BioCryst shares are blasted as a PhIII HAE 'success' underwhelms analysts
It was designed to be the oral fix for hereditary angioedema (HAE) — a rare, potentially life-threatening genetic disorder — but BioCryst Pharmaceutical’s experimental capsule has underwhelmed in a pivotal late-stage study, despite meeting the primary endpoint.
Two doses of the oral drug, BCX7353, (110 mg and 150 mg) were investigated in a 121-patient, placebo-controlled trial called APeX-2 for the prevention of angioedema attacks — recurrent episodes of severe swelling commonly in the limbs, face, intestinal tract, and airway.
Both doses met the main goal of reducing the rate of attacks. The higher dose diminished the attack rate by 44% versus placebo (p<0.001), while the lower dose cut the attack rate by 30% (p=0.024). That didn’t cut it on Wall Street, though. Analysts have long suggested a rate of at least 50% would ensure BCX7353 would be competitive in a market that is currently monopolized by highly effective injectables.
Shares of North Carolina-based BioCryst $BCRX were halved in premarket trading.
Injectable medications, such as Shire’s 2018-approved lanadelumab, can reduce HAE attack frequency by about 80%. “(W)e would’ve thought the drug’s efficacy would’ve needed to at least near that of (Shire’s older HAE injectable) Cinryze in order for it to be disruptive to the HAE treatment landscape,” Stifel analysts wrote in a note.
Needham analysts noted recently:
Based on discussions with KOLs, efficacy rates in the 50%-range would be sufficient for an oral product to play a significant role in HAE prophylaxis market given the high demand for oral alternatives. Recall, Cinryze was a game-changer for HAE patients despite IV dosing of every 3-4 days and efficacy rates of ~50%. The potential of BCX7353 in HAE prophylaxis hinges on meeting stat sig in APeX-2, with an efficacy rate of at least 50% and a clean safety/tolerability profile.
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