Verastem(US:VSTM)2024-11-06 21:22Clinical Trial Results - The combination of avutometinib and defactinib has shown a confirmed overall response rate (ORR) of 31% in evaluable patients with measurable disease in the RAMP 201 study[138]. - Among patients with KRAS mutant LGSOC, the confirmed ORR was 44%, while for KRAS wild-type patients, it was 17%[138]. - The median duration of response was 31.1 months for all evaluable patients, with 22 months for KRAS mutant and 9.2 months for KRAS wild-type populations[138]. - The median progression-free survival was 12.9 months overall, with 22 months for KRAS mutant and 12.8 months for KRAS wild-type populations[138]. - Initial results from the RAMP 203 study showed a confirmed ORR of 25% in patients treated with the combination of avutometinib and LUMAKRAS[144]. - The RAMP 205 trial, supported by a $3.8 million grant from PanCAN, reported an overall response rate (ORR) of 83% in the dose level 1 cohort[147]. - The RAMP 301 study is currently enrolling patients with recurrent LGSOC regardless of KRAS mutation status, aiming for full approval of the combination therapy[143]. - The RAMP 204 study was closed to further enrollment to prioritize the RAMP 203 study, which includes treatment-naïve patients[146]. - The company expects to report updated data from the RAMP 205 study in the first quarter of 2025[147]. Market Opportunity - The total annual incident addressable market opportunity for the combination is estimated at approximately $300 million for KRAS mutant and $374 million for KRAS wild-type populations[140]. - The total prevalent addressable market opportunity is estimated at approximately $1.7 billion for KRAS mutant and $1.6 billion for KRAS wild-type populations[140]. Financial Performance - The company reported a net loss of $24.0 million for the three months ended September 30, 2024, compared to a net loss of $20.0 million for the same period in 2023, representing a 20% increase in losses[152]. - Total operating expenses for Q3 2024 were $37.0 million, a 74% increase from $21.3 million in Q3 2023[157]. - Total revenue for the nine months ended September 30, 2024, was $10.0 million, a 100% increase compared to $0.0 million for the same period in 2023[170]. - Research and development expenses increased by 77% to $24.8 million in Q3 2024 from $13.9 million in Q3 2023, driven by higher costs in contract research organizations, consulting, and investigator fees[157]. - Research and development expense for the 2024 Period was $60.5 million, representing a 56% increase from $38.9 million in the 2023 Period[171]. - Selling, general and administrative expense for the 2024 Period was $32.8 million, a 49% increase from $22.1 million in the 2023 Period[174]. - Interest income decreased to $3.2 million in the 2024 Period from $4.3 million in the 2023 Period, a decline of 27%[176]. - Interest expense increased to $3.4 million in the 2024 Period from $3.0 million in the 2023 Period, a rise of 13%[177]. - The change in fair value of preferred stock tranche liability resulted in $4.2 million income for the 2024 Period compared to $0.3 million expense in the 2023 Period[178]. - The change in fair value of warrant liability was $13.5 million for the 2024 Period, with no warrant liability outstanding during the 2023 Period[179]. Cash Flow and Financing - As of September 30, 2024, the company had $113.2 million in cash, cash equivalents, and investments[182]. - For the nine months ended September 30, 2024, the company reported a net cash used in operating activities of $79.7 million, compared to $56.8 million for the same period in 2023[184]. - Cash provided by investing activities for the 2024 Period was $60.0 million, primarily from maturities of investments[185]. - The company raised $53.8 million in net proceeds from the issuance of common stock and warrants in July 2024[186]. - The company has borrowed $40.0 million under the Loan Agreement, with interest rates subject to fluctuations based on market conditions[191]. - The cash outflow from net losses adjusted for non-cash charges was $78.7 million for the 2024 Period, compared to $55.8 million for the 2023 Period[184]. - The cash outflow due to changes in operating assets and liabilities for the 2024 Period was primarily driven by a decrease of $2.7 million in accounts payable[184]. - The company expects to finance a portion of its business through future potential milestones and royalties from the Secura APA[181]. Collaboration and Development - The company entered into a collaboration agreement with GenFleet for exclusive rights to develop oncology programs targeting RAS pathway-driven cancers, with a lead program VS-7375/GFH375 selected in December 2023[149]. - The Phase 1 study for VS-7375/GFH375 in China began in July 2024, evaluating safety and efficacy in patients with KRAS G12D mutations[150]. - The company anticipates filing an IND for VS-7375/GFH375 in the United States by the first quarter of 2025[150]. Going Concern - The company had an accumulated deficit of $891.0 million as of September 30, 2024, raising substantial doubt about its ability to continue as a going concern[152]. - The company is exposed to market risk related to changes in interest rates, with an immediate 100 basis point change not expected to materially affect the fair market value of its investment portfolio[189]. - The company has not reported any material changes in its contractual obligations and commitments since the last annual report[188].