Floatz Rating
B59/100
Confidence
Indicative
Sponsor
BioMarin Pharmaceutical
Modality
Small molecule
Development Phase
Phase 4 / Approved
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
49Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT07477691CT.gov Immune Modulation During Palynziq® Treatment in Adults (IMPALA) | — | Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Phenylketonuria
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Methotrexate (this asset) | BioMarin Pharmaceutical | Approved | B · 59 |
| Sapropterin Dihydrochloride | Sohag University | P4 | A |
| Phenylase | BioMarin Pharmaceutical | P4 | A |
| Ravpal-Peg | BioMarin Pharmaceutical | P2 | BB |
| Repinatrabit | Otsuka Pharmaceutical Development & Commercialization, Inc. | P3 | BB |
| Escherichia Coli Strain Synb1618 | Synlogic | P2 | BB |
| L-Carnitine | Mansoura University | P4 | BB |
| Bmn 165 | BioMarin Pharmaceutical | P3 | B |
| Sepiapterin | PTC Therapeutics | P3 | B |
+18 more in the Phenylketonuria cohort
Other indications for Methotrexate
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Adult Acute Lymphoblastic Leukemia | — | P4 | BBB · 77 |
| B-Cell Childhood Acute Lymphoblastic Leukemia | — | P3 | BBB · 76 |
| Acute Promyelocytic Leukemia | — | P3 | BBB · 73 |
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. Methotrexate in Phenylketonuria. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 13, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/methotrexate-phenylketonuria
Are you the sponsor?
Submit your data room for a Verified Rating. The public-data rating remains visible alongside.
Contact →