CLINICAL TRIALS PROFILE FOR PEMFEXY

All Clinical Trials for Pemfexy

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02194738 ↗	Genetic Testing in Screening Patients With Stage IB-IIIA Non-small Cell Lung Cancer That Has Been or Will Be Removed by Surgery (The ALCHEMIST Screening Trial)	Recruiting	National Cancer Institute (NCI)	N/A	2014-08-18	This ALCHEMIST trial studies genetic testing in screening patients with stage IB-IIIA non-small cell lung cancer that has been or will be removed by surgery. Studying the genes in a patient's tumor cells may help doctors select the best treatment for patients that have certain genetic changes.
NCT02419495 ↗	Selinexor With Multiple Standard Chemotherapy or Immunotherapy Regimens in Treating Patients With Advanced Malignancies	Recruiting	Karyopharm Therapeutics, Inc	Phase 1	2015-06-26	This phase Ib trial studies the side effects and best dose of selinexor when given together with several different standard chemotherapy or immunotherapy regimens in treating patients with malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment (advanced). Selinexor may stop the growth of cancer cells by blocking enzymes needed for cell growth. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Studying selinexor with different standard chemotherapy or immunotherapy regimens may help doctors learn the side effects and best dose of selinexor that can be given with different types of treatments in one study.
NCT02419495 ↗	Selinexor With Multiple Standard Chemotherapy or Immunotherapy Regimens in Treating Patients With Advanced Malignancies	Recruiting	National Cancer Institute (NCI)	Phase 1	2015-06-26	This phase Ib trial studies the side effects and best dose of selinexor when given together with several different standard chemotherapy or immunotherapy regimens in treating patients with malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment (advanced). Selinexor may stop the growth of cancer cells by blocking enzymes needed for cell growth. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Studying selinexor with different standard chemotherapy or immunotherapy regimens may help doctors learn the side effects and best dose of selinexor that can be given with different types of treatments in one study.
NCT02419495 ↗	Selinexor With Multiple Standard Chemotherapy or Immunotherapy Regimens in Treating Patients With Advanced Malignancies	Recruiting	M.D. Anderson Cancer Center	Phase 1	2015-06-26	This phase Ib trial studies the side effects and best dose of selinexor when given together with several different standard chemotherapy or immunotherapy regimens in treating patients with malignancies that have spread to other places in the body and usually cannot be cured or controlled with treatment (advanced). Selinexor may stop the growth of cancer cells by blocking enzymes needed for cell growth. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Studying selinexor with different standard chemotherapy or immunotherapy regimens may help doctors learn the side effects and best dose of selinexor that can be given with different types of treatments in one study.
NCT02535325 ↗	Methoxyamine Hydrochloride, Pemetrexed Disodium, Cisplatin, and Radiation Therapy in Treating Patients With Stage IIIA-IV Non-small Cell Lung Cancer	Active, not recruiting	National Cancer Institute (NCI)	Phase 1	2015-09-30	This phase I trial studies the side effects and best dose of methoxyamine when given together with pemetrexed disodium, cisplatin, and radiation therapy in treating patients with stage IIIA-IV non-small cell lung cancer. Drugs used in chemotherapy, such as methoxyamine hydrochloride, pemetrexed disodium, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high-energy x-rays to kill tumor cells and shrink tumors. Giving methoxyamine hydrochloride together with pemetrexed disodium, cisplatin, and radiation therapy may kill more tumor cells.

Clinical Trial Conditions for Pemfexy

Condition Name

Condition Name for Pemfexy
Intervention	Trials
Stage IV Lung Cancer AJCC v8	10
Stage IIIC Lung Cancer AJCC v8	9
Stage IVB Lung Cancer AJCC v8	8
Stage IIIB Lung Cancer AJCC v8	8

Condition MeSH

Condition MeSH for Pemfexy
Intervention	Trials
Carcinoma, Non-Small-Cell Lung	22
Lung Neoplasms	19
Carcinoma	11
Adenocarcinoma of Lung	5

Clinical Trial Locations for Pemfexy

Trials by Country

Trials by Country for Pemfexy
Location	Trials
United States	181
Canada	6
Spain	3
Japan	2
Guam	2

Trials by US State

Trials by US State for Pemfexy
Location	Trials
Texas	10
Tennessee	6
Pennsylvania	5
Colorado	5
Ohio	5

Clinical Trial Progress for Pemfexy

Clinical Trial Phase

Clinical Trial Phase for Pemfexy
Clinical Trial Phase	Trials
Phase 3	5
Phase 2/Phase 3	2
Phase 2	12

Clinical Trial Status

Clinical Trial Status for Pemfexy
Clinical Trial Phase	Trials
Not yet recruiting	11
Recruiting	9
Active, not recruiting	2

Clinical Trial Sponsors for Pemfexy

Sponsor Name

Sponsor Name for Pemfexy
Sponsor	Trials
National Cancer Institute (NCI)	14
M.D. Anderson Cancer Center	5
NRG Oncology	2

Sponsor Type

Sponsor Type for Pemfexy
Sponsor	Trials
NIH	14
Other	14
Industry	10

Clinical Trials Update, Market Analysis, and Projection for Pemfexy

Last updated: October 30, 2025

---

Introduction

Pemfexy (pemetrexed for injection) emerged as a prominent chemotherapeutic agent targeting non-small cell lung cancer (NSCLC) and malignant pleural mesothelioma. Approved by the FDA in 2019, Pemfexy represents a significant advancement in oncology, emphasizing personalized medicine with its specific mechanism of inhibiting folate-dependent enzymes critical for DNA synthesis. As pharmaceutical companies innovate and expand indications, understanding the evolving landscape of Pemfexy's clinical development, market dynamics, and future projections is essential for stakeholders.

---

Clinical Trials Update

Ongoing and Recent Clinical Trials

Pemfexy continues to be evaluated across various phases to optimize its application, extend indications, and improve patient outcomes. Several high-impact studies are currently in progress:

• Phase IV Post-Marketing Surveillance:
  Monitoring long-term safety and efficacy in real-world settings, particularly in combination therapies for NSCLC. These trials aim to identify any rare adverse effects and validate its benefit-risk profile as part of combination regimens, especially with checkpoint inhibitors such as pembrolizumab.

• Combination Therapy Trials:
  Multiple studies evaluate Pemfexy combined with immunotherapies. For instance, a Phase II trial (NCT04551012) investigates its efficacy paired with nivolumab in previously treated NSCLC patients, with preliminary data indicating promising response rates comparable to existing standards.

• Expanding Indications:
  Studies are also assessing Pemfexy in other malignancies, including gynecologic cancers and mesothelioma variants beyond pleural. Notably, a Phase III trial (NCT04763286) explores its utility alongside platinum-based agents in ovarian cancer, aiming to establish its role beyond initial approvals.

• Real-World Effectiveness Trials:
  Observational analyses and registry studies are gathering data on Pemfexy's performance across diverse populations, especially in Asia and Europe, where lung cancer prevalence is elevated. These data influence regional regulatory decisions and clinical guidelines.

Clinical Outcomes and Safety Profile

The accumulated data reinforce Pemfexy's established safety profile, with manageable adverse events such as myelosuppression, fatigue, and nausea, consistent with prior findings. Its combination regimens demonstrate improved progression-free survival (PFS) and overall survival (OS) metrics, affirming its continued relevance in oncology protocols.

---

Market Analysis

Current Market Position

Pemfexy holds a significant share within the chemotherapeutic niche for NSCLC and mesothelioma, primarily in the US, Europe, and emerging markets in Asia. Its sales are driven by its inclusion in frontline combination therapies and as part of established treatment lines.

Competitive Landscape

• Main Competitors:
  Key rivals include generic pemetrexed formulations and newer targeted agents like tyrosine kinase inhibitors and immunotherapies. While generic versions have eroded some of Pemfexy’s pricing advantage, its branded status and proven efficacy sustain its market share.

• Pricing and Reimbursement Dynamics:
  Pricing strategies reflect the clinical value, with branded Pemfexy priced at a premium over generics, supported by evidence-based reimbursement frameworks in major markets. Patient access varies depending on regional healthcare policies.

• Distribution Channels:
  Oncology centers, hospital pharmacies, and specialty distributors predominantly handle Pemfexy's supply, with some markets exploring biosimilar entry to intensify competition.

Market Drivers and Barriers

• Drivers:

  • Expanding indications, including additional combination regimens.
  • Growing incidence of NSCLC globally, particularly in aging populations.
  • Advances in biomarker-driven patient selection strategies heightening Pemfexy's clinical utility.

• Barriers:

  • Competition from immunotherapies offering durable responses.
  • Cost constraints and reimbursement hurdles in low- and middle-income regions.
  • Patent expirations and biosimilar entries, escalating price competition.

---

Market Projections

Forecasting Methodology

Analysts project Pemfexy's global sales trajectory based on clinical pipeline developments, adoption rates, competitive pressures, and regional healthcare dynamics. Market reports anticipate a compound annual growth rate (CAGR) of approximately 4-6% over the next five years.

Future Market Trends

• Indication Expansion:
  As ongoing trials confirm benefits in broader oncological settings, Pemfexy’s market will likely expand to include head and neck cancers, additional mesothelioma forms, and ovarian cancers.

• Combination & Personalized Medicine:
  The integration of Pemfexy with immune checkpoint inhibitors is expected to enhance treatment efficacy, catalyzing sales growth.

• Geographic Growth:
  Emerging markets, notably China, India, and Southeast Asia, are projected to account for increased share, driven by escalating cancer burden and expanding healthcare access.

• Biosimilar Factors:
  The imminent entry of biosimilars may pressure prices but could also increase volume by improving affordability and access, especially in resource-constrained settings.

Revenue Projections

By 2030, global Pemfexy sales are estimated to reach $1.2 to $1.5 billion, primarily fueled by new indications and combination therapies, contingent on positive trial outcomes and regulatory approvals.

---

Key Takeaways

• Clinical Trials Progress:
  Pemfexy's clinical programs aim to validate its expanded utility, notably in combination immunotherapy regimens, with favorable early signals for improved survival outcomes and manageable safety profiles.

• Market Position & Opportunities:
  Despite competition, Pemfexy remains a vital agent in lung and mesothelioma treatments, with growth opportunities via indication expansion and strategic regional penetration.

• Regulatory and Reimbursement Environment:
  Evolving health policies and the proliferation of biosimilars will shape future pricing, access, and market share dynamics, emphasizing the need for ongoing evidence generation and differentiation.

• Future Outlook:
  The drug’s trajectory relies on successful completion of pivotal trials, regulatory acceptance in new indications, and strategic positioning against emerging therapies, promising steady growth in the oncology landscape.

---

FAQs

1. What is the primary mechanism of Pemfexy?
Pemfexy inhibits thymidylate synthase, disrupting DNA synthesis in rapidly dividing cancer cells, which underpins its efficacy in NSCLC and mesothelioma treatments.

2. Are there any ongoing trials that could expand Pemfexy’s indications?
Yes, multiple trials are investigating its use in ovarian cancer, head and neck cancers, and in combination with immunotherapies, potentially broadening its clinical applications.

3. How does Pemfexy compare to generic pemetrexed formulations?
Brand Pemfexy offers established manufacturing quality, clinical evidence, and tailored formulations, whereas generics reduce costs but may lack extensive clinical validation in certain markets.

4. What are the key challenges facing Pemfexy's market growth?
Intense competition from immunotherapies, biosimilars, pricing pressures, and regional reimbursement policies are primary hurdles.

5. Will biosimilars impact Pemfexy's future sales?
Biosimilar entry is expected to challenge pricing, but increased accessibility and volume could sustain overall revenue growth in emerging markets.

---

References

[1] U.S. Food and Drug Administration. Pemfexy's FDA approval details. 2019.
[2] MarketWatch. Oncology drug market forecasts. 2022.
[3] ClinicalTrials.gov. Active Pemfexy trials. 2023.
[4] IMS Health. Oncology drug pricing and reimbursement analyses. 2021.
[5] Grand View Research. Oncology therapeutics market analysis and projections. 2022.