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MHU650

Phase 1

Macular Edema | Small molecule | Ophthalmology |Novartis AG|Last Updated: May 18, 2025

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment21

FDA Designations

No designations recorded

Clinical trial landscape

MHU650 · 1 trial · 4 indications

Phase 1 1
NCT04635800A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of IVT MHU650 in Macular Edema PatientsMacular Edema
COMPLETED21 Analytics
PHASE1COMPLETED
A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of IVT MHU650 in Macular Edema Patients
Macular EdemaUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of participants with ocular and nonocular adverse events
Day 1 to Day 60

An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.

Change from baseline in Best Correct Visual Acuity (BCVA ) by dose level and timepoint - Study Eye
Day 1 to Day 60

Best Correct Visual Acuity was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts.

Change from baseline in Central subfield thickness (CSFT) (µm) in the study eye by dose level and timepoint
Day 1 to Day 60

Central subfield thickness was measured by spectral domain optical coherence tomography (SD-OCT).

Change from baseline in Intraocular pressure (IOP) (mmHg) in the study eye by dose level and timepoint
Day 1 to Day 60

Intraocular pressure was measured per the sites' local standard operating procedures, via tonometry.

Secondary Endpoints

Pharmacokinetics of single dose of MHU650 - Cmax
Days 1, 2, 5, 15, 29, 43 and 60
Pharmacokinetics of single dose of MHU650 - Tmax
Days 1, 2, 5, 15, 29, 43 and 60
Pharmacokinetics of single dose of MHU650 - T1/2
Days 1, 2, 5, 15, 29, 43 and 60
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1EXPERIMENTALCohort 1; open-label, non-randomized, single administration
Cohort 2EXPERIMENTALCohort 2; open-label, non-randomized, single administration
Cohort 3EXPERIMENTALCohort 3, open-label; non-randomized, single administration
Cohort 4EXPERIMENTALCohort 4, open-label, non-randomized, single administration

Interventions

NameTypeDescription
MHU650DRUGMHU650 powder for solution for injection
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Eligibility Criteria

Age Range18 Years to 90 Years
SexALL
Healthy VolunteersNo
Study Sites6

Key Inclusion Criteria: * Patients with macular edema in at least one eye, including those with focal or diffuse diabetic macular edema (DME), neovascular age-related macular degeneration (nAMD), or retinal vein occlusion (RVO). In the opinion of the investigator, the decrease in vision in the stud...

Countries:United StatesPuerto Rico
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Frequently asked questions about MHU650

What is MHU650 used for?

MHU650 is an investigational small molecule being developed for the treatment of macular edema, a condition involving fluid accumulation in the macula of the eye. It is being studied in patients with macular edema, including those with diabetic macular edema, neovascular age-related macular degeneration, and retinal vein occlusion.

Who makes MHU650?

MHU650 is being developed by Novartis AG, a multinational pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS. The company is conducting clinical research to evaluate the safety, tolerability, and pharmacokinetics of this investigational therapy.

What phase is MHU650 in?

MHU650 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A Phase 1 study evaluating the drug has been completed, and it remains in early-stage clinical development for the treatment of macular edema.

What clinical trials is MHU650 in?

MHU650 has one completed Phase 1 clinical trial, identified as NCT04635800. This study evaluated the safety, tolerability, and pharmacokinetics of intravitreal MHU650 in patients with macular edema, including those with diabetic macular edema, neovascular age-related macular degeneration, and retinal vein occlusion. The trial enrolled 21 participants.

How is MHU650 administered?

MHU650 is administered via intravitreal injection, meaning it is injected directly into the vitreous humor of the eye. This route of administration is commonly used for ophthalmic therapies to deliver the drug directly to the site of disease in the retina.