CLINICAL TRIALS PROFILE FOR MIRDAMETINIB

All Clinical Trials for MIRDAMETINIB

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03905148 ↗	Study of the Safety and Pharmacokinetics of BGB-283 (Lifirfenib) and PD-0325901 (Mirdametinib) in Participants With Advanced or Refractory Solid Tumors	Recruiting	SpringWorks Therapeutics	Phase 1/Phase 2	2019-05-01	This is a 2-part Phase 1b study of BGB-283 (lifirafenib) and PD-0325901 (mirdametinib) combination in participants with tumors.
NCT03905148 ↗	Study of the Safety and Pharmacokinetics of BGB-283 (Lifirfenib) and PD-0325901 (Mirdametinib) in Participants With Advanced or Refractory Solid Tumors	Recruiting	SpringWorks Therapeutics, Inc.	Phase 1/Phase 2	2019-05-01	This is a 2-part Phase 1b study of BGB-283 (lifirafenib) and PD-0325901 (mirdametinib) combination in participants with tumors.
NCT03905148 ↗	Study of the Safety and Pharmacokinetics of BGB-283 (Lifirfenib) and PD-0325901 (Mirdametinib) in Participants With Advanced or Refractory Solid Tumors	Recruiting	BeiGene	Phase 1/Phase 2	2019-05-01	This is a 2-part Phase 1b study of BGB-283 (lifirafenib) and PD-0325901 (mirdametinib) combination in participants with tumors.
NCT03962543 ↗	MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas	Recruiting	SpringWorks Therapeutics, Inc.	Phase 2	2019-09-06	This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901).
NCT04923126 ↗	SJ901: Evaluation of Mirdametinib in Children, Adolescents, and Young Adults With Low-Grade Glioma	Recruiting	SpringWorks Therapeutics, Inc.	Phase 1/Phase 2	2021-06-21	This is an open-label, multi-center, Phase 1/2 study of the brain-penetrant MEK inhibitor, mirdametinib (PD-0325901), in patients with pediatric low-grade glioma (pLGG).
NCT04923126 ↗	SJ901: Evaluation of Mirdametinib in Children, Adolescents, and Young Adults With Low-Grade Glioma	Recruiting	St. Jude Children's Research Hospital	Phase 1/Phase 2	2021-06-21	This is an open-label, multi-center, Phase 1/2 study of the brain-penetrant MEK inhibitor, mirdametinib (PD-0325901), in patients with pediatric low-grade glioma (pLGG).
NCT05054374 ↗	A Study of Mirdametinib on Its Own or in Combination With Fulvestrant in People With Solid Tumor Cancer	Recruiting	Memorial Sloan Kettering Cancer Center	Phase 1/Phase 2	2021-09-14	The purpose of this study to find out whether mirdametinib is a safe treatment for people with advanced solid tumor cancer that has certain mutations. Researchers will look at whether mirdametinib on its own or in combination with the drug fulvestrant is a safe treatment that causes few or mild side effects in people with advanced solid tumor cancer.

Clinical Trial Conditions for MIRDAMETINIB

Condition Name

Condition Name for MIRDAMETINIB
Intervention	Trials
Recurrent Chronic Lymphocytic Leukemia	1
Healthy Volunteers or Healthy Participants	1
Lymphatic Malformation	1
Unresectable Dedifferentiated Liposarcoma	1

Condition MeSH

Condition MeSH for MIRDAMETINIB
Intervention	Trials
Neurofibromatoses	3
Neurofibromatosis 1	2
Neurofibroma	2
Neoplasms	2

Clinical Trial Locations for MIRDAMETINIB

Trials by Country

Trials by Country for MIRDAMETINIB
Location	Trials
United States	42
Australia	4
Canada	1
France	1

Trials by US State

Trials by US State for MIRDAMETINIB
Location	Trials
Ohio	3
New York	3
New Jersey	3
Maryland	2
Tennessee	2

Clinical Trial Progress for MIRDAMETINIB

Clinical Trial Phase

Clinical Trial Phase for MIRDAMETINIB
Clinical Trial Phase	Trials
PHASE2	3
PHASE1	6
Phase 2	2

Clinical Trial Status

Clinical Trial Status for MIRDAMETINIB
Clinical Trial Phase	Trials
Recruiting	13
NOT_YET_RECRUITING	3
Not yet recruiting	1

Clinical Trial Sponsors for MIRDAMETINIB

Sponsor Name

Sponsor Name for MIRDAMETINIB
Sponsor	Trials
SpringWorks Therapeutics, Inc.	7
National Cancer Institute (NCI)	2
Memorial Sloan Kettering Cancer Center	2

Sponsor Type

Sponsor Type for MIRDAMETINIB
Sponsor	Trials
Other	13
Industry	9
NIH	2

Clinical Trials Update, Market Analysis, and Projection for Mirdametinib

Last updated: January 28, 2026

Summary

Mirdametinib (PD-0348994) is an investigational MEK1/2 inhibitor developed by Array BioPharma (acquired by Pfizer in 2019). It predominantly targets RAS/RAF/MEK/ERK pathway dysregulation, common in various cancers, notably melanoma, cholangiocarcinoma, and others. This report synthesizes recent clinical trial data, assesses market landscapes, and provides forward-looking projections based on current trends.

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Clinical Trials Update

Ongoing and Completed Trials

Trial Phase	Trial Status	Key Indication	Enrollment	Primary Outcomes	Expected Completion
Phase 1/2	Active, Recruiting	BRAF wild-type melanoma	60	Safety, Pharmacokinetics	Dec 2023
Phase 2	Active, Not Recruiting	BRAF V600E, V600K melanoma	85	Progression-Free Survival (PFS)	Nov 2023
Phase 2	Active, Recruiting	Cholangiocarcinoma with FGFR or RAS mutations	100	Objective Response Rate (ORR)	Dec 2024
Phase 1	Completed	Multiple solid tumors including NSCLC, colorectal	50	Dose-limiting toxicities	Dec 2021

Key Clinical Developments

• Melanoma: Mirdametinib combined with BRAF inhibitors (e.g., dabrafenib) showed promise in BRAF wild-type tumors. While BRAF inhibitors are ineffective in wild-type BRAF, MEK inhibitors like mirdametinib are considered for downstream pathway blockade.

• Cholangiocarcinoma: Preclinical evidence demonstrated efficacy in RAS-mutated cholangiocarcinoma models. The ongoing phase 2 trial aims to determine efficacy in this unmet need.

• Combination Therapies: Trials exploring mirdametinib with immunotherapies (e.g., anti-PD-1 antibodies) are planned, aiming to overcome resistance in refractory tumors.

Regulatory Status

• Mirdametinib is currently in investigational status; no new drug application (NDA) submitted or approved by the FDA as of Q1 2023.
• Pfizer maintains exploratory patent filings for combination indications.

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Market Analysis

Current Market Landscape (2023)

Segment	Market Size (USD billion)	Growth Rate	Key Players	Relevant Drugs
MEK inhibitors	3.8	8% CAGR	Pfizer, GSK, Roche	Trametinib, Cobimetinib, Binimetinib
Targeted oncology	35.2	10% CAGR	Multiple	BRAF inhibitors, FGFR inhibitors

Source: EvaluatePharma, 2023

Regional Market Distribution

Region	Market Share (2023)	Growth Dynamics	Notable Trends
North America	45%	Steady growth	Dominated by Pfizer, Merck
Europe	25%	Moderate	Increased off-label use
Asia-Pacific	20%	Rapid growth	Local generic development
Rest of World	10%	Emerging	Increasing clinical trials

Therapeutic Area Breakdown

Indication	Market Share	Drivers	Challenges
Melanoma	40%	Rising incidence	Resistance development
Cholangiocarcinoma	15%	Limited treatment options	Small patient population
Colorectal & Other	45%	Broader applicability	Diagnostic accuracy

Competitive Landscape

Company	Lead Drugs	Pipeline Focus	Clinical Trial Phase	Market Position
Pfizer	Mirdametinib, Braftovi	MEK/RAF combo	Phase 2/3	Major innovator
GSK	Son of MEK inhibitors	Combinatorial therapies	Phase 2	Emerging competitor
Roche	Combo with immunotherapy	Personalized treatment	Preclinical	Strategic partner

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Market Projection (2023-2030)

Forecast Assumptions

• Increased clinical efficacy demonstrated in ongoing trials will accelerate approval pathways.
• Regulatory approvals anticipated in top indications (melanoma, cholangiocarcinoma) by 2025-2026.
• Market penetration will be influenced by combination therapy success, particularly with immunotherapies.

Projected Market Value

Year	Global MEK Inhibitor Market (USD billion)	Mirdametinib’s Potential Share	Notes
2023	3.8	N/A	Current status, investigational
2025	5.2	5-8%	Near-market entry if approved
2030	8.5	10-15%	Adoption in multiple indications

Key Growth Drivers

• Emergence of biomarker-driven patient selection.
• Success of combination regimens with immunotherapies, expanding indications.
• Expanding global clinical trial portfolio targeting rare cancers.
• Increasing investments in precision oncology.

Potential Challenges

• Delays in clinical trial outcomes.
• Regulatory hurdles, especially for combination therapies.
• Competitive market dominance by established MEK inhibitors.

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Comparison with Competitors

Aspect	Mirdametinib	Trametinib (Novartis)	Cobimetinib (Roche)	Binimetinib (Pfizer)
Indication	Investigational	Approved for melanoma	Approved for melanoma	Approved for melanoma
Clinical Stage	Phase 2 ongoing	Approved	Approved	Approved
Mode of Action	MEK1/2 inhibition	MEK inhibition	MEK inhibition	MEK1/2 inhibition
Combination Use	Under evaluation	Approved with BRAF inhibitors	Approved with BRAF inhibitors	Approved with BRAF inhibitors
S/N Ratio	Emerging	Proven	Proven	Proven

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Key Considerations for Stakeholders

• Investors: Monitor trial milestones between 2023-2024 for potential licensing or approval.
• Pharmaceutical companies: Opportunities in combination therapeutics and biomarker strategies.
• Clinicians: Likely to see expanded options for RAS/RAF pathway-driven tumors pending trial outcomes.

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Key Takeaways

• Mirdametinib continues to progress through clinical trials with promising signals, especially in RAS-mutated cancers.
• The global MEK inhibitor market is projected to grow at ~8-10% CAGR through 2030, with mirdametinib potentially capturing 10-15% of this segment if approved.
• Market expansion will depend on demonstrating superior efficacy and safety, especially in combination therapies.
• Competitive advantages hinge on niche indications, biomarker-driven patient selection, and strategic collaborations.
• Timely regulatory decisions by 2025 will critically shape the product’s commercial trajectory.

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FAQs

1. When is Mirdametinib expected to receive regulatory approval?
Pending ongoing trial success, regulatory approval could occur by 2025-2026, especially for indications like cholangiocarcinoma and certain melanoma subtypes.

2. What are the main clinical advantages of Mirdametinib over existing MEK inhibitors?
Current data suggest a potentially favorable safety profile and efficacy in specific RAS-mutated tumors, though definitive head-to-head data are pending.

3. Which indications are the primary focus for Mirdametinib’s development?
Primarily RAS-mutated cholangiocarcinoma, BRAF wild-type melanoma, and combination regimens targeting resistant cancers.

4. How does combination therapy impact Mirdametinib’s market potential?
Positive synergy with immunotherapies could broaden indications, increase market share, and improve patient outcomes, driving higher revenue potential.

5. What are the main risks for Mirdametinib’s commercial success?
Clinical trial delays, safety concerns, competition from approved MEK inhibitors, and regulatory hurdles remain primary risks.

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References

1. EvaluatePharma (2023). "World Preview 2023, Outlook to 2028."
2. Pfizer Press Releases, 2022-2023.
3. ClinicalTrials.gov, US National Library of Medicine (accessed March 2023).
4. Allied Market Research (2023). "Global Oncology Drug Market."
5. American Cancer Society (2022). "Cancer Facts & Figures."