What happened

Shares of the rare-disease drugmaker BioCryst Pharmaceuticals (NASDAQ:BCRX) gained as much as 44% today in early-morning trading on sky-high volume. The catalyst behind this surge higher is the news that the company's experimental hereditary angioedema (HAE) drug candidate, BCX7353, continued to post impressive results in a mid-stage dose ranging study based on a second interim analysis.

Specifically, the company reported that the intent-to-treat population exhibited significant reductions in HAE attacks for both the 125 mg (p=0.004) and the 350 mg (p=0.014) doses, compared to placebo. Equally as important, there were no serious safety issues related to treatment with BCX7353, according to BioCryst. 

Stock chart.

image source: Getty Images.

So what

Although BioCryst is attempting to carve into a key drug market for the far larger Shire (NASDAQ: SHPG) with BCX7353, this oral HAE medication could provide a more convenient alternative to traditional injected or infused therapies. Most importantly, an oral competitor with a similar efficacy and safety profile relative to Shire's Food and Drug Administration-approved Cinryze, as well as its late-stage candidate lanadelumab, might produce higher adherence rates among HAE patients. 

Now what

The key take home message is that BCX7353's real-world value proposition will ultimately depend on how it stacks up against Shire's HAE drugs in terms of both safety and efficacy. And that question won't be answered until BioCryst advances BCX7353 into a pivotal-stage trial later on down the road. 

Unfortunately, BioCryst will need to raise capital well before initiating a late-stage trial for BCX7353, given its last stated cash position of $98 million and burn rate that already exceeds $43 million per year. In other words, there's a good chance that the company is going to take advantage of this spike in its share price to raise capital, perhaps within the next few days. So, until then, investors interested in this small-cap biotech stock may want to bide their time for a more attractive entry point. 

This article represents the opinion of the writer, who may disagree with the “official” recommendation position of a Motley Fool premium advisory service. We’re motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.