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BIIB094

Phase 1

Parkinson's Disease | Small molecule | Neurology |Ionis Pharmaceuticals, Inc.|Last Updated: Sep 23, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment62

FDA Designations

No designations recorded

Clinical trial landscape

BIIB094 · 1 trial · 1 indication

Phase 1 1
NCT03976349A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's DiseaseParkinson's Disease
COMPLETED62 Analytics
PHASE1COMPLETED
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
Parkinson's DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Part A: Screening (Day -42) up to Day 85, Part B: Screening (Day -77) up to Day 253

An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); however, this does not include an event that, had it occurred in a more severe form, might have caused death; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; is a medically important event.

Secondary Endpoints

Serum Concentrations of BIIB094
Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
Area Under the Concentration-Time Curve From Time Zero Extrapolated to Infinity (AUCinf) of BIIB094
Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
Area Under the Concentration-Time Curve From Time Zero to Last Quantifiable Concentration (AUClast) of BIIB094
Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part A (SAD): BIIB094 Dose 1EXPERIMENTALParticipants will receive a single IT injection of BIIB094 during Part A \[Single Ascending Dose (SAD)\].
Part A (SAD): BIIB094 Dose 2EXPERIMENTALParticipants will receive a single IT injection of BIIB094 during Part A (SAD).
Part A (SAD): BIIB094 Dose 3EXPERIMENTALParticipants will receive a single IT injection of BIIB094 during Part A (SAD).
Part A (SAD): BIIB094 Dose 4EXPERIMENTALParticipants will receive a single IT injection of BIIB094 during Part A (SAD).
Part A (SAD): BIIB094 Dose 5EXPERIMENTALParticipants will receive a single IT injection of BIIB094 during Part A (SAD).
Part A (SAD): BIIB094 Dose 6EXPERIMENTALParticipants will receive a single IT injection of BIIB094 during Part A (SAD).
Part B (MAD): BIIB094 Dose 1EXPERIMENTALParticipants will receive a single IT injection of BIIB094 on multiple days during Part B \[Multiple Ascending Dose (MAD)\].
Part B (MAD): BIIB094 (Non LRRK2) Dose 2EXPERIMENTALParticipants \[Non leucine-rich repeat kinase 2 (Non LRRK2)\] will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Part B (MAD): BIIB094 (LRRK2) Dose 2EXPERIMENTALParticipants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Part B (MAD): BIIB094 (Non LRRK2) Dose 3EXPERIMENTALParticipants (Non LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Part B (MAD): BIIB094 (LRRK2) Dose 3EXPERIMENTALParticipants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).
Part A (SAD): Matching PlaceboPLACEBO_COMPARATORParticipants will receive matching placebo during Part A \[Single Ascending Dose (SAD)\].
Part B (MAD): Matching PlaceboPLACEBO_COMPARATORParticipants will receive matching placebo on multiple days during Part B (MAD).

Interventions

NameTypeDescription
BIIB094DRUGAdministered as specified in the treatment arm.
PlaceboDRUGAdministered as specified in the treatment arm.
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Eligibility Criteria

Age Range35 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites18

Key Inclusion Criteria: * Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulations. * Diagnosed with PD within 7 years at the ...

Countries:United StatesCanadaIsraelNorwaySpainUnited Kingdom
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Frequently asked questions about BIIB094

What is BIIB094 used for in Parkinson's Disease?

BIIB094 is an investigational small molecule being developed for the treatment of Parkinson's Disease. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied to evaluate its safety, tolerability, and pharmacokinetics in adults with Parkinson's Disease.

What does BIIB094 target?

The molecular target of BIIB094 has not been disclosed in available information. The drug is being studied for its effects in Parkinson's Disease, but its specific mechanism of action is not publicly detailed. Clinical trials are focused on assessing safety, tolerability, and how the drug is processed in the body.

Who makes BIIB094?

BIIB094 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker IONS. The company is conducting clinical research on this investigational drug for Parkinson's Disease, with the drug currently in Phase 1 development.

What phase is BIIB094 in?

BIIB094 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or other regulatory agencies. The Phase 1 trial has been completed, and the drug remains in early-stage clinical research for Parkinson's Disease.

What clinical trials is BIIB094 in?

BIIB094 has one completed clinical trial registered as NCT03976349, titled "A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease." This Phase 1 trial enrolled 62 participants across the United States, Canada, Israel, Norway, Spain, and the United Kingdom.

Is BIIB094 the same as any other drug?

No alternative names for BIIB094 have been disclosed. The drug is identified solely by its development code BIIB094 in clinical trial registrations and public information. It is being studied as a distinct investigational compound for Parkinson's Disease.