On March 18, 2021 Amplia Therapeutics (ATX) reported that it has signed a term sheet for a collaboration agreement with the Garvan Institute of Media Research in Sydney (Press release, Amplia Therapeutics, MAR 18, 2021, View Source;[email protected] [SID1234576887]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The term sheet maps out a collaborative research and clinical development program, focused on the use of Amplia’s FAK inhibitor, AMP945, to treat patients with pancreatic cancer.

The collaboration will initially look at new treatment regiments and patient selection strategies for Amplia’s clinical trials of AMP945 in pancreatic cancer patients later this year.

The collaboration will combine the Garvan Institute’s research strength in FAK biology and its extensive clinical research network with Amplia’s proprietary FAK inhibitors and drug development capability.

"The purpose of this collaboration is to optimise our planned clinical program for AMP945," said Amplia’s CEO and Managing Director, Dr John Lambert.

"Clearly, Garvan’s team has an unsurpassed understanding of FAK biology and access to an extensive clinical network. By leveraging Garvan’s deep understanding of the different biological roles that FAK can play, we aim to optimise the design of our planned clinical trials, recruit the right pancreatic cancer patients and treat them in the right way, giving AMP945 the best chance for success."

Professor Paul Timpson, Cancer Research Theme Leader at Garvan will be the lead scientist in the collaborative research program, after collaborating with Amplia for over a year.

The terms of the agreement are confidential but include future payments by Amplia to Garvan based on AMP945 achieving specified clinical, regulatory and commercial sales milestones.

The final Collaboration Agreement is expected to be signed within 90 days. While the terms of the agreement are confidential, they do include future, success-based payments by Amplia to Garvan on AMP945 achieving specified clinical, regulatory and commercial sales milestones.

This ASX announcement was approved and authorised for release by the Board of Amplia Therapeutics

On March 18, 2021 Aktis Oncology, a biotechnology company discovering and developing a novel class of targeted radiopharmaceuticals to treat a broad range of solid tumor cancers, reported completion of a $72 million Series A financing (Press release, Aktis Oncology, MAR 18, 2021, View Source [SID1234576886]). The company was founded and incubated by MPM Capital, which co-led the financing with EcoR1 Capital and Vida Ventures, with participation from Octagon Capital, TCG Crossover, Novartis and Bristol Myers Squibb.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Aktis Oncology is building on recent successes in the radiopharmaceutical field, using a novel approach to extend the efficacy of targeted radiopharmaceuticals into a wide range of solid tumors," said Todd Foley, Chairman of Aktis Oncology, and Managing Director of MPM Capital. "We’re incredibly excited by the science, the team and a world class syndicate of institutional and strategic investors that share in our vision to realize the potential of targeted radiotherapy to become a mainstream anti-cancer modality."

Aktis Oncology is developing novel alpha radiotherapies to improve upon the efficacy of conventional solid tumor therapies, while limiting toxicity. The company has developed proprietary platforms to discover best-in-class tumor targeting agents designed with optimal pharmacology for delivering alpha radiotherapy. Aktis Oncology targeting agents are highly tumor penetrant but clear from other areas of the body quickly, to maximize tumor elimination while minimizing potential side effects of treatment.

"Alpha radiotherapy is the future of radiopharmaceuticals," said Matthew Roden, PhD, President and Chief Executive Officer of Aktis Oncology. "By harnessing the power of alpha particles – with nearly 1000 times the potency of beta particles – we believe our approach has the potential to deliver game-changing results for patients."

Aktis Oncology’s theranostic approach would also enable clinicians to visualize and verify target engagement prior to exposure to therapeutic radioisotopes, allowing for better selection of patients most likely to benefit from therapy. The company plans to produce multiple development candidates with potential for efficacy against well-validated cancer targets.

Aktis Oncology was founded by Brian Goodman, PhD, Patrick Baeuerle, PhD, and Todd Foley, of MPM Capital. The company is led by:

Matthew Roden, PhD, President and Chief Executive Officer of Aktis Oncology. He is an Executive Partner of MPM Capital, and serves as Chairman of Turmeric Acquisition Corporation, and is a member of the boards of iTeos Therapeutics and NextPoint Therapeutics. Prior to joining MPM, Dr. Roden held senior leadership positions in the biopharma industry, most recently as Senior Vice President and Head of Enterprise Strategy at Bristol Myers Squibb; he held senior roles in the capital markets, including serving as head of biotechnology equity research at UBS and as a senior biotechnology equity analyst at J.P. Morgan; and has scientific training in immunology, structural biology, and cancer biology.
Paul Feldman, PhD, Chief Scientific Officer of Aktis Oncology. Prior to joining Aktis Oncology Dr. Feldman was the Head of Discovery and Translational Medicine at Intarcia Therapeutics. An accomplished drug discoverer and executive in the biopharmaceutical industry, he previously served as Senior Vice President at GlaxoSmithKline, leading the Enteroendocrine Discovery Performance Unit and GSK’S enterprise Chemistry Council, before retiring to co-found Phoundry Pharmaceuticals, a peptide hormone therapeutic company.
Brian Goodman, PhD, Head of Operations and Corporate Development of Aktis Oncology. As part of the Investment and Operations Team at MPM Capital, he is responsible for investment identification, due diligence, business development, and new company creation at a number of MPM portfolio companies. He serves as a board observer for Orna Therapeutics and Triplet Therapeutics. Prior to joining MPM Capital, Dr. Goodman co-founded and served as Head of Technology and Innovation at Evelo Biosciences, a company focused on the pharmacology of microbial drugs across multiple therapeutic areas; and was a Senior Associate at Flagship Pioneering.
Aktis Oncology is headquartered in Cambridge, Mass. with laboratory operations in Research Triangle Park, NC.

On March 18, 2021 Sutro Biopharma, Inc. (NASDAQ: STRO), a clinical-stage drug discovery, development and manufacturing company focused on the application of precise protein engineering and rational design to create next-generation cancer and autoimmune therapeutics, reported its financial results for the year ended December 31, 2020, its recent business highlights, and provided a preview of anticipated selected milestones in 2021 (Press release, Sutro Biopharma, MAR 18, 2021, View Source [SID1234576885]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2020 was a highly productive year for Sutro as we advanced the clinical development of our programs and navigated through the pandemic. Four product candidates that have now entered the clinic, two of which are partnered programs, were discovered and developed using our cell-free protein synthesis platform, showcasing its strength and versatility," said Bill Newell, Sutro’s Chief Executive Officer. "We were particularly encouraged to see meaningful clinical benefit for women with advanced platinum-resistant and refractory ovarian cancer who were part of the Phase 1 dose-escalation study for STRO-002, our FolRα-targeted ADC. We dosed the first patient in dose-expansion in January 2021 and are rapidly moving this program forward. Additionally, we are progressing well on our partnerships – our Bristol Myers Squibb and EMD Serono collaboration programs are enrolling patients in Phase 1 studies and we are continuing to work with Merck on the cytokine derivatives collaboration to bring its first program to the clinic. We look forward to continued progress on our clinical programs and utilizing our industry leading cell-free platform to advance additional product candidates to benefit patients with unmet medical needs."

Recent Business Highlights and Expected 2021 Milestones

STRO-002: Continued progress in Phase 1 trial of STRO-002, folate receptor-alpha (FolRα)-targeted antibody-drug conjugate (ADC) for patients with recurrent platinum resistant or refractory ovarian cancer.

Dose-escalation portion of the Phase 1 trial completed enrollment as of August 31, 2020 and interim data as of October 30, 2020 were presented by key opinion leaders (KOL) and management at the KOL Discussion of STRO-002 Data Event in December 2020.
Additional data from dose-escalation, with extended follow-up on patients remaining on study, is expected in the first half of 2021.
Dose-expansion portion of the Phase 1 trial began enrolling patients in January 2021 and initial dose-expansion data is expected to be reported in the second half of 2021.
Dose-expansion data is expected to inform regulatory interactions, potentially accelerate development of our registration strategy, and enable identification of the broadest population that may benefit from STRO-002.
STRO-001: Phase 1 dose-escalation continues for STRO-001, a CD74-targeted ADC for development in B-cell malignancies.

Dose-escalation in the Phase 1 trial enrolling patients with lymphoma and multiple myeloma is ongoing and the maximum tolerated dose has not yet been reached.
Interim data from the dose-escalation portion of the trial in patients with non-Hodgkin lymphoma and preclinical data from our collaboration with Fred Hutchinson Cancer Research Center were presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020.
The dose-expansion portion of the Phase 1 trial is expected to begin enrolling patients in the second half of 2021.
Merck collaboration: Working collaboratively with Merck to advance two cytokine derivative programs towards the clinic.

Sutro is continuing to work with Merck to discover new therapeutics for cancer and autoimmune diseases. The collaboration is advancing two cytokine-derivative programs through the research phase.
In March 2020, Merck extended by one year the research term of the collaboration’s first program, which included a $5.0 million payment to Sutro.
In August 2020, Sutro entered into a supply agreement with Merck, giving Sutro responsibility for manufacturing pre-clinical and clinical supply for products emerging from the collaboration.
Bristol Myers Squibb (BMS) collaboration: Phase 1 trial for CC-99712, a BCMA-targeted ADC, is continuing to enroll multiple myeloma patients.

Since initiation of Phase 1 in the second half of 2019, BMS has been enrolling patients in a dose-escalation/expansion trial to assess treatment of relapsed and refractory multiple myeloma, with the last reported dose level at 3.0 mg/kg.
CC-99712 was granted Orphan Drug Designation by the FDA for multiple myeloma.
BMS is responsible for the worldwide clinical development and commercialization of CC–99712. Sutro is responsible for clinical supply manufacturing and certain development services for CC-99712 and is entitled to development and regulatory milestone or contingent payments and tiered royalties on sales ranging from mid to high single digit percentages.
Merck KGaA, EMD Serono (EMD Serono) collaboration: Phase 1 trial for M1231, a first-in-class bispecific ADC targeting MUC1–EGFR for development in solid tumors, was initiated in the first quarter of 2021.

EMD Serono is enrolling patients in the dose-escalation portion of a Phase 1 trial of M1231 for treatment of metastatic solid tumors including non-small cell lung cancer (NSCLC) and esophageal squamous cell carcinoma.
Sutro is responsible for manufacturing early clinical supply of M1231 and is eligible for milestone or contingent payments and tiered royalties.
Vaxcyte relationship: Potential vaccine application demonstrates the power of Sutro’s cell-free technology in conjugated vaccines.

Under a license from Sutro, Vaxcyte has the right to use the XpressCF and XpressCF+ platforms to discover and develop vaccine candidates for the treatment or prophylaxis of infectious diseases.
Vaxcyte is progressing their broader spectrum pneumococcal conjugate vaccine (VAX–24) through preclinical development.
Sutro is eligible to receive four percent (4%) royalties on worldwide net sales of any licensed vaccine candidates. Sutro retains the right to discover and develop vaccines for treatment or prophylaxis of any disease not caused by an infectious pathogen, including cancer.
In June 2020, Vaxcyte completed an initial public offering of its common stock. Sutro owns approximately 1.6 million shares of Vaxcyte common stock as of December 31, 2020.
Key 2020 financings

In May 2020 and December 2020, Sutro closed public offerings of its common stock, with gross proceeds of approximately $98.0 million and approximately $144.9 million, respectively.
Sutro ended 2020 with cash, cash equivalents & marketable securities of $326.5 million, with projected runway into the second half of 2023, based on current business plans and assumptions, and not including the value of its holdings of Vaxcyte common stock.
Full Year 2020 Financial Highlights

Cash, Cash Equivalents and Marketable Securities

As of December 31, 2020, Sutro had cash, cash equivalents and marketable securities of $326.5 million, as compared to $133.5 million as of December 31, 2019, which represents a net cash increase of $193.0 million during 2020. The cash, cash equivalents and marketable securities balance noted above does not include the value associated with Sutro’s holdings of approximately 1.6 million shares of Vaxcyte common stock. As of December 31, 2020, the fair value of the Vaxcyte common stock held by Sutro was $41.6 million.

Unrealized Gain from Increase in Value of Vaxcyte Common Stock

The non-operating, unrealized gain of $41.5 million in 2020 consisted of $41.6 million due to the increase in the estimated fair value of Sutro’s holdings of approximately 1.6 million shares of Vaxcyte common stock, partially offset by approximately $0.1 million in adjustments related to revaluations of certain Vaxcyte equity items. Vaxcyte common stock held by Sutro will be measured at fair value based on the closing price of Vaxcyte’s common stock on the last trading day of each reporting period, with any non-operating, unrealized gains and losses recorded in Sutro’s statements of operations.

Revenue

Revenue was $42.7 million in each of the year ended December 31, 2020 and the year ended December 31, 2019, related principally to the Merck, BMS, and EMD Serono collaborations. Future collaboration revenue from Merck, BMS, and EMD Serono, and from any future collaboration partners, will fluctuate as a result of the amount and timing of revenue recognition of upfront, milestones and other collaboration agreement payments.

Operating Expenses

Total operating expenses for the year ended December 31, 2020 were $113.8 million, as compared to $98.2 million in 2019, including non-cash stock-based compensation of $11.9 million and $10.3 million, and depreciation and amortization expense of $4.3 million and $4.8 million, in 2020 and 2019, respectively. Total operating expenses for 2020 were comprised of research and development expenses of $77.0 million and general and administrative expenses of $36.8 million, which are expected to increase in future periods as Sutro’s internal product candidates advance in clinical development and additional general and administrative expenses are incurred as a public company.

On March 18, 2021 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that the Annual Report for 2020 has been published (Press release, Alligator Bioscience, MAR 18, 2021, View Source [SID1234576884]). The Annual Report is attached as a PDF and is available on the company’s website, View Source

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The information was submitted for publication, through the agency of the contact person set out above, at 4:30 p.m. CET on March 18, 2021.

On March 18, 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 and full year ending December 31, 2020, and highlighted its key corporate objectives for 2021 and early 2022 (Press release, Verastem, MAR 18, 2021, View Source [SID1234576883]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are encouraged as we continue to gain greater understanding of the profile of our RAF/MEK inhibitor VS-6766 and its role in blocking multiple nodes in the RAS pathway without the resistance and tolerability issues that have historically been challenges. Based on its unique profile, we believe that VS-6766 could play a significant role in combination therapy across multiple tumor types and where patients need better options," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "We have a strong balance sheet to execute on our corporate objectives and report across multiple key catalysts. Our annual spend is projected to be approximately $50 million for 2021, and we still anticipate being comfortably funded until at least 2024."

In Q4 2020, the Company advanced VS-6766 into two Phase 2 registration-directed clinical studies: RAMP 201 in patients with recurrent low-grade serous ovarian cancer (LGSOC) and RAMP 202 in patients with KRAS-G12V mutant non-small cell lung cancer (NSCLC). Both are evaluating VS-6766 with the Company’s FAK inhibitor, defactinib. The combination of VS-6766 and defactinib had been found to be clinically active in patients with KRAS mutant tumors through an ongoing investigator-initiated Phase 1/2 FRAME study. The FRAME study is evaluating the combination in patients with recurrent LGSOC, KRAS mutant NSCLC, KRAS-G12V mutant NSCLC, pancreatic cancer and KRAS mutant endometrioid cancer.

"In the FRAME study, additional patients with recurrent LGSOC have now responded to treatment with clinically meaningful results for patients with KRAS wild-type tumors, which represents approximately 70% of all LGSOC patients. The results add to the robust and durable responses that have been demonstrated for patients with KRAS mutations," said Jonathan Pachter, Chief Scientific Officer of Verastem Oncology. "The majority of LGSOC cases are RAS pathway-driven, and these new data support the premise that VS-6766 could be a treatment for all LGSOC patients. Therefore, we are expanding the selection phase of our company-sponsored RAMP 201 study to now include both patient populations (KRAS mutant and KRAS wild-type) to determine the optimal go-forward regimen for both."

In an updated December 2020 read-out of the FRAME study LGSOC cohort (n=24), the overall response rate (ORR) is 52% (11 of 21 response evaluable patients), with KRAS mutant ORR at 70% (7 of 10 response evaluable patients), KRAS wild-type ORR at 44% (4 of 9 response evaluable patients) and KRAS status undetermined ORR at 0% (0 of 2 response evaluable patients). As reported previously, the most common side effects seen in the study were rash, creatine kinase elevation, nausea, hyperbilirubinemia and diarrhea, most being NCI CTC Grade 1/2 and all were reversible. The data from the LGSOC cohort are anticipated to be presented at a major medical meeting during the second half of 2021.

Recent Corporate Highlights

Phase 2 registration-directed (RAMP 201) study underway investigating VS-6766 alone and in combination with defactinib for the treatment of patients with recurrent LGSOC.

Supportive updated data from LGSOC cohort of the investigator-sponsored Phase 1/2 FRAME study continues to show encouraging clinical activity, durability and a favorable safety profile in both the overall patient population and in the subgroup of patients with KRAS mutant LGSOC, including patients who had previously progressed following treatment with a MEK inhibitor.

Ongoing data from the FRAME study are demonstrating clinically meaningful results in patients with KRAS wild-type LGSOC in addition to those with KRAS mutation as more patients have had an opportunity to respond to treatment.
Phase 2 registration-directed (RAMP 202) study underway investigating VS-6766 alone and in combination with defactinib for the treatment of patients with recurrent KRAS-G12V mutant NSCLC.
Closed COPIKTRA sale to Secura Bio, Inc. (Secura) in a deal valued at up to $311 million, plus royalties.
Ended 2020 with $147.2 million in cash, cash equivalents and investments.
Upcoming Milestones and Key Priorities for 2021-2022

LGSOC

Amend RAMP 201 protocol to add enrollment of patients with KRAS wild-type LGSOC in selection phase.
Report top-line results from the selection phase of RAMP 201 and commence expansion phase during the first half of 2022.
Report updated data from Phase 1/2 FRAME study LGSOC cohort, including the KRAS wild-type population, during the second half of 2021.
NSCLC

Report top-line results from the selection phase of RAMP 202 and commence expansion phase during the first half of 2022.
Updated data from the Phase 1/2 FRAME study NSCLC cohort will be presented by the investigator at the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The presentation will include an update on all variants of mutant KRAS, including further updates on the KRAS G12V NSCLC patients who had been previously highlighted. As no new KRAS G12V patients were enrolled in the original FRAME study, a KRAS G12V-specific NSCLC cohort of FRAME has been opened in addition to the Company-sponsored RAMP 202 registration-directed trial.
Corporate and Financial

Expect 2021 annual operating expenses of approximately $50 million.
Fourth Quarter 2020 Financial Results

Total revenue for the three months ended December 31, 2020 (2020 Quarter) was $0.5 million, compared to $3.6 million for the three months ended December 31, 2019 (2019 Quarter).

Total research and development (R&D) and selling, general and administrative (SG&A) expenses for the 2020 Quarter were $17.2 million, compared to $36.2 million for the 2019 Quarter.

R&D expense for the 2020 Quarter was $10.1 million, compared to $12.5 million for the 2019 Quarter. The decrease of $2.4 million, or 18.5%, was primarily related to a reduction in contract research organization costs partially offset by an increase in drug substance and drug product manufacturing costs.

SG&A expense for the 2020 Quarter was $7.1 million, compared to $23.7 million for the 2019 Quarter. The decrease of $16.6 million, or 70.1%, primarily resulted from the Company’s shift in strategic direction and COPIKTRA sale to Secura which led to lower employee related expenses and consulting and professional fees.

Net loss for the 2020 Quarter was $(19.9) million, or $(0.12) per share (basic and diluted), compared to $(38.8) million, or $(0.51) per share (basic and diluted), for the 2019 Quarter.

For the 2020 Quarter, non-GAAP adjusted net loss was $(14.8) million, or $(0.09) per share (diluted), compared to non-GAAP adjusted net loss of $(30.3) million, or $(0.40) per share (diluted), for the 2019 Quarter. Please refer to the GAAP to Non-GAAP Reconciliation attached to this press release.

Full-Year 2020 Financial Results

Verastem Oncology ended 2020 with cash, cash equivalents and investments of $147.2 million.

During the year ended December 31, 2020 (2020 Period), Verastem Oncology repaid in full all principal, accrued and unpaid interest, fees, and expenses under the Amended Loan Agreement with Hercules in an aggregate amount of $37.4 million and the Amended Loan Agreement was terminated along with Hercules’ commitment to provide funding under any future term loans. All liens on substantially all of the Company’s assets to secure the loans under the Amended Loan Agreement have been terminated and released.

Total revenue for the 2020 Period was $88.5 million, compared to $17.5 million for the year ended December 31, 2019 (2019 Period).

Sale of COPIKTRA license and related assets revenue for the 2020 Period was $70.0 million, compared to $0.0 million for the 2019 Period. The 2020 Period was comprised of a $70.0 million upfront payment received as part of the COPIKTRA sale to Secura.

Net product revenue for the 2020 Period was $15.2 million, compared to $12.3 million for the 2019 Period. License and collaboration revenue for the 2020 Period was $2.9 million, compared to $5.1 million for the 2019 Period.

Total R&D and SG&A expenses for the 2020 Period were $104.1 million, compared to $147.0 million for the 2019 Period.

R&D expense for the 2020 Period was $41.4 million, compared to $45.8 million for the 2019 Period. The decrease of $4.4 million, or 9.6%, was primarily related to a reduction in contract research organization costs and employee related expense, partially offset by the $3.0 million up-front non-refundable payment made to Chugai Pharmaceuticals, Co. Ltd. for the VS-6766 license in the first quarter of 2020.

SG&A expense for the 2020 Period was $62.7 million, compared to $101.2 million for the 2019 Period. The decrease of $38.5 million, or 38.0%, was primarily due to the Company’s shift in strategic direction and sale of COPIKTRA business which led to lower employee related expense and consulting and professional fees.

Net loss for the 2020 Period was $(67.7) million, or $(0.44) per share (basic and diluted), compared to $(149.2) million, or $(2.00) per share (basic and diluted), for the 2019 Period.

For the 2020 Period, non-GAAP adjusted net loss was $(37.8) million, or $(0.25) per share (diluted), compared to non-GAAP adjusted net loss of $(126.0) million, or $(1.69) per share (diluted), for the 2019 Period. 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 and year ended December 31, 2020 and 2019 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).