The company noted recent dose-related observations in an ongoing BCX10013 nonclinical study would delay the clinical program. BioCryst planned to advance BCX10013 into patient studies in mid-2023, including in patients with paroxysmal nocturnal hemoglobinuria (PNH), to evaluate once-daily dosing. In January, the company announced initial data from ongoing phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) trials in healthy volunteers. It showed rapid, sustained, and >97 percent suppression of the complement system’s alternative pathway (AP) 24 hours following a single 110 mg dose and that BCX10013 has been safe and generally well-tolerated at all doses studied to date. Needham writes that they believe the sell-off was overdone and that BCRX’s 25% YTD decline brought its shares to the level that fails to adequately capture Orladeyo’s sales (2023 sales guidance of ≥$320 million, +27%Y/Y) and growth trajectory. The analyst upgraded the stock to Buy from Hold with a price target of $14. The analyst expects Orladeyo can attain peak sales of $700 million, given that competition from another oral HAE prophylactic is unlikely for at least 5-7 years. BCRX’s OpEx spending is high, but the company is well-funded, with a cash balance of $443.9 million. The BCX10013 […]