Delving into the Latest Updates on Mirdametinib with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Mirdametinib (USAN), PD 901, PD-0325901

+ [3]

Target

MEK1 x MEK2

Action

inhibitors

Mechanism

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 DiseasesCongenital Disorders+ [6]

Active Indication

Neurofibromatosis 1NF1 mutant Plexiform NeurofibromaDedifferentiated Liposarcoma+ [11]

Inactive Indication

Advanced breast cancerAdvanced cancerAdvanced Lung Non-Small Cell Carcinoma+ [4]

Originator Organization

Springworks Therapeutics, Inc.

Active Organization

BeiGene Ltd.

Springworks Therapeutics, Inc.SPRINGWORKS THERAPEUTICS IRELAND LTD

Inactive Organization

Pfizer Inc.

License Organization

SpringWorks Therapeutics LLCMerck KGaA

Springworks Therapeutics, Inc.

+ [2]

Drug Highest PhaseApproved

First Approval Date

United States (11 Feb 2025),

Neurofibromatosis 1

RegulationFast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Priority Review (United States)

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Structure/Sequence

Molecular FormulaC16H14F3IN2O4

InChIKeySUDAHWBOROXANE-SECBINFHSA-N

CAS Registry391210-10-9

Background:
Multiple myeloma (MM) is a type of blood cancer that affects a person s immunity. MM returns after treatment (relapse) in almost all people; MM may also not respond to initial treatment (refractory). Many people with relapsed refractory MM (RRMM) also have changes in their KRAS and NRAS genes. Researchers want to try a new drug treatment that targets cancer with these changed genes.
Objective:
To test 2 drugs (mirdametinib and sirolimus) in people with RRMM.
Eligibility:
People aged 18 and older with RRMM who have changes in their KRAS or NRAS genes.
Design:
Participants will be screened. They will have blood tests and imaging scans. They will have an eye exam and a test of their heart function. They will need to provide proof of their disease status and of their KRAS or NRAS status. If neither is available, the tests will be repeated.
Participants will have a bone marrow biopsy: A needle will be inserted into a hipbone to draw out some soft tissue.
This study will be done in two parts. In the first part of this study, we will find a safe dose of mirdametinib combined with sirolimus. In the second part, we will learn more about how mirdametinib combined with sirolimus may work against RRMM.
Mirdametinib (capsules) and sirolimus (tablets) are taken by mouth. Participants will take both drugs at home on a 4-week cycle. They will take mirdametinib twice a day for the first 3 weeks of each cycle. They will take sirolimus once a day, every day, during each cycle.
Participants will have study visits once a week during the first cycle, and then on the first day of subsequent cycles. Blood, heart, imaging scans, and other tests will be repeated.
Treatment with the study drugs will go on for 1 year. Then participants will have follow-up visits every 3 months for 4 more years.

Start Date28 May 2025

Sponsor / Collaborator

National Cancer Institute

NCT06666348

/ Not yet recruitingPhase 1/2

A Phase 1/2 Study of Mirdametinib and Vinblastine for Newly Diagnosed or Previously Untreated Patients With Pediatric Low-grade Glioma and Activation of the MAPK Pathway

This is a 3-part open-label study (feasibility phase, treatment phase and follow-up phase) of orally administered mirdametinib in combination with intravenous vinblastine chemotherapy in patients with PLGG with activation of MAPK pathway.
Feasibility Phase:
The maximum tolerated/recommended phase 2 dose (MTD/RP2D) of the mirdametinib plus vinblastine combination will be assessed using a modified Rolling-6 design.
Treatment Phase:
Patients will receive mirdametinib twice daily (continuously) at a fixed dose (2mg/m2 po BID up to 4 mg BID) for a total of 13 cycles (28 days cycle). Weekly intravenous vinblastine at MTD will be given for a total of 17 cycles. If adverse events occur, two dose reductions are allowed.
Follow-up Phase:
Following the end of treatment, patients will be scheduled for a follow-up visit every 6 months for 36 months to evaluate PFS, TTP and OS.

Start Date01 Apr 2025

Sponsor / Collaborator

C17 Council

NCT06843967

/ RecruitingPhase 1/2IIT

A Phase Ib/II Study of the Mirdametinib in Combination With Palbociclib in Patients With Advanced Dedifferentiated Liposarcoma

The purpose of this study is to find out whether mirdametinib in combination with palbociclib is an effective and safe treatment for people with metastatic, recurrent, and unresectable liposarcoma. This study will test different doses of mirdametinib in combination with a fixed dose of palbociclib to find the best safe dose for further testing.

Start Date19 Feb 2025

Sponsor / Collaborator

Memorial Sloan Kettering Cancer Center

[+1]

100 Clinical Results associated with Mirdametinib

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100 Translational Medicine associated with Mirdametinib

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100 Patents (Medical) associated with Mirdametinib

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986

Literatures (Medical) associated with Mirdametinib

01 Jun 2025·Animal Bioscience

Key events in the process of sex determination and differentiation in early chicken embryos

Article

Author: Gong, Wei ; Sun, Hongyan ; Li, Zeyu ; Qian, Hongwu ; Liu, Xin ; Zuo, Qisheng ; Li, Bichun ; Han, Wei ; Song, Jiuzhou ; Liu, Guanzheng ; Niu, Yingjie ; Chen, Guo Hong ; Zhu, Xilin ; Lv, Xiaoqian ; Jin, Kai ; Wu, Jun ; Sun, Changhua

Objective: Current understanding of sex determination and differentiation mechanisms during early chicken embryonic development remains incomplete. To address this, we applied RNA sequencing to identify male-female expression differences at critical developmental stages (E0 blastocysts, E3.5-E6.5 genital ridges, E18.5 gonads), focusing on glycolysis, histone acetylation, and DNA methylation. This approach aims to unravel key regulatory mechanisms and advance developmental biology insights.Methods: We analyzed molecular mechanisms of chicken sex determination at key stages (E0 blastocysts, E3.5-E6.5 genital ridges, E18.5 gonads) using RNA sequencing. Glycolysis, histone acetylation, and DNA methylation levels were assessed in embryonic stem cells and chicken embryonic fibroblasts. E18.5 gonads were treated with glycolytic activators (SB431542 and PD0325901 [2i]), a DNA demethylation activator (Vitamin C [Vc]), or an inhibitors of histone acetylation modification (valproic acid [VPA]). Sex-related gene expression, hormone levels, and gonad morphology were evaluated to determine treatment effects.Results: Key findings revealed that sex differences emerged as early as the blastocyst stage, intensified with embryonic development and were marked by a surge in sexually dimorphic gene expression. Gene Ontology and Kyoto encyclopedia of genes and genomes analyses highlighted the pivotal roles of energy metabolism and epigenetic modification process during this critical period. 2i, VC, or VPA interventions targeting E18.5 embryo gonads, induced a remarkable transformation of ovarian tissue into a testis-like structure, characterized by cortical thinning, medulla densification, downregulation of female-specific genes (FOXL2, WNT4), upregulation of male-specific genes (SOX9, AMH), and increased testosterone secretion. This phenotypic and molecular shift underscores the ability of metabolic and epigenetic modulators to reprogram ovarian development towards a male-like pattern, preserving male sexual characteristics.Conclusion: Our study establishes energy metabolism and epigenetic regulation as central drivers of avian sex determination. These findings advance understanding of vertebrate developmental biology and provide a framework for dissecting regulatory networks in avian sexual development.

01 Jun 2025·PHARMACOLOGICAL RESEARCH

Properties of FDA-approved small molecule protein kinase inhibitors: A 2025 update

Review

Author: Roskoski, Robert

Because of the deregulation of protein kinase action in many inflammatory diseases and cancer, the protein kinase family has become one of the most significant drug targets in the 21st century. There are 85 FDA-approved protein kinase antagonists that target about two dozen different enzymes and four of these drugs were approved in 2024 and a fifth was approved in 2025. Of these drugs, five target dual specificity protein kinases (MEK1/2), fourteen inhibit protein-serine/threonine protein kinases, twenty-one block nonreceptor protein-tyrosine kinases, and 45 target receptor protein-tyrosine kinases. The data indicate that 75 of these drugs are prescribed for the treatment of neoplasms. Seven drugs (abrocitinib, baricitinib, deucravacitinib, deuruxolitinib, ritlecitinib, tofacitinib, upadacitinib) are prescribed for the management of inflammatory diseases (atopic dermatitis, rheumatoid arthritis, psoriasis, alopecia areata, and ulcerative colitis). Of the 85 FDA-approved agents, about two dozen are used in the treatment of multiple diseases. The following four drugs received FDA approval in 2024 - deuruxolitinib (alopecia areata), ensartinib and lazertinib (non-small cell lung cancer), and tovorafenib (pediatric glioma) while mirdametinib was approved in 2025 for the treatment of type I neurofibromatosis (von Recklinghausen disease). Apart from netarsudil, temsirolimus, and trilaciclib, the approved protein kinase blockers are orally bioavailable. This article summarizes the physicochemical properties of all 85 FDA-approved small molecule protein kinase inhibitors including the molecular weight, number of hydrogen bond donors/acceptors, ligand efficiency, lipophilic efficiency, polar surface area, and solubility. A total of 39 of the 85 FDA-approved drugs have a least one Lipinski rule of 5 violation.

01 May 2025·BIOCHEMICAL PHARMACOLOGY

Basroparib overcomes acquired resistance to MEK inhibitors by inhibiting Wnt-mediated cancer stemness in KRAS-mutated colorectal cancer

Article

Author: Kim, Jae-Sung ; Kim, Song Hyun ; Kim, Dong Young ; Kim, Ye-Hyun ; Kim, Kyungjin ; Kwon, Young-Ju ; Kim, Uk-Il

Mitogen-activated protein kinase (MEK) inhibitors show promise in treating KRAS-mutated cancers, including colorectal cancer (CRC). However, acquired resistance to MEK inhibitors often results in failure of effective treatment in patients with KRAS-mutated CRC. Here, we demonstrated that basroparib overcomes MEK inhibitor resistance in KRAS-G12V- or -G12D-mutated CRC. This basroparib-mediated sensitization was dependent on the KRAS mutation status and was enhanced specifically in KRAS-G12V- or -G12D-mutated cells. Additionally, when combined with MEK inhibitors, basroparib significantly reduced tumor growth in KRAS-G12V-mutated CRC xenograft models, but not in KRAS-G13D-mutated and -wild-type models. Furthermore, basroparib sensitized KRAS-G12V-mutated CRC cells with acquired resistance to MEK inhibitors. Notably, when combined with MEK inhibitors, basroparib not only suppressed tumor regrowth but also prolonged the survival of tumor models with acquired resistance to MEK inhibitors. Mechanistically, basroparib suppressed Wnt-mediated cancer stemness, a bypass mechanism for acquired resistance to MEK inhibitors in KRAS-mutated CRC. This study provides the first preclinical evidence of the relevance of basroparib in treating CRC with acquired resistance to MEK inhibitors due to APC and KRAS mutations, possibly by reducing the Wnt-mediated cancer stemness.

79

News (Medical) associated with Mirdametinib

23 May 2025

·firstwordpharma.com

Blenrep comeback continues with EU recommendation, plus a thumbs up for Itovebi

GSK's bid to bring back multiple myeloma therapy Blenrep (belantamab mafodotin) got a major boost on Friday when the antibody-drug conjugate (ADC) received a positive opinion from the EU's drug advisory panel. Also receiving a positive vote from the European Medicine Agency's Committee for Medicinal Products for Human Use (CHMP) was Roche's breast cancer drug Itovebi (inavolisib). Survival data for the PI3K inhibitor was recently spotlighted in an abstract released ahead of the American Society of Clinical Oncology (ASCO) meeting (see – ASCO25 abstract roundup: Roche's Itovebi cuts risk of death).Autolus' CAR-T cell therapy Aucatzyl (obecabtagene autoleucel) also received a thumbs up from CHMP, as well as mirdametinib, a MEK inhibitor recently acquired by Merck KGaA in its $3.9-billion takeout of SpringWorks Therapeutics. Blenrep is backBlenrep was withdrawn from global markets in 2022 following disappointing results from a confirmatory trial, but after positive readouts from two pivotal Phase III trials — DREAMM-7 and DREAMM-8 — GSK has been working to bring the BCMA-targeted ADC back. Specifically, CHMP recommended Blenrep to treat relapsed or refractory multiple myeloma in combination with bortezomib and dexamethasone (BVd) for adults who have received at least one prior therapy; and in combination with Bristol Myers Squibb's Pomalyst (pomalidomide) plus dexamethasone for patients who have received at least one prior treatment, including BMS's Revlimid (lenalidomide). In DREAMM-7, Blenrep plus BVd achieved a progression-free survival (PFS) of 36.6 months, versus 13.4 months for the comparator regimen of Johnson & Johnson's Darzalex (daratumumab) plus BVd.DREAMM-8 compared Blenrep to bortezomib, both in combination with Pomalyst and dexamethasone. At nearly 22 months follow-up, median PFS was not yet reached with Blenrep compared to 12.7 months in the control arm.In April, the UK became the first country to reauthorise Blenrep. Regulatory applications for the ADC are under review in a dozen other countries, including the US, Switzerland, Japan, China and Canada. The FDA is expected to make a decision by July 23, although there is concern that recent mass layoffs at the FDA could slow down the review process (see – Spotlight On: Could key drug approvals be put at risk of delay by FDA layoffs?).GSK is positioning the drug as a key growth driver, projecting peak annual sales exceeding £3 billion (see – Spotlight On: Can GSK deliver much needed pipeline momentum?).Itovebi impressesItovebi scored a positive opinion for its use in combination with palbociclib and fulvestrant to treat PIK3CA‑mutated, ER‑positive/HER2‑negative, locally advanced or metastatic breast cancer, following recurrence on or within 12 months of completing adjuvant endocrine treatment.The PI3K inhibitor won a similar approval in the US last October based on data from the Phase III INAVO120 study showing a 57% benefit for the Itovebi triplet on the primary endpoint of PFS compared to palbociclib and fulvestrant alone. Patients achieved PFS of 15 months in the experimental arm versus 7.3 months for placebo.At ASCO, Roche plans to share updated survival data for Itovebi. The abstract revealed that patients who received the triplet experienced a median overall survival of 34 months compared to 27 months for controls, representing a 33% reduction in the risk of death. Additionally, the combination therapy delayed the time to chemotherapy from 12.6 months in the control arm to 35.6 months with Itovebi.Go-ahead for Aucatzyl, mirdametinibA CHMP recommendation also went to Aucatzyl to treat patients aged 26 and older with relapsed or refractory B cell precursor acute lymphoblastic leukaemia (ALL).Autolus' CD19-directed cell therapy was cleared by the FDA in November for a similar indication, based on data from the Phase Ib/II FELIX trial. Of 65 patients with ALL who received the CAR-T, 27 (42%) achieved a complete response (CR) within three months, with a median CR duration of 14.1 months.Meanwhile, Merck KGaA's newly gained mirdametinib received a positive opinion to treat symptomatic, inoperable plexiform neurofibromas (PN) in paediatric and adult patients with neurofibromatosis type 1 (NF1) aged 2 years and above. If fully approved by the European Commission, it will have the brand name of Ezmekly.In February, the MEK inhibitor was approved in the US for the same patient population, where it's marketed as Gomekli.

Clinical ResultDrug ApprovalPhase 3AcquisitionASCO

28 Apr 2025

·www.fiercepharma.com

Merck KGaA springs back into M&A game with $3.9B buyout of Pfizer spinout SpringWorks

Germany's Merck KGaA has bought out rare disease specialist SpringWorks for $3.9 billion. Connecticut-based SpringWorks is a 2017 spin out of Pfizer. Merck KGaA’s long courtship of SpringWorks Therapeutics has come to fruition as the German drugmaker has bought out the Connecticut biopharma for an equity value of $3.9 billion, the companies said on Monday.The deal, which works out to $47 per share, enhances Merck KGaA’s presence in the U.S. and bolsters its oncology portfolio, as SpringWorks brings two approved drugs and a handful of rare disease pipeline candidates. When word of the potential acquisition first surfaced in early February, SpringWorks’ share price shot up to $60 and its market cap rose from $3 billion to $4 billion. But as the rumors had cooled by early this month, SpringWorks’ value had returned to its previous level.Then last week came reports of renewed interest from the companies in pulling off the transaction.Merck KGaA said that it paid a premium of 26% based on SpringWorks’ volume-weighted price of $37.38 on February 7, which was the day prior to the initial market speculation about a potential deal.Over the last five days, as deal talks ginned back up, Merck KGaA’s share price has increased by 6%, while that of SpringWorks grew by 21%.In a statement, Merck KGaA CEO Belén Garijo called the buyout “a major step in our active portfolio strategy to position our company as a globally diversified, innovation and technology powerhouse.”Outside of Merck KGaA's electronics business, the SpringWorks deal represents the company's largest acquisition since its 2015 buyout of St. Louis manufacturing specialist Sigma-Aldrich for $17 billion. The German company also acquired Massachusetts manufacturer Millipore for $7 billion in 2010 and Swiss biotech Serono for $13.3 billion in 2006.SpringWorks traces its roots to 2017, when it was spun out of Pfizer with two drugs then in phase 3, which have since been approved. “We have successfully launched two best-in-class medicines in the United States, and with the aspiration to deliver our therapies worldwide, our journey is at a pivotal juncture,” Saqib Islam, CEO of SpringWorks, said in a release. In late 2023, SpringWorks earned an FDA nod for Ogsiveo, a treatment for ultra-rare desmoid tumors. The drug is off to a promising launch, achieving sales of $61 million in the fourth quarter and $172 million for 2024.Two months ago, SpringWorks gained FDA approval for MEK inhibitor Gomekli to treat neurofibromatosis type 1 (NF1), a rare genetic disease. It is the first treatment for adults and children with the disorder and is competing with AstraZeneca and Merck's (U.S.) drug Koselugo, which was approved for pediatric patients and rang up sales of $631 million in 2024. Pipeline candidates brought by SpringWorks include cancer drug brimarafenib, an RAF dimer inhibitor which the company is developing along with BeiGene, and SW-682, a TEAD inhibitor which is being investigated for Hippo-mutant solid tumors.Analysts at ODDO BHF gave the acquisition a thumbs up, writing to clients that it “brings to the healthcare business some positive prospects again.”

Drug ApprovalAcquisitionPhase 3

28 Apr 2025

·pharma.economictimes.indiatimes.com

Germany's Merck KGaA in $3.9 billion deal to buy US biotech firm SpringWorks

Frankfurt: Merck KGaA has struck a deal to buy U.S. biotech company SpringWorks Therapeutics for an equity value of $3.9 billion, as the German company seeks to acquire treatments for rare tumours to boost its cancer drugs business. The family-controlled company said in a statement on Monday the purchase price of $47 per share in cash represents an equity value of about $3.9 billion, equivalent to an enterprise value of $3.4 billion (3.0 billion euros), when SpringWorks' cash holdings are deducted. The deal is one of the largest in years for Merck, but its price tag was 22 per cent lower than the about $60 per share analysts had expected after a Reuters report on February 10 that the companies were in advanced talks. Merck on April 24 lowered price expectations, saying the two companies were in late-stage discussions over a bid of around $47 per SpringWorks share. Merck's shares were down 0.4 per cent in early trade, underperforming the broader German stock market. The deal will be funded with available cash and new debt. It is expected to be accretive to Merck's earnings per share, adjusted for special items, in 2027, said a statement from the German group, based in Darmstadt, near Frankfurt. It added it would still be able to pursue larger transactions. Stamford, Connecticut-based SpringWorks, which listed its shares in New York in 2019, develops drugs to treat cancer and rare types of tumour. It has two products on the market: Ogsiveo with 2024 sales of $172 million to treat desmoid tumours, an aggressive disease affecting soft tissue, as well as Gomekli, which was approved in February to treat NF1-PN, characterised by nerve sheath tumours. "We have the unique opportunity with SpringWorks to establish a leadership position in rare tumours and build a strong foundation for further investments in this area," Peter Guenter, head of healthcare at Merck, said. The transaction will likely close during the second half of 2025, subject to approval of SpringWorks' shareholders and regulatory clearance, Merck added. TRUMP DISRUPTION JP Morgan analyst Anupam Rama previously said the lack of other serious bidders and the challenging macroeconomic environment had probably lowered the valuation. This year had been expected to be stellar for mergers and acqusitions in the sector until the disruption caused by U.S. President Donald Trump's policies, including massive layoffs at the U.S. Food and Drug Administration. That has brought uncertainty to the drug approval process, hampering projected drug sales and affecting valuations. Merck is particularly keen to strengthen its drug development pipeline after high-profile setbacks in late-stage drug trials, including a decision last year to halt development of head and neck cancer drug Xevinapant. A major trial testing multiple sclerosis drug Evobrutinib failed in December 2023. In 2015, the company agreed to buy U.S. lab equipment supplier Sigma-Aldrich for $17 billion, its biggest deal to date. In 2019, Merck acquired U.S. electronics materials manufacturer Versum for around $6.5 billion. (Reporting by Ludwig Burger, Emma-Victoria Farr in Frankfurt and Sabrina Valle in New York; Editing by Kirsten Donovan and Barbara Lewis)

AcquisitionDrug Approval

100 Deals associated with Mirdametinib

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KEGG	Wiki	ATC	Drug Bank
D11675	-	-	DB07101

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Neurofibromatosis 1	United States	Springworks Therapeutics, Inc.	11 Feb 2025

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
NF1 mutant Plexiform Neurofibroma	NDA/BLA	United States	Springworks Therapeutics, Inc.	04 Mar 2024
Dedifferentiated Liposarcoma	Phase 2	United States	Springworks Therapeutics, Inc.	19 Feb 2025
Myxoid Liposarcoma	Phase 2	United States	Springworks Therapeutics, Inc.	19 Feb 2025
Advanced Malignant Solid Neoplasm	Phase 2	United States	Springworks Therapeutics, Inc.	03 Feb 2023
Advanced Malignant Solid Neoplasm	Phase 2	Australia	Springworks Therapeutics, Inc.	03 Feb 2023
Melanoma, Cutaneous Malignant	Phase 2	United States	Springworks Therapeutics, Inc.	03 Feb 2023
Melanoma, Cutaneous Malignant	Phase 2	Australia	Springworks Therapeutics, Inc.	03 Feb 2023
Low grade glioma	Phase 2	United States	Springworks Therapeutics, Inc.	21 Jun 2021
Colorectal Cancer	Phase 2	Netherlands	Pfizer Inc.	01 Jan 2014
KRAS mutant Non-small Cell Lung Cancer	Phase 2	Netherlands	Pfizer Inc.	01 Jan 2014

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03962543 (ReNeu, AAN2025)	Phase 2	NF1 mutant Plexiform Neurofibroma	-	Mirdametinib capsule	jphoqdbugf(slpuwrdxsy) = unygeijxwy bphcyyqafb (yoltstqolk, 29% - 55%) View more	Positive	07 Apr 2025
				Mirdametinib dispersible tablet	jphoqdbugf(slpuwrdxsy) = yiactitokl bphcyyqafb (yoltstqolk, 38% - 65%) View more
NCT03962543 (ReNeu, Pubmed \| J Clin Oncol)	Phase 2	NF1 mutant Plexiform Neurofibroma	-	Mirdametinib capsules	nyokaeatjo(pfmyiyqavv) = uqznxlwdtq uexibhjrpx (uombedfomo )	Positive	20 Feb 2025
				Mirdametinib tablets for oral suspension	nyokaeatjo(pfmyiyqavv) = avaxfxsyky uexibhjrpx (uombedfomo )
NCT04923126 (SJ901, SNO2024) Manual	Phase 1	Low grade glioma BRAF alteration \| FGFR1 alteration \| NF1 alteration ... View more	23	MIRDAMETINIB	xevmklfuyb(eehpsytxpv) = ywrftiysap kudfywnpmt (jkbcjkqzgs ) View more	Positive	11 Nov 2024
NCT03962543 (ReNeu, SNO2024) Manual	Phase 2	NF1 mutant Plexiform Neurofibroma	114	MIRDAMETINIB (adults)	uwdhkybgna(obaihwylro) = Clinically meaningful improvement was defined as change from baseline >9.7 points for adults, >8.5 points for child self-report, and >9.0 points for parent proxy-report. PedsQL-TS improvement (least-squares mean, LSM [SE] change) from baseline at C13 was 3.9 (1.6; P=.018; n=34) for adults, 4.0 (2.4; P=.096; n=38) for children by self-report, and 5.6 (1.9; P=.005; n=43) by parent proxy-report. PedsQL-TS improvements for adults and children by parent proxy-report (not by children self-report) were observed early (at C5 and C3, respectively) and sustained through C24. Clinically meaningful improvement in PedsQL-TS from baseline at C13 was achieved by 37% (10/27) adults, 45% (13/29) children by self-report, and 47% (15/32) children by parent proxy-report (among patients who could have achieved a clinically meaningful change from baseline). Significant (P<.05) improvement from baseline to C13 was reported for physical (adults, children, and parent proxy-report), school/work (adults), and emotional and social (parent proxy-report) subscales. cuxbcvakto (uthmdrrzre )	Positive	11 Nov 2024
				MIRDAMETINIB (children)
NCT03962543 (ReNeu, SNO2024 \| FDA_CDER) Manual	Phase 2	Neurofibromatosis 1	114	mirdametinib (adults)	lziqtlblob(wrcebnrnzx) = dxlliqqlpc tbxwdqduxs (ulrifjjlbe, 29 - 55) View more	Positive	11 Nov 2024
				mirdametinib (children)	lziqtlblob(wrcebnrnzx) = dhijvawjce tbxwdqduxs (ulrifjjlbe, 38 - 65) View more
NCT03962543 (ReNeu, SNO2024)	Phase 2	Plexiform Neurofibroma	12	MIRDAMETINIB (Prepubescent patients)	kklsnbwudo(kkgowjsoeh) = tzxvqysdmc immdztsqip (wwzoqbdhjr )	-	11 Nov 2024
NCT03962543 (RENEU, EANO2024)	Phase 2	NF1 mutant Plexiform Neurofibroma	114	Mirdametinib 2 mg/m^2 BID	agwohijrob(fuxeigkrej) = zkfdbrdsbk ghfvhfgphl (ajaofscjvc, 29 - 55) View more	Positive	17 Oct 2024
				Placebo	agwohijrob(fuxeigkrej) = njiqrbsvmo ghfvhfgphl (ajaofscjvc, 38 - 65) View more
NCT05054374 (CTgov)	Phase 1/2	Advanced Malignant Solid Neoplasm \| Metastatic breast cancer \| HER2-negative breast cancer	6	Fulvestrant+Mirdametinib (Arm 1, Part 1 - Mirdametinib in Combination With Fulvestrant)	hbuntjqkma(pbwtmdticn) = ognlhrdsqx xutvquciui (lqyysyrcca, jfcybmqoth - bpphhoaapb) View more	-	09 Jul 2024
				Fulvestrant+Mirdametinib (Arm 1, Part 2 - Mirdametinib in Combination With Fulvestrant)	hbuntjqkma(pbwtmdticn) = uuoglhxhio xutvquciui (lqyysyrcca, mxzduygzkw - avnwyyjjfy) View more
NCT03962543 (ReNeu, ASCO2024) Manual	Phase 2	NF1 mutant Plexiform Neurofibroma	114	Mirdametinib 2 mg/m2 BID	lfwvmrynrm(gflhcxvibv) = oiyggxxwec yumtijwvyd (pjgepkvjtk, 29 - 55) View more	Positive	24 May 2024
				Mirdametinib (pediatric)	lfwvmrynrm(gflhcxvibv) = ncssxhauao yumtijwvyd (pjgepkvjtk, 38 - 65) View more
NCT03962543 (ReNeu, GlobeNewswire) Manual	Phase 2	Plexiform Neurofibroma NF1	114	Mirdametinib (pediatric patients)	fetpcssvqi(zuahggxepa) = brqfpexphy aqbtfcxcwp (uioiwjpzyl ) View more	Positive	16 Nov 2023
				Mirdametinib (adult patients)	fetpcssvqi(zuahggxepa) = gafqklkpaz aqbtfcxcwp (uioiwjpzyl ) View more