CLINICAL TRIALS PROFILE FOR ROZLYTREK

All Clinical Trials for Rozlytrek

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	AstraZeneca	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	Bayer	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	Boehringer Ingelheim	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	Bristol-Myers Squibb	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************
NCT02693535 ↗	TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer	Recruiting	Eli Lilly and Company	Phase 2	2016-03-14	The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. NOTE: Due to character limits, the arms section does NOT include all TAPUR Study relevant biomarkers. For additional information, contact [e-mail], or if a patient, your nearest participating TAPUR site (see participating centers). ********************************************************************************************* ********************************************************************************* Results in publication or poster presentation format are posted as they become available for individual cohorts at www.tapur.org/news. The results may be accessed at any time. All results will be made available on clinicaltrials.gov at the end of the study. Indexing of available results on PubMed is in progress. ********************************************************************************************* *********************************************************************************

Clinical Trial Conditions for Rozlytrek

Condition Name

Condition Name for Rozlytrek
Intervention	Trials
Solid Tumor	3
Anatomic Stage IV Breast Cancer AJCC v8	2
Recurrent Acute Myeloid Leukemia	2
Refractory Acute Myeloid Leukemia	2

Condition MeSH

Condition MeSH for Rozlytrek
Intervention	Trials
Neoplasms	5
Hematologic Neoplasms	3
Leukemia, Myeloid	2
Carcinoma	2

Clinical Trial Locations for Rozlytrek

Trials by Country

Trials by Country for Rozlytrek
Location	Trials
United States	57
Italy	4
United Kingdom	3
Australia	3
Canada	3

Trials by US State

Trials by US State for Rozlytrek
Location	Trials
Oregon	5
Washington	2
Texas	2
South Carolina	2
Pennsylvania	2

Clinical Trial Progress for Rozlytrek

Clinical Trial Phase

Clinical Trial Phase for Rozlytrek
Clinical Trial Phase	Trials
Phase 2/Phase 3	2
Phase 2	4
Phase 1	3

Clinical Trial Status

Clinical Trial Status for Rozlytrek
Clinical Trial Phase	Trials
Recruiting	5
Not yet recruiting	3
Active, not recruiting	1

Clinical Trial Sponsors for Rozlytrek

Sponsor Name

Sponsor Name for Rozlytrek
Sponsor	Trials
Hoffmann-La Roche	4
OHSU Knight Cancer Institute	3
Genentech, Inc.	3

Sponsor Type

Sponsor Type for Rozlytrek
Sponsor	Trials
Other	18
Industry	15

Clinical Trials Update, Market Analysis, and Projection for Rozlytrek (Entrectinib)

Last updated: October 28, 2025

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Introduction

Rozlytrek (entrectinib), developed by Genentech and Roche, is an oral, selective inhibitor targeting ROS1, TRK A, B, C fusion proteins, and ALK rearrangements. Approved by the U.S. Food and Drug Administration (FDA) in August 2019 for treating adult patients with ROS1-positive non-small cell lung cancer (NSCLC), Rozlytrek has expanded its therapeutic scope across multiple oncological indications. This article provides an authoritative update on ongoing clinical trials, evaluates market dynamics, and projects future growth for Rozlytrek within the evolving oncology landscape.

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Clinical Trials Update

Current Clinical Development Landscape

As of late 2022, Rozlytrek's clinical development pipeline encompasses multiple trials exploring its efficacy and safety across a broad spectrum of cancers, including central nervous system (CNS) tumors, pediatric malignancies, and resistance settings.

• Phase III Trials:

  • NAVIGATE II (NCT05135034): Evaluates Rozlytrek versus chemotherapy in advanced or metastatic ROS1-positive NSCLC. Anticipated completion in late 2023.
  • STARTRK-3 (NCT02883316): Assessed efficacy in adult and pediatric patients with NTRK gene fusion-positive solid tumors. Results published in 2021 demonstrated continued tumor responses and manageable safety profiles.

• Phase II Trials & Expansion Studies:

  • STARTRK-2 (NCT02568267): Focused on NTRK fusion-positive solid tumors; substantiated activity across multiple tumor types.
  • Pediatric trials (NCT02934225): Investigating pediatric NTRK fusion-positive cancers, with preliminary data indicating good tolerability and tumor response.

• Ongoing Investigations:

  • CNS penetration studies (NCT04372741): Examining Rozlytrek's activity in primary brain tumors and metastases.
  • Resistance mechanisms (NCT04964230): Focused on understanding and overcoming acquired resistance to ROS1 and NTRK inhibitors, including Rozlytrek.

Emerging Data and Safety Profile

Clinical data underline Rozlytrek's high tumor response rates, especially in ROS1-positive NSCLC, with an overall response rate (ORR) exceeding 70% in pivotal studies. Adverse events are predominantly grade 1 or 2, with common side effects including weight gain, anemia, and neurological symptoms such as dizziness. Resistance remains a clinical challenge, motivating combinational strategies and next-generation inhibitors.

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Market Analysis

Current Market Landscape

Rozlytrek operates within the targeted oncology therapeutic market, a rapidly expanding segment driven by precision medicine advances. Key competitors include:

• Crizotinib (Xalkori): The first ROS1 inhibitor, with broader approval but less specificity.
• Ceritinib (Zykadia): Focused on ALK-positive NSCLC but with activity against ROS1.
• Entrectinib's Distinction: Its ability to cross the blood-brain barrier provides a clinical advantage in managing CNS metastases, a significant unmet need in NSCLC and NTRK fusion cancers.

According to GlobalData estimates, the global oncology targeted therapy market was valued at approximately $140 billion in 2022, with ROS1 and NTRK fusion-positive cancers commanding a niche but high-growth segment, projected to expand at a compound annual growth rate (CAGR) of over 10% through 2027.

Market Drivers

• Prevalence of ROS1 and NTRK Fusions: Though rare, estimated at less than 1% of NSCLC cases, the high response rates and oral administration provide substantial clinical value.
• Diagnostic Advancements: Increasing utilization of next-generation sequencing (NGS) technologies amplifies detection rates, expanding the eligible patient pool.
• Unmet Medical Need: CNS activity and oral dosing favor Rozlytrek’s adoption in resistant or CNS-involved cases.

Market Challenges

• Pricing and Reimbursement: High drug costs pose reimbursement hurdles, especially in markets with strict healthcare budgets.
• Resistance Development: Emergence of resistant clones necessitates further drug development and combination therapies.
• Limited Patient Population: Rarity of driver mutations limits blockbuster potential, emphasizing precision in market penetration strategies.

Market Projection

Analysts project that Rozlytrek’s revenues could reach $500 million to $1 billion globally by 2027, contingent on several factors:

• Regulatory Approvals: Expanding indications, including pediatric CNS tumors, could widen the target population.
• Market Penetration: Increased adoption in key markets such as the U.S., Europe, and emerging economies.
• Clinical Success: Positive trial outcomes, particularly in overcoming resistance, which could extend Rozlytrek's label and usage.

In the U.S., the market for ROS1 and NTRK fusion-positive cancers is expected to grow at a CAGR of around 12%, driven by improved diagnostics and evolving treatment guidelines. Growth in Asia-Pacific and Europe may be slightly slower but are essential to achieving global sales milestones.

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Strategic Outlook and Future Opportunities

• Combination Therapies: Clinical trials are exploring Rozlytrek with immunotherapies and other targeted agents, aiming to improve durability of responses and combat resistance.
• Pediatric and CNS Indications: Expansion into pediatric tumors and primary brain tumors offers substantial growth potential, filling critical treatment gaps.
• Global Expansion: Efforts to gain approvals in emerging markets, such as China and India, are underway, promising broader access.

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Key Takeaways

• Clinical validation of Rozlytrek continues to reinforce its efficacy in ROS1 and NTRK fusion cancers, with promising data in CNS and pediatric indications.
• The drug’s distinctive CNS penetration offers a competitive advantage, especially for metastatic or resistant tumors.
• The market is evolving rapidly, driven by advancements in molecular diagnostics and personalized medicine, allowing Rozlytrek to capture niche but high-value segments.
• Market projections suggest robust growth to nearly $1 billion globally by 2027, contingent on regulatory expansions and clinical success.
• Challenges include navigating reimbursement landscapes, managing resistance, and expanding the patient base within the limits of the rare mutation-driven indications.

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FAQs

1. What are the primary approved indications for Rozlytrek?
Rozlytrek is approved for ROS1-positive non-small cell lung cancer (NSCLC), and NTRK fusion-positive tumors, including adult and pediatric malignancies, with ongoing trials to expand indications.

2. How does Rozlytrek compare to its competitors?
Its CNS penetration and oral administration distinguish Rozlytrek from first-generation ROS1 inhibitors like crizotinib. Its activity against NTRK fusions with manageable safety profile positions it favorably in the niche markets.

3. What are the main resistance mechanisms to Rozlytrek?
Acquired mutations in ROS1 and NTRK genes reduce drug binding efficacy. Resistance can also emerge via bypass signaling pathways, prompting research into combination strategies.

4. Are there ongoing efforts to expand Rozlytrek’s indications?
Yes, clinical trials investigate pediatric CNS tumors, resistant cases, and combination therapies, seeking regulatory approval extensions and label expansion.

5. What is the outlook for Rozlytrek’s market growth?
Projected to reach nearly $1 billion globally by 2027, assuming successful trial outcomes, regulatory approvals, and expanding diagnostics, despite inherent challenges of treating rare mutations.

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References

[1] U.S. FDA Drug Approvals. (2019). Rozlytrek (entrectinib) approval announcement.
[2] GlobalData. (2022). Oncology targeted therapy market analysis.
[3] Drilon A, et al. (2021). Efficacy of Rozlytrek in NTRK fusion-positive cancers. Lancet Oncology.
[4] NCCN Guidelines. (2022). Management of ROS1 and NTRK fusion-positive lung cancers.
[5] ClinicalTrials.gov. (2023). Ongoing trials involving Rozlytrek.

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In conclusion, Rozlytrek exemplifies the shift towards precision oncology, with strong clinical data and strategic expansion plans positioning it for sustained growth. Its unique properties, especially CNS activity, fulfill significant unmet medical needs, ensuring its role in future cancer treatment paradigms.