- The FDA lifted a partial clinical hold on BioCryst Pharmaceuticals Inc's (NASDAQ: BCRX) BCX9930 program. The company will resume enrollment under revised protocols at a reduced dose of 400 mg twice daily of BCX9930.
- This includes the REDEEM-1 & REDEEM-2 trials in paroxysmal nocturnal hemoglobinuria and the RENEW trial in C3 glomerulopathy, immunoglobulin A nephropathy, and primary membranous nephropathy.
- Clinical evidence and recent laboratory studies have informed the company's hypothesis that crystals form in the kidneys of some patients.
- The company believes that lowering the dose to 400 mg and ensuring adequate hydration dilutes the drug concentration in the urine below the threshold where crystals can form.
- In April, BioCryst voluntarily paused enrollment in BCX9930 trials while investigating elevated serum creatinine (SCr) in some patients at the 500 mg twice daily dose.
- The FDA subsequently placed the program on partial clinical hold.
- PNH patients naive to C5 inhibitor therapy who received 400 mg twice daily in the Phase 1 program (n=7) experienced a mean change from baseline increase in hemoglobin of 4.3 g/dL and required no transfusions.
- No patients experienced an elevation in their SCr during dose-ranging at the 400 mg twice daily dose. All observed cases of SCr elevations occurred at 500 mg twice daily.
- Q2 Orladeyo revenue reached $65.2 million, with total sales of $65.5 million, +31.2% Y/Y, beating the consensus of $64.31 million.
- EPS loss came in at $(0.32) up from $(0.24) a year ago, beating the consensus of $(0.34).
- Guidance: BioCryst expects FY22 Orladeyo sales of $255 million - $265 million.
- Price Action: BCRX shares are up 20.6% at $13.45 during the market session on the last check Thursday.
Read at Benzinga