” said Dan Paterson, president and chief executive officer of Verastem Oncology. “2024 is expected to be a pivotal year with the planned NDA submission for our avutometinib and defactinib combination in recurrent LGSOC, and multiple clinical data readouts across our programs including initial data from the RAMP 205 trial in metastatic pancreatic cancer and data from the combination with G12C inhibitors in NSCLC. We look forward to continuing to deliver results across our programs.”
Fourth Quarter 2023 and Recent Highlights
Avutometinib and Defactinib Combination in Low-Grade Serous Ovarian Cancer (LGSOC)
- Initiated international confirmatory Phase 3 RAMP 301 trial evaluating the avutometinib and defactinib combination versus standard of care chemotherapy or hormonal therapy for the treatment of recurrent LGSOC in December 2023 to support potential full approval.
- Reported results of a planned subgroup analysis of Part A of the Phase 2 RAMP 201 trial evaluating avutometinib and defactinib combination in recurrent LGSOC, which demonstrated promising efficacy in patients regardless of number and class of prior therapies including after poor response to prior therapy at the Annual Global Meeting of the International Gynecologic Cancer Society meeting in November 2023.
- Launched patient and healthcare professional programs, including Let’s Talk About LGSOC, to support clinicians in the diagnosis and management of LGSOC and provide information and resources to patients.
-
Received Orphan Drug Designation from the
U.S. Food and Drug Administration (FDA) in March 2024 for avutometinib alone or in combination with defactinib for the treatment of all patients with recurrent LGSOC.
Verastem anticipates the following milestones for avutometinib and defactinib combination in LGSOC in 2024:
-
On track to submit a rolling New Drug Application (NDA) for Accelerated Approval for the avutometinib and defactinib combination in LGSOC in H1 2024. Preparations for a potential
U.S. commercial launch in 2025 are ongoing and plans to initiate discussions with European and Japanese regulatory authorities to address patient needs outside theU.S. continue to advance. - Plan to announce updated topline data from RAMP 201 trial in LGSOC in H1 2024.
Avutometinib in Combination with KRAS G12C Inhibitors in Non-Small Cell Lung Cancer (NSCLC)
- Received Fast Track designation from the FDA for avutometinib, in combination with Amgen’s G12C inhibitor, LUMAKRAS™ (sotorasib), for the treatment of patients with KRAS G12C-mutant metastatic NSCLC who have received at least one prior systemic therapy and have not been previously treated with a KRAS G12C inhibitor, in January 2024.
-
Presented initial results from Phase 1/2 RAMP 203 trial evaluating the efficacy and safety of avutometinib and sotorasib in patients with KRAS G12C-mutant NSCLC who have or have not been previously treated with a KRAS G12C inhibitor at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in October 2023. The confirmed objective rate of response (ORR) was
25% with responses observed in both KRAS G12C inhibitor resistant and naïve patients, and a recommended Phase 2 dose was selected. - Added defactinib to RAMP 203 trial of avutometinib with sotorasib based on stronger tumor regressions in KRAS G12C-mutant NSCLC preclinical models when FAKi is added along with G12Ci + avutometinib.
Verastem anticipates the following milestones for avutometinib in combination with KRAS G12C inhibitors in 2024:
- Data updates from patients with KRAS G12C-mutant NSCLC in the Phase 1/2 RAMP 203 trial evaluating avutometinib and sotorasib and the Phase 1/2 RAMP 204 trial evaluating avutometinib and adagrasib are planned for mid-2024.
Avutometinib and Defactinib Combination in Frontline Metastatic Pancreatic Cancer
- Plan to present initial safety and efficacy results from RAMP 205 trial of avutometinib and defactinib in combination with current standard of care gemcitabine and nab-paclitaxel in frontline metastatic pancreatic cancer in H1 2024.
GFH375 (VS-7375): Oral KRAS G12D (ON/OFF) Inhibitor
- Completed investigational new drug (IND)-enabling studies for oral KRAS G12D (ON/OFF) inhibitor GFH375 (VS-7375), the lead program in the collaboration with GenFleet Therapeutics (“GenFleet”).
-
GenFleet is expected to submit an IND application for GFH375 (VS-7375) in
China in H1 2024 with plans to begin a Phase 1 trial in H2 2024. Discovery/lead optimization continues for second and third programs.
Upcoming Presentations
Verastem previously announced the acceptance of multiple abstracts for presentation at upcoming medical conferences:
-
Multiple abstracts were selected for oral and poster presentations at the Society of Gynecologic Oncology (SGO) 2024 Annual Meeting on Women’s Cancer on March 16-18 in
San Diego . These presentations will include a late-breaking oral presentation on a planned subgroup analysis of Part A of the Phase 2 RAMP 201 trial of avutometinib and defactinib combination of heavily pretreated patients with LGSOC and a plenary oral presentation of preclinical efficacy data of avutometinib in combination with a FAK inhibitor in recurrent LGSOC as well as a trials-in-progress poster about the Phase 3 RAMP 301 trial. See press release here. -
Five preclinical data abstracts were accepted for poster presentation at the American Association for Cancer Research (AACR) Annual Meeting 2024 on April 5-10 in
San Diego . These presentations will highlight anti-tumor efficacy data of GFH375 (VS-7375), data on RAF/MEK clamp avutometinib and FAK inhibition in pancreatic ductal adenocarcinoma models supporting the ongoing RAMP 205 trial, and avutometinib and a FAK inhibitor combination in cutaneous melanoma models to overcome resistance to BRAF and MEK inhibitors, resistance to immunotherapy, and brain metastasis. See press release here.
Corporate Updates
- Strengthened the executive leadership team with appointments of Mike Crowther to Chief Commercial and Business Strategy Officer and the promotion of Dan Calkins to Chief Financial Officer in October 2023.
Fourth Quarter 2023 Financial Results
Verastem Oncology ended the fourth quarter of 2023 with cash, cash equivalents and investments of
Total operating expenses for the three months ended December 31, 2023 (the “2023 Quarter”) were
Research & development expenses for the 2023 Quarter were
Selling, general & administrative expenses for the 2023 Quarter were
Net loss for the 2023 Quarter was
For the 2023 Quarter, non-GAAP adjusted net loss was
Full-Year 2023 Financial Results
Total operating expenses for the year ended December 31, 2023 (the “2023 Period”) were
Research & development expenses for the 2023 Period were
Selling, general & administrative expenses for the 2023 Period were
Net loss for the 2023 Period was
For the 2023 Period, non-GAAP adjusted net loss was
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 the Avutometinib and Defactinib Combination
Avutometinib is a RAF/MEK clamp that induces inactive complexes of MEK with ARAF, BRAF and CRAF potentially creating a more complete and durable anti-tumor response through maximal RAS/MAPK pathway inhibition. In contrast to currently available MEK-only inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other MEK-only inhibitors. The
Verastem Oncology is currently conducting clinical trials with its RAF/MEK clamp avutometinib in RAS/MAPK driven tumors as part of its (Raf And Mek Program). RAMP 301 (NCT06072781) is a Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent LGSOC. RAMP 201 (NCT04625270) is a Phase 2 registration-directed trial of avutometinib in combination with defactinib in patients with recurrent LGSOC and enrollment has been completed in each of the dose optimization and expansion phases and the low-dose evaluation.
Verastem Oncology has established clinical collaborations with Amgen and Mirati to evaluate LUMAKRAS™ (sotorasib) in combination with avutometinib and defactinib and KRAZATI™ (adagrasib) in combination with avutometinib in KRAS G12C mutant NSCLC as part of the RAMP 203 (NCT05074810) and RAMP 204 (NCT05375994) trials, respectively. Supported by the “Therapeutic Accelerator Award” received from PanCAN, Verastem Oncology is conducting RAMP 205 (NCT05669482), a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer.
About GFH375 (VS-7375)
GFH375 (VS-7375) is a potential best-in-class, potent and selective oral KRAS G12D (ON/OFF) inhibitor, identified as the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. GenFleet plans to submit an IND in
About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a late-stage development biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with cancer. Our pipeline is focused on novel small molecule drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition and FAK inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn.
Forward-Looking Statements Notice
This press release includes forward-looking statements about Verastem Oncology’s strategy, future plans and prospects, including statements related to the expected outcome and benefits of the collaboration with GenFleet, the potential clinical value of various of its clinical trials, the timing of commencing and completing trials, including topline data reports, interactions with regulators, the potential for and timing of commercialization of product candidates and potential for additional development programs involving Verastem Oncology’s lead compound. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," “can,” “promising” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement.
Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the success in the development and potential commercialization of our product candidates, including avutometinib in combination with other compounds, including defactinib, LUMAKRAS™ and others; the uncertainties inherent in research and development, such as negative or unexpected results of clinical trials, the occurrence or timing of applications for our product candidates that may be filed with regulatory authorities in any jurisdictions; whether and when regulatory authorities in any jurisdictions may approve any such applications that may be filed for our product candidates, and, if approved, whether our product candidates will be commercially successful in such jurisdictions; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the scope, timing, and outcome of any legal proceedings; 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 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; that our product candidates will 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 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 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 will 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 will take longer or cost more than planned, including as a result of conducting additional studies; that we may not have sufficient cash to fund our contemplated operations; that we may not attract and retain high quality personnel; that we or Chugai Pharmaceutical Co., Ltd. will fail to fully perform under the avutometinib license agreement; that our target market for our product candidates might be smaller than we are presently estimating; that Secura Bio, Inc. will fail to fully perform under the asset purchase agreement with Secura Bio, Inc., including in relation to milestone payments; that we will not see a return on investment on the payments we have and may continue to make pursuant to the collaboration and option agreement with GenFleet or that GenFleet will fail to fully perform under the agreement; 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 will not pursue or submit regulatory filings for our product candidates; and that our product candidates will 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, 2023 as filed with the Securities and Exchange Commission (SEC) on March 14, 2024 and in any subsequent filings with the SEC. The forward-looking statements contained in this press release reflect Verastem Oncology’s views as of the date hereof, and the Company does not assume and specifically disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.
Verastem Oncology
|
|||||
|
December 31, 2023 |
|
December 31, 2022 |
||
|
|
|
|
|
|
Cash, cash equivalents, & investments |
$ |
137,129 |
|
$ |
87,894 |
Accounts receivable, net |
|
— |
|
|
31 |
Prepaid expenses and other current assets |
|
6,553 |
|
|
4,945 |
Property and equipment, net |
|
37 |
|
|
92 |
Right-of-use asset, net |
|
1,171 |
|
|
1,789 |
Restricted cash and other assets |
|
4,828 |
|
|
299 |
Total assets |
$ |
149,718 |
|
$ |
95,050 |
|
|
|
|
|
|
Current Liabilities |
$ |
26,380 |
|
$ |
21,663 |
Long term debt |
|
40,086 |
|
|
24,526 |
Lease liability, long-term |
|
530 |
|
|
1,470 |
Preferred stock tranche liability |
|
4,189 |
|
|
— |
Convertible preferred stock |
|
21,159 |
|
|
— |
Stockholders’ equity |
|
57,374 |
|
|
47,391 |
Total liabilities, convertible preferred stock and stockholders’ equity |
$ |
149,718 |
|
$ |
95,050 |
Verastem Oncology
|
||||||||||||
|
|
Three months ended December 31, |
|
Year ended December 31, |
||||||||
|
|
2023 |
|
2022 |
|
2023 |
|
2022 |
||||
Revenue: |
|
|
|
|
|
|
|
|
|
|
|
|
Sale of COPIKTRA license and related assets revenue |
|
$ |
— |
|
$ |
— |
|
$ |
— |
|
$ |
2,596 |
Total revenue |
|
|
— |
|
|
— |
|
|
— |
|
|
2,596 |
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
22,502 |
|
|
10,740 |
|
|
61,356 |
|
|
50,558 |
Selling, general and administrative |
|
|
8,637 |
|
|
6,106 |
|
|
30,728 |
|
|
24,975 |
Total operating expenses |
|
|
31,139 |
|
|
16,846 |
|
|
92,084 |
|
|
75,533 |
Loss from operations |
|
|
(31,139) |
|
|
(16,846) |
|
|
(92,084) |
|
|
(72,937) |
Other income (expense) |
|
|
(49) |
|
|
(7) |
|
|
(109) |
|
|
47 |
Interest income |
|
|
1,869 |
|
|
769 |
|
|
6,214 |
|
|
1,215 |
Interest expense |
|
|
(1,120) |
|
|
(724) |
|
|
(4,139) |
|
|
(2,137) |
Change in fair value of preferred stock tranche liability |
|
|
3,071 |
|
|
— |
|
|
2,751 |
|
|
— |
Net loss |
|
$ |
(27,368) |
|
$ |
(16,808) |
|
$ |
(87,367) |
|
$ |
(73,812) |
Net loss per share—basic and diluted |
|
$ |
(1.02) |
|
$ |
(0.99)(1) |
|
$ |
(3.96)(1) |
|
$ |
(4.57)(1) |
Weighted average common shares outstanding used in computing: |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share – basic and diluted |
|
|
26,808 |
|
|
17,042(1) |
|
|
22,054(1) |
|
|
16,138(1) |
(1) Amounts have been retroactively restated to reflect the 1-for-12 reverse stock split effected on May 31, 2023
Verastem Oncology
|
||||||||||||
|
|
Three months ended December 31, |
|
Year ended December 31, |
||||||||
|
|
2023 |
|
2022 |
|
2023 |
|
2022 |
||||
Net loss reconciliation |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss (GAAP basis) |
|
$ |
(27,368) |
|
$ |
(16,808) |
|
$ |
(87,367) |
|
$ |
(73,812) |
Adjust: |
|
|
|
|
|
|
|
|
|
|
|
|
Stock-based compensation expense |
|
|
1,598 |
|
|
1,287 |
|
|
5,860 |
|
|
6,047 |
Non-cash interest, net |
|
|
(837) |
|
|
(3) |
|
|
(1,132) |
|
|
228 |
Change in fair value of preferred stock tranche liability |
|
|
(3,071) |
|
|
— |
|
|
(2,751) |
|
|
— |
Severance and other |
|
|
113 |
|
|
109 |
|
|
199 |
|
|
109 |
Adjusted net loss (non-GAAP basis) |
|
$ |
(29,565) |
|
$ |
(15,415) |
|
$ |
(85,191) |
|
$ |
(67,428) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Reconciliation of net loss per share |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share – diluted (GAAP Basis) |
|
$ |
(1.02) |
|
$ |
(0.99)(1) |
|
$ |
(3.96)(1) |
|
$ |
(4.57)(1) |
Adjust per diluted share: |
|
|
|
|
|
|
|
|
|
|
|
|
Stock-based compensation expense |
|
|
0.06 |
|
|
0.09(1) |
|
|
0.26(1) |
|
|
0.38(1) |
Non-cash interest, net |
|
|
(0.03) |
|
|
—(1) |
|
|
(0.05)(1) |
|
|
0.01(1) |
Change in fair value of preferred stock tranche liability |
|
|
(0.11) |
|
|
— |
|
|
(0.12)(1) |
|
|
— |
Severance and other |
|
|
— |
|
|
— |
|
|
0.01(1) |
|
|
0.01(1) |
Adjusted net loss per share – diluted (non-GAAP basis) |
|
$ |
(1.10) |
|
$ |
(0.90)(1) |
|
$ |
(3.86)(1) |
|
$ |
(4.18)(1) |
Weighted average common shares outstanding used in computing net loss per share—diluted |
|
|
26,808 |
|
17,042(1) |
|
|
22,054(1) |
|
|
16,138(1) |
|
(1) Amounts have been retroactively restated to reflect the 1-for-12 reverse stock split effected on May 31, 2023
View source version on businesswire.com: https://www.businesswire.com/news/home/20240314845611/en/
For Investor and Media Inquiries:
Julissa Viana
Vice President, Corporate Communications and Investor Relations
investors@verastem.com or
media@verastem.com
Source: Verastem Oncology
FAQ
What Phase 3 trial did Verastem Oncology initiate for recurrent low-grade serous ovarian cancer?
What is the expected timeline for the submission of the rolling NDA for accelerated approval?
For which cancer type did Verastem Oncology receive FDA Fast Track designation?
What is the lead program in Verastem Oncology's collaboration with GenFleet Therapeutics?
What are the anticipated data readouts in 2024 for Verastem Oncology's combination therapies?
What were some of the key highlights for Verastem Oncology in Q4 2023?
Who is the president and CEO of Verastem Oncology?
What financial results were reported by Verastem Oncology for the three months and full year ended December 31, 2023?
