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Atovaquone

Phase 1

Locally Advanced Non-Small Cell Lung Cancer | Small molecule | Oncology |United Therapeutics Corporation|Last Updated: Jan 29, 2026

Success Probability

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Trial Design

CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment21

FDA Designations

No designations recorded

Clinical trial landscape

Atovaquone · 1 trial · 1 indication

Phase 1 1
NCT04648033Atovaquone With Radical ChemorADIotherapy in Locally Advanced NSCLCLocally Advanced Non-Small Cell Lung Cancer
COMPLETED21 Analytics
PHASE1COMPLETED
Atovaquone With Radical ChemorADIotherapy in Locally Advanced NSCLC
Locally Advanced Non-Small Cell Lung CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Dose Limiting Toxicities in Patients Taking Atovaquone in Combination With Radical Concurrent Chemoradiotherapy for Non-small Cell Lung Cancer.
From first dose of atovaquone to 3-month follow up visit (up to 25 weeks)

To determine the maximum tolerated dose level (and therefore recommended phase II dose) of atovaquone when administered concomitantly with radical concurrent chemoradiotherapy (CRT) in patients with non-small cell lung cancer (NSCLC). This is the dose of atovaquone associated with no more than 48% dose limiting toxicity (DLT) rate (target toxicity level).

Secondary Endpoints

Severity of Worst Adverse Events Per Dose Level of Atovaquone Administered in Combination With Radical Concurrent Chemotherapy for NSCLC According to CTCAE V4.03
From first dose of atovaquone until last follow up visit at 6 months post completion of CRT (up to 38 weeks)
Number of Patients for Whom it Was Possible to Derive a Hypoxia Metagene Signature Score From 3'RNA-Seq of Genetic Material From Archival Tumour Samples
At baseline (diagnosis)
Mean Baseline Tumour Hypoxia Level (TBRvol) Assessed by F18-FMISO PET-CT
At baseline (prior to atovaquone treatment)
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Dose level 1 - 450 mg BD atovaquone + concurrent CRTEXPERIMENTALAtovaquone: * Taken during an initial run in period (2 weeks +/- 7 days), then continued during standard of care chemoradiotherapy. * One of 4 dose levels is allocated to each patient by a TiTE-CRM statistical model which takes into account all toxicity data to date. * Dose levels - 450 mg, 600 mg, 675 mg or 750 mg (all doses PO BD). * Last dose atovaquone taken on the last day of radiotherapy. * Total duration of atovaquone treatment is 59 days (+/- 7 days), unless stopped earlier for toxicity or any other reason. Chemotherapy: * 2 x 21-day cycles. * 80 mg/m2 cisplatin on day 1 and 22 of chemoradiotherapy. * 15 mg/m2 vinorelbine on days 1,8, 22 and 29 of chemoradiotherapy. Radiotherapy: * 66 Gy in 33 fractions. * Delivered once daily, 5 days a week (Monday-Friday) for 6.5 weeks.
Dose level 2 - 600 mg BD atovaquone + concurrent CRTEXPERIMENTALAtovaquone: * Taken during an initial run in period (2 weeks +/- 7 days), then continued during standard of care chemoradiotherapy. * One of 4 dose levels is allocated to each patient by a TiTE-CRM statistical model which takes into account all toxicity data to date. * Dose levels - 450 mg, 600 mg, 675 mg or 750 mg (all doses PO BD). * Last dose atovaquone taken on the last day of radiotherapy. * Total duration of atovaquone treatment is 59 days (+/- 7 days), unless stopped earlier for toxicity or any other reason. Chemotherapy: * 2 x 21-day cycles. * 80 mg/m2 cisplatin on day 1 and 22 of chemoradiotherapy. * 15 mg/m2 vinorelbine on days 1,8, 22 and 29 of chemoradiotherapy. Radiotherapy: * 66 Gy in 33 fractions. * Delivered once daily, 5 days a week (Monday-Friday) for 6.5 weeks.
Dose level 3 - 675 mg BD atovaquone + concurrent CRTEXPERIMENTALAtovaquone: * Taken during an initial run in period (2 weeks +/- 7 days), then continued during standard of care chemoradiotherapy. * One of 4 dose levels is allocated to each patient by a TiTE-CRM statistical model which takes into account all toxicity data to date. * Dose levels - 450 mg, 600 mg, 675 mg or 750 mg (all doses PO BD). * Last dose atovaquone taken on the last day of radiotherapy. * Total duration of atovaquone treatment is 59 days (+/- 7 days), unless stopped earlier for toxicity or any other reason. Chemotherapy: * 2 x 21-day cycles. * 80 mg/m2 cisplatin on day 1 and 22 of chemoradiotherapy. * 15 mg/m2 vinorelbine on days 1,8, 22 and 29 of chemoradiotherapy. Radiotherapy: * 66 Gy in 33 fractions. * Delivered once daily, 5 days a week (Monday-Friday) for 6.5 weeks.
Dose level 4 - 750 mg BD atovaquone + concurrent CRTEXPERIMENTALAtovaquone: * Taken during an initial run in period (2 weeks +/- 7 days), then continued during standard of care chemoradiotherapy. * One of 4 dose levels is allocated to each patient by a TiTE-CRM statistical model which takes into account all toxicity data to date. * Dose levels - 450 mg, 600 mg, 675 mg or 750 mg (all doses PO BD). * Last dose atovaquone taken on the last day of radiotherapy. * Total duration of atovaquone treatment is 59 days (+/- 7 days), unless stopped earlier for toxicity or any other reason. Chemotherapy: * 2 x 21-day cycles. * 80 mg/m2 cisplatin on day 1 and 22 of chemoradiotherapy. * 15 mg/m2 vinorelbine on days 1,8, 22 and 29 of chemoradiotherapy. Radiotherapy: * 66 Gy in 33 fractions. * Delivered once daily, 5 days a week (Monday-Friday) for 6.5 weeks.

Interventions

NameTypeDescription
Atovaquone Oral SuspensionDRUGAtovaquone, cisplatin and vinorelbine are all considered Investigational Medicinal Products (IMPs) in this trial due to the investigation of these drugs in a novel combination. Patients will be allocated one of four doses of atovaquone: 450 mg, 600 mg, 675 mg or 750 mg (all doses PO BD).
Standard of care chemotherapyDRUGAtovaquone, cisplatin and vinorelbine are all considered Investigational Medicinal Products (IMPs) in this trial due to the investigation of these drugs in a novel combination. Patients will receive two 21-day cycles of cisplatin and vinorelbine chemotherapy, comprising 80 mg/m2 cisplatin on days 1 \& 22 of their CRT treatment and 15 mg/m2 vinorelbine on days 1, 8, 22 \& 29.
Standard of care radiotherapyRADIATIONThoracic radiotherapy will commence on day one of chemotherapy and be delivered in 66 Gy in 33 fractions, once daily, 5 days a week (Monday-Friday) for 6.5 weeks.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: A patient will be eligible for inclusion in this study if all of the following criteria apply: 1. Histologically or cytologically confirmed diagnosis of locally advanced NSCLC and selected for treatment with full dose radical concurrent CRT 2. At least one measurable lesion gre...

Countries:United Kingdom
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Frequently asked questions about Atovaquone

What is Atovaquone used for in Locally Advanced Non-Small Cell Lung Cancer?

Atovaquone is being studied for use in locally advanced non-small cell lung cancer. It is an investigational small molecule being evaluated in combination with radical chemoradiotherapy. The drug is currently in Phase 1 clinical development for this indication, and it has not been approved for this use.

What does Atovaquone target?

Atovaquone is a small molecule that targets mitochondrial cytochrome bc1 complex, inhibiting mitochondrial electron transport. This mechanism is being investigated for its potential anti-cancer effects in locally advanced non-small cell lung cancer. The drug is being studied in combination with standard chemoradiotherapy in a clinical trial setting.

Who makes Atovaquone?

Atovaquone is being developed by United Therapeutics Corporation, which trades under the ticker UTHR. The company is conducting clinical research on this drug for the treatment of locally advanced non-small cell lung cancer. Atovaquone is currently in Phase 1 clinical development for this indication.

What phase is Atovaquone in?

Atovaquone is in Phase 1 clinical development for locally advanced non-small cell lung cancer. It is an investigational drug and has not been approved by regulatory authorities for this use. The Phase 1 trial has been completed, and the drug remains in early-stage clinical investigation.

What clinical trials is Atovaquone in?

Atovaquone has been studied in a Phase 1 clinical trial with the identifier NCT04648033, titled "Atovaquone With Radical ChemorADIotherapy in Locally Advanced NSCLC." This trial was conducted in the United Kingdom and enrolled 21 participants. The study has been completed, and it evaluated atovaquone in combination with radical chemoradiotherapy.

Is Atovaquone the same as Mepron?

Atovaquone is the generic name for a drug that is also known by the brand name Mepron. In the context of this clinical development program, Atovaquone is being investigated for a new use in locally advanced non-small cell lung cancer. The drug is being developed by United Therapeutics Corporation for this oncology indication.