CLINICAL TRIALS PROFILE FOR TEMSIROLIMUS

505(b)(2) Clinical Trials for Temsirolimus

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Indication	NCT02093598 ↗	POEM STUDY: A Phase IIa Trial in Endometrial Carcinoma With Temsirolimus	Completed	MedSIR	Phase 2	2012-05-01	Type of Application: Clinical trial of new indication. Experimental drug: The study dose of temsirolimus will be 25 mg administered intravenously, infused over a 30- to 60-minute period once weekly for 28 days (Total doses: 4 doses). Temsirolimus is a selective inhibitor of mTOR (mammalian target of rapamycin). Pharmacotherapeutic group: Protein Kinase Inhibitors; ATC code: L01X E09. Primary Objective: - To identify in tumor samples future biomarkers associated with a short term exposure to temsirolimus. - This is an exploratory clinical study. No efficacy objectives are included in this clinical trial. Secondary Objectives: - To estimate the tolerability for all temsirolimus-treated patients throughout the study and up to 28 days after the last dose of temsirolimus. - To correlate observed changes with the different type of endometrial carcinoma (type I and type II), with regard to proteins related to mTOR (p4EBP1, pS6K1, c-MYC, cyclin D, p27, BAD, p53, Bcl-2 PTEN, pAKT, mTOR), - To estimate the potential predictive value of some biomarkers (immunostaining for PTEN, pAKT, mTOR), relevant mutations in PTEN, PI3KCA, k-RAS, CTNNB1, and microsatellite instability status. - To estimate the prognostic value of Ki67 expression after short-term presurgical therapy exposure - To collect data about the differences in expression profile, assessed by RNA microarrays
New Combination	NCT03571438 ↗	Evaluation of a Promising New Combination of Protein Kinase Inhibitors on Organotypic Cultures of Human Renal Tumors	Recruiting	University Hospital, Grenoble	N/A	2017-10-16	The investigators objective is to test the combination directly on organotypic cultures of tumors from patients after their excision in the Department of Urology and Renal Transplantation of the University Hospital of Grenoble and to compare their efficacy with that of currently selected treatments in the clinic. The population targeted by the combination for use in clinical practice is patients with metastatic clear cell renal cell carcinoma. Current treatments for these patients are Sunitinib, Pazopanib and Temsirolimus.

All Clinical Trials for Temsirolimus

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00003712 ↗	CCI-779 in Treating Patients With Advanced Solid Tumors	Completed	National Cancer Institute (NCI)	Phase 1	2001-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of CCI-779 in treating patients who have advanced solid tumors.
NCT00003712 ↗	CCI-779 in Treating Patients With Advanced Solid Tumors	Completed	University of Texas	Phase 1	2001-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of CCI-779 in treating patients who have advanced solid tumors.
NCT00003712 ↗	CCI-779 in Treating Patients With Advanced Solid Tumors	Completed	Wyeth is now a wholly owned subsidiary of Pfizer	Phase 1	2001-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of CCI-779 in treating patients who have advanced solid tumors.
NCT00003712 ↗	CCI-779 in Treating Patients With Advanced Solid Tumors	Completed	The University of Texas Health Science Center at San Antonio	Phase 1	2001-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of CCI-779 in treating patients who have advanced solid tumors.
NCT00012142 ↗	CCI-779 in Treating Patients With Prostate Cancer	Completed	National Cancer Institute (NCI)	Phase 2	2000-09-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Randomized phase II trial to determine the effectiveness of CCI-779 in treating patients who have progressive prostate cancer.

Clinical Trial Conditions for Temsirolimus

Condition Name

Condition Name for Temsirolimus
Intervention	Trials
Renal Cell Carcinoma	13
Unspecified Adult Solid Tumor, Protocol Specific	10
Advanced Cancer	10
Kidney Cancer	8

Condition MeSH

Condition MeSH for Temsirolimus
Intervention	Trials
Carcinoma	51
Carcinoma, Renal Cell	42
Neoplasms	38
Lymphoma	25

Clinical Trial Locations for Temsirolimus

Trials by Country

Trials by Country for Temsirolimus
Location	Trials
United States	980
Canada	104
Germany	37
Australia	29
France	29

Trials by US State

Trials by US State for Temsirolimus
Location	Trials
Texas	62
California	48
New York	43
Pennsylvania	42
Illinois	40

Clinical Trial Progress for Temsirolimus

Clinical Trial Phase

Clinical Trial Phase for Temsirolimus
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	2
Phase 3	9

Clinical Trial Status

Clinical Trial Status for Temsirolimus
Clinical Trial Phase	Trials
Completed	139
Terminated	31
Active, not recruiting	15

Clinical Trial Sponsors for Temsirolimus

Sponsor Name

Sponsor Name for Temsirolimus
Sponsor	Trials
National Cancer Institute (NCI)	86
Pfizer	33
Wyeth is now a wholly owned subsidiary of Pfizer	24

Sponsor Type

Sponsor Type for Temsirolimus
Sponsor	Trials
Other	185
Industry	111
NIH	87

Clinical Trials Update, Market Analysis, and Future Projection for Temsirolimus

Last updated: October 28, 2025

Introduction

Temsirolimus, marketed under the brand name Torisel, is an mTOR (mammalian target of rapamycin) inhibitor developed primarily for the treatment of various cancers. Originally approved by the FDA in 2007 for advanced renal cell carcinoma (RCC), its therapeutic potential extends into other oncological indications. As cancer treatments evolve with targeted therapies, understanding Temsirolimus’s current clinical development, market positioning, and future outlook is critical for stakeholders ranging from pharmaceutical companies to investors.

Clinical Trials Update

Current Clinical Development Landscape

Temsirolimus’s clinical development pipeline has seen a strategic shift from initial indications like RCC toward exploring its efficacy across a broader spectrum of cancers, including melanoma, breast cancer, and atypical teratoid/rhabdoid tumors. According to ClinicalTrials.gov, as of late 2022, there are approximately 10 active or recruiting trials investigating Temsirolimus in various settings.

Notable Trials and Results

• Combination Therapy in Solid Tumors: A phase 1/2 trial (NCT03453203) evaluated Temsirolimus with everolimus in advanced solid tumors, showing some promise in overcoming resistance mechanisms seen with mTOR inhibition alone.

• Breast Cancer: A phase 2 study (NCT02364935) assessed Temsirolimus in triple-negative breast cancer (TNBC) with limited efficacy, highlighting the need for biomarker-driven patient stratification.

• Pediatric Tumors: Trials, such as NCT01351350, explore Temsirolimus in pediatric atypical teratoid/rhabdoid tumors, demonstrating a modest response rate, reinforcing its potential in specific subpopulations.

Regulatory Activities and Outcomes

While the FDA approved Temsirolimus for RCC, recent regulatory activities focus on its investigational use in combination therapies for refractory cancers. Some trials have faced challenges with limited efficacy signals, leading to early terminations or pauses, such as the shut down of certain pediatric studies due to insufficient responses.

Emerging Data and Future Directions

Preliminary data suggest that combining Temsirolimus with immune checkpoint inhibitors (e.g., nivolumab) may enhance therapeutic activity, especially in highly resistant tumors. These innovative combination strategies are currently under Phase 1/2 evaluation, with outcomes anticipated in 2023–2024.

Market Analysis

Current Market Position

Temsirolimus’s first-mover advantage in RCC helped establish a profitable niche. However, the landscape has become highly competitive with the advent of newer targeted agents and immune therapies, such as cabozantinib, nivolumab, and pembrolizumab, which have shown superior efficacy profiles and broader indications.

Market Size and Revenue

According to global market research reports, the oncology mTOR inhibitors segment was valued at approximately $2.5 billion in 2022 and is projected to grow at a CAGR of around 8% through 2030[1]. Temsirolimus’s revenue contribution has waned, attributed to:

• Increased competition.
• Expiration of patents in some markets, fostering biosimilar entry.
• Limited pipeline expansion.

Competitive Landscape

Key competitors include everolimus (Afinitor), which has a broader approved indication spectrum and more extensive clinical data. The rise of immunotherapies has also reduced reliance on mTOR inhibitors for certain cancers, impacting market share.

Distribution and Market Access

While Temsirolimus retains approval for RCC, its usage is largely confined to specialized oncology centers. Reimbursement policies and trial outcomes influence its market penetration further, especially in regions with cost-sensitive healthcare systems.

Market Projection and Future Outlook

Growth Drivers

• Emerging Combination Regimens: Trials combining Temsirolimus with immune checkpoint inhibitors and other targeted agents could rejuvenate interest, especially if positive results support regulatory approval.
• Rare and Pediatric Cancers: Narrow indications such as atypical teratoid/rhabdoid tumors remain niche but could offer high-value opportunities pending better trial outcomes.

Challenges

• Market Saturation: Dominance of newer agents with better efficacy profiles puts pressure on Temsirolimus’s market share.
• Limited Patent Life: Patent expiration reduces exclusivity, enabling biosimilar competition.
• Efficacy Limitations: Modest activity in some cancers restricts broader adoption.

Forecast (2023–2030)

Projections estimate that Temsirolimus’s global sales will decline marginally until 2025 but could stabilize or slightly increase if successful in novel combination trials. Overall, its role will likely shift from a first-line option to a niche agent within combination regimens or in rare cancer indications, with annual revenues potentially remaining below $300 million in mature markets.

Strategic Considerations

Pharmaceutical developers are evaluating re-synthesis of Temsirolimus for precision oncology, focusing on biomarkers for response prediction. Partnering with biotech firms or academic institutions for innovative trials could extend its lifecycle.

Key Takeaways

• Clinical Pipeline Reality: Temsirolimus’s ongoing trials reflect an emphasis on combination therapies and rare tumors. While existing data is mixed, innovative strategies could unlock new avenues.
• Market Position: The drug faces stiff competition from more versatile mTOR inhibitors and immune therapies. Its niche status persists in specific oncology settings.
• Growth Opportunities: Success hinges on bridging efficacy gaps via biomarker-driven patient selection and strategic collaborations.
• Challenges: Patent expirations and market saturation forecast a decline in prominence without significant trial-driven breakthroughs.
• Future Outlook: Focused development in niche indications and combination regimens may sustain modest growth, but the global market for Temsirolimus is expected to contract relative to its peak years.

FAQs

1. What are the primary current indications for Temsirolimus?
   Temsirolimus remains approved mainly for advanced renal cell carcinoma (RCC), with investigational use in other cancers like breast cancer, melanoma, and pediatric tumors.

2. Have recent clinical trials demonstrated significant efficacy?
   Overall, recent trials have shown limited efficacy in unselected patient populations, but biomarker-driven approaches and combination therapies hold promise for improved outcomes.

3. What factors threaten Temsirolimus’s market future?
   Market threats include newer, more effective therapies, patent expirations leading to biosimilar competition, and limited success in expanding indications.

4. Is Temsirolimus effective when combined with other therapies?
   Emerging evidence suggests potential benefits when combined with immune checkpoint inhibitors, but definitive clinical validation is pending.

5. What strategic moves could extend Temsirolimus's lifecycle?
   Developing biomarker-based patient selection, engaging in strategic partnerships for novel combination trials, and targeting niche, hard-to-treat cancers could prolong its relevance.

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Sources:

[1] MarketWatch. "Global mTOR inhibitors Market Size, Share & Trends Analysis Report." 2022.