Shares of BioCryst Pharmaceuticals spiked 19% after the biotech company won US regulatory approval for Orladeyo, (berotralstat), its lead drug candidate for the prevention of attacks in hereditary angioedema (HAE) patients.
BioCryst (BCRX) said that the US Food and Drug Administration (FDA) approved the oral, once-daily Orladeyo as a treatment to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. Orladeyo will be available by the end of December ahead of a formal commercial launch in early-2021.
HAE is a rare disease defined by unpredictable and possibly life-threatening periodic swelling attacks and affects approximately one in 50,000 people. The most common attacks are on the arms and legs, but the condition can also cause irritation to the intestinal tract while the lungs’ airways can also be affected.
“The FDA approval of Orladeyo fulfills a promise BioCryst made to HAE patients that we were committed to helping them achieve the dream of an oral, once-daily medicine to prevent and reduce the burden of their attacks,” said BioCryst CEO Jon Stonehouse.
The approval was based on a pivotal Phase 3 trial, which showed that Orladeyo significantly lowered attacks at 24 weeks, and this reduction was sustained through 48 weeks. HAE patients who completed 48 weeks of treatment saw reductions in their HAE attack rates, from a mean of 2.9 attacks per month at baseline to a mean of 1.0 attacks per month after 48 weeks of therapy. In the long-term open label trial, patients completing 48 weeks of therapy had a mean attack rate of 0.8 attacks per month.
Orladeyo demonstrated to be safe and well tolerated in both trials. The most frequently reported adverse reactions in patients receiving Orladeyo compared with placebo were gastrointestinal reactions. These reactions generally occurred early after initiation of treatment with Orladeyo, became less frequent with time and eventually self-resolved.
Following the approval, Needham analyst Serge Belanger reiterated a Hold rating on the stock, noting that he “remains skeptical of the product’s overall potential given significantly lower efficacy levels (~44% attack rate reduction in ph 3 trials) vs. current injectable therapies with 80%-90% efficacy.”
“We maintain our Hold rating given our skepticism around Orladeyo’s sales potential and a current valuation approaching $1B that captures a significant portion of the product’s upside,” Belanger wrote in a note to investors. (See BioCryst price targets and analyst ratings on TipRanks)
Turning now to the rest of the Street, BCRX’s Moderate Buy consensus rating breaks down into 5 Buys and 2 Holds. With shares up 78% so far this this, the average price target of $8.75 implies shares could soar another 43% in the next year.
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