Glycerol Phenylbutyrate
Developmental And Epileptic Encephalopathy
Explore 7 assets in Developmental And Epileptic Encephalopathy →Floatz Rating
C39/100
Confidence
Indicative
Sponsor
Weill Medical College of Cornell University
Modality
Small molecule
Development Phase
Early Phase 1
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
24Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04937062CT.gov Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Developmental And Epileptic Encephalopathy
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Glycerol Phenylbutyrate (this asset) | Weill Medical College of Cornell University | Early P1 | C · 39 |
| Bexicaserin | Longboard Pharmaceuticals | P3 | BB |
| Benzeneethanamine, N-Ethyl-?-Methyl-4-(Trifluoromethyl)-, Hydrochloride, (±)- | Zogenix, Inc. | P3 | BB |
| Elsunersen | Praxis Precision Medicines | P3 | B |
| Nbi-827104 | Neurocrine Biosciences | P2 | CCC |
| Melatonin | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | P2 | CCC |
| Soticlestat | Takeda | P1/2 | CCC |
| S230815 | Institut de Recherches Internationales Servier | P1/2 | CCC |
| Stiripentol | Biocodex | P1 | CC |
+1 more in the Developmental And Epileptic Encephalopathy cohort
Other indications for Glycerol Phenylbutyrate
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Hepatic Encephalopathy | — | P2 | B · 61 |
| Cirrhosis Of Liver | — | P2 | CCC · 54 |
| Urea Cycle Disorder | — | P1 | CC · 47 |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Glycerol Phenylbutyrate in Developmental And Epileptic Encephalopathy. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/glycerol-phenylbutyrate-developmental-and-epileptic-encephalopathy
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