On August 7, 2025 K36 Therapeutics, Inc. ("K36"), a privately held clinical-stage biotechnology company developing novel, targeted therapies for cancers with unmet medical need, reported the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) Application for KTX-2001, a selective, oral inhibitor of NSD2 (nuclear receptor-binding SET domain protein 2), a histone methyltransferase and oncogene that activates gene expression in some cancers (Press release, K36 Therapeutics, AUG 7, 2025, View Source [SID1234655011]).

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!

With this clearance, KTX-2001 becomes the second NSD2 inhibitor from K36 to advance into clinical development. This Phase 1 program will evaluate KTX-2001 both as a monotherapy and in combination with Bayer’s androgen receptor inhibitor, darolutamide, for metastatic castration-resistant prostate cancer (mCRPC) NCT07103018.

"We are proud to announce the FDA cleared the IND for our second clinical program, KTX-2001, on July 3rd. This achievement demonstrates our team’s efficiency and focus on advancing KTX-2001 and positively impacting the lives of patients with mCRPC," said Terry Connolly, Ph.D., President and Chief Executive Officer of K36. "Along with this significant milestone, I am delighted to announce the Company has entered into a clinical trial collaboration agreement with Bayer for supply of darolutamide for the combination with KTX-2001 in our trial."

Under the terms of the agreement, K36 will conduct and sponsor the trial and Bayer will supply darolutamide. K36 maintains development and commercial rights to KTX-2001. KTX-2001 is a highly potent and selective inhibitor of NSD2 that is being developed for the treatment of solid tumors, initially mCRPC, and compliments K36’s first clinical candidate, KTX-1001, that is being developed for the treatment of relapsed and/or refractory multiple myeloma patients with genetic translocation t(4;14).

Separately, K36 announced it has selected the Prostate Cancer Clinical Trials Consortium (PCCTC) as the Contract Research Organization (CRO) who will operationalize the KTX-2001 clinical program, STRIKE-001. "We are pleased to partner with K36 as its CRO. PCCTC has established a robust network of academic and community sites, and has significant expertise in managing innovative, multisite prostate cancer clinical trials and are uniquely positioned to support the efficient launch and execution of the STRIKE-001 trial," said Jake Vinson, CEO of the PCCTC.

Jason Redman, MD, Senior Medical Director at K36 Therapeutics and leader of the KTX-2001 prostate cancer program stated, "Targeting NSD2 inhibition as a first-in-class, oral therapy represents a fundamentally new approach to treating prostate cancer by modulating the epigenetic drivers of tumor progression. This novel mechanism is distinct from other epigenetic therapies and addresses the underlying biology in a new way. The STRIKE-001 Phase 1 trial marks an important step forward, bringing a promising new option to patients with limited treatment alternatives."

About KTX-2001

KTX-2001 is a small molecule, specific inhibitor of the nuclear receptor binding SET domain protein 2 (NSD2) (also known as multiple myeloma [MM] SET domain-containing protein [MMSET]/Wolf-Hirschhorn syndrome candidate 1 protein [WHSC1]). Both KTX-1001 and KTX-2001 specifically inhibit NSD2 methylation of histone H3 at lysine 36 (H3K36) and disrupts aberrant NSD2-dependent oncogenic pathways.

About the KTX-2001 Phase 1 Clinical Trial

The Phase 1 clinical trial STRIKE-001 NCT07103018 is a multi-center, open label dose escalation of KTX-2001 single agent (Part A) and KTX-2001 combination with darolutamide (Part B). Part A will evaluate the safety and tolerability, maximum tolerated dose and recommended phase 2 dose(s) of KTX-2001 monotherapy in participants with mCRPC. Part B will study the safety and tolerability of KTX-2001+darolutamide to determine the recommended Phase 2 dose(s) of KTX-2001+darolutamide in participants with mCRPC. Additionally, objectives for Parts A and B include pharmacokinetics, pharmacodynamics and preliminary clinical activity. K36 expects to enroll approximately 140 participants with mCRPC who have progressed on a second generation or later androgen receptor inhibitor (AR) targeted therapy/androgen biosynthesis inhibitor starting in 2H2025.

On August 7, 2025 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported business highlights and financial results for the second quarter ended June 30, 2025 (Press release, Arcellx, AUG 7, 2025, View Source [SID1234654961]).

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 data presented for all 117 patients enrolled in the registrational iMMagine-1 study continue to demonstrate anito-cel’s potential to be a life-changing therapy for multiple myeloma patients," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "Along with our partners at Kite, we are planning to execute our anticipated 2026 commercial launch with the goal of ensuring access to as many patients as could benefit as rapidly as possible. To that end, we expect to launch in over 160 authorized treatment centers in the United States within the first year on the market and to have an adequate supply to meet physician expectations. We’re committed to delivering on the potential of anito-cel by ensuring best-in-class support and operational execution alongside our partners at Kite, who are the leaders in cell therapy. We are grateful for the patients, caregivers, and physicians who participated in our multiple myeloma anito-cel program, providing us an opportunity to advance this therapy to more patients in need. We look forward to sharing longer-term data from the iMMagine-1 study later this year. Additionally, it’s exciting to have engaged with the Food and Drug Administration and have our IND cleared earlier than expected for our next AML clinical program targeting CD33 and CD123 utilizing our ARC-SparX platform."

Recent Business Progress

Presented positive preliminary data for the Phase 2 pivotal iMMagine-1 study of anito-cel in patients with relapsed or refractory multiple myeloma (RRMM) at EHA (Free EHA Whitepaper)2025. The Phase 2 iMMagine-1 data were from a May 1, 2025 data cutoff date, including all 117 patients with a median follow-up of 12.6 months and a minimum follow-up of four months after treatment with anito-cel. All patients received a single infusion of anito-cel (target dose of 115×106 CAR+ viable T cells). 100 of 117 patients (85%) were triple refractory, and 47 of 117 patients (40%) were penta refractory. Patients received a median of three prior lines of therapy, with 60 of 117 patients (51%) having received three prior lines.

Overall response rate (ORR) was 97% (114/117) with a complete response/stringent complete response (CR/sCR) rate of 68% (79/117) and a very good partial response or higher (>VGPR) rate of 85% (100/117), per International Myeloma Working Group (IMWG) criteria as investigator-assessed. Of those evaluable for minimal residual disease (MRD) testing at the time of this data cut, 93.3% (70/75) achieved MRD negativity at a minimum of 10-5 sensitivity. Six-month, 12-month, and 18-month progression-free survival (PFS) rates were 92%, 79% and 66%, respectively, and six-month, 12-month, and 18-month overall survival (OS) rates were 97%, 95%, and 90% respectively. Median PFS and median OS have not been reached.

No delayed or non-immune effector cell-associated neurotoxicity syndrome (ICANS) neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, and no immune-mediated enterocolitis were observed with anito-cel. No additional treatment- or therapy-related deaths or Grade ≥3 cytokine release syndrome (CRS) or ICANS events occurred since the previous data presentation in December 2024.

Received clearance from the Food and Drug Administration for the clearance of an Investigational New Drug application for ACLX-004 targeting CD33 and CD123 utilizing the Company’s ARC-SparX platform.

Second Quarter 2025 Financial Highlights

Cash, cash equivalents, and marketable securities:
As of June 30, 2025, Arcellx had cash, cash equivalents, and marketable securities of $537.6 million. Arcellx anticipates that its cash, cash equivalents, and marketable securities will fund its operations into 2028.

Collaboration revenue:
Collaboration revenue was $7.6 million and $27.4 million for the quarters ended June 30, 2025 and 2024, respectively, a decrease of $19.8 million. This decrease was primarily driven by completion of dosing and manufacturing of anito-cel in the iMMagine-1 trial in the fourth quarter of 2024.

R&D expenses:
Research and development expenses were $37.6 million and $41.0 million for the quarters ended June 30, 2025 and 2024, respectively, a decrease of $3.4 million. This decrease was primarily driven by completion of dosing and manufacturing of anito-cel in the iMMagine-1 trial in the fourth quarter of 2024, partially offset by increased personnel costs, which includes non-cash stock-based compensation expense.

G&A expenses:
General and administrative expenses were $28.7 million and $21.4 million for the quarters ended June 30, 2025 and 2024, respectively, an increase of $7.3 million. This increase was primarily driven by increased commercial readiness costs and personnel costs, which includes non-cash stock-based compensation expense.

Net income or loss:
Net loss was $52.8 million and $27.2 million for the quarters ended June 30, 2025 and 2024, respectively.

On August 7, 2025 Genmab reported interim report for the first half ended June 30, 2025 (Press release, Genmab, AUG 7, 2025, View Source [SID1234654977]).

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!

Highlights

Epcoritamab advancing to earlier lines of therapy with the submission of a sBLA to the FDA for epcoritamab plus R2 in patients with relapsed or refractory FL
Rinatabart sesutecan (Rina-S) continues to progress, demonstrating encouraging antitumor activity in endometrial cancer in data presented at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting
Data from over 40 abstracts highlighting the depth, breadth and strength of Genmab’s comprehensive epcoritamab development program presented at multiple medical conferences
Genmab revenue increased 19% compared to the first six months of 2024, to $1,640 million
"In the first half of the year we continued to make progress towards our strategic priorities as we strive towards our goal of bringing our innovative therapies to additional patients in need. We further maximized the potential of our commercialized medicines with an additional sBLA submission for EPKINLY (epcoritamab-bysp) and the launch of Tivdak (tisotumab vedotin) in Japan. We also accelerated the development of our late-stage pipeline through both encouraging data presentations and, for Rina-S, the announcement of additional planned Phase 3 clinical trials," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Financial Performance First Half of 2025

Revenue was $1,640 million for the first six months of 2025 compared to $1,382 million for the first six months of 2024. The increase of $258 million, or 19%, was primarily driven by higher DARZALEX and Kesimpta royalties achieved under our collaborations with Johnson & Johnson (J&J) and Novartis Pharma AG (Novartis), respectively, and higher EPKINLY net product sales.
Royalty revenue was $1,378 million in the first six months of 2025 compared to $1,111 million in the first six months of 2024, an increase of $267 million, or 24%. The increase in royalties was driven by higher net sales of DARZALEX and Kesimpta.
Net sales of DARZALEX (daratumumab), including sales of the subcutaneous (SC) product (daratumumab and hyaluronidase-fihj, sold under the tradename DARZALEX FASPRO in the U.S.) by J&J were $6,776 million in the first six months of 2025 compared to $5,570 million in the first six months of 2024, an increase of $1,206 million or 22%.
Total costs and operating expenses were $1,092 million in the first six months of 2025 compared to $1,030 million in the first six months of 2024. The increase of $62 million, or 6%, was driven by the expansion of our product pipeline, including advancement of Rina-S, the continued development of Genmab’s broader organizational capabilities as well as profit-sharing amounts payable to AbbVie Inc. (AbbVie) related to EPKINLY sales.
Operating profit was $548 million in the first six months of 2025 compared to $352 million in the first six months of 2024.
Net financial items resulted in income of $119 million for the first six months of 2025 compared to $204 million in the first six months of 2024. The decrease was primarily due to a decrease in foreign exchange impacts driven by the change in functional currency of Genmab A/S on January 1, 2025, as well as a decrease in interest income for the first six months of 2025 compared to the first six months of 2024 related to average lower cash balances.

Outlook
Genmab is updating its revenue and operating profit guidance for 2025. The improved guidance is driven by higher total royalty revenues from DARZALEX.

2025 FULL YEAR OUTLOOK

(USD million) Revised Guidance Revised Mid-Point Previous Guidance Guidance Mid-Point
Revenue 3,500 – 3,700 3,600 3,340 – 3,660 3,500
Royalties 2,945 – 3,090 3,017 2,785 – 3,015 2,900
Net product sales/Collaboration revenue* 425 – 465 445 415 – 460 438
Milestones/Reimbursement revenue 130 – 145 138 140 – 185 162
Gross profit** 3,280 – 3,460 3,370 3,120 – 3,420 3,270
Operating expenses** (2,055) – (2,225) (2,140) (2,055) – (2,225) (2,140)
Operating profit 1,055 – 1,405 1,230 895 – 1,365 1,130
Net Product Sales and Collaboration Revenue consists of EPKINLY Net Product Sales in the U.S. and Japan and Tivdak (Genmab’s share of net profits) in the U.S. and Net Product Sales in Japan
Operating Expenses Range excludes Cost of Product Sales Range, which is included in Gross Profit Range

Other Matters
Both the functional currency of the Genmab A/S legal entity and the presentation currency of the condensed consolidated financials statements have been changed from DKK to USD effective January 1, 2025. The change in functional currency has been implemented with prospective effect. The change in presentation currency has been implemented with retrospective effect. Comparative figures for prior periods have been restated accordingly.

Conference Call
Genmab will hold a conference call to discuss the results for the first half of 2025 today, Thursday, August 7, at 6:00 pm CEST, 5:00 pm BST or 12:00 pm EDT. To join the call please use the below registration link. Registered participants will receive an email with a link to access dial-in information as well as a unique personal PIN: View Source A live and archived webcast of the call and relevant slides will be available at View Source

On August 7, 2025 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported pipeline and business achievements, reiterated key anticipated milestones, and announced second quarter 2025 financial results (Press release, Nuvalent, AUG 7, 2025, View Source [SID1234654993]).

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!

"With the initiation of our rolling NDA submission for zidesamtinib in TKI pre-treated advanced ROS1-positive NSCLC and the dosing of the first patient in our ALKAZAR Phase 3 trial of neladalkib in TKI-naïve advanced ALK-positive NSCLC, 2025 has been marked by transformative milestones towards our mission to discover, develop, and deliver precisely targeted therapies for patients with cancer," said Alexandra Balcom, Chief Financial Officer of Nuvalent. "We remain focused on continuing to deliver value that is grounded in prioritizing patient impact with topline pivotal data from our ALKOVE-1 trial of neladalkib in TKI pre-treated advanced ALK-positive NSCLC expected by year-end and the first report of preliminary data for other ALK-positive solid tumors at ESMO (Free ESMO Whitepaper), continued momentum in our HEROEX-1 trial of NVL-330 for HER2-altered NSCLC, and a robust discovery pipeline."

"In parallel to our continued execution against development and regulatory milestones, we are actively building the strong commercial infrastructure needed to achieve our vision of becoming a fully integrated, commercial-stage biopharmaceutical company," said James Porter, Ph.D., Chief Executive Officer of Nuvalent. "Today we are proud to announce the promotion of Jason Waters to Senior Vice President, Commercial, in recognition of his leadership in establishing our commercial strategy and shaping a launch-ready organization that extends from our founding values of Patient Impact, Empowerment, and Collaboration. Supported by a growing team and strong cash runway into 2028, we believe we are well-positioned to achieve our goals."

Recent Pipeline Achievements and Anticipated Milestones

ROS1 Program

•
The company has initiated its rolling NDA submission for zidesamtinib, a novel ROS1-selective inhibitor, in tyrosine kinase inhibitor (TKI) pre-treated patients with advanced ROS1-positive non-small cell lung cancer (NSCLC). The FDA agreed to accept the NDA for participation in the Real-Time Oncology Review (RTOR) pilot program, which facilitates earlier submission of topline efficacy and safety results prior to the submission of the complete application, to support
an earlier start to the FDA’s evaluation of the application. Completion of the NDA submission is targeted for the third quarter of 2025.
•
The NDA submission is based on positive pivotal data for TKI pre-treated patients with advanced ROS1-positive NSCLC enrolled in the global ARROS-1 Phase 1/2 clinical trial. These data were recently reported along with preliminary data from the ongoing Phase 2 TKI-naïve cohort of ARROS-1, in which a total of 104 patients had been enrolled as of June 16, 2025. The company continues to engage with the FDA on potential opportunities for line-agnostic expansion.
ALK Program

•
Nuvalent recently announced the dosing of the first patient in ALKAZAR, the company’s global Phase 3 randomized, controlled trial designed to evaluate neladalkib for the treatment of patients with TKI-naïve ALK-positive NSCLC. Patients will be randomized 1:1 to receive neladalkib monotherapy or alectinib monotherapy, a front-line standard of care, reflecting input from collaborating physician-scientists and alignment with global regulatory agencies.
•
Evaluation of neladalkib is ongoing in the ALKOVE-1 Phase 1/2 trial for patients with advanced ALK-positive NSCLC and other solid tumors:
o
The company expects to report pivotal data for TKI pre-treated patients with advanced ALK-positive NSCLC by year-end 2025.
o
The company will present preliminary data from the Phase 2 exploratory cohort for patients with ALK-positive solid tumors beyond NSCLC during a poster presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025, taking place October 17-21, 2025, in Berlin, Germany. Details of the presentation are as follows:
Title: Neladalkib (NVL-655) efficacy and safety in patients with ALK-positive solid tumors in the ALKOVE-1 study
Presentation Number: 972P
Session Category: Poster Session
Session Title: Developmental Therapeutics
Presentation Date and Time: Sunday, October 19, 2025, 12:00 – 12:45 CEST
Location: Hall 25
Presenter: Benjamin J. Solomon, MBBS, Ph.D. (Peter MacCallum Cancer Centre, Melbourne, Australia)

HER2 Program

•
Enrollment is ongoing in the HEROEX-1 Phase 1a/1b clinical trial evaluating the overall safety and tolerability of NVL-330 for pre-treated patients with HER2-altered NSCLC. Additional objectives include determination of the recommended Phase 2 dose, characterization of NVL-330’s pharmacokinetic profile, and preliminary evaluation of anti-tumor activity. The company expects to continue to progress the HEROEX-1 trial throughout 2025.

Business Updates

•
Jason Waters, MBA, Promoted to Senior Vice President, Commercial: Jason joined Nuvalent in 2024, bringing more than 20 years of experience in biopharma, and 15 years in commercial oncology focused on product launches and commercialization. Most recently, Jason served as the Head of Commercial at Mersana Therapeutics, where he led launch preparedness efforts and companion diagnostic strategy for the market entry of a novel antibody-drug conjugate. Prior to this, he held various commercial leadership roles at GSK, including Field Vice President and US Brand Lead for ZEJULA, where he led the integration and multiple launches of the brand following the acquisition of TESARO in acquisition in 2019. At TESARO, Jason served as the Global Brand Lead for ZEJULA, coordinating its EU launch. Earlier in his career, Jason was Senior Director, Sales Strategy at Takeda Oncology, where he contributed to the launches of subcutaneous VELCADE and NINLARO. He also held various sales, operational, and leadership roles at Sanofi, Aventis, and Abbott Laboratories.
•
Christy Oliger Appointed to Board of Directors: Nuvalent announced the appointment of Christy Oliger to its board of directors. Ms. Oliger brings more than 30 years of commercial and business experience in the pharmaceutical and biotechnology industry to the Nuvalent board. Most recently, Ms. Oliger served as Senior Vice President of the Oncology Business Unit at Genentech, where she was responsible for all commercial activities in the U.S. During her 20-year tenure at Genentech, Ms. Oliger held a number of senior leadership roles in both commercial and research and development across a variety of therapeutic areas, including oncology, neurology, rare disease, respiratory, dermatology and immunology. Prior to Genentech, she held management positions at Schering-Plough.

Second Quarter 2025 Financial Results

•
Cash Position: Cash, cash equivalents and marketable securities were $1.0 billion as of June 30, 2025. Nuvalent continues to believe its existing cash, cash equivalents and marketable securities will be sufficient to fund its current operating plan into 2028.
•
R&D Expenses: Research and development (R&D) expenses were $80.9 million for the second quarter of 2025.
•
G&A Expenses: General and administrative (G&A) expenses were $23.7 million for the second quarter of 2025.
•
Net Loss: Net loss was $99.7 million for the second quarter of 2025.

On August 7, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported business updates and announced financial results for the second quarter ended June 30, 2025 (Press release, Verastem, AUG 7, 2025, View Source [SID1234655012]).

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!

"In the second quarter of 2025, AVMAPKI FAKZYNJA CO-PACK became the first-ever treatment approved by the FDA specifically for use in patients with KRAS-mutated recurrent low-grade serous ovarian cancer, and we are off to a strong start with the launch of this innovative combination therapy," said Dan Paterson, president and chief executive officer of Verastem Oncology. "In the quarter, we also made significant progress across our pipeline programs with the first patient dosed in the U.S. Phase 1/2a trial for VS-7375, our potential best-in-class KRAS G12D (ON/OFF) inhibitor, and with positive updated data from our RAMP 205 trial in the front-line setting of metastatic pancreatic cancer. Our focus for the second half of the year is to continue to build on the positive launch momentum, continue to advance RAMP 205 and RAMP 301 clinical trials, and enroll patients in both the VS-7375 monotherapy and combination cohorts to unlock new opportunities with our RAS/MAPK-pathway focused portfolio."

Second Quarter 2025 and Recent Updates

AVMAPKI FAKZYNJA CO-PACK (avutometinib in combination with defactinib) U.S. Launch

Received U.S. Food and Drug Administration (FDA) approval for AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy on May 8, 2025, approximately two months in advance of the Prescription Drug User Fee Act (PDUFA) action date of June 30, 2025.
Achieved net product revenue of $2.1 million in the first six weeks of launch. AVMAPKI FAKZYNJA CO-PACK was launched in the U.S. within one week of FDA Approval.
AVMAPKI FAKZYNJA CO-PACK is now available through a distribution network in the U.S. that includes specialty pharmacies, specialty distributors, and group purchasing agreements that are in place.
Prescriptions for patients are being received from both academic and community centers, including both repeat prescriptions from physicians prescribing to multiple patients and refills for individual patients and there has been broad payer coverage and reimbursement.
A support program for patients prescribed AVMAPKI FAKZYNJA CO-PACK, called Verastem Cares, went operational immediately following launch.
Highlighted the update to the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines) in May 2025, which recommends avutometinib in combination with defactinib as a Category 2A recommendation for the treatment of KRAS-mutated recurrent LGSOC, which is aligned to the FDA-approved indication.
Submitted to the NCCN the publications of the RAMP 201 and FRAME studies in support of its consideration of the inclusion of the KRAS wild-type population evaluated in these trials in the NCCN Guidelines in July 2025. The NCCN Committee for Ovarian Cancer will hold its annual meeting in October 2025, and the Company has been informed that its submission will be reviewed at that time.
Avutometinib and Defactinib Combination in LGSOC

In the ongoing Phase 3 RAMP 301 trial, planned enrollment of the 270 patients is nearing completion. A pre-planned Interim Analysis (IA) by an Independent Data Monitoring Committee (IDMC) will be conducted to determine if a sample-size re-estimation is recommended for the RAMP 301 trial. The IA is intended to ensure the trial is adequately powered for success and preserves the trial’s integrity. The Company will be blinded to the specific results of the IA.
Enrollment in the Phase 2 RAMP 201J trial in Japan has been completed, and the Company has activated sites in Japan to join the global RAMP 301 trial.
Announced that the primary analysis from the RAMP 201 clinical trial was published in the Journal of Clinical Oncology on July 11, 2025.
Granted Orphan Drug Designation for avutometinib plus defactinib for the treatment of ovarian cancer by the European Commission based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products in July 2025.
Announced that the primary analysis from the FRAME study was published in Nature Medicine on June 27, 2025.
Presented an abstract at the ESMO (Free ESMO Whitepaper) Gynaecological Cancers Congress 2025 titled "Blood ctDNA vs tumor tissue screening for the detection of KRAS mutations in low-grade serous ovarian cancer" in June 2025.
Shared multiple oral and poster presentations at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 on April 25-30, highlighting the exploration of the mechanisms by which the Company’s FAK inhibitor increases the anti-tumor efficacy of avutometinib.
Key Milestones Expected for the Second Half of 2025:

Report outcome of the IDMC’s sample-size re-estimation recommendation for RAMP 301 in Q4 2025.
Report initial data from the RAMP 201J Phase 2 clinical trial being conducted in Japan with JGOG in Q4 2025.
Continue to advance the regulatory pathway in Japan and Europe.
VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor, in Advanced Solid Tumors

Announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to VS-7375, a potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, for the first-line treatment of patients with KRAS G12D-mutated locally advanced or metastatic adenocarcinoma of the pancreas (PDAC) and for the treatment of patients with KRAS G12D-mutated locally advanced or metastatic PDAC who have received at least one prior line of standard systemic therapy in July 2025.
Announced that the first patient has been dosed in the monotherapy portion of VS-7375-101, the U.S. Phase 1/2a clinical trial evaluating VS-7375 in patients with advanced KRAS G12D mutant solid tumors in June 2025.
The Company announced today the addition of four new cohorts in the VS-7375-101 trial to evaluate VS-7375 in combination with other treatments in various settings in advanced non-small cell lung cancer (NSCLC) and PDAC, and as monotherapy for advanced solid tumor types other than PDAC, NSCLC, or colorectal cancer (CRC) that harbor a KRAS G12D mutation.
Announced updated data from partner GenFleet Therapeutics’ Phase 1 study of VS-7375 in China (known as GFH375) at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June. In the trial, GFH375 demonstrated an overall response rate (ORR) of 52% in patients with PDAC and an ORR of 42% in NSCLC.
Announced that the FDA had cleared the Company’s Investigational New Drug (IND) application for VS-7375, enabling a Phase 1/2a trial in advanced solid tumors in the U.S. in April 2025.
Shared a presentation at the AACR (Free AACR Whitepaper) Annual Meeting 2025 in April, which highlighted that the KRAS G12D dual ON/OFF inhibitor VS-7375 was found to be more efficacious than KRAS G12D and pan-RAS ON-only inhibitors in preclinical models.
Key Milestones Expected for the Second Half of 2025:

Report a preliminary update on the Phase 1 monotherapy dose escalation in Q4 2025.
Subject to the results of the Phase 1 monotherapy dose escalation, the Company plans to initiate monotherapy expansion cohorts in both advanced PDAC and NSCLC.
Initiate the dose escalation cohorts in combination with cetuximab, chemotherapy, and chemotherapy with checkpoint-inhibitor for CRC, PDAC, and NSCLC, respectively, in Q4 2025.
Subject to the results of the Phase 1 dose escalation combinations cohorts with VS-7375, the Company plans to initiate a combination expansion cohort in CRC, PDAC, and NSCLC.
RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in First-Line Metastatic PDAC

Announced positive updated safety and efficacy results from the RAMP 205 Phase 1/2 trial evaluating avutometinib plus defactinib in combination with gemcitabine and Nab-paclitaxel in the front-line for patients with metastatic PDAC in May 2025. As of April 25, 2025, patients in the dose level 1 cohort, which was selected as the recommended Phase 2 dose (RP2D), achieved a confirmed ORR of 83% (10/12).
The RAMP 205 study has met the pre-defined criteria to advance beyond the first stage of the expansion study and enrollment is continuing in the expansion cohort for up to 29 patients at the RP2D.
Key Milestones Expected for the Second Half of 2025:

Complete enrollment in the RAMP 205 expansion cohort in Q3 2025.
RAMP 203: Avutometinib Plus Defactinib in Combination with a KRAS G12C Inhibitor in NSCLC

Patients continue to be evaluated in both the doublet and triplet combination cohorts of the study.
Key Milestones Expected for 2025:

Report an interim update on the safety and efficacy results in RAMP 203 from both the doublet and triplet combinations in Q4 2025.
Second Quarter 2025 Financial Results

Total Revenue for the three months ended June 30, 2025 (the "2025 Quarter") was $2.1 million, compared to $10.0, million for the three months ended June 30, 2024 (the "2024 Quarter"). Net Product Revenue for the 2025 Quarter was $2.1 million, compared to $0.0 million for the 2024 Quarter. The Company began commercial sales of the AVMAPKI FAKZYNJA CO-PACK within the United States following receipt of FDA approval in May 2025. Sale of COPIKTRA license and related assets revenue was $0.0 million for the 2025 Quarter, compared to $10.0 million for the 2024 Quarter. Revenue for the 2024 Quarter was comprised of one sales milestone payment of $10.0 million due upon Secura achieving cumulative worldwide net sales of COPIKTRA exceeding $100.0 million.

Total operating expenses for the 2025 Quarter were $45.9 million, compared to $28.3 million for the 2024 Quarter.

Cost of sales associated with product revenue was $0.4 million for the 2025 Quarter, compared to $0.0 for the 2024 Quarter.

Research & development expenses for the 2025 Quarter were $24.8 million, compared to $18.1 million for the 2024 Quarter. The increase of $6.7 million, or 37.0%, was primarily related to increased contract research organization costs and increased drug substance and drug product costs.

Selling, general & administrative expenses for the 2025 Quarter were $20.7 million, compared to $10.2 million for the 2024 Quarter. The increase of $10.5 million, or 102%, was primarily related to additional costs in anticipation of a potential launch of avutometinib and defactinib in KRAS-mutated LGSOC, increased personnel costs, including non-cash stock compensation, consulting, and professional fees.

Net loss for the 2025 Quarter was $25.9 million, or $0.39 per share (basic), compared to $8.3 million, or $0.31 per share (basic and diluted) for the 2024 Quarter.

For the 2025 Quarter, non-GAAP adjusted net loss was $41.4 million, or $0.63 per share (diluted) compared to non-GAAP adjusted net loss of $16.5 million, or $0.61 per share (diluted), for the 2024 Quarter. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.

Verastem Oncology ended the second quarter of 2025 with cash, cash equivalents and investments of $164.3 million.

Conference Call and Webcast

Verastem will host a conference call and webcast today at 4:30 pm ET to review the second quarter 2025 financial results and recent business updates. To access the conference call, please dial (800) 715-9871 (U.S.) or (646) 307-1963 (international) and enter the passcode 1210516 at least 10 minutes prior to the event start time. A live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source A replay of the webcast will be archived and available following the event.