Verastem, Inc.
For the period ended Mar 31, 2026. Filed May 7, 2026.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2026 · Filed May 7, 2026
VSTM$8.41-10.3%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-09-30; filed 2025-11-04.
0001104659-25-106279
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-03-31; filed 2026-05-07.
0001104659-26-057114
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 88 previous paragraphs and 70 current paragraphs.
18 unchanged; 23 reworded; 26 added; 39 removed; 8 previous and 3 current paragraphs rearranged.
Tables are not compared: 11 previous and 19 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (26)
Current report
Our pipeline includes clinical-stage programs, preclinical research programs and externally partnered early-stage programs. Our focus is on novel small molecule drugs developed both as monotherapy and in combination, which inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including targeting RAS directly with KRAS G12D inhibition, targeting the pathway downstream with RAF/MEK inhibition, and targeting the parallel pathway that drives resistance with FAK inhibition. Our focus is to expeditiously develop and deliver transformative therapies that truly change outcomes for people living with RAS/MAPK pathway-driven cancers.
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COMMERCIAL PRODUCTS
Current report
AVMAPKI FAKZYNJA CO-PACK
Current report
The combination is being evaluated in an ongoing international Phase 3 trial, RAMP 301, in recurrent LGSOC with or without a KRAS mutation. The trial was fully enrolled, as of December 2025, and will serve as a confirmatory study for the initial indication and has the potential to expand the indication regardless of KRAS mutation status. The results will also be leveraged for potential geographic expansion.
Current report
CLINICAL PIPELINE
Current report
VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 has a differentiated profile compared to other RAS inhibitors. Based on preclinical data, VS-7375 offers dual, potent inhibition of both ON and OFF states of KRAS G12D. We believe this correlates with better in vivo efficacy and durability versus ON-only RAS inhibitors. VS-7375 has demonstrated a high affinity for KRAS G12D with long residence time (18-24 hours) in preclinical models. We believe this correlates with a more rapid and durable suppression of pERK signaling (which controls growth and cell survival) when compared to other ON-only KRAS G12D inhibitors in tumor cell lines. The selective inhibition of VS-7375 to KRAS G12D has shown, in preclinical models, to spare T cell proliferation to maintain a normal healthy immune response, versus a RAS-multi-inhibitor which impairs T cell proliferation at increasing concentrations of drug. The once daily oral dosing of VS-7375 achieves exposures corresponding to maximal tumor regressions across preclinical models for pancreatic, lung and colorectal cancers. Verastem announced in April 2025 that the U.S. Investigational New Drug (IND) application for (VS-7375-101) was cleared, and initiated a Phase 1/2 clinical trial in June 2025 in patients with advanced KRAS G12D mutant solid tumors, including pancreatic ductal adenocarcinoma (“PDAC”), non-small cell lung cancer (“NSCLC”) and colorectal cancer (“CRC”).
Current report
In the Phase 1/2 study the Company cleared multiple monotherapy dose levels with no dose-limiting toxicities (DLTs) and no major toxicities and continues to dose-escalate. The Company also cleared multiple dose levels in combination with cetuximab with no DLTs and higher doses are being evaluated. The Company continues to enroll patients across various cohorts in the Phase 1/2 study. Following recent feedback from the FDA, the Company is amending its Phase 1/2 trial protocol to separate out disease-specific Phase 2 registration-directed trials for KRAS G12D mutated 2L PDAC, 2L/3L NSCLC and 2L+ CRC.
Current report
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 our partner in China. We also reported that the FDA requested the Company develop separate Phase 2 trial protocols for their trials in PDAC, NSCLC and CRC, where we are seeking marketing authorization.
Current report
GENFLEET THERAPEUTICS (SHANGHAI), Inc
Current report
We shared multiple updates from GenFleet Therapeutics, our partner developing VS-7375, known as GFH375, in China.
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FINANCIAL OPERATIONS OVERVIEW
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In our Annual Report on Form 10-K for the year ended December 31, 2025, filed on March 4, 2026, we disclosed that our existing cash resources, including proceeds from exercise of warrants in January 2026, along with revenue we expected to generate from sales of AVMAPKI FAKZYNJA CO-PACK, was expected to be sufficient to fund our planned operations through twelve months from the date of issuance of those consolidated financial statements. As of March 4, 2026, our liquidity analysis excluded projected expenses for the Phase 2 studies entitled TARGET-D 201, TARGET-D 202, and TARGET-D 203, as we had not yet formally committed to the spend for those studies. Subsequent to the filing of our Annual Report on Form 10-K for the year ended December 31, 2025, we initiated TARGET-D-201, TARGET-D 202, and TARGET-D 203, resulting in a significant increase in projected research and development expenses. Consequently, the significant increase in costs we now expect to incur raises substantial doubt about our ability to continue as a going concern within the twelve months after the date of the issuance of these consolidated financial statements.
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“N/M” - Percentage change is not meaningful (N/M) where the prior period amount is zero or not comparable.”
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Product Revenue, Net.
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Costs of sales – product.
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Costs of sales – intangible amortization.
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Research and development expense.
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Selling, general and administrative expense* *
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Other expense
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Interest income
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Interest expense
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Interest expense for the 2026 Quarter was $0.4 million compared to $0.2 million for the 2025 Quarter. The $0.2 million increase from the 2025 Quarter was primarily driven by interest expense incurred as part of the vendor financing arrangement resulting in an increase of interest expense recorded in the 2026 Quarter.
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Operating activities
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Investing activities
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The cash used in investing activities for the 2026 Quarter relates to the purchase of investments of $16.0 million. There were no cash inflows or outflows related to investing activities during the 2025 Quarter.
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Financing activities
Removed paragraphs (39)
Previous report
Avutometinib and Defactinib
Previous report
On May 8, 2025, the FDA approved AVMAPKI FAKZYNJA CO-PACK for the treatment of adult patients with “KRAS mt” recurrent LGSOC who received prior systemic therapy. The KRAS mutant recurrent LGSOC indication has been granted approval by the FDA under the accelerated approval pathway based on the tumor response rate and duration of response. Continued approval for this indication is contingent upon verification and description of clinical benefit in a confirmatory trial.
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Avutometinib and Defactinib Combination in LGSOC
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In the fourth quarter of 2020, we commenced a registration-directed trial investigating avutometinib in combination with defactinib for the treatment of patients with recurrent LGSOC entitled RAMP 201 study. We use the term “RAMP” to refer to our RAF and MEK Program. The RAMP 201 study is an adaptive two-part multicenter, parallel cohort, randomized, open label trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent LGSOC. The primary analysis from both the FRAME trial and RAMP 201 trial has been published.
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In December 2023, we announced the initiation of a confirmatory Phase 3 trial to evaluate the combination of avutometinib and defactinib for the treatment of patients with recurrent LGSOC entitled RAMP 301. RAMP 301 is a randomized global confirmatory trial, which is evaluating the efficacy and safety of avutometinib and defactinib versus standard of care chemotherapy or hormonal therapy in patients with recurrent LGSOC with and without a KRAS mutation. RAMP 301 will serve as the confirmatory study required by the FDA for the combination of avutometinib and defactinib for the initial indication of recurrent KRAS mutant LGSOC to potentially receive full approval and has the potential to support an expanded indication regardless of KRAS mutation status. RAMP 301 is a global study with enrollment open in the United States, Australia, Canada, Europe, United Kingdom, New Zealand, Japan, and Korea.
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Enrollment of the planned 270 patients was completed a full quarter early in September of 2025. A pre-planned Interim Analysis by an Independent Data Monitoring Committee (“IDMC”) was completed, and the IDMC recommended a modest one-time increase in enrollment to the RAMP 301 trial of 29 patients across KRAS mutation status. We expect to complete enrollment of the IDMC recommended patients by the first quarter of 2026.
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On May 8, 2025, the FDA approved AVMAPKI FAKZYNJA CO-PACK in advance of its Prescription Drug User Fee Act or PDUFA action date. Following this approval, we submitted a letter to the National Comprehensive Cancer Network (“NCCN”) seeking inclusion of avutometinib plus defactinib combination therapy in its LGSOC treatment guidelines. NCCN included AVMAPKI FAKZYNJA CO-PACK in its LGSOC treatment guidelines consistent with the product’s approved indication. In July 2025, we submitted to NCCN the publications for RAMP 201 and FRAME in support of our request to NCCN that they consider the inclusion of AVMAPKI FAKZYNJA CO-PACK in its LGSOC treatment guidelines for the KRAS wild-type population evaluated in these trials. We were informed that our submission would be reviewed at the NCCN Committee for Ovarian Cancer annual meeting, which was held in October 2025. The results from the meeting have not yet been made public.
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In October 2024, the Japanese Gynecologic Oncology Group dosed the first patient in a Phase 2 trial called RAMP 201J, evaluating the safety and efficacy of avutometinib in combination with defactinib in recurrent LGSOC in Japan. Preliminary safety and efficacy results from the Phase 2 RAMP 201J trial in Japan was accepted as an E-Poster at the International Gynecologic Cancer Society 2025 Annual Meeting. In the published abstract, with a data extract date of April 11, 2025, no dose limiting toxicities were observed and avutometinib and defactinib drug exposure levels were comparable to those observed in the global RAMP 201 study. Additional data, including efficacy (response rates) and updated safety will be available on November 5, 2025, when the embargo lifts.
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Phase 1/2 Trial (known as RAMP 203 Study) - Avutometinib and Defactinib in Combination with Amgen’s LUMAKRAS* *(Sotorasib) in Patients with KRAS G12C NSCLC
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In September 2021, we entered into a clinical collaboration agreement with Amgen, Inc. (“Amgen”) to evaluate the combination of avutometinib with Amgen’s KRAS G12C inhibitor LUMAKRAS® (sotorasib) in a Phase 1/2 study entitled RAMP 203. The Phase 1/2 trial began evaluating the safety, tolerability and efficacy of avutometinib in combination with LUMAKRAS in patients with KRAS G12C non-small cell lung cancer (“NSCLC”) who have not been previously treated with a KRAS G12C inhibitor, as well as in patients who have progressed on a KRAS G12C inhibitor. The trial built upon initial preclinical data showing enhanced anti-tumor efficacy with the combination of LUMAKRAS (KRAS G12C inhibition) and avutometinib (RAF/MEK inhibition) relative to either agent alone. In January 2024, the FDA granted fast track designation for combination of avutometinib and LUMAKRAS 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. The RAMP 203 study has progressed to the recommended Phase 2 dose of 4 mg avutometinib in combination with 960 mg of LUMAKRAS for the doublet of avutometinib and LUMAKRAS. Enrollment of patients without prior G12C treatment and patients who have experienced disease progression on a KRAS G12C inhibitor in the doublet dose expansion phase has been completed. Patients in both doublet cohorts continue to be followed for safety and efficacy.
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Based on emerging data demonstrating improved tumor regressions in KRAS G12C-mutant NSCLC preclinical models when a FAK inhibitor is combined with a G12C inhibitor and avutometinib, defactinib was added to the RAMP 203 study in new triplet cohorts in 2024. In December 2024, we announced three patients whose cancer previously progressed on a G12C inhibitor have been treated with the triplet combination of sotorasib 960 mg administered daily (“QD”) on a continuous schedule and avutometinib 3.2 mg twice-weekly plus defactinib 200 mg twice-daily. Avutometinib and defactinib are administered on a three out of four weeks schedule. There were no dose limiting toxicities observed in the first triplet combination cohort. Planned dose level evaluation cohorts for the triplet combination completed enrollment in first quarter of 2025. We expect to present an interim update of safety and efficacy from both the doublet and triplet data in the fourth quarter of 2025.
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Phase 1/2 Trial (known as RAMP 205 Study) - Avutometinib and Defactinib in Combination with Gemcitabine/Nab-Paclitaxel in Patients with Metastatic Adenocarcinoma of the Pancreas
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In May 2022, we received the first “Therapeutic Accelerator Award” from Pancreatic Cancer Network (“PanCAN”) for up to $3.8 million. The grant is supporting a Phase 1b/2 clinical trial of avutometinib in combination with defactinib entitled RAMP 205. RAMP 205 is evaluating the safety, tolerability and efficacy of avutometinib and defactinib in combination with GEMZAR® (gemcitabine) and ABRAXANE® (Nab-paclitaxel) in patients with previously untreated metastatic adenocarcinoma of the pancreas. The RAMP 205 trial is evaluating whether combining avutometinib (to target mutant KRAS which is mutated in more than 90% of pancreatic adenocarcinomas) and defactinib (to reduce stromal density and adaptive resistance to avutometinib) to the standard GEMZAR/ABRAXANE regimen improves outcomes for patients with pancreatic adenocarcinoma. In August 2022, PanCAN agreed to provide us with an additional $0.5 million for the collection and translational analysis of patient samples. Combination dose evaluation is ongoing.
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A dose level “0” has been added to the RAMP 205 study protocol that evaluates 3.2 mg of avutometinib twice a week, 200 mg of defactinib twice a day for three weeks out of every four weeks with 800 mg/m2 of gemcitabine and 100 mg/m2 of Nab-paclitaxel on a schedule of day 1, day 8, and day 15. All dose levels have been expanded to 12 patients each, including six additional patients recently enrolled to dose level 1, where it was reported five out of six patients reported an objective response rate at the American Society of Clinical Oncology (“ASCO”) 2024 Annual Meeting. At dose level 1, of the six additional patients, five remained on therapy and continued to be monitored for response given the initial length of time to response. We completed enrollment in quarter one of 2025. In May 2025, we announced as of April 25, 2025, 60 patients (12 per cohort) had been treated in one of five dose regimens with the combination of avutometinib and defactinib with gemcitabine and Nab-paclitaxel in frontline metastatic pancreatic ductal adenocarcinoma (“PDAC”). In the dose level 1 cohort, 12 patients received 2.4 mg of avutometinib twice a week, 200 mg of defactinib twice a day for 3 weeks out of every 4 and 800 mg/m2 of gemcitabine and 125 mg/m2 of Nab-paclitaxel on a schedule of day 1, day 8 and day 15. In dose level 1, 83% (10/12) of patients achieved partial responses (8 confirmed and 2 unconfirmed who remained on treatment). Given these results, we have selected dose level 1 as the recommended phase 2 dose to expand enrollment from the initial 12 patients to a total of 29 patients.
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We have completed enrollment of all 29 patients at the recommended phase 2 dose as of the end of the third quarter of 2025. We expect to report an update on the safety and efficacy of the RAMP 205 expansion cohort in the first half of 2026.
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Furthermore, avutometinib and defactinib are currently being investigated in combination with immunotherapeutic and other agents through investigator sponsored trials for the treatment of various solid tumors, including, but not limited to, colorectal cancer (“CRC”), gynecological cancer with MAPK pathway alterations, breast cancer, thyroid cancer and melanoma.
Previous report
In August 2023, we entered into a collaboration and option agreement (the “GenFleet Agreement”) with GenFleet Therapeutics (Shanghai), Inc. (“GenFleet”)pursuant to which GenFleet granted us options to obtain exclusive development and commercialization rights worldwide outside of mainland China, Hong Kong, Macau, and Taiwan for up to three oncology programs targeting RAS pathway driven cancers (the “GenFleet Options”). Under the GenFleet Agreement, we can exercise our GenFleet Options on a program-by-program basis. The collaboration builds on the strengths of both companies in oncology small molecule drug development, enabling us to partner our clinical development and regulatory expertise with GenFleet’s accomplished discovery capabilities. This synergistic collaboration includes our experience and established network of collaborators, including scientific and clinical experts in RAS biology and RAS pathway-driven cancers and GenFleet’s accomplishments with its KRAS G12C inhibitor program. In December 2023, we announced the selection of an oral and selective KRAS G12D (ON/OFF) inhibitor entitled VS-7375 (known as GFH375 in China) with a potential best-in-class profile as the lead program from our collaboration with GenFleet. An investigational new drug (“IND”) application by GenFleet in China for VS-7375 was cleared in June 2024. In July 2024, GenFleet began dosing several patients in a Phase 1/2 trial in China that is evaluating VS-7375 in patients with KRAS G12D-mutated advanced solid tumors. The Phase 1/2 study is being conducted in
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approximately 20 hospitals in China. The Phase 1 study was used to determine the recommended Phase 2 dose, and the Phase 2 study will further evaluate the efficacy and safety of VS-7375 in patients with advanced solid tumors, such as PDAC, CRC, and NSCLC.
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On January 14, 2025, we announced the early exercise of the GenFleet Option with respect to VS-7375. We filed an IND application in the United States for VS-7375 during the first quarter of 2025. In April 2025 we announced the FDA clearance of the IND, and in June 2025 we announced we dosed our first patient with VS-7375 in our U.S. Phase 1/2a clinical trial evaluating VS-7375 in patients with advanced KRAS G12D solid tumors entitled VS-7375-101.
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At the ASCO 2025 Annual Meeting, GenFleet announced updated data from the dose escalation phase of the Phase 1/2 trial of GFH375 (known as VS-7375 in the U.S.). GenFleet reported that 62 patients were enrolled in the Phase 1 portion of the study in China, receiving oral doses ranging from 100 to 900 mg QD. In the study, 98% of patients had metastatic disease, and 75% had received ≥2 prior lines of therapy. As of the data cutoff of May 16, 2025, 23 efficacy-evaluable patients with PDAC and 12 efficacy-evaluable patients with NSCLC, who received daily dosages of 400 or 600 mg and had at least one post-treatment tumor assessment, achieved an objective response rate (“ORR”) of 52% and a disease control rate (“DCR”) of 100%, and an ORR of 42% and a DCR of 83%, respectively. As of the cutoff date of March 31, 2025, GenFleet reported that there were no dose-limiting toxicities observed across all dose levels (100-900 mg QD), and the treatment-related adverse events (“TRAEs”) were mostly Grade 1/2. The most common TRAEs occurring in at least 20% of patients were diarrhea, nausea, vomiting, and anemia. TRAEs ≥ Grade 3 consisted mainly of decreased neutrophil count (8%) and diarrhea (5%). No TRAE-related deaths were reported.
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On September 8, 2025 updated data from GenFleet’s Phase 1/2 study of GFH375 in China was featured in a mini oral presentation at the IASLC 2025 World Conference on Lung Cancer (WCLC). At the recommended phase 2 dose of 600 mg QD, the ORR was 68.8%, both confirmed and unconfirmed, and the DCR was 93.8%. Additionally, among the 26 evaluable patients with NSCLC treated across all dose levels, the ORR was 57.7%, both confirmed and unconfirmed, and the DCR was 88.5%.
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On October 19, 2025 at the European Society for Medical Oncology (“ESMO”) congress, GenFleet announced updated efficacy data from the dose expansion phase of the Phase 1/2 trial of GFH375 (known as VS-7375 in the U.S.) As of the data cutoff on September 27, 2025 GFH375 resulted in a 41% ORR and a disease control rate (“DRR”) of 96.7% at the 600 mg QD for patients with heavily pre-treated pancreatic ductal adenocarcinoma of which, the majority of patients 91.5% experienced a reduction in target lesions. 68% of the pretreated patients had received 2 or more lines of anticancer therapies with 92.4% of patients receiving gemcitabine-based regimens and more than 50% receiving fluorouracil or irinotecan-containing regimens. At month four, the overall survival (“OS”) rate was 92.2%, however, median OS has not been reached as of the announcement. The median progression-free survival (“PFS”) was 5.52 months with a median follow-up time of 5.65 months and a 4-month PFS rate of 78.2%. At evaluation, 31 (47%) of patients were still on treatment with the longest duration of treatment eclipsing one year (367 days). The most recent safety data reported was as of the data cutoff on August 27, 2025 where Grade 3 TRAEs were reported in 20 patients (30.3%) and a Grade 4 TRAE (neutropenia) occurred in one patient (1.5%). Of the 66 patients in the safety population, four patients (6.1%) had a dose reduction and two patients (3%) discontinued due to TRAEs. No TRAE-related deaths were reported. The mean relative dose intensity was 93%.
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On October 27, 2025, GenFleet shared additional analyses from previously presented data at recent medical congresses evaluating GFH375 in both advanced KRAS G12D mutant PDAC and NSCLC. In a subgroup analysis, 12 patients with second line PDAC at 600 mg QD achieved an ORR of 58.3% and a DCR of 100%. In the third line or later setting, 47 PDAC patients receiving 600 mg QD achieved an ORR of 36.2% and a DCR of 95.7%. In the second line subgroup, the median progression-free survival (“mPFS”) and median overall survival (“mOS”) have not been reached. An additional analysis of gastrointestinal disorders, hematological toxicities, and liver enzyme abnormalities in 2L+ patients with PDAC (n=66) at 600 mg QD showed no adverse events Grade ≥3 occurred at rates above 8.0%. In an analysis of pre-treated patients with NSCLC at 600 mg QD, the four-month PFS rate was >75% and the mPFS has not been reached. The median follow-up time was 4.2 months. GenFleet announced that the first patient has been dosed in a Phase 1b/2 study of GFH375 combined with cetuximab or chemotherapy for advanced solid tumors on October 22, 2025.
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On October 23, 2025, we announced a preliminary update on our U.S. Phase 1/2a monotherapy dose escalation trial of VS-7375 in patients with previously treated advanced KRAS G12D mutant solid tumors. In the study, VS-7375 cleared both the 400 mg QD and the 600 mg QD monotherapy doses with no dose-limiting toxicities (DLTs) observed. At the two dose levels evaluated in the U.S. cohort, no nausea, vomiting, or diarrhea greater than Grade 1 were reported. The study’s dose escalation continues with evaluation of the monotherapy 900 mg QD dose level. Of the five efficacy evaluable patients in the VS-7375-101 study, with at least one scan, four out of five patients have had a tumor reduction and are still on treatment. The remaining patients receiving either the 400 mg QD or 600 mg QD doses have not yet reached their first response assessment. In addition, no new safety signals have been observed relative to earlier data presentations in both PDAC and NSCLC by our partner, GenFleet, in its ongoing Phase 1/2 clinical study in China evaluating VS-7375 (known as GFH375).
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We plan to report an interim safety and efficacy update on the Phase 1/2a trial of VS-7375 in the first half of 2026. We expect to select the recommended phase 2 dose (“RP2D”) and plan to initiate the monotherapy expansion cohorts in advanced PDAC, NSCLC and other solid tumors in the first half of 2026. We announced that we have initiated patient enrollment for the first dose escalation combination cohort evaluating VS-7375 with cetuximab in patients with advanced solid tumors, including colorectal cancer. Additionally, we expect to initiate the dose escalation cohorts in combination with chemotherapy for PDAC, and chemotherapy plus anti-PD-1 for NSCLC in the fourth quarter 2025. We expect to select the recommended phase 2 dose (“RP2D”) and plan to initiate the combination expansion cohorts in CRC, PDAC, and NSCLC in the first half of 2026. We also plan to engage with the FDA in the first half of 2026 to discuss our development path forward, including potential registration-directed clinical trials in PDAC and NSCLC.
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*Selling, general and administrative expense.Selling, general and administrative expense for the 2025 Quarter was $21.0 million compared to $12.3 million for the 2024 Quarter. The increase of $8.7 million from the 2024 Quarter to the 2025 Quarter primarily resulted from the increase of $8.2 million of [consulting and professional fees] primarily related to the launch of AVMAPKI FAKZYNJA CO-PACK for the treatment of KRAS mutant recurrent LGSOC, an increase of $2.3 million in personnel costs including non-cash based stock compensation, an increase of $1.2 million in commercial operations expenses, partially offset by a decrease of $3.0 million in July 2024 Offering financing fees in the 2024 Quarter. *
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*Change in fair value of warrant liability.*The change in fair value of the warrant liability was $55.9 million expense for the 2025 Quarter compared to $13.5 million income for the 2024 Quarter. The $55.9 million expense for the 2025 Quarter was comprised of the mark-to-market adjustment for the liability classified Warrants issued as part of the July 2024 Offering. The increase was driven by an increase in fair value per Warrant from June 30, 2025 to the exercise date for the 5,166,666 Warrants exercised during the 2025 Quarter and an increase in fair value per Warrant for the
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8,962,503 Warrants that remain outstanding at September 30, 2025, due to an increase in our stock price resulting in the $55.9 million expense during the 2025 Quarter. The $13.5 million income for the 2024 Quarter was comprised of the mark-to-market adjustment for the warrants issued as part of the July 2024 Offering which decreased primarily due to the decrease in our stock price from July 23, 2024 to end of 2024 Quarter.
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Comparison of the nine months ended September 30, 2025 and 2024
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*Sale of COPIKTRA license and related assets revenue.*Sale of COPIKTRA license and related assets revenue for the 2025 Period was $0.0 million compared to $10.0 million for the 2024 Period. Sale of COPIKTRA license and related assets revenue for the 2024 Period was comprised of one sales milestone payment of $10.0 million due to Secura achieving cumulative worldwide net sales of COPIKTRA exceeding $100.0 million during the 2024 Quarter. We received the $10.0 million milestone payment in July 2024.
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Research and development expense.* *Research and development expense for the Period was $82.9 million compared to $60.5 million for the 2024 Period. The $22.4 million increase in R&D expense from the 2024 period to the 2025 period was primarily driven by a $7.4 million increase in CRO costs, a $5.1 million increase in drug substance and drug product manufacturing costs, a $4.2 million increase in investigator fees, a $3.6 million increase in personnel costs, including non-cash stock-based compensation, a$6.0 million GenFleet Option payment related to VS-7375 in the 2025 Period, a $0.8 million increase in clinical supply costs, and a $0.7 million increase in pre-clinical expenses. These increases were partially offset by a $3.0 million milestone payment made to GenFleet in the 2024 Period, and a $2.4 million decrease in consulting expenses.
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The $9.8 million increase in avutometinib+ defactinib – LGSOC is primarily driven by an increase in RAMP 301 trial costs as the trial continues to advance and enroll more patients. The $1.9 million decrease in avutometinib+ defactinib – NSCLC is primarily driven by a decrease in RAMP 202 trial costs. The $1.6 million increase in avutometinib + defactinib – pancreatic cancer is primarily driven by fully utilizing the PanCAN Grant in the second quarter of 2024 and therefore there was no offsetting reduction in expense in the 2025 Period. The $4.4 million decrease in avutometinib and defactinib manufacturing and non-clinical trial specific expenses was driven by decreased drug metabolism and pharmacokinetics testing costs, decreased consulting costs, and decreased drug substance manufacturing costs. The $12.1 million increase in GenFleet/ VS-7375 related expenses is driven primarily by the $6.0 million GenFleet Option payment with respect to VS-7375 the 2025 Period, drug substance and product manufacturing costs and
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CRO costs for the VS-7375-101 trial, partially offset by a $3.0 million development milestone payment made during the 2024 Period pursuant to the GenFleet Agreement.
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*Interest income.*Interest income for the 2025 Period was $3.0 million compared to $3.2 million for the 2024 Period. The decrease of $0.2 million from the 2024 Period to the 2025 Period in interest income was primarily due to the decrease in investment balances on short term investments and cash equivalents and a decrease in interest rates on securities during each respective period.
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*Interest expense.*Interest expense for the 2025 Period was $0.7 million compared to $3.4 million for the 2024 Period. The decrease of $2.7 million from the 2024 Periodwas primarily driven by termination of the Loan Agreement with Oxford on January 13, 2025 resulting in a reduction of interest expense recorded in the 2025 Period. The Company has elected to record interest expense associated with the Notes within change in fair value of Notes on the condensed statements of operations and comprehensive loss.
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*Loss on debt extinguishment.*The loss on debt extinguishment for the 2025 Period of $1.8 million represents the loss recognized on early extinguishment of our Loan Agreement. On January 13, 2025, we repaid in full all principal, accrued and unpaid interest, fees, and expenses under the Loan Agreement in an aggregate amount of $42.7 million (the Payoff Amount). The Payoff Amount, excluding accrued interest, exceeded the carrying amount of the Term Loans on January 13, 2025 by $1.8 million which was recorded as a loss on debt extinguishment. There was no loss on debt extinguishment in the 2024 Period.
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*Change in fair value of preferred stock tranche liability.*The change in fair value of the preferred stock tranche liability was $4.2 million income for the 2024 Period. The change in fair value of preferred stock tranche liability was comprised of the mark-to-market adjustment related to the second tranche right issued as part of the Securities Purchase Agreement. The preferred stock tranche liability expired in July 2024 and therefore was not outstanding during the 2025 Period. The fair value of the preferred stock tranche liability decreased from $4.2 million at the beginning of the 2024 Period to $0.0 million at the end of the 2024 Period resulting in $4.2 million income in the 2024 Period.
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RAMP 205 study. Cash used in operating activities was $107.6 million and $79.7 million for the 2025 Period and the 2024 Period, respectively.
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*Investing activities.*The cash used in investing activities for the 2025 Period relates to the purchase of intangible assets of $8.3 million. The cash provided by investing activities for the 2024 Period relates to maturities of investments of $60.0 million, partially offset by a purchase of property and equipment of less than $0.1 million.
Reworded paragraphs (23)
Previous wording
We are a biopharmaceutical company committed to the development and commercialization of new medicines to improve the lives of patients diagnosed with RAS MAPK pathway-driven cancers. We market AVMAPKI™ FAKZYNJA™ CO-PACK (avutometinib capsules; defactinib tablets) in the United States. 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, FAK inhibition and KRAS G12D inhibition.
Current wording
We are a biopharmaceutical company committed to developing and commercializing new medicines to improve the lives of patients diagnosed with challenging RAS/MAPK pathway-driven cancers. We market AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) in the United States (“U.S.”), the first treatment specifically FDA-approved for adults with KRAS-mutated recurrent LGSOC who have received prior systemic therapy.
Previous wording
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/ extracellular-signal-regulated-kinase (“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 is designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors. Avutometinib, alone or in combination with defactinib, has received orphan drug designation for the treatment of all patients with LGSOC in the United States. Defactinib has received orphan drug designation in ovarian cancer in the United States, the European Union, and Australia. In addition, the FDA granted orphan drug designation to avutometinib, in combination with defactinib, for the treatment of pancreatic cancer.
Current wording
AVMAPKI FAKZYNJA CO-PACK is the first treatment specifically approved by the FDA for adults with KRAS-mutated recurrent LGSOC who have received prior systemic 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.
Previous wording
As of September 30, 2025, we had an accumulated deficit of $1,132.1 million. Our net loss was $98.5 million, $176.6 million, $24.0 million and $66.1 million for the three and nine months ended September 30, 2025 and 2024, respectively. As of September 30, 2025, we had cash, cash equivalents, and investments of $137.7 million. In accordance with applicable accounting standards, we evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about our ability to continue as a going concern within 12 months after the date of the issuance of the consolidated financial statements. We anticipate operating losses may continue for the foreseeable future and we continue to incur operating costs to execute our strategic plan, including costs related to research and development of our product candidates and commercial activities. As a result of the assessment in accordance with the applicable accounting standards, these conditions raise substantial doubt about our ability to continue as a going concern for 12 months after the date the condensed consolidated financial statements are issued.
Current wording
As of March 31, 2026, we had an accumulated deficit of $1,201.6 million. Our net loss was $36.6 million and $52.1 million for the three months ended March 31, 2026 and 2025, respectively. As of March 31, 2026, we had cash, cash equivalents, and investments of $181.7 million. In accordance with applicable accounting standards, we are required to evaluate whether there are conditions and events, considered in the aggregate, that raise substantial doubt about our ability to continue as a going concern within twelve months after the date of the issuance of these condensed consolidated financial statements.
Previous wording
We expect to finance our operations with our existing cash, cash equivalents and investments, through potential future milestones and royalties received pursuant to the Secura APA, through the Note Purchase Agreement, through future product revenues or through other strategic financing opportunities that could include, but are not limited to collaboration agreements, offerings of our equity, or the incurrence of debt. However, given the risks associated with these potential strategic or financing opportunities, they are not deemed probable for purposes of the going concern assessment. If we fail to obtain additional capital or generate sufficient revenue from our commercialization activities in
Current wording
We expect to finance our operations with our existing cash, cash equivalents and investments, through potential future milestones and royalties received pursuant to the Secura APA, through the Note Purchase Agreement, through future product revenues or through other strategic financing opportunities that could include, but are not limited to collaboration agreements, offerings of our equity, or the incurrence of debt. However, given the risks associated with these potential strategic or financing opportunities, they are not deemed probable for purposes of the going concern assessment. If we fail to obtain additional capital or generate sufficient revenue from our commercialization activities in the future, we may be unable to complete our planned preclinical studies and clinical trials and obtain approval of certain investigational product candidates from the FDA or foreign regulatory authorities. Therefore, there is substantial doubt about our ability to continue as a going concern.
Previous wording
Our discussion and analysis of our financial condition and results of operations is based upon our condensed consolidated financial statements prepared in accordance with U.S. generally accepted accounting principles. The preparation of these condensed consolidated financial statements requires us to make estimates and judgments that affect the reported amounts of certain assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements, and the amounts of revenues and expenses during the reported periods.
Current wording
preparation of these condensed consolidated financial statements requires us to make estimates and judgments that affect the reported amounts of certain assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements, and the amounts of revenues and expenses during the reported periods.
Previous wording
The critical accounting policies we identified in our most recent Annual Report on Form 10-K for the fiscal year ended December 31, 2024, related to revenue recognition, collaborative agreements, accrued and prepaid research and development expenses, and stock-based compensation. During the nine months ended September 30, 2025, there were no material changes to our critical accounting policies.
Current wording
The critical accounting policies we identified in our most recent Annual Report on Form 10-K for the fiscal year ended December 31, 2025, related to revenue recognition, accrued and prepaid research and development expenses, stock-based compensation and fair value of notes.
Previous wording
*Product Revenue, Net.*We began commercial sales of AVMAPKI FAKZYNJA CO-PACK within the United States in May 2025, following receipt of FDA marketing approval on May 8, 2025. For the three months ended September 30, 2025 (the “2025 Quarter”) we recorded approximately $11.2 million of net product revenue. We had no product revenue during the three months ended September 30, 2024 (the “2024 Quarter”).
Current wording
We began commercial sales of AVMAPKI FAKZYNJA CO-PACK within the United States in May 2025, following receipt of FDA marketing approval on May 8, 2025. For the three months ended March 31, 2026 (the “2026 Quarter”) we recorded approximately $18.7 million of net product revenue. We did not recognize any product revenue during the three months ended March 31, 2025 (the “2025 Quarter”).
Previous wording
*Costs of sales – product.*Costs of sales – product for the 2025 Quarter of approximately $1.7 million consisted of costs associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK, royalties owed to Chugai, Pfizer, and the Institute for Cancer Research (“ICR”) on such sales, and certain period costs. The Company began capitalizing
Current wording
Costs of sales – product for the 2026 Quarter of approximately $2.8 million consisted of costs associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK, royalties owed on such sales, and certain period costs including inventory write downs. The Company began capitalizing inventory upon receiving FDA approval for AVMAPKI FAKZYNJA CO-PACK on May 8, 2025. Prior to the FDA approval of AVMAPKI FAKZYNJA CO-PACK, expenses associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK were recorded as research and development
Previous wording
inventory upon receiving FDA approval for AVMAPKI FAKZYNJA CO-PACK on May 8, 2025. Prior to the FDA approval of AVMAPKI FAKZYNJA CO-PACK, expenses associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK were recorded as research and development expense. Certain of the costs of AVMAPKI FAKZYNJA CO-PACK units recognized as revenue during the 2025 Quarter, or approximately $0.1 million, were expensed prior to obtaining regulatory approval, therefore, are not included in cost of sales- product during this period. We expect cost of sales - product to increase in relation to product revenues as we deplete these inventories. We had no cost of sales – product during the 2024 Quarter.
Current wording
expense. Certain costs of AVMAPKI FAKZYNJA CO-PACK units recognized as revenue during the 2026 Quarter, or approximately $0.1 million, were expensed prior to obtaining regulatory approval, therefore, are not included in cost of sales*–* product during this period. We expect cost of sales - product to increase in relation to product revenues as we deplete these inventories. We did not recognize any cost of sales – product during the 2025 Quarter.
Previous wording
*Costs of sales –intangible amortization.*Cost of sales – intangible amortization for the 2025 Quarter of approximately $0.3 million was related to finite-lived intangible assets related to AVMAPKI FAKZYNJA CO-PACK which we recognized and began amortizing during the second quarter of 2025. There was no cost of sales – intangible amortization the 2024 Quarter.
Current wording
Cost of sales – intangible amortization for the 2026 Quarter of approximately $0.3 million was related to finite-lived intangible assets related to AVMAPKI FAKZYNJA CO-PACK which we recognized and began amortizing during the second quarter of 2025. We did not recognize any cost of sales – intangible amortization for the 2025 Quarter.
Previous wording
*Research and development expense.*Research and development expense for the 2025 Quarter was $29.0 million, compared to $24.8 million for the 2024 Quarter. The $4.2 million increase was primarily driven by a $3.0 million increase in drug substance/product manufacturing costs, a $2.6 million increase in contract research organization (“CRO”) costs, and a $1.6 million increase in investigator fees. These increases were partially offset by a $3.0 million milestone payment made during the 2024 Quarter pursuant to the GenFleet Agreement.
Current wording
Research and development expense for the 2026 Quarter was $38.2 million, compared to $29.2 million for the 2025 Quarter. The $9.1 million increase was primarily driven by a $4.7 million increase in investigator fees, a $3.7 million increase in drug substance and drug product manufacturing costs, a $3.1 million increase in clinical supply costs, a $2.7 million increase in contract research organization (“CRO”) costs, and a $1.9 million increase in personnel costs, including stock-based compensation. These increases were partially offset by a $6.0 million decrease in license fees, reflecting an Option exercise fee payment made during the 2025 Quarter pursuant to the GenFleet Agreement, and a $1.0 million decrease in consulting expenses.
Previous wording
The $1.6 million increase in avutometinib+ defactinib – LGSOC was primarily driven by an increase in RAMP 301 trial costs as the trial continues to advance and enroll more patients. The $1.7 million decrease in avutometinib and defactinib manufacturing and non-clinical trial specific expenses was primarily driven by decreased drug substance costs and decreased consulting costs. The $2.3 million increase in GenFleet and VS-7375 related expenses was primarily driven by increased drug substance costs and CRO costs for the 7375-101 study, partially offset by a $3.0 million development milestone payment made during the 2024 Quarter pursuant to the GenFleet Agreement.
Current wording
The $1.0 million increase in avutometinib+ defactinib – LGSOC was primarily driven by an increase in RAMP 301 trial costs as the study continues to advance and enroll more patients. The $0.9 million decrease in avutometinib and defactinib manufacturing and non-clinical trial specific expenses was primarily driven by decreases in drug substance costs, drug product costs and consulting costs. The $7.2 million increase in GenFleet and VS-7375 related expenses was primarily driven by increased drug substance, drug product, CRO and trial specific investigator costs for the 7375-101 study, partially offset by the $6.0 million Option exercise fee incurred to license VS-7375 in the 2025 Quarter pursuant to the GenFleet Agreement. The $1.7 million increase in personnel costs is driven by increased headcount.
Previous wording
*Selling, general and administrative expense.Selling, general and administrative expense for the 2025 Period was $56.7 million compared to $32.8 million for the 2024 Period. The increase of $23.9 million from the 2024 Period to the 2025 Period was primarily driven by a $19.9 million increase in consulting and professional fees related to the launch of AVMAPKI FAKZYNJA CO-PACK for the treatment of KRAS mutant recurrent LGSOC, and a $5.9 million increase in personnel costs including non-cash stock-based compensation. These increases were partially offset by a $1.4 million decrease in commercial operations expenses and a $0.5 million decrease in in travel, conference, and other expenses. *
Current wording
Selling, general and administrative expense for the 2026 Quarter was $22.3 million compared to $15.0 million for the 2025 Quarter. The $7.3 million increase was primarily driven by a $4.7 million increase in personnel costs, including non cash based stock-based compensation expense, a $2.2 million increase in commercial operations expenses, a $0.9 million increase in travel and conferences and other general and administrative expenses, and a $0.3 million increase in consulting and professional fees. These increases were partially offset by a $0.8 million decrease in financing fees related to the Note Purchase Agreement incurred in the 2025 Quarter.
Previous wording
Other expense. Other expense for the 2025 Quarter was less than $0.1 million compared to $0.1 million for the 2024 Quarter. Other expense for the 2025 Quarter and 2024 Quarter was comprised of transaction losses due to changes in foreign currency exchange rates.
Current wording
Other expense for the 2026 Quarter was less than $0.1 million compared to $0.1 million for the 2025 Quarter. Other expense for the 2026 Quarter and 2025 Quarter was comprised of transaction losses due to changes in foreign currency exchange rates.
Previous wording
Interest income. Interest income for the 2025 Quarter was $1.2 million, compared to $0.8 million for the 2024 Quarter. The $0.4 million increase was primarily driven by higher balances of cash equivalents during the 2025 Quarter compared to the 2024 Quarter.
Current wording
Interest income for the 2026 Quarter was $1.3 million, compared to $1.0 million for the 2025 Quarter. The $0.3 million increase was primarily driven by higher balances of cash equivalents during the 2026 Quarter compared to the 2025 Quarter.
Previous wording
*Interest expense.*Interest expense for the 2025 Quarter was $0.3 million compared to $1.1 million for the 2024 Quarter. The $0.8 million decrease from the 2024 Quarter was primarily driven by termination of the Loan Agreement with Oxford on January 13, 2025 resulting in a reduction of interest expense recorded in the 2025 Quarter. The Company has elected to record interest expense associated with the Notes within change in fair value of Notes on the condensed statements of operations and comprehensive loss.
Current wording
Change in fair value of warrant liability
Previous wording
*Change in fair value of warrant liability.*The change in fair value of the warrant liability was $38.0 million expense for the 2025 Period compared to $13.5 million income for the 2024 Period. The $38.0 million expense for the 2025 period was comprised of the mark-to-market adjustment for the liability classified warrants issued as part of the July 2024 Offering. The increase was driven by an increase in fair value per Warrant from December 31, 2025 to the exercise date for the 9,120,831 Warrants exercised during the 2025 Period and an increase in fair value per Warrant for the 8,962,503 Warrants that remain outstanding at September 30, 2025, due to an increase in our stock price resulting in the $38.0 million expense during the 2025 Period. The $13.5 million income for the 2024 Period was comprised of the mark-to-market adjustment for the warrants issued as part of the July 2024 Offering which decreased primarily due to the decrease in our stock price from July 23, 2024 to end of the 2024 Period.
Current wording
The change in fair value of the warrant liability was $9.3 million income for the 2026 Quarter compared to $2.4 million expense for the 2025 Quarter. The $9.3 million income for the 2026 Quarter and $2.4 million expense for the 2025 Quarter was comprised of the mark-to-market adjustment for the liability classified Warrants issued as part of the July 2024 Offering. The liability classified warrants decreased in value from December 31, 2025 to when 8,391,666 Warrants were exercised in January 2025 primarily driven by a reduction our stock price and the expiration of 37,500 Warrants, resulting in $9.3 million income during the 2026 Quarter. The liability classified warrants increased in value from December 31, 2024 to when 1,166,666 Warrants were exercised in March 2025 and March 31, 2025 primarily driven by an increase in our stock price, resulting in a $2.4 million expense during the 2025 Quarter.
Previous wording
*Change in fair value of Notes.*We elected the fair value option for the Notes and therefore the changes in fair value, including interest, other than changes that are directly attributable to instrument specific credit risk are recorded as change in fair of Notes in the condensed statements of operations and comprehensive loss. The change in fair value for the 2025 Quarter of $2.7 million was primarily driven by interest on the Notes and a reduction in the risk-free rate during the 2025 Quarter. There were no Notes outstanding in the 2024 Quarter.
Current wording
The change in fair value of Notes was $1.9 million for the 2026 Quarter compared to $4.4 million during the 2025 Quarter. We elected the fair value option for the Notes and therefore changes in fair value, including interest, other than changes that are directly attributable to instrument specific credit risk are recorded as change in fair value of Notes in the condensed statements of operations and comprehensive loss. The decrease in change in fair value in the 2026 Quarter is a result of the 2025 Quarter change in fair value being higher due to the revaluation of the note from its notional value to its fair value following issuance of the Notes in January 2025.
Previous wording
We have financed our operations to date primarily through public and private offerings of our common stock, pre-funded warrants, and warrants, offerings of convertible notes, sales of common stock under our at-the-market equity offering program, our loan and security agreement executed with Hercules in March 2017, as amended, the Loan Agreement with Oxford, our Note Purchase Agreement, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC, and Yakult, the upfront payment and milestone payments received under the Secura APA, and sales of Series B Convertible Preferred Stock. Additionally, we have financed a portion of our operations through product revenue, including from AVMAPKI FAKZYNJA CO-PACK, beginning with our U.S. commercial launch in May 2025 and from COPIKTRA, from its U.S. commercial launch in September 2018 through our sale of the COPIKTRA license in September 2020. We expect to finance a portion of our business through future potential milestones and royalties received pursuant to the Secura APA.
Current wording
We have financed our operations to date primarily through public and private offerings of our common stock, pre-funded warrants, and warrants, offerings of convertible notes, sales of common stock under our at-the-market equity offering program, our Note Purchase Agreement, former loan agreements, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC, and Yakult, the upfront payment and milestone payments received under the Secura APA, and sales of Series B Convertible Preferred Stock. Additionally, we have financed a portion of our operations through product revenue, including from AVMAPKI FAKZYNJA CO-PACK, beginning with our U.S. commercial launch in May 2025, and from COPIKTRA, from its U.S. commercial launch in September 2018 through our sale of the COPIKTRA license in September 2020. We expect to finance a portion of our business through future potential milestones and royalties received pursuant to the Secura APA.
Previous wording
As of September 30, 2025, we had $137.7 million of cash, cash equivalents, and investments. We primarily invest our cash, cash equivalents and investments in U.S. Government money market funds, U.S. government agency bonds, corporate bonds and commercial paper of publicly traded companies.
Current wording
As of March 31, 2026, we had $181.7 million in cash, cash equivalents, and investments. We primarily invest our cash, cash equivalents and investments in U.S. Government money market funds, government bonds, corporate bonds and commercial paper of publicly traded companies.
Previous wording
The following table sets forth the primary sources and uses of cash for the 2025 Period and the 2024 Period (in thousands):
Current wording
The following table sets forth the primary sources and uses of cash for the 2026 Quarter and the 2025 Quarter (in thousands):
Previous wording
Operating activities. The use of cash in both periods resulted primarily from our net losses adjusted for non-cash charges and changes in the components of working capital. Our cash outflow from net losses adjusted for non-cash charges and adjustments was $123.3 million and $78.7 million for the 2025 Period and the 2024 Period, respectively. Non-cash charges and adjustments for the 2025 Period were primarily related to the change in fair value of warrant liability, non-cash changes in fair value of the Notes, loss on debt extinguishment and stock-based compensation expense. Non-cash charges and adjustments for the 2024 Period were primarily related to the changes in fair value of the preferred stock tranche liability, the change in fair value of warrant liability and stock-based compensation expense. Our cash inflow from operating activities due to changes in operating assets and liabilities was $15.8 million for the 2025 Period primarily driven by an increase of $21.6 million in accrued expenses and other liabilities, and an increase of $4.5 million in accounts payable, partially offset by an increase of $6.7 million in accounts receivable, an increase of $1.9 million in prepaid expenses, other current assets and other assets, and an increase of $1.8 million in inventory. Ourcash outflow due to changes in operating assets and liabilities was $0.9 million for the 2024 Period was primarily driven by a decrease of $2.7 million in accounts payable, an increase of $1.9 million in prepaid expenses, other current assets and other assets, a decrease of $0.3 million in deferred liabilities, and an increase of $0.2 million in grant receivable partially offset by an increase of $4.2 million of accrued expenses and other liabilities. The increases or decreases in both periods in prepaid expenses, other current assets, and other asserts are exclusive of cash received from PanCAN and used on the
Current wording
The use of cash in both periods resulted primarily from our net losses adjusted for non-cash charges and changes in the components of working capital. Our cash outflow from net losses adjusted for non-cash charges and adjustments was $43.5 million and $42.9 million for the 2026 Quarter and the 2025 Quarter, respectively. Non-cash charges and adjustments for the 2026 Quarter were primarily related to the change in fair value of warrant liability and stock-based compensation expense. Non-cash charges and adjustments for the 2025 Quarter were primarily related to non-cash changes in fair value of the Notes, the change in fair value of warrant liability, loss on debt extinguishment and stock-based compensation expense.
Previous wording
Financing activities. The cash provided by financing activities for the 2025 Period represents $100.1 million of proceeds from issuance of common stock and pre-funded warrants, $75.0 million of proceeds received pursuant to the Note Purchase Agreement, $31.9 million of proceeds from the exercise of Warrants, $1.2 million of proceeds received from insurance premium financing, and less than $0.1 million of proceeds received from exercise of stock options and employee stock purchase program, partially offset by the $42.6 million repayment of our Loan Agreement, and $1.0 million of payments for insurance premium financing. The cash provided by financing activities for the 2024 Period represents $53.8 million of net proceeds received from the issuance of shares of common stock, pre-funded warrants, and warrants as part of the July 2024 Offering, $1.3 million of proceeds received from insurance premium financing and $0.2 million of proceeds received from exercise of stock options and our employee stock purchase plan, partially offset by $1.2 million of payments on insurance premium financing, and $0.2 million of fees paid to the Lenders to amend our Loan Agreement with Oxford. Refer toNote 10. Debt to our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the Note Purchase Agreement and Loan Agreement;Note 15. Capital Stock to our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the 2025 Private Placement, the Note Purchase Agreement, and the Warrants;Note 12. Notes Payableto our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the finance agreement with First Insurance related to insurance premium financing and the monthly payments of principal and interest related thereto.
Current wording
The cash provided by financing activities for the 2026 Period represents $29.4 million of proceeds from the exercise of Warrants, $0.7 million of proceeds received from insurance premium financing, and less than $0.1 million of proceeds received from exercise of stock options and employee stock purchase program, partially offset by the $1.3 million repayment of our vendor financing arrangement, and $0.2 million of payments for insurance premium financing. The cash provided by financing activities for the 2025 Quarter represents $75.0 million of proceeds received pursuant to the Note Purchase Agreement, $22.7 million of proceeds received under the August 2021 ATM, $7.4 million of proceeds received pursuant to the Stock Purchase Agreement, $4.1 million of proceeds from the exercise of Warrants, $1.2 million of proceeds received from insurance premium financing, and less than $0.1 million of proceeds received related to our employee stock purchase plan, partially offset by the $42.6 million repayment of our Loan Agreement, and $0.4 million of payments for insurance premium financing. Refer toNote 10. Debt to our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the Note Purchase Agreement and Loan Agreement;Note 15. Capital Stock to our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the 2025 Private Placement, the Note Purchase Agreement, and the Warrants;Note 12. Notes Payable to our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the finance agreement with First Insurance related to insurance premium financing and the monthly payments of principal and interest related thereto;Note 13. Vendor Financing Arrangement to our unaudited condensed consolidated financial statements included in this quarterly report for additional details on the vendor finance agreement with IQVIA related to the master service agreement.
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Current report — wording carried from elsewhere
Our discussion and analysis of our financial condition and results of operations is based upon our condensed consolidated financial statements prepared in accordance with U.S. generally accepted accounting principles. The
Current report — wording carried from elsewhere
Change in fair value of Notes
Current report — wording carried from elsewhere
Our cash outflow from operating activities due to changes in operating assets and liabilities was $8.6 million for the 2026 Quarter, primarily driven by a $3.1 million decrease in accrued expenses and other liabilities, $2.8 million decrease in accounts payable, a $1.3 million increase in accounts receivable, a $0.9 million increase in prepaid expenses, other current assets and other assets, and $0.5 million increase in inventory. Our cash inflow from operating activities due to changes in operating assets and liabilities was $4.3 million for the 2025 Quarter. Cash inflow due to changes in operating assets and liabilities for the 2025 Quarter was primarily driven by an increase of $3.2 million in accounts payable and an increase of $2.7 million in accrued expense and other liabilities, partially offset by an increase of $1.6 million in prepaid expenses, other current assets and other assets. Cash used in operating activities was $52.1 million and $38.7 million for the 2026 Quarter and the 2025 Quarter, respectively.
Rearranged previous paragraphs (8)
Previous report — wording carried elsewhere
Our operations to date have been focused on organizing and staffing our company, business planning, raising capital, identifying and acquiring potential product candidates, undertaking preclinical studies and clinical trials for our product candidates and initiating U.S. commercial operations following the approval of COPIKTRA through our ownership period ending in September 2020 and in anticipation of and following the approval of AVMAPKI FAKZYNJA CO-PACK in May 2025. We have financed our operations to date primarily through public and private offerings of our common stock, pre-funded warrants, and warrants, offerings of convertible notes, sales of common stock under our at-the-market equity offering program, our loan and security agreement executed with Hercules in March 2017, as amended, the Loan Agreement with Oxford, our Note Purchase Agreement, the upfront payments and milestone payments under our license and collaboration agreements with Sanofi, CSPC, and Yakult, the upfront payment and milestone payments received under the Secura APA, and sales of Series B Convertible Preferred Stock. Additionally, from our U.S. commercial launch of COPIKTRA on September 24, 2018, through our ownership period ending in September 2020, we financed a portion of our operations through product revenue. From our U.S. commercial launch of AVMAPKI FAKZYNJA CO-PACK on May 8 th, 2025, we are financing a portion of our operations through product revenue.
Previous report — wording carried elsewhere
the future, we may be unable to complete our planned preclinical studies and clinical trials and obtain approval of certain investigational product candidates from the FDA or foreign regulatory authorities. Therefore, there is substantial doubt about our ability to continue as a going concern.
Previous report — wording carried elsewhere
*Product Revenue, Net.*We began commercial sales of AVMAPKI FAKZYNJA CO-PACK within the United States in May 2025, following receipt of FDA marketing approval on May 8, 2025. For the nine months ended September 30, 2025 (the “2025 Period”) we recorded approximately $13.4 million of net product revenue. We had no product revenue during the nine months ended September 30, 2024 (the “2024 Period”).
Previous report — wording carried elsewhere
*Costs of sales – product.*Costs of sales – product for the 2025 Period of approximately $2.0 million consisted of costs associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK, royalties owed to Chugai, Pfizer, and ICR on such sales, and certain period costs. We began capitalizing inventory upon receiving FDA approval for AVMAPKI FAKZYNJA CO-PACK on May 8, 2025. Prior to the FDA approval of AVMAPKI FAKZYNJA CO-PACK, expenses associated with the manufacturing of AVMAPKI FAKZYNJA CO-PACK were recorded as research and development expense. Certain of the costs of AVMAPKI FAKZYNJA CO-PACK units recognized as revenue during the 2025 Period, or approximately $0.1 million, were expensed prior to obtaining regulatory approval, therefore, are not included in cost of sales - product during this period. We expect cost of sales - product to increase in relation to product revenues as we deplete these inventories. We had no cost of sales – product during the 2024 Period.
Previous report — wording carried elsewhere
*Costs of sales –intangible amortization.*Cost of sales – intangible amortization for the 2025 Period of approximately $0.4 million was related to finite-lived intangible assets related to AVMAPKI FAKZYNJA CO-PACK which we recognized and began amortizing during the second quarter of 2025. There was no cost of sales – intangible amortization during the 2024 Period.
Previous report — wording carried elsewhere
The table below summarizes our direct research and development expenses for our product/ product candidates/ projects and our unallocated research and development costs for the 2025 Period and the 2024 Period.
Previous report — wording carried elsewhere
Other expense. Other expense for the 2025 Period was $0.2 million compared to $0.1 million for 2024 Period. Other expense for the 2025 Period and 2024 Period was comprised of transaction losses due to changes in foreign currency exchange rates.
Previous report — wording carried elsewhere
*Change in fair value of Notes.*We elected the fair value option for the Notes and therefore the changes in fair value, including interest, other than changes that are directly attributable to instrument specific credit risk are recorded as change in fair of Notes in the condensed statements of operations and comprehensive loss. The change in fair value for the 2025 Period of $10.2 million was primarily driven by interest on the Notes and a reduction in the risk-free rate during the 2025 Period. There were no Notes outstanding in the 2024 Period.