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DNL343

Phase 1

Amyotrophic Lateral Sclerosis | Small molecule | Neurology |Denali Therapeutics Inc.|Last Updated: Sep 19, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment29
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05006352A Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral SclerosisPHASE1 COMPLETED 29Aug 11, 2021Jun 5, 2024Sep 19, 20247 United States, Netherlands
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Study Endpoints
Primary Endpoints
Incidence of treatment-emergent adverse events (TEAEs) throughout the double-blind period
28 Days
Secondary Endpoints
PK parameter: Maximum concentration (Cmax) of DNL343 in plasma
19 months
PK parameter: Time to reach maximum concentration (tmax) of DNL343 in plasma
19 months
PK parameter: Trough concentration (Ctrough) of DNL343 in plasma
19 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
DNL343 (High Dose)EXPERIMENTAL -
DNL343 (Low Dose)EXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
DNL343DRUGOral repeating dose
PlaceboDRUGOral repeating dose
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Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites7

Key Inclusion Criteria: * Diagnosis of sporadic or familial ALS * ≤ 4 years since ALS symptom onset * Stable doses of approved ALS treatments (riluzole and/or edaravone) for at least 2 months prior to screening * Participants must be able to swallow the study intervention * Vital capacity \>50% pre...

Countries:United StatesNetherlands
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