MEK1 inhibitors(Dual specificity mitogen-activated protein kinase kinase 1 inhibitors), MEK2 inhibitors(Dual specificity mitogen-activated protein kinase kinase 2 inhibitors)

Therapeutic Areas

NeoplasmsNervous System DiseasesRespiratory Diseases+ [9]

Active Indication

BRAF Fusion GliomaLow grade gliomaBRAF mutation positive hairy cell leukemia+ [122]

Inactive Indication

Adenocarcinoma of LungAdult Acute Myeloblastic LeukemiaAdvanced biliary tract cancer+ [41]

Originator Organization

GSK Plc

Active Organization

National Cancer Institute

Novartis Pharmaceuticals Corp.

Novartis AG

+ [10]

Inactive Organization

GlaxoSmithKline Trading Services Ltd.

GlaxoSmithKline (China) Investment Co., Ltd.

Drug Highest PhaseApproved

First Approval Date

United States (29 May 2013),

BRAF V600E Mutation-Positive MelanomaBRAF V600K Mutation-Positive Melanoma

RegulationFast Track (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Conditional marketing approval (China), Orphan Drug (South Korea), Priority Review (Australia), Breakthrough Therapy (United States), Orphan Drug (Japan)

Structure/Sequence

Molecular FormulaC28H29FIN5O5S

InChIKeyOQUFJVRYDFIQBW-UHFFFAOYSA-N

CAS Registry1187431-43-1

300

Clinical Trials associated with Trametinib dimethyl sulfoxide

NCT06119789

/ RecruitingPhase 2IIT

The MATRIX Clinical Studies of Precision Cancer Therapy for Rare Cancers

Some rare cancers are hard-to-treat and patients have a poor prognosis. It is known that some of these patients have targetable molecular alterations, and some benefit from targeted drugs. However in many cases these drugs are not approved for the rare cancers.
In this study the aim is to do advanced molecular diagnostics to identify possible targets for therapy, and to treat accordingly.

Start Date20 Mar 2025

Sponsor / Collaborator

Oslo universitetssykehus HF

[+9]

NCT06819605

/ Not yet recruitingPhase 2/3IIT

Neoadjuvant Therapy With Dabrafenib and Trametinib Followed by Curettage/Fenestration Surgery Versus Up-front Curettage and Fenestration Surgery in BRAF V600E Mutated Ameloblastoma: A Randomized Controlled Clinical Trial

Ameloblastoma is the most common benign odontogenic tumor, characterized by high local invasiveness and a high recurrence rate. Currently, surgical treatment is the standard treatment modality. The main surgical approaches include radical resection represented by local extended resection (with a safety margin of more than 2 cm) and conservative procedures represented by curettage and fenestration. Although radical resection can effectively treat the disease, it often leads to severe jaw bone defects and even disrupts the continuity of the jaw bone. However, the conservative procedures have a recurrence rate of approximately 40%.
This study aims to reduce the recurrence rate of conservative procedures by using neoadjuvant therapy with dabrafenib combined with trametinib, and to reduce surgical trauma while improving the radical cure effect. The endpoints of this study are the 3-year and 5-year recurrence rates, as well as the effectiveness and safety of the neoadjuvant therapy.

Start Date01 Mar 2025

Sponsor / Collaborator

Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine

NCT06804824

/ RecruitingPhase 1

A Phase 1/1b, Open-Label, Multicenter, First-in-Human Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Anti-Tumor Activity of VVD-159642, a RAS-PI3Kα Inhibitor, as a Single Agent and in Combination in Participants With Advanced Solid Tumors

A FIH study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary anti-tumor activity of VVD-159642, a rat sarcoma viral oncogene-phosphatidylinositol 3-kinase alpha (RAS-PI3Kα) inhibitor, as a single agent and in combination with either sotorasib or trametinib in participants with advanced solid tumors.

Start Date25 Feb 2025

Sponsor / Collaborator

Vividion Therapeutics, Inc.

100 Clinical Results associated with Trametinib dimethyl sulfoxide

100 Translational Medicine associated with Trametinib dimethyl sulfoxide

100 Patents (Medical) associated with Trametinib dimethyl sulfoxide

3,965

Literatures (Medical) associated with Trametinib dimethyl sulfoxide

01 Dec 2025·CANCER CHEMOTHERAPY AND PHARMACOLOGY

Real-world pharmacokinetics of trametinib in pediatric low-grade glioma

Article

Author: Faure-Conter, Cécile ; Guitton, Jérôme ; Ceraulo, Antony ; Pagnot, Laurie ; Philippe, Michael ; Granger, Isaline ; Leblond, Pierre ; Favier, Bertrand

PURPOSE:

Trametinib, a MEK1/2 inhibitor, has emerged as a promising treatment for pediatric patients with low-grade gliomas (LGG). However, trametinib exhibits significant inter-individual pharmacokinetic (PK) variability, and studies in adults demonstrated an exposure-efficacy relationship. This study aimed to evaluate the PK profile of trametinib in pediatric routine care and explore potential exposure-outcome relationships.

METHODS:

We analyzed PK data from 65 blood samples from 19 children receiving trametinib, either as single agent or in combination with dabrafenib. A trough concentration (Cmin) range of 8-15 ng/mL was considered, based on average exposure reported in the largest pediatric study.

RESULTS:

The mean Cmin was 8.82 ng/ml, with 64.6% of samples falling within the predefined target range, while 35.4% were below it. Regarding tolerance, 84.2% of patients experienced treatment-related toxicities, predominantly skin and subcutaneous tissue disorders. Efficacy data were limited.

CONCLUSION:

These findings underscore the necessity of therapeutic drug monitoring in pediatric patients to optimize treatment efficacy and minimize toxicity, highlighting trametinib's potential for personalized dosing strategies in this population.

01 Dec 2025·CHILDS NERVOUS SYSTEM

Efficacy and safety of dabrafenib plus trametinib in pediatric versus adult gliomas: a systematic review and meta-analysis

Review

Author: Tos, Salem M ; Kooshki, Alireza ; Najari, Dorsa ; Hajikarimloo, Bardia ; Zare, Amir Hessam ; Alvani, Mohammadamin Sabbagh ; Hasanzade, Arman ; Habibi, Mohammad Amin ; Zare, Amir Hossein

BACKGROUND:

The clinical course and therapeutic outcomes of pediatric and adult gliomas vary. Dabrafenib plus trametinib is a new therapeutic option for the management of gliomas. This study aimed to compare the outcomes of co-administration of dabrafenib and trametinib in pediatric and adult gliomas.

METHODS:

Systematic search was conducted in four electronic databases encompassing Pubmed, Embase, Scopus, and Web of Science. Publications that assessed the role of dabrafenib plus trametinib in adults or pediatrics were included.

RESULTS:

Eight studies with 243 individuals, encompassing 161 pediatrics and 82 adults, were included in our study. We demonstrated that despite a higher objective response rate (ORR) (53% [95% CI, 44-61%] vs. 39% [95% CI, 26-54%], P = 0.11) and clinical benefit rate (CBR) (87% [95% CI, 72-95%] vs. 73% [95% CI, 54-86%], P = 0.16) among pediatrics, the difference was insignificant. We exhibited that younger age, BRAF V600 mutation, and longer therapy periods were accompanied by better radiological outcomes among pediatrics, and the female gender was correlated with better radiological results in adults. Our findings showed that the pooled adverse event (AE) rate was 96% (95% CI, 69-100%) in pediatrics and 83% (95% CI, 42-97%) among adults; however, there was no meaningful difference (P = 0.30).

CONCLUSION:

Co-administration of dabrafenib and trametinib is accompanied by promising results among pediatrics and adults diagnosed with glioma. However, the comparison results should be interpreted meticulously due to limitations that may affect the generalizability of the findings.

01 May 2025·INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES

Effects of F53 hotspot mutations on the molecular dynamics of MEK1 protein and the binding of its FDA-approved inhibitors

Article

Author: Vural, Berkin Ersin ; Yandım, Cihangir

MEK1 (MAP2K1) could emerge as an oncogenic protein in the presence of certain mutations, and could be inhibited by FDA-approved drugs (trametinib, cobimetinib, binimetinib and selumetinib). However, how the mutations on MEK1's hotspot residue F53 affect the bindings of these therapeutic molecules remained largely unexplored. We performed molecular dynamics (MD) simulations with wild-type and mutated (F53L/V/C/I/Y) MEK1 structures in the presence and absence of these drugs and observed changes on the motion of MEK1. A longer duration in the lowest energy state conformation was observed during the simulations in the presence of F53 mutations. This was complemented by cross-correlated motions of amino acids of MEK1. More importantly, the binding affinities of inhibitors were affected. There was a marked reduction in the calculated binding affinity of trametinib in the presence of F53C mutation. On the other hand, the binding affinities of cobimetinib and selumetinib could overcome F53 mutations on MEK1. Binimetinib interestingly exhibited an increased binding affinity when F53C/I mutations were present. Taken together, our results provide a comprehensive perspective on the structural and drug-binding effects of observed mutations on the hotspot residue F53 within MEK1; warranting further research in stratifying F53 hotspot mutations for effective drug binding.

128

News (Medical) associated with Trametinib dimethyl sulfoxide

20 Mar 2025

·investors.erasca.com

Erasca Reports Fourth Quarter and Full Year 2024 Business Updates and Financial Results

Potentially best-in-class RAS-targeting franchise advancing with both ERAS-0015 and ERAS-4001 expected to enter the clinic in 2025 Ongoing Phase 3 SEACRAFT-2 registrational trial progressing well with Stage 1 randomized data expected in H2 2025 Robust balance sheet with cash, cash equivalents, and marketable securities of $440 million as of December 31, 2024 is expected to fund operations into H2 2027 SAN DIEGO, March 20, 2025 (GLOBE NEWSWIRE) -- Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers, today provided business updates and reported financial results for the fiscal quarter and full year ended December 31, 2024. “In 2024, we prepared our RAS-targeting franchise to enter the clinic and further advanced our registrational program for naporafenib. The high enthusiasm for our RAS-targeting franchise is encouraging, particularly as our potential best-in-class pan-RAS molecular glue ERAS-0015 and our potential first-in-class pan-KRAS inhibitor ERAS-4001 approach the clinic. The high prevalence of KRAS alterations and the significant unmet need among these patients offer substantial opportunities for both candidates in key indications, namely colorectal, lung, pancreatic, and other solid tumor cancers,” said Jonathan E. Lim, M.D., Erasca’s chairman, CEO, and co-founder. “The SEACRAFT-2 trial is progressing well, and with FDA Fast Track Designation (FTD) granted in NRASm melanoma, this combination has the potential to be first-to-market in this area of high unmet need with no approved targeted therapies.” Dr. Lim added, “We have an exciting year ahead focused on shutting down RAS and expect multiple value drivers in 2025 and beyond. In the second half of 2025, we expect Stage 1 randomized data from SEACRAFT-2 in patients with NRASm melanoma. For our RAS-targeting franchise, we expect investigational new drug (IND) submissions for ERAS-0015 in mid-second quarter and for ERAS-4001 in the second quarter of 2025. We continue to be well capitalized and capital efficient and have revised our cash runway guidance from the first half of 2027 to the second half of 2027.” Research and Development (R&D) Highlights RAS-Targeting Franchise Announced Progress Across RAS-Targeting Franchise: In October 2024, Erasca presented a program update for the pan-RAS molecular glue ERAS-0015 and pan-KRAS inhibitor ERAS-4001 as part of a virtual investor event. The updates highlighted the rapid progress observed across both programs including in-house confirmation of potential best-in-class profiles for both agents and completion of many key activities to support IND submissions. In-Licensed Potential Best-in-Class and First-in-Class RAS-Targeting Franchise: In May 2024, Erasca announced exclusive license agreements for two preclinical RAS programs—a potential best-in-class pan-RAS molecular glue (ERAS-0015) and a potential first-in-class pan-KRAS inhibitor (ERAS-4001). ERAS-0015 and ERAS-4001 are potent, orally bioavailable molecules with complementary RAS inhibitory mechanisms. ERAS-0015 has the potential to address unmet medical needs in approximately 2.7 million patients who are diagnosed annually worldwide with RAS-mutant tumors, including the more than 2.2 million patients with KRAS-mutant tumors whom ERAS-4001 could also address. Naporafenib Program Presented Promising SEACRAFT-1 Phase 1b Data: In October 2024, Erasca announced promising initial Phase 1b data for naporafenib plus trametinib (MEKINIST®) in the melanoma cohort of SEACRAFT-1 at the 36th EORTC-NCI-AACR (ENA) Symposium on Molecular Targets and Cancer Therapeutics. Efficacy and tolerability data supported the rationale for pursuing an NRASm melanoma tissue-specific indication and reinforced the potential of the ongoing Phase 3 SEACRAFT-2 registrational trial, which was initiated in the second quarter of 2024. Analysis of Median Overall Survival (mOS) Data for Naporafenib and Trametinib: In March 2024, a pooled analysis of patients with NRASm melanoma dosed with the combination of naporafenib and trametinib at two different doses across two different trials (Phase 1b and Phase 2) showed an mOS of 13.0 and 14.1 months, respectively. The pooled dataset compared favorably relative to historical benchmarks. Corporate Highlights Extended Cash Runway into H2 2027: In 2024, Erasca raised $251 million in equity financings, including a $45 million oversubscribed private placement financing and a $184 million oversubscribed underwritten offering, both of which were led by high-quality new and existing healthcare-focused investors and both of which helped extend our anticipated cash runway into the second half of 2027. Strengthened Leadership Team and RDCAB: Erasca strengthened its leadership team and Research, Development, and Commercial Advisory Board (RDCAB) with three key appointments. Michael Humphries, Ph.D., was appointed as vice president of medical affairs, bringing to Erasca over 15 years of medical affairs, clinical development, and drug discovery experience at Array Biopharma, Bayer Oncology, ARIAD Pharmaceuticals, Takeda, AnHeart Therapeutics, and Nuvation Bio. His leadership includes three U.S. launches of next-generation tyrosine kinase inhibitors in three variants of oncogene-addicted non-small cell lung cancer (NSCLC). In his most recent role at Nuvation Bio, Dr. Humphries served as vice president head of medical affairs where he built out the medical affairs strategy, infrastructure, culture, and personnel. He earned his Ph.D. in cell and developmental biology from the University of Colorado at Denver. Yifah Yaron, M.D., Ph.D., was appointed as vice president of clinical development, bringing over 18 years of experience in clinical drug development focusing on oncology. She most recently led clinical teams at Harpoon Therapeutics (acquired by Merck), and served in clinical development roles at Cytomx Therapeutics, Exelixis, and Genentech. During her time at Exelixis, Dr. Yaron held increasing leadership responsibilities on the cabozantinib program, including serving as a member of the clinical filing team for the first indication in medullary thyroid cancer. Dr. Yaron completed an oncology fellowship at UCSF and an internal medicine residency at University of Rochester Strong Memorial Hospital. She earned her M.D. and Ph.D. degrees (Ph.D. in biological chemistry) as well as her B.S. in biology from the University of California, Los Angeles. Jean-Michel Vernier, Ph.D., was appointed as senior chemistry advisor with 25+ years of drug discovery and development experience. He currently serves as senior vice president of chemistry at Radionetics Oncology and previously served as Erasca’s vice president of chemistry. He held positions of increasing responsibilities at several companies, including Ignyta, Ardea Biosciences, Merck Research Laboratories, and SIBIA Neurosciences. Throughout his career, Dr. Vernier has led departments engaged in early-stage drug discovery programs and drug substance GMP production. He has co-authored more than 30 peer-reviewed publications and is an inventor on more than 50 U.S. patents. Dr. Vernier earned his Ph.D. in synthetic organic chemistry from the University Louis Pasteur, Strasbourg, France, and was a postdoctoral fellow at Colorado State University. Michael Humphries, Ph.D., was appointed as vice president of medical affairs, bringing to Erasca over 15 years of medical affairs, clinical development, and drug discovery experience at Array Biopharma, Bayer Oncology, ARIAD Pharmaceuticals, Takeda, AnHeart Therapeutics, and Nuvation Bio. His leadership includes three U.S. launches of next-generation tyrosine kinase inhibitors in three variants of oncogene-addicted non-small cell lung cancer (NSCLC). In his most recent role at Nuvation Bio, Dr. Humphries served as vice president head of medical affairs where he built out the medical affairs strategy, infrastructure, culture, and personnel. He earned his Ph.D. in cell and developmental biology from the University of Colorado at Denver. Yifah Yaron, M.D., Ph.D., was appointed as vice president of clinical development, bringing over 18 years of experience in clinical drug development focusing on oncology. She most recently led clinical teams at Harpoon Therapeutics (acquired by Merck), and served in clinical development roles at Cytomx Therapeutics, Exelixis, and Genentech. During her time at Exelixis, Dr. Yaron held increasing leadership responsibilities on the cabozantinib program, including serving as a member of the clinical filing team for the first indication in medullary thyroid cancer. Dr. Yaron completed an oncology fellowship at UCSF and an internal medicine residency at University of Rochester Strong Memorial Hospital. She earned her M.D. and Ph.D. degrees (Ph.D. in biological chemistry) as well as her B.S. in biology from the University of California, Los Angeles. Jean-Michel Vernier, Ph.D., was appointed as senior chemistry advisor with 25+ years of drug discovery and development experience. He currently serves as senior vice president of chemistry at Radionetics Oncology and previously served as Erasca’s vice president of chemistry. He held positions of increasing responsibilities at several companies, including Ignyta, Ardea Biosciences, Merck Research Laboratories, and SIBIA Neurosciences. Throughout his career, Dr. Vernier has led departments engaged in early-stage drug discovery programs and drug substance GMP production. He has co-authored more than 30 peer-reviewed publications and is an inventor on more than 50 U.S. patents. Dr. Vernier earned his Ph.D. in synthetic organic chemistry from the University Louis Pasteur, Strasbourg, France, and was a postdoctoral fellow at Colorado State University. Key Upcoming Milestones SEACRAFT-2: Randomized pivotal Phase 3 trial for naporafenib plus trametinib in patients with NRASm melanoma Phase 3 Stage 1 randomized dose optimization data expected in H2 2025 AURORAS-1: Phase 1 trial for ERAS-0015 (pan-RAS molecular glue) in patients with RASm solid tumors IND filing expected in mid-Q2 2025 Initial Phase 1 monotherapy data in relevant tumor types expected in 2026 BOREALIS-1: Phase 1 trial for ERAS-4001 (pan-KRAS inhibitor) in patients with KRASm solid tumors IND filing expected in Q2 2025 Initial Phase 1 monotherapy data in relevant tumor types expected in 2026 Phase 3 Stage 1 randomized dose optimization data expected in H2 2025 IND filing expected in mid-Q2 2025 Initial Phase 1 monotherapy data in relevant tumor types expected in 2026 IND filing expected in Q2 2025 Initial Phase 1 monotherapy data in relevant tumor types expected in 2026 Fourth Quarter and Full Year 2024 Financial Results Cash Position: Cash, cash equivalents, and marketable securities were $440.5 million as of December 31, 2024, compared to $322.0 million as of December 31, 2023. Research and Development (R&D) Expenses: R&D expenses were $26.1 million for the quarter ended December 31, 2024, compared to $24.8 million for the quarter ended December 31, 2023. The increase was primarily driven by increases in expenses incurred in connection with clinical trials, preclinical studies, discovery activities, and outsourced services and consulting fees. R&D expenses were $115.4 million for the full year ended December 31, 2024, compared to $103.8 million for the full year ended December 31, 2023. Erasca also recorded $22.5 million of in-process R&D expense during the year ended December 31, 2024 for upfront payments under Erasca’s ERAS-0015 and ERAS-4001 license agreements. General and Administrative (G&A) Expenses: G&A expenses were $9.6 million for the quarter ended December 31, 2024, compared to $9.1 million for the quarter ended December 31, 2023. The increase was primarily driven by an increase in personnel costs, including stock-based compensation expense. G&A expenses were $41.7 million for the full year ended December 31, 2024, compared to $37.7 million for the full year ended December 31, 2023. Net Loss: Net loss was $32.2 million for the quarter ended December 31, 2024, compared to $29.7 million for the quarter ended December 31, 2023. For the full year ended December 31, 2024, Erasca reported a net loss of $161.7 million, or $(0.69) per basic and diluted share, compared to a net loss of $125.0 million, or $(0.83) per basic and diluted share, for the full year ended December 31, 2023. About Erasca At Erasca, our name is our mission: To erase cancer. We are a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers. Our company was co-founded by leading pioneers in precision oncology and RAS targeting to create novel therapies and combination regimens designed to comprehensively shut down the RAS/MAPK pathway for the treatment of patients with cancer. We have assembled one of the deepest RAS/MAPK pathway-focused pipeline in the industry. We believe our team’s capabilities and experience, further guided by our scientific advisory board which includes the world’s leading experts in the RAS/MAPK pathway, uniquely position us to achieve our bold mission of erasing cancer. Cautionary Note Regarding Forward-Looking Statements Erasca cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. The forward-looking statements are based on our current beliefs and expectations and include, but are not limited to: our expectations regarding the potential therapeutic benefits and potential patient population for each of our product candidates, including naporafenib, ERAS-0015, and ERAS-4001; the planned advancement of our development pipeline, including the anticipated timing of data readouts for the SEACRAFT-2, AURORAS-1, and BOREALIS-1 trials; the anticipated timing of the IND submissions for the AURORAS-1 and BOREALIS-1 trials; and the sufficiency of our cash, cash equivalents, and marketable securities to fund operations into the second half of 2027. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in our business, including, without limitation: our approach to the discovery and development of product candidates based on our singular focus on shutting down the RAS/MAPK pathway, a novel and unproven approach; preliminary results of clinical trials are not necessarily indicative of final results and one or more of the clinical outcomes may materially change as patient enrollment continues, following more comprehensive reviews of the data and more patient data become available; the analysis of pooled Phase 1 and Phase 2 naporafenib plus trametinib data covers two clinical trials with different designs and inclusion criteria, which cannot be directly compared, and therefore may not be a reliable indicator of survival data; due to differences between trial designs and subject characteristics, comparing clinical data across different trials may not be a reliable indicator of such data; results from preclinical studies or early clinical trials not necessarily being predictive of future results; we only have one product candidate in clinical development and all of our other development efforts are in the preclinical or development stage; our SEACRAFT trials may not support the registration of naporafenib; our assumptions around which programs may have a higher probability of success may not be accurate, and we may expend our limited resources to pursue a particular product candidate and/or indication and fail to capitalize on product candidates or indications with greater development or commercial potential; potential delays in the commencement, enrollment, data readout, and completion of clinical trials and preclinical studies; our dependence on third parties in connection with manufacturing, research, and preclinical and clinical testing; unexpected adverse side effects or inadequate efficacy of our product candidates that may limit their development, regulatory approval, and/or commercialization, or may result in recalls or product liability claims; unfavorable results from preclinical studies or clinical trials; the inability to realize any benefits from our current licenses, acquisitions, and collaborations, and any future licenses, acquisitions, or collaborations, and our ability to fulfill our obligations under such arrangements; regulatory developments in the United States and foreign countries; later developments with the FDA or EU health authorities may be inconsistent with the feedback received to date regarding our development plans and trial designs; Fast Track Designation may not lead to a faster development or regulatory review or approval process, and does not increase the likelihood that our product candidates will receive marketing approval; our ability to obtain and maintain intellectual property protection for our product candidates and maintain our rights under intellectual property licenses; our ability to fund our operating plans with our current cash, cash equivalents, and marketable securities; and other risks described in our prior filings with the Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in our annual report on Form 10-K for the year ended December 31, 2024, and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and we undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. MEKINIST® is a registered trademark owned by or licensed to Novartis AG, its subsidiaries, or affiliates. Contact: Joyce Allaire LifeSci Advisors, LLC jallaire@lifesciadvisors.com Source: Erasca, Inc. Source: Erasca, Inc.

Phase 1Executive ChangeFinancial StatementLicense out/in

27 Feb 2025

·ir.c4therapeutics.com

C4 Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Business Highlights

Planning Activities Are Ongoing for Next Phase of Cemsidomide Clinical Development in Multiple Myeloma and Non-Hodgkin’s Lymphoma; Trials Expected to Initiate in Early 2026 CFT1946 Phase 1/2 Trial Continues to Progress Across Multiple Cohorts; Data in Melanoma and Colorectal Cancer Expected in Second Half of 2025 Cash, Cash Equivalents and Marketable Securities of $267.3 million as of December 31, 2024; Expected to Provide Runway into 2027 WATERTOWN, Mass., Feb. 27, 2025 (GLOBE NEWSWIRE) -- C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, today reported financial results for the year ended December 31, 2024, as well as business updates. “In 2024, C4T made significant progress across our degrader portfolio and our discovery collaborations. We shared clinical data from our two lead programs, where cemsidomide demonstrated a potential best-in-class profile, and CFT1946 demonstrated proof of mechanism and early signs of anti-tumor activity, and a third program, CFT8919, entered clinical development in Greater China. We also delivered two development candidates to Biogen and initiated a new discovery collaboration with Merck KGaA, Darmstadt, Germany,” said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. “Entering 2025, we continue to advance these clinical programs and operationalize the next phase of cemsidomide development to enable patient dosing in early 2026. We remain focused on unlocking value of cemsidomide and generating data from the Phase 1 trials of CFT1946 and CFT8919 to inform future development strategies for these programs.” FOURTH QUARTER 2024 HIGHLIGHTS AND RECENT ACHIEVEMENTS Cemsidomide: Multiple Myeloma (MM) In December 2024, at the Annual Society of Hematology (ASH) meeting, presented data from the ongoing dose escalation trial with cemsidomide in combination with dexamethasone. As of the data cutoff date of October 11, 2024, the 75 µg once daily (QD) dose level achieved an overall response rate (ORR) of 36 percent. Cemsidomide was well-tolerated across all dose levels. The maximum tolerated dose has not yet been reached. Patients are enrolling at the 100 µg QD dose level. Non-Hodgkin’s Lymphoma (NHL) In December 2024, at ASH, presented data from the ongoing cemsidomide monotherapy dose escalation trial. As of the data cutoff date of October 11, 2024, cemsidomide demonstrated an ORR of 38 percent across all subtypes and doses studied. In peripheral T-cell lymphoma (PTCL), cemsidomide achieved an ORR of 44 percent and a 25 percent complete metabolic response rate. The maximum tolerated dose has not yet been reached. Patients are enrolling at the 87.5 µg QD dose level. CFT1946: In October 2024, at the Targeted Protein Degradation Summit, presented new preclinical data demonstrating CFT1946 crosses the blood-brain barrier, with Kpuu values of 0.34-0.88, an important feature as a portion of patients with BRAF V600 mutant solid tumors develop brain metastases. Continued to enroll patients with BRAF V600 mutations in the ongoing Phase 1/2 trial across multiple cohorts including monotherapy in melanoma, in combination with trametinib in melanoma and in combination with cetuximab in colorectal cancer (CRC). CFT8919: In November 2024, Betta Pharmaceuticals, with C4T support, initiated the Phase 1 clinical trial of CFT8919 in Greater China. CORPORATE UPDATE: Continued to evolve the Board of Directors with the appointment of biotechnology executive, Steve Hoerter, who has over three decades of executive management, commercial and board experience in oncology. KEY UPCOMING MILESTONES Cemsidomide: Complete Phase 1 dose escalation and present data in MM and NHL in the second half of 2025. Open expansion cohort(s) in PTCL as part of the current Phase 1/2 trial in the second half of 2025. Enable initiation of the next phase of clinical development for MM and PTCL, with new studies expected to initiate in early 2026. CFT1946: Complete monotherapy Phase 1 dose escalation in BRAF V600 mutant solid tumors in the first half of 2025. Present Phase 1 data in the second half of 2025, which will include: (1) CFT1946 monotherapy in BRAF V600 mutant solid tumors, (2) CFT1946 monotherapy expansion in melanoma and (3) CFT1946 in combination with cetuximab in CRC. UPCOMING INVESTOR EVENTS March 3, 2025 at 9:10 am ET: Management will be present at TD Cowen 44th Annual Healthcare Conference taking place March 3 – 5, 2025 in Boston, MA. March 10, 2025: Management will participate in the Leerink Partners Global Healthcare Conference taking place March 9 – 12, 2025 in Miami, FL. FOURTH QUARTER AND FULL YEAR 2024 FINANCIAL RESULTS Revenue: Total revenue for the fourth quarter and full year ended December 31, 2024 was $5.2 million and $35.6 million, respectively, compared to $3.3 million and $20.8 million for the prior year periods. The increase in revenue was primarily due to new collaborations with Merck KGaA, Darmstadt, Germany (MKDG) and Merck, as well as revenue related to our ongoing collaboration with Betta Pharmaceuticals. Total revenue for the full year 2024 reflects revenue recognized under our collaborations with Biogen, Betta Pharmaceuticals, Merck, MKDG and Roche, and total revenue recognized for the full year 2023 reflects revenue recognized under collaboration agreements with Biogen, Roche and Calico. Research and Development (R&D) Expense: R&D expense for the fourth quarter and full year ended December 31, 2024 was $32.5 million and $110.6 million, respectively, compared to $30.4 million and $117.7 million for the prior year periods. The increase in R&D expense for the fourth quarter was primarily related to clinical trial expense as cemsidomide and CFT1946 continue to advance. The decrease in R&D expense for the full year was primarily due to reduced headcount and external services resulting from restructuring activities that occurred in January 2024. General and Administrative (G&A) Expense: G&A expense for the fourth quarter and full year ended December 31, 2024 was $10.4 million and $42.1 million, respectively, remaining relatively flat compared to $10.3 million and $42.1 million for the prior year periods. Net Loss and Net Loss per Share: Net loss for the fourth quarter and full year ended December 31, 2024 was $34.6 million and $105.3 million, respectively, compared to $34.8 million and $132.5 million for the prior year periods. Net loss per share for the fourth quarter and full year ended December 31, 2024 was $0.49 and $1.52, respectively, compared to $0.68 and $2.67 for the prior year periods. Cash Position and Financial Guidance: Cash, cash equivalents and marketable securities as of December 31, 2024 were $267.3 million, compared to $281.7 million as of December 31, 2023. The reduction in cash, cash equivalents and marketable securities during 2024 was primarily the result of $65.2 million of cash used in operating activities (net of $16 million received in milestone payments from Biogen), partially offset by $24.4 million in proceeds from the sale of shares of our common stock through our “at-the-market” offering arrangement and $20 million received under the Betta stock purchase agreement, all of which were previously disclosed. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2024 will enable the company to fund its operating plan into 2027. About C4 TherapeuticsC4 Therapeutics (C4T) (Nasdaq: CCCC) is a clinical-stage biopharmaceutical company dedicated to delivering on the promise of targeted protein degradation science to create a new generation of medicines that transforms patients’ lives. C4T is progressing targeted oncology programs through clinical studies and leveraging its TORPEDO® platform to efficiently design and optimize small-molecule medicines to address difficult-to-treat diseases. C4T’s degrader medicines are designed to harness the body’s natural protein recycling system to rapidly degrade disease-causing proteins, offering the potential to overcome drug resistance, drug undruggable targets and improve patient outcomes. For more information, please visit www.c4therapeutics.com. About CemsidomideCemsidomide is an investigational, orally bioavailable small-molecule degrader designed to be a more potent and selective degrader of IKZF1/3, transcription factors that drive multiple myeloma (MM) and non-Hodgkin’s lymphomas (NHL), with unique pharmacokinetic properties. Clinical data has shown that cemsidomide is well-tolerated. In MM, cemsidomide displays evidence of anti-myeloma activity and immunomodulatory effects. In NHL, cemsidomide displays evidence of anti-lymphoma activity. More information may be accessed at www.clinicaltrials.gov (identifier: NCT04756726). About CFT1946CFT1946 is an investigational, orally bioavailable brain penetrant small molecule degrader of BRAF V600 mutations in solid tumors currently being evaluated in a Phase 1/2 global clinical trial in patients refractory to BRAF inhibitors. CFT1946 is designed to be potent and selective against the BRAF V600 mutant form. Initial clinical data from the Phase 1 trial demonstrate that CFT1946 has a well-tolerated safety profile, demonstrates dose-dependent bioavailability and degradation of BRAF V600E protein, and demonstrates evidence of monotherapy anti-tumor activity. CFT1946 is the only degrader of BRAF V600 mutant solid tumors in clinical trials. More information about this trial may be accessed at www.clinicaltrials.gov (identifier: NCT05668585). About CFT8919CFT8919 is an orally bioavailable allosteric degrader that is designed to be potent and selective against EGFR bearing an oncogenic L858R mutation. In preclinical studies, CFT8919 is active in in vitro and in vivo models of L858R driven non-small cell lung cancer. Importantly, CFT8919 retains full activity against additional EGFR mutations that confer resistance against approved EGFR inhibitors including L858R-C797S, L858R-T790M and L858R-T790M-C797S. Forward-Looking StatementsThis press release contains “forward-looking statements” of C4 Therapeutics, Inc. within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements may include, but may not be limited to, express or implied statements regarding our ability to develop potential therapies for patients; the design and potential efficacy of our therapeutic approaches; the predictive capability of our TORPEDO® platform in the development of novel, selective, orally bioavailable BiDAC™ and MonoDAC™ degraders; the potential timing, design and advancement of our preclinical studies and clinical trials, including the potential timing for and receipt of regulatory authorization related to clinical trials and other clinical development activities including clinical trial commencement or cohort initiation; our ability and the potential to successfully manufacture and supply our product candidates for clinical trials; our ability to replicate results achieved in our preclinical studies or clinical trials in any future studies or trials; our ability to replicate interim or early-stage results from our clinical trials in the results obtained when those clinical trials are completed or when those therapies complete later-stage clinical trials; regulatory developments in the United States and foreign countries; the anticipated timing and content of presentations of data from our clinical trials; and our ability to fund our future operations. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties related to the initiation, timing, advancement and conduct of preclinical and clinical studies and other development requirements for our product candidates; the risk that any one or more of our product candidates will cost more to develop or may not be successfully developed and commercialized; and the risk that sufficient capital to fund our future operations will be available to us on acceptable terms or at the times required. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in C4 Therapeutics’ most recent Annual Report on Form 10-K and/or Quarterly Report on Form 10-Q, as filed with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and C4 Therapeutics undertakes no duty to update this information unless required by law. Contacts:Investors:Courtney SolbergAssociate Director, Investor RelationsCSolberg@c4therapeutics.com Media:Loraine SpreenSenior Director, Corporate Communications & Patient AdvocacyLSpreen@c4therapeutics.com

Phase 1Financial StatementClinical Result

14 Jan 2025

·ir.c4therapeutics.com

C4 Therapeutics Announces 2025 Milestones Across Clinical Portfolio of Degrader Medicines Pursuing Targets of High Unmet Need in Oncology

Cemsidomide Data Presented at American Society of Hematology (ASH) Annual Meeting Support Best-in-Class Profile; Program Advancing to Next Phase of Clinical Development in Multiple Myeloma and Non-Hodgkin’s Lymphoma CFT1946 Phase 1 Trial Continues to Progress in BRAF V600X Solid Tumors With Monotherapy Dose Escalation Expected to Complete in 1H 2025; Data in Melanoma and Colorectal Cancer Expected in Second Half of 2025 CFT8919 Progressing Through Phase 1 Dose Escalation in Greater China; Phase 1 Data Will Inform Future Development Plans Outside of China Cash Runway Expected to Fund Operations Into 2027 WATERTOWN, Mass., Jan. 14, 2025 (GLOBE NEWSWIRE) -- C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, today announced its anticipated 2025 milestones as it continues its evolution into becoming a fully integrated biotechnology company focused on orally bioavailable degraders. “Stellar execution in 2024 has set up 2025 to be a pivotal year for the company as we work to generate important data that will position us to advance programs and bring degrader medicines to patients searching for new therapeutic options,” said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. “The cemsidomide data presented at the ASH Annual Meeting in December support a potentially best-in-class profile and we are preparing for the next phase of development of this molecule. We continue to progress the CFT1946 Phase 1/2 study and will leverage data from the tumor specific cohorts to determine the development path for this program. In addition, we expect data from the CFT8919 Phase 1 dose escalation study run by our partner Betta Pharmaceuticals in China, which will define its potential for non-small cell lung cancer patients with the EGFR L858R mutation. We are excited about the degrader rationale for these programs, which we believe have the potential to deliver value for patients, caregivers, physicians and shareholders.” ANTICIPATED 2025 MILESTONES Cemsidomide: Cemsidomide is an oral degrader of IKZF1/3 for the potential treatment of relapsed/refractory (R/R) multiple myeloma (MM) and R/R non-Hodgkin’s lymphoma (NHL). Multiple Myeloma Enable initiation of the next phase of clinical development to investigate cemsidomide in combination with dexamethasone in the late-line setting, and in combination with other MM agents for earlier lines of treatment. These new studies are expected to initiate in early 2026. Complete Phase 1 dose escalation and present data in the second half of 2025. Non-Hodgkin’s Lymphoma Complete Phase 1 dose escalation and present data in the second half of 2025. Open expansion cohort(s) of the current Phase 1/2 trial in patients with peripheral T-cell lymphoma (PTCL) in the second half of 2025. Enable initiation of the next phase of clinical development to investigate cemsidomide monotherapy in later lines of therapy in PTCL. This new study is expected to initiate in early 2026. CFT1946: CFT1946 is an oral degrader targeting BRAF V600 mutations for the potential treatment of solid tumors including colorectal cancer (CRC), melanoma and other malignancies with V600 mutations. Complete monotherapy Phase 1 dose escalation in BRAF V600 mutant solid tumors in the first half of 2025. Generate data from the Phase 1 cohorts exploring monotherapy CFT1946 in melanoma, CFT1946 in combination with cetuximab in CRC and CFT1946 in combination with trametinib in melanoma. Data from these cohorts will define and enable the next phase of development. Present Phase 1 data in the second half of 2025. These presentations will include: (1) monotherapy in BRAF V600 mutant solid tumors, (2) monotherapy expansion cohorts in melanoma, and (3) in combination with cetuximab in CRC. CFT8919: CFT8919 is an oral degrader targeting EGFR bearing an oncogenic L858R mutation for the potential treatment of non-small cell lung cancer (NSCLC). Evaluate data from the Phase 1 dose escalation study in Greater China, which is led by partner Betta Pharmaceuticals, in patients with locally or advanced metastatic NSCLC harboring an EGFR L858R mutation. These data will be used to determine the next phase of development. Discovery: C4T will continue to utilize its TORPEDO® platform to develop orally bioavailable degraders for oncology and non-oncology targets for internal research and collaboration programs. To further highlight its deep expertise in drug discovery, C4T plans to: Present and publish preclinical work from its internal pipeline and TORPEDO® platform. Advance internal and collaboration programs to key milestones. 2024 ACCOMPLISHMENTS Cemsidomide: C4T advanced the Phase 1/2 clinical trial and delivered data reinforcing the potential of cemsidomide to become a backbone therapy of choice in MM and NHL where IKZF1/3 degradation is warranted. Multiple Myeloma At ASH, presented data on cemsidomide in combination with dexamethasone. As of the data cutoff date of October 11, 2024, the dose level exploring 75 µg once daily (QD) achieved an overall response rate (ORR) of 36 percent. Cemsidomide was well-tolerated across all dose levels. The maximum tolerated dose has not yet been reached. Patients are enrolling in the 100 µg QD cohort. Non-Hodgkin’s Lymphoma At ASH, presented data on cemsidomide monotherapy. As of the data cutoff date of October 11, 2024, cemsidomide demonstrated a 38 percent ORR across all subtypes and doses studied. In PTCL, cemsidomide achieved a 44 percent ORR and a 25 percent complete metabolic response rate. The maximum tolerated dose has not yet been reached. Patients are enrolling in the 75 µg QD cohort. CFT1946: C4T advanced the Phase 1/2 clinical trial across multiple arms and delivered monotherapy data demonstrating proof of mechanism and early evidence of proof of concept. At the European Society of Medical Oncology (ESMO) Congress, presented monotherapy data demonstrating CFT1946 is well tolerated across all dose levels and demonstrates initial signs of anti-tumor activity across all dose levels. At the TPD Summit, presented new preclinical data demonstrating CFT1946 has the ability to cross the blood-brain barrier, with Kpu,u values in the range of 0.34 to 0.88. Progressed the Phase 1 monotherapy dose escalation study. Began enrolling patients across multiple exploratory cohorts: CFT1946 monotherapy in melanoma, CFT1946 in combination with trametinib in melanoma, and CFT1946 in combination with cetuximab in CRC. CFT8919: Betta Pharmaceuticals, with C4T support, initiated the Phase 1 clinical trial of CFT8919 in Greater China. Discovery: C4T further validated its TORPEDO® platform and advanced key research efforts. Delivered two development candidates for non-oncology targets to collaborator Biogen. Established a new collaboration with Merck KGaA, Darmstadt, Germany focused on two critical oncogenic proteins. Continued to progress its internal discovery portfolio of orally bioavailable degraders. Corporate: C4T further strengthened its leadership across its management team and Board of Directors to supports its evolution into a fully integrated biotechnology company. Paige Mahaney, Ph.D., was appointed chief scientific officer. Dr. Mahaney is an experienced drug developer who has helped leading pharmaceutical companies build clinical portfolios across a wide range of disease indications and treatment modalities. C4T continued to evolve its governance by appointing three new members to its Board of Directors who bring deep experience across drug discovery, commercialization and lifecycle management. Cash Guidance C4T expects that its cash, cash equivalents and marketable securities as of December 31, 2024, together with anticipated collaboration expense reimbursements, but excluding any collaboration option or milestone payments, will enable the company to fund its operating plan into 2027. JP Morgan Presentation C4T will present at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025 at 2:15 pm PST (5:15 pm EST). A live webcast will be available under “Events & Presentations” in the Investors section of the company’s website at www.c4therapeutics.com. A replay of the webcast will be archived on the C4T website for at least two weeks following the presentation. About C4 Therapeutics C4 Therapeutics (C4T) (Nasdaq: CCCC) is a clinical-stage biopharmaceutical company dedicated to delivering on the promise of targeted protein degradation science to create a new generation of medicines that transforms patients’ lives. C4T is progressing targeted oncology programs through clinical studies and leveraging its TORPEDO® platform to efficiently design and optimize small-molecule medicines to address difficult-to-treat diseases. C4T’s degrader medicines are designed to harness the body’s natural protein recycling system to rapidly degrade disease-causing proteins, offering the potential to overcome drug resistance, drug undruggable targets and improve patient outcomes. For more information, please visit www.c4therapeutics.com. About Cemsidomide Cemsidomide is an investigational, orally bioavailable small-molecule degrader designed to be a more potent and selective degrader of IKZF1/3, transcription factors that drive multiple myeloma (MM) and non-Hodgkin’s lymphomas (NHL), with unique pharmacokinetic properties. Clinical data has shown that cemsidomide is well-tolerated. In MM, cemsidomide displays evidence of anti-myeloma activity and immunomodulatory effects. In NHL, cemsidomide displays evidence of anti-lymphoma activity. More information may be accessed at www.clinicaltrials.gov (identifier: NCT04756726). About CFT1946 CFT1946 is an investigational, orally bioavailable brain penetrant small molecule degrader of BRAF V600 mutations in solid tumors currently being evaluated in a Phase 1/2 global clinical trial in patients refractory to BRAF inhibitors. CFT1946 is designed to be potent and selective against the BRAF V600 mutant form. Initial clinical data from the Phase 1 trial demonstrate that CFT1946 has a well-tolerated safety profile, demonstrates dose-dependent bioavailability and degradation of BRAF V600E protein, and demonstrates evidence of monotherapy anti-tumor activity. CFT1946 is the only degrader of BRAF V600 mutant solid tumors in clinical trials. More information about this trial may be accessed at www.clinicaltrials.gov (identifier: NCT05668585). About CFT8919 CFT8919 is an orally bioavailable allosteric degrader that is designed to be potent and selective against EGFR bearing an oncogenic L858R mutation. In preclinical studies, CFT8919 is active in in vitro and in vivo models of L858R driven non-small cell lung cancer. Importantly, CFT8919 retains full activity against additional EGFR mutations that confer resistance against approved EGFR inhibitors including L858R-C797S, L858R-T790M, and L858R-T790M-C797S. Forward-Looking Statements This press release contains “forward-looking statements” of C4 Therapeutics, Inc. within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements may include, but may not be limited to, express or implied statements regarding our ability to develop potential therapies for patients; the design and potential efficacy of our therapeutic approaches; the predictive capability of our TORPEDO® platform in the development of novel, selective, orally bioavailable BiDAC™ and MonoDAC™ degraders; the potential timing, design and advancement of our preclinical studies and clinical trials, including the potential timing for and receipt of regulatory authorization related to clinical trials and other clinical development activities including clinical trial commencement or cohort initiation; our ability and the potential to successfully manufacture and supply our product candidates for clinical trials; our ability to replicate results achieved in our preclinical studies or clinical trials in any future studies or trials; our ability to replicate interim or early-stage results from our clinical trials in the results obtained when those clinical trials are completed or when those therapies complete later-stage clinical trials; regulatory developments in the United States and foreign countries; the anticipated timing and content of presentations of data from our clinical trials; and our ability to fund our future operations. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties related to the initiation, timing, advancement and conduct of preclinical and clinical studies and other development requirements for our product candidates; the risk that any one or more of our product candidates will cost more to develop or may not be successfully developed and commercialized; and the risk that sufficient capital to fund our future operations will be available to us on acceptable terms or at the times required. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in C4 Therapeutics’ most recent Annual Report on Form 10-K and/or Quarterly Report on Form 10-Q, as filed with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and C4 Therapeutics undertakes no duty to update this information unless required by law. Contacts: Investors: Courtney Solberg Senior Manager, Investor Relations CSolberg@c4therapeutics.com Media: Loraine Spreen Senior Director, Corporate Communications & Patient Advocacy LSpreen@c4therapeutics.com

Phase 1Executive ChangeClinical Result

100 Deals associated with Trametinib dimethyl sulfoxide

External Link

KEGG	Wiki	ATC	Drug Bank
D10176	Trametinib dimethyl sulfoxide	L01XE25	DB08911

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
BRAF Fusion Glioma	Japan	Novartis Pharma AG	24 Sep 2024
Low grade glioma	European Union	Novartis Europharm Ltd.	05 Jan 2024
Low grade glioma	Iceland	Novartis Europharm Ltd.	05 Jan 2024
Low grade glioma	Liechtenstein	Novartis Europharm Ltd.	05 Jan 2024
Low grade glioma	Norway	Novartis Europharm Ltd.	05 Jan 2024
BRAF mutation positive hairy cell leukemia	Japan	Novartis Pharma AG	24 Nov 2023
BRAF mutation Solid Tumors	Japan	Novartis Pharma AG	24 Nov 2023
BRAF V600E mutation-positive low grade glioma	United States	Novartis Pharmaceuticals Corp.	16 Mar 2023
BRAF Mutation Non-small Cell Lung Cancer	Japan	Novartis Pharma AG	28 Mar 2016
BRAF mutation positive Melanoma	Japan	Novartis Pharma AG	28 Mar 2016
BRAF V600E Mutation-Positive Solid Tumors	South Korea	Novartis Korea Ltd.	01 Oct 2015
BRAF V600 Mutation-Positive Non-small Cell Lung Cancer	European Union	Novartis Europharm Ltd.	30 Jun 2014
BRAF V600 Mutation-Positive Non-small Cell Lung Cancer	Iceland	Novartis Europharm Ltd.	30 Jun 2014
BRAF V600 Mutation-Positive Non-small Cell Lung Cancer	Liechtenstein	Novartis Europharm Ltd.	30 Jun 2014
BRAF V600 Mutation-Positive Non-small Cell Lung Cancer	Norway	Novartis Europharm Ltd.	30 Jun 2014
BRAF V600 mutation low-grade glioma	Australia	Novartis Pharmaceuticals Australia Pty Ltd.	14 Feb 2014
BRAF V600 mutation-positive Melanoma	Australia	Novartis Pharmaceuticals Australia Pty Ltd.	14 Feb 2014
BRAF V600E mutant Non-small Cell Lung Cancer	Australia	Novartis Pharmaceuticals Australia Pty Ltd.	14 Feb 2014
BRAF V600E Mutation-Positive Anaplastic Thyroid Cancer	Australia	Novartis Pharmaceuticals Australia Pty Ltd.	14 Feb 2014
High grade glioma	Australia	Novartis Pharmaceuticals Australia Pty Ltd.	14 Feb 2014

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Glioma	Phase 3	United States	Novartis AG	10 Jun 2022
Differentiated Thyroid Gland Carcinoma	Phase 3	United States	Novartis Pharmaceuticals Corp.	15 Nov 2021
Differentiated Thyroid Gland Carcinoma	Phase 3	China	Novartis Pharmaceuticals Corp.	15 Nov 2021
Differentiated Thyroid Gland Carcinoma	Phase 3	Argentina	Novartis Pharmaceuticals Corp.	15 Nov 2021
Differentiated Thyroid Gland Carcinoma	Phase 3	Brazil	Novartis Pharmaceuticals Corp.	15 Nov 2021
Differentiated Thyroid Gland Carcinoma	Phase 3	Canada	Novartis Pharmaceuticals Corp.	15 Nov 2021
Differentiated Thyroid Gland Carcinoma	Phase 3	India	Novartis Pharmaceuticals Corp.	15 Nov 2021
Differentiated Thyroid Gland Carcinoma	Phase 3	Malaysia	Novartis Pharmaceuticals Corp.	15 Nov 2021
Differentiated Thyroid Gland Carcinoma	Phase 3	Taiwan Province	Novartis Pharmaceuticals Corp.	15 Nov 2021
Differentiated Thyroid Gland Carcinoma	Phase 3	Turkey	Novartis Pharmaceuticals Corp.	15 Nov 2021

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03668431 (NEWS) Manual	Phase 2	BRAF V600E mutant Colorectal Cancer BRAF V600E	37	Dabrafenib + Trametinib + Spartalizumab	htqarrhuoz(wcgdmwrzum) = nqxlatzdvp ceopltxzgc (zsyqtdbwhm ) View more	Positive	01 Mar 2025
NCT03148275 (SARC033, CTgov)	Phase 2	Epithelioid Hemangioendothelioma	44	Questionnaire Administration+Trametinib	ofimwoiclb = pkyfqwfjrq nudrmrnrtt (hjwvwddgrc, zzemmnbjff - bfnnidhufk) View more	-	26 Nov 2024
NCT02974725 (Pubmed \| Lung Cancer) Manual	Phase 1	NRAS Mutant Melanoma \| KRAS mutant Non-small Cell Lung Cancer \| BRAF Mutation Non-small Cell Lung Cancer KRAS Mutation \| BRAF Mutation \| NRAS Mutation	216	Naporafenib + Rineterkib	fixgfkvotq(rcrckrygks) = tdhzzufruf tianhfncch (iymvvenriw ) View more	Negative	01 Nov 2024
				Naporafenib + Trametinib	fixgfkvotq(rcrckrygks) = sgoixossuk tianhfncch (iymvvenriw ) View more
NCT04310397 (CTgov)	Phase 2	BRAF V600K Mutation-Positive Melanoma \| Melanoma, Cutaneous Malignant	4	Spartalizumab+Dabrafenib+trametinib	fqlrsdbzid(wroorbusaa) = steqjmnxgq sozudqygdu (qilyjpvdjn, ndzllhyyfh - amerkgisms) View more	-	30 Oct 2024
P18.17.B (EANO2024)	Not Applicable	Recurrent Glioblastoma BRAFV600E mutation \| NRTK fusion	17	Dabrafenib-trametinib	cmdezatyhx(mrlxfncprv) = No grade 3-4 drug-related adverse events were observed in either subgroups; in any case target therapy was interrupted for toxicity dvuhmvdlxc (lfzfvajmod )	Positive	17 Oct 2024
				Larotrectinib
TRAIN (EANO2024)	Phase 2	Plexiform Neurofibroma	30	Trametinib 2 mg daily	hixuvextct(jjqrlxmtef) = gffjgctryd pooggxiwfx (yafkcwvhjp )	Positive	17 Oct 2024
NCT02097225 (Cancer Therapy Evaluation Program study 9557, CTgov)	Phase 1	BRAF V600E Mutation-Positive Melanoma \| Melanoma, Cutaneous Malignant \| Metastatic Solid Tumor ... View more	22	Dabrafenib+Trametinib+Onalespib (Treatment (Dabrafenib, Trametinib, Onalespib))	mqnmuntgia = caxualctet uzrgaasbch (ewvvslulsh, yobrdmzgvz - cjlkrmodoe) View more	-	16 Oct 2024
				Dabrafenib+Trametinib+Onalespib (Dose Level 1: Dabrafenib = 150 mg, Trametinib = 1 mg, Onalespib = 180 mg/m2)	vyyyyfdwau = pwpdhgijmd gnldqzhhle (hdovoucjym, pgwhixvxcn - wfkufxntxf) View more
ESMO2024 Manual	Not Applicable	Differentiated Thyroid Gland Carcinoma Second line \| First line BRAFV600E mutation	37	Dabrafenib trametinibinib (D+T)	ypngnaojvo(luorduwjqm) = lfszaqheow vuyubyweke (bpcbdenduo, 49.2 - 95.3) View more	Positive	16 Sep 2024
				Dabrafenib+trametinib (≥ 2nd line)	ypngnaojvo(luorduwjqm) = zbuyszoozf vuyubyweke (bpcbdenduo, 16.3 - 61.6) View more
ESMO2024 Manual	Not Applicable	Recurrent Glioblastoma BRAF V600E	17	Dabrafenib-trametinib	njefwkenbk(nvotkjvmxk) = None btgibaywhy (zijefwrseh )	Positive	16 Sep 2024
				Larotrectinib
NCT04544111 (ESMO2024) Manual	Phase 2	Refractory Thyroid Gland Carcinoma	19	Trametinib	fsreoopolb(kasospkodq) = ayhgiutsas ghtrdqsdaq (bngiezntga ) View more	Positive	16 Sep 2024
				dabrafenib+PDR001 (BRAFV600E- tumors after progression on BRAF-directed therapy)	fsreoopolb(kasospkodq) = mbigftqvvh ghtrdqsdaq (bngiezntga ) View more

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