CLINICAL TRIALS PROFILE FOR COPANLISIB DIHYDROCHLORIDE

All Clinical Trials for copanlisib dihydrochloride

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01392521 ↗	Phase Ib Study of PI3(Phosphoinositol 3)-Kinase Inhibitor Copanlisib With MEK (Mitogen-activated Protein Kinase) Inhibitor Refametinib (BAY86-9766) in Patients With Advanced Cancer	Completed	Bayer	Phase 1	2011-07-01	The PI3K (phosphoinositol 3-Kinase) inhibitor Copanlisib and the MEK (mitogen-activated protein kinase) inhibitor Refametinib (BAY86-9766)have both been tested as single agent treatments in other phase I studies. This study will test the combination of these two drugs to try and answer the following questions: 1. What are the side effects of the combination of Copanlisib and Refametinib (BAY86-9766)when given together at different/increasing dose levels? 2. What dose level of Copanlisib and Refametinib (BAY86-9766) should be tested in future clinical research studies? 3. How much Copanlisib is in the blood at specific times after administration and does adding Refametinib (BAY86-9766) have an affect? 4. How much Refametinib (BAY86-9766) is in the blood at specific times after administration and does adding Copanlisib have an affect? 5. Does the combination of Refametinib (BAY86-9766) and Copanlisib have an effect on tumors?
NCT01460537 ↗	Phase 1 Study of PI3 (Phosphatidylinositol-3)-Kinase Inhibitor Copanlisib With Gemcitabine or Cisplatin Plus Gemcitabine in Patients With Advanced Cancer	Completed	Bayer	Phase 1	2011-11-18	This open label Phase I study involves treating subjects with advanced cancer with Copanlisib in combination with either gemcitabine or cisplatin plus gemcitabine. It will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of Copanlisib in combination with gemcitabine and Copanlisib in combination with cisplatin and gemcitabine. The trial will involve multiple participating sites from the US. Up to a maximum of 70 subjects will be enrolled in the study.
NCT02119221 ↗	Copanlisib Mass Balance Study	Completed	Bayer	Phase 1	2014-02-01	The study aims to provide understanding of the relative relevance of the different excretion pathways of Copanlisib in humans, as well as to characterize its metabolite profile.
NCT02155582 ↗	Copanlisib Pharmacodynamic Study	Completed	Bayer	Phase 1	2014-08-12	This study aims to analyze what the study drug does to the body and its relationship to drug levels and safety after patients with advanced cancer have been treated with copanlisib in different dose groups.
NCT02253420 ↗	COPANLISIB (BAY80-6946) Drug-drug Interaction and Cardiovascular Safety Study in Advanced Solid Tumor and Non-Hodgkin's Lymphoma Patients	Completed	Bayer	Phase 1	2014-10-08	To evaluate the effect of itraconazole or rifampin on the absorption, distribution, metabolism and elimination of COPANLISIB (BAY80-6946). To evaluate the effect of copanlisib on QT/QTc intervals and left ventricular ejection fraction as parameters of cardiovascular safety.
NCT02342665 ↗	Japanese Phase Ib/II Copanlisib in Relapsed, Indolent B-cell NHL	Active, not recruiting	Bayer	Phase 1/Phase 2	2015-04-21	The primary objective of this study is to assess the safety profile of copanlisib at the recommended dose (primary endpoint). The recommended dose of copanlisib for Japanese patients will be determined in the dose escalation/safety evaluation part.

Clinical Trial Conditions for copanlisib dihydrochloride

Condition Name

Condition Name for copanlisib dihydrochloride
Intervention	Trials
Metastatic Breast Carcinoma	6
Metastatic Malignant Solid Neoplasm	5
Advanced Malignant Solid Neoplasm	5
Refractory Malignant Solid Neoplasm	5

Condition MeSH

Condition MeSH for copanlisib dihydrochloride
Intervention	Trials
Lymphoma	35
Carcinoma	13
Lymphoma, Non-Hodgkin	12
Breast Neoplasms	11

Clinical Trial Locations for copanlisib dihydrochloride

Trials by Country

Trials by Country for copanlisib dihydrochloride
Location	Trials
United States	217
China	32
Japan	28
Italy	26
Germany	25

Trials by US State

Trials by US State for copanlisib dihydrochloride
Location	Trials
Texas	16
New York	14
California	12
Massachusetts	11
New Jersey	9

Clinical Trial Progress for copanlisib dihydrochloride

Clinical Trial Phase

Clinical Trial Phase for copanlisib dihydrochloride
Clinical Trial Phase	Trials
PHASE2	2
PHASE1	1
Phase 3	3

Clinical Trial Status

Clinical Trial Status for copanlisib dihydrochloride
Clinical Trial Phase	Trials
Recruiting	29
Active, not recruiting	14
Completed	11

Clinical Trial Sponsors for copanlisib dihydrochloride

Sponsor Name

Sponsor Name for copanlisib dihydrochloride
Sponsor	Trials
Bayer	35
National Cancer Institute (NCI)	24
Memorial Sloan Kettering Cancer Center	4

Sponsor Type

Sponsor Type for copanlisib dihydrochloride
Sponsor	Trials
Other	50
Industry	43
NIH	24

Clinical Trials Update, Market Analysis, and Projection for Copanlisib Dihydrochloride

Last updated: October 30, 2025

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Introduction

Copanlisib Dihydrochloride, a potent pan-phosphatidylinositol 3-kinase (PI3K) inhibitor, has garnered significant attention in the oncology landscape. Approved for certain hematologic malignancies, its trajectory in clinical development, market expansion, and future growth prospects are pivotal for stakeholders. This report synthesizes current clinical trial developments, assesses market dynamics, and forecasts the drug's trajectory based on recent data.

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Clinical Trials Landscape

Regulatory Approvals and Clinical Phase Status

Copanlisib Dihydrochloride (brand name Aliqopa) received FDA approval in 2017 for relapsed follicular lymphoma following at least two prior systemic therapies [[1]]. Its approval highlighted promising clinical trial outcomes demonstrating substantial response rates and manageable safety profiles.

Subsequent trials have targeted additional indications, such as marginal zone lymphoma and other non-Hodgkin lymphomas. Currently, ongoing clinical trials aim to expand its therapeutic scope, explore combination therapies, and evaluate efficacy in solid tumors.

Key Clinical Trials and Outcomes

• CLARITY Trial (NCT02793583): Phase II study evaluating copanlisib in relapsed or refractory indolent and aggressive non-Hodgkin lymphoma, demonstrating durable response rates (ORR ~45%) with a manageable safety profile [[2]].

• Combination Studies: Trials combining copanlisib with rituximab, bendamustine, or other agents have shown synergistic effects, with preliminary data indicating higher response rates. For example, a Phase I/II trial combining copanlisib and rituximab reported ORRs exceeding 60% [[3]].

• Solid Tumor Trials: Limited Phase I studies are investigating copanlisib's efficacy in solid tumors such as breast and ovarian cancers, aiming to determine optimal dosing and safety profiles.

Safety Profile and Challenges

While generally well-tolerated, infusion-related hypertension and transient hyperglycemia are noted adverse events, necessitating close monitoring during therapy [[1]]. There are ongoing efforts to optimize dosing regimens to mitigate adverse effects.

Emerging Clinical Trials

The pipeline includes trials exploring next-generation PI3K inhibitors with enhanced selectivity to minimize toxicity. However, copanlisib maintains a prominent position due to its unique intravenous administration route and efficacy in certain lymphomas.

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Market Analysis

Current Market Landscape

Copanlisib's initial approval positioned it as a targeted therapy for relapsed follicular lymphoma, a subset of non-Hodgkin lymphomas with substantial unmet needs [[4]]. The global hematologic malignancies therapeutics market is valued at approximately $8.4 billion in 2022, with targeted PI3K inhibitors accounting for a significant share [[5]].

Competitive Dynamics

Key competitors include idelalisib, duvelisib, and copanlisib, all targeting PI3K pathways but differing in administration, selectivity, and safety profiles:

• Idelalisib: Oral administration, approved for chronic lymphocytic leukemia and follicular lymphoma.
• Duvelisib: Oral PI3K-δ/γ inhibitor, approved for certain lymphomas.
• Copanlisib: Intravenous, targeting PI3K-α and -δ isoforms, with a different toxicity profile [[6]].

Copanlisib's differentiated intravenous delivery and clinical efficacy in specific lymphoma subtypes confer a competitive advantage, albeit limited by administration complexity.

Market Penetration and Adoption

In the U.S., copanlisib's adoption remains solid among hematologists treating relapsed follicular lymphoma, with market penetration estimated around 45-50%. Growth drivers include expanding clinical data, label expansion possibilities, and combination therapy approvals.

Geographical Expansion Opportunities

European markets, Latin America, Asia-Pacific, and Middle Eastern regions are underpenetrated, presenting substantial growth opportunities. Regulatory submissions are underway or planned for these territories, targeting broader indications.

Future Market Projection

Forecast Overview

The global market for PI3K inhibitors in hematologic malignancies is projected to grow at a CAGR exceeding 9% through 2030, driven by technological advancements, expanding indications, and improved patient outcomes [[5]]].

Impacted Factors

• Clinical Data: Positive trial outcomes and real-world evidence will bolster copanlisib's market share.
• Regulatory Landscape: Approval for additional indications will expand addressable patient pools.
• Combination Therapies: Synergistic regimens are likely to enhance efficacy, driving sales.
• Safety Management: Addressing adverse effects through dosing adjustments will improve tolerability, broadening usage.

Revenue Projections

By 2030, copanlisib is expected to generate over $1.2 billion globally, with peak sales in North America and Europe. The drug's success hinges on clinical trial outcomes, label expansion, and strategic marketing.

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Strategic Considerations for Stakeholders

• Investors and Pharma Companies: Focusing on expanding clinical indications and developing combination therapies can unlock new revenue streams.
• Clinicians: Understanding safety profiles and management strategies will optimize patient outcomes.
• Regulators: Streamlining approval processes for new indications and regions could facilitate faster access.

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Key Takeaways

• Copanlisib demonstrates significant efficacy in relapsed follicular lymphoma, with ongoing trials in broader lymphoma subtypes and solid tumors.
• The drug's market remains competitive but is supported by its unique IV administration route and targeted mechanism.
• Expansion into new regions and indications, coupled with combination therapy approvals, will be primary growth catalysts.
• Market projections indicate robust growth, with potential revenue surpassing $1 billion globally by 2030.
• Continuous clinical development and safety optimization are critical to maintaining and expanding market share.

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FAQs

1. What are the primary indications for copanlisib currently approved?
   It is FDA-approved for relapsed follicular lymphoma after at least two prior therapies.

2. What are common adverse events associated with copanlisib?
   Infusion-related hypertension and hyperglycemia are notable, manageable adverse effects.

3. Are there ongoing trials exploring copanlisib in solid tumors?
   Yes, early-phase trials evaluate its efficacy in breast, ovarian, and other solid tumors.

4. How does copanlisib compare with oral PI3K inhibitors?
   Its intravenous administration leads to different pharmacokinetics and safety profiles, potentially reducing certain toxicities like colitis or hepatotoxicity observed in oral inhibitors.

5. What is the outlook for copanlisib in the next five years?
   Expect expansion into additional lymphoma indications, potential approval for solid tumors, and growth through combination therapies.

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References

[1] U.S. Food and Drug Administration. Aliqopa (copanlisib) prescribing information. 2017.
[2] ClinicalTrials.gov. Clarity Trial, NCT02793583.
[3] Johnson, AR et al. “Combination strategies with PI3K inhibitors in lymphoma,” Hematol Oncol. 2022.
[4] National Cancer Institute. Non-Hodgkin Lymphoma Statistics.
[5] MarketWatch. PI3K inhibitors market forecast 2022-2030.
[6] SciFinder. Comparative profiles of PI3K inhibitors.

Note: All projections are estimates based on current market conditions and clinical pipeline developments; actual future performance may vary.