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BIIB104
BIIB104 is a LRRK2 kinase inhibitor Small molecule drug developed by Biogen. It is currently in Phase 2 development for LRRK2-associated Parkinson's disease. Also known as: PF-04958242.
BIIB104 is a selective inhibitor of leucine-rich repeat kinase 2 (LRRK2) that reduces pathological protein aggregation in neuronal cells.
BIIB104 is a small molecule that acts as a positive allosteric modulator of the glutamate receptor ionotropic AMPA. It is being studied in a Phase 2 clinical trial for the treatment of cognitive impairment associated with schizophrenia.
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Baseline phase 2 → approval rate
+15.3pp
Industry-wide phase 2 drugs reach approval ~15.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
CNS / neurology attrition
-3.0pp
CNS drugs have historically high Phase 3 failure rates (notably in Alzheimer disease + major depression). -
Big-pharma sponsor
+3.0pp
Biogen is a top-20 pharma sponsor — historical approval rates run ~3pp above average due to scale, regulatory experience, and trial-design quality.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2031–2034 | — |
| EMA | EU | 2032–2035 | +0.7 yr |
| MHRA | GB | 2032–2035 | +0.7 yr |
| Health Canada | CA | 2032–2036 | +0.9 yr |
| TGA | AU | 2032–2036 | +1.2 yr |
| PMDA | JP | 2032–2036 | +1.5 yr |
| NMPA | CN | 2033–2037 | +2.3 yr |
| MFDS | KR | 2032–2036 | +1.4 yr |
| CDSCO | IN | 2032–2037 | +1.8 yr |
| ANVISA | BR | 2033–2037 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | BIIB104 |
|---|---|
| Also known as | PF-04958242 |
| Sponsor | Biogen |
| Drug class | LRRK2 kinase inhibitor |
| Target | LRRK2 (Leucine-rich repeat kinase 2) |
| Modality | Small molecule |
| Therapeutic area | Neurology |
| Phase | Phase 2 |
Mechanism of action
BIIB104 targets LRRK2, a kinase implicated in Parkinson's disease pathogenesis. By inhibiting LRRK2 activity, the drug aims to reduce the accumulation of alpha-synuclein and other pathological proteins that contribute to neurodegeneration. This mechanism is intended to slow or halt disease progression in LRRK2-associated Parkinson's disease.
Approved indications
- LRRK2-associated Parkinson's disease
Common side effects
- Headache
- Nausea
- Diarrhea
Key clinical trials
- A Study to Evaluate the Safety and Efficacy of BIIB104 in Participants With Cognitive Impairment Associated With Schizophrenia (CIAS) (PHASE2)
- A Study to Evaluate Safety and Pharmacokinetics of BIIB104 in Healthy Japanese and Non-Japanese Participants (PHASE1)
- A Study to Evaluate the Relative Bioavailability of Two Formulations of BIIB104 and Assess the Effect of Food on the Pharmacokinetics of BIIB104 Following Administration of a Single, Fixed, Oral Dose of BIIB104 in Healthy Participants (PHASE1)
- Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB104 in Healthy Japanese and Non-Japanese Participants (PHASE1)
- A Study to Explore Pharmacodynamic Effects of BIIB104 on Brain Circuitry in Healthy Participants (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- BIIB104 CI brief — competitive landscape report
- BIIB104 updates RSS · CI watch RSS
- Biogen portfolio CI
Frequently asked questions about BIIB104
What is BIIB104?
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Is BIIB104 also known as anything else?
What drug class is BIIB104 in?
What development phase is BIIB104 in?
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What does BIIB104 target?
Related
- Drug class: All LRRK2 kinase inhibitor drugs
- Target: All drugs targeting LRRK2 (Leucine-rich repeat kinase 2)
- Manufacturer: Biogen — full pipeline
- Therapeutic area: All drugs in Neurology
- Indication: Drugs for LRRK2-associated Parkinson's disease
- Also known as: PF-04958242
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing