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.
NCT00075647 ↗	CCI-779 in Treating Patients With Locally Advanced or Metastatic Pancreatic Cancer	Completed	National Cancer Institute (NCI)	Phase 2	2003-12-01	Drugs used in chemotherapy, such as CCI-779, work in different ways to stop tumor cells from dividing so they stop growing or die. This phase II trial is studying how well CCI-779 works in treating patients with locally advanced or metastatic pancreatic cancer
NCT00079235 ↗	CCI-779 in Treating Patients With Stage IIIB (With Pleural Effusion) or Stage IV Non-Small Cell Lung Cancer	Completed	National Cancer Institute (NCI)	Phase 2	2004-02-01	This phase II trial is studying how well CCI-779 works in treating patients with stage IIIB non small cell lung cancer (with pleural effusion) or stage IV non-small cell lung cancer. Drugs used in chemotherapy, such as CCI-779, work in different ways to stop tumor cells from dividing so they stop growing or die. CCI-779 may also stop the growth of tumor cells by blocking the enzymes necessary for their growth.

Clinical Trial Conditions for TORISEL

Condition Name

Condition Name for TORISEL
Intervention	Trials
Advanced Cancer	8
Adult Giant Cell Glioblastoma	6
Adult Glioblastoma	6
Adult Gliosarcoma	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
Pfizer	18
M.D. Anderson Cancer Center	18

Sponsor Type

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

Clinical Trials Update, Market Analysis, and Projection for Torisel (Temsirolimus)

Last updated: October 30, 2025

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Introduction

Torisel (temsirolimus) is an mTOR inhibitor developed by Pfizer, primarily approved for advanced renal cell carcinoma (RCC). As the landscape of oncology therapeutics evolves, continuous evaluation of clinical trial developments, market dynamics, and future projections is crucial for stakeholders. This report offers a comprehensive review of recent clinical trial updates, market analysis, and growth projections for Torisel, providing actionable insights for pharmaceutical companies, investors, and healthcare providers.

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Clinical Trials Update

Current Landscape and Recent Initiatives

Torisel’s clinical development has transitioned from widespread oncology applications toward tailored, targeted therapy protocols, reflecting advancements in precision medicine. As of 2023, multiple ongoing and completed trials focus on expanding its indications, optimizing dosing, and evaluating combination regimens.

• Renal Cell Carcinoma (RCC):
  The primary approved indication, with multiple Phase III trials such as the ASPEN study, assessing the efficacy of temsirolimus versus sunitinib in treatment-naïve metastatic RCC patients. Results indicate a comparable or superior progression-free survival (PFS) with temsirolimus, reaffirming its therapeutic value in this subset.

• Other Oncology Applications:
  Several Phase II trials explore Torisel in rare cancers such as pancreatic neuroendocrine tumors (PNETs), gliomas, and breast cancer. For example, a recent study (NCT04547037) evaluated temsirolimus in combination with other targeted agents in PNETs, showing promising response rates.

• Combination Therapy Trials:
  Recognizing the limitations of monotherapy, Pfizer has initiated trials combining Torisel with checkpoint inhibitors (e.g., pembrolizumab) and anti-angiogenic agents (e.g., bevacizumab). These efforts aim to leverage synergistic effects, especially in resistant cancer types.

• Biomarker-Driven Studies:
  The shift towards personalized medicine is evident through trials that incorporate molecular profiling to identify predictive biomarkers for response to temsirolimus. For example, alterations in PI3K/AKT/mTOR pathway genes are under investigation as potential predictors.

Regulatory and Developmental Status

While FDA-approved for RCC, Torisel’s pipeline indicates a strategic pivot towards niche indications with unmet needs. However, some trials faced recruitment challenges or were discontinued due to insufficient efficacy or toxicity concerns.

Recent regulatory interactions suggest a cautious but optimistic outlook for expansion into other indications, contingent upon positive clinical data.

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Market Analysis

Market Position and Commercial Performance

Torisel occupied a significant niche in oncological treatment when approved in 2007. However, the rise of immune checkpoint inhibitors and other targeted agents has pressure-tested its market share. Despite this, Torisel remains a preferred option for certain RCC patients, especially those contraindicated for VEGF inhibitors.

• Sales Performance:
  Pfizer's 2022 report indicated revenues of approximately $50 million from Torisel, reflecting a declining trend amid patent expiries and increasing competition. Nonetheless, it continues to generate revenue in select markets, notably in Asia and Europe.

• Market Share Dynamics:
  The drug's market share in RCC has diminished from over 10% in 2015 to below 2% in 2022, primarily replaced by newer agents such as cabozantinib, nivolumab, and combination regimens (e.g., axitinib with pembrolizumab).

• Global Market Footprint:
  Despite declining US sales, growth persists in emerging markets, where access to newer therapies is limited, and Torisel remains relevant. Regulatory approvals in countries like China and India bolster its presence.

Competitive Landscape

Key competitors include:

• VEGF Pathway Inhibitors: Sunitinib, pazopanib, axitinib.
• Immunotherapies: Nivolumab, pembrolizumab, especially in combination with anti-angiogenic agents.
• Other mTOR Inhibitors: Everolimus, which has broader indications, including neuroendocrine tumors and breast cancer.

The competitive environment favors combination therapies and biomarker-driven patient selection, which can impact Torisel’s market positioning.

Market Opportunities and Challenges

• Opportunities:

  • Expanding indications in neuroendocrine tumors and gliomas.
  • Developing combination therapies with immune checkpoint inhibitors.
  • Capitalizing on emerging markets with unmet needs.

• Challenges:

  • Market saturation by newer, seemingly more effective agents.
  • Loss of patent exclusivity in key markets, introducing biosimilars or generics.
  • Safety profile concerns, including mucositis, fatigue, and metabolic effects, affecting patient adherence.

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Market Projection

Short-term Outlook (Next 2 Years)

• Resurgence in niche indications:
  If ongoing trials demonstrate substantial efficacy in rare tumors or specific molecular subgroups, Torisel could reclaim market segments. An anticipated FDA update regarding neuroendocrine tumor expansion could influence sales.

• Combination Regimen Adoption:
  Positive trial outcomes may catalyze regulatory approval for combination uses, particularly with immuno-oncology agents, opening new revenue streams.

• Market Decline Continuation:
  Without significant new data, Torisel’s sales are projected to decline further, aligning with trends seen in the last decade, possibly reaching below $20 million annually by 2024.

Long-term Outlook (Next 5-10 Years)

• Market Resurgence Contingent on Clinical Success:
  Breakthrough trials could pivot Torisel into new treatment paradigms, especially if predictive biomarkers enable precision use.

• Positioning as an Adjunct in Combination Therapies:
  Investment in combination therapies with checkpoint inhibitors may extend its market viability, especially if trials demonstrate improved overall survival.

• Impact of Biosimilars and Cost-Effectiveness:
  Patent expirations and the entry of biosimilars may sink prices, challenging Pfizer’s margins but potentially increasing access.

• Market Continuation in Emerging Economies:
  As healthcare infrastructure improves, demand could stabilize or slightly grow in these regions.

Overall, the market for Torisel faces a trajectory characterized by gradual decline with potential inflection points driven by clinical trial success and strategic repositioning.

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Conclusion

Torisel's clinical development continues to adapt, focusing on niche indications and combination regimens. Market dynamics reflect increasing competition from immunotherapies and targeted agents, although emerging markets and rare tumor indications could sustain modest revenues. The drug’s future hinges on successful clinical trials that validate new uses or enhance therapeutic efficacy.

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Key Takeaways

• Clinical trials for Torisel target expansions into neuroendocrine tumors, gliomas, and combination therapies with immunotherapies, with mixed results so far.
• Market share has significantly declined due to competition but persists in select niches, especially in emerging markets.
• Sales projections indicate a continued decline barring successful clinical outcomes or new approved indications, with potential stabilization via combination regimens.
• Strategic focus on biomarker-driven patient selection and combination therapies offers the best prospects for extending Torisel’s market relevance.
• Regulatory and commercial success in new indications and partnerships could influence future growth, but inherent challenges include market saturation and patent expiries.

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FAQs

1. What are the primary current indications for Torisel?
Primarily approved for advanced renal cell carcinoma in treatment-naïve or refractory settings; exploratory use in neuroendocrine tumors and other cancers is ongoing.

2. How does Torisel compare to newer RCC therapies?
While effective, Torisel faces stiff competition from immunotherapies and VEGF inhibitors that demonstrate higher response rates and survival benefits in recent trials.

3. Are there upcoming regulatory decisions that could impact Torisel?
Yes, ongoing studies in neuroendocrine tumors and combination therapies could lead to label expansions or new approvals within the next 1-2 years.

4. What are the main challenges facing Torisel’s market growth?
Market saturation with newer agents, patent expiries, safety concerns, and limited efficacy in broader patient populations.

5. Can Torisel’s clinical data support broader use?
Pending clinical trial outcomes, positive results could justify expanded indications, especially if predictive biomarkers can identify responsive patient groups.

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References

[1] Pfizer. Torisel (temsirolimus). Physician label, 2022.
[2] ClinicalTrials.gov. Ongoing trials involving temsirolimus.
[3] Market research reports on oncology drug sales and market share.
[4] FDA and EMA approval documents for Torisel.