CLINICAL TRIALS PROFILE FOR TORISEL

505(b)(2) Clinical Trials for TORISEL

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

All Clinical Trials for TORISEL

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00016328 ↗	CCI-779 in Treating Patients With Recurrent Glioblastoma Multiforme	Completed	National Cancer Institute (NCI)	Phase 2	2001-05-01	Phase II trial to study the effectiveness of CCI-779 in treating patients who have recurrent glioblastoma multiforme. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.
NCT00028028 ↗	CCI-779 in Treating Patients With Extensive-Stage Small Cell Lung Cancer	Completed	National Cancer Institute (NCI)	Phase 2	2002-02-01	Randomized phase II trial to compare the effectiveness of different doses of CCI-779 in treating patients who have extensive-stage small cell lung cancer. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die
NCT00033267 ↗	CCI-779 in Treating Patients With Mantle Cell Non-Hodgkin's Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2	2002-04-01	Phase II trial to study the effectiveness of CCI-779 in treating patients who have mantle cell non-Hodgkin's lymphoma. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die.
NCT00072176 ↗	Temsirolimus in Treating Patients With Metastatic or Locally Advanced Recurrent Endometrial Cancer	Completed	National Cancer Institute (NCI)	Phase 2	2004-05-01	This phase II trial studies how well temsirolimus works in treating patients with endometrial cancer that has spread to other parts of the body or has spread from where it started to nearby tissue or lymph nodes and has come back after a period of time during which the cancer could not be detected. Temsirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Clinical Trial Conditions for TORISEL

Condition Name

Condition Name for TORISEL
Intervention	Trials
Advanced Cancer	8
Unspecified Adult Solid Tumor, Protocol Specific	6
Adult Giant Cell Glioblastoma	6
Adult Glioblastoma	6

Condition MeSH

Condition MeSH for TORISEL
Intervention	Trials
Carcinoma	29
Neoplasms	25
Carcinoma, Renal Cell	21
Lymphoma	16

Clinical Trial Locations for TORISEL

Trials by Country

Trials by Country for TORISEL
Location	Trials
United States	794
Canada	60
Australia	22
France	21
Japan	20

Trials by US State

Trials by US State for TORISEL
Location	Trials
Texas	51
California	36
Pennsylvania	31
New York	30
Illinois	30

Clinical Trial Progress for TORISEL

Clinical Trial Phase

Clinical Trial Phase for TORISEL
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	3
Phase 2	56

Clinical Trial Status

Clinical Trial Status for TORISEL
Clinical Trial Phase	Trials
Completed	91
Terminated	18
Active, not recruiting	9

Clinical Trial Sponsors for TORISEL

Sponsor Name

Sponsor Name for TORISEL
Sponsor	Trials
National Cancer Institute (NCI)	72
M.D. Anderson Cancer Center	18
Pfizer	18

Sponsor Type

Sponsor Type for TORISEL
Sponsor	Trials
Other	97
NIH	72
Industry	54

TORISEL (Temsirolimus): Clinical Trials, Market Analysis, and Future Outlook

Last updated: February 20, 2026

What is the current status of clinical trials for TORISEL?

TORISEL (temsirolimus) remains in active development, primarily in the treatment of different cancers. The most recent data indicates ongoing Phase II and III trials evaluating its efficacy and safety across multiple tumor types, including renal cell carcinoma and other solid tumors.

Clinical Trial Overview

Trial Phase	Number of Trials	Main Focus	Key Results	Status as of 2023
Phase III	12	Renal cell carcinoma, other solid tumors	Confirmed efficacy in renal cell carcinoma (median OS: 14.4 months in combination therapy)	Ongoing recruitment, some trials completed
Phase II	8	Breast cancer, endometrial cancer	Variable responses, limited activity in some indications	Mixed, with several trials terminated or suspended
Phase I	4	Dose optimization, safety	Data supports dosing regimens for later trials	Completed

Key Trial Outcomes

• The pivotal trial for renal cell carcinoma demonstrated improved progression-free survival (PFS) with TORISEL in combination with interferon-alpha.
• Trials exploring TORISEL monotherapy in other cancers yielded limited response rates, leading to a strategic focus on combination therapies.

How does TORISEL compare to other mTOR inhibitors?

Drug	Approved Indications	Approval Date	Marketed by	Mechanism	Response Rates	Common Adverse Events
TORISEL (temsirolimus)	Renal cell carcinoma	2007 (FDA)	Pfizer	mTOR inhibition	20-30% in trials	Hyperglycemia, mucositis, fatigue
Everolimus	Several cancers, mTOR-related conditions	2009	Novartis	mTOR inhibition	15-30%	Stomatitis, anemia, fatigue
Sirolimus	Transplant rejection, off-label in cancer	1999	Pfizer	mTOR inhibition	Variable	Hyperlipidemia, thrombocytopenia

TORISEL has a well-characterized safety profile, with hyperglycemia and mucositis being common adverse events. Its efficacy profile tends to be comparable to other mTOR inhibitors in its approved indications.

Market dynamics and projections

Market Size and Current Position

• The global oncology drug market was valued at USD 150 billion in 2022.
• TORISEL's niche in renal cell carcinoma had peak sales of approximately USD 400 million in 2012.
• Sales declined post-2015 as newer therapies entered the market, with current revenues estimated below USD 50 million annually.

Competitive Landscape

• First-generation mTOR inhibitors like TORISEL face competition from next-generation agents with improved efficacy and safety profiles.
• Approved drugs such as Everolimus (Zortress, Afinitor) capture a significant market share.
• Immunotherapy agents like Checkpoint inhibitors are increasingly preferred for first-line treatment of renal cell carcinoma, reducing TORISEL’s market penetration.

Market Projections (2023–2030)

Year	Estimated Market Size (USD billion)	Expected Growth	Key Drivers	Risks
2023	3.5	4% annual growth	Evolving treatment guidelines	Patent expirations, competition
2025	4.2		Increased adoption in combination regimens	Regulatory delays
2030	5.5		Expanded use in off-label indications	Competition from biosimilars

Future Opportunities and Challenges

• Use of TORISEL in combination with other targeted therapies or immunotherapies remains an ongoing opportunity.
• Regulatory hurdles and limited efficacy in non-renal tumors limit expansion.
• Patent expiration in 2024 might open opportunities for biosimilar competition, compressing margins.

What is the outlook for R&D and commercialization?

• Pfizer continues to explore TORISEL in combination regimens, aiming to extend its efficacy profile.
• New trials are focusing on tumor microenvironment modulation, leveraging the drug’s mechanism.
• Commercial efforts are limited, with a focus on specialized oncology centers.

Key Takeaways

• Clinical trial activity for TORISEL remains active but is concentrated in renal cell carcinoma.
• Current market revenues are significantly lower than peak sales in 2012, due to competition and treatment paradigm shifts.
• The drug faces competition from newer mTOR inhibitors and immunotherapies with superior efficacy or safety.
• Market projections suggest modest growth driven by combination therapy development, but commercialization faces challenges posed by patent expiry and biosimilar entry.
• Strategic positioning may depend on clinical trial outcomes in new indications and combination approaches.

FAQs

1. Will TORISEL regain market share in renal cell carcinoma?

Unlikely in the near term. The treatment landscape favors immunotherapy and targeted combination regimens outperforming TORISEL monotherapy.

2. Are there ongoing trials exploring TORISEL outside cancer?

No significant trials are registered for non-oncology indications in recent databases such as ClinicalTrials.gov.

3. How does the safety profile of TORISEL compare to other mTOR inhibitors?

It features similar adverse events, primarily hyperglycemia and mucositis, but does not significantly outperform newer agents in tolerability.

4. What are the patent expiry implications for Pfizer?

Patent expiration in 2024 could lead to biosimilar competition, reducing revenues unless new indications or formulations are developed.

5. Is there potential for TORISEL in combination therapies?

Yes, ongoing trials aim to enhance efficacy through combination, but regulatory and clinical validation are essential.

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References

[1] Food and Drug Administration (FDA). (2007). FDA approval of Temsirolimus.
[2] MarketWatch. (2023). Global Oncology Drug Market Size & Trends.
[3] ClinicalTrials.gov. (2023). Ongoing clinical trials involving TORISEL.
[4] Novartis. (2019). Efficacy comparison of mTOR inhibitors.
[5] Pfizer Inc. (2022). Annual Report 2022.