BioCryst Announces Publication of Data from Open-label Extension of the APeX-2 Pivotal Trial of ORLADEYO® (berotralstat)
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announces publication of data from the open-label extension (OLE) of the APeX-2 trial of ORLADEYO for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older. Results show rapid and sustained reductions in HAE attacks and improved quality of life over 96 weeks of treatment.
The publication of the APeX-2 trial results for ORLADEYO, as a prophylactic treatment for hereditary angioedema (HAE), represents a significant milestone for BioCryst Pharmaceuticals. The trial's focus on long-term safety and efficacy is a critical aspect for both clinicians and patients when considering treatment options. The reported reductions in HAE attacks and improvements in quality of life over 96 weeks suggest that ORLADEYO could offer a substantial benefit to patients suffering from this chronic condition.
From an investor's perspective, these positive results could potentially lead to increased adoption of the treatment, thus driving revenue growth for BioCryst. Additionally, the emphasis on long-term data is likely to strengthen the drug's market position against competitors, particularly if the safety profile remains robust over extended use. The impact on the stock market for BioCryst should be monitored closely as further real-world evidence is presented at medical congresses, which may further solidify the drug's reputation and financial prospects.
In the context of the broader healthcare market, the successful long-term results of ORLADEYO are indicative of a trend towards chronic disease management with a focus on patient quality of life. This aligns with the industry's pivot to value-based care, where outcomes and patient satisfaction are increasingly prioritized. The data suggests that ORLADEYO could become a key player in the HAE prophylactic space, potentially influencing treatment guidelines and insurance coverage decisions.
For stakeholders, including healthcare providers and payers, the demonstrated efficacy and safety over a 96-week period provide a strong argument for the inclusion of ORLADEYO in treatment regimens. However, it is essential to consider the cost-effectiveness of the drug in comparison to existing therapies, as this will be a significant factor in its market penetration and, consequently, its impact on BioCryst's financial performance.
The methodology and results of the APeX-2 trial underscore the importance of long-term clinical data in the assessment of a drug's therapeutic value. The transition from a double-blind, placebo-controlled setup to an open-label extension is a standard approach to understanding the prolonged effects of a medication. The primary and secondary endpoints chosen, focusing on safety, tolerability, attack rates and quality of life, are particularly relevant to HAE, a condition that significantly impacts patients' daily living.
It is noteworthy that the study has been published in a reputable journal, which adds to the credibility of the findings. However, it is crucial to critically appraise the data, considering the open-label nature of the extension phase, which can introduce bias. Furthermore, the real-world application of the drug may reveal additional insights into its performance, which could either corroborate or challenge the trial's findings.
12/19/2023 - 07:00 AM
– Results published in JACI: In Practice show rapid and sustained reductions in HAE attacks and improved quality of life over 96 weeks of treatment with ORLADEYO –
RESEARCH TRIANGLE PARK, N.C., Dec. 19, 2023 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that data from the open-label extension (OLE) of the APeX-2 trial of oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older have been published online by the Journal of Allergy and Clinical Immunology: In Practice (JACI: In Practice ).
“As detailed in this manuscript, long-term prophylaxis with ORLADEYO has enabled patients with HAE to better manage their condition, including reducing the number of HAE attacks they experience and demonstrating clinically meaningful improvement in their quality of life. I have seen firsthand in my practice evidence that the longer patients remain on ORLADEYO, the better their outcomes are,” said William R. Lumry, M.D., clinical professor of internal medicine at the University of Texas Southwestern Medical School.
“We are pleased to share long-term efficacy and safety data from APeX-2 as published in JACI: In Practice . These data further illustrate the potential lasting outcomes that can be appreciated by patients who are treated with oral, once-daily ORLADEYO. We continue to see long-term safety and effectiveness data that reinforce ORLADEYO as an important treatment option for patients with HAE, and we look forward to sharing additional real-world evidence at upcoming medical congresses,” said Dr. Ryan Arnold, chief medical officer of BioCryst.
APeX-2 was a Phase 3, double-blind, placebo-controlled, parallel-group, three-part study evaluating ORLADEYO versus placebo for the prevention of HAE attacks in 121 patients with HAE Type I or Type II. In part 3 of the study (weeks 49-96), all patients (n=81) were treated with open-label ORLADEYO at 150 mg. The primary endpoint of the OLE was long-term safety and tolerability, while secondary endpoints included HAE attack rates and quality of life (QoL).
The authors concluded that ORLADEYO was generally well tolerated, provided rapid and sustained reductions in HAE attacks and improved QoL over the study duration of 96 weeks. The results from part 3 of APeX-2 were first presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress in July 2021.
Additional details can be found in the manuscript, which is available online at jaci-inpractice.org . JACI: In Practice is an official journal of the American Academy of Allergy, Asthma, and Immunology (AAAAI).
About ORLADEYO ® (berotralstat) ® (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 ® (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
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).
Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. 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 www.fda.gov/medwatch .
Please see full Prescribing Information .
About BioCryst Pharmaceuticals ® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn .
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.
BCRXW
Contact : jbluth@biocryst.com
BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) is the company behind the ORLADEYO treatment for hereditary angioedema (HAE).
The key findings from the APeX-2 trial of ORLADEYO include rapid and sustained reductions in HAE attacks and improved quality of life over 96 weeks of treatment.
The primary endpoint of the OLE was long-term safety and tolerability, while secondary endpoints included HAE attack rates and quality of life (QoL).
The manuscript with additional details from the APeX-2 trial can be found online at jaci-inpractice.org.
