- BioCryst has two approved products.
- ORLADEYO beat street expectations today with its first full quarter revenue figures.
- BCX9930 will start a pivotal trial soon.
- Looking for more investing ideas like this one? Get them exclusively at The Total Pharma Tracker. Learn More »
BioCryst Pharmaceuticals (NASDAQ:BCRX) has two approved products and a diverse pipeline targeting rare diseases. ORLADEYO or berotralstat is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. It was approved in the US in early December 2020, and was also approved in Japan and the EU this month. The company’s other approved drug is RapiVab, which was approved for influenza back in 2014. Its pipeline looks like this:
There are three drugs in development, BCX9930, BCX9250, AND Galidesivir. All are in early stages. That means, after the recent approval, there are no major pivotal catalysts for a number of years.
Complement mediated disease assets
BCX9930 is a Factor D inhibitor. Factor D is essential to the alternative pathway, so inhibiting it works well for blocking all downstream processes. A number of diseases have their origin in this pathway, so potentially all these diseases come under the purview of a factor D inhibitor. These diseases are, PNH and aHUS in hematology, ANCA and IgAN vasculitis and Lupus Nephritis in rheumatology, and in nephrology, IgAN, C3G and PMN, among others. Preclinical data has shown that oral BCX9930 monotherapy can control both Intravascular (IVH) and Extravascular (EVH) Hemolysis.
In a proof-of-concept phase 1 trial, 100 percent of treatment-naïve patients and 83 percent of C5 inadequate response patients were transfusion-free following doses of 400 mg bid or 500 mg bid of oral BCX9930. These patients had severe disease prior to treatment, as seen below:
Meaningful changes in key biomarkers were observed after treatment, which indicated control of hemolysis; many patients did not need transfusions to maintain adequate hemoglobin levels. The drug was safe and well-tolerated.
“The significant reduction in transfusions and increases in hemoglobin seen in this trial with an oral medicine address an unmet need for patients and physicians -- a PNH therapy that can maximize hematological benefit through the control of both intravascular and extravascular hemolysis,” said Antonio Risitano, M.D., Ph.D., San Giuseppe Moscati Hospital, Avellino, Italy, and principal investigator of the trial.
Key highlights from this small, 15-patient trial were:
Through their last study visit, 100 percent of treatment-naïve patients and 83 percent of C5 inhibitor inadequate response patients were transfusion-free. Prior to the trial, 22 percent of treatment-naïve patients and 17 percent of C5 inadequate responders were transfusion-free.
Hemoglobin levels increased by a mean of 3.5 g/dL in treatment-naïve patients and 3.2 g/dL in C5 inhibitor inadequate response patients; at last visit, mean hemoglobin levels were 11.8 g/dL and 12.2 g/dL, respectively.
Relative red blood cell clone size, a marker of hemolytic control, also increased from 53 to 92 percent in treatment-naïve patients and from 50 to 80 percent in C5 inhibitor inadequate response patients.
C3 opsonization, a key marker of extravascular hemolysis, declined to less than two percent by the eight-week visit in five of six C5 inadequate response patients following BCX9930 therapy.
In C5 inhibitor naïve patients, clinical chemistry biomarkers of intravascular hemolysis, including lactate dehydrogenase (LDH), aspartate aminotransferase (AST) and bilirubin, all declined. Mean LDH x ULN declined by 73 percent from 7.5x ULN at baseline to 2.0x ULN.
BCX9930 has both Fast Track status and Orphan Drug Designation in PNH. BioCryst plans to begin (2H 2021) pivotal trials in PNH patients at selected dose level. In an important development, the FDA has agreed with BioCryst that change from baseline in hemoglobin is the primary endpoint for the pivotal trial.
BCX9250 is the company’s other asset. It is an oral ALK-2 inhibitor targeting a rare but terrible disease called Fibrodysplasia Ossificans Progressiva (FOP). Only about 1 in 2 million get this disease, which results in loss of function, deformities, and a severely disabling condition; there are no FDA approved treatments.
The company says - “Specific activating mutations of the ALK-2 gene are seen in all cases of FOP. An activating mutation in ALK-2 is necessary for the disease to occur, making the ALK-2 enzyme an ideal drug target for treatment of FOP.”
A phase 1 trial with two cohorts, single and multiple ascending doses, in healthy subjects, saw a dose-dependent increase in exposure to the drug. No efficacy data is available from this healthy subject trial. The company plans further trials. The drug was safe and well-tolerated.
BRCX has a market cap of $1.96bn and a cash reserve of $244mn; 70% of the company stock is held by funds and institutions. Insiders have bought and sold the stock.
BCRX's share price went up 20% just today after releasing Q1 earnings that beat street expectations on the back of outstanding revenue from ORLADEYO’s first quarter in the market. The company's Q1 revenue increased +295.4% Y/Y to $19.06M, beating analysts' average estimate by $9.46M. Hereditary Angioedema treatment ORLADEYO brought in $10.9M in its first full quarter after launch. The majority of this revenue came from new patients who switched to ORLADEYO from either injectable/infused prophylactic medications or from acute-only treatment. The remainder came from patients transitioning from clinical trials and the company’s early access program.
The company expects its cash runway to take it into 2023, based on its revenue estimates and an option to access an additional $75M from its existing credit facility.
BCRX was an excellent trading stock to me over the years, way back when it did not have ORLADEYO approved. Today, it is a growth stock, with one approved drug with excellent introductory revenue figures, and new geographies opening up with new approvals. It also has a second drug with an upcoming pivotal trial to start soon in a multibillion dollar market where it has already shown solid data with respect to the agreed upon endpoint. I expect PDUFA sometime in early 2023, and the company has enough cash to last that long. The stock is up 20% today; however, this is a stock to be slowly accumulated for long-term growth.
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This article was written by
Avisol Capital Partners is made up of a team of medical experts, finance professionals and techies, all of whom invest their own money in the picks they share. They aim to help readers find the middle ground between value and growth investing, as they demystify the biopharma industry.They lead the investing group Total Pharma Tracker where they offer a monthly updated catalyst database, an investability scoring system for quick reference ideas, and direct access in chat for dialogue and questions. Learn more.
Analyst’s Disclosure: I/we have no positions in any stocks mentioned, but may initiate a long position in BCRX over the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.
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