BCRX is a commercial stage company that focuses on difficult to develop oral therapies for rare disease with a productive drug development team. From a bullish perspective, they own the first approved oral HAE therapy, ORLADEYO, which will hit over $250M in sales this year, in only its 2nd full year since approval. The company projects that it will hit $1B in peak ORLADEYO sales, which make the current ~$2.5B enterprise value appear low.
For the bull case, what makes BCRX's valuation appear particular low is that in addition to its approved potentially blockbuster drug, the company has 2 compounds in the clinic currently, and has pre-clinical candidates too. BCX9930, an oral Factor D inhibitor has shown promising efficacy in early clinical trials. And although kidney safety issues arose, which led to an FDA clinical hold, from a bull's perspective, those appear to be easily avoided by using a lower, but effective dose (400 mg), and assuring that the drug is taken with water.
Finally, from a bullish perspective, the company has a productive drug development team that adds significant value. There is another internally developed drug candidate, BCX9250, an ALK-2 inhibitor for treating the rare disease FOP, that will enter phase 2 likely in 2023 for another rare disease. Furthermore, the company appears poised to introduce at least 1 more home-grown candidate into the clinic by the end of 2023. Thus, for BCRX bulls, there is a case to be made.
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