Floatz Rating
CCC53/100
Confidence
Indicative
Sponsor
Amylyx Pharmaceuticals Inc.
Modality
Small molecule
Development Phase
Phase 2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
38Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05676034CT.gov AMX0035 in Adult Patients With Wolfram Syndrome | — | 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 · Wolfram Syndrome
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Taurursodiol (this asset) | Amylyx Pharmaceuticals Inc. | P2 | CCC · 53 |
| Valproic Acid | University of Birmingham | P2 | CCC |
| Tirzepatide | Ospedale San Raffaele | P2 | CCC |
| Depakine | Centre d'Etude des Cellules Souches | P2 | CCC |
| Dantrolene | Washington University School of Medicine | P1/2 | CCC |
| Exenatide | Hadassah Medical Organization | N/A | D |
Other indications for Taurursodiol
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Amyotrophic Lateral Sclerosis | — | P3 | BBB · 74 |
| Primary Biliary Cholangitis | — | P3 | BB · 64 |
| Wild Type ATTR Amyloidosis | — | P3 | BB · 64 |
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. Taurursodiol in Wolfram Syndrome. 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/taurursodiol-wolfram-syndrome
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