On May 11, 2021 Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer, reported recent business progress and first quarter financial results (Press release, Kronos Bio, MAY 11, 2021, View Source [SID1234579668]).

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"With continued progress in our preclinical and clinical programs and significant milestones expected this year, we look forward to hosting a research and development day to present our pipeline and unveil our development strategy for both of our clinical-stage SYK inhibitors entospletinib and lanraplenib," said Norbert Bischofberger, Ph.D., president and CEO. "We recently showcased progress with our CDK9 program at the AACR (Free AACR Whitepaper) annual meeting in April, where we presented preclinical data indicating that KB-0742 could have utility in the treatment of MYC-amplified cancers. We look forward to sharing more about our development plans for this compound at our research and development day."

Dr. Bischofberger added: "I would also like to take this opportunity to reflect on the recent passing of our Board Member, Dr. John C. Martin, a dear friend and mentor, and to convey our deepest sympathies to John’s family and everyone who was fortunate enough to have known him."

Recent Highlights

Presented preclinical data for KB-0742, the company’s potent oral, highly selective cyclin dependent kinase 9 (CDK9) inhibitor, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data showed that CDK9 inhibition on an intermittent dosing schedule with KB-0742 resulted in sustained inhibition of tumor growth in multiple types of solid tumors, and suggested that genomic amplification of MYC, a well-characterized transcription factor and a long-recognized driver of cancer, is a key feature in defining sensitivity to CDK9 inhibition.

Announced the appointment of Taiyin Yang, Ph.D., to its board of directors. Dr. Yang currently serves as the executive vice president of Pharmaceutical Development and Manufacturing at Gilead Sciences, Inc., and has more than four decades of experience developing and manufacturing medicines in a variety of therapeutic categories.

Announced a positive End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for spleen tyrosine kinase (SYK) inhibitor entospletinib. Kronos Bio will proceed with its plan to assess measurable residual disease (MRD) negative complete response (CR) as the primary endpoint in a registrational Phase 3 trial to support potential accelerated approval of entospletinib in patients newly diagnosed with NPM1-mutated acute myeloid leukemia (AML). The company plans to initiate the Phase 3 trial in mid-2021, with MRD negative CR data expected in the second half of 2023.

Announced the first patient was dosed in the Phase 1/2 clinical trial of KB-0742, which is being developed to treat MYC-amplified solid tumors.
Pipeline updates planned at upcoming Virtual R&D Day on May 25, 2021

Unveil the development strategy for the company’s SYK inhibitors entospletinib and lanraplenib in AML. Kronos Bio executives will be joined by Eytan M. Stein, M.D., assistant attending physician and director, Program for Drug Development in Leukemia, Leukemia Service, Department of Medicine at Memorial Sloan Kettering Cancer Center, who will provide an overview of AML and the current treatment landscape.

Highlight the opportunity to target MYC through CDK9 inhibition with KB-0742, including a review of preclinical data, expectations for initial safety, pharmacokinetic and pharmacodynamic data anticipated in the fourth quarter of 2021 and potential populations for the expansion cohorts in the second stage of the company’s Phase 1/2 clinical trial.

Provide an overview of the company’s differentiated drug discovery platform and potential future pipeline programs.
The live webinar will begin at 1 p.m. ET on Tuesday, May 25, 2021, and will conclude at approximately 4:00 p.m. ET. Registration is accessible on the Investors & Media section of the company’s website at www.kronosbio.com. A replay of the webcast will be archived and available following the event.

First Quarter Financial Highlights

Cash, Cash Equivalents and Investments: As of March 31, 2021, cash, cash equivalents and investments totaled $440.6 million.

R&D Expenses: Research and development expenses were $17.6 million for the first quarter of 2021, which includes $2.5 million in non-cash stock-based compensation expense. R&D expenses for the quarter were primarily driven by costs associated with initiating and conducting the company’s clinical trials.

G&A Expenses: General and administrative expenses were $8.6 million for the first quarter of 2021, which includes $2.7 million in non-cash stock-based compensation expense.

Net Loss: Net loss for the first quarter of 2021 was $26.1 million, or $0.48 per share, including non-cash stock-based compensation expense of $5.2 million.

On May 11, 2021 Dynavax Technologies Corporation ("Dynavax") (Nasdaq: DVAX) reported the pricing of $200.0 million aggregate principal amount of 2.50% convertible senior notes due 2026 (the "notes") in a private placement to persons reasonably believed to be qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act") (Press release, Dynavax Technologies, MAY 11, 2021, View Source [SID1234579684]). Dynavax also granted the initial purchasers of the notes an option to purchase up to an additional $30.0 million aggregate principal amount of notes. The sale of the notes is expected to close on May 13, 2021, subject to customary closing conditions.

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The notes will be general unsecured obligations of Dynavax and will accrue interest payable semiannually in arrears on May 15 and November 15 of each year, beginning on November 15, 2021, at a rate of 2.50% per year. The notes will mature on May 15, 2026, unless earlier converted, redeemed or repurchased. The initial conversion rate of the notes will be 95.5338 shares of Dynavax’s common stock per $1,000 principal amount of notes (equivalent to an initial conversion price of approximately $10.47 per share of Dynavax’s common stock). The initial conversion price of the notes represents a premium of approximately 32.5% over the last reported sale price of Dynavax’s common stock on May 10, 2021. The notes will be convertible by the holders thereof into cash, shares of Dynavax’s common stock or a combination of cash and shares of Dynavax’s common stock, at Dynavax’s election.

Dynavax may redeem for cash all or any portion of the notes, at its option on or after May 20, 2024 and prior to the 31st scheduled trading day immediately preceding the maturity date, if the last reported sale price of Dynavax’s common stock has been at least 130% of the conversion price then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period (including the last trading day of such period) ending on, and including, the trading day immediately preceding the date on which Dynavax provides notice of redemption, at a redemption price equal to 100% of the principal amount of the notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date.

If Dynavax undergoes a "fundamental change" (as defined in the indenture governing the notes), subject to certain conditions and exceptions, noteholders may require Dynavax to repurchase for cash all or any portion of their notes at a fundamental change repurchase price equal to 100% of the principal amount of the notes to be repurchased, plus accrued and unpaid interest to, but excluding, the fundamental change repurchase date. In addition, following certain corporate events that occur prior to the maturity date or if Dynavax delivers a notice of redemption, Dynavax will, in certain circumstances, increase the conversion rate for a noteholder who elects to convert its notes in connection with such a corporate event or convert its notes called (or deemed called) for redemption in connection with such notice of redemption, as the case may be.

Dynavax estimates that the net proceeds from the offering of the notes will be approximately $195.1 million (or approximately $224.4 million if the initial purchasers exercise their option to purchase additional notes in full), after deducting the initial purchasers’ discounts and commissions and estimated offering expenses payable by Dynavax. Dynavax expects to use the net proceeds from the offering of the notes, together with cash on hand, to repay in full the outstanding debt and other obligations under Dynavax’s term loan agreement and to pay the costs of the capped call transactions described below. Dynavax expects that approximately $190.2 million will be needed to repay the outstanding debt and other obligations under the term loan agreement and approximately $24.2 million will be needed to pay the cost of the capped call transactions.

If the initial purchasers exercise their option to purchase additional notes in full, Dynavax expects to use approximately $3.6 million of the net proceeds from the sale of such additional notes to enter into additional capped call transactions. Dynavax expects to use any remaining net proceeds from the sale of such additional notes for general corporate purposes.

In connection with the pricing of the notes, Dynavax entered into capped call transactions with one of the initial purchasers and other financial institutions (the "option counterparties"). The capped call transactions will cover, subject to customary adjustments, the number of shares of Dynavax’s common stock that initially underlie the notes. The capped call transactions are expected to offset the potential dilution to Dynavax’s common stock as a result of any conversion of notes, with such offset subject to a cap initially equal to $15.80 (which represents a premium of 100% over the last reported sale price of Dynavax’s common stock on May 10, 2021).

In connection with establishing their initial hedges of the capped call transactions, Dynavax has been advised that the option counterparties and/or their respective affiliates expect to enter into various derivative transactions with respect to Dynavax’s common stock concurrently with or shortly after the pricing of the notes and/or purchase shares of Dynavax’s common stock concurrently with or shortly after the pricing of the notes. This activity could increase (or reduce the size of any decrease in) the market price of Dynavax’s common stock or the trading price of the notes at that time.

In addition, the option counterparties and/or their respective affiliates may modify their hedge positions by entering into or unwinding various derivatives with respect to Dynavax’s common stock and/or purchasing or selling Dynavax’s common stock or other securities of Dynavax in secondary market transactions following the pricing of the notes and prior to the maturity of the notes (and are likely to do so on each exercise date of the capped call transactions, which are expected to occur during the 30 trading day period beginning on the 31st scheduled trading day prior to the maturity date of the notes, or following any termination of any portion of the capped call transactions in connection with any repurchase, redemption or early conversion of the notes). This activity could also cause or avoid an increase or a decrease in the market price of Dynavax’s common stock or the notes, which could affect a noteholder’s ability to convert its notes and, to the extent the activity occurs during any observation period related to a conversion of notes, it could affect the amount and value of the consideration that a noteholder will receive upon conversion of such notes.

Neither the notes, nor any shares of Dynavax’s common stock issuable upon conversion of the notes, have been registered under the Securities Act or any state securities laws, and unless so registered, may not be offered or sold in the United States absent registration or an applicable exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and other applicable securities laws.

This press release is neither an offer to sell nor a solicitation of an offer to buy any securities, nor shall it constitute an offer, solicitation or sale of the securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

On May 11, 2021 Aptevo Therapeutics Inc. ("Aptevo" or "the Company") (NASDAQ:APVO), a clinical-stage biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported its financial results for the quarter ended March 31, 2021 (Press release, Aptevo Therapeutics, MAY 11, 2021, View Source [SID1234579700]).

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"We are pleased to report progress in the clinical trial of our ADAPTIR platform candidate APVO436, with the dose limiting toxicity (DLT) evaluation in cohorts 1 through 9 now completed and 44 patients enrolled to date in cohorts 1-10. We remain confident in the clinical impact potential of APVO436 and our platform technologies, and look forward to sharing interim data readouts from our ongoing APVO436 study later this year," said Mr. Marvin White, President and CEO of Aptevo. "The transaction with HCR, which we completed in March 2021, provided significant non-dilutive funding, which adds to our cash runway and helps fund the company through the next twelve months."

First Quarter 2021 Financial Results Summary

As announced in March, Aptevo received $35 million ("the Investment Amount") from its sale of the right to royalty payments made by Pfizer Inc. ("Pfizer") with respect to net sales of RUXIENCE to an entity managed by HealthCare Royalty Management, LLC ("HCR"). Aptevo is eligible to receive additional payments in aggregate of up to an additional $32.5 million based on the achievement of sales milestones in 2021, 2022, and 2023 (collectively, the "Milestone Amounts"). The Royalty Purchase Agreement further provides that, once HCR reaches aggregate royalty payments totaling 190% of the Investment Amount plus the Milestone Amounts to the extent paid by HCR to the Company, Aptevo will be entitled to receive 50% of any additional royalty payments by Pfizer thereafter. In connection with this royalty purchase agreement, Aptevo amended its term loan agreement with MidCap Financial and used $10 million of the proceeds received to pay down outstanding principal.

Cash Position: Aptevo had cash and cash equivalents as of March 31, 2021 totaling $58.8 million, including restricted cash of $1.3 million. The restricted cash is expected to be released over the next twelve months.

Royalty Revenue: Royalty revenue was $2.4 million for the three months ended March 31, 2021, related to the royalty from Pfizer on global net sales of RUXIENCE, a biosimilar to the drug RITUXAN, launched by Pfizer in early 2020. RUXIENCE is a trademark of Pfizer; RITUXAN is a trademark of Biogen. Due to our continuing involvement under the Definitive Agreement originally between Trubion and Wyeth, we continue to recognize royalty revenue on net sales of RUXIENCE and record the royalty payments to HCR as a reduction of the liability when paid. As such payments are made to HCR, the balance of the liability will be effectively repaid over the life of the Royalty Purchase Agreement.

Research and Development Expenses: Research and development expenses increased to $5.4 million for the three months ended March 31, 2021 from $4.0 million for the three months ended March 31, 2020 as we continue to invest in the APVO436 clinical trial and our preclinical candidates, including ALG.APV-527, APVO603 and APVO442.

General and Administrative Expenses: For the three months ended March 31, 2021, general and administrative expenses increased to $3.9 million from $3.6 million for March 31, 2020, with higher costs for professional services.

Other Expense, Net: Other expense, net consists primarily of interest on debt and costs related to debt extinguishment. Other expense, net was $0.8 million for the three months ended March 31, 2021 and $2.4 million for the three months ended March 31, 2020. A slight increase in interest expense this quarter was offset by a significant decrease due to a loss on extinguishment of debt of $2.1 million recognized in the first quarter of 2020 when we repaid our previous loan to MidCap Financial using the proceeds from the sale of Aptevo BioTherapeutics LLC.

Discontinued Operations: Income from discontinued operations was $0.4 million for the three months ended March 31, 2021 and $12.9 million for the three months ended March 31, 2020. For the three months ended March 31, 2021, we collected a deferred payment of $0.2 million from Medexus related to fourth quarter 2020 IXINITY sales and $0.2 million from the Saol Therapeutics, related to the 2017 sale of the Hyperimmune Business to them. For the three months ended March 31, 2020, we recognized net income from discontinued operations totaling $12.9 million. This included the gain on the sale of Aptevo BioTherapeutics LLC of $14.3 million and net operating losses from Aptevo BioTherapeutics LLC of $1.6 million related to the period prior to the sale on February 28, 2020.

Net Income (Loss): Aptevo’s net loss for the three-month period ended March 31, 2021 was $(7.3) million or $(1.64) per share, as compared to a net income of $2.9 million or $0.89 per share for the corresponding period in 2019. Our net loss from continuing operations for the first quarter of 2021 was $(7.6) million compared to $(10.0) million in the first quarter of 2020.

Liability Related to Sale of Future Royalties: We treat the Royalty Purchase Agreement with HCR as a debt financing, amortized under the effective interest rate method over the estimated life of the related expected royalty stream. The liabilities related to sale of future royalties and the debt amortization are based on our current estimates of future royalties expected to be paid over the life of the arrangement. We will periodically assess the expected royalty payments using projections from external sources. To the extent our estimates of future royalty payments are greater or less than previous estimates or the estimated timing of such payments is materially different than previous estimates, we will adjust the effective interest rate and recognize related non-cash interest expense on a prospective basis. We are not obligated to repay the proceeds received under the Royalty Purchase Agreement with HCR.

On May 11, 2021 Ultimovacs ASA ("Ultimovacs") (OSE ULTI), a clinical stage leader in immune stimulatory vaccines for cancer, reported the publication in Frontiers in Immunology of its positive long-term Overall Survival (OS) data from the Phase I trial evaluating the Company’s universal cancer vaccine, UV1, in combination with checkpoint inhibitor ipilimumab in patients with metastatic malignant melanoma (Press release, Ultimovacs, MAY 11, 2021, View Source [SID1234579716]). As published in the journal, in addition to the achievement of the primary endpoints of safety and tolerability, 50% of the patients were still alive at the data cut-off, supporting the combination of the Company’s proprietary UV1 vaccine with ipilimumab, a CTLA-4 checkpoint inhibitor and standard-of-care treatment, in this late-stage patient population.

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"Publishing our clinical trial data in such a prestigious peer-reviewed publication adds validation for UV1 as a promising therapeutic option for cancer patients. As we continue to evaluate UV1 in various combinations and indications, it is valuable to gain increased international recognition from the clinical community for this study," stated Jens Bjørheim, Chief Medical Officer at Ultimovacs. "Historical data on the use of ipilimumab as monotherapy in malignant melanoma have shown a 5-year survival rate below 20%, therefore the results published today reinforce UV1’s potential in this indication."

The data published in Frontiers in Immunology covers 4.8 years of follow-up on the total of 12 metastatic malignant melanoma patients that were enrolled in the Phase I trial. As reported in the journal, the OS was 50% at 4.8 years, which was confirmed by the results of 5 years of follow-up announced by the Company in December 2020.

Building on these promising Phase I results, Ultimovacs is currently enrolling INITIUM, its Phase II clinical trial evaluating UV1 in combination with ipilimumab and nivolumab in patients with metastatic malignant melanoma. The company expects to announce data on the trial’s primary endpoint in 2H2022. In addition, Ultimovacs has an ongoing and fully-enrolled Phase I trial evaluating UV1 in combination with pembrolizumab, a PD-1 checkpoint inhibitor, as a first line treatment in metastatic malignant melanoma patients.

The publication in Frontiers in Immunology can be found under doi: 10.3389/fimmu.2021.663865.

About UV1

UV1 is a peptide-based vaccine inducing a specific T cell response against the universal cancer antigen telomerase. UV1 is being developed as an "off-the-shelf" therapeutic cancer vaccine which may serve as a platform for use in combination with other immunotherapy which requires an ongoing T cell response for their mode of action. To date, UV1 has been tested in four phase I clinical trials in a total of 82 patients and maintained a positive safety and tolerability profile as well as encouraging signals of efficacy.

About UV1 Clinical Programs

As a universal cancer vaccine, UV1’s unique mechanism of action has the potential to be applicable across most cancer types. The clinical development of the UV1 vaccine includes four randomized, multinational, Phase II combination trials: INITIUM, NIPU, DOVACC and FOCUS, recruiting over 500 patients in total. The INITIUM trial is an Ultimovacs-sponsored clinical trial recruiting 154 patients with metastatic malignant melanoma to evaluate UV1 in combination with ipilimumab and nivolumab as first-line treatment. The NIPU study is testing UV1 in combination with checkpoint inhibitors ipilimumab and nivolumab as second-line treatment in 118 patients with advanced malignant pleural mesothelioma, a rare lung cancer. The study is sponsored by Oslo University Hospital and Bristol-Myers Squibb is providing the checkpoint inhibitors for this study. The DOVACC study is sponsored by the Nordic Society of Gynaecological Oncology. In total, 184 patients with high-grade ovarian cancer will be enrolled to evaluate UV1 in combination with durvalumab and olaparib, both provided by AstraZeneca. FOCUS is an investigator-sponsored, randomized clinical trial enrolling 75 patients with metastatic head and neck cancer receiving pembrolizumab as standard of care, and will evaluate the impact of adding UV1 to this regimen. Ultimovacs anticipates announcing data on the primary endpoints for the NIPU and INITIUM studies in 2H2022 and for the DOVACC and FOCUS studies in 2023.

On May 11, 2021 Verastem, Inc. (Nasdaq: VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to advancing new medicines for patients battling cancer, reported financial results for the three months ended March 31, 2021, highlighted recent progress and outlined key corporate objectives (Press release, Verastem, MAY 11, 2021, View Source [SID1234579808]).

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"During the first quarter of 2021, we expanded the selection portion of the Phase 2 RAMP 201 study to include all recurrent low-grade serous ovarian cancer types. This decision was based on the positive updated data from the LGSOC cohort of the Phase 1/2 FRAME which continues to show strong response rates across both KRAS mutant and wild-type recurrent LGSOC, along with robust durability and a favorable tolerability profile," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "We closed the first quarter with just over $127 million in cash, cash equivalent and investments, leaving us well positioned to execute on our two ongoing Phase 2 studies evaluating VS-6766 and defactinib in LGSOC and KRAS G12V non-small cell lung cancer (NSCLC), as well as our other key corporate objectives."

Recent Corporate Highlights

LGSOC

Reported updated data from the LGSOC cohort of the ongoing, investigator-sponsored Phase 1/2 FRAME study evaluating VS-6766 in combination with defactinib in patients with recurrent LGSOC. Combination continues to demonstrate activity, durability and a favorable tolerability profile, including in patients who have progressed following treatment with a MEK inhibitor.

Overall response rate (ORR) across all patients was 52% (11 of 21 patients).

ORR for patients with KRAS mutant LGSOC was 70% (7 of 10 patients).

ORR for patients with wild type LGSOC was 44% (4 of 9 patients).

The most common side effects were Grade 1/2 rash, creatine kinase elevation, nausea, hyperbilirubinemia and diarrhea, which were reversible.
Company-sponsored, registration-directed Phase 2 study (RAMP 201) underway investigating VS-6766 alone and in combination with defactinib for the treatment of recurrent LGSOC. Study recently expanded to include both KRAS mutant and KRAS wild-type patients in the selection phase to determine the optimal go-forward regimen for both types of LGSOC.
KRAS G12V Mutant NSCLC

Company-sponsored, registration-directed Phase 2 study (RAMP 202) underway investigating VS-6766 alone and in combination with defactinib for the treatment of patients with KRAS G12V mutant NSCLC.
Upcoming Milestones and Key Priorities for 2021-2022

LGSOC

Updated data from Phase 1/2 FRAME study to be submitted for presentation at a major medical meeting during the second half of 2021.
Complete selection portion of RAMP 201 during first half of 2022; commence expansion portion.
G12V NSCLC

Complete selection portion of RAMP 202 during first half of 2022; commence expansion portion.
First Quarter 2021 Financial Results

Verastem Oncology ended the first quarter 2021 with cash, cash equivalents and investments of $127.1 million.

Total revenue for the three months ended March 31, 2021 (2021 Quarter) was $1.0 million, compared to $5.1 million for the three months ended March 31, 2020 (2020 Quarter).

Total operating expenses for the 2021 Quarter were $15.1 million, compared to $31.4 million for the 2020 Quarter.

Selling, general and administrative expenses for the 2021 Quarter were $6.2 million, compared to $19.6 million for the 2020 Quarter. The decrease of $13.4 million, or 68.4%, primarily resulted from the Company’s shift in strategic direction and COPIKTRA sale to Secura Bio, Inc., which led to lower employee related expenses and consulting and professional fees.

Research and development expense for the 2021 Quarter was $8.9 million, compared to $10.9 million for the 2020 Quarter. The decrease of $2.0 million, or 18.3%, was primarily related to the upfront non-refundable payment of $3.0 million to Chugai Pharmaceutical Co., Ltd for the VS-6766 license in the 2020 Quarter and decreased contract research organization costs, partially offset by increased drug substance and drug product costs and increased investigator sponsored trial expenses.

Net loss for the 2021 Quarter was $15.0 million, or $0.09 per share (basic and diluted), compared to $38.0 million, or $0.35 per share (basic and diluted), for the 2020 Quarter.

For the 2021 Quarter, non-GAAP adjusted net loss was $12.4 million, or $0.07 per share (diluted), compared to non-GAAP adjusted net loss of $21.3 million, or $0.20 per share (diluted), for the 2020 Quarter. Please refer to the GAAP to Non-GAAP Reconciliation attached to this press release.

Financial Guidance and Outlook

With the proceeds from the sale of COPIKTRA, Verastem Oncology expects that it will have a cash runway until at least 2024 to deliver on the current programs for VS-6766 and defactinib, including clinical and regulatory milestones and development in LGSOC and KRAS mutant NSCLC. Verastem Oncology expects its 2021 annual operating expenses to be approximately $50 million.

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 the United States (GAAP), the Company uses the following non-GAAP financial measures in this press release: non-GAAP adjusted net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company’s GAAP financial statements, because it provides greater transparency and period-over-period comparability with respect to the Company’s operating performance and can enhance investors’ ability to identify operating trends in the Company’s business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company’s operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months ended March 31, 2021 and 2020 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

About VS-6766

VS-6766 (formerly known as CH5126766 and RO5126766) is a unique inhibitor of the RAF/MEK signaling pathway. In contrast to other MEK inhibitors in development, VS-6766 blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows VS-6766 to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors.

About Defactinib

Defactinib (VS-6063) is an oral small molecule inhibitor of FAK and PYK2 that is currently being evaluated as a potential combination therapy for various solid tumors. The Company has received Orphan Drug designation for defactinib in ovarian cancer and mesothelioma in the US, EU and Australia. Preclinical research by Verastem Oncology scientists and collaborators at world-renowned research institutions has described the effect of FAK inhibition to enhance immune response by decreasing immuno-suppressive cells, increasing cytotoxic T cells, and reducing stromal density, which allows tumor-killing immune cells to enter the tumor.1,2

About the VS-6766/Defactinib Combination

RAS mutant tumors are present in ~30% of all human cancers, have historically presented a difficult treatment challenge and are often associated with significantly worse prognosis. Challenges associated with identifying new treatment options for these types of cancers include resistance to single agents, identifying tolerable combination regimens with MEK inhibitors and new RAS inhibitors in development addressing only a minority of all RAS mutated cancers.

The combination of VS-6766 and defactinib has been found to be clinically active in patients with KRAS mt tumors. In an ongoing investigator-initiated Phase 1/2 FRAME study, the combination of VS-6766 and defactinib is being evaluated in patients with LGSOC, KRAS mt NSCLC and colorectal cancer (CRC). The FRAME study was expanded to include new cohorts in pancreatic cancer, KRASmt endometrioid cancer and KRAS-G12V NSCLC. Verastem Oncology is also supporting an investigator-initiated Phase 2 trial evaluating VS-6766 with defactinib in patients with metastatic uveal melanoma.

Verastem Oncology has initiated Phase 2 registration-directed trials of VS-6766 with defactinib in patients with recurrent LGSOC and in patients with recurrent KRAS-G12V NSCLC as part of its RAMP (Raf And Mek Program).