BioCryst designs novel drugs

Climbing High

Based in Research Triangle Park, North Carolina, BioCryst Pharmaceuticals, Inc. uses crystallography and structure-based drug design for the development of novel therapeutics to treat cancer, cardiovascular diseases, autoimmune diseases and viral infections. The company is advancing multiple internal programs toward potential commercialization including Fodosine in oncology, BCX-4208 in transplantation and autoimmune diseases and peramivir in seasonal and life-threatening influenza.

BioCryst discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO™ (berotralstat) is approved in the United States, European Union, Japan and the United Kingdom for the prevention of a rare immune system disorder called hereditary angioedema (HAE) attacks in adults and pediatric patients 12 years and older.

BioCryst has a worldwide partnership with Roche for the development and commercialization BCX-4208. The company is collaborating with Mundipharma for the development and commercialization of Fodosine in markets across Europe, Asia, Australia and certain neighboring countries.

Additionally, BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever.

RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea. Post-marketing commitments for RAPIVAB are ongoing.

Shares of BioCryst have risen by about 180 percent since the company's first rare-disease drug earned FDA approval in December. Another potential blockbuster rolling through the company's pipeline has the potential to be an even bigger winner. In spite of strong gains durring the past six months, BioCryst's market cap is still lower than the market caps of many clinical-stage biotech companies.

In December, the FDA approved Orladeyo, a once-daily capsule designed to prevent painful swelling episodes caused by HAE. Patients with HAE have random swelling episodes usually limited to their limbs and face. Sometimes, they can cause severe pain when they occur in the intestines. Occasionally, swelling episodes that constrict patients' airways can be fatal. HAE patients need prophylactic treatments like Orladeyo that can safely be taken regularly for years or even decades. Despite competition from existing HAE treatments like Takhzyro from Takeda, BioCryst reported an encouraging $11 million in sales during Orladeyo's first full quarter on the market.

Since Takhzyro earned regulatory approval in 2018 as a treatment to prevent swelling attacks in HAE patients, the drug has been successful for Takeda. During the first three months of 2021, sales of the injection reached an annualized rate of $3.2 billion. So for BioCryst, capturing just a fraction of the addressable patient population for Orladeyo would amount to enormous sales growth.

In the second half of 2021, BioCryst will begin a pivotal trial of BCX9930, a new drug candidate for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). In March, results from an early-stage study of BCX9930 showed significantly increased levels of hemoglobin for PNH patients, suggesting it could become the first oral treatment for PNH.

If BCX9930 is approved to treat PNH and related disorders, it could be a blockbuster for BioCryst. Soliris and Ultomiris, which AstraZeneca acquired from Alexion earlier this year -- and which are also treatments for PNH and other blood-based immune-system disorders -- generated a combined $5.1 billion in revenue in 2020.

There is a great deal of competition for HAE treatments, and the clinical data for Orladeyo is not as good as that of Takhzyro. As an easy-to-swallow capsule, Orladeyo has some potential to differentiate itself from competing drugs -- but perhaps not for much longer. In February, KalVista reported positive results from a phase 2 trial of KVD900, an orally available treatment candidate for acute HAE attacks.

As an acute HAE treatment, KVD900 will not compete directly with Orladeyo, which aims to prevent attacks. Unfortunately for BioCryst, KalVista is also developing a prophylactic HAE treatment dubbed KVD824 that could end up competing directly with Orladeyo. Kalvista's success with KVD900 so far suggests that there is a good chance that KVD824 will become a thorn in BioCryst's side in the future.

Selling small-molecule drugs that can be distributed by pharmacists is relatively easy compared to selling treatments that need to be infused or injected. Engineering new small-molecule drugs that can alter the function of a specific protein is a multidisciplinary challenge.

There are few companies with the proven ability to do what BioCryst has already accomplished, and none trade as inexpensively. At the moment, the company has a relatively modest $2.5 billion market cap despite having one drug that has earned regulatory approval and another potential blockbuster in the pipeline advancing into late-stage development.

BioCryst finished last quarter with $227 million in cash and equivalents on the books, after losing $64 million during the period. If the company cannot encourage investors with more successes from its BCX990 program or begin to make ends meet with Orladeyo sales before operations claim its cash cushion, the stock could take a dip. In recent trading session, BioCryst saw 2,555,113 shares changing hands with its beta currently measuring 2.63. Recent per share price level of $14.61 trading at $1 or 7.35 percent gives it a market valuation of $2.61 billion. That most recent trading price of stock is at a discount of -2.46 percent from its 52-week high price of $14.97 and is indicating a premium of 77.41 percent from its 52-week low price of $3.3. Taking a look at company’s average trading volume for the last 10 days demonstrates a volume of 3.42 million shares.

For BioCryst , the analysts’ consensus is at an average recommendation of buy while assigning it a mean rating of 2. Splitting up the data highlights that, out of 11 analysts covering the stock, none rated the stock as a sell while none recommended an overweight rating for the stock. Two suggested the stock as a hold whereas nine see the stock as a buy. No analysts advised it as an underweight. The company is expected to be making an EPS of -$0.25 in the current quarter.