Core Viewpoint - Verastem, Inc. is a clinical-stage biopharmaceutical company focused on developing cancer treatments targeting the RAS/MAPK pathway, with a promising combination therapy of Avutometinib and Defactinib for recurrent low-grade serous ovarian cancer (LGSOC) expected to receive FDA approval by 2H2024 [1][12][30] Company Overview - Founded in 2010 and headquartered in Needham, Massachusetts, Verastem develops oncology treatments using small-molecule inhibitors targeting specific cancer pathways, particularly the RAS/MAPK pathway [2] - The company’s leading therapy aims to suppress uncontrolled cancerous cell proliferation associated with mutations in the RAS/MAPK pathway, which are involved in several cancer types [2] Clinical Program - The combination of Avutometinib and Defactinib is currently in a rolling NDA submission process, with expectations for completion by 2H2024 [1][4] - The clinical pipeline includes multiple trials for various indications, including recurrent LGSOC, metastatic pancreatic cancer, and mKRAS G12C non-small cell lung cancer (NSCLC) [3][6][10] Pipeline and Collaborations - The RAMP 301 trial is focused on recurrent LGSOC, while RAMP 203 and RAMP 204 are collaborations with Amgen and Mirati Therapeutics, respectively, targeting mKRAS G12C NSCLC [7][10] - The company has received Fast Track Designation from the FDA for the combination therapy of Avutometinib and Sotorasib for mKRAS G12C NSCLC [9] Market Opportunity - The total addressable market for LGSOC is substantial, with an estimated annual opportunity of $570 million and a prevalent market potential of $1.7 billion for KRAS mutant patients [12][26] - The combination therapy has shown promising early results, with an overall response rate of 45% and 60% for KRAS-mutated patients, significantly higher than current treatment options [13][26] Financial Position - As of June 30, 2024, Verastem reported cash, cash equivalents, and investments totaling $144.5 million, providing a cash runway through the anticipated FDA approval decision by 1H2025 [21][22] - The company’s market cap is approximately $98.2 million, indicating it trades below its cash value, which may present a speculative investment opportunity [20][22] Conclusion - Verastem's combination therapy of Avutometinib and Defactinib is positioned as a potentially groundbreaking treatment for recurrent LGSOC, with a viable path to regulatory approval and significant market potential [30] - The company is viewed as a speculative buy, given its promising clinical data and financial resources to support ongoing development [29][30]

Clinical Development and Market Opportunity - The combination of avutometinib and defactinib has shown a confirmed objective response rate of 27% in all patients with recurrent LGSOC, with rates of 37% in KRAS mutant and 15% in KRAS wild-type patients [112]. - The total annual incident addressable market opportunity for the combination is estimated to be approximately $300 million for KRAS mutant and $270 million for KRAS wild-type populations [114]. - The total prevalent addressable market opportunity is estimated to be approximately $1.7 billion for KRAS mutant and $1.1 billion for KRAS wild-type populations [114]. - The estimated median duration of therapy for KRAS mutant patients is 14 months, while for KRAS wild-type patients it is 7 months [117]. - The FDA granted breakthrough designation for the combination of avutometinib and defactinib for the treatment of all patients with recurrent LGSOC [110]. - The RAMP 301 study has been initiated to evaluate the combination of avutometinib and defactinib against standard chemotherapy or hormonal therapy in recurrent LGSOC patients [119]. - The RAMP 203 study has shown a confirmed overall response rate of 25% in patients with KRAS G12C NSCLC [120]. - The company plans to seek accelerated approval from the FDA for the combination of avutometinib and defactinib for patients with recurrent KRAS mutant LGSOC [113]. - The combination of avutometinib and defactinib has received orphan drug designation for the treatment of LGSOC in the United States [110]. - The company received a Therapeutic Accelerator Award from PanCAN for up to $3.8 million to support a Phase 1b/2 clinical trial of avutometinib in combination with defactinib [122]. - The RAMP 205 trial reported a confirmed overall response rate (ORR) of 83% (5/6) for patients receiving the combination of avutometinib and defactinib with gemcitabine and Nab-paclitaxel [123]. - The Phase 1 study for GFH375/VS-7375 is being conducted in approximately 20 hospitals in China, evaluating safety and efficacy in patients with advanced KRAS G12D mutant solid tumors [125]. - The collaboration with GenFleet includes options for exclusive development rights for up to three oncology programs targeting RAS pathway-driven cancers [125]. Financial Performance - Total revenue for the three months ended June 30, 2024, was $10.0 million, a 100% increase compared to $0.0 million for the same period in 2023 [132]. - Research and development expenses increased by 40% to $18.1 million for the 2024 Quarter, up from $12.9 million in the 2023 Quarter [133]. - The net loss for the three months ended June 30, 2024, was $8.3 million, a 66% improvement compared to a net loss of $24.3 million for the same period in 2023 [132]. - The company had an accumulated deficit of $867.0 million as of June 30, 2024 [127]. - The company received net proceeds of approximately $51.1 million from the sale of 13,333,334 shares of common stock in July 2024 [128]. - Total revenue for the six months ended June 30, 2024, was $10.0 million, a 100% increase compared to $0.0 million for the same period in 2023 [143]. - Research and development expenses for the 2024 Period were $35.8 million, up from $24.9 million in the 2023 Period, representing a 44% increase [144]. - Selling, general and administrative expenses increased to $20.6 million in the 2024 Period from $14.7 million in the 2023 Period, a rise of 40% [147]. - Interest income for the 2024 Period was $2.4 million, compared to $2.1 million in the 2023 Period, marking a 12% increase [148]. - Interest expense rose to $2.3 million in the 2024 Period from $1.9 million in the 2023 Period, reflecting a 20% increase [149]. - The change in fair value of preferred stock tranche liability resulted in an income of $4.2 million for the 2024 Period, compared to an expense of $0.5 million in the 2023 Period [150]. - Cash outflow from operating activities was $55.9 million for the 2024 Period, compared to $40.2 million for the 2023 Period [154]. - As of June 30, 2024, the company had $83.4 million in cash, cash equivalents, and investments [152]. - The company received a $10.0 million milestone payment in July 2024 due to Secura achieving cumulative worldwide net sales of COPIKTRA exceeding $100.0 million [143]. - 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 [150]. - Net cash used in operating activities for six months ended June 30, 2024, was $(55,877) thousand, compared to $(40,190) thousand for the same period in 2023, indicating a decline in operational cash flow [157]. - Cash, cash equivalents, and investments totaled $83.4 million as of June 30, 2024, primarily consisting of interest-bearing assets [159]. - The company has borrowed $40.0 million under a Loan Agreement, with interest rates subject to fluctuations based on the one-month CME Secured Overnight Financing Rate plus a margin [161]. - The increase in cash, cash equivalents, and restricted cash for the six months ended June 30, 2024, was $4,536 thousand, a significant decrease from $108,537 thousand in the prior year [157]. - Financing activities resulted in cash inflow of $441 thousand for the six months ended June 30, 2024, a significant decrease from $135,531 thousand in the prior year, indicating reduced financing activities [157]. Risk Factors - The company anticipates continued operating losses due to ongoing research and development costs and lack of regulatory approval for product candidates [129]. - The company is exposed to interest rate risk, but a 100 basis point change in interest rates would not materially affect the fair market value of its investment portfolio due to its short-term duration [159]. - As of June 30, 2024, an immaterial amount of total liabilities were denominated in foreign currencies, indicating limited exposure to foreign currency fluctuations [160]. - The company’s available-for-sale securities are subject to interest rate risk, which could lead to a decline in value if market interest rates increase [159]. - A 10% increase in current interest rates would result in an immaterial increase in cash interest expense due to the overall interest rate floor and cap in the Loan Agreement [161].

Verastem Oncology (VSTM) announced that it has dosed the first patient in a phase I/II study evaluating its KRAS G12D inhibitor, GFH375/VS-7375, for treating KRAS G12D mutant solid tumors. Verastem's collaboration partner GenFleet Therapeutics is conducting the study in China. We note that after evaluating the initial dose escalation data from the above mentioned phase I study in China, VSTM plans to expedite the development path ahead for GFH375/VS-7375 in the United States. In May 2024, VSTM initiated the ...

LOS ANGELES, July 10, 2024 (GLOBE NEWSWIRE) -- The Portnoy Law Firm advises Verastem, Inc. ("Verastem" or the "Company") (NASDAQ: VSTM) investors that the firm has initiated an investigation into possible securities fraud and may file a class action on behalf of investors. Verastem investors that lost money on their investment are encouraged to contact Lesley Portnoy, Esq. Please visit our website to review more information and submit your transaction information. Investors can contact the law firm at no co ...

Group 1 - Pomerantz LLP is investigating claims on behalf of investors of Verastem, Inc. regarding potential securities fraud or unlawful business practices by the company and its officers or directors [1] - Following the news of the investigation, Verastem's stock price dropped by $8.06 per share, representing a decline of 66.17%, closing at $4.12 per share on May 24, 2024 [3] - On May 23, 2024, Verastem announced interim safety and efficacy results from the RAMP 205 Phase 1/2 clinical trial, revealing that 12 patients experienced 19 treatment-emergent serious adverse events [5] Group 2 - Pomerantz LLP is recognized as a leading firm in corporate, securities, and antitrust class litigation, with a history of recovering multimillion-dollar damages for victims of securities fraud and corporate misconduct [4]

On this news, Verastem's stock price fell $8.06 per share, or 66.17%, to close at $4.12 per share on May 24, 2024. Attorney advertising. Prior results do not guarantee similar outcomes. [Click here for information about joining the class action] Pomerantz LLP, with offices in New York, Chicago, Los Angeles, London, Paris, and Tel Aviv, is acknowledged as one of the premier firms in the areas of corporate, securities, and antitrust class litigation. Founded by the late Abraham L. Pomerantz, known as the dean ...

NEW YORK, June 30, 2024 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Verastem, Inc. ("Verastem" or the "Company") (NASDAQ: VSTM). Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980. On May 23, 2024, Verastem issued a press release "announc[ing] the initial interim safety and efficacy results from the ongoing RAMP 205 Phase 1/2 clinical trial evaluating avutometinib plus defactinib in combination with gemcitabine and ...

For the transition period from to Commission file number: 001-35403 Table of Contents Verastem, Inc. UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 For the quarterly period ended March 31, 2024 FORM 10-Q OR (Mark One) ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 02494 (Zip Code) (781) 292-4200 (Registrant's telephone number, including area code) Sec ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-35403 Verastem, Inc. (Exact name of registrant as specified in its charter) Delaware (State or other jurisdiction o ...

Financial Performance - The company has an accumulated deficit of $797.5 million as of September 30, 2023[151]. - The net loss for the three months ended September 30, 2023, was $20.0 million, compared to $60.0 million for the same period in 2022[151]. - Total revenue for the nine months ended September 30, 2023 was $0.0 million, a decrease of 100% from $2.6 million in the same period of 2022[168]. - Net loss for Q3 2023 was $20.0 million, an increase of 11% compared to a net loss of $18.1 million in Q3 2022[157]. - Research and development expenses for Q3 2023 were $13.9 million, an increase of 24% from $11.3 million in Q3 2022[157]. - Total operating expenses for Q3 2023 were $21.3 million, up 20% from $17.7 million in Q3 2022[157]. - Research and development expenses for the nine months ended September 30, 2023 were $38.9 million, a slight decrease of 2% from $39.8 million in the same period of 2022[169]. - Selling, general and administrative expenses for the nine months ended September 30, 2023 were $22.1 million, an increase of 17% from $18.9 million in the same period of 2022[168]. - Selling, general and administrative expenses increased to $22.1 million in 2023 from $18.9 million in 2022, a rise of 16.9%[174]. - Interest income for Q3 2023 was $2.2 million, a significant increase of 611% from $0.3 million in Q3 2022[165]. - Interest expense for Q3 2023 was $1.1 million, up 57% from $0.7 million in Q3 2022[166]. - Interest income surged to $4.3 million in 2023, up from $0.4 million in 2022, reflecting a significant increase of 975% driven by higher interest rates and investment balances[176]. - Interest expense rose to $3.0 million in 2023 compared to $1.4 million in 2022, marking an increase of 114.3% primarily due to a loan agreement with Oxford[177]. - Net cash used in operating activities was $56.8 million for the 2023 period, compared to $47.1 million in 2022, indicating a 20.4% increase in cash outflow[182]. - Cash provided by financing activities in 2023 was $134.6 million, significantly higher than $51.8 million in 2022, reflecting a 159.5% increase[184]. - The company expects existing cash resources to fund planned operations for at least 12 months from the date of the financial statements[151]. - The company is exposed to interest rate risk, with a total of $165.7 million in cash and investments as of September 30, 2023, which could be affected by changes in U.S. interest rates[191]. - As of September 30, 2023, the company had $165.7 million in cash, cash equivalents, and investments[181]. Clinical Trials and Research - The combination of avutometinib and defactinib demonstrated an overall objective response rate (ORR) of 45% in patients with recurrent low-grade serous ovarian cancer (LGSOC) in the RAMP 201 trial[142]. - Among patients with KRAS mutant LGSOC, the ORR was 60%, while it was 29% for KRAS wild-type LGSOC[143]. - The FRAME study showed an ORR of 42% in evaluable patients with LGSOC, with a median duration of response of 26.9 months[144]. - The company plans to initiate a confirmatory Phase 3 trial (RAMP 301) to evaluate the efficacy and safety of the combination therapy in recurrent LGSOC in the second half of 2023[145]. - The RAMP 203 trial, evaluating the combination of avutometinib with Amgen's KRAS G12C inhibitor, has shown a confirmed ORR of 25% across efficacy-evaluable patients[146]. - The company received a grant of up to $3.8 million from the Pancreatic Cancer Network to support the RAMP 205 trial, which will evaluate the combination of avutometinib and defactinib in pancreatic cancer[148]. Strategic Partnerships - The company entered into a collaboration agreement with GenFleet Therapeutics, involving an upfront payment of $2.0 million and potential milestone payments of up to $622.0 million[186][187]. - The company expects to finance operations through future potential milestones and royalties from the Secura APA[180]. - The company may terminate the GenFleet Agreement with 90 days written notice, allowing flexibility in its strategic partnerships[188]. - The company made a $2.0 million upfront payment in September 2023 under the GenFleet Agreement, contributing to increased R&D costs[162].