GROWTHFORGE · BEACON · SOURCE-BACKED SIGNAL

BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy

NCT05116787 is on the Cheap-to-Truth Radar because its public ClinicalTrials.gov record contains a recent development signal worth inspecting. This page is not a recommendation or diligence conclusion.

Priority to inspect82/100
StatusTERMINATED
PhasePHASE2
Lead sponsorBioCryst Pharmaceuticals
Last update2026-10-29

Public-record facts

InterventionPlacebo (DRUG), BCX9930 monotherapy (DRUG)
ConditionParoxysmal Nocturnal Hemoglobinuria (PNH)
Enrollment12
Why stoppedSponsor decision
Results postedYes

Why Beacon surfaced it

What Cheap-to-Truth would verify next

  1. Was the stopping reason scientific, operational, financial, strategic, or mixed?
  2. What human safety and efficacy evidence actually exists beyond the registry summary?
  3. Who controls the relevant rights, and are they practically obtainable?
  4. Is there an indication and endpoint where a smaller experiment could answer the key decision?
  5. Would the resulting data be usable by the regulator that matters?

Source: ClinicalTrials.gov NCT05116787. Registry information is sponsor/investigator submitted; verify primary evidence before making development decisions.

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