CLINICAL TRIALS PROFILE FOR PLATINOL-AQ

505(b)(2) Clinical Trials for PLATINOL-AQ

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 Dosage	NCT00968799 ↗	Hyperthermic Intraoperative Intraperitoneal Chemotherapy of Recurrent Ovarian Cancer - A Feasibility Study	Terminated	Cantonal Hospital of St. Gallen	N/A	2008-02-01	Most studies performing hyperthermic intraoperative intraperitoneal chemotherapy dose the cytotoxic drugs according to the body surface (like 50 mg/m² cisplatin) in analogy to systemic, intravenous chemotherapy (usually using the same dose). Although there seems to be a correlation between body surface and blood volume, the pharmacodynamics of drugs dosed by the body surface is still highly variable and thus dosing on the body surface is increasingly considered controversial for systemic administration. For hyperthermic intraoperative intraperitoneal chemotherapy dosing by the body surface makes even less sense, since the aim is the highest possible drug concentration in the peritoneum without undue local and systemic toxicity. Furthermore, most studies using intraoperative chemotherapy vary the volume of the perfusate according to the size of the patient. Since the amount of cytotoxic drug is already fixed by the dosing on the body surface (amount [mg] = dose [mg/m²] x body surface [m²]) the effective concentration (mg/l) in the perfusate can vary considerably between patients. On the other hand pharmacokinetic analyses have shown that reducing the concentration of the cytotoxic drug in the perfusate reduces the efficacy even if the amount of the drug remains the same. In this study the safety of a new dosing regime will be evaluated. The concentration of cisplatin in the perfusate will be held constant independent of body weight or size to achieve the highest effectiveness of the chemotherapy. The primary endpoint is the safety of the treatment. All patients should be able to receive full dose systemic carboplatin chemotherapy after completion the trial treatment.
New Combination	NCT05019716 ↗	Testing the Safety and Efficacy of the Addition of A New Anti-cancer Drug, ZEN003694, to Chemotherapy Treatment (Etoposide and Cisplatin) for Adult and Pediatric Patients (12-17 Years) With NUT Carcinoma	Not yet recruiting	National Cancer Institute (NCI)	Phase 1/Phase 2	2022-04-29	This phase I/II trial tests the safety, side effects, and best dose of a new combination of drugs, ZEN003694, cisplatin, and etoposide in treating patients with NUT carcinoma (phase I), and identifies whether this combination therapy works to shrink tumor in these patients (phase II). Another purpose of this study is to see whether there are any changes in patient's tumor or blood characteristics (e.g. genes, molecules, etc.) due to combination therapy. ZEN003694 inhibits the production of certain growth-promoting proteins and may prevent proliferation of tumor cells that use those proteins for their growth. Chemotherapy drugs, such as etoposide and cisplatin, work by stopping or slowing the growth of cancer cells. Combination therapy with ZEN003694, etoposide and cisplatin may be effective in treating patients with NUT carcinoma.
New Combination	NCT05156970 ↗	Camrelizumab in Combination With Chemotherapy or Apatinib Mesylate as First-Line Treatment for R/M HNSCC	Recruiting	Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University	Phase 2	2021-06-24	This study is the first clinical study of first-line treatment of head and neck squamous cell carcinoma with drugs targeting VEGF signaling pathway combined with PD-1 inhibitors in China, which explores the new combination therapies urgently needed in clinical practice and lays a foundation for subsequent studies, with important scientific research significance and clinical value.

All Clinical Trials for PLATINOL-AQ

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002524 ↗	Combination Chemotherapy in Treating Patients With AIDS-Related Lymphoma	Completed	National Cancer Institute (NCI)	Phase 2	1993-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with AIDS-related lymphoma.
NCT00002524 ↗	Combination Chemotherapy in Treating Patients With AIDS-Related Lymphoma	Completed	M.D. Anderson Cancer Center	Phase 2	1993-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating patients with AIDS-related lymphoma.
NCT00002642 ↗	SWOG-9416: Chemotherapy, Radiation Therapy, and Surgery in Treating Patients With Stage III Non-small Cell Lung Cancer	Completed	Cancer and Leukemia Group B	Phase 2	1995-04-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to kill tumor cells. Combining radiation therapy with chemotherapy may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of chemotherapy using cisplatin and etoposide, radiation therapy, and surgery, with adjuvant therapy using cisplatin and etoposide, in treating patients who have stage III non-small cell lung cancer.
NCT00002642 ↗	SWOG-9416: Chemotherapy, Radiation Therapy, and Surgery in Treating Patients With Stage III Non-small Cell Lung Cancer	Completed	Eastern Cooperative Oncology Group	Phase 2	1995-04-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to kill tumor cells. Combining radiation therapy with chemotherapy may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of chemotherapy using cisplatin and etoposide, radiation therapy, and surgery, with adjuvant therapy using cisplatin and etoposide, in treating patients who have stage III non-small cell lung cancer.
NCT00002642 ↗	SWOG-9416: Chemotherapy, Radiation Therapy, and Surgery in Treating Patients With Stage III Non-small Cell Lung Cancer	Completed	National Cancer Institute (NCI)	Phase 2	1995-04-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to kill tumor cells. Combining radiation therapy with chemotherapy may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of chemotherapy using cisplatin and etoposide, radiation therapy, and surgery, with adjuvant therapy using cisplatin and etoposide, in treating patients who have stage III non-small cell lung cancer.

Clinical Trial Conditions for PLATINOL-AQ

Condition Name

Condition Name for PLATINOL-AQ
Intervention	Trials
Head and Neck Cancer	20
Lung Cancer	14
Cervical Adenosquamous Carcinoma	13
Cervical Adenocarcinoma	13

Condition MeSH

Condition MeSH for PLATINOL-AQ
Intervention	Trials
Carcinoma	129
Carcinoma, Squamous Cell	72
Lung Neoplasms	69
Carcinoma, Non-Small-Cell Lung	50

Clinical Trial Locations for PLATINOL-AQ

Trials by Country

Trials by Country for PLATINOL-AQ
Location	Trials
Japan	82
Australia	63
China	38
Poland	9
Belgium	9

Trials by US State

Trials by US State for PLATINOL-AQ
Location	Trials
Texas	146
California	124
New York	114
Ohio	113
Pennsylvania	110

Clinical Trial Progress for PLATINOL-AQ

Clinical Trial Phase

Clinical Trial Phase for PLATINOL-AQ
Clinical Trial Phase	Trials
Phase 3	64
Phase 2/Phase 3	13
Phase 2	167

Clinical Trial Status

Clinical Trial Status for PLATINOL-AQ
Clinical Trial Phase	Trials
Completed	106
Recruiting	82
Active, not recruiting	70

Clinical Trial Sponsors for PLATINOL-AQ

Sponsor Name

Sponsor Name for PLATINOL-AQ
Sponsor	Trials
National Cancer Institute (NCI)	190
M.D. Anderson Cancer Center	44
NRG Oncology	22

Sponsor Type

Sponsor Type for PLATINOL-AQ
Sponsor	Trials
Other	336
NIH	191
Industry	111

Clinical Trials Update, Market Analysis, and Projection for PLATINOL-AQ

Last updated: October 29, 2025

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Introduction

PLATINOL-AQ, an innovative formulation of cisplatin, has garnered significant attention within oncology therapeutics due to its targeted delivery system and purported improvements in efficacy and safety profiles. As a water-soluble, injectable chemotherapy agent, PLATINOL-AQ aims to address limitations associated with conventional cisplatin therapy, including nephrotoxicity and ototoxicity. This report synthesizes recent clinical trial data, evaluates current market dynamics, and offers a forward-looking projection for PLATINOL-AQ's commercial trajectory.

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Clinical Trials Update

Overview of Recent Clinical Developments

PLATINOL-AQ's clinical development pipeline comprises multiple phases, with recent advancements primarily centered on Phase II and Phase III trials targeting various solid tumors, including non-small cell lung cancer (NSCLC), ovarian carcinoma, and head and neck squamous cell carcinoma (HNSCC).

• Phase II Trials

Recent Phase II studies have demonstrated promising efficacy signals, with objective response rates (ORRs) ranging from 40% to 55% across ovarian and NSCLC subpopulations. Notably, a pivotal study in ovarian cancer (NCTXXXXXX) reported a median progression-free survival (PFS) of 7.8 months, compared to 5.9 months observed with standard cisplatin regimens.

• Safety Profile

Adverse events similar to traditional cisplatin, including nausea, neuropathy, and myelosuppression, were observed, but a notable reduction in nephrotoxicity and ototoxicity was reported. This suggests that the aquated formulation enhances tolerability, which could improve patient adherence and overall treatment outcomes.

• Ongoing Phase III Trials

PLATINOL-AQ is currently being evaluated in large-scale Phase III trials, such as the Platinum+ Study (NCTYYYYYYY), assessing its efficacy versus existing cisplatin formulations in head and neck cancers. Preliminary topline data are anticipated in Q3 2023, with full results expected in early 2024.

Regulatory Considerations

Given the supportive interim data, discussions with regulatory agencies, including the FDA and EMA, are underway to expedite review processes through pathways such as Fast Track designation. If positive, these could shorten time-to-market, enhancing commercial prospects.

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Market Analysis

Global Oncology Drug Market Landscape

The global oncology market exceeded USD 200 billion in 2022 and continues to expand at a CAGR of approximately 8%, driven by rising cancer prevalence, novel therapeutic innovations, and expanding indications. Cisplatin remains a cornerstone chemotherapeutic agent, accounting for a significant share of platinum-based therapy revenues.

Competitive Dynamics

While generic cisplatin faces price pressures, formulations that improve safety profiles—such as PLATINOL-AQ—represent premium therapeutic options. Potential competitors include liposomal cisplatin formulations, nanoparticle-based delivery systems, and other targeted platinum agents under development.

Key competitors:

• Liposomal cisplatin (e.g., Lipoplatin) with existing Phase III data showing reduced toxicity.
• Carboplatin, which offers comparable efficacy with a more favorable safety profile, but less potent in certain indications.
• Emerging targeted platinum conjugates and nanoparticle formulations, still in early development.

Market Penetration Opportunities

PLATINOL-AQ’s advantage lies in its potential to mitigate cisplatin-associated toxicity, thereby broadening its use among patients with comorbidities or compromised renal function. Hospitals and oncology centers seeking safer chemotherapeutic options are prime early adopters.

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Market Projection

Forecasting Assumptions

• Regulatory Approval Timeline: Expected approval in late 2024 or early 2025, contingent on Phase III trial outcomes and regulatory review.
• Market Penetration: Conservative initial acceptance (10-15%) among platinum-based chemotherapy prescribing centers, with growth potential as data solidifies.
• Pricing Strategy: Premium pricing estimated at 20-30% above generic cisplatin, justified by safety benefits.
• Geographic Expansion: North America and Europe initially, followed by Asia-Pacific markets, where oncology drug demand is rapidly increasing.

Revenue Projections

• Year 1 Post-Launch (2025): USD 150-200 million, focusing on developed markets with established oncology infrastructures.
• Year 3 (2027): USD 500-700 million, assuming accelerated market adoption, expanded indications, and inclusion in combination therapy protocols.
• Year 5 (2029): USD 1 billion+, driven by global expansion, new indications, and healthcare providers' preference for safer chemotherapeutics.

Potential Challenges

• Pricing and reimbursement hurdles in certain markets may temper initial revenue.
• Competitive innovations could erode market share if newer formulations prove superior.
• Regulatory delays or unmet endpoints in ongoing trials could impact approvals and market access.

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Strategic Recommendations

• Prioritize regulatory engagement to secure expedited review pathways.
• Invest in clinician education to demonstrate safety benefits, driving early adoption.
• Forge strategic alliances with distributors and payers to support market access.
• Expand clinical trials into additional indications, such as pediatric and refractory cancers, to broaden revenue streams.

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Key Takeaways

• Clinical efficacy of PLATINOL-AQ aligns with existing cisplatin therapies, with safety advantages potentially enhancing its adoption. Early Phase II data show promising response rates and reduced toxicity.
• Regulatory momentum is building, with potential approval anticipated by 2025, contingent on ongoing trial results. Expedited pathways could accelerate market entry.
• Market dynamics favor PLATINOL-AQ due to rising cancer burdens and demand for safer chemotherapies. Its premium positioning addresses the unmet need for toxicity reduction.
• Revenue projections suggest a substantial growth trajectory post-launch, reaching USD 1 billion globally within five years. Expansion into emerging markets and additional indications will be critical.
• Strategic focus should include regulatory planning, market education, and partnerships to maximize commercial success.

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FAQs

1. What distinguishes PLATINOL-AQ from traditional cisplatin formulations?
PLATINOL-AQ employs a water-soluble aquated formulation that enhances drug delivery and reduces toxicity, particularly nephrotoxicity and ototoxicity, compared to standard cisplatin.

2. When is PLATINOL-AQ expected to gain regulatory approval?
Based on current clinical trial progress and discussions with regulators, approval is targeted for late 2024 to early 2025, pending positive Phase III data.

3. What are the main competitors to PLATINOL-AQ in the platinum-based chemotherapeutic space?
Key competitors include liposomal cisplatin (Lipoplatin), carboplatin, and emerging nanoparticle-based platinum agents, each with varying safety and efficacy profiles.

4. How might safety advantages influence PLATINOL-AQ’s market uptake?
Enhanced safety can expand its use to patients with renal impairment and reduce hospitalizations due to toxicity, thereby increasing prescriber preference and market penetration.

5. What strategies can companies adopt to maximize PLATINOL-AQ’s commercial success?
Engaging regulatory agencies for expedited review, clinician education, early payer engagement, and expanding indication portfolio are critical strategies.

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References

1. Market data sourced from GlobalData, 2022.
2. Clinical trial updates derived from ClinicalTrials.gov, ongoing Phase II and III studies (NCTXXXXXX, NCTYYYYYYY).
3. Regulatory guidance from FDA and EMA communication channels.