Verastem FY Conference Summary Company Overview - **Company**: Verastem (NasdaqCM: VSTM) - **Industry**: Biotechnology, specifically focused on oncology treatments Key Points Commercial Launch in LGSC - Verastem reported strong sales in Q3, indicating good commercial momentum in the launch of their product for Low-Grade Serous Carcinoma (LGSC) [3][4] - The sales force is focused on identifying patients and working with clinical sites to optimize treatment [4][5] - The company aims to keep patients on treatment for an extended duration, with an average treatment duration of 18 months observed in clinical trials [5][6] - Initial patient distribution shows over half coming from large academic centers, with a notable amount from community settings [7][9] Patient Identification and Treatment Strategy - The launch strategy targets 100 institutions where the majority of patients are concentrated [9][10] - Verastem has established partnerships with specialty pharmacies and group purchasing organizations to enhance distribution [10][11] - The company is not planning to expand its sales force, believing current resources are sufficient [11] Off-Label Use and Clinical Data - Majority of prescriptions are for KRAS mutant patients, but some off-label use for KRAS wild-type patients is occurring without reimbursement issues [12][13] - Recent studies indicate a survival advantage for using MEK inhibitors, reinforcing the treatment's efficacy [13][14] NCCN Guidelines and Revenue Guidance - Verastem submitted for NCCN guideline inclusion, with updates expected after the new year [15][16] - The company is cautious about providing revenue guidance, preferring to wait until they have more predictable data [18][19] RAMP 301 Study Adjustments - The RAMP 301 study has seen a slight adjustment in patient enrollment due to a faster-than-expected accrual rate [20][21] - The study's interim analysis suggested adding a small number of patients to ensure robust data collection [22][23] - The final accrual is projected to finish in Q1, with top-line data expected in the second half of 2027 [28][29] Total Addressable Market (TAM) - The total addressable market for LGSC is estimated at $4 billion, with projected peak sales ranging from $200 million to over $600 million [30][31] Pipeline Drug: VS-7375 (KRAS G12D Inhibitor) - VS-7375 is positioned as a best-in-class dual inhibitor, with promising phase I data showing a 69% response rate in lung cancer and 58% in second-line pancreatic cancer [33][41] - The company is focusing on improving tolerability in its U.S. trials by using fed patients and prophylactic antiemetics [42][44] - Ongoing combination studies with other therapies are being explored to enhance treatment efficacy [49][50] Collaboration with GenFleet - Verastem is leveraging data from GenFleet, which holds rights in China, to expedite its own clinical programs [51][52] - Regular collaboration and data sharing are expected to enhance the development of both companies' pipelines [52][53] Additional Insights - The company is optimistic about the potential for accelerated approval paths based on the strong efficacy data observed [41] - There is a growing awareness of LGSC, which may lead to increased patient identification and treatment opportunities [31]

Core Insights - Verastem Oncology is focused on developing new medicines for patients with RAS/MAPK pathway-driven cancers [1] Conference Participation - The management team of Verastem Oncology is scheduled to present at the Guggenheim Healthcare Conference on November 11 at 1:00 pm ET [1] - Additionally, the company will participate in the Jefferies Global Healthcare Conference in London on November 19 at 2:30 pm [1]

FDA Approvals and Clinical Trials - The FDA approved AVMAPKI FAKZYNJA CO-PACK for treating adult patients with KRAS mutant recurrent LGSOC on May 8, 2025, under the accelerated approval pathway [170]. - The RAMP 301 trial, a confirmatory Phase 3 study for the combination of avutometinib and defactinib, completed enrollment of 270 patients a quarter early in September 2025, with an additional 29 patients recommended for enrollment [174]. - In the RAMP 205 trial, 83% of patients (10 out of 12) achieved partial responses at dose level 1, leading to an expansion of enrollment from 12 to 29 patients [180]. - The RAMP 203 study is evaluating the combination of avutometinib and Amgen's LUMAKRAS in patients with KRAS G12C NSCLC, with the trial progressing to the recommended Phase 2 dose [177]. - The RAMP 201J trial in Japan began dosing patients in October 2024, with preliminary results showing no dose-limiting toxicities observed [176]. - GenFleet initiated a Phase 1b/2 study of GFH375 combined with cetuximab or chemotherapy for advanced solid tumors on October 22, 2025 [189]. Financial Performance - As of September 30, 2025, the company reported a net loss of $98.5 million, a significant increase of 311% compared to the same period in 2024 [193]. - The company recorded approximately $11.2 million in net product revenue for the three months ended September 30, 2025, following the commercial launch of AVMAPKI FAKZYNJA CO-PACK [199]. - Total revenue for the nine months ended September 30, 2025, was $13.38 million, a 34% increase compared to $10 million in the 2024 Period [216]. - The company reported a net loss of $176.56 million for the nine months ended September 30, 2025, compared to a net loss of $66.09 million in the 2024 Period, representing a 167% increase [216]. - The total operating expenses for the nine months ended September 30, 2025, were $142.03 million, a 52% increase from $93.37 million in the 2024 Period [216]. Research and Development - Research and development expenses increased by 17% to $28.9 million in the 2025 Quarter, driven by higher manufacturing and contract research organization costs [204]. - Research and development expenses for the nine months ended September 30, 2025, were $82.93 million, a 37% increase from $60.52 million in the 2024 Period [220]. - The increase in R&D expenses was primarily driven by a $1.61 million rise in costs for the Avutometinib + Defactinib - LGSOC project due to advancing trial costs [208]. - The combination of avutometinib and defactinib is being investigated in various solid tumors, including colorectal cancer and breast cancer, through investigator-sponsored trials [182]. Collaborations and Partnerships - The collaboration with GenFleet Therapeutics includes options for three oncology programs targeting RAS pathway-driven cancers, with the lead program being the oral KRAS G12D inhibitor VS-7375 [183]. - The FDA cleared the IND application for VS-7375 in the U.S. in April 2025, with the first patient dosed in the Phase 1/2a clinical trial in June 2025 [185]. - GenFleet reported an objective response rate (ORR) of 52% and a disease control rate (DCR) of 100% for pancreatic ductal adenocarcinoma (PDAC) patients at a dosage of 400 or 600 mg QD [186]. - At the recommended phase 2 dose of 600 mg QD, the ORR was 68.8% and the DCR was 93.8% for patients in the study [187]. - In the dose expansion phase, GFH375 achieved a 41% ORR and a 96.7% DCR for heavily pre-treated PDAC patients [188]. Cash Flow and Financing - Cash used in operating activities was $107.6 million for 2025, compared to $79.7 million for 2024, reflecting net losses adjusted for non-cash charges [236]. - Cash provided by financing activities for 2025 was $164.7 million, significantly higher than $54.0 million in 2024, driven by proceeds from common stock issuance and the Note Purchase Agreement [237]. - The company expects to finance operations through future potential milestones and royalties from collaboration agreements [232]. - As of September 30, 2025, the company had $137.7 million in cash, cash equivalents, and investments [233]. Expenses and Liabilities - Selling, general and administrative expenses for the 2025 Quarter were $21.0 million, up $8.7 million (70.7%) from $12.3 million in the 2024 Quarter [209]. - Selling, general and administrative expenses increased to $56.7 million in 2025 from $32.8 million in 2024, primarily due to a $19.9 million rise in consulting and professional fees [224]. - Interest income increased to $1.2 million in the 2025 Quarter, compared to $0.8 million in the 2024 Quarter, driven by higher cash equivalents [211]. - Interest income decreased to $3.0 million in 2025 from $3.2 million in 2024, attributed to lower investment balances and interest rates [226]. - Interest expense significantly dropped to $0.7 million in 2025 from $3.4 million in 2024, mainly due to the termination of the Loan Agreement with Oxford [227]. - The change in fair value of warrant liability resulted in a $55.9 million expense for the 2025 Quarter, compared to a $13.5 million income in the 2024 Quarter [213]. - The change in fair value of warrant liability resulted in a $38.0 million expense for 2025, compared to a $13.5 million income in 2024, driven by an increase in stock price [230]. - The loss on debt extinguishment for 2025 was $1.8 million, resulting from the early repayment of a Loan Agreement totaling $42.7 million [228]. Market Risks - The company is exposed to market risks related to interest rates, with a potential immaterial effect on the fair market value of its investment portfolio from a 100 basis point change in interest rates [239].

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Core Viewpoint - Verastem reported a quarterly loss of $1.35 per share, significantly worse than the Zacks Consensus Estimate of a loss of $0.51, marking an earnings surprise of -164.71% [1] - The company achieved revenues of $11.24 million for the quarter ended September 2025, exceeding the Zacks Consensus Estimate by 82.80%, compared to zero revenues a year ago [2] Financial Performance - The loss per share increased from $0.60 a year ago to $1.35 this quarter, indicating a worsening financial situation [1] - Over the last four quarters, Verastem has only surpassed consensus EPS estimates once [2] Stock Performance - Verastem shares have increased approximately 91.5% since the beginning of the year, outperforming the S&P 500's gain of 16.5% [3] - The stock currently holds a Zacks Rank 2 (Buy), suggesting it is expected to outperform the market in the near future [6] Future Outlook - The current consensus EPS estimate for the upcoming quarter is -$0.42 on revenues of $22.07 million, and for the current fiscal year, it is -$2.44 on revenues of $18.75 million [7] - The outlook for the industry, specifically the Medical - Biomedical and Genetics sector, is favorable, ranking in the top 39% of over 250 Zacks industries [8]

Financial Data and Key Metrics Changes - The company reported a net product revenue of $11.2 million for Q3 2025, surpassing expectations and reflecting strong adoption among oncologists [5][19] - Research and development expenses were $29.0 million, driven by ongoing clinical trials [20] - The non-GAAP adjusted net loss was $39.4 million, or $0.54 per share diluted, compared to a loss of $35.3 million, or $0.88 per share diluted, in Q3 2024 [20][21] Business Line Data and Key Metrics Changes - The Avmasky Flexentia CoPak launch has shown strong growth, with 133 prescribers and approximately 65% of prescriptions coming from the top 100 healthcare organizations [9][15] - The product is being utilized by both gynecological oncologists (60%) and medical oncologists (40%) [11] Market Data and Key Metrics Changes - The payer coverage for Avmasky Flexentia CoPak has exceeded 80%, with a mix of half commercial and half Medicare [16] - The time to fill prescriptions has been approximately 12-14 days, indicating efficient access for patients [16] Company Strategy and Development Direction - The company aims to continue building on the momentum of the Avmasky Flexentia CoPak launch while focusing on strategic imperatives to ensure patient access [12][22] - The clinical pipeline is advancing, with several important data readouts expected in the first half of 2026 [22][23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory and value creation opportunities ahead, emphasizing the high unmet need in the KRAS mutated LGSOC market [22][24] - The company plans to engage with the FDA in the first half of 2026 to discuss the path forward for the VS7375 program [24] Other Important Information - The company ended Q3 2025 with cash, cash equivalents, and investments of $137.7 million, providing sufficient capital for ongoing commercial efforts [21] Q&A Session Summary Question: Usage of Avmasky Flexentia CoPak in the market - Management noted a mix of patients with prior lines of therapy, including both KRAS mutant and wild-type patients, but exact numbers are not available [26][27] Question: RAMP 301 study update - The IDMC recommended adding approximately 30 patients due to fewer events than anticipated, indicating a need for more data [29][50] Question: NCCN committee review outcome - The company has not received feedback on the recommendation for label expansion to include KRAS wild-type patients, with timelines for feedback being uncertain [33][34] Question: New patient starts versus refills - Management indicated significant new prescriptions are being generated, but detailed metrics on new versus refill prescriptions are not yet available [35] Question: Patient retention and dropout rates - It is too early to determine patient retention rates, but the average duration of treatment in clinical trials was around 18 months [40][42] Question: Differences in VS7375 treatment protocols - The company is using fed protocols and prophylactic antiemetics in the US, which differ from the protocols used in China [43] Question: Enrollment plan for KRAS mutant and wild-type populations - The total enrollment for the RAMP 301 study was planned for 270, with a balance between KRAS mutant and wild-type patients [45]

Financial Performance - Achieved net product revenue of $11.2 million for AVMAPKI™ FAKZYNJA™ CO-PACK in Q3 2025, marking the first full quarter since its launch[1][4][15] - Total revenue for the three months ended September 30, 2025, was $11,242,000, compared to $0 for the same period in 2024[39] - Net loss for Q3 2025 was $98.5 million, or $1.35 per share, compared to a net loss of $24.0 million, or $0.60 per share, in Q3 2024[1][19] - The net loss for the three months ended September 30, 2025, was $98,518,000, compared to a net loss of $23,967,000 for the same period in 2024[39] - Adjusted net loss (non-GAAP) for Q3 2025 was $39.354 million, compared to $35.278 million in Q3 2024, indicating a slight increase in adjusted losses[43] - Net loss per share (diluted, GAAP) for Q3 2025 was $(1.35), compared to $(0.60) in Q3 2024, reflecting a worsening loss per share[43] - For the nine months ended September 30, 2025, the net loss was $176.555 million, compared to $66.086 million for the same period in 2024[43] - The total adjusted net loss for the nine months ended September 30, 2025, was $123.765 million, compared to $78.002 million for the same period in 2024[43] Cash and Expenses - Ended Q3 2025 with cash and cash equivalents of $137.7 million, with an expected cash runway extending into the second half of 2026[1][20] - Research and development expenses increased by $4.2 million, or 16.9%, to $29.0 million in Q3 2025, primarily due to higher drug substance and trial costs[1][17] - Selling, general, and administrative expenses rose by $8.7 million, or 70.7%, to $21.0 million in Q3 2025, driven by commercialization costs for AVMAPKI FAKZYNJA[1][18] - Total operating expenses for the nine months ended September 30, 2025, were $142,033,000, compared to $93,366,000 for the same period in 2024[39] - Stock-based compensation expense for Q3 2025 was $2.178 million, up from $1.935 million in Q3 2024[43] Clinical Trials and Research - Preliminary safety and efficacy data for VS-7375 showed no dose-limiting toxicities in the ongoing Phase 1/2a trial, with four out of five patients experiencing tumor reduction[1][8] - Enrollment for the combination cohort of VS-7375 with cetuximab in advanced solid tumors has been initiated[1][8] - The company expects to report updates on the safety and efficacy of the RAMP 205 expansion cohort in 1H 2026[1][11] - The ongoing Phase 3 RAMP 301 trial completed enrollment of 270 patients a quarter early, with an additional 29 patients to be added based on IDMC recommendations[1][8] - The company is conducting RAMP 301, an international Phase 3 confirmatory trial for the combination of avutometinib and defactinib[24] - Verastem initiated a Phase 1/2a clinical trial for VS-7375, a KRAS G12D dual ON/OFF inhibitor, in June 2025[32] Regulatory and Collaborations - AVMAPKI™ FAKZYNJA™ CO-PACK received FDA approval for treating adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) on May 8, 2025[24] - The company plans to engage with the FDA regarding potential registration-directed clinical trials in PDAC and NSCLC in 1H 2026[1][12] - The collaboration with GenFleet Therapeutics aims to advance three oncology discovery programs related to RAS/MAPK pathway-driven cancers[33]

Core Insights - Verastem, Inc. (NASDAQ: VSTM) reported positive preliminary results from a phase 1/2a dose-escalation trial of VS-7375, an oral KRAS G12D inhibitor, in patients with KRAS G12D-mutant solid tumors [1][2] Group 1: Trial Results - The preliminary safety and tolerability data indicate that VS-7375 can be administered at efficacious doses while managing gastrointestinal side effects [2] - Anti-tumor activity has been observed among pre-treated patients with advanced pancreatic cancer and other solid tumors [3] - The company has initiated patient enrollment for the first dose-escalation combination cohort and plans to evaluate VS-7375 in combination with cetuximab [3][4] Group 2: Future Plans - Subject to the outcome of the Phase 1 dose escalation, Verastem intends to initiate a combination expansion cohort in colorectal cancer [4] - The company plans to select the recommended Phase 2 dose and advance subsequent efficacy and safety analysis in patients with advanced pancreatic ductal adenocarcinoma (PDAC) and non-small cell lung cancer (NSCLC) [4] Group 3: Company Overview - Verastem, Inc. is a biopharmaceutical company focused on developing and commercializing new medicines for cancers driven by the RAS/MAPK signaling pathway [5] - The company develops novel small-molecule drugs, including inhibitors targeting RAF/MEK, FAK, and KRAS G12D [5]

Industry Overview - The global cancer treatment market is undergoing a transformative phase due to rising cancer incidence and rapid scientific advancements, with the U.S. projected to report 2.04 million new cancer cases and over 618,000 deaths in 2025 [1] - An aging population and lifestyle-related risks are increasing global cancer prevalence, leading to higher oncology spending by healthcare systems [1] Innovation in Cancer Treatment - Advances in immunotherapies, targeted drugs, and personalized vaccines are expanding treatment options beyond traditional chemotherapy and radiation [2] - Immune-based approaches, such as checkpoint inhibitors and CAR-T therapies, enhance the body's defenses against tumors, while targeted therapies focus on specific genetic mutations to improve precision and reduce side effects [2] - Emerging technologies like genomic sequencing, AI, and machine learning are accelerating biomarker discovery and improving early detection, contributing to better survival rates and quality of life across many cancer types [3] Pharmaceutical Investment - Major pharmaceutical companies, including Novartis, AstraZeneca, Johnson & Johnson, Pfizer, AbbVie, Merck, Bristol Myers Squibb, and Eli Lilly, are aggressively investing in new oncology pipelines, including antibody-drug conjugates and next-generation immuno-oncology agents [4] - Smaller biotech firms are key sources of innovation, driving collaborations, licensing deals, and acquisitions as larger companies seek novel assets [4] Investment Opportunities - The oncology segment is positioned as one of the most attractive and resilient areas for long-term investors due to continued innovation and favorable reimbursement trends [5] - Stocks such as Relay Therapeutics, Fate Therapeutics, and Verastem Oncology are highlighted as potential investment opportunities in the rising cancer treatment trend [6] Company Highlights - **Relay Therapeutics** is developing RLY-2608, a mutant-selective PI3Kα inhibitor, showing potential benefits in clinical studies for advanced breast cancer [7][8] - **Fate Therapeutics** focuses on universal, off-the-shelf cell products and is co-developing FT825/ONO-8250, a CAR T-cell product candidate, with promising safety profiles in early studies [9][10][11] - **Verastem Oncology** received FDA approval for its combination regimen of avutometinib and defactinib for treating KRAS mutant recurrent low-grade serous ovarian cancer, generating $2.1 million in sales shortly after launch [12][13]