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DNL593

Phase 1

Frontotemporal Dementia | Small molecule | Neurology |Denali Therapeutics Inc.|Last Updated: Jan 15, 2026

Target and mechanism

Molecular targetPGRN
Target classProtein
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment85

FDA Designations

ORPHAN_DRUG

Clinical trial landscape

DNL593 · 1 trial · 1 indication

Phase 1 1
NCT05262023A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia (FTD-GRN)Frontotemporal Dementia
ACTIVE NOT_RECRUITING85 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia (FTD-GRN)
Frontotemporal Dementia
NCT0526202385
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Study Endpoints

Primary Endpoints

Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)
up to 18 months
Incidence of treatment-emergent clinically significant abnormalities in safety laboratory values
up to 18 months
Change from baseline in vital sign measurements: systolic and diastolic blood pressure
up to 18 months
Change from baseline in vital sign measurements: heart rate
up to 18 months
Change from baseline in vital sign measurements: respiratory rate
up to 18 months
Change from baseline in vital sign measurements: body temperature
up to 18 months
Change from baseline in electrocardiogram (ECG) results including PR, QRS, and QTcF intervals
up to 18 months
Incidence of treatment-emergent clinically significant abnormalities in physical/neurological examination findings
up to 18 months
Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS; Parts B and C only)
up to 18 months

Secondary Endpoints

PK Parameter: Maximum concentration (Cmax) of DNL593 in serum
up to 18 months
PK Parameter: Time to reach maximum concentration (tmax) of DNL593 in serum
up to 18 months
PK Parameter: Area under the concentration-time curve (AUC) from time zero to time of last measurable concentration (AUClast) of DNL593 in serum
up to 18 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
DNL593 (Healthy Participant)EXPERIMENTAL -
Placebo (Healthy Participant)PLACEBO_COMPARATOR -
DNL593 (Participants with FTD)EXPERIMENTAL -
Placebo (Participants with FTD)PLACEBO_COMPARATOR -

Interventions

NameTypeDescription
DNL593DRUGAscending single doses (for healthy participants) and multiple doses (for participants with FTD)
PlaceboDRUGAscending single doses (for healthy participants) and multiple doses (for participants with FTD)
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Eligibility Criteria

Age Range18 Years to 80 Years
SexALL
Healthy VolunteersYes
Study Sites26

Key Inclusion Criteria: Part A: * Women of non-childbearing potential (surgically sterilized or post menopausal) or men, aged ≥18 to ≤ 55 years * BMI of ≥ 18 to ≤ 32 kg/m² * When engaging in sex with a woman of child bearing potential, two forms of birth control are required Part B: * Women of n...

Countries:United StatesBelgiumBrazilColombiaCzechiaFranceItalyNetherlandsPortugalSpainTurkey (Türkiye)United Kingdom
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Frequently asked questions about DNL593

What is DNL593 used for?

DNL593 is an investigational small molecule being developed for the treatment of Frontotemporal Dementia (FTD), a neurodegenerative condition. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied in patients with FTD-GRN, a genetic form of the disease.

What does DNL593 target?

DNL593 targets PGRN, a protein involved in cellular processes. By modulating PGRN, the drug aims to address the underlying pathology of Frontotemporal Dementia. This mechanism is being evaluated in clinical trials to assess its safety and efficacy in patients with FTD-GRN.

Who makes DNL593?

DNL593 is being developed by Denali Therapeutics Inc., a biopharmaceutical company focused on neurological diseases. Denali Therapeutics is publicly traded under the ticker symbol DNLI on the NASDAQ stock exchange.

What phase is DNL593 in?

DNL593 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The Phase 1 trial is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug in healthy participants and those with Frontotemporal Dementia.

What clinical trials is DNL593 in?

DNL593 is being studied in a Phase 1 clinical trial with the identifier NCT05262023. This trial is titled "A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia (FTD-GRN)." The study is active but not recruiting and has an enrollment of 85 participants.

Is DNL593 the same as any other drug?

DNL593 is not known to have any alternative names. It is a unique investigational compound developed by Denali Therapeutics. The drug is being studied under the clinical trial identifier NCT05262023 for the treatment of Frontotemporal Dementia.