BioCryst: Factor D Inhibitor Data In 2024 Should Be Watched Closely (NASDAQ:BCRX)

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) has made great progress in advancing its pipeline. That’s because it is currently evaluating the use of oral D Factor inhibitor, known as BCX10013, for the treatment of complemented mediated disorders. There was some data presented from a completed 160 mg multiple ascending dose cohort from a healthy volunteer study already.

However, why I believe there is great potential here with respect to this program is for two reasons. The first reason is because the company plans to release proof-of-concept data from this phase 1 trial in 2024. Secondly, the biotech is taking a bold move in that it hopes to establish a competitive advantage over other alternative pathway inhibitors in terms of dosing.

The goal is to have BCX10013 dosed once daily, whereas many others in clinical development are being advanced as twice-daily dosing drugs for patients. Another aspect to consider is that the stock price traded lower on the back of news from another biotech company advancing its very own oral drug for Hereditary Angioedema [HAE]. This is not good news, because it would compete directly with BioCryst’s ORLADEYO. However, BioCryst is still already far in the game, and is even in the process of running a phase 3 study to expand the label of its drug to target the pediatric HAE patient population.

BCX10013 Has Potential To Do Well In The Complement Mediated Disorders Space

As I stated above, BioCryst Pharmaceuticals is in the process of advancing the use of its drug BCX10013 for the treatment of patients with complement mediated disorders. At the R&D Day it had, it presented data from a completed 160 mg multiple ascending dose cohort from a healthy volunteer trial. Again, why I believe it has value is because of the release of proof-of-concept data from the ongoing phase 1 study. Should this data be positive, not only would it cause the stock price to trade higher, but it would also be confirmation of being able to create a competitive advantage over other alternative pathway inhibitors in clinical development. That’s because many of the drugs from other pharmaceutical companies being advanced now, targeting the alternative pathway, are being developed to be given as twice-daily dosing.

If BioCryst can prove to inhibit this same pathway with once-daily type of dosing, then it would have a huge competitive advantage over all the other companies in the same space. The thing is that BioCryst is not just going after the complement mediated disorders market with BCX10013 alone. It is advancing three other drugs to target such disorders, which are an oral C5 inhibitor, oral C2 inhibitor and bifunctional complement inhibitor. It is expected that phase 1 studies, targeting complement mediated disorders with the oral C5 inhibitor and bifunctional complement inhibitor, will be initiated in 2025. Then, the oral C2 inhibitor is going to be advanced for these patients in a phase 1 study in 2026. The point being that there are going to be multiple shots on goal in being able to target the complement mediated disorders market.

ORLADEYO Expansion For Hereditary Angioedema Is In Motion

BioCryst already has received approval as the first oral, once-daily plasma kallikrein inhibitor in the United States and other global markets for people with Hereditary Angioedema [HAE] age 12 and older. The stock traded lower on the back of news that a competing pharmaceutical company by the name of Pharvaris (PHVS) achieved the primary endpoint of a phase 2 study known as CHAPTER-1. This mid-stage study recruited a total of 34 HAE patients type 1 and type 2 who were randomized to receive one of the following:

• 20 mg/day of deucrictibant
• 40 mg/day of deucrictibant
• Placebo.

Patients were randomized to receive one of these dosing regimens for a total of 12 weeks. The final outcome was that 40 mg deucrictibant achieved the primary endpoint of reduced mean monthly attack rate compared to placebo by 84.5%. Thus, the primary endpoint was achieved with a statistically significant p-value of p=0.0008.

This data is very good, and over the long term this doesn’t bode well for BioCryst. However, that’s just it, this was only a phase 2 study for the time being. Pharvaris states that it will have to send this data to the FDA, and from there, should everything be okay in terms of results, then it will likely need to then complete a larger phase 3 study which is going to take several years. By then, I believe that ORLADEYO would have captured a good chunk of the market.

Not only that, but BioCryst is already in the process of testing its oral kallikrein inhibitor drug for the treatment of pediatric patients ages 2 to <12 years of age in the ongoing APeX-P trial. The goal of this phase 3 study is to ultimately allow the biotech to expand its label towards targeting another chunk of the HAE patient population.

Financials

According to the 10-Q SEC Filing, BioCryst Pharmaceuticals had cash, cash equivalents, restricted cash, and investments of $399.2 million as of September 30, 2023. This biotech is already generating revenue through sales of its approved drug ORLADEYO for the treatment of patients with HAE. Net revenue for Q3 of 2023 came in at $85.7 million, which was a 29.8% year-over-year increase. This momentum should continue in the following quarters, and the company believes that it is on a good track to reach its trajectory of $1 billion in peak revenue.

BioCryst Pharmaceuticals, Inc. is still in a place where it needs to raise cash to keep itself funded. That’s because it notes in its 10-Q SEC Filing that it has enough cash to fund its operations for at least the next 12 months.

Risks To Business

There are several risks that investors should be aware of before investing in BioCryst Pharmaceuticals. The first risk to consider would be with respect to the advancement of BCX10013 for the treatment of complement mediated disorders. While healthy volunteer data were released from the 160 mg dose cohort, there is no guarantee that proof-of-concept phase 1 data will turn out to be successful.

A second risk to consider would be with respect to ORLADEYO sales themselves. There is no assurance that sales of ORLADEYO will continue to grow at the expected pace to reach $1 billion in peak sales, nor that the ongoing phase 3 APeX-P trial will allow it to expand its label.

A third risk to consider would then be competition such as Pharvaris, which just achieved positive results from its phase 2 study using deucrictibant for the treatment of patients with HAE. The thing is that whether or not this ends up being a competitor will largely depend upon whether or not it ultimately succeeds in phase 3 testing and eventually receives regulatory approval. In addition to the fact that it still several years away from reaching the market.

The fourth and final risk to consider would be with respect to the financial position that it is in. That’s because it believes that its cash on hand will only be enough to fund its operations for at least the next 12 months. This means it is highly likely going to need to raise additional funds. In my opinion, I believe that it will raise cash at least by the 1st half of 2024.

Conclusion

BioCryst Pharmaceuticals has made great progress in being able to advance its pipeline. That’s because it is advancing an oral Factor D inhibitor by the name of BCX10013 for the treatment of patients with complement mediated disorders. Results from an ongoing phase 1 proof-of-concept study using this drug to treat these patients are expected to be released in 2024.

The whole hope of the pipeline is not just on this one clinical product. It hopes to advance several other complement inhibitors in the coming years. This is evidenced by the initiation of studies for the oral C5 inhibitor and bifunctional complement inhibitor to treat these disorders in 2025. A third clinical candidate, which is a C2 inhibitor targeting the same pathway, is expected to start being developed in 2026.

In the meantime, the company is doing well to potentially reach its goal of obtaining $1 billion in peak sales for its oral HAE drug ORLADEYO. Lastly, there is another clinical candidate being advanced in the pipeline, known as BCX17725. This clinical drug is being advanced for the treatment of patients with Netherton Syndrome.