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Floatz Rating
BB64/100
Confidence
Indicative
v0.2
Sponsor
St. Jude Children's Research Hospital
Modality
protein_peptide
Development Phase
Phase 2/3
Status
Active
Evidence ledger · v0.2

Clinical Evidence

Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
51High confidence
Detailed axis rationale is planned and will be published soon.
TrialPhaseStatusNPrimary endpointReadout
Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia
Completed
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
Active Not Recruiting
Stem Cell Transplant for Hemoglobinopathy
Completed
Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies
Terminated

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 · Thalassemia

AssetSponsorPhaseRating
Nivestim (this asset)St. Jude Children's Research HospitalP2/3BB · 64
DeferasiroxNovartis PharmaceuticalsP4BBB
FludarabineMemorial Sloan Kettering Cancer CenterP2/3BB
HydroxyureaRiphah International UniversityP4BB
Ppsv23Fakultas Kedokteran Universitas IndonesiaP4BB
Zinc SulfateFakultas Kedokteran Universitas IndonesiaP4BB
EtavopivatNovo Nordisk A/SP3BB
Conjugated Pneumococcal VaccineFakultas Kedokteran Universitas IndonesiaP4BB
BusulfanumUniversity of California, San FranciscoP2/3BB

+42 more in the Thalassemia cohort

Other indications for Nivestim

IndicationSponsorPhaseRating
Burkitt LymphomaP4BBB · 78
NeutropeniaP4BBB · 76
Mycosis FungoidesP4BBB · 75

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. Nivestim in Thalassemia. 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/n-met-g-csf-thalassemia

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