BioCryst Presents Real-World Data Showing Rapid and Sustained HAE Attack Rate Reduction After Beginning ORLADEYO® (berotralstat), Regardless of Prior Prophylactic Therapy

RESEARCH TRIANGLE PARK, N.C., Nov. 10, 2022 (GLOBE NEWSWIRE) -- (Nasdaq: BCRX) today announced new real-world data demonstrating rapid, sustained reduction of patient-reported HAE attacks and consistently low attack rates among patients 12 years and older who started on oral, once-daily ORLADEYO^® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE), including patients who switched from other prophylactic therapies.

The data are being presented at the 2022 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is being conducted at the Kentucky International Convention Center in Louisville, Kentucky, from November 10-14, 2022.

“These data are particularly exciting because, consistent with our long-term clinical program data, they show that patients on ORLADEYO sustain, and even improve, attack control the longer they are on therapy,” said Dr. Ryan Arnold, chief medical officer of BioCryst.

“It is notable that this real-world evidence suggests that people living with HAE can maintain or improve control of their disease on ORLADEYO, regardless of prior treatment history,” Arnold added.

“Every HAE patient has a unique experience with their therapy, and these data demonstrate that ORLADEYO can be a very effective treatment option for patients regardless of their reported prior attack rates or prophylactic therapy history. The sustained, long-term attack rate reductions we are seeing illustrate the durability of this efficacy for patients who are looking to improve control over their HAE by switching to a therapy with a less burdensome route of administration than subcutaneous or intravenous prophylactic therapies,” said William R. Lumry, M.D., clinical professor of internal medicine at the University of Texas Southwestern Medical School.

BioCryst ACAAI 2022 Presentation Highlights

The presentations at ACAAI are based on analyses from patient-reported results collected in the real-world clinical setting from BioCryst’s sole-source pharmacy, including HAE Type I and Type II patients in the United States who actively received ORLADEYO between December 16, 2020, and May 20, 2022.

• Consistently Low Hereditary Angioedema Attack Rates Observed with Berotralstat Regardless of Previous Prophylaxis: Real-World Outcomes (poster #P062); Saturday, November 12, 11:35 a.m. ET; Monitor 10, Exhibit Hall
  
  
  
  
  • This analysis assessed patient-reported HAE attack rates of patients on ORLADEYO 110 mg or 150 mg who were previously on another prophylactic therapy (n=129), including lanadelumab (n=53), a subcutaneous (SC) C1 esterase inhibitor (n=31), danazol (n=15) and an intravenous (IV) C1 esterase inhibitor (n=21). Nine patients were on a combination of prophylactic therapies.
    
    
    
    
  • Regardless of prior prophylaxis, a rapid reduction in median attack rates was observed early (1.67 attacks/month at baseline to a median attack rate of 0.33 attacks/month in days 1-90). The reduction of median attack rates was sustained throughout the 360-day treatment period.
    
    
    
    
  • Upon initiating ORLADEYO treatment, the reduction in median attack rates from baseline over the 1–360 days period was consistent for patients regardless of their prior prophylaxis therapy. The reductions after starting ORLADEYO for each prior prophylactic therapy were:
    
    
    
    
    • 77 percent reduction for patients previously on lanadelumab;
      
      
      
      
    • 64 percent reduction for patients previously on SC C1-INH;
      
      
      
      
    • 70 percent reduction for patients previously on danazol; and
      
      
      
      
    • 72 percent reduction previously on IV C1-INH.
      
      
      
      
  • The incidence of AEs reported was lower than the incidence reported in clinical trials.
    
    
    
    
• Rapid and Sustained Reductions in Hereditary Angioedema Attack Rates with Long-term Berotralstat: Real-World Outcomes (Distinguished Industry Oral Presentation); Session A; Saturday, November 12, 4:30-5:30 p.m. ET; Room M103-M10
  
  
  
  
  • This analysis assessed the efficacy of ORLADEYO 110 mg or 150 mg in patients (n=128) for a treatment period of more than 270 days.
    
    
    
    
  • Patients reported a meaningful reduction in HAE attack rates when treated with ORLADEYO. An 80 percent average reduction from median baseline attack rate (1.67 median attacks/month at baseline vs. 0.33 median attacks/month during days 1-90) was observed during the initial 90-day treatment period. Regardless of baseline attack rate, patients reported a reduction in attack rates when treated with ORLADEYO.
    
    
    
    
  • The incidence of adverse events (AEs) reported was consistent with the incidence reported in clinical trials.
    
    

About ORLADEYO^® (berotralstat)

ORLADEYO^® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.

U.S. Indication and Important Safety Information

INDICATION

ORLADEYO^® (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.

Limitations of use

The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.

IMPORTANT SAFETY INFORMATION

An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.

The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C) and in patients taking chronically administered P-glycoprotein (P-gp) or breast cancer resistance protein (BCRP) inhibitors (eg, cyclosporine).

Berotralstat is a substrate of P-gp and BCRP. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.

ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO.

The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established.

There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production.

To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or .

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About BioCryst Pharmaceuticals

BioCryst Pharmaceuticals 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 and many global markets. BioCryst has active programs to develop oral medicines for multiple targets across the complement system, including oral factor D inhibitors BCX9930 and BCX10013, which are in clinical development. RAPIVAB® (peramivir injection) is approved in the U.S. and multiple global markets, with post-marketing commitments ongoing. For more information, please visit the company’s website at .

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst’s business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst’s and its partners’ development, regulatory processes and supply chains, negatively impact BioCryst’s ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst files periodically with the Securities and Exchange Commission; BioCryst’s ability to successfully implement its commercialization plans for, and to commercialize, ORLADEYO, which could take longer or be more expensive than planned; the commercial viability of ORLADEYO, including its ability to achieve market acceptance; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst’s business; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management's expected ranges. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst’s forward-looking statements.

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