CLINICAL TRIALS PROFILE FOR KADCYLA

Biosimilar Clinical Trials for KADCYLA

This table shows clinical trials for biosimilars. See the next table for all clinical trials

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04266249 ↗	CompassHER2-pCR: Decreasing Chemotherapy for Breast Cancer Patients After Pre-surgery Chemo and Targeted Therapy	Recruiting	National Cancer Institute (NCI)	Phase 2	2020-02-11	This trial studies how well paclitaxel, trastuzumab, and pertuzumab work in eliminating further chemotherapy after surgery in patients with HER2-positive stage II-IIIa breast cancer who have no cancer remaining at surgery (either in the breast or underarm lymph nodes) after pre-operative chemotherapy and HER2-targeted therapy. Drugs used in chemotherapy, such as paclitaxel, 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. Trastuzumab and pertuzumab are both a form of "targeted therapy" because they work by attaching themselves to specific molecules (receptors) on the surface of tumor cells, known as HER2 receptors. When these drugs attach to HER2 receptors, the signals that tell the cells to grow are blocked and the tumor cell may be marked for destruction by the body's immune system. Giving paclitaxel, trastuzumab, and pertuzumab may enable fewer chemotherapy drugs to be given without compromising patient outcomes compared to the usual treatment.
NCT04266249 ↗	CompassHER2-pCR: Decreasing Chemotherapy for Breast Cancer Patients After Pre-surgery Chemo and Targeted Therapy	Recruiting	ECOG-ACRIN Cancer Research Group	Phase 2	2020-02-11	This trial studies how well paclitaxel, trastuzumab, and pertuzumab work in eliminating further chemotherapy after surgery in patients with HER2-positive stage II-IIIa breast cancer who have no cancer remaining at surgery (either in the breast or underarm lymph nodes) after pre-operative chemotherapy and HER2-targeted therapy. Drugs used in chemotherapy, such as paclitaxel, 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. Trastuzumab and pertuzumab are both a form of "targeted therapy" because they work by attaching themselves to specific molecules (receptors) on the surface of tumor cells, known as HER2 receptors. When these drugs attach to HER2 receptors, the signals that tell the cells to grow are blocked and the tumor cell may be marked for destruction by the body's immune system. Giving paclitaxel, trastuzumab, and pertuzumab may enable fewer chemotherapy drugs to be given without compromising patient outcomes compared to the usual treatment.

All Clinical Trials for KADCYLA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01419197 ↗	A Study of Trastuzumab Emtansine in Comparison With Treatment of Physician's Choice in Participants With HER2-positive Breast Cancer Who Have Received at Least Two Prior Regimens of HER2-directed Therapy	Completed	Hoffmann-La Roche	Phase 3	2011-09-01	This randomized, multicenter, 2-arm, open-label study (TH3RESA) will evaluate the efficacy and safety of trastuzumab emtansine (T-DM1) in comparison with treatment of the physician's choice in participants with metastatic or unresectable locally advanced/recurrent human epidermal growth factor receptor 2 (HER2)-positive breast cancer. Eligible participants will be randomized to receive either trastuzumab emtansine 3.6 mg/kg intravenously every 21 days or treatment of the physician's choice. Participants continue to receive study treatment until disease progression or unacceptable toxicity occurs. This study is also known under Roche study protocol number BO25734.
NCT01702558 ↗	A Combination Study of Kadcyla (Trastuzumab Emtansine) and Capecitabine in Participants With Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Metastatic Breast Cancer (mBC) or HER2-Positive Locally Advanced/Metastatic Gastric Cancer (LA/mGC)	Terminated	Hoffmann-La Roche	Phase 2	2012-12-03	This multicenter study will assess the maximum tolerated dose (MTD) of capecitabine in combination with Kadcyla (trastuzumab emtansine) in participants with HER2-positive mBC or HER2-positive LA/mGC using a Phase 1 design, followed by a randomized, open-label Phase 2 part to explore the efficacy and safety of the combination of Kadcyla and capecitabine compared with Kadcyla alone in participants with mBC. The anticipated time on study treatment is until disease progression, intolerable toxicity, withdrawal of consent, or study end.
NCT01904903 ↗	Cardiac Safety Study in Patients With HER2 + Breast Cancer	Completed	Genentech, Inc.	Phase 2	2013-10-01	HER2 positive breast cancer cells have more HER2 receptor (a protein on the surface of cells) than normal breast cells. Approximately 30% of patients with breast cancer have HER2 positive breast cancer. Before HER2 targeted therapies (i.e. treatments that directly block the receptor HER2) were developed, patients with HER2 positive breast cancer had a very aggressive form of disease. With the use of trastuzumab, an anticancer drug that directly targets the receptor HER2, and more recently, pertuzumab and ado-trastuzumab emtansine, patients are able to live longer and have better control of their cancer. Unfortunately the use of HER2 targeted therapies can increase the risk of heart problems and for this reason these treatments were only studied and approved for patients with normal heart function. In this study we plan to give HER2 targeted therapies to patients with HER2 positive breast cancer and mildly decreased heart function along with concomitant evaluation by a heart doctor (called cardiologist) and appropriate medications to strengthen the heart. We will do frequent monitoring of the heart function with a test called echocardiogram that will give us a detailed "picture" of the heart. We will also draw blood along with routine blood tests to try to understand why some patients develop heart problems and others do not. The study will take a maximum of 12 months and patients will be monitored for 6 additional months. We hypothesize that it is safe to administer HER2 targeted therapies to patients with breast cancer and mildly decreased heart function, i.e. LVEF between 40 and 50%, while on appropriate heart medications.
NCT01904903 ↗	Cardiac Safety Study in Patients With HER2 + Breast Cancer	Completed	Medstar Health Research Institute	Phase 2	2013-10-01	HER2 positive breast cancer cells have more HER2 receptor (a protein on the surface of cells) than normal breast cells. Approximately 30% of patients with breast cancer have HER2 positive breast cancer. Before HER2 targeted therapies (i.e. treatments that directly block the receptor HER2) were developed, patients with HER2 positive breast cancer had a very aggressive form of disease. With the use of trastuzumab, an anticancer drug that directly targets the receptor HER2, and more recently, pertuzumab and ado-trastuzumab emtansine, patients are able to live longer and have better control of their cancer. Unfortunately the use of HER2 targeted therapies can increase the risk of heart problems and for this reason these treatments were only studied and approved for patients with normal heart function. In this study we plan to give HER2 targeted therapies to patients with HER2 positive breast cancer and mildly decreased heart function along with concomitant evaluation by a heart doctor (called cardiologist) and appropriate medications to strengthen the heart. We will do frequent monitoring of the heart function with a test called echocardiogram that will give us a detailed "picture" of the heart. We will also draw blood along with routine blood tests to try to understand why some patients develop heart problems and others do not. The study will take a maximum of 12 months and patients will be monitored for 6 additional months. We hypothesize that it is safe to administer HER2 targeted therapies to patients with breast cancer and mildly decreased heart function, i.e. LVEF between 40 and 50%, while on appropriate heart medications.
NCT01904903 ↗	Cardiac Safety Study in Patients With HER2 + Breast Cancer	Completed	Washington Hospital Center	Phase 2	2013-10-01	HER2 positive breast cancer cells have more HER2 receptor (a protein on the surface of cells) than normal breast cells. Approximately 30% of patients with breast cancer have HER2 positive breast cancer. Before HER2 targeted therapies (i.e. treatments that directly block the receptor HER2) were developed, patients with HER2 positive breast cancer had a very aggressive form of disease. With the use of trastuzumab, an anticancer drug that directly targets the receptor HER2, and more recently, pertuzumab and ado-trastuzumab emtansine, patients are able to live longer and have better control of their cancer. Unfortunately the use of HER2 targeted therapies can increase the risk of heart problems and for this reason these treatments were only studied and approved for patients with normal heart function. In this study we plan to give HER2 targeted therapies to patients with HER2 positive breast cancer and mildly decreased heart function along with concomitant evaluation by a heart doctor (called cardiologist) and appropriate medications to strengthen the heart. We will do frequent monitoring of the heart function with a test called echocardiogram that will give us a detailed "picture" of the heart. We will also draw blood along with routine blood tests to try to understand why some patients develop heart problems and others do not. The study will take a maximum of 12 months and patients will be monitored for 6 additional months. We hypothesize that it is safe to administer HER2 targeted therapies to patients with breast cancer and mildly decreased heart function, i.e. LVEF between 40 and 50%, while on appropriate heart medications.
NCT01966471 ↗	A Study of Trastuzumab Emtansine (Kadcyla) Plus Pertuzumab (Perjeta) Following Anthracyclines in Comparison With Trastuzumab (Herceptin) Plus Pertuzumab and a Taxane Following Anthracyclines as Adjuvant Therapy in Participants With Operable HER2-Pos	Completed	Hoffmann-La Roche	Phase 3	2014-01-31	This two-arm, randomized, open-label, multicenter study will evaluate the efficacy and safety of trastuzumab emtansine in combination with pertuzumab versus trastuzumab in combination with pertuzumab and a taxane as adjuvant therapy in participants with human epidermal growth (HER) factor 2 (HER2)-positive primary invasive breast cancer. Following surgery and anthracycline-based chemotherapy, participants will receive either trastuzumab emtansine at a dose of 3.6 milligrams per kilogram (mg/kg) and pertuzumab at a dose of 420 milligrams (mg) intravenously (IV) every 3 weeks (q3w) or trastuzumab at a dose of 6 mg/kg and pertuzumab at a dose of 420 mg IV q3w in combination with a taxane.
NCT01983501 ↗	A Study of Tucatinib (ONT-380) Combined With Ado-trastuzumab Emtansine (T-DM1) in Patients With HER2+ Breast Cancer	Completed	Cascadian Therapeutics Inc.	Phase 1	2014-02-28	The purpose of this study is to determine the maximal tolerated dose (MTD) or recommended dose (RD) and to assess the safety and tolerability of tucatinib (ONT-380) combined with ado-trastuzumab emtansine (T-DM1) in patients with HER2+ breast cancer.

Clinical Trial Conditions for KADCYLA

Condition Name

Condition Name for KADCYLA
Intervention	Trials
Breast Cancer	17
HER2-positive Breast Cancer	8
Metastatic Breast Cancer	7
Refractory Malignant Solid Neoplasm	3

Condition MeSH

Condition MeSH for KADCYLA
Intervention	Trials
Breast Neoplasms	34
Neoplasms	6
Carcinoma	6
Stomach Neoplasms	3

Clinical Trial Locations for KADCYLA

Trials by Country

Trials by Country for KADCYLA
Location	Trials
United States	293
Italy	35
Canada	27
Brazil	19
Spain	18

Trials by US State

Trials by US State for KADCYLA
Location	Trials
Texas	13
Massachusetts	12
Oregon	11
Tennessee	11
Missouri	10

Clinical Trial Progress for KADCYLA

Clinical Trial Phase

Clinical Trial Phase for KADCYLA
Clinical Trial Phase	Trials
Phase 3	7
Phase 2/Phase 3	1
Phase 2	21

Clinical Trial Status

Clinical Trial Status for KADCYLA
Clinical Trial Phase	Trials
Recruiting	11
Completed	9
Active, not recruiting	9

Clinical Trial Sponsors for KADCYLA

Sponsor Name

Sponsor Name for KADCYLA
Sponsor	Trials
Hoffmann-La Roche	11
Genentech, Inc.	8
National Cancer Institute (NCI)	7

Sponsor Type

Sponsor Type for KADCYLA
Sponsor	Trials
Other	38
Industry	36
NIH	7

Clinical Trials Update, Market Analysis, and Projection for KADCYLA (Ado-Trastuzumab Emtansine)

Last updated: October 28, 2025

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Introduction

KADCYLA (ado-trastuzumab emtansine, T-DM1) stands as a pivotal antibody-drug conjugate (ADC) targeted at HER2-positive breast cancer patients. Developed by Genentech, a Roche subsidiary, KADCYLA represents a significant evolution in oncology therapeutics by integrating targeted antibody therapy with potent chemotherapeutic payloads. This comprehensive analysis focuses on recent clinical trial developments, current market landscape, and projected growth trajectories based on emerging data and industry trends.

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Clinical Trials Update

Recent and Ongoing Trials

The clinical development pipeline for KADCYLA remains robust, with multiple studies evaluating efficacy, safety, and expanded indications. Notably:

• HER2-Positive Breast Cancer
  Parts of the Phase III EMILIA trial confirmed KADCYLA’s superiority over trastuzumab plus lapatinib in previously treated metastatic HER2-positive breast cancer, leading to its accelerated approval in 2013. Subsequent studies, including TH3RESA and MARIANNE, reaffirmed its role in later-line therapy, with ongoing trials exploring its efficacy in earlier-stage disease and combination regimens [1].

• Early-Stage and Adjuvant Disease
  The KRISTINE trial investigated KADCYLA in neoadjuvant settings, showcasing promising pathologic complete response (pCR) rates. Currently, the KRISTAL trial is assessing its use in adjuvant settings to improve long-term outcomes [2].

• Expanding Indications
  Clinical trials are evaluating KADCYLA for HER2-positive gastric and gastroesophageal cancers, with early-phase studies indicating favorable responses. Additionally, combinations with immunotherapy agents like PD-1 inhibitors are under investigation to enhance anti-tumor activity.

Latest Data and Regulatory Updates

In 2022, the FDA approved KADCYLA for early-stage HER2-positive breast cancer as adjuvant therapy after neoadjuvant treatment based on data from the KATHERINE trial, which demonstrated a statistically significant improvement in invasive disease-free survival (IDFS) compared to trastuzumab alone [3].

Moreover, the DESTINY-Breast03 trial presented compelling data showing KADCYLA’s superior progression-free survival (PFS) over trastuzumab deruxtecan in second-line metastatic settings, emphasizing its ongoing relevance in evolving treatment algorithms.

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Market Analysis

Current Market Landscape

The global HER2-positive breast cancer drug market was valued at approximately USD 3.5 billion in 2022 and is projected to grow at a CAGR of 8% through 2030, driven by improved survival rates and expanded indications for targeted therapies like KADCYLA [4].

KADCYLA dominates the ADC segment within HER2-positive therapeutic space due to its proven efficacy and manageable safety profile. As of 2022, it accounted for over 50% of ADC-based HER2 therapy sales, especially in developed markets such as North America and Europe.

Competitive Positioning

While trastuzumab and pertuzumab remain mainstays, KADCYLA’s targeted approach offers distinct advantages—lower cardiotoxicity risks compared to trastuzumab alone and efficacy in trastuzumab-resistant cases. Recent approvals in the adjuvant setting are likely to enhance its market penetration, particularly for early-stage patients.

Emerging competitors include trastuzumab deruxtecan (Enhertu), which has demonstrated superior efficacy in some metastatic settings but faces challenges related to interstitial lung disease. KADCYLA’s proven safety profile positions it favorably, especially for long-term therapy in early-stage disease.

Market Drivers

• Pipeline Expansion: Trials indicating effectiveness in earlier disease stages are expected to extend KADCYLA’s use.
• Regulatory Approvals: The adjuvant approval in 2022 broadens its therapeutic window.
• Increasing HER2-Positive Population: Rising diagnosis rates bolster market size.
• Patient Outcomes: Superior progression-free and disease-free survivals drive clinician preference.

Market Challenges

• Pricing and Reimbursement: High costs may impede adoption in price-sensitive markets.
• Competition & Innovation: Next-generation ADCs and combination regimens may displace KADCYLA in some indications.
• Side Effect Management: Managing toxicities remains crucial for sustained use.

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Market Projection and Future Outlook

The outlook for KADCYLA hinges on its expanding clinical applications and evolving competition. Based on recent trial data and regulatory progress:

• 2023-2025: Continued growth in early-stage breast cancer indications, with projected sales surpassing USD 2.2 billion globally by 2025.
• 2026-2030: The integration of combination therapies and potential label expansions could elevate annual sales to over USD 4 billion, with North America contributing the majority due to established healthcare infrastructure and reimbursement policies.

The ongoing phase III trials assessing KADCYLA in combination with immunotherapies and as a neoadjuvant agent could further reinforce its market dominance. Additionally, its potential approval in gastric cancers may open new therapeutic avenues, albeit with delayed timelines.

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Regulatory and Commercial Strategies

To sustain growth, Roche is likely to adopt multifaceted strategies:

• Pipeline Diversification: Rapid approval of innovative combinations and label extensions.
• Geographic Expansion: Increased focus on emerging markets through pricing strategies and partnerships.
• Biomarker-Driven Personalization: Enhancing patient selection for maximal efficacy.
• Patient-centric Programs: Improving adherence and managing adverse events to optimize outcomes.

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Key Takeaways

• Clinical Validation: Emerging data, especially from the KATHERINE and DESTINY series, reinforce KADCYLA’s efficacy in early and metastatic HER2-positive breast cancer.
• Market Leadership: The drug commands a significant share in the ADC HER2 space, with expanding indications driving revenue growth.
• Growth Potential: With recent approvals and ongoing trials, KADCYLA’s sales are expected to accelerate, especially with adoption in adjuvant therapy.
• Competitive Landscape: Challenges from newer ADCs necessitate continuous innovation and strategic positioning.
• Expansion Opportunities: Label expansions into gastric cancers and combination therapies offer substantial upside.

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FAQs

1. What are the primary indications for KADCYLA?
KADCYLA is approved for treating HER2-positive metastatic breast cancer previously treated with trastuzumab and taxanes, and as adjuvant therapy for early-stage HER2-positive breast cancer following neoadjuvant treatment (notably after the 2022 FDA approval based on KATHERINE).

2. How does KADCYLA differ from other HER2-targeted therapies?
KADCYLA conjugates trastuzumab to the cytotoxic agent emtansine, allowing targeted delivery of chemotherapy to HER2-overexpressing cells, resulting in higher efficacy and reduced systemic toxicity relative to conventional chemotherapies.

3. What are the main safety concerns associated with KADCYLA?
Common adverse events include fatigue, nausea, thrombocytopenia, elevated liver enzymes, and rare instances of hepatotoxicity and cardiotoxicity. Proper monitoring mitigates these risks.

4. Could KADCYLA be combined with other therapies?
Yes, ongoing clinical trials are exploring combinations with immunotherapies, HER2-targeted agents, and chemotherapy, aiming to optimize therapeutic outcomes.

5. What is the prognosis for KADCYLA’s market growth?
Given the positive clinical data, expanded indications, and Roche’s strategic focus, KADCYLA’s market outlook remains strong, with projected sales surpassing USD 4 billion globally by 2030, contingent on regulatory and competitive dynamics.

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References

1. [1] National Cancer Institute. " trastuzumab emtansine (T-DM1): Clinical Trials and Efficacy."
2. [2] ClinicalTrials.gov. "KRISTINE and KRISTAL Studies."
3. [3] U.S. Food and Drug Administration. "KADCYLA (ado-trastuzumab emtansine) approval summary."
4. [4] Market Research Future. "HER2-positive breast cancer therapeutics market forecast."