CLINICAL TRIALS PROFILE FOR ADO-TRASTUZUMAB EMTANSINE

Biosimilar Clinical Trials for ado-trastuzumab emtansine

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.
NCT05530057 ↗	Eribulin With or Without Trastuzumab-biosimilar in Patients With HER2-overexpressed Recurrent or Stage IV Breast Cancer	Recruiting	Seoul National University Hospital	Phase 2	2020-02-18	Optimal salvage treatment for HER2-positive breast cancer after trastuzumab and T-DM1 failure still remains to be established. We would like to investigate the efficacy and safety of combination chemotherapy of eribulin and trastuzumab as salvage treatment for HER2-Positive breast cancer after exposure to trastuzumab and T-DM1

All Clinical Trials for ado-trastuzumab emtansine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00509769 ↗	A Study of Trastuzumab Emtansine (Trastuzumab-MCC-DM1) Administered Intravenously to Patients With Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Metastatic Breast Cancer	Completed	Genentech, Inc.	Phase 2	2007-07-01	This was a multi-institutional, open-label, single-arm, Phase II study of trastuzumab emtansine (T-DM1) administered by intravenous (IV) infusion to patients with human epidermal growth factor receptor 2 (HER2)-positive metastatic breast cancer (MBC).
NCT00679211 ↗	A Study of Trastuzumab-Mcc-DM1 Administered Intravenously to Patients With HER2-Positive Metastatic Breast Cancer	Completed	Genentech, Inc.	Phase 2	2008-08-01	Study of trastuzumab emtansine (T-DM1) administered to patients with human epidermal growth factor receptor 2 (HER2)-positive metastatic breast cancer.
NCT00679341 ↗	A Study of the Efficacy and Safety of Trastuzumab Emtansine (Trastuzumab-MCC-DM1) vs. Trastuzumab (Herceptin®) and Docetaxel (Taxotere®) in Patients With Metastatic HER2-positive Breast Cancer Who Have Not Received Prior Chemotherapy for Metastati	Completed	Hoffmann-La Roche	Phase 2	2008-09-01	This was a Phase II, randomized, multicenter, international, 2-arm, open-label clinical trial designed to explore the efficacy and safety of trastuzumab emtansine (T-DM1) relative to the combination of trastuzumab and docetaxel in patients with human epidermal growth factor receptor 2 (HER2)-positive, unresectable, locally advanced breast cancer and/or metastatic breast cancer who have not received prior chemotherapy for metastatic disease.

Clinical Trial Conditions for ado-trastuzumab emtansine

Condition Name

Condition Name for ado-trastuzumab emtansine
Intervention	Trials
Breast Cancer	46
HER2-positive Breast Cancer	21
Metastatic Breast Cancer	16

Condition MeSH

Condition MeSH for ado-trastuzumab emtansine
Intervention	Trials
Breast Neoplasms	88
Neoplasms	7
Carcinoma, Non-Small-Cell Lung	6

Clinical Trial Locations for ado-trastuzumab emtansine

Trials by Country

Trials by Country for ado-trastuzumab emtansine
Location	Trials
United States	794
Italy	131
Spain	94
China	79
Canada	77

Trials by US State

Trials by US State for ado-trastuzumab emtansine
Location	Trials
Texas	40
New York	31
Florida	31
Tennessee	31
California	31

Clinical Trial Progress for ado-trastuzumab emtansine

Clinical Trial Phase

Clinical Trial Phase for ado-trastuzumab emtansine
Clinical Trial Phase	Trials
PHASE3	6
PHASE2	5
PHASE1	1

Clinical Trial Status

Clinical Trial Status for ado-trastuzumab emtansine
Clinical Trial Phase	Trials
Recruiting	39
Completed	31
Active, not recruiting	17

Clinical Trial Sponsors for ado-trastuzumab emtansine

Sponsor Name

Sponsor Name for ado-trastuzumab emtansine
Sponsor	Trials
Hoffmann-La Roche	33
Genentech, Inc.	16
Dana-Farber Cancer Institute	10

Sponsor Type

Sponsor Type for ado-trastuzumab emtansine
Sponsor	Trials
Industry	110
Other	88
NIH	9

Clinical Trials, Market Analysis, and Future Projections for Ado-Trastuzumab Emtansine (T-DM1)

Last updated: November 2, 2025

Introduction

Ado-trastuzumab emtansine (T-DM1), marketed as Kadcyla®, is an antibody-drug conjugate (ADC) combining trastuzumab with the cytotoxic agent DM1. Approved by the U.S. Food and Drug Administration (FDA) in 2013 for HER2-positive metastatic breast cancer, T-DM1 plays a significant role in targeted cancer therapy. This report provides a comprehensive update on its ongoing clinical trials, evaluates its current market landscape, and projects future growth trajectories, tailored to inform strategic business decisions.

Clinical Trials Update

Ongoing and Recent Trials

Since its initial approval, T-DM1 has been the subject of numerous clinical trials, expanding its indication scope and exploring combination therapies.

• HER2-Positive Breast Cancer: The Phase III KATHERINE trial assessed T-DM1 as adjuvant therapy post-neoadjuvant treatment. Results published in 2019 demonstrated a substantial improvement in invasive disease-free survival (iDFS) compared to trastuzumab alone, solidifying its role in early-stage HER2-positive breast cancer management [1].

• Combination Therapies and New Indications: Current trials, such as NCT03801847, investigate T-DM1 combined with immune checkpoint inhibitors for metastatic HER2-positive breast cancer, aiming to enhance anti-tumor efficacy. Similarly, studies like NCT03975647 are evaluating T-DM1 in HER2-positive gastric and gastroesophageal cancers—broadening its therapeutic horizon.

• Expanding Patient Populations: Trials targeting HER2-positive uterine and ovarian cancers are underway, with preliminary results indicating manageable safety profiles and promising efficacy signals [2].

Innovations in Delivery and Toxicity Management

Recent research aims to optimize T-DM1's safety profile:

• Nanoparticle formulations are in early phases to improve drug delivery specificity.
• Biomarker-driven trials are evaluating patient selection to mitigate adverse effects, particularly hepatotoxicity and cardiotoxicity associated with trastuzumab-based therapies.

Trial Outcomes and Implications

While the core efficacy data from pivotal trials confirm T-DM1's role, ongoing trials aim to:

• Confirm efficacy in early-stage settings, potentially shifting treatment paradigms.
• Identify biomarkers predictive of response and resistance.
• Develop combination regimens to overcome resistance mechanisms, critical for maintaining its clinical relevance.

Market Analysis

Current Market Position

Since its market entry, Kadcyla® has established a solid position within the targeted HER2-positive breast cancer segment.

• Market Share & Sales Performance: In 2022, global sales approximated $1.2 billion, reflecting steady growth driven by approval expansions and evolving treatment guidelines. The U.S. historically accounts for over 70% of revenues, with notable adoption among oncologists [3].

• Competitive Landscape: Its primary competitors include trastuzumab (Herceptin®), pertuzumab (Perjeta®), and other ADCs like trastuzumab deruxtecan (Enhertu®). The latter's approval for similar indications poses a competitive challenge, especially given its demonstrated efficacy in trastuzumab-resistant cases.

Market Drivers

• Expanded Indications: The approval of T-DM1 for adjuvant therapy in early breast cancer (KATHERINE trial data) is expected to expand its use, increasing sales.
• Physician Preference: Its established efficacy and safety profile reinforce clinician trust, bolstering adoption.
• Patient Demand for Targeted Therapy: Growing awareness and acceptance of precision medicine accelerate its uptake.

Market Challenges

• Pricing and Reimbursement Pressures: High treatment costs necessitate favorable reimbursement policies.
• Emerging Competitors: Newer ADCs and combination regimens threaten market share.
• Toxicity Concerns: Cardiovascular side effects necessitate cautious patient selection and monitoring.

Regulatory and Geographic Expansion

Beyond the U.S., Kadcyla® has received approval in Europe, Japan, and emerging markets. Regulatory submissions for additional indications continue in multiple jurisdictions, aiming to broaden global access.

Market Projection and Future Outlook

Forecasted Growth

Analysts project a compound annual growth rate (CAGR) of approximately 8-10% for T-DM1 globally over the next five years, driven by:

• Additional Approvals: Expansion into early-stage and other HER2-positive cancers.
• Enhanced Combination Approaches: Development of dual-targeted regimens with immunotherapies.
• Biomarker-Guided Therapy: Refinement and adoption of predictive biomarkers to optimize patient selection.

Potential Market Limitations

• Emerging Therapies: The arrival of newer ADCs with better tolerability or efficacy, such as trastuzumab deruxtecan, could limit T-DM1's growth.
• Resistance Development: Understanding and overcoming mechanisms of resistance remains critical for sustained market relevance.

Strategic Opportunities

• Adjuvant and Neoadjuvant Expansion: Continued evidence supporting earlier interventions will increase prescription volumes.
• Combination Regimens: Integrated therapies with immune checkpoint inhibitors show promise, enhancing therapeutic outcomes and market share.
• Geographic Diversification: Penetrating emerging markets via strategic partnerships can unlock new revenue streams.

Conclusion

Ado-trastuzumab emtansine continues to solidify its position as a cornerstone in HER2-positive breast cancer therapy. Ongoing clinical trials promise to expand its indications, improve its safety profile, and refine its role within combination therapies. Market-wise, Kadcyla® maintains steady growth, with future prospects buoyed by regulatory expansions and therapeutic innovations. However, competition and resistance mechanisms pose ongoing challenges requiring strategic adaptation.

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Key Takeaways

• Clinical Validation: The KATHERINE trial confirms T-DM1’s efficacy as adjuvant therapy, potentially revolutionizing early HER2-positive breast cancer treatment.
• Market Leadership: Kadcyla® holds a strong position driven by expanding indications, but faces competition from newer ADCs like trastuzumab deruxtecan.
• Growth Drivers: Expansion into early-stage settings, combination therapies, and personalized medicine will underpin future market growth.
• Challenges: High costs, toxicity concerns, and emerging competitors necessitate ongoing innovation and strategic positioning.
• Strategic Outlook: Emphasizing biomarker-driven patient selection, international expansion, and therapeutic combinations will be critical for maximizing clinical and commercial potential.

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Frequently Asked Questions

1. What are the primary indications for ado-trastuzumab emtansine?
Currently approved for HER2-positive metastatic breast cancer, especially after trastuzumab and taxane therapy, and as adjuvant therapy in early breast cancer following the KATHERINE trial.

2. How does T-DM1 compare to other HER2-targeted therapies?
T-DM1 combines targeted antibody therapy with a cytotoxic payload, offering a favorable safety profile and proven efficacy in resistant cases, though newer agents like trastuzumab deruxtecan demonstrate higher response rates but also pose different toxicity considerations.

3. What are the major side effects associated with T-DM1?
Common adverse effects include fatigue, nausea, elevated liver enzymes, and thrombocytopenia. Cardiotoxicity remains a concern but is less prevalent than with trastuzumab alone.

4. Which upcoming clinical trial results could influence T-DM1’s market position?
Results from trials exploring T-DM1 in early-stage disease, combination with immunotherapies, and in other HER2-expressing cancers could significantly expand its therapeutic footprint.

5. What is the outlook for T-DM1’s market growth over the next five years?
Projected growth is optimistic, with an estimated CAGR of 8-10%, driven by indication expansion, combination strategies, and geographic penetration, barring unforeseen competitive disruptions.

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References

[1] von Minckwitz G, et al. "Adjuvant Trastuzumab Emtansine in HER2-Positive Early Breast Cancer." New England Journal of Medicine, 2019.

[2] Geyer CE, et al. "Evaluation of HER2-Targeted Antibody–Drug Conjugates in Ovarian and Gastric Cancers." Journal of Clinical Oncology, 2021.

[3] IQVIA. "Pharmaceutical Market Reports," 2022.