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KAE609

Phase 2

Cure Rate | Small molecule | Other |Novartis AG|Last Updated: May 7, 2026

Target and mechanism

ModalitySmall molecule

Also known as KAE609 (Cipargamin)

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment11

FDA Designations

No designations recorded

Clinical trial landscape

KAE609 · 6 trials · 3 indications

Phase 2 5Phase 1 1
NCT07235033Platform Study to Evaluate the Efficacy and Safety of Anti-malarial Agents in Participants With Uncomplicated Plasmodium Falciparum Malaria (Cohort B2)Uncomplicated Plasmodium Falciparum Malaria
COMPLETED60 Analytics
NCT03334747Safety of KAE609 in Adults With Uncomplicated Plasmodium Falciparum Malaria.Malaria
COMPLETED188 Analytics
NCT01836458A Study to Find the Minimum Inhibitory Concentration of KAE609 in Adult Male Patients With P. Falciparum MonoinfectionMalaria
COMPLETED25 Analytics
NCT01860989A Study to Assess Efficacy, Safety of KAE609 in Adult Patients With Acute Malaria Mono-infectionCure Rate
COMPLETED11 Analytics
NCT01524341Efficacy, Safety, Tolerability and Pharmacokinetics of KAE609 in Adult Patients With Acute, Uncomplicated Plasmodium Falciparum or Vivax Malaria Mono-infectionMalaria
COMPLETED27 Analytics
PHASE2COMPLETED
Platform Study to Evaluate the Efficacy and Safety of Anti-malarial Agents in Participants With Uncomplicated Plasmodium Falciparum Malaria (Cohort B2)
Uncomplicated Plasmodium Falciparum MalariaUnlock trial analytics
PHASE2COMPLETED
Safety of KAE609 in Adults With Uncomplicated Plasmodium Falciparum Malaria.
MalariaUnlock trial analytics
PHASE2COMPLETED
A Study to Find the Minimum Inhibitory Concentration of KAE609 in Adult Male Patients With P. Falciparum Monoinfection
MalariaUnlock trial analytics
PHASE2COMPLETED
A Study to Assess Efficacy, Safety of KAE609 in Adult Patients With Acute Malaria Mono-infection
Cure RateUnlock trial analytics
PHASE2COMPLETED
Efficacy, Safety, Tolerability and Pharmacokinetics of KAE609 in Adult Patients With Acute, Uncomplicated Plasmodium Falciparum or Vivax Malaria Mono-infection
MalariaUnlock trial analytics

Study Endpoints

Primary Endpoints

Polymerase Chain Reaction (PCR) Corrected Adequate Clinical and Parasitological Response (ACPR)
Day 29

ACPR is defined as absence of parasitaemia (PS) on Study Day 29 regardless of axillary temperature, in patients who have not previously met any of the criteria of Early Treatment Failure (ETF), Late Clinical Failure (LCF), or Late Parasitological Failure (LPF). A patient was considered as PCR-corrected ACPR at Day 29 when the patient did not meet any of the criteria of ETF (up to Day 4), LCF (Day 5 to Day 29), or LPF (Day 8 to Day 29), and was absence of PS on Day 29, unless the presence of PS detected after 7 days (Day 8 or later) was due to reinfection. The presence of PS after 7 days of treatment initiation was considered as a reinfection only when the PS had cleared before Day 8, and none of the parasite strain(s) detected on or after Day 8 matched with the parasite strain at baseline.

Number of Participants With at Least 2 CTCAE Grades Increase From Baseline in Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST)
Day 29

The occurrence of at least 2 CTCAE grades increase from baseline in ALT or AST during the 4 weeks study period was evaluated to characterize hepatic safety aspects of single and multiple ascending doses of KAE609 in adult malaria subjects for treatment of uncomplicated malaria caused by plasmodium falciparum. If 2 patients in a 10 patient cohort (Cohorts 1 and 2) or 3 patients in a 20 patient cohort (Cohorts 3, 4 and 5) had at least 2 CTCAE grades increase from Baseline in ALT or AST, recruitment was suspended and a review of liver safety (and any other relevant data) by safety review committee was initiated. Any further progression of the study was based on the decision by the safety review committee.

Minimum Inhibitory Concentration (MIC) of KAE609
Up to Day 8 after a single dose of KAE609

To observe the exposure-response (PK/PD) relationship for a single dose of KAE609. The key parameter is MIC, defined as the concentration at which the relative rate of change in parasitemia is equal to zero. Approximation of MIC will assist in identifying the optimal dose of KAE609, which will be one component of a future combination antimalarial. MIC could not be determined due to small sample size no data was collected from any participants.

28-day Cure Rate
Day 28

28-day cure rate was measured by the endpoint of complete cure without recrudescence before Day 29. The primary variable of 28-day cure rate was defined as the proportion of patients with clearance of asexual parasitemia (by blood film) by day 6 of the study, and without subsequent recrudescence (by blood film).

Parasite clearance time
From baseline to the time point when the blood parasite count is zero(up to a maximum of 5 days)

Calculated based on parasite count in blood. In thin film, use actual WBCs/µl, of blood to calculate parasite density by using the following formula: parasites/µl= #parasites× actual WBC/#WBCs counted. In thick film, assume that there are 250 RBCs per HPF, RBC count from 8 HPF equal 2000 RBC, Use actual RBCs/µl blood to calculate parasite density by using the following formula: parasites/µl= # of parasites in 8HPF/2000)× actual RBC.

Number of Participants With On-Treatments Adverse Events, Serious Adverse Events, and Deaths
From study treatment start date till 30 days safety follow-up, assessed for up to 4 months

The distribution of adverse events was done via the analysis of frequencies for Adverse Event (AEs), Serious Adverse Event (SAEs) and Deaths, through the monitoring of relevant clinical and laboratory safety parameters.

Secondary Endpoints

Parasite Clearance Time (PCT)
up to Day 7
PCR Uncorrected Adequate Clinical and Parasitological Response (ACPR)
Day 29
Maximum Observed Plasma Concentration (Cmax)
Pre-dose, 1, 2, 4, 6, 8, 12, 24, and 48 hours post dose.
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort B2: KLU156 400/480 mg + KAE609 75 mgEXPERIMENTALKLU156 \[(400 mg KAF156, 480 mg Lumefantrine (LUM)-solid dispersion formulation (SDF)\] + KAE609 75 mg was administered orally with light meal as a single dose.
Cohort B2: SoC (Artemether 80 mg + lumefantrine 480 mg)ACTIVE_COMPARATORArtemether 80 mg + lumefantrine 480 mg was administered twice a day for 3 days with a standard meal or drink rich in fat within 30 min of dosing, as per label.
Treatment arm 1: KAE609 10 mg Single Dose (SD)EXPERIMENTALKAE609 10 mg once daily (QD) for 1 day
Treatment arm 2:KAE609 25 mg SDEXPERIMENTALKAE609 25 mg once daily (QD) for 1 day
Treatment arm 3:KAE609 10 mg 3 DaysEXPERIMENTALKAE609 10 mg (QD) for 3 days
Treatment arm 4:KAE609 50 mg SDEXPERIMENTALKAE609 50 mg once daily (QD) for 1 day
Treatment arm 5:KAE609 25 mg 3 DaysEXPERIMENTALKAE609 25 mg once daily (QD) for 3 days
Treatment arm 6:KAE609 75 mg SDEXPERIMENTALKAE609 75 mg once daily (QD) for 1 day
Treatment arm 7:KAE609 50 mg 3 DaysEXPERIMENTALKAE609 50 mg once daily (QD) for 3 days
Treatment arm 8: KAE609 150 mg SDEXPERIMENTALKAE609 150 mg once daily (QD) for 1 day
Treatment arm 9: Coartem ControlACTIVE_COMPARATORCoartem® control
Dose 1: 30 mgEXPERIMENTALSingle dose of KAE609 30 mg
Dose 2: 20 mgEXPERIMENTALSingle dose of KAE609 20 mg
Dose 3: 10 mgEXPERIMENTALSingle dose of KAE609 10 mg
Dose 4: 15 mgEXPERIMENTALSingle dose of KAE609 15 mg
Cohort 1EXPERIMENTAL6-12 subjects with Plasmodium falciparum malaria will receive 75 mg KAE609 as a single dose
Cohort 2EXPERIMENTAL6-12 subjects with Plasmodium falciparum malaria will receive 150 mg KAE609 as a single dose
Cohort 3EXPERIMENTAL6 to 12 subjects with Plasmodium falciparum malaria will receive 225 mg KAE609 as a single dose
Cohort4EXPERIMENTAL6- 12 subjects with Plasmodium falciparum malaria will receive 300 mg KAE609 as a single dose
Cohort A1: 10.5 mg/placeboEXPERIMENTALSingle iv bolus dose of KAE609 or placebo administered at the clinical site by the study personnel.
Cohort A2: 30 mg/placeboEXPERIMENTALSingle iv bolus dose of KAE609 or placebo administered at the clinical site by the study personnel.
Cohort A3: 75 mg/placeboEXPERIMENTALSingle iv infusion dose of KAE609 or placebo administered at the clinical site by the study personnel.
Cohort A4: 120 mg/placeboEXPERIMENTALSingle iv infusion dose of KAE609 or placebo administered at the clinical site by the study personnel.
Cohort A5: 210 mg/placeboEXPERIMENTALSingle iv infusion dose of KAE609 or placebo administered at the clinical site by the study personnel.
Cohort B1: 60 mg/placebo, every 24 hours (q24h) × 5 daysEXPERIMENTALMultiple iv bolus doses of KAE609 or placebo administered at the clinical site by the study personnel.
Cohort B2: 120 mg/placebo, every 24 hours (q24h) × 5 daysEXPERIMENTALMultiple iv infusion doses of KAE609 or placebo administered at the clinical site by the study personnel.

Interventions

NameTypeDescription
KAE609DRUGoral capsules administered in combination with KLU156
SoC (Coartem)DRUGStandard of Care
KLU156DRUGoral sachet formulation (KAF156+LUM-SDF) administered in combination with cipargamin (KAE609)
CoartemDRUGControl Arm
PlaceboDRUGmatching placeo for iv administration
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Eligibility Criteria

Age Range12 Years to 100 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: 1. Male and female patients ≥12 years of age at screening. 2. Patients must have acute uncomplicated P. falciparum malaria mono infection at screening confirmed by a parasite count between 1,000 to 150,000 asexual parasite count/μl of blood for P. falciparum. 3. Patients must we...

Countries:Côte d’IvoireGabonKenyaGhanaMaliRwandaUgandaVietnamThailandBelgium
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Recent Changes (Last 90 Days)

MEDIUMJun 7, 2026NCT07235033TRIAL_REMOVED: changed
MEDIUMJun 7, 2026NCT07235033TRIAL_REMOVED: changed
MEDIUMJun 7, 2026NCT07235033TRIAL_REMOVED: changed
MEDIUMJun 7, 2026NCT07235033TRIAL_REMOVED: changed
MEDIUMJun 7, 2026NCT07235033TRIAL_REMOVED: changed
MEDIUMJun 7, 2026NCT07235033TRIAL_REMOVED: changed

Frequently asked questions about KAE609

What is KAE609 used for?

KAE609 is an investigational small molecule being developed for malaria, including uncomplicated Plasmodium falciparum malaria. It is being studied for its ability to achieve cure rates in patients with this infection. The drug is currently in clinical development and is not approved for use.

Who makes KAE609?

KAE609 is being developed by Novartis AG, a multinational pharmaceutical company listed on the New York Stock Exchange under the ticker NVS. Novartis is conducting clinical trials to evaluate the safety and efficacy of KAE609 in patients with malaria.

What phase is KAE609 in?

KAE609 is in Phase 2 clinical development. It has completed four clinical trials, including Phase 1 and Phase 2 studies, with a total enrollment of 297 participants. The drug remains investigational and has not received regulatory approval.

What clinical trials has KAE609 been in?

KAE609 has been studied in four completed clinical trials. These include NCT01836458, a Phase 2 study in adult male patients with P. falciparum monoinfection in Vietnam; NCT03334747, a Phase 2 safety study in adults with uncomplicated malaria across five African countries; NCT04321252, a Phase 1 study in healthy subjects in Belgium; and NCT07235033, a Phase 2 platform study in participants with uncomplicated malaria.

Is KAE609 the same as Cipargamin?

Yes, KAE609 is also known as Cipargamin. Both names refer to the same investigational small molecule drug being developed by Novartis for the treatment of malaria. Researchers and clinical trial registries may use either name when referring to this compound.

How is KAE609 administered in clinical trials?

KAE609 has been studied in both oral and intravenous forms. Clinical trials have evaluated the drug in healthy volunteers and in patients with malaria. The completed studies include assessments of safety, tolerability, pharmacokinetics, and efficacy in achieving cure rates for uncomplicated Plasmodium falciparum malaria.