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BCX10013 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| BCX10013 | EXPERIMENTAL | Participants with PNH will receive BCX10013 daily for 4 weeks before dose escalation may occur. |
| Name | Type | Description |
|---|---|---|
| BCX10013 | DRUG | Multiple dose levels may be tested in this study. |
Key Inclusion Criteria: 1. Male or non-pregnant, non-lactating female adults ≥ 18 years old. 2. Documented diagnosis of PNH confirmed by flow cytometry. 3. Body mass index (BMI) ≤ 40 kg/m\^2. 4. Are either: (a) naïve to treatment with a complement inhibitor; or (b) have received no treatment with r...
BCX10013 is an investigational small molecule being developed for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare hematologic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
BCX10013 is being developed by BioCryst Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BCRX. The company is conducting clinical trials to evaluate the safety and tolerability of this investigational drug.
BCX10013 is in Phase 1 clinical development. A Phase 1 dose escalation study has been completed, and the drug remains investigational. It has not received regulatory approval and is not yet available for clinical use outside of trials.
BCX10013 has one completed Phase 1 clinical trial, NCT06100900, titled 'Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH)'. The study enrolled 8 participants with PNH at sites in Malaysia and South Africa.
No alternative names for BCX10013 have been disclosed. The drug is identified solely by its investigational code name BCX10013, and no other brand names or aliases are currently associated with it in public clinical trial registries.